1. Challenges in the
treatment of hemophilia
Renchi Yang, MD
Institute of Hematology and Blood
Diseases Hospital, Chinese Academy
of Medical Sciences

2. Healthcare Delivery System
• Developed countries
• Emerging countries
• Developing countries
• Undeveloped countries

3. Challenges
• Complications: inhibitor development, viral
infections (HIV, Hepatitis, etc) ；
hemarthropathy/pseudotumor
• Aging issue
• Affordable?
• Available?
• Compliance

4. Adherence rate toAdherence rate to secondsecondaryary prophylaxisprophylaxis
among pamong patienatientsts with severe hemowith severe hemo philiaphilia AA
Ono O et al., Haemophilia 2009;15:1032–8
100 ％
90 ％
80 ％
70 ％
60 ％
50 ％
40 ％
30 ％
20 ％
10 ％
0 ％ 3-5 6-12 13-18 19-29 30-39 40-49 50-
yrs of age
＞＝ 50 ％ 75-51 ％ 90-76 ％ ＞ 90 ％

5. Obstacles against introduction ofObstacles against introduction of
thethe prophylaxis by ageprophylaxis by age
Ono O et al., Haemophilia 2009;15:1032–8yrs of age
Total score
Difficulty in venous access
Risk for appearance of
inhibitors
Mental stress on the
patient
Unwillingness of family
members
Feeling no necessity
Inadequate system for
guidance
Problems with drug safety
Poor adherence to
replacement therapy
Others
250
200
150
100
50
0
＜ 3 3-5 6-12 13-

6. Hemarthropathy in different age goups
Zhang L, et al. Hemophilia 2003,9(6):696-702

7. Pseudotumor

8. BeijingBeijing
TianjinTianjin
HefeiHefei
JinanJinan
GuangzhouGuangzhou
Hong KongHong Kong
ShanghaiShanghai
CHINACHINA
Area: 9 600 000 KmArea: 9 600 000 Km22
Population: 1.3 billionPopulation: 1.3 billion

9. ● 6 initial network
● 17 new centres
Haemophilia care situation
• Low diagnosis rate
• Lack of specialised hospitals –clinical
and lab expertise
• Complex medical insurance system
• Access and availbility of treatment
• Lack of awareness

10. Current Status of treatment
Ø Low supply of plasma derived
concentrates
Ø High cost of concentrates
Ø Low coverage and reimbursement
by Insurance

11. “Planned care
is more effective than
random care”

12. WFH
HTCCNC
Secretary: Renchi Yang
Vice secretary: Xuefeng Wang
Tianjin
Institute of
Hematology
Beijing
PUMC
Hospital
Shanghai
Ruijin
Hospital
Guangzhou
Nanfang
Hospital
Hefei
Anhui
Provincial
Hospital
Jinan
Shandong
Blood center
National
Hemophilia
Registry
Center
Coagulation
Testing
Training
Center
Hemophilia
Nurse
Training
Center
Physiotherapist
Training
Center
Chinese Society of Hematology
Thrombosis and Hemostasis Committee

13. Regional Hemophilia Network
Beijing ：
Henan, Shanxi, Gansu
Qinghai, Ningxia,
Xinjiang
Tianjin ：
Hebei, Shaanxi,
Inner Mongolia
Liaoning, Jilin,
Helongjiang
Guangzhou ：
Guangxi, Hainan
Yunnan, Guizhou
Shanghai ：
Jiangsu, Zhejiang,
Fujian
Sichuan, Chongqing
Anhui ：
Jiangxi, Hunan
Hubei
Shandong ：
Tibet

14. HTCCNC meeting
• 1st HTCCNC meeting: Jinan, 2004
• 2nd HTCCNC meeting: Shanghai, 2005
• 3rd HTCCNC meeting: Hefei, 2006
• 4th HTCCNC meeting: Beijing, 2007
• 5th HTCCNC meeting: Tianjin, 2008
• 6th HTCCNC meeting: Guangzhou, 2009
• 7th HTCCNC meeting: Jinan, 2010
• 8th HTCCNC meeting: Wuhan, 2012

15. Evolution of Product Availability
– Before 1995 ： Cryoprecipitates, FFP,
plasma derived concentrates (non virus-
inactivated)
– 1995 ： Domestic produced plasma
derived concentrates (virus-inactivated)
– 2002 ： rhFVIIa （ NovoSeven ）
– 2007 ： rhFVIII （ Kogenate FS® ）
– 2013: Adavate, Xyntha, Benefix

16. Evolution of Medical Insurance
– Before 2001 ： No national medical insurance
program
– 2002 ： Some hemophilia patients covered by
medical insurance
– 2003 ： FVIII concentrates / PCC covered by medical
insurance in some cities (Tianjin, Beijing, Shanghai,
etc.)
– 2005 ： FVIII concentrates covered by national
medical insurance all over the country
– 2007 ： outpatient covered as inpatient
– 2008 ： rhFVIII concentrates covered by medical
insurance in some cities (Guangzhou, Tianjin, etc.)

17. Electronic patient registry

18. 2010-Q42010-Q4 ：各中：各中
心电脑和打印机心电脑和打印机
到位到位
2010-3-72010-3-7 ：血友病病例：血友病病例
信息报送培训会信息报送培训会
2010-4-162010-4-16 ：人民网“名：人民网“名
医大讲堂”介绍国家血医大讲堂”介绍国家血
友病登记系统友病登记系统
2010-1-122010-1-12 ：百特与卫生：百特与卫生
部签署捐赠协议部签署捐赠协议
2010-4-13 ， News conference
2010-7-6, Provincial center
workshop

19. Inhibitor rate in China
• 1435 hemophilia （ 16/3/2007-5/6/2008 ）
： 1108 severe ， 249 moderate ， 78
mild. 1363 no relation.
• 56/1435 （ 3.9% ） inhibitor ， 18/56 （ 3
2.1% ） high titer （≥ 5BU/ml ） .
• Severe ： 48/1108 （ 4.3% ）；
Moderate ： 6/249 （ 2.4% ）； Mild ：
2/78 （ 2.5% ）。
Wang XF, et al. Haemophilia 2010, 16(4):632-9

20. Key players & contributers
l Beijing
l Henan
l Xinjiang
l Xi’an
l Ningxia
l Lanzhou
l Qinghai
l Tianjin
l Ha’erbin
l Shanxi
l Changchun
l Dalian
l Hebei
l Shenyang
l Guangzhou
l Guiyang
l Shenzhen
l Hainan
l Kunming
l Guangxi
l Shanghai
l Fujian
l Wenzhou
l HangzhouX2
l Suzhou
l Chongqing
l ChengduX2
l Yangzhou
l Hefei
l Changsha
l WuhanX2
l Jiangxi
l Shandong
l Beijing
l Henan
l Xinjiang
l Xi’an
l Ningxia
l Lanzhou
l Qinghai
l Tianjin
l Ha’erbin
l Shanxi
l Changchun
l Dalian
l Hebei
l Shenyang
l Guangzhou
l Guiyang
l Shenzhen
l Hainan
l Kunming
l Guangxi
l Shanghai
l Fujian
l Wenzhou
l HangzhouX2
l Suzhou
l Chongqing
l ChengduX2
l Yangzhou
l Hefei
l Changsha
l WuhanX2
l Jiangxi
l Shandong
Organizations
• National Haemophilia Study Group since 1985
• Haemophilia Home of China established in 2000
• Haemophilia Treatment Centre
• Collaborative Network of China established in 2004
Development support
• WFH since 2001
• NNHF fellowships & projects since 2006
• GAP programme started in 2009
• Grants by pharmaceutical companies

21. Acknowledgement
• Members of the HTCCNC: Dr. Xinsheng Zhang, Dr. Jing Sun,
Dr. Xuefeng Wang, Dr. Jingsheng Wu, Dr. Runhui Wu, Dr.
Renchi Yang and Dr. Yongqiang Zhao
• Consultants: Prof. Man-Chiu Poon, Calgary, CANADA, Dr. K.H.
Luke, Ottawa, CANADA, Prof. Changgeng Ruan and Prof.
Hongli Wang
• Hemophilia Patients Union of China
• World Federation of Hemophilia, Novo Nordisk Hemophilia
Foundation
• Baxter, Bayer, Novo Nordisk, Pfizer
• Ministry of Health, PRC

22. THANKSTHANKS ！！