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

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

doi:10.1002/rth2.12552

Research and Practice in Thrombosis and Haemostasis (Aug 2021)

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

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

Runhui Wu: Hemophilia Comprehensive Care Center Hematology Department Beijing Children’s HospitalCapital Medical UniversityNational Center for Children’s Health Beijing China
Xiaojing Li: Hemophilia Diagnosis and Treatment Center Department of Hematology and Oncology Chengdu Women’s and Children’s Central HospitalSchool of MedicineUniversity of Electronic Science and Technology of China Chengdu China
Wanru Yao: Hemophilia Comprehensive Care Center Hematology Department Beijing Children’s HospitalCapital Medical UniversityNational Center for Children’s Health Beijing China
Qing Zhang: Hemophilia Diagnosis and Treatment Center Department of Hematology and Oncology Chengdu Women’s and Children’s Central HospitalSchool of MedicineUniversity of Electronic Science and Technology of China Chengdu China
Min Zhou: Hemophilia Diagnosis and Treatment Center Department of Hematology and Oncology Chengdu Women’s and Children’s Central HospitalSchool of MedicineUniversity of Electronic Science and Technology of China Chengdu China
Ningning Zhang: Hemophilia Comprehensive Care Center, Radiology Center Beijing Children’s HospitalCapital Medical UniversityNational Center for Children’s Health Beijing China
Sheng Yang: Hemophilia and Diagnosis Treatment Center Department of Ultrasound Chengdu Women’s and Children’s Central HospitalSchool of MedicineUniversity of Electronic Science and Technology of China Chengdu China
Zhenping Chen: Hemophilia Comprehensive Care Center Hematology Department Beijing Children’s HospitalCapital Medical UniversityNational Center for Children’s Health Beijing China
Yan Wang: Hemophilia Comprehensive Care Center, Rehabilitation Department Beijing Children’s HospitalCapital Medical UniversityNational Center for Children’s Health Beijing China
Yangying Kuang: Hemophilia Diagnosis and Treatment Center Department of Radiology Chengdu Women’s and Children’s Central HospitalSchool of MedicineUniversity of Electronic Science and Technology of China Chengdu China
Ling Tang: Hemophilia Comprehensive Care Center Hematology Department Beijing Children’s HospitalCapital Medical UniversityNational Center for Children’s Health Beijing China
Yingzi Zhen: Hemophilia Comprehensive Care Center Hematology Department Beijing Children’s HospitalCapital Medical UniversityNational Center for Children’s Health Beijing China
Audrey Abad: Child Health Evaluative Sciences, Research InstituteThe Hospital for Sick Children Toronto ON Canada
Andrea S. Doria: Department of Diagnostic Imaging Medical Imaging Department The Hospital for Sick ChildrenUniversity of Toronto Toronto Canada
Pamela Hilliard: Child Health Evaluative Sciences, Research InstituteThe Hospital for Sick Children Toronto ON Canada
Danial M. Ignas: Child Health Evaluative Sciences, Research InstituteThe Hospital for Sick Children Toronto ON Canada
Prasad Mathew: Division of Pediatric Hematology/Oncology Presbyterian Health Services Hospital Albuquerque NM USA
Derek Stephens: The Hospital for Sick Children Research InstituteUniversity of Toronto Toronto ON Canada
Victor S. Blanchette: Division of Hematology/Oncology The Hospital for Sick Children and Department of Pediatrics University of Toronto Toronto ON Canada
Koon‐Hung Luke: Department of Pediatrics Laboratory Medicine and Pathology Division of Hematology and Oncology University of OttawaChildren Hospital of Eastern Ontario Ottawa ON Canada

DOI: https://doi.org/10.1002/rth2.12552
Journal volume & issue: Vol. 5, no. 6
pp. n/a – n/a

Abstract

Read online

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%, 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.

Published in Research and Practice in Thrombosis and Haemostasis

ISSN: 2475-0379 (Online)
Publisher: Elsevier
Country of publisher: United States
LCC subjects: Medicine: Internal medicine: Specialties of internal medicine: Diseases of the blood and blood-forming organs
Website: https://www.sciencedirect.com/journal/research-and-practice-in-thrombosis-and-haemostasis

About the journal

Abstract

Keywords