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Tytuł:
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Significant reduction in hemarthrosis in boys with severe hemophilia A: The China hemophilia individualized low-dose secondary prophylaxis study.
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Autorzy:
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Wu R; Hemophilia Comprehensive Care Center Hematology Department Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.
Li X; Hemophilia Diagnosis and Treatment Center Department of Hematology and Oncology Chengdu Women's and Children's Central Hospital School of Medicine University of Electronic Science and Technology of China Chengdu China.; Hemophilia Treatment Center Department of Pediatrics New Century Women's and Children's Hospital Chengdu China.
Yao W; Hemophilia Comprehensive Care Center Hematology Department Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.
Zhang Q; Hemophilia Diagnosis and Treatment Center Department of Hematology and Oncology Chengdu Women's and Children's Central Hospital School of Medicine University of Electronic Science and Technology of China Chengdu China.
Zhou M; Hemophilia Diagnosis and Treatment Center Department of Hematology and Oncology Chengdu Women's and Children's Central Hospital School of Medicine University of Electronic Science and Technology of China Chengdu China.
Zhang N; Hemophilia Comprehensive Care Center, Radiology Center Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.
Yang S; Hemophilia and Diagnosis Treatment Center Department of Ultrasound Chengdu Women's and Children's Central Hospital School of Medicine University of Electronic Science and Technology of China Chengdu China.
Chen Z; Hemophilia Comprehensive Care Center Hematology Department Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.
Wang Y; Hemophilia Comprehensive Care Center, Rehabilitation Department Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.
Kuang Y; Hemophilia Diagnosis and Treatment Center Department of Radiology Chengdu Women's and Children's Central Hospital School of Medicine University of Electronic Science and Technology of China Chengdu China.
Tang L; Hemophilia Comprehensive Care Center Hematology Department Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.
Zhen Y; Hemophilia Comprehensive Care Center Hematology Department Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.
Abad A; Child Health Evaluative Sciences, Research Institute The Hospital for Sick Children Toronto ON Canada.
Doria AS; Department of Diagnostic Imaging Medical Imaging Department The Hospital for Sick Children University of Toronto Toronto Canada.; The Hospital for Sick Children Research Institute University of Toronto Toronto ON Canada.
Hilliard P; Child Health Evaluative Sciences, Research Institute The Hospital for Sick Children Toronto ON Canada.
Ignas DM; Child Health Evaluative Sciences, Research Institute The Hospital for Sick Children Toronto ON Canada.
Mathew P; Division of Pediatric Hematology/Oncology Presbyterian Health Services Hospital Albuquerque NM USA.
Stephens D; The Hospital for Sick Children Research Institute University of Toronto Toronto ON Canada.
Blanchette VS; Division of Hematology/Oncology The Hospital for Sick Children and Department of Pediatrics University of Toronto Toronto ON Canada.
Luke KH; Department of Pediatrics Laboratory Medicine and Pathology Division of Hematology and Oncology University of Ottawa Children Hospital of Eastern Ontario Ottawa ON Canada.
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Źródło:
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Research and practice in thrombosis and haemostasis [Res Pract Thromb Haemost] 2021 Sep 18; Vol. 5 (6), pp. e12552. Date of Electronic Publication: 2021 Sep 18 (Print Publication: 2021).
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Typ publikacji:
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Journal Article
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Język:
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English
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Imprint Name(s):
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Publication: 2023- : [New York] : Elsevier
Original Publication: Hoboken, NJ : John Wiley & Sons, Inc., [2017]-
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Contributed Indexing:
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Keywords: China; health care; hemarthrosis; hemophilia A; outcome assessment; prophylaxis studies; ultrasonography
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Entry Date(s):
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Date Created: 20210927 Latest Revision: 20240403
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Update Code:
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20240403
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PubMed Central ID:
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PMC8449287
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DOI:
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10.1002/rth2.12552
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PMID:
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34568725
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Introduction: In countries with restricted access to clotting factor concentrates, early implementation of low-dose prophylaxis is recommended over episodic treatment.
Objective: The objective of this 1-year prospective secondary prophylaxis study was to evaluate the efficacy of a dose/frequency escalating protocol in young boys with hemophilia A in China.
Methods: Boys were started on a low-dose protocol (minimum 10-15 IU/kg of factor VIII [FVIII] twice weekly). Escalation was based on index joint bleeding, swelling/persistent joint swelling, and serial ultrasound (gray scale and color Doppler) examinations of index joints.
Results: Thirty-three boys, median age 4.8 years (interquartile range, 3.8-6.1) were enrolled in a 3-month observation period that preceded a 1-year prophylaxis phase. A significant reduction in total bleeding events (43.0%, P = .001), index joint bleeds (53.2%, P = .002), and target index joint bleeds (70.0%, P = 0.02) was observed during the prophylaxis phase. During the prophylaxis period, 40% of target joints resolved. The percentage of boys with zero index joint bleeds increased significantly ( P = .004) from 51.5% during the observation phase to 81.8% in last quarter of the prophylaxis phase (months 10-12). There was no progression of arthropathy based on physical examination (Hemophilia Joint Health Score), X-ray, and ultrasound obtained at entry into the prophylaxis phase and at study exit. The median FVIII consumption over the prophylaxis phase was 1786 IU/kg/y.
Conclusion: A low-dose, individualized prophylaxis protocol, guided by individual bleeding profiles and serial assessment of joint status, enables escalation of treatment intensity in boys with severe hemophilia A, leading to a significant reduction in bleeding events and reduction in target joint bleeding.
(© 2021 The Authors. Research and Practice in Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis (ISTH).)
