Premium
Comparison of the efficacy and safety of 12‐month low‐dose factor VIII tertiary prophylaxis vs on‐demand treatment in severe haemophilia A children
Author(s) -
Chozie Novie A.,
Primacakti Fitri,
Gatot Djajadiman,
Setiabudhy Rahajuningsih D.,
Tulaar Angela B. M.,
Prasetyo Marcel
Publication year - 2019
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1111/hae.13770
Subject(s) - medicine , haemophilia , haemophilia a , arthropathy , group b , adverse effect , pediatrics , surgery , randomized controlled trial , hemarthrosis , osteoarthritis , alternative medicine , pathology
Prophylaxis has commonly become standard treatment for severe haemophilia patients. The World Federation of Hemophilia (WFH) recommends low‐dose prophylaxis in countries with resource constraints. Objective To determine efficacy and safety of low‐dose factor VIII (FVIII) tertiary prophylaxis compared to on‐demand treatment in severe haemophilia A children in Indonesia. Methods Eligible patients were randomly assigned to prophylaxis and on‐demand groups. Patients in the prophylaxis group received infusion of FVIII 10 IU/kg body weight, two times per week. Primary outcomes were the numbers of joint bleeding and total bleeding episodes; secondary outcomes were evidence of FVIII inhibitor, Hemophilia Joint Health Score (HJHS) and Hemophilia Early Arthropathy Detection Ultrasound (HEAD‐US) score. Patients were monitored for 12 months. Results Fifty patients, all with tertiary prophylaxis, 4‐18 years of age, were randomized into prophylaxis (n = 25) and on‐demand (n = 25) groups. The mean follow‐up time was 12.8 ± 0.86 vs 12.3 ± 0.54 months, respectively. Numbers of total and joint bleeding episodes were significantly lower in the prophylaxis group ( P < 0.001, 95% CI −24.6;−10.7 and P < 0.001, 95% CI −14;−3, respectively) . The prophylaxis group showed improvement of joint function ( P = 0.004; CI 95% −3;−0.5); on the contrary, we found deterioration in the on‐demand group ( P = 0.001; CI 95% 1;3). HEAD‐US scores showed improvement at month 6 in the prophylaxis group, but there was no significant difference between groups at month 12. Conclusion Low‐dose FVIII tertiary prophylaxis was effective in reducing joint bleeding episodes and improvement of HJHS compared to on‐demand FVIII treatment in severe haemophilia A children.