Young Shil Park, Ji Kyoung Park, Jeong A Park, Hee Jo Baek, Jae Hee Lee, Chur Woo You, Chuhl Joo Lyu, Eun Jin Choi
{"title":"韩国 B 型血友病患者治疗方案和用药的临床数据。","authors":"Young Shil Park, Ji Kyoung Park, Jeong A Park, Hee Jo Baek, Jae Hee Lee, Chur Woo You, Chuhl Joo Lyu, Eun Jin Choi","doi":"10.1007/s44313-024-00024-8","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>To investigate the clinical treatment status, such as treatment regimen, bleeding events, and drug dose, in patients with hemophilia B in South Korea.</p><p><strong>Methods: </strong>In this retrospective chart review, data of patients with hemophilia B from eight university hospitals were collected. Demographic and clinical data, treatment data, such as regimen and number of injections, dose of factor IX concentrate, and bleeding data were reviewed. Descriptive analyses were performed with annual data for 2019, 2020, and 2021, as well as the three years consecutively.</p><p><strong>Results: </strong>The medical records of 150 patients with hemophilia B between January 1, 2019, and December 31, 2021, were collected. Among these, 72 (48.0%) were severe, 47 (31.3%) were moderate, and 28 (18.7%) were mild. The results showed approximately two times more patients receiving prophylaxis as those receiving on-demand therapy, with 66.1% of patients receiving prophylaxis in 2019, 64.9% in 2020, and 72.1% in 2021. Annualized bleeding rates were 2.2% (± 3.1) in 2019, 1.8% (± 3.0) in 2020, and 1.8% (± 2.9) in 2021 among patients receiving prophylaxis. For the doses of factor IX concentrate, patients receiving prophylaxis received an average of 41.6 (± 11.9) IU/Kg/Injection in 2019, 45.7 (± 12.9) IU/Kg/Injection in 2020, and 60.1 (± 24.0) IU/Kg/Injection in 2021.</p><p><strong>Conclusions: </strong>Clinically, prophylaxis is more prevalent than reported. Based on insights gained from current clinical evidence, it is expected that the unmet medical needs of patients can be identified, and physicians can evaluate the status of patients and actively manage hemophilia B using more effective treatment strategies.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.3000,"publicationDate":"2024-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11310372/pdf/","citationCount":"0","resultStr":"{\"title\":\"Clinical data on treatment regimen and use of medication among patients with hemophilia B in Korea.\",\"authors\":\"Young Shil Park, Ji Kyoung Park, Jeong A Park, Hee Jo Baek, Jae Hee Lee, Chur Woo You, Chuhl Joo Lyu, Eun Jin Choi\",\"doi\":\"10.1007/s44313-024-00024-8\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>To investigate the clinical treatment status, such as treatment regimen, bleeding events, and drug dose, in patients with hemophilia B in South Korea.</p><p><strong>Methods: </strong>In this retrospective chart review, data of patients with hemophilia B from eight university hospitals were collected. Demographic and clinical data, treatment data, such as regimen and number of injections, dose of factor IX concentrate, and bleeding data were reviewed. Descriptive analyses were performed with annual data for 2019, 2020, and 2021, as well as the three years consecutively.</p><p><strong>Results: </strong>The medical records of 150 patients with hemophilia B between January 1, 2019, and December 31, 2021, were collected. Among these, 72 (48.0%) were severe, 47 (31.3%) were moderate, and 28 (18.7%) were mild. The results showed approximately two times more patients receiving prophylaxis as those receiving on-demand therapy, with 66.1% of patients receiving prophylaxis in 2019, 64.9% in 2020, and 72.1% in 2021. Annualized bleeding rates were 2.2% (± 3.1) in 2019, 1.8% (± 3.0) in 2020, and 1.8% (± 2.9) in 2021 among patients receiving prophylaxis. For the doses of factor IX concentrate, patients receiving prophylaxis received an average of 41.6 (± 11.9) IU/Kg/Injection in 2019, 45.7 (± 12.9) IU/Kg/Injection in 2020, and 60.1 (± 24.0) IU/Kg/Injection in 2021.</p><p><strong>Conclusions: </strong>Clinically, prophylaxis is more prevalent than reported. Based on insights gained from current clinical evidence, it is expected that the unmet medical needs of patients can be identified, and physicians can evaluate the status of patients and actively manage hemophilia B using more effective treatment strategies.</p>\",\"PeriodicalId\":46224,\"journal\":{\"name\":\"Blood Research\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":2.3000,\"publicationDate\":\"2024-08-08\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11310372/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Blood Research\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1007/s44313-024-00024-8\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Blood Research","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1007/s44313-024-00024-8","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
背景:调查韩国血友病 B 患者的临床治疗状况,如治疗方案、出血事件和药物剂量:调查韩国 B 型血友病患者的临床治疗状况,如治疗方案、出血事件和药物剂量:在这项回顾性病历审查中,收集了 8 家大学医院的 B 型血友病患者数据。方法:在这项回顾性病历审查中,收集了 8 家大学医院的血友病 B 患者数据,审查了人口统计学和临床数据、治疗数据(如治疗方案和注射次数、浓缩因子 IX 的剂量)以及出血数据。对 2019 年、2020 年和 2021 年以及连续三年的年度数据进行了描述性分析:收集了 150 名 B 型血友病患者在 2019 年 1 月 1 日至 2021 年 12 月 31 日期间的医疗记录。其中,72 人(48.0%)为重度,47 人(31.3%)为中度,28 人(18.7%)为轻度。结果显示,接受预防性治疗的患者约为按需治疗患者的两倍,2019 年接受预防性治疗的患者占 66.1%,2020 年占 64.9%,2021 年占 72.1%。在接受预防性治疗的患者中,2019 年的年化出血率为 2.2%(± 3.1),2020 年为 1.8%(± 3.0),2021 年为 1.8%(± 2.9)。就浓缩因子 IX 的剂量而言,接受预防性治疗的患者在 2019 年平均接受 41.6(± 11.9)IU/Kg/注射,2020 年平均接受 45.7(± 12.9)IU/Kg/注射,2021 年平均接受 60.1(± 24.0)IU/Kg/注射:在临床上,预防性治疗比报告的更为普遍。结论:临床上,预防性治疗比报告的更为普遍。根据从当前临床证据中获得的见解,预计可以确定患者未满足的医疗需求,医生可以评估患者的状况,并采用更有效的治疗策略积极管理 B 型血友病。
Clinical data on treatment regimen and use of medication among patients with hemophilia B in Korea.
Background: To investigate the clinical treatment status, such as treatment regimen, bleeding events, and drug dose, in patients with hemophilia B in South Korea.
Methods: In this retrospective chart review, data of patients with hemophilia B from eight university hospitals were collected. Demographic and clinical data, treatment data, such as regimen and number of injections, dose of factor IX concentrate, and bleeding data were reviewed. Descriptive analyses were performed with annual data for 2019, 2020, and 2021, as well as the three years consecutively.
Results: The medical records of 150 patients with hemophilia B between January 1, 2019, and December 31, 2021, were collected. Among these, 72 (48.0%) were severe, 47 (31.3%) were moderate, and 28 (18.7%) were mild. The results showed approximately two times more patients receiving prophylaxis as those receiving on-demand therapy, with 66.1% of patients receiving prophylaxis in 2019, 64.9% in 2020, and 72.1% in 2021. Annualized bleeding rates were 2.2% (± 3.1) in 2019, 1.8% (± 3.0) in 2020, and 1.8% (± 2.9) in 2021 among patients receiving prophylaxis. For the doses of factor IX concentrate, patients receiving prophylaxis received an average of 41.6 (± 11.9) IU/Kg/Injection in 2019, 45.7 (± 12.9) IU/Kg/Injection in 2020, and 60.1 (± 24.0) IU/Kg/Injection in 2021.
Conclusions: Clinically, prophylaxis is more prevalent than reported. Based on insights gained from current clinical evidence, it is expected that the unmet medical needs of patients can be identified, and physicians can evaluate the status of patients and actively manage hemophilia B using more effective treatment strategies.