Hemophilia A Clinical Trial
Official title:
Evaluate Efficacy and Safety of ADVATE in the Standard Prophylaxis Treatment of Severe or Moderately Severe Hemophilia A
Based on the successful pioneer work of Dr Nilsson and her colleagues in Sweden that started
in the late 1950's, prophylaxis is recommend as the standard of care for boys with severe
haemophilia by WHO and World Federation of Hemophilia (WFH). The efficacy and safety of
prophylaxis in preventing bleeds and arthropathy in patients with hemophilia has been
confirmed in well-designed clinical studies.To keep the factor level above 1%, the standard
dosage for patients with severe hemophilia A is 20-40 Units /kg/infusion (average 30 Units
/kg) every other day or three times a week. This dosage has a very high consumption of
factor, up to 5000-6000 international unit(IU)/kg/year. The high consumption of factor and
cost present a major barrier to use the standard prophylaxis in many countries particularly
in the developing world.
But after the Recombinant Human Coagulation Factor VIII for injection (ADVATE) Patient
Assistance Program(Golden Key) was launched in 24 Apr 2014 in Nanjing China, the
affordability of patients was solved and many patients will get more chance to receive
standard prophylaxis.
This study is designed to evaluate the Annual Bleeding rate (ABR), joint health outcomes and
QoL outcomes in subjects using standard prophylaxis under the conditions of routine
practice.
Hemophilia A is an X-linked recessive, congenital bleeding disorder caused by deficient or
defective coagulation factor VIII (FVIII). The absence or severe reduction of FVIII leads to
'spontaneous' bleeding episodes (occurring primarily in joints, muscles, and less commonly,
in soft tissues) and to excessive bleeding following trauma or injury. Estimations based on
the WFH's annual global surveys indicate that the number of people with hemophilia in the
world is approximately 400,000. [1] China accounts for a quarter of about 100,000.
Currently, based on the successful pioneer work of Dr Nilsson and her colleagues in Sweden
that started in the late 1950's, prophylaxis is recommend as the standard of care for boys
with severe haemophilia by WHO and WFH. [2] The efficacy and safety of prophylaxis in
preventing bleeds and arthropathy in patients with hemophilia has been confirmed in
well-designed clinical studies.[3,4,5]To keep the factor level above 1%, the standard dosage
for patients with severe hemophilia A is 20-40 Units /kg/infusion (average 30 Units /kg) [6]
every other day or three times a week. This dosage has a very high consumption of factor, up
to 5000-6000 international unit(IU)/kg/year. [7] The high consumption of factor and cost
present a major barrier to use the standard prophylaxis in many countries particularly in
the developing world. [8] Many families are looking forward to standard prophylaxis to
reducing bleeding episodes, stop or slow the deterioration of joint disease in their sons
with severe hemophilia and thus improving their quality of life (QoL). But in China the
majority of boys with severe hemophilia A cannot afford the high costs of standard
prophylaxis .They can only pay for on-demand treatment or low-dose prophylaxis. But after
the Advate Patient Assistance Program(Golden Key) was launched in 24 Apr 2014 in Nanjing
China, the affordability of patients was solved and many patients will get more chance to
receive standard prophylaxis.
This study is designed to evaluate the Annual Bleeding rate (ABR), joint health outcomes and
QoL outcomes in subjects using standard prophylaxis under the conditions of routine
practice.
;
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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