Transcript Document

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 prophylaxis
among patients with severe hemophilia A
＞＝50％
75-51％
3-5
13-18
90-76％
＞90％
100％
90％
80％
70％
60％
50％
40％
30％
20％
10％
0％
yrs of age
6-12
19-29
30-39
40-49
50-
Ono O et al., Haemophilia 2009;15:1032–8
250
Obstacles against introduction of
the prophylaxis by age
Total score
Difficulty in venous access
200
Risk for appearance of
inhibitors
Mental stress on the
patient
150
Unwillingness of family
members
Feeling no necessity
100
Inadequate system for
guidance
Problems with drug safety
50
Poor adherence to
replacement therapy
Others
0
＜3
yrs of age
3-5
6-12
13-
Ono O et al., Haemophilia 2009;15:1032–8
Hemarthropathy in different age goups
Zhang L, et al. Hemophilia 2003,9(6):696-702
Pseudotumor
CHINA
Beijing
Area: 9 600 000 Km2
Population: 1.3 billion
Jinan
Tianjin
Hefei
Shanghai
Guangzhou
Hong Kong
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
● 6 initial network
● 17 new centres
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”
Chinese Society of Hematology
WFH
Thrombosis and Hemostasis Committee
HTCCNC
Secretary: Renchi Yang
Vice secretary: Xuefeng Wang
Tianjin
Institute of
Hematology
National
Hemophilia
Registry
Center
Shanghai
Ruijin
Hospital
Coagulation
Testing
Training
Center
Guangzhou
Nanfang
Hospital
Hemophilia
Nurse
Training
Center
Beijing
PUMC
Hospital
Hefei
Anhui
Provincial
Hospital
Physiotherapist
Training
Center
Jinan
Shandong
Blood center
Tianjin：
Regional Hemophilia Network
Hebei, Shaanxi,
Inner Mongolia
Liaoning, Jilin,
Helongjiang
Beijing：
Henan, Shanxi, Gansu
Qinghai, Ningxia,
Xinjiang
Shanghai：
Jiangsu, Zhejiang,
Fujian
Sichuan, Chongqing
Anhui：
Shandong：
Tibet
Jiangxi, Hunan
Hubei
Guangzhou：
Guangxi, Hainan
Yunnan, Guizhou
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 virusinactivated)
– 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
2010-4-13，News conference
2010-7-6, Provincial center
workshop
2010-1-12：百特与卫生部
签署捐赠协议
2010-3-7：血友病病例信 2010-4-16：人民网“名
息报送培训会
医大讲堂”介绍国家血友
病登记系统
2010-Q4：各中心
电脑和打印机到位
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
Organizations
• National Haemophilia Study Group since 1985
• Haemophilia Home of China established in 2000
• Haemophilia Treatment Centre
• Collaborative Network of China established in 2004
l
l
l
l
l
l
l
Beijing
Henan
Xinjiang
Xi’an
Ningxia
Lanzhou
Qinghai
l
l
l
l
l
l
l
Tianjin
Ha’erbin
Shanxi
Changchun
Dalian
Hebei
Shenyang
l S handong
l
l
l
l
l
l
l
l
Development support
• WFH since 2001
• NNHF fellowships & projects since 2006
• GAP programme started in 2009
• Grants by pharmaceutical companies
l
l
l
l
l
l
Guangzhou
Guiyang
Shenzhen
Hainan
Kunming
Guangxi
l
l
l
l
Shanghai
Fujian
Wenzhou
HangzhouX2
Suzhou
Chongqing
ChengduX2
Yangzhou
Hefei
Changsha
WuhanX2
Jiangxi
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
THANKS！