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.

BackgroundNasopharyngeal carcinoma (NPC) is endemic in Indonesia and 20% of the patients are diagnosed before the age of 31. This study evaluates presentation and treatment outcome of young patients in Jakarta, in a tertiary referral centre.MethodsForty-nine patients under the age of 31, diagnosed with NPC between July 2004 and January 2007, were evaluated. Baseline data included histological type, stage of disease and presenting symptoms. We intended to follow all patients after diagnosis to reveal treatment outcome and overall survival (OS).ResultsAll but two patients had advanced stage disease (94%), 7 (14%) had distant metastasis. The median interval between start of complaints and diagnosis was 9 months. Forty-two patients were planned for curative intent treatment. Eleven patients (26%) never started treatment, 2 patients did not complete treatment and 3 patients did not return after finishing treatment. Four patients died before radiation could start. Three patients died within 4 months after treatment. Nine patients (21%) had a complete response. Due to the high number of patients who were lost to follow-up (LFU), OS was analyzed as follows: a best-case (patients censored at last contact) and a worst-case scenario (assuming that patients who did not finish treatment or had disease at last contact would have died). The 2-year OS for patients without distant metastases was 39–71%.ConclusionTreatment outcome for young patients with NPC in this institute was poor. Improvement can be achieved when NPC is diagnosed at an earlier stage and when there is better treatment compliance.

21,1%;31,2%) dan event-free survival (EFS) 6,5% (95% IK: 3,1%;6,2%). Angka loss to follow-up mencapai sepertiga jumlah pasien yang mempengaruhi hasil estimasi kesintasan (overall survival). Rendahnya EFS disebabkan oleh angka kematian yang tinggi mencapai 50 dari 93 pasien dan 62% di antaranya disebabkan oleh sepsis. Kesimpulan. Keberhasilan pengobatan LMA masih sangat rendah dibandingkan laporan penelitian dari negara lain. Faktor yang paling berperan terhadap hal ini adalah kematian yang tinggi akibat infeksi berat atau sepsis. Komplikasi infeksi mungkin terjadi akibat toksisitas obat dan fasilitas perawatan untuk pasien LMA kurang memadai. Stratifikasi risiko pasien LMA dan evaluasi protokol kemoterapi yang diberikan serta penyediaan fasilitas perawatan yang baik akan memperbaiki hasil pengobatan LMA.