Introduction 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.

Background: Recurrent joint bleeds leading to arthropathy is the main problem in severe hemophilia children. This study aimed to investigate joint status in severe hemophilia A children receiving episodic treatment in Cipto Mangunkusumo Hospital, Jakarta.Methods: A cross-sectional study was conducted in Cipto Mangunkusumo Hospital as Indonesian National Hemophilia Treatment Center on children (4–18 years) with severe hemophilia A, who previously received episodic treatment, with no history of inhibitor factor VIII. Hemophilia Joint Health Score was evaluated according to HJHS version 2.1 2011. Joint ultrasonography was done for six index joints (bilateral elbows, knees and ankles) using Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) methods. Data of age of first joint bleed, number of target joints and inhibitor factor VIII were obtained from the Pediatric Hemophilia Registry and medical records.Results: There were 59 subjects aged 4 to 18 years. Twenty-nine out of 59 (49.2%) subjects experienced first joint bleed before of 2 years of age. The most common of joint bleeds was a right ankle. Mean total HJHS was 8.71±8.73. Subjects aged 4–10 years showed lower HJHS (4.6±3.7) as compared to subjects aged >10–18 years (12.3±10.3), p<0.001; 95% CI=4.9–13. Mean HEAD-US scores in subjects aged 4–10 years (18.7±5.6) was lower than in subjects aged >10–18 years (28±7.9), p<0.001, 95% CI= -12.9–-5.6.Conclusion: HJHS and HEAD-US scores of severe hemophilia A children receiving episodic treatment aged 4–10 years are lower compared to subjects aged >10–18 years, indicating more severe joint destruction in older children and progressivity of joint damage over time. It is important to start prophylactic treatment to prevent progressivity of joint damage.

Background Repeated bleeding in hemophilic arthropathy (HA) may result in severe degenerative changes and joint destruction. The gradient-recalled echo (GRE) sequence MR is proved to be the best method to detect hemosiderin deposition. However, MR is not widely available in developing countries, including Indonesia. Some studies have proposed ultrasonography (US) as an alternative tool in evaluating hemophilic joint. However, there is still some disagreement on the ability of US to detect hemosiderin deposition. Objective To evaluate the association between US and GRE-sequence MR imaging in detecting hemosiderin deposition in hemophilic ankle joint. Material and methods A total of 102 sites from 17 ankle joints of 11 boys with severe hemophilia A underwent US examination using a high-frequency linear array transducer. GRE-sequence MR examination was performed in sagittal view consistent with the sites scanned by US. Both examinations were performed on the same day, but MR interpretation was performed blindly at different times. The association between US and GRE-sequences in detecting hemosiderin deposition was analyzed using McNemar’s test. Results Statistical analysis showed a significant association (p value < 0.001) between US and GRE MR in detecting hemosiderin deposition, but the association is weak (R = 0.26). Sensitivity and specificity of US for detecting hemosiderin deposition were 46.84% (95%CI: 35.51–58.40) and 95.65% (95%CI: 78.05–99.89), respectively, with positive predictive value 97.37% (95%CI: 84.29–99.61), negative predictive value 34.38% (95%CI: 29.50–39.60) and accuracy 57.84% (95%CI: 47.66–67.56). Conclusion There was a weak association between US and GRE-sequences in detecting hemosiderin deposition of hemophilic ankle joint. ​​