Abstract：
BACKGROUND: Regular prophylaxis with activated prothrombin complex concentrates (aPCCs) is effective in adult patients with hemophilia with inhibitors; however, data in children are scarce.
METHODS: This was a single-center retrospective study at Saitama Children\'s Medical Center. Patients with severe and moderate hemophilia with inhibitors aged <15 years at the start of aPCCs prophylaxis were included. Medical records were retrospectively reviewed.
RESULTS: We treated nine pediatric patients with hemophilia with inhibitors (median age, 1.9 years; age range, 1.3-12.9 years; inhibitor titers before treatment with aPCCs, 5.9-69 BU/mL) using prophylactic aPCCs (doses, 50-100 U/kg; 2-3 times/week). The median prophylactic period was 13 months (range: 5-31 months). The median annualized bleeding rate (ABR) during prophylactic treatment with aPCCs was 2 (range, 0-17). In four patients, ABR was reduced by 19%-100% with prophylactic aPCCs compared to on-demand aPCCs. An adverse effect of treatment was that a patient with hemophilia B developed nephrotic syndrome 34 months after starting regular prophylaxis with aPCCs.
CONCLUSIONS: Regular prophylactic aPCCs reduced the ABR even in younger children with hemophilia A and B. Serious adverse events include nephrotic syndrome, which requires caution.
METHODS: This was a single-center retrospective study at Saitama Children\'s Medical Center. Patients with severe and moderate hemophilia with inhibitors aged <15 years at the start of aPCCs prophylaxis were included. Medical records were retrospectively reviewed.
RESULTS: We treated nine pediatric patients with hemophilia with inhibitors (median age, 1.9 years; age range, 1.3-12.9 years; inhibitor titers before treatment with aPCCs, 5.9-69 BU/mL) using prophylactic aPCCs (doses, 50-100 U/kg; 2-3 times/week). The median prophylactic period was 13 months (range: 5-31 months). The median annualized bleeding rate (ABR) during prophylactic treatment with aPCCs was 2 (range, 0-17). In four patients, ABR was reduced by 19%-100% with prophylactic aPCCs compared to on-demand aPCCs. An adverse effect of treatment was that a patient with hemophilia B developed nephrotic syndrome 34 months after starting regular prophylaxis with aPCCs.
CONCLUSIONS: Regular prophylactic aPCCs reduced the ABR even in younger children with hemophilia A and B. Serious adverse events include nephrotic syndrome, which requires caution.
摘要：
背景：使用活化的凝血酶原复合物浓缩物（aPCCs）定期预防对使用抑制剂的成年血友病患者有效；然而，儿童的数据很少。
方法：这是一项在Sa玉儿童医疗中心进行的单中心回顾性研究。包括在aPCCs预防开始时年龄<15岁的重度和中度血友病患者。对病历进行回顾性审查。
结果：我们用抑制剂治疗了9例血友病患儿（中位年龄，1.9岁；年龄范围，1.3-12.9年；用aPCCs治疗前的抑制剂滴度，5.9-69BU/mL）使用预防性aPCCs（剂量，50-100U/kg；2-3次/周）。中位预防期为13个月（范围：5-31个月）。aPCCs预防性治疗期间的中位年出血率（ABR）为2（范围，0-17）。在四名患者中，与按需aPCCs相比，预防性aPCCs的ABR降低了19％-100％。治疗的不利影响是血友病B患者在开始定期预防aPCCs后34个月发展为肾病综合征。
结论：即使在患有血友病A和B的年幼儿童中，定期预防性aPCCs也能降低ABR。严重的不良事件包括肾病综合征，这需要谨慎。
方法：这是一项在Sa玉儿童医疗中心进行的单中心回顾性研究。包括在aPCCs预防开始时年龄<15岁的重度和中度血友病患者。对病历进行回顾性审查。
结果：我们用抑制剂治疗了9例血友病患儿（中位年龄，1.9岁；年龄范围，1.3-12.9年；用aPCCs治疗前的抑制剂滴度，5.9-69BU/mL）使用预防性aPCCs（剂量，50-100U/kg；2-3次/周）。中位预防期为13个月（范围：5-31个月）。aPCCs预防性治疗期间的中位年出血率（ABR）为2（范围，0-17）。在四名患者中，与按需aPCCs相比，预防性aPCCs的ABR降低了19％-100％。治疗的不利影响是血友病B患者在开始定期预防aPCCs后34个月发展为肾病综合征。
结论：即使在患有血友病A和B的年幼儿童中，定期预防性aPCCs也能降低ABR。严重的不良事件包括肾病综合征，这需要谨慎。
