Overview of scleroderma manifestations, organ involvement, brief classifications (limited, diffuse, sine scleroderma). Overview of current treatment options, need for additional therapies. Overview of plan for multi-disciplinary scleroderma center at the University of Chicago. Potential future therapies in the literature at large. Planned trials/future treatment options at the University of Chicago.
For more info about scleroderma and the foundation, head to www.stopscleroderma.org
This talk was presented at the Scleroderma Patient Education Conference on May 4, 2024, hosted by the Scleroderma Foundation of Greater Chicago.
1362463849 derangement of wound healing in diabetic neuropathydfsimedia
This document discusses factors that impair wound healing in diabetes, including peripheral neuropathy, wound hypoxia, abnormal cellular pathways, excess inflammation, and deficient precursor cells. It then summarizes the results of a study on the role of inflammation in delayed wound healing of diabetic patients. The study found that matrix metalloproteinases MMP-9 and MMP-2, which degrade the extracellular matrix, were overexpressed in the tissue and serum of diabetic patients with infected foot ulcers compared to those without infection. This overexpression contributes to delayed wound healing.
1362463849 derangement of wound healing in diabetic neuropathydfsimedia
This document discusses factors that impair wound healing in diabetes, including peripheral neuropathy, wound hypoxia, abnormal cellular pathways, excess inflammation, and deficient precursor cells. It then summarizes the results of a study on the role of inflammation in delayed wound healing of diabetic patients. The study found that matrix metalloproteinases MMP-9 and MMP-2, which degrade the extracellular matrix, were overexpressed in the tissue and serum of diabetic patients with infected foot ulcers compared to those without infection. This overexpression contributes to delayed wound healing.
PANEL DISCUSSION ON ENDOMETRIOSIS IN ADOLESCENTS (2018 )Lifecare Centre
PANEL DISCUSSION ON ENDOMETRIOSIS IN ADOLESCENTS (2018 ) MODERATOR
DR SHARDA JAIN
DR ILA GUPTA
DR DIPTI NABH
panelist
UMA RAI
RAJ BOKARIA
JYOTI AGARWAL
JYOTI BHASKER
RENU CHAWLA
DIPTI NABH
VANDANA GUPTA
PANEL DISCUSSION ON ENDOMETRIOSIS IN ADOLESCENTS (2018 )Lifecare Centre
PANEL DISCUSSION ON ENDOMETRIOSIS IN ADOLESCENTS (2018 ) MODERATOR
DR SHARDA JAIN
DR ILA GUPTA
DR DIPTI NABH
panelist
UMA RAI
RAJ BOKARIA
JYOTI AGARWAL
JYOTI BHASKER
RENU CHAWLA
DIPTI NABH
VANDANA GUPTA
This document discusses the pharmacologic management of rheumatoid arthritis and osteoarthritis. It provides an overview of the drug therapies used for each condition. For rheumatoid arthritis, it covers the pathogenesis and classification criteria. It then discusses the goals of drug treatment and the main categories of drugs used - NSAIDs, glucocorticoids, and disease-modifying antirheumatic drugs (DMARDs). For osteoarthritis, it provides background on the condition and notes that treatment focuses more on non-pharmacologic measures rather than inflammation. Drug classes discussed for osteoarthritis include acetaminophen, NSAIDs, viscosupplementation with hyaluronan, and dietary supplements like glucosamine and chondroitin sulfate
This document discusses the pharmacologic management of rheumatoid arthritis and osteoarthritis. It provides an overview of the drug therapies used for each condition. For rheumatoid arthritis, it covers the pathogenesis and classification criteria. It then discusses the goals of drug treatment and the main categories of drugs used - NSAIDs, glucocorticoids, and disease-modifying antirheumatic drugs (DMARDs). For osteoarthritis, it provides background on the condition and notes that treatment focuses more on non-pharmacologic measures rather than inflammation. Drug classes discussed for osteoarthritis include acetaminophen, NSAIDs, viscosupplementation with hyaluronan, and dietary supplements like glucosamine and chondroitin sulfate
1362463849 derangement of wound healing in diabetic neuropathydfsimedia
This document discusses factors that impair wound healing in diabetes, including peripheral neuropathy, wound hypoxia, abnormal cellular pathways, excess inflammation, and deficient precursor cells. It then summarizes the results of a study on the role of inflammation in delayed wound healing of diabetic patients. The study found that matrix metalloproteinases MMP-9 and MMP-2, which degrade the extracellular matrix, were overexpressed in the tissue and serum of diabetic patients with infected foot ulcers compared to those without infection. This overexpression contributes to delayed wound healing.
1362463849 derangement of wound healing in diabetic neuropathydfsimedia
This document discusses factors that impair wound healing in diabetes, including peripheral neuropathy, wound hypoxia, abnormal cellular pathways, excess inflammation, and deficient precursor cells. It then summarizes the results of a study on the role of inflammation in delayed wound healing of diabetic patients. The study found that matrix metalloproteinases MMP-9 and MMP-2, which degrade the extracellular matrix, were overexpressed in the tissue and serum of diabetic patients with infected foot ulcers compared to those without infection. This overexpression contributes to delayed wound healing.
PANEL DISCUSSION ON ENDOMETRIOSIS IN ADOLESCENTS (2018 )Lifecare Centre
PANEL DISCUSSION ON ENDOMETRIOSIS IN ADOLESCENTS (2018 ) MODERATOR
DR SHARDA JAIN
DR ILA GUPTA
DR DIPTI NABH
panelist
UMA RAI
RAJ BOKARIA
JYOTI AGARWAL
JYOTI BHASKER
RENU CHAWLA
DIPTI NABH
VANDANA GUPTA
PANEL DISCUSSION ON ENDOMETRIOSIS IN ADOLESCENTS (2018 )Lifecare Centre
PANEL DISCUSSION ON ENDOMETRIOSIS IN ADOLESCENTS (2018 ) MODERATOR
DR SHARDA JAIN
DR ILA GUPTA
DR DIPTI NABH
panelist
UMA RAI
RAJ BOKARIA
JYOTI AGARWAL
JYOTI BHASKER
RENU CHAWLA
DIPTI NABH
VANDANA GUPTA
This document discusses the pharmacologic management of rheumatoid arthritis and osteoarthritis. It provides an overview of the drug therapies used for each condition. For rheumatoid arthritis, it covers the pathogenesis and classification criteria. It then discusses the goals of drug treatment and the main categories of drugs used - NSAIDs, glucocorticoids, and disease-modifying antirheumatic drugs (DMARDs). For osteoarthritis, it provides background on the condition and notes that treatment focuses more on non-pharmacologic measures rather than inflammation. Drug classes discussed for osteoarthritis include acetaminophen, NSAIDs, viscosupplementation with hyaluronan, and dietary supplements like glucosamine and chondroitin sulfate
This document discusses the pharmacologic management of rheumatoid arthritis and osteoarthritis. It provides an overview of the drug therapies used for each condition. For rheumatoid arthritis, it covers the pathogenesis and classification criteria. It then discusses the goals of drug treatment and the main categories of drugs used - NSAIDs, glucocorticoids, and disease-modifying antirheumatic drugs (DMARDs). For osteoarthritis, it provides background on the condition and notes that treatment focuses more on non-pharmacologic measures rather than inflammation. Drug classes discussed for osteoarthritis include acetaminophen, NSAIDs, viscosupplementation with hyaluronan, and dietary supplements like glucosamine and chondroitin sulfate
This document discusses idiopathic inflammatory myopathies (IIM), including their classification, clinical features, epidemiology, and relevant literature. The main types of IIM are polymyositis (PM), dermatomyositis (DM), necrotizing autoimmune myopathy (NAM), and sporadic inclusion body myositis (sIBM). DM is characterized by a skin rash in addition to muscle weakness. NAM presents with severe weakness but no rash. sIBM typically affects older males and causes both proximal and distal weakness. The document provides details on clinical manifestations and diagnostic criteria for each IIM subtype.
This document discusses idiopathic inflammatory myopathies (IIM), including their classification, clinical features, epidemiology, and relevant literature. The main types of IIM are polymyositis (PM), dermatomyositis (DM), necrotizing autoimmune myopathy (NAM), and sporadic inclusion body myositis (sIBM). DM is characterized by a skin rash in addition to muscle weakness. NAM presents with severe weakness but no rash. sIBM typically affects older males and causes both proximal and distal weakness. The document provides details on clinical manifestations and diagnostic criteria for each IIM subtype.
This document reviews glomerulonephritis as a cause of acute kidney injury. It discusses several types of glomerulonephritis that can lead to AKI, including anti-GBM disease, small vessel vasculitis, lupus nephritis, IgA nephropathy, and post-streptococcal glomerulonephritis. Kidney biopsy is important for diagnosis but not always possible in critically ill patients. Treatment depends on the specific type of glomerulonephritis and may include corticosteroids, cyclophosphamide, plasma exchange, and immunosuppression. Prognostic markers include the extent of glomerular lesions, need for dialysis, and creatin
This document reviews glomerulonephritis as a cause of acute kidney injury. It discusses several types of glomerulonephritis that can lead to AKI, including anti-GBM disease, small vessel vasculitis, lupus nephritis, IgA nephropathy, and post-streptococcal glomerulonephritis. Kidney biopsy is important for diagnosis but not always possible in critically ill patients. Treatment depends on the specific type of glomerulonephritis and may include corticosteroids, cyclophosphamide, plasma exchange, and immunosuppression. Prognostic markers include the extent of glomerular lesions, need for dialysis, and creatin
Drug-induced hypersensitivity syndrome (DIHS)/Drug reaction with eosinophilia and systemic symptoms (DRESS)
Presented by Pongsawat Rodsaward, MD.
December 17, 2021
Drug-induced hypersensitivity syndrome (DIHS)/Drug reaction with eosinophilia and systemic symptoms (DRESS)
Presented by Pongsawat Rodsaward, MD.
December 17, 2021
The document discusses mast cell disorders and their classification. It describes localized mastocytosis, which includes cutaneous mastocytosis subtypes like urticaria pigmentosa and diffuse cutaneous mastocytosis. Systemic mastocytosis is classified into several subtypes based on severity from indolent to aggressive forms. The pathogenesis involves mutations in the KIT gene in most cases that lead to mast cell proliferation. Clinical features can include systemic symptoms from mast cell mediators as well as dermatologic findings like the rash of urticaria pigmentosa.
The document discusses mast cell disorders and their classification. It describes localized mastocytosis, which includes cutaneous mastocytosis subtypes like urticaria pigmentosa and diffuse cutaneous mastocytosis. Systemic mastocytosis is classified into several subtypes based on severity from indolent to aggressive forms. The pathogenesis involves mutations in the KIT gene in most cases that lead to mast cell proliferation. Clinical features can include systemic symptoms from mast cell mediators as well as dermatologic findings like the rash of urticaria pigmentosa.
Rheumatoid arthritis is a systemic inflammatory disease that causes swelling and tenderness of the joints. It results from a breakdown of immune tolerance that leads to inflammation of the synovial tissue lining joints. The disease predominantly affects females and occurs most often between the ages of 35-50. Treatment involves both pharmacological and non-pharmacological approaches. Pharmacological treatment starts with NSAIDs and progresses to DMARDs like methotrexate and biologics if needed to reduce inflammation, prevent further joint damage, and control symptoms.
Rheumatoid arthritis is a systemic inflammatory disease that causes swelling and tenderness of the joints. It results from a breakdown of immune tolerance that leads to inflammation of the synovial tissue lining joints. The disease predominantly affects females and occurs most often between the ages of 35-50. Treatment involves both pharmacological and non-pharmacological approaches. Pharmacological treatment starts with NSAIDs and progresses to DMARDs like methotrexate and biologics if needed to reduce inflammation, prevent further joint damage, and control symptoms.
The document discusses principles of treating infectious illnesses in critical care, with a focus on antibiotic resistance and choice of antibiotics. It covers several topics: the impact of antibiotic use on resistance; choosing initial antibiotics and tailoring treatment based on culture results; applying pharmacology and pharmacodynamics to optimize bacterial killing; and reviewing guidelines for specific infections. It also provides an overview of antibiotic classes, mechanisms of action, considerations for dosing in renal impairment, and highlights specific agents like penicillins, cephalosporins, and vancomycin.
The document discusses principles of treating infectious illnesses in critical care, with a focus on antibiotic resistance and choice of antibiotics. It covers several topics: the impact of antibiotic use on resistance; choosing initial antibiotics and tailoring treatment based on culture results; applying pharmacology and pharmacodynamics to optimize bacterial killing; and reviewing guidelines for specific infections. It also provides an overview of antibiotic classes, mechanisms of action, considerations for dosing in renal impairment, and highlights specific agents like penicillins, cephalosporins, and vancomycin.
Presentazione a cura del Professor Franco Scaldaferri - M.A.S.T.E.R. ECM in Gastroenterologia: Approccio personalizzato alla complessità in Gastroenterologia - Fondazione Santa Lucia - Roma 19/01/2018
Presentazione a cura del Professor Franco Scaldaferri - M.A.S.T.E.R. ECM in Gastroenterologia: Approccio personalizzato alla complessità in Gastroenterologia - Fondazione Santa Lucia - Roma 19/01/2018
20 July 2022 Diagnosis and Treatment of Multiple Sclerosis.pptxmanjujanhavi
This document provides an overview of the diagnosis and treatment of multiple sclerosis (MS). It discusses the epidemiology, pathophysiology, clinical presentation, diagnosis using the McDonald criteria, disease course classifications (relapsing-remitting vs progressive), natural history, outcomes measures, and available treatment options for MS. Treatment options include disease-modifying therapies (DMTs) such as injectable medications (interferons, glatiramer acetate), oral medications (fingolimod, dimethyl fumarate, teriflunomide), monoclonal antibody therapies (ocrelizumab, natalizumab), and management of relapses, symptoms, and comorbidities. The goal of treatment is to reduce rel
20 July 2022 Diagnosis and Treatment of Multiple Sclerosis.pptxmanjujanhavi
This document provides an overview of the diagnosis and treatment of multiple sclerosis (MS). It discusses the epidemiology, pathophysiology, clinical presentation, diagnosis using the McDonald criteria, disease course classifications (relapsing-remitting vs progressive), natural history, outcomes measures, and available treatment options for MS. Treatment options include disease-modifying therapies (DMTs) such as injectable medications (interferons, glatiramer acetate), oral medications (fingolimod, dimethyl fumarate, teriflunomide), monoclonal antibody therapies (ocrelizumab, natalizumab), and management of relapses, symptoms, and comorbidities. The goal of treatment is to reduce rel
Abstract—In Italy the hydatid disease is more prevalent and new cases are highlighted more frequently in Sicily, Sardinia, (Italy). Aim of this study is to put the indication in search of iaditea nature in both spleen swelling and muscle tendon.
Material and Method Patients observed during the period 2007-2009 at the Surgical Clinic III and Digestive Surgery, Policlinico G Rodolico were explored for Hydatid cyste at various sites. Diagnosis of cysts ecchinococcus occurred primarily for various four reasons either for compression of bodies involved or for eosinophilia or for instrumental investigation or for anaphylactic reaction to rupture of cysts. Biological diagnosis is based on serology rather than isolation of the parasite (indirect diagnosis);
Results Patients attended during the period 2007-2009 Hydatid cyst was found in 0.5% of all cases in liver along with 4 in the lung, 3 in splenic, 2 in the mammary and 2 in the chest wall No 2. The Surgical treatment with the complete removal of the cyst with a satisfactory postoperative course in the absence of cases of relapse of the disease and by following the therapeutic act, the assumption of mebendazole 50mg / kg / day for 3 weeks at a dose of 400mg for 4 months
Conclusions There is a need to define diagnostic methods with high specificity and sensitivity, which can provide a valid diagnostic aid for the cases clinically difficult to diagnose. And the final diagnosis must then also be based on the development of immunological methods that allow the determination of specific antibodies in the serum and their titration and / or the circulating antigen determination.
Abstract—In Italy the hydatid disease is more prevalent and new cases are highlighted more frequently in Sicily, Sardinia, (Italy). Aim of this study is to put the indication in search of iaditea nature in both spleen swelling and muscle tendon.
Material and Method Patients observed during the period 2007-2009 at the Surgical Clinic III and Digestive Surgery, Policlinico G Rodolico were explored for Hydatid cyste at various sites. Diagnosis of cysts ecchinococcus occurred primarily for various four reasons either for compression of bodies involved or for eosinophilia or for instrumental investigation or for anaphylactic reaction to rupture of cysts. Biological diagnosis is based on serology rather than isolation of the parasite (indirect diagnosis);
Results Patients attended during the period 2007-2009 Hydatid cyst was found in 0.5% of all cases in liver along with 4 in the lung, 3 in splenic, 2 in the mammary and 2 in the chest wall No 2. The Surgical treatment with the complete removal of the cyst with a satisfactory postoperative course in the absence of cases of relapse of the disease and by following the therapeutic act, the assumption of mebendazole 50mg / kg / day for 3 weeks at a dose of 400mg for 4 months
Conclusions There is a need to define diagnostic methods with high specificity and sensitivity, which can provide a valid diagnostic aid for the cases clinically difficult to diagnose. And the final diagnosis must then also be based on the development of immunological methods that allow the determination of specific antibodies in the serum and their titration and / or the circulating antigen determination.
Understanding the molecular biology of intervertebral disc degeneration and p...CarlosAlbertoMontesM
This document reviews gene therapy strategies for treating intervertebral disc degeneration. It discusses the molecular biology and etiology of disc degeneration, which involves an imbalance between catabolism and anabolism leading to loss of extracellular matrix. Current treatments only relieve symptoms and do not address the underlying pathology. The review examines growth factor therapy, cell therapy, and gene therapy approaches for regeneration. Gene therapy was initially mediated by viral vectors but recent advances allow for non-viral methods to avoid risks of mutagenesis and infection. With further progress, non-viral gene therapy may prove effective for treating disc degeneration.
Understanding the molecular biology of intervertebral disc degeneration and p...CarlosAlbertoMontesM
This document reviews gene therapy strategies for treating intervertebral disc degeneration. It discusses the molecular biology and etiology of disc degeneration, which involves an imbalance between catabolism and anabolism leading to loss of extracellular matrix. Current treatments only relieve symptoms and do not address the underlying pathology. The review examines growth factor therapy, cell therapy, and gene therapy approaches for regeneration. Gene therapy was initially mediated by viral vectors but recent advances allow for non-viral methods to avoid risks of mutagenesis and infection. With further progress, non-viral gene therapy may prove effective for treating disc degeneration.
This talk was presented by Michael Macklin, MD from the University of Chicago at the Scleroderma Patient Education Conference on May 4, 2024, hosted by the Scleroderma Foundation of Greater Chicago. This talk includes:
Overview of scleroderma manifestations, organ involvement, brief classifications (limited, diffuse, sine scleroderma)
Overview of current treatment options, need for additional therapies
Overview of plan for multi-disciplinary scleroderma center at the University of Chicago
Potential future therapies in the literature at large
Planned trials/future treatment options at the University of Chicago
For more info about scleroderma and the foundation, head to www.stopscleroderma.org
Interstitial lung disease (ILD) is a common complication of scleroderma that leads to inflammation and scarring of the lungs. In this session, we will review the prevalence of scleroderma-associated ILD (SSc-ILD), classic symptoms, and the approach to evaluating patients with suspected disease. In addition, we will cover various treatments available for patients with SSc-ILD.
This talk was presented at the Scleroderma Patient Education Conference on May 4, 2024, hosted by the Scleroderma Foundation of Greater Chicago.
For more info about scleroderma and the foundation, head to www.stopscleroderma.org
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This document discusses idiopathic inflammatory myopathies (IIM), including their classification, clinical features, epidemiology, and relevant literature. The main types of IIM are polymyositis (PM), dermatomyositis (DM), necrotizing autoimmune myopathy (NAM), and sporadic inclusion body myositis (sIBM). DM is characterized by a skin rash in addition to muscle weakness. NAM presents with severe weakness but no rash. sIBM typically affects older males and causes both proximal and distal weakness. The document provides details on clinical manifestations and diagnostic criteria for each IIM subtype.
This document discusses idiopathic inflammatory myopathies (IIM), including their classification, clinical features, epidemiology, and relevant literature. The main types of IIM are polymyositis (PM), dermatomyositis (DM), necrotizing autoimmune myopathy (NAM), and sporadic inclusion body myositis (sIBM). DM is characterized by a skin rash in addition to muscle weakness. NAM presents with severe weakness but no rash. sIBM typically affects older males and causes both proximal and distal weakness. The document provides details on clinical manifestations and diagnostic criteria for each IIM subtype.
This document reviews glomerulonephritis as a cause of acute kidney injury. It discusses several types of glomerulonephritis that can lead to AKI, including anti-GBM disease, small vessel vasculitis, lupus nephritis, IgA nephropathy, and post-streptococcal glomerulonephritis. Kidney biopsy is important for diagnosis but not always possible in critically ill patients. Treatment depends on the specific type of glomerulonephritis and may include corticosteroids, cyclophosphamide, plasma exchange, and immunosuppression. Prognostic markers include the extent of glomerular lesions, need for dialysis, and creatin
This document reviews glomerulonephritis as a cause of acute kidney injury. It discusses several types of glomerulonephritis that can lead to AKI, including anti-GBM disease, small vessel vasculitis, lupus nephritis, IgA nephropathy, and post-streptococcal glomerulonephritis. Kidney biopsy is important for diagnosis but not always possible in critically ill patients. Treatment depends on the specific type of glomerulonephritis and may include corticosteroids, cyclophosphamide, plasma exchange, and immunosuppression. Prognostic markers include the extent of glomerular lesions, need for dialysis, and creatin
Drug-induced hypersensitivity syndrome (DIHS)/Drug reaction with eosinophilia and systemic symptoms (DRESS)
Presented by Pongsawat Rodsaward, MD.
December 17, 2021
Drug-induced hypersensitivity syndrome (DIHS)/Drug reaction with eosinophilia and systemic symptoms (DRESS)
Presented by Pongsawat Rodsaward, MD.
December 17, 2021
The document discusses mast cell disorders and their classification. It describes localized mastocytosis, which includes cutaneous mastocytosis subtypes like urticaria pigmentosa and diffuse cutaneous mastocytosis. Systemic mastocytosis is classified into several subtypes based on severity from indolent to aggressive forms. The pathogenesis involves mutations in the KIT gene in most cases that lead to mast cell proliferation. Clinical features can include systemic symptoms from mast cell mediators as well as dermatologic findings like the rash of urticaria pigmentosa.
The document discusses mast cell disorders and their classification. It describes localized mastocytosis, which includes cutaneous mastocytosis subtypes like urticaria pigmentosa and diffuse cutaneous mastocytosis. Systemic mastocytosis is classified into several subtypes based on severity from indolent to aggressive forms. The pathogenesis involves mutations in the KIT gene in most cases that lead to mast cell proliferation. Clinical features can include systemic symptoms from mast cell mediators as well as dermatologic findings like the rash of urticaria pigmentosa.
Rheumatoid arthritis is a systemic inflammatory disease that causes swelling and tenderness of the joints. It results from a breakdown of immune tolerance that leads to inflammation of the synovial tissue lining joints. The disease predominantly affects females and occurs most often between the ages of 35-50. Treatment involves both pharmacological and non-pharmacological approaches. Pharmacological treatment starts with NSAIDs and progresses to DMARDs like methotrexate and biologics if needed to reduce inflammation, prevent further joint damage, and control symptoms.
Rheumatoid arthritis is a systemic inflammatory disease that causes swelling and tenderness of the joints. It results from a breakdown of immune tolerance that leads to inflammation of the synovial tissue lining joints. The disease predominantly affects females and occurs most often between the ages of 35-50. Treatment involves both pharmacological and non-pharmacological approaches. Pharmacological treatment starts with NSAIDs and progresses to DMARDs like methotrexate and biologics if needed to reduce inflammation, prevent further joint damage, and control symptoms.
The document discusses principles of treating infectious illnesses in critical care, with a focus on antibiotic resistance and choice of antibiotics. It covers several topics: the impact of antibiotic use on resistance; choosing initial antibiotics and tailoring treatment based on culture results; applying pharmacology and pharmacodynamics to optimize bacterial killing; and reviewing guidelines for specific infections. It also provides an overview of antibiotic classes, mechanisms of action, considerations for dosing in renal impairment, and highlights specific agents like penicillins, cephalosporins, and vancomycin.
The document discusses principles of treating infectious illnesses in critical care, with a focus on antibiotic resistance and choice of antibiotics. It covers several topics: the impact of antibiotic use on resistance; choosing initial antibiotics and tailoring treatment based on culture results; applying pharmacology and pharmacodynamics to optimize bacterial killing; and reviewing guidelines for specific infections. It also provides an overview of antibiotic classes, mechanisms of action, considerations for dosing in renal impairment, and highlights specific agents like penicillins, cephalosporins, and vancomycin.
Presentazione a cura del Professor Franco Scaldaferri - M.A.S.T.E.R. ECM in Gastroenterologia: Approccio personalizzato alla complessità in Gastroenterologia - Fondazione Santa Lucia - Roma 19/01/2018
Presentazione a cura del Professor Franco Scaldaferri - M.A.S.T.E.R. ECM in Gastroenterologia: Approccio personalizzato alla complessità in Gastroenterologia - Fondazione Santa Lucia - Roma 19/01/2018
20 July 2022 Diagnosis and Treatment of Multiple Sclerosis.pptxmanjujanhavi
This document provides an overview of the diagnosis and treatment of multiple sclerosis (MS). It discusses the epidemiology, pathophysiology, clinical presentation, diagnosis using the McDonald criteria, disease course classifications (relapsing-remitting vs progressive), natural history, outcomes measures, and available treatment options for MS. Treatment options include disease-modifying therapies (DMTs) such as injectable medications (interferons, glatiramer acetate), oral medications (fingolimod, dimethyl fumarate, teriflunomide), monoclonal antibody therapies (ocrelizumab, natalizumab), and management of relapses, symptoms, and comorbidities. The goal of treatment is to reduce rel
20 July 2022 Diagnosis and Treatment of Multiple Sclerosis.pptxmanjujanhavi
This document provides an overview of the diagnosis and treatment of multiple sclerosis (MS). It discusses the epidemiology, pathophysiology, clinical presentation, diagnosis using the McDonald criteria, disease course classifications (relapsing-remitting vs progressive), natural history, outcomes measures, and available treatment options for MS. Treatment options include disease-modifying therapies (DMTs) such as injectable medications (interferons, glatiramer acetate), oral medications (fingolimod, dimethyl fumarate, teriflunomide), monoclonal antibody therapies (ocrelizumab, natalizumab), and management of relapses, symptoms, and comorbidities. The goal of treatment is to reduce rel
Abstract—In Italy the hydatid disease is more prevalent and new cases are highlighted more frequently in Sicily, Sardinia, (Italy). Aim of this study is to put the indication in search of iaditea nature in both spleen swelling and muscle tendon.
Material and Method Patients observed during the period 2007-2009 at the Surgical Clinic III and Digestive Surgery, Policlinico G Rodolico were explored for Hydatid cyste at various sites. Diagnosis of cysts ecchinococcus occurred primarily for various four reasons either for compression of bodies involved or for eosinophilia or for instrumental investigation or for anaphylactic reaction to rupture of cysts. Biological diagnosis is based on serology rather than isolation of the parasite (indirect diagnosis);
Results Patients attended during the period 2007-2009 Hydatid cyst was found in 0.5% of all cases in liver along with 4 in the lung, 3 in splenic, 2 in the mammary and 2 in the chest wall No 2. The Surgical treatment with the complete removal of the cyst with a satisfactory postoperative course in the absence of cases of relapse of the disease and by following the therapeutic act, the assumption of mebendazole 50mg / kg / day for 3 weeks at a dose of 400mg for 4 months
Conclusions There is a need to define diagnostic methods with high specificity and sensitivity, which can provide a valid diagnostic aid for the cases clinically difficult to diagnose. And the final diagnosis must then also be based on the development of immunological methods that allow the determination of specific antibodies in the serum and their titration and / or the circulating antigen determination.
Abstract—In Italy the hydatid disease is more prevalent and new cases are highlighted more frequently in Sicily, Sardinia, (Italy). Aim of this study is to put the indication in search of iaditea nature in both spleen swelling and muscle tendon.
Material and Method Patients observed during the period 2007-2009 at the Surgical Clinic III and Digestive Surgery, Policlinico G Rodolico were explored for Hydatid cyste at various sites. Diagnosis of cysts ecchinococcus occurred primarily for various four reasons either for compression of bodies involved or for eosinophilia or for instrumental investigation or for anaphylactic reaction to rupture of cysts. Biological diagnosis is based on serology rather than isolation of the parasite (indirect diagnosis);
Results Patients attended during the period 2007-2009 Hydatid cyst was found in 0.5% of all cases in liver along with 4 in the lung, 3 in splenic, 2 in the mammary and 2 in the chest wall No 2. The Surgical treatment with the complete removal of the cyst with a satisfactory postoperative course in the absence of cases of relapse of the disease and by following the therapeutic act, the assumption of mebendazole 50mg / kg / day for 3 weeks at a dose of 400mg for 4 months
Conclusions There is a need to define diagnostic methods with high specificity and sensitivity, which can provide a valid diagnostic aid for the cases clinically difficult to diagnose. And the final diagnosis must then also be based on the development of immunological methods that allow the determination of specific antibodies in the serum and their titration and / or the circulating antigen determination.
Understanding the molecular biology of intervertebral disc degeneration and p...CarlosAlbertoMontesM
This document reviews gene therapy strategies for treating intervertebral disc degeneration. It discusses the molecular biology and etiology of disc degeneration, which involves an imbalance between catabolism and anabolism leading to loss of extracellular matrix. Current treatments only relieve symptoms and do not address the underlying pathology. The review examines growth factor therapy, cell therapy, and gene therapy approaches for regeneration. Gene therapy was initially mediated by viral vectors but recent advances allow for non-viral methods to avoid risks of mutagenesis and infection. With further progress, non-viral gene therapy may prove effective for treating disc degeneration.
Understanding the molecular biology of intervertebral disc degeneration and p...CarlosAlbertoMontesM
This document reviews gene therapy strategies for treating intervertebral disc degeneration. It discusses the molecular biology and etiology of disc degeneration, which involves an imbalance between catabolism and anabolism leading to loss of extracellular matrix. Current treatments only relieve symptoms and do not address the underlying pathology. The review examines growth factor therapy, cell therapy, and gene therapy approaches for regeneration. Gene therapy was initially mediated by viral vectors but recent advances allow for non-viral methods to avoid risks of mutagenesis and infection. With further progress, non-viral gene therapy may prove effective for treating disc degeneration.
Similar to Scleroderma: Treatment Options and a Look to the Future - Dr. Macklin (20)
This talk was presented by Michael Macklin, MD from the University of Chicago at the Scleroderma Patient Education Conference on May 4, 2024, hosted by the Scleroderma Foundation of Greater Chicago. This talk includes:
Overview of scleroderma manifestations, organ involvement, brief classifications (limited, diffuse, sine scleroderma)
Overview of current treatment options, need for additional therapies
Overview of plan for multi-disciplinary scleroderma center at the University of Chicago
Potential future therapies in the literature at large
Planned trials/future treatment options at the University of Chicago
For more info about scleroderma and the foundation, head to www.stopscleroderma.org
Interstitial lung disease (ILD) is a common complication of scleroderma that leads to inflammation and scarring of the lungs. In this session, we will review the prevalence of scleroderma-associated ILD (SSc-ILD), classic symptoms, and the approach to evaluating patients with suspected disease. In addition, we will cover various treatments available for patients with SSc-ILD.
This talk was presented at the Scleroderma Patient Education Conference on May 4, 2024, hosted by the Scleroderma Foundation of Greater Chicago.
For more info about scleroderma and the foundation, head to www.stopscleroderma.org
This session will discuss modalities and demonstrate exercises to improve movement and function in the hands, face and mouth. Suggestions will be also be provided on the use of assistive devices and alternate techniques to accomplish tasks of daily living to increase independence and protect the hands.
This presentation was held on May 4, 2024 by the Scleroderma Foundation of Greater Chicago.
For more information on the foundation and scleroderma, head to our website at www.stopscleroderma.org
Chronic pain is common. If we don’t suffer from it ourselves, chances are we know someone who does. Changes in the structure and function of the brain are thought to underlie chronic pain. The good news is that these changes are not hardwired. Many things can be done to influence how the brain processes pain signals including exercise, healthy eating, and better sleep, as well as thinking more adaptive thoughts, positive emotions, and feeling love and connected. This session will highlight the neuroscience related to chronic pain and how engaging in simple self-management strategies can result in less pain and a more rewarding life.
This presentation comes from the Spring Patient Education conference presented by the Scleroderma Patient Education Conference presented by the Scleroderma Foundation of Greater Chicago.
See the slides from the Scleroderma Foundation of Greater Chicago's Workshop: Improving Mental Health with Chronic Illness. This presentation was held by the mental health professionals at Ellie Mental Health.
Learn from Bethany Doerfler, MS, RD, LDN, a registered dietitian whose clinical practice and research focuses on providing wellness-based medical nutrition therapy for digestive disorders and allergic bowel diseases. She currently practices in the Division of Gastroenterology and Hepatology at Northwestern Medicine in Chicago, IL. She is the first dietitian to be fully integrated into a gastroenterology division for both research and patient care. This presentation is optimized for Scleroderma patients to learn about their diet options to improve scleroderma symptoms and their gut health.
This presentation covers gastrointestinal issues, which are commonly experienced by those living with scleroderma. This session is set to be an invaluable resource for patients and caregivers, as it will provide crucial insights and approaches to managing GI issues effectively. Dr. Khanna's vast knowledge and experience make this talk a must-attend event for anyone seeking to enhance their understanding and management of GI symptoms in scleroderma.
This document discusses calcinosis, a complication of scleroderma where calcium deposits form in the soft tissues. It outlines that calcinosis most commonly affects the hands, joints, knees and muscles. While the calcium levels in the blood are normal, inflammation and low blood flow in tissues are thought to contribute to calcinosis formation. The deposits contain bone-like proteins and crystals. 18F-NaF PET imaging may help detect early or active calcinosis. Treatments discussed include protecting joints, surgical removal of deposits, topical or injected sodium thiosulfate, medications like tofacitinib, NSAIDs, colchicine and minimizing scleroderma complications to reduce calcinosis risk over
This talk will center around the crucial topic of interstitial lung disease (ILD). Gain invaluable insights into the latest advancements in ILD management, potential treatment options, and the importance of clinical trials in advancing care for scleroderma patients.
Dr. Cuttica and Dr. Mylvaganam will co-lead an insightful talk on pulmonary hypertension (PH). Attendees will have the opportunity to learn about pulmonary hypertension, one of the most serious conditions that impact individuals with scleroderma. The talk will give an overview of pulmonary hypertension and potential treatment options.
In this talk we will discuss the most common findings associated with scleroderma. We will discuss some of the methods your dental team can utilize to help manage your condition, and also some ways that you can help yourself and your dental team manage your condition. We will discuss some unique methods for maintaining your oral health care and will conclude with an open Q&A session.
Virtually every aspect of systemic sclerosis can be beneficially impacted by exercise: inflammation, circulation, body warmth, GI, skin, musculoskeletal and lung health.
The therapeutic underpinnings of exercise target the specific mechanisms behind the pervasive SSc-disease biological, physical and psychological manifestations.
This session is intended to empower people living with scleroderma with knowledge of systemic sclerosis and the anticipated impact exercise and physical activity can have on the many manifestations of systemic sclerosis.
At the end of this session, attendees should have a better understanding of the extent of SSc and feel confident in constructing an exercise regimen generally and for their particular needs related to scleroderma.
This talk will review the best practices for monitoring for the early detection of interstitial lung disease (ILD) and pulmonary hypertension (PH), the two most common and serious lung diseases that occur in patients with scleroderma. It will also cover the many new medications approved for the treatment of ILD and PH and when these medications are indicated. The goal is for patients with scleroderma to understand the recent advances in the diagnosis and treatment of scleroderma-associated lung diseases that are leading to improved outcomes.
In this talk, Dr. Brown will expand your knowledge of how scleroderma impacts the GI tract. This presentation is crucial as an estimated 90% of scleroderma patients suffer from gastrointestinal complications.
Dr. Brown is well-known for his exceptional ability to make complex medical information easy to understand.
Presented by Murray Baron, MD at the Scleroderma Patient Education Conference, hosted by the Scleroderma Foundation Greater Chicago Chapter on Saturday, October 12, 2019 in Chicago, IL. For more about the foundation visit scleroderma.org/chicago.
Presented by Jennifer Mundt, PhD at the Scleroderma Patient Education Conference, hosted by the Scleroderma Foundation Greater Chicago Chapter on Saturday, October 12, 2019 in Chicago, IL.
This document summarizes a presentation on recent developments in treating scleroderma interstitial lung disease. It discusses that ILD affects the lung interstitium and causes decreased oxygen transfer and stiff lungs. ILD risk is higher in patients with autoantibodies like anti-Scl-70. New data shows that long-term lung function loss predicts benefit from anti-fibrotic drugs. The Senscis trial found that nintedanib slowed lung function decline in SSc-ILD patients. Hematopoietic stem cell transplant trials like ASTIS and SCOT showed reduced mortality compared to controls, but transplant-related mortality remains a concern. A new Scleroderma Lung Study trial will test pirfen
This document provides an overview of gastrointestinal manifestations and treatment in scleroderma. It discusses how 60-90% of scleroderma patients experience GI involvement, most commonly affecting the esophagus, stomach, small intestine, and colon. For the esophagus, it covers GERD, dysphagia, and their diagnostic tests and treatments like PPIs. For the stomach, it discusses gastroparesis, GAVE, and treatments like prokinetic agents and APC. It reviews SIBO, CIPO, and treatments for the small intestine. For the colon and anus, it covers constipation, fecal incontinence, diagnostic tests, and treatments including laxatives, bio
Presented by Dr. JoAnna Harper, PharmD at the Scleroderma Patient Education Conference hosted by the Scleroderma Foundation Greater Chicago Chapter on April 27, 2019 in Oakbrook, IL.
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low birth weight presentation. Low birth weight (LBW) infant is defined as the one whose birth weight is less than 2500g irrespective of their gestational age. Premature birth and low birth weight(LBW) is still a serious problem in newborn. Causing high morbidity and mortality rate worldwide. The nursing care provide to low birth weight babies is crucial in promoting their overall health and development. Through careful assessment, diagnosis,, planning, and evaluation plays a vital role in ensuring these vulnerable infants receive the specialize care they need. In India every third of the infant weight less than 2500g.
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Respiratory issues like asthma are the most sensitive issue that is affecting millions worldwide. It hampers the daily activities leaving the body tired and breathless.
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These lecture slides, by Dr Sidra Arshad, offer a simplified look into the mechanisms involved in the regulation of respiration:
Learning objectives:
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7. Explain the role of peripheral chemoreceptors in regulation of respiration
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3. Chapter 13, Human Physiology by Lauralee Sherwood, 9th edition
Test bank for karp s cell and molecular biology 9th edition by gerald karp.pdfrightmanforbloodline
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The skin is the largest organ and its health plays a vital role among the other sense organs. The skin concerns like acne breakout, psoriasis, or anything similar along the lines, finding a qualified and experienced dermatologist becomes paramount.
Lecture 6 -- Memory 2015.pptlearning occurs when a stimulus (unconditioned st...AyushGadhvi1
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The development of nanogold-based cancer therapy could revolutionize oncology by providing a more targeted, less invasive treatment option. This project contributes to the growing body of research aimed at harnessing nanotechnology for medical applications, paving the way for future clinical trials and potential commercial applications.
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- Video recording of this lecture in English language: https://youtu.be/Pt1nA32sdHQ
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Travel Clinic Cardiff offers comprehensive travel health services, including vaccinations, travel advice, and preventive care for international travelers. Our expert team ensures you are well-prepared and protected for your journey, providing personalized consultations tailored to your destination. Conveniently located in Cardiff, we help you travel with confidence and peace of mind. Visit us: www.nxhealthcare.co.uk
2. OBJECTIVES
• Classification and manifestations of scleroderma
• Overview of treatment options for scleroderma
• Potential treatments on the horizon at large and at the University of Chicago
• Multi disciplinary care in scleroderma
3. ETIOLOGY
• Scleroderma is an incompletely understood disease
• There is clearly an autoimmune basis with vascular dysfunction, and activation of
fibroblasts leading to fibrosis
4. Benfaremo D, Svegliati S, Paolini C, Agarbati S, Moroncini G. Systemic Sclerosis: From Pathophysiology to Novel Therapeutic Approaches. Biomedicines. 2022; 10(1):163. https://doi.org/10.3390/biomedicines10010163
5. CLASSIFICATION
• Limited: Skin involvement that does not go past elbows or knees for limbs, no trunk
involvement, face/neck can be involved
• Diffuse: Skin involvement that does go past elbows or knees for limbs, trunk
involvement, face/neck can be involved
• Sine scleroderma: No skin involvement, still has Raynaud's, antibodies, internal
organ involvement
6. CLASSIFICATION
• Limited and diffuse tend to have different antibody profiles (SCL-70 for diffuse,
centromere for limited)
• Diffuse tends to have an aggressive course initially then becomes less active
overtime while limited is more slowly but consistently progressive
• Diffuse has more direct lung damage (interstitial lung disease) while limited has
more lung blood vessel involvement (pulmonary artery hypertension)
• Diffuse in general is more “fibrotic”, limited in general is more “vascular”
8. Mammino , J. (n.d.). Systemic sclerosis . American Osteopathic College of Dermatology . https://www.aocd.org/page/SystemicSclerosis
9. McMichael , J. (2018, November 7). Prayer sign. Twitter . https://twitter.com/globaldermie
10. July 22, 2021,N Engl J Med 2021; 385:357DOI: 10.1056/NEJMicm2103390
11. Barrett, C. (2017, August 16). Clinical Signs of Systemic Sclerosis. Stanford Medicine 25. https://stanfordmedicine25.stanford.edu/blog/archive/2017/scleroderma.html
12. YAMAMOTO T. MULTIPLE CALCINOSIS CUTIS AS A RESULT OF KÖBNER PHENOMENON IN A PATIENT WITH SYSTEMIC SCLEROSIS. OUR DERMATOL ONLINE. 2020;11(E):E176.1-E176.2
14. (2014, SEPTEMBER 20). GAVE (GASTRIC ANTRAL VASCULAR ECTASIA). UPDATE IN GASTROENTEROLOGY. HTTPS://UPTODATEGASTRO.WORDPRESS.COM/2014/09/20/GAVE-GASTRIC-ANTRAL-
VASCULAR-ECTASIA/
15. LUNG INVOLVEMENT
• Interstitial lung disease (Inflammation and fibrosis within the lung tissue)
• Pulmonary artery hypertension (high blood pressure in the pulmonary artery)
16. Weerakkody, Y. (2023, May 15). Scleroderma (pulmonary manifestations): Radiology reference article.
Radiopaedia. https://radiopaedia.org/articles/scleroderma-pulmonary-manifestations-1
Di Muzio B, Normal chest CT. Case study, Radiopaedia.org (Accessed on 09 Feb
2024) https://doi.org/10.53347/rID-41162
17. Phillips, Q. (2022, July 18). What is pulmonary arterial hypertension (PAH)? Symptoms, Causes, Diagnosis, Treatment, and Prevention. EverydayHealth.com.
https://www.everydayhealth.com/pulmonary-arterial-hypertension
18. KIDNEY
• Scleroderma renal crisis
USMLE® Step 2 CK question of the day: Scleroderma renal crisis . Osmosis . (2023, February 22). https://www.osmosis.org/blog/2021/12/15/usmle-step-2-ck-
question-of-the-day-scleroderma-renal-crisis
19. CARDIAC
• Myocarditis (inflammation of the heart muscle)
• Pericarditis (inflammation of the sac around the heart)
• Heart failure
• Arrythmias (Abnormal heart rhythms)
20. MUSCLE
• Myositis (inflammation in muscles) in overlap
Srikantharajah, D., Lloyd, M.E. & Kiely, P.D.W. Rituximab and intravenous
immunoglobulin treatment in PM/Scl antibody-associated disease: case-based
review. Rheumatol Int 42, 359–364 (2022). https://doi.org/10.1007/s00296-021-
05075-z
22. October 24, 2013, N Engl J Med 2013; 369:1638, DOI: 10.1056/NEJMicm1304702
23. TREATMENT
• Much progress has been made
• Treatment generally guided by manifestations present
• Some organ involvement still has few options available
24. SKIN THICKENING
Treatment options include:
• Methotrexate (anti metabolite)
• Mycophenolate (anti metabolite)
• Rituximab? (biologic, anti B-Cell)
• Tocilizumab? (biologic, anti inflammatory signal molecule, IL-6)
25. SKIN THICKENING
• Immunoglobulin therapy? (blood product)
• Cyclophosphamide rarely (DNA disrupting agent)
• Area of significant need overall
27. TELANGIECTASIA
Graham Dinsdale, Andrea Murray, Tonia Moore, Janice Ferguson, Jack Wilkinson, Helen Richards, Christopher E. M. Griffiths, Ariane L Herrick, A
comparison of intense pulsed light and laser treatment of telangiectases in patients with systemic sclerosis: a within-subject randomized trial,
Rheumatology, Volume 53, Issue 8, August 2014, Pages 1422–1430, https://doi.org/10.1093/rheumatology/keu006
28. CALCINOSIS
• Area of significant need
• Injections or compounded sodium thiosulfate cream around area
• Minocycline (oral, antibiotic/general anti-inflammatory properties)
• Tofacitinib? (Oral, targeted selective molecule, JAK inhibitor), data from myositis
• Surgical removal
29. GAVE (WATERMELON STOMACH)
• Argon plasma coagulation (laser to burn off blood vessels)
• Therapy used for other manifestations may also help
30. GAVE (WATERMELON STOMACH)
Ohira, Tetsuya et al. “Detection of active bleeding from gastric antral vascular ectasia by capsule endoscopy.” World journal of gastrointestinal endoscopy vol. 5,3 (2013): 138-40. doi:10.4253/wjge.v5.i3.138
31. DYSMOTILITY IN THE
GASTROINTESTINAL SYSTEM
• An area of great unmet need
• Proton pump inhibitors for reflux (pantoprazole, esomeprazole, etc)
• Various promotility drugs (Metoclopramide, erythromycin, pyridostigmine,
prucalopride)
• Immunoglobulin therapy? (blood product)
Nassar M, Ghernautan V, Nso N, et al. Gastrointestinal involvement in systemic sclerosis: An updated review. Medicine (Baltimore). 2022;101(45):e31780.
doi:10.1097/MD.0000000000031780
Matsuda, Kazuki M et al. “Rapid improvement of systemic sclerosis-associated intestinal pseudo-obstruction with intravenous immunoglobulin administration.” Rheumatology (Oxford,
England) vol. 62,9 (2023): 3139-3145. doi:10.1093/rheumatology/kead093
32. DYSMOTILITY IN THE
GASTROINTESTINAL SYSTEM
• Over the counter laxatives can be helpful in early stages (senna, polyethylene glycol,
etc.)
• Prolonged dysmotility can lead to diarrhea from small intestinal bowel overgrowth,
easily treatable with antibiotics
• Severe dysmotility can lead to malnutrition, need to bypass intestines, total parental
nutrition (TPN)
33. MUSCLE INVOLVEMENT
• Inflammatory myositis is also undergoing a treatment revolution
• Immunoglobulin therapy (blood product) now standard of care
• Other therapies if myositis is a predominant feature
34. SCLERODERMA RENAL CRISIS
• Prevention is important, association with high prednisone doses, especially in
patients who are RNA polymerase III positive
• Treated with high doses of blood pressure medicine, mainly angiotensin converting
enzyme inhibitors (ACEI) such as lisinopril, captopril
35. INTERSTITIAL LUNG DISEASE
(INFLAMMATION AND FIBROSIS WITHIN
THE LUNG TISSUE)
• Mycophenolate (anti metabolite) standard of care
• Azathioprine (anti metabolite), may be inferior to other options
• Rituximab (biologic, anti B-Cell)
• Tocilizumab (biologic, anti inflammatory signal molecule, IL-6)
• Tocilizumab is one of two FDA approved therapies for scleroderma, specifically for ILD (2021)
Roofeh, David et al. “Tocilizumab Prevents Progression of Early Systemic Sclerosis-Associated Interstitial Lung Disease.” Arthritis & rheumatology (Hoboken, N.J.) vol. 73,7 (2021): 1301-
1310. doi:10.1002/art.41668
Kaufman M. FDA Approves Tocilizumab to Treat Systemic Sclerosis-Associated ILD. The Rheumatologist. Published March 25, 2021. Accessed February 17, 2024. https://www.the-
rheumatologist.org/article/fda-approves-tocilizumab-to-treat-systemic-sclerosis-associated-ild/
36. INTERSTITIAL LUNG DISEASE
(INFLAMMATION AND FIBROSIS WITHIN
THE LUNG TISSUE)
• Cyclophosphamide rarely (DNA disrupting agent)
• Combination therapy (Mycophenolate + biologic)?
• Nintedanib in cases of significant fibrosis (anti fibrotic), generally added to
immunosuppression
• Nintedanib is the only other FDA approved therapy, ILD (2019)
Putman M. FDA Approves Nintedanib for SSc-ILD, But Temper Your Expectations. The Rheumatologist. Published December 18, 2019. https://www.the-
rheumatologist.org/article/fda-approves-nintedanib-for-ssc-ild-but-temper-your-expectations/
37. PULMONARY ARTERY HYPERTENSION
(HIGH BLOOD PRESSURE IN THE
PULMONARY ARTERY)
• Managed by a pulmonary hypertension specialist (pulmonary and/or cardiology
depending on center)
• Combination therapy generally used
• Sildenafil, tadalafil (Phosphodiesterase V inhibitor) (vasodilator)
• Ambrisentan, macitentan, bosentan (endothelin receptor antagonist) (prevents
vasoconstriction)
Kuwana, Masataka et al. “Initial combination therapy of ambrisentan and tadalafil in connective tissue disease-associated pulmonary arterial hypertension (CTD-PAH) in the modified intention-to-treat population of
the AMBITION study: post hoc analysis.” Annals of the rheumatic diseases vol. 79,5 (2020): 626-634. doi:10.1136/annrheumdis-2019-216274
38. PULMONARY ARTERY HYPERTENSION
(HIGH BLOOD PRESSURE IN THE
PULMONARY ARTERY)
• Epoprostenol, treprostinil, iloprost (prostacyclin analogues) (vasodilator)
39. HEART INVOLVMENT
• An area of great unmet need
Myocarditis (inflammation of the heart muscle)
• Immunosuppression seems to work, unknown best drug
Pericarditis (inflammation of the sac around the heart)
• Treated like pericarditis outside of scleroderma (Naproxen/ibuprofen, colchicine)
40. HEART INVOLVMENT
Heart failure/arrythmia (Abnormal heart rhythms)
• Treated like causes outside of scleroderma
• Significant heart failure from scleroderma carries a very poor prognosis
41. RAYNAUD'S
• Lifestyle modification (gloves, cold avoidance, hand warmers, etc)
• Amlodipine, nifedipine (Calcium channel blockers)
• Sildenafil, tadalafil (Phosphodiesterase V inhibitor)
• Fluoxetine? (SSRI)
• Losartan? (ARB)
• Smoking cessation!
Dziadzio, M et al. “Losartan therapy for Raynaud's phenomenon and scleroderma: clinical and biochemical findings in a fifteen-week, randomized, parallel-group, controlled trial.” Arthritis and rheumatism vol. 42,12 (1999):
2646-55. doi:10.1002/1529-0131(199912)42:12<2646::AID-ANR21>3.0.CO;2-T
Coleiro, B et al. “Treatment of Raynaud's phenomenon with the selective serotonin reuptake inhibitor fluoxetine.” Rheumatology (Oxford, England) vol. 40,9 (2001): 1038-43. doi:10.1093/rheumatology/40.9.1038
42. RAYNAUD'S
• Botox? (controversial, may actually worsen)
• Fat transfer with plastic surgery?
• Digital sympathectomy? (surgical stripping of sympathetic nervous system)
• Bosentan (endothelin receptor antagonist) (prevents vasoconstriction)
• Epoprostenol (prostacyclin analogue) (vasodilator)
• Peripheral nerve block (temporary)
Senet, Patricia et al. “Efficacy and Safety of Botulinum Toxin in Adults with Raynaud's Phenomenon Secondary to Systemic Sclerosis: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study.” Arthritis &
rheumatology (Hoboken, N.J.) vol. 75,3 (2023): 459-467. doi:10.1002/art.42342
43. FUTURE TREATMENT
• Many potential treatment options are in development!
• Some are specific for ILD, other multiple system involvement
44. FT011
• Oral drug, Phase 2 in diffuse scleroderma
• Is a G protein-coupled receptor 68 protein sensing antagonist, inhibits pro-fibrotic and
inflammatory pathways
• Improved a variety of outcomes including lung function, skin thickening vs placebo
• GI side effects and photosensitivity seem to be the most common side effects
• Phase 3 to come
Khanna D, Spino C, Bernstein E, Goldin J, Tashkin D, roth M, SLS III Investigators O. Combination Therapy of Mycophenolate Mofetil and Pirfenidone vs. Mycophenolate Alone: Results from the
Scleroderma Lung Study III [abstract]. Arthritis Rheumatol. 2022; 74 (suppl 9). https://acrabstracts.org/abstract/combination-therapy-of-mycophenolate-mofetil-and-pirfenidone-vs-mycophenolate-
alone-results-from-the-scleroderma-lung-study-iii/. Accessed February 16, 2024
45. GUSELKUMAB
• IL-23 inhibitor (biologic)
• Used in psoriatic arthritis, psoriasis, data in Crohn’s
• Phase 2 trial in Japan
• Primary outcome is skin thickening
https://clinicaltrials.gov/study/NCT04683029
46. ANIFROLUMAB
• Type 1 interferon blocker (biologic)
• Used in Lupus, especially for skin involvement
• Phase 3 study
• Evaluating effect on lung function and skin thickening mainly
• Chicago site is planned, not yet recruiting
https://clinicaltrials.gov/study/NCT05925803
47. IXAZOMIB
• Ixazomib is an oral proteosome inhibitor
• Currently used in multiple myeloma
• Phase 2 study
• Recruiting at Mayo Clinic Arizona
• Evaluating effect on ILD and skin thickening
https://www.clinicaltrials.gov/study/NCT04837131
48. BARICITINIB
• A JAK inhibitor used in COVID-19, RA
• Phase 4 trial in China evaluating use in ILD and skin thickening compared with
cyclophosphamide
https://clinicaltrials.gov/study/NCT05300932
49. RO7303509
• RO7303509 is a biologic that targets TGF-β3 (antifibrotic)
• Phase 1A already done, recruiting for Phase 1B
• Sites include DC, Boston, Rochester MN, NYC
Han L, Jamalian S, Pan L, et alPOS1266 A PHASE IA, RANDOMIZED STUDY TO EVALUATE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SINGLE ASCENDING
DOSES OF RO7303509, AN ANTI-TGFΒ3 MONOCLONAL ANTIBODY, IN HEALTHY VOLUNTEERS Annals of the Rheumatic Diseases 2023;82:976-977.
https://clinicaltrials.gov/study/NCT05462522
50. INEBILIZUMAB
• Anti CD19 biologic (B cell targeting)
• Phase 3 placebo-controlled study
• Evaluating skin thickening
• Taking place in Japan
• Already approved for Neuromyelitis Optica (NMO)
https://clinicaltrials.gov/study/NCT05198557
51. BELIMUMAB AND RITUXIMAB
• Belimumab is an anti B-lymphocyte stimulator (BLyS) biologic
• Currently used in Lupus
• Combination of belimumab and rituximab vs placebo
• Phase 2, recruiting in NYC
• Evaluating ILD and skin thickening
https://clinicaltrials.gov/study/NCT03844061
52. BELIMUMAB
• Primary outcome is lung function
• Phase 3 study, placebo controlled
• Northwestern is a site-recruiting per clinicaltrials.gov
https://clinicaltrials.gov/study/NCT05878717
53. IFETROBAN
• Thromboxane receptor antagonist
• Oral drug, phase 2 study, placebo controlled
• Looking specifically at both diffuse scleroderma and at patients with confirmed
pulmonary artery hypertension
• Secondary endpoints include skin thickening, lung function
• Closest sites include NYC, Philadelphia, Omaha, Boston, Baltimore
https://clinicaltrials.gov/study/NCT02682511
54. HZN-825
• HZN-825 is a lysophosphatidic acid receptor blocker, lysophosphatidic acid is
thought to be involved in fibrosis
• Phase 2 placebo-controlled study in diffuse scleroderma
• Evaluating lung function, skin thickening
• Sites include Ann Arbor, NYC, Boston, Philadelphia, Rochester MN
https://clinicaltrials.gov/study/NCT04781543
55. TULISOKIBART
• Tulisokibart is biologic targeting tumor necrosis factor (TNF)-like cytokine 1A
• Also being evaluated in IBD
• Placebo controlled phase 2 in patients with diffuse disease with ILD
• Mainly looking at lung function
• Recruiting centers include DC, Baltimore, Boston, Ann Arbor, Cleveland, Toledo,
Milwaukee, Pittsburgh, NYC
https://clinicaltrials.gov/study/NCT05270668
56. VIXARELIMAB
• Vixarelimab blocks the oncostatin M receprot, blocking signaling of oncostatin M
and interleukin 31
• Oncostatin M/IL-31 are thought to be involved in itch, inflammation and fibrosis
• Primarily evaluating lung function in ILD
• Sites include Northwestern-Glenview, Muncie IN
Sofen, Howard et al. “Efficacy and safety of vixarelimab, a human monoclonal oncostatin M receptor β antibody, in moderate-to-severe prurigo nodularis: a randomised, double-blind, placebo-
controlled, phase 2a study.” EClinicalMedicine vol. 57 101826. 3 Feb. 2023, doi:10.1016/j.eclinm.2023.101826
https://clinicaltrials.gov/study/NCT05785624
57. AUTOLOGOUS STEM CELL
TRANSPLANT
Chatterjee S, Majhail N. Autologous Hematopoietic Cell Transplantation: Way of the Future in Severe Systemic Sclerosis? Cleveland Clinic. Published November 30, 2018. Accessed February 17, 2024.
https://consultqd.clevelandclinic.org/autologous-hematopoietic-cell-transplantation-way-of-the-future-in-severe-systemic-sclerosis
58. AUTOLOGOUS STEM CELL
TRANSPLANT
• Has shown benefit for a variety of manifestations in scleroderma
• May be reasonable in rapidly progressive cases
• Exact patient selection evolving
• George Georges, MD at Northwestern is running a trial through heme/onc
• University of Chicago will be referring to him as appropriate
Bruera, Sebastian et al. “Stem cell transplantation for systemic sclerosis.” The Cochrane database of systematic reviews vol. 7,7 CD011819. 29 Jul. 2022, doi:10.1002/14651858.CD011819.pub2
Sullivan KM, Majhail NS, Bredeson C, et al. Systemic Sclerosis as an Indication for Autologous Hematopoietic Cell Transplantation: Position Statement from the American Society for Blood and Marrow Transplantation. Biol
Blood Marrow Transplant. 2018;24(10):1961-1964. doi:10.1016/j.bbmt.2018.06.025
Sullivan KM, Shah A, Sarantopoulos S, Furst DE. Review: Hematopoietic Stem Cell Transplantation for Scleroderma: Effective Immunomodulatory Therapy for Patients With Pulmonary Involvement. Arthritis Rheumatol.
2016;68(10):2361-2371. doi:10.1002/art.39748
Panopoulos, Stylianos T et al. “Outcomes of conventionally-treated systemic sclerosis patients eligible for autologous haematopoietic stem cell transplantation.” Clinical and experimental rheumatology vol. 39 Suppl 131,4
(2021): 29-33. doi:10.55563/clinexprheumatol/dhn3mb
59. USE OF SPINAL STIMULATORS FOR
REFRACTORY RAYNAUD’S IN
CONNECTIVE TISSUE DISEASE
• Planned study in collaboration with pain management at the University of Chicago
• Planned for patient's intolerant to, or refractory to drugs such as
sildenafil/amlodipine
60. USE OF SPINAL STIMULATORS FOR
REFRACTORY RAYNAUD’S IN
CONNECTIVE TISSUE DISEASE
Wu M, Linderoth B, Foreman RD. Putative mechanisms behind effects of spinal cord stimulation on vascular diseases: a review of experimental studies. Auton
Neurosci. 2008;138(1-2):9-23. doi:10.1016/j.autneu.2007.11.001
Fiks V. Spinal cord stimulation can treat Peripheral Vascular Disease. www.apmconline.org. Accessed February 17, 2024.
https://www.apmconline.org/blog/spinal-cord-stimulation-can-treat-peripheral-vascular-disease
61. CAR T THERAPY
• University of Chicago will be a site in partnership with Cabaletta Bio Inc for
scleroderma, myositis, lupus
Klebanoff, C., Yamamoto, T. & Restifo, N. Treatment of aggressive lymphomas with anti-CD19 CAR T cells. Nat Rev Clin Oncol 11, 685–686 (2014).
https://doi.org/10.1038/nrclinonc.2014.190
62. CAR T THERAPY
• CAR T therapy may be superior to antibody targeted therapies at CD19 (B cells)
given ability of CAR T cells to move throughout body
• Skin involvement, and ILD will be main manifestations evaluated, along with cardiac
and/or renal involvement
• Significant pulmonary artery hypertension is a major exclusion criteria
63.
64. CAR T THERAPY
• Four patients with scleroderma described
• All 4 were able to discontinue other immunosuppression
Müller, Fabian et al. “CD19 CAR T-Cell Therapy in Autoimmune Disease - A Case Series with Follow-up.” The New
England journal of medicine vol. 390,8 (2024): 687-700. doi:10.1056/NEJMoa2308917
65. CONQUEST
• University of Chicago will be a site in partnership with the Scleroderma Research
Foundation for COUNQUEST
• Multi arm study, 2 arms at UChicago, placebo controlled
• Evaluating ILD
• Background mycophenolate allowed
66. CONQUEST
• One arm will include Amlitelimab, a biologic that targets OX40L which is involved in
cytokine release and T-cell activation
• Another arm will include BI 1015550, an oral PDE4B inhibitor, which helps
downregulate a variety of proinflammatory cytokines
• BI 1015550 is similar to a drug used for psoriatic arthritis called apremilast, though
is more selective which is thought to make it have less gastrointestinal side effects
67. MULTIDISCIPLINARY SCLERODERMA
CARE
• Scleroderma is clearly a complex disorder, effecting nearly every organ in the body
• Multiple fields are needed to work together, with rheumatology often being the
primary specialty involved
• University of Chicago will be forming a multidisciplinary scleroderma program to
help improve care for scleroderma patients, and improve access in Chicago
69. MULTIDISCIPLINARY SCLERODERMA
CARE
• Overtime will involve dedicated time in Hyde Park on Mondays with coordination of
other fields as needed
• I will also be in Crown Point, IN and River East
• Need for other fields will be coordinated at Hyde Park