Efficacy and Cost-Effectiveness of Low-Dose vs Standard Dose Prophylaxis for Hemophilia in Indonesia: A Systematic Review.

Abstract

Studies on high-dose prophylaxis therapy using Factor VIII show promising decrease in the Annual Bleeding Rates (ABR) in hemophilia patients. However, the greater dose and frequency raise concerns on cost considerations and adherence of the patients, especially in several countries where resources are limited. Other data has proven that the low dose prophylaxis is also promising regarding the decrease of ABR. The purpose of this systematic review is to conduct a comprehensive analysis of the lower dosage formulation used for prophylaxis in hemophilia. A PubMed and Embase database search was performed based on articles that met the following criteria: written in English language and published within the last 10 years. Consequently, the following key terms were used in combination: 'high dose', 'low dose', 'recombinant', 'prophylaxis', and 'hemophilia' in different combinations. 19 articles were included for this review. 10 of the investigated papers demonstrated decrease in ABR, functional improvement of affected joints, alleviation of pain, and a better quality of life in hemophilia patients. Low dose prophylaxis has proven to significantly improve symptoms, lower ABR and preserve joint and bone health compared to episodic or on-demand treatment. Furthermore, low dose prophylaxis (LDP) was also observed to be cost-effective and more convenient in certain countries, especially in south-east Asia where resources are limited. Overall, low dose prophylaxis appears to be non-inferior in improving the overall Quality of Life in people with hemophilia, and therefore could be a beneficial alternative in countries of the south east Asian region.