Challenges in the treatment of hemophilia
Renchi Yang, MD
Institute of Hematology and Blood Diseases Hospital, Chinese Academy
of Medical Sciences
Healthcare Delivery System
• Developed countries
• Emerging countries
• Developing countries
• Undeveloped countries
Challenges
• Complications: inhibitor development, viral infections (HIV, Hepatitis, etc) ;hemarthropathy/pseudotumor
• Aging issue
• Affordable?
• Available?
• Compliance
Adherence rate to secondary Adherence rate to secondary prophylaxis among patients with prophylaxis among patients with
severe hemophilia Asevere hemophilia A
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 %
Obstacles against introduction ofObstacles against introduction ofthe prophylaxis by agethe prophylaxis by age
Ono O et al., Haemophilia 2009;15:1032–8yrs of age
Total score
Difficulty in venous access
Risk for appearance ofinhibitors
Mental stress on thepatient
Unwillingness of familymembers
Feeling no necessity
Inadequate system forguidance
Problems with drug safety
Poor adherence toreplacement therapy
Others
250
200
150
100
50
0< 3 3-5 6-12 13-
Hemarthropathy in different age goups
Zhang L, et al. Hemophilia 2003,9(6):696-702
Pseudotumor
BeijingBeijing
TianjinTianjin
HefeiHefei
JinanJinan
GuangzhouGuangzhou
Hong KongHong Kong
ShanghaiShanghai
CHINACHINAArea: 9 600 000 KmArea: 9 600 000 Km22
Population: 1.3 billionPopulation: 1.3 billion
● 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
Current Status of treatment
Ø Low supply of plasma derived concentrates
Ø High cost of concentrates
Ø Low coverage and reimbursement by Insurance
“Planned care is more effective than
random care”
WFH
HTCCNC Secretary: Renchi YangVice secretary: Xuefeng Wang
Tianjin Institute of Hematology
BeijingPUMC Hospital
Shanghai Ruijin Hospital
Guangzhou Nanfang Hospital
HefeiAnhui Provincial Hospital
JinanShandong Blood center
NationalHemophiliaRegistryCenter
CoagulationTestingTraining Center
HemophiliaNurseTrainingCenter
PhysiotherapistTrainingCenter
Chinese Society of Hematology
Thrombosis and Hemostasis Committee
Regional Hemophilia Network
Beijing:Henan, Shanxi, GansuQinghai, Ningxia, Xinjiang
Tianjin:Hebei, Shaanxi,Inner MongoliaLiaoning, Jilin, Helongjiang
Guangzhou :Guangxi, HainanYunnan, Guizhou
Shanghai :Jiangsu, Zhejiang,FujianSichuan, Chongqing
Anhui :Jiangxi, HunanHubei
Shandong :Tibet
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
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
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.)
Electronic patient registry
2010-Q42010-Q4 :各中心:各中心电脑和打印机到位电脑和打印机到位
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”介绍国家血友医大讲堂”介绍国家血友病登记系统病登记系统
2010-1-122010-1-12 :百特与卫生部:百特与卫生部签署捐赠协议签署捐赠协议
2010-4-13 , News conference2010-7-6, Provincial center workshop
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 ( 32.1% ) 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
Key players & contributers
l Beijingl Henanl Xinjiang l Xi’anl Ningxial Lanzhoul Qinghai
l Tianjin l Ha’erbinl Shanxil Changchunl Dalianl Hebeil Shenyang
l Guangzhoul Guiyangl Shenzhenl Hainanl Kunmingl Guangxi
l Shanghail Fujianl Wenzhoul HangzhouX2l Suzhoul Chongqingl ChengduX2l Yangzhou
l Hefeil Changshal WuhanX2l Jiangxi
l Shandong
l Beijingl Henanl Xinjiang l Xi’anl Ningxial Lanzhoul Qinghai
l Tianjin l Ha’erbinl Shanxil Changchunl Dalianl Hebeil Shenyang
l Guangzhoul Guiyangl Shenzhenl Hainanl Kunmingl Guangxi
l Shanghail Fujianl Wenzhoul HangzhouX2l Suzhoul Chongqingl ChengduX2l Yangzhou
l Hefeil Changshal WuhanX2l 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
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
THANKSTHANKS!!
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