Low-dose prophylaxis and its impact on the health of haemophilia patients.

Abstract

There is convincing evidence to show that low-dose prophylaxis (LDP) results in reduction in annualized bleeding rate (ABR) and better health-related quality of life (HRQoL) compared with on-demand or episodic treatment (ET) in haemophilia patients. The aim is to review various LDP protocols practised for the treatment of haemophilia, specifically in resource-limited countries. A literature survey was made of articles published in English language in PubMed and EMBASE without any time limit using keywords 'low dose', 'prophylaxis' and 'haemophilia' in different combinations. A total of 19 reports involving LDP in patients with haemophilia were included in this review. Almost all studies reported reduction in ABR, improvement in joint function, pain and HRQoL compared with ET, but this did not fully translate into significant improvement in structural arthropathy already caused by earlier bleeds, suggesting that LDP may be less or ineffective in either stopping or reversing the damage. Individualized dose escalation protocols based on pharmacokinetic (PK) or clinical parameters were found to be superior to fixed LDP protocols and cost-effective compared with standard dose protocols. The developing countries can initiate LDP as the first step of prophylaxis, but certainly this should not be the final goal of the health care system in any country. Due to the complex pathophysiological mechanisms underlying haemophilic arthropathy, long-term data on LDP in haemophilia patients are warranted.