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Pediatric Vasculitis Initiative (PedVas) A CIHR Emerging Team Grant in Rare Disease (2012)

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Canadian Organization for Rare Disorders
Canadian Organization for Rare Disorders

Rare Disease Day 2015 Conference Day 2: Susa Benseler

Health & Medicine
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Pediatric Vasculitis Initiative (PedVas) Update for CORD Rare Disease Conference, March 2015 Susa Benseler, MD PhD Chief Rheumatology, Alberta Children’s Hospital on behalf of David Cabral Dirk Foell Jinko Graham Bob Hancock Raashid Luqmani Colin Ross International childhood vasculitis investigators contributing to ARChiVe, BrainWorks & DCVAS A CIHR Emerging Team Grant in Rare Disease (2012)
Pediatric Vasculitis Initiative (PedVas) • CIHR- , Vasculitis foundation and CORD- supported opportunity to support existing clinical childhood vasculitis networks with a translational research component involving collection & analysis of biological samples and Knowledge Translation • International & multi-disciplinary collaboration between bench scientists and clinicians
9 year old girl • ER: presented with weeks of fever on and off, fatigue, congestion, swollen glands, nosebleeds and cough • Previously perfectly healthy
Laboratory test results • High levels of inflammatory markers • No evidence of infection • Positive ANCA antibodies
Chest imaging Cavitating lung lesions and nodules
Destructive disease Destroyed sinus walls and saddle nose deformity
Diagnosis: Childhood vasculitis
6 year old girl • For 2 weeks complaining of nausea, headaches, mood changes, school concentration difficulties • ? Virus infection (pediatrician) • ER: blood work and CT normal
• Perfectly health in the past medical history • No travel, no infection, no injury 6 year old girl
Laboratory test results • High levels of inflammatory markers • No evidence of brain infection or bleed • Spinal tap: inflammation/irritation
Acute worsening • Vomiting, headache, • Decreased level of conciousness, • neck stiffness • Seizure status • No fever, no other symptoms
MRI brain imaging • Wide spread inflammation • No stroke • No tumor
Elective brain biopsy Central Nervous System (CNS) vasculitis
Diagnosis: Childhood vasculitis
Questions asked by families and health care providers • What is childhood vasculitis? • What caused it? Are siblings at risk? • How to best make the diagnosis and avoid invasive tests? • Is there a cure? • Which treatment to choose? Are the treatments dangerous? • How will this affect her future?
A registry for childhood systemic vasculitis • 40 US/Canadian centers • Launched 2008 A prospective registry for CNS vasculitis • 210 centers around the word • Launched 2010 Building international networks… David Cabral and Susa Benseler
• Approach: Learn from each and every child • Web-based, password secured prospective data collections that connect physicians of many disciplines around the world • Provides treatment protocols, SOPs, review scales, outcome tools, literature updates and second opinions • Publications, advocacy, data ownership rule
Righ t Righ t
Right
116 28 85 37 8 5 4 19 36 28 33 NP-cPACNS P-cPACNS SV-cPACNS Neuronal antibody mediated IBrainD Demyelinating brain disease T-cell mediated IBrainD Granulomatous disease Secondary IBrainD to infection Secondary IBrainD to underlying systemic disease Moya Moya syndrome Childhood inflammatory brain diseases N=416, Feb 2015
116 28 85 37 8 5 4 19 36 28 33 NP-cPACNS P-cPACNS SV-cPACNS Neuronal antibody mediated IBrainD Demyelinating brain disease T-cell mediated IBrainD Granulomatous disease Secondary IBrainD to infection Secondary IBrainD to underlying systemic disease N=416, Feb 2015 Angiography-positive primary childhood CNS vasculitis N=144
116 28 85 37 8 5 4 19 36 28 33 NP-cPACNS P-cPACNS SV-cPACNS Neuronal antibody mediated IBrainD Demyelinating brain disease T-cell mediated IBrainD Granulomatous disease Secondary IBrainD to infection Secondary IBrainD to underlying systemic disease Moya Moya syndrome Angiography-negative small vessel primary childhood CNS vasculitis N=85
Questions asked by families and health care providers • What is childhood vasculitis? • What caused it? Are siblings at risk? • How to best make the diagnosis and avoid invasive tests? • Which treatment to choose? Are the treatments dangerous? • How will this affect her future? • Is there a cure?
What is vasculitis?
Vasculitis is a devastating disease
Clinical diagnosis N = 165 Cellucci, 2013
Questions asked by families and health care providers • What is childhood vasculitis? • What caused it? Are her siblings at risk? • How to best make the diagnosis and avoid invasive tests? • Which treatment to choose? Are the treatments dangerous? • How will this affect her future? • Is there a cure?
vWF: A potential biomarker • N= 102 cPACNS patients Cellucci, 2012 Rheumatology
Fresh CNS vasculitis
Longstanding disease
Canadian Neuropathology study • Neuropathologists across Canada, external experts including Harry Vinters, lead: Cynthia Hawkins • Tasks: – development and validation of a Brain Biopsy Review Tool for Childhood CNS vasculitis – Defining childhood CNS vasculitis histologically Tool development completed, RedCap version Validation: 50 biopsies (cPACNS, other IBrainD, epilepsy surgery specimens) • Consensus meeting, Manuscripts in preparation
Towards a histological diagnosis of cPACNS 30 SVcPACNS cases 10 epilepsy controls 10 inflammatory controls
Questions asked by families and health care providers • What is childhood vasculitis? • What caused it? Are her siblings at risk? • How to best make the diagnosis and avoid invasive tests? • Is there a cure? • Which treatment to choose? Are the treatments dangerous? • How will this affect her future?
Small vessel cPACNS treatment
Treatment protocol Small vessel cPACNS
http://www.sickkids.ca/Research/Brainworks/Welcome/Welcome.html …or google childhood CNS vasculitis BrainWorks
http://www.sickkids.ca/Research/Brainworks/Welcome/Welcome.html …or google childhood CNS vasculitis BrainWorks
Small vessel cPACNS outcome Hutchinson, Lancet Neurology 2010
SVcPACNS Flares Cyclophophamid Prednisone MMF Azathioprine Relapse: ON Diagnosis 6 Months 12 Months 18 Months 24 Months
Anti-TNFα Therapy in refractory SVcPACNS Bhattish, 2012, J Rheumatology
BrainWorks website
Twilt, Nature Reviews 2012
http://www.sickkids.ca/Research/Brainworks/Welcome/Welcome.html …or google childhood CNS vasculitis
Successful partnerships N=501 adult encephalitis patients N=177 < 18 years of age Titulaer, Lancet Neurology 2014
Titulaer, Lancet Neurology2014 Outcome: Relapses
• Assessing pediatric phenotype & outcomes. • Assessing, developing clinical tools (classification, severity, activity, damage) & treatment protocols to help physicians • Embedded comparative study with adults & DCVAS • Re-evaluating ‘classification’ using bioinformatics to evaluate clinical data with linked gene expression profiles. • Biomarker discovery PedVas
Current Status Site recruitment – 48 sites from 10 countries collecting clinical data – 21 sites from 7 countries with ethics for biosampling Clinical data – 381 systemic vasculitis patients – 228 primary CNS vasculitis patients (Approx 90% have follow-up data up to 12 months) Biological samples – 93 patients contributed (RNA, DNA, serum, plasma, urine) • First adult (DCVAS) samples collected in Aug ’14 • Abstracts 5, manuscripts: 4 published, 3 in preparation
Supports existing vasculitis registries to collect & analyse clinical data, biological samples & KT. PedVas
Challenges Difficulties in cIinical data versus biosamples! • ethics approval challenges for blood collection and shipping across borders • contract negotiations between universities because funding is being provided • workload (& costs) for bio-sampling and shipping • Some countries sites prohibit release of biosamples Study start-up activities taking up to 12 months per site (ethics approval, contract negotiation, laboratory set-up, training)
Site Initiation Timeline New site or Existing ARChiVe/ Brainworks site Ethics amendment or Brand new application Agreements Review -Funds Transfer Letter - Material Transfer Agreement (if required) - UBC UILO Office - Site Legal Office • Time to ethics approval: 10 months (Ranges 2 - 19 m (some pending for > 20 months) • Time to fully signed agreement: 4 months (Ranges 1 to 8 m (some pending for > 13 months) Average time: 4 months Average time: 10 months Site Study Start-up - Laboratory Set-up - Site Initiation Call/Training - Database access - Supplies mailed Recruitment! Approx 1 month Approx 1 month
Preliminary Data: Early outcomes in pediatric systemic vasculitis Morishita K, et al at ACR Boston 2014 • 79 children: 6- and 12-month outcomes • Primary findings: – Majority had severe disease with kidney failure and lung bleeds disease – Only half achieved remission by 6 months – Only two thirds achieved remission by 12 months – One in two children relapses – A significant proportion of patients have evidence of damage even by 12 months
Preliminary Data: S100 A12 Biomarkers S100A12 measured healthy people and children with vasculitis Serum S100A12 levels are highest at the time of diagnosis when vasculitis is most active. (More sensitive than ESR & CRP) Levels decline after treatment, but rarely reaches levels measured in serum from healthy individuals
Biomarker discovery EGF: epidermal growth factor Eotaxin FGF-2(basic) Fractalkine GM-CSF IFNalpha2 and IFNgamma IL-1alpha, IL-1beta, IL-1Ra IL-2,IL-3, IL-5, IL-6, IL-7, IL-8, IL-9, IL-10, IL-12, IL-13, IL-15, IL-17 IP-10 MCP-3 MDC MIP1alpha PDGF-AA/AB/BB CD40L (CD154) sIL-2Ralpha TGFalpha TNFbeta ENA-78 SDF-1alpha + beta
Inflammatory Biomarker Profiles Mineyko, 2012
Preliminary Data: gene expression profiles
Thank you…

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Pediatric Vasculitis Initiative (PedVas) A CIHR Emerging Team Grant in Rare Disease (2012)

  • 1. Pediatric Vasculitis Initiative (PedVas) Update for CORD Rare Disease Conference, March 2015 Susa Benseler, MD PhD Chief Rheumatology, Alberta Children’s Hospital on behalf of David Cabral Dirk Foell Jinko Graham Bob Hancock Raashid Luqmani Colin Ross International childhood vasculitis investigators contributing to ARChiVe, BrainWorks & DCVAS A CIHR Emerging Team Grant in Rare Disease (2012)
  • 2. Pediatric Vasculitis Initiative (PedVas) • CIHR- , Vasculitis foundation and CORD- supported opportunity to support existing clinical childhood vasculitis networks with a translational research component involving collection & analysis of biological samples and Knowledge Translation • International & multi-disciplinary collaboration between bench scientists and clinicians
  • 3.
  • 4. 9 year old girl • ER: presented with weeks of fever on and off, fatigue, congestion, swollen glands, nosebleeds and cough • Previously perfectly healthy
  • 5. Laboratory test results • High levels of inflammatory markers • No evidence of infection • Positive ANCA antibodies
  • 6. Chest imaging Cavitating lung lesions and nodules
  • 7. Destructive disease Destroyed sinus walls and saddle nose deformity
  • 9. 6 year old girl • For 2 weeks complaining of nausea, headaches, mood changes, school concentration difficulties • ? Virus infection (pediatrician) • ER: blood work and CT normal
  • 10. • Perfectly health in the past medical history • No travel, no infection, no injury 6 year old girl
  • 11. Laboratory test results • High levels of inflammatory markers • No evidence of brain infection or bleed • Spinal tap: inflammation/irritation
  • 12. Acute worsening • Vomiting, headache, • Decreased level of conciousness, • neck stiffness • Seizure status • No fever, no other symptoms
  • 13. MRI brain imaging • Wide spread inflammation • No stroke • No tumor
  • 14. Elective brain biopsy Central Nervous System (CNS) vasculitis
  • 16. Questions asked by families and health care providers • What is childhood vasculitis? • What caused it? Are siblings at risk? • How to best make the diagnosis and avoid invasive tests? • Is there a cure? • Which treatment to choose? Are the treatments dangerous? • How will this affect her future?
  • 17. A registry for childhood systemic vasculitis • 40 US/Canadian centers • Launched 2008 A prospective registry for CNS vasculitis • 210 centers around the word • Launched 2010 Building international networks… David Cabral and Susa Benseler
  • 18. • Approach: Learn from each and every child • Web-based, password secured prospective data collections that connect physicians of many disciplines around the world • Provides treatment protocols, SOPs, review scales, outcome tools, literature updates and second opinions • Publications, advocacy, data ownership rule
  • 19.
  • 20.
  • 22. Right
  • 23.
  • 24.
  • 25.
  • 26. 116 28 85 37 8 5 4 19 36 28 33 NP-cPACNS P-cPACNS SV-cPACNS Neuronal antibody mediated IBrainD Demyelinating brain disease T-cell mediated IBrainD Granulomatous disease Secondary IBrainD to infection Secondary IBrainD to underlying systemic disease Moya Moya syndrome Childhood inflammatory brain diseases N=416, Feb 2015
  • 27. 116 28 85 37 8 5 4 19 36 28 33 NP-cPACNS P-cPACNS SV-cPACNS Neuronal antibody mediated IBrainD Demyelinating brain disease T-cell mediated IBrainD Granulomatous disease Secondary IBrainD to infection Secondary IBrainD to underlying systemic disease N=416, Feb 2015 Angiography-positive primary childhood CNS vasculitis N=144
  • 28. 116 28 85 37 8 5 4 19 36 28 33 NP-cPACNS P-cPACNS SV-cPACNS Neuronal antibody mediated IBrainD Demyelinating brain disease T-cell mediated IBrainD Granulomatous disease Secondary IBrainD to infection Secondary IBrainD to underlying systemic disease Moya Moya syndrome Angiography-negative small vessel primary childhood CNS vasculitis N=85
  • 29.
  • 30. Questions asked by families and health care providers • What is childhood vasculitis? • What caused it? Are siblings at risk? • How to best make the diagnosis and avoid invasive tests? • Which treatment to choose? Are the treatments dangerous? • How will this affect her future? • Is there a cure?
  • 33.
  • 34. Clinical diagnosis N = 165 Cellucci, 2013
  • 35. Questions asked by families and health care providers • What is childhood vasculitis? • What caused it? Are her siblings at risk? • How to best make the diagnosis and avoid invasive tests? • Which treatment to choose? Are the treatments dangerous? • How will this affect her future? • Is there a cure?
  • 36.
  • 37. vWF: A potential biomarker • N= 102 cPACNS patients Cellucci, 2012 Rheumatology
  • 40. Canadian Neuropathology study • Neuropathologists across Canada, external experts including Harry Vinters, lead: Cynthia Hawkins • Tasks: – development and validation of a Brain Biopsy Review Tool for Childhood CNS vasculitis – Defining childhood CNS vasculitis histologically Tool development completed, RedCap version Validation: 50 biopsies (cPACNS, other IBrainD, epilepsy surgery specimens) • Consensus meeting, Manuscripts in preparation
  • 41. Towards a histological diagnosis of cPACNS 30 SVcPACNS cases 10 epilepsy controls 10 inflammatory controls
  • 42. Questions asked by families and health care providers • What is childhood vasculitis? • What caused it? Are her siblings at risk? • How to best make the diagnosis and avoid invasive tests? • Is there a cure? • Which treatment to choose? Are the treatments dangerous? • How will this affect her future?
  • 43. Small vessel cPACNS treatment
  • 47. Small vessel cPACNS outcome Hutchinson, Lancet Neurology 2010
  • 48. SVcPACNS Flares Cyclophophamid Prednisone MMF Azathioprine Relapse: ON Diagnosis 6 Months 12 Months 18 Months 24 Months
  • 49. Anti-TNFα Therapy in refractory SVcPACNS Bhattish, 2012, J Rheumatology
  • 51.
  • 52.
  • 54.
  • 55.
  • 57.
  • 58.
  • 59.
  • 60. Successful partnerships N=501 adult encephalitis patients N=177 < 18 years of age Titulaer, Lancet Neurology 2014
  • 62.
  • 63. • Assessing pediatric phenotype & outcomes. • Assessing, developing clinical tools (classification, severity, activity, damage) & treatment protocols to help physicians • Embedded comparative study with adults & DCVAS • Re-evaluating ‘classification’ using bioinformatics to evaluate clinical data with linked gene expression profiles. • Biomarker discovery PedVas
  • 64. Current Status Site recruitment – 48 sites from 10 countries collecting clinical data – 21 sites from 7 countries with ethics for biosampling Clinical data – 381 systemic vasculitis patients – 228 primary CNS vasculitis patients (Approx 90% have follow-up data up to 12 months) Biological samples – 93 patients contributed (RNA, DNA, serum, plasma, urine) • First adult (DCVAS) samples collected in Aug ’14 • Abstracts 5, manuscripts: 4 published, 3 in preparation
  • 65. Supports existing vasculitis registries to collect & analyse clinical data, biological samples & KT. PedVas
  • 66. Challenges Difficulties in cIinical data versus biosamples! • ethics approval challenges for blood collection and shipping across borders • contract negotiations between universities because funding is being provided • workload (& costs) for bio-sampling and shipping • Some countries sites prohibit release of biosamples Study start-up activities taking up to 12 months per site (ethics approval, contract negotiation, laboratory set-up, training)
  • 67. Site Initiation Timeline New site or Existing ARChiVe/ Brainworks site Ethics amendment or Brand new application Agreements Review -Funds Transfer Letter - Material Transfer Agreement (if required) - UBC UILO Office - Site Legal Office • Time to ethics approval: 10 months (Ranges 2 - 19 m (some pending for > 20 months) • Time to fully signed agreement: 4 months (Ranges 1 to 8 m (some pending for > 13 months) Average time: 4 months Average time: 10 months Site Study Start-up - Laboratory Set-up - Site Initiation Call/Training - Database access - Supplies mailed Recruitment! Approx 1 month Approx 1 month
  • 68. Preliminary Data: Early outcomes in pediatric systemic vasculitis Morishita K, et al at ACR Boston 2014 • 79 children: 6- and 12-month outcomes • Primary findings: – Majority had severe disease with kidney failure and lung bleeds disease – Only half achieved remission by 6 months – Only two thirds achieved remission by 12 months – One in two children relapses – A significant proportion of patients have evidence of damage even by 12 months
  • 69. Preliminary Data: S100 A12 Biomarkers S100A12 measured healthy people and children with vasculitis Serum S100A12 levels are highest at the time of diagnosis when vasculitis is most active. (More sensitive than ESR & CRP) Levels decline after treatment, but rarely reaches levels measured in serum from healthy individuals
  • 70. Biomarker discovery EGF: epidermal growth factor Eotaxin FGF-2(basic) Fractalkine GM-CSF IFNalpha2 and IFNgamma IL-1alpha, IL-1beta, IL-1Ra IL-2,IL-3, IL-5, IL-6, IL-7, IL-8, IL-9, IL-10, IL-12, IL-13, IL-15, IL-17 IP-10 MCP-3 MDC MIP1alpha PDGF-AA/AB/BB CD40L (CD154) sIL-2Ralpha TGFalpha TNFbeta ENA-78 SDF-1alpha + beta
  • 73.