Hemophilia is a common hereditary coagulation disorder due to deficiency or reduce activity of clotting factor VIII or clotting factor IX. This disorder is a X- linked recessive disorder. Types: Hemophilia A- deficiency of clotting factors VIII Hemophilia B- deficiency of clotting factors IX Hemophilia C- deficiency of clotting factors XI Parahaemophilia- deficiency of clotting factor V Causes of hemophilia Hemophilia has a sex-linked recessive inheritance. In most cases Hemophilia caused by a mutation in a gene that encodes for one of the clotting factors . Since the hemophilia gene is located on the X chromosome, Hemophilia usually occurs in males, and Female is the carrier of hemophilia. Diagnosis Complete blood cell count Coagulation studies FVIII assay Normal values for FVIII assays are 50-150%. Values in hemophilia are as follows: Mild: >5% Moderate: 1-5% Severe: <1% Treatment of Hemophilia Other Types of Treatment Desmopressin (DDAVP) Antifibrinolytic Medicines Vaccinations- hepatitis A and B. Gene Therapy Gene Therapy New Drugs for Hemophilia treatment New Drugs for Hemophilia treatment Bangladesh perspectives Bangladesh would have 10800 hemophiliacs. But, initially the patients does not concern about hemophilia. Patients are usually diagnosed only after bleeding episode and sometimes the episode are causes serious consequences. Conclusion Primary diagnosis with the success of gene therapy and availability of the new bioengineered products the prospect of the hemophiliacs will be brighter in near future.

1. Md.Waliullah Wali
waliseu31@gmail.com

2. CONTENTS
2

3. 3

4. 4

5. 5
Spontaneous bleeding....

6. 6
How Pathetic !!!

7. 7
Hemophilia is that kind of disorder ,
Which may cause this !!!

8. Introduction
8
Hemophilia is a common hereditary coagulation
disorder due to deficiency or reduce activity of
clotting factor VIII or clotting factor IX.
This disorder is a X- linked recessive disorder.
Fibrin Mesh
Normal Hemophilia
Incomplete

9. Types:
9
 Hemophilia A- deficiency of clotting factors VIII
 Hemophilia B- deficiency of clotting factors IX
 Hemophilia C- deficiency of clotting factors XI
 Parahaemophilia- deficiency of clotting factor V

10. History of hemophilia
10
 Hemophilia has been called a "royal disease“.
Queen Victoria

11. 11

12.  Excessive bleeding
 Easy bruising
Signs and Symptoms of Hemophilia
12
 Sudden pain, swelling and warmth
in large joints
 Painful, prolonged headache
 Repeated vomiting
 Extreme fatigue
 Neck pain
 Double vision

13. Causes of hemophilia
13
 Hemophilia has a sex-linked recessive
inheritance.
 In most cases Hemophilia caused by a mutation
in a gene that encodes for one of the clotting
factors .
 Since the hemophilia gene is located on the X
chromosome, Hemophilia usually occurs in
males, and Female is the carrier of hemophilia.

14. Causes of hemophilia
14

15. Diagnosis
15
 Complete blood cell count
 Coagulation studies
 FVIII assay
Normal values for FVIII assays are 50-150%. Values
in hemophilia are as follows:
 Mild: >5%
 Moderate: 1-5%
 Severe: <1%

16. Treatment of Hemophilia
16
Treatment with Replacement Therapy

17. Treatment of Hemophilia
17

18. Other Types of Treatment
18
 Desmopressin (DDAVP)
 Antifibrinolytic Medicines
 Vaccinations- hepatitis A and B.

19. Gene Therapy
19

20. Gene Therapy
20

21. New Drugs for Hemophilia treatment
21
Drug Company Clinical Trial
NOVOEIGHT (Recombinant Factor
VIII)
Novo Nordisk FDA Approved October
16, 2013. Anticipated
launch after April 2015.
Recombinant FVIII, glycopegylated,
long-acting
Novo Nordisk Phase III (started
January 2012)
Human-cl rhFVIII, recombinant,
human cell line
Octapharma Phase III (started June
2011)
Drugs for Hemophilia A
In Clinical Trial

22. New Drugs for Hemophilia treatment
22
Drugs for Hemophilia B
In Clinical Trial
Drug Company Clinical Trial
Recombinant Factor IX Cangene
Corporation
Phase III (as of July
2013)
Recombinant factor IX,
glycopegylated, long-acting
Novo Nordisk Phase III
Recombinant factor IX,
albumin fusion, long-acting
CSL Behring Phase III

23. Bangladesh perspectives
23
 Bangladesh would have 10800 hemophiliacs.
 But, initially the patients does not concern about
hemophilia.
 Patients are usually diagnosed only after bleeding
episode and sometimes the episode are causes
serious consequences.
 As per report of the annual global survey 2009 by
WFH, only three hemophilia treatment centers are
existing in Bangladesh.
 However these centers are not well equipped with
diagnostic facilities and adequate trained manpower
and most of all is in capital city.

24. Conclusion
24
Primary diagnosis with the success of gene
therapy and availability of the new
bioengineered products the prospect of the
hemophiliacs will be brighter in near future.

25. 25
Thank you All