Long-term joint outcomes of regular low-dose prophylaxis in Chinese children with severe haemophilia A
Yuefang Wu
Department of Pediatrics, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China
Department of Pediatrics, Beijing Shijitan Hospital, Capital Medical University, Beijing, China
Search for more papers by this authorJingjing Lu
Department of Radiology, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China
Search for more papers by this authorYin Zhou
Department of Pediatrics, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China
Search for more papers by this authorKuixing Li
Department of Hematology, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences & Peking Union Medical College, Beijing, China
Search for more papers by this authorYing Liu
Department of Physical Medical Rehabilitation, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China
Search for more papers by this authorShufen Liu
Department of Physical Medical Rehabilitation, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China
Search for more papers by this authorZhuo Li
Department of Pediatrics, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China
Search for more papers by this authorYongqiang zhao
Department of Hematology, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences & Peking Union Medical College, Beijing, China
Search for more papers by this authorMan-Chiu Poon
Department of Medicine, Pediatrics and Oncology, University of Calgary Cumming School of Medicine, Southern Alberta Rare Blood and Bleeding Disorders Comprehensive Care Program, Foothills Hospital, Calgary, Alberta, Canada
Search for more papers by this authorCorresponding Author
Juan Xiao
Department of Pediatrics, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China
Correspondence
Juan Xiao, Department of Pediatrics, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences & Peking Union Medical College, No.1 Shuaifuyuan Wangfujing Dongcheng District, Beijing 100730, China.
Email: [email protected]
Search for more papers by this authorYuefang Wu
Department of Pediatrics, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China
Department of Pediatrics, Beijing Shijitan Hospital, Capital Medical University, Beijing, China
Search for more papers by this authorJingjing Lu
Department of Radiology, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China
Search for more papers by this authorYin Zhou
Department of Pediatrics, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China
Search for more papers by this authorKuixing Li
Department of Hematology, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences & Peking Union Medical College, Beijing, China
Search for more papers by this authorYing Liu
Department of Physical Medical Rehabilitation, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China
Search for more papers by this authorShufen Liu
Department of Physical Medical Rehabilitation, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China
Search for more papers by this authorZhuo Li
Department of Pediatrics, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China
Search for more papers by this authorYongqiang zhao
Department of Hematology, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences & Peking Union Medical College, Beijing, China
Search for more papers by this authorMan-Chiu Poon
Department of Medicine, Pediatrics and Oncology, University of Calgary Cumming School of Medicine, Southern Alberta Rare Blood and Bleeding Disorders Comprehensive Care Program, Foothills Hospital, Calgary, Alberta, Canada
Search for more papers by this authorCorresponding Author
Juan Xiao
Department of Pediatrics, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences and Peking Union Medical College, Beijing, China
Correspondence
Juan Xiao, Department of Pediatrics, Peking Union Medical College Hospital, Chinese Academe of Medical Sciences & Peking Union Medical College, No.1 Shuaifuyuan Wangfujing Dongcheng District, Beijing 100730, China.
Email: [email protected]
Search for more papers by this authorAbstract
Objectives
To explore the long-term joint outcomes of low-dose prophylaxis in Chinese children with severe haemophilia A and to analyse their related factors.
Methods
We retrospectively analysed follow-up data from 21 severe haemophilia A children on regular low-dose prophylaxis for 6–10 years. We used International Prophylaxis Study Group magnetic resonance imaging score (IPSG MRI score), Hemophilia Joint Health Score (HJHS), number of target joints, and Hemophilia-Specific Quality of Life Index (Haemo-QoL) to evaluate joint outcomes. Factors associated with these outcomes were evaluated by statistical analysis.
Results
(1) The children were 1.75 to 17 years age at prophylaxis initiation. Median prophylactic factor VIII dose was 22.9 IU/kg per week. (2) At the end of follow-up: (a) The total IPSG MRI scores were 2–24 with 90.5% children exhibiting moderate to severe joint involvement (score 7–24); (b) The HJHS ranged 2–27, with 0–10 for 46.7% children and >10 for 53.3% children. There was a positive correlation between the MRI score and HJHS (p < .05); (c) Compared to their on-demand treatment period before prophylaxis, target joints numbers decreased, and no child needed auxiliary devices to walk; (d) Joint outcomes were positively correlated with the age at initiation of low-dose prophylaxis (p < .05) and negatively correlated with the treatment dose.
Conclusion
Long-term low-dose prophylaxis had positive effect on joint outcomes compared with on-demand treatment. However, a certain degree of joint damage remained in all children indicating the need for improving the current strategy of low-dose prophylaxis.
DISCLOSURES
The authors stated that they had no interests which might be perceived as posing a conflict or bias.
Open Research
DATA AVAILABILITY STATEMENT
All research data in this study are available and can be referenced if necessary.
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