A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children

Brief Title

A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children

Official Title

Hematology Oncology Center

Brief Summary

      In the past, due to economic and medical resource constraints, the hemophilia comprehensive
      care in China was suboptimal. The BCH data of both retrospective and prospective studies
      reveals that for 4-6y and 6-9y patients with severe hemophilia respectively: 45% and 82% of
      the patients have suffered from joint bleeding, with mean AJBR of 4.18 and 4.95; and 24.2%
      and 33.3% of them with AJBR>10 times, led to arthropathies and made their quality of life be
      heavy affected. Now, with the development of economy and medical science in China,
      prophylaxis regimens have been initiated in more and more children with hemophilia.
      Considering the difference between prophylaxis regimens, the frequency of joint bleeding was
      reduced significantly, the quality of life of hemophilia kids improved. An assessment scoring
      system for the appropriate validation of individualized prophylaxis treatment regimens are
      urgently needed.

      Before, the most important assessed indication for hemophilia prophylaxis was the frequency
      of joint bleeding. But increasing evidences are showing that there is a discrepancy between
      real joint damage and joint bleeding frequency. The single indicator of joint bleeding
      frequency is not sufficient to evaluate the joint status of hemophilia children.

      Under the World Health Organization's ICF guidelines, the assessment scoring system for
      selecting prophylaxis for children with hemophilia should include the tools currently
      available for assessment of structure/function of the joint, patient activities and patient
      participation in hemophilia healthcare. According to ICF of WHO, the most common bleeding
      parts are elbows, knees and ankles, therefore the assessment of children with hemophilia
      should include the evaluation of the structure, the function of these 6 Index joints, the
      capacity of activities and the capacity of participation of children. These will constitute a
      comprehensive hemophilia evaluation system.

      In China, exploration of the optimal and individualized prophylaxis regimen is urgent, and
      the comprehensive evaluation system should include joint structure and function, body's
      activities and individual participation, thus may be more appropriate for the individualized
      prophylaxis for Chinese children with hemophilia.

Study Phase

Phase 4

Study Type

Interventional

Primary Outcome

Change of Ultrasound HEAD-US score of index joints

Condition

Haemophilia

Intervention

Observe the patient's condition and then change the regimen

Study Arms / Comparison Groups

 single arm

Description:

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Other

Estimated Enrollment

Start Date

August 2016

Completion Date

December 2017

Primary Completion Date

September 2017

Eligibility Criteria

        Inclusion Criteria:

          1. Severe hemophilia A (FVIII: C<2%),

          2. Age 1-7y

          3. Historical bleeding in any knee, elbow and ankle joint (s),

          4. >50 EDs irrespective of FVIII product, including human coagulate factor and
             recombinant factor VIII

          5. No inhibitor present or history/family history,

          6. On-demand or low-dose prophylaxis (the dose per kg.week less or as Step 1 prophylaxis
             regimens factor consumption)

          7. Regular clinical visit with accessible data,

          8. Informed consent will be obtained from patient legal guardians before the enrollment.

        Exclusion Criteria:

          -  1. Other bleeding disorders or systemic disorders, or don't fit for enrollment
             according to the investigator, 2. FVIII inhibitor development:>0.6 BU (confirmed by
             two separate tests), 3. Unable to follow the protocol or refuse to continue the
             participation.

Gender

All

Ages

1 Year - 7 Years

Accepts Healthy Volunteers

No

Contacts

Wu Runhui, MD, PhD, 0086-010-59617621, [email protected]

Location Countries

China

Location Countries

China

Administrative Informations

NCT ID

NCT02999308

Organization ID

CHIPS

Responsible Party

Principal Investigator

Study Sponsor

Beijing Children's Hospital

Study Sponsor

Wu Runhui, MD, PhD, Principal Investigator, Beijing Children's Hospital

Verification Date

December 2016