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！！