Impact of Low Dose Prophylaxis (LDP) on spontaneous bleeds in Hemophilia-A: A Bicentre study from Pakistan

Abstract

Background & Objectives: Hemophilia-A is a bleeding disorder characterized by musculoskeletal bleeding. Standard dose factor replacement (SDP) is the mainstay of treatment for severe or moderately severe HA patients however it is fraught with increased cost and burden on health care system. Low dose prophylaxis (LDP) has gained popularity in low- and middle-income settings (LMIC) as being more feasible and cost effective. The aim of this study was to establish efficacy of low dose prophylaxis of clotting factor concentrates (CFCs) as primary or secondary prophylaxis strategy on the bleeding outcomes of pediatric severe HA patients.

Methodology: This bicentre prospective collaborative study from the Hemophilia Patient Welfare Society (HPWS) and the World Federation of Hemophilia (WFH) commenced at Rawalpindi & Lahore Hemophilia Treatment Centers (HTCs) in Pakistan from January 2017 to January 2023.This study assessed the annualized bleeding rates (ABR) before and after LDP in pediatric HA patients as primary or secondary prophylaxis.

Results: This bicenter study from Lahore and Rawalpindi reports statistically significant reduction in ABR after LDP (p=<0.0001) in 46 pediatric male patients. The study reports good compliance, reduced ABR and no development of new inhibitors in these patients all followed for one year post LDP start.

Conclusion: This study shows the efficacy of LDP in pediatric HA patients in reducing ABR. This also gives a safety signal in terms of no major breakthrough bleeds and no new inhibitor formation.