OBJECTIVES

: Prophylactic treatment of patients with hemophilia using factor VIII and IX infusions is considered to be a standard approach of care by World Federation of Hemophilia. However, hemophilia patients from Southeast Asian countries have to rely on ‘on-demand’ therapy due to high out-of-pocket expenses associated with prophylactic therapy. Therefore, we conducted a systematic review to compare the efficacy with prophylactic and on-demand factor VIII and IX therapy in hemophilia patients from Southeast Asia to address the need of evidence based reimbursement decision making in hemophilia. Risk of inhibitor development against both therapies was also compared.

METHODS

: A systematic literature search was conducted using PubMed, Cochrane, and Google Scholar. English language, full text articles, comparing the efficacy and inhibitor development with prophylactic vs on demand factor VIII and IX in Southeast Asian patients with hemophilia were included.

RESULTS

: Of the 98 retrieved studies, 8 (8.16%) met the inclusion criteria (6 RCTs, 2 uncontrolled), including a total of 663 patients. Four studies (50.0%) were from Malaysia, two (25.0%) were from China, and one (12.5%) each from Hong Kong and Singapore. Out of 7 (7.14%) studies that evaluated the efficacy, mean/median annualized bleeding rate (ABR) was the most common efficacy endpoint (assessed by 100% studies). Improvement in ABR was greater for prophylaxis therapy (mean 2.93-11.8, median 0-3.9) vs on-demand therapy (mean 31.14-41.5, median 15.58-57.5). Out of 6 (6.12%) studies that evaluated inhibitor development, none of the studies reported inhibitor development with prophylaxis or on-demand therapy.

CONCLUSIONS

: The evidence suggest that prophylactic factor VIII/IX treatment in Southeast Asian patients have better efficacy compared with the on-demand therapy. These results suggest to make policy changes for offering financial protection to patients with hemophilia.