Regular prophylaxis with activated prothrombin complex concentrates in pediatric hemophilia.

IF 1 4区医学 Q3 PEDIATRICS

Pediatrics International Pub Date : 2024-01-01 DOI:10.1111/ped.15774

Ayumi Horiguchi, Yuki Arakawa, Yuichi Mitani, Kiyotaka Isobe, Makiko Mori, Kohei Fukuoka, Koichi Oshima, Katsuyoshi Koh

{"title":"Regular prophylaxis with activated prothrombin complex concentrates in pediatric hemophilia.","authors":"Ayumi Horiguchi, Yuki Arakawa, Yuichi Mitani, Kiyotaka Isobe, Makiko Mori, Kohei Fukuoka, Koichi Oshima, Katsuyoshi Koh","doi":"10.1111/ped.15774","DOIUrl":null,"url":null,"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.","PeriodicalId":20039,"journal":{"name":"Pediatrics International","volume":null,"pages":null},"PeriodicalIF":1.0000,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pediatrics International","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1111/ped.15774","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"PEDIATRICS","Score":null,"Total":0}

引用次数: 0

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.

查看原文本刊更多论文

小儿血友病患者定期使用活性凝血酶原复合物浓缩物进行预防。

背景：定期使用活化凝血酶原复合物浓缩物（aPCCs）进行预防治疗对患有抑制剂的成人血友病患者有效，但儿童患者的数据却很少：这是在琦玉儿童医疗中心进行的一项单中心回顾性研究。研究对象：重度和中度血友病伴抑制剂老年患者：我们使用预防性 aPCCs（剂量为 50-100 U/kg；2-3 次/周）治疗了 9 名患有抑制剂的儿童血友病患者（中位年龄为 1.9 岁；年龄范围为 1.3-12.9 岁；使用 aPCCs 治疗前抑制剂滴度为 5.9-69 BU/mL）。中位预防期为 13 个月（5-31 个月）。在使用 aPCCs 预防性治疗期间，年化出血率（ABR）的中位数为 2（范围：0-17）。与按需使用 aPCCs 相比，在四名患者中，预防性使用 aPCCs 的 ABR 降低了 19%-100%。治疗的一个不良反应是，一名血友病 B 患者在开始定期预防性使用 aPCCs 34 个月后出现了肾病综合征：结论：即使是年龄较小的 A 型和 B 型血友病患儿，定期预防性使用 aPCC 也能降低 ABR。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Pediatrics International 医学-小儿科

CiteScore

2.00

自引率

7.10%

发文量

519

审稿时长

12 months

期刊介绍： Publishing articles of scientific excellence in pediatrics and child health delivery, Pediatrics International aims to encourage those involved in the research, practice and delivery of child health to share their experiences, ideas and achievements. Formerly Acta Paediatrica Japonica, the change in name in 1999 to Pediatrics International, reflects the Journal''s international status both in readership and contributions (approximately 45% of articles published are from non-Japanese authors). The Editors continue their strong commitment to the sharing of scientific information for the benefit of children everywhere. Pediatrics International opens the door to all authors throughout the world. Manuscripts are judged by two experts solely upon the basis of their contribution of original data, original ideas and their presentation.