Significant Reduction in Hemarthrosis in Boys with Severe Hemophilia A: The China Hemophilia Individualized Low-dose Secondary Prophylaxis Study

Overview

Journal Res Pract Thromb Haemost

Publisher Elsevier

Date 2021 Sep 27

PMID 34568725

Citations 5

Authors

Runhui Wu

Xiaojing Li

Wanru Yao

Qing Zhang

Min Zhou

Ningning Zhang

Sheng Yang

Zhenping Chen

Yan Wang

Yangying Kuang

Ling Tang

Yingzi Zhen

Audrey Abad

Andrea S Doria

Pamela Hilliard

Danial M Ignas

Prasad Mathew

Derek Stephens

Victor S Blanchette

Koon-Hung Luke

Affiliations

Soon will be listed here.

Abstract

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%, = .001), index joint bleeds (53.2%, = .002), and target index joint bleeds (70.0%, = 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 (= .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.

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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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