A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children (CHIPS)
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|ClinicalTrials.gov Identifier: NCT02999308|
Recruitment Status : Unknown
Verified December 2016 by Runhui WU, Beijing Children's Hospital.
Recruitment status was: Recruiting
First Posted : December 21, 2016
Last Update Posted : December 21, 2016
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.
|Condition or disease||Intervention/treatment||Phase|
|Haemophilia||Other: Observe the patient's condition and then change the regimen||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Hematology Oncology Center|
|Study Start Date :||August 2016|
|Estimated Primary Completion Date :||September 2017|
|Estimated Study Completion Date :||December 2017|
|Experimental: single arm||
Other: Observe the patient's condition and then change the regimen
The patients will be assessed every 3 months and the treatment regimen will be modified by the result of assessment.
- Change of Ultrasound HEAD-US score of index joints [ Time Frame: baseline,3 months, 6 months,9 months and 12 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02999308
|Contact: Wu Runhui, MD, PhDfirstname.lastname@example.org|
|Beijing Children's Hospital, Capital Medical University||Recruiting|
|Beijing, Beijing, China, 100054|
|Contact: Wu Runhui, MD, PhD 0086-010-59617621 email@example.com|
|Principal Investigator:||Wu Runhui, MD, PhD||Beijing Children's Hospital|