Significant reduction in hemarthrosis in boys with severe hemophilia A: The China hemophilia individualized low-dose secondary prophylaxis study.

China health care hemarthrosis hemophilia A outcome assessment prophylaxis studies ultrasonography

Journal

Research and practice in thrombosis and haemostasis
ISSN: 2475-0379
Titre abrégé: Res Pract Thromb Haemost
Pays: United States
ID NLM: 101703775

Informations de publication

Date de publication:
Aug 2021
Historique:
received: 30 12 2020
revised: 23 04 2021
accepted: 29 04 2021
entrez: 27 9 2021
pubmed: 28 9 2021
medline: 28 9 2021
Statut: epublish

Résumé

In countries with restricted access to clotting factor concentrates, early implementation of low-dose prophylaxis is recommended over episodic treatment. 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. 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. 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%, 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.

Identifiants

DOI: 10.1002/rth2.12552 PMID: 34568725 PMC: PMC8449287
pubmed: 34568725
doi: 10.1002/rth2.12552
pii: S2475-0379(22)01436-4
pmc: PMC8449287
doi:

Types de publication

Journal Article

Langues

eng

Pagination

e12552

Informations de copyright

© 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).

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Auteurs

Runhui Wu (R)

Hemophilia Comprehensive Care Center Hematology Department Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.
ORCID: 0000-0003-4030-209X

Xiaojing 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.

Wanru Yao (W)

Hemophilia Comprehensive Care Center Hematology Department Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.

Qing 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.

Min 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.

Ningning Zhang (N)

Hemophilia Comprehensive Care Center, Radiology Center Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.

Sheng 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.

Zhenping Chen (Z)

Hemophilia Comprehensive Care Center Hematology Department Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.
ORCID: 0000-0002-5273-145X

Yan Wang (Y)

Hemophilia Comprehensive Care Center, Rehabilitation Department Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.

Yangying 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.

Ling Tang (L)

Hemophilia Comprehensive Care Center Hematology Department Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.

Yingzi Zhen (Y)

Hemophilia Comprehensive Care Center Hematology Department Beijing Children's Hospital Capital Medical University National Center for Children's Health Beijing China.

Audrey Abad (A)

Child Health Evaluative Sciences, Research Institute The Hospital for Sick Children Toronto ON Canada.
ORCID: 0000-0001-8396-3168

Andrea S 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.

Pamela Hilliard (P)

Child Health Evaluative Sciences, Research Institute The Hospital for Sick Children Toronto ON Canada.
ORCID: 0000-0002-3123-3400

Danial M Ignas (DM)

Child Health Evaluative Sciences, Research Institute The Hospital for Sick Children Toronto ON Canada.
ORCID: 0000-0002-3036-9541

Prasad Mathew (P)

Division of Pediatric Hematology/Oncology Presbyterian Health Services Hospital Albuquerque NM USA.

Derek Stephens (D)

The Hospital for Sick Children Research Institute University of Toronto Toronto ON Canada.

Victor S Blanchette (VS)

Division of Hematology/Oncology The Hospital for Sick Children and Department of Pediatrics University of Toronto Toronto ON Canada.
ORCID: 0000-0003-3341-5010

Koon-Hung 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.

Classifications MeSH