The findings have implications for low resource countries, the researchers reported.
Novie A. Chozie, MD, of Universitas Indonesia in Jakarta, along with colleagues, conducted a parallel-group, randomized controlled study of 50 children with severe hemophilia A. Study participants were randomized to receive either low‐dose FVIII prophylaxis (n = 25) or on‐demand treatment (n = 25) for a total of 12 months. The findings were published in.
Participants in the prophylaxis arm received FVIII at a dose of 10 IU/kg, infused twice weekly. If patients experienced an episode of acute bleeding, prophylaxis was delayed until the episode resolved.
The primary outcome was the number of total and joint bleeding events from the start of therapy to 12 months. Secondary outcomes included evidence of FVIII inhibitor, Hemophilia Joint Health Score (HJHS), and Hemophilia Early Arthropathy Detection Ultrasound (HEAD‐US) score.
After analysis, the team found that the number of joint and total bleeding events was significantly lower in the prophylaxis group. For total bleeding events, there was a median of 8 events with the prophylaxis group, compared with 25 in the on-demand treatment group (P less than .001). There was a median of three joint bleeding events in the prophylaxis group versus nine in the on-demand group (P less than .001).
Patients in the prophylaxis arm also showed improved joint function (P = .004), while those in the on‐demand arm showed evidence of deterioration (P = .001).
Two key limitations of the study were short duration of follow-up and single‐center design, which could have limited the generalizability of the results.
“In countries with limited resources, low‐dose prophylaxis is strongly recommended as a therapeutic option for severe haemophilia A [in] children,” the researchers wrote.
The study was funded by Grifols. The authors reported having no conflicts of interest.
SOURCE: Chozie NA et al. Haemophilia. 2019 May 2. .