Low Dose Prophylaxis (LDP) in Haemophilia- by Using Extended Half- Life (EHL) Factor Concentrates

Abstract

Objective: To assess the effectiveness of low dose prophylaxis  in Haemophilia, as compared to episodic treatment

Methodology:In this prospective cohort study twenty one patients with severe Haemophilia A, registered with Haemophilia Treatment Centre Rawalpindi, were enrolled . According to Low Dose Prophylaxis (LDP) protocol, patients were started on a uniform dose of extended half life (EHL), recombinant, Factor VIII concentrate (Eloctate) ,250 IU, intravenous , once weekly, escalated to twice weekly if a patient experienced more than two spontaneous bleeds in three months, and thrice weekly if bleeding continued after initial dose escalation. Twenty one patients ,receiving episodic treatment (treatment as per requirement of bleeding episode) were also included . Primary outcome was the total spontaneous bleeds. Secondary outcome was presence of factor VIII inhibitors.All the  patients were assessed every  four months for spontaneous bleeds . Inhibitors were screened in serum six monthly .Patients were followed for twenty four months and Annulized Bleeding Rate (ABR)was calculated .

Results: Eleven patients were on once weekly , nine on twice weekly and one thrice weekly infusions. Age range was two to  nine years (mean 4.07 years).  Low dose prophylaxis dose per Kg of body weight per week was calculated by multiplying the number of times the dose was given in a week with 250 IU and the result divided by the body weight in Kg. Mean Annualized Bleeding Rate (ABR) ,during study period was 0.07, which is significantly less as compared to episodic treatment group (p <0.001). Development of inhibitors was not found in any patient on prophylactic treatment. In prophylactic group majority (91.66%) did not showed any bleeding episode during treatment period .

Conclusion:Low Dose prophylaxis significantly reduces Annualized bleeding Rate(ABR) and production of inhibitors, as compared to those who are on episodic treatment .