Manco-Johnson Marilyn J, Le Binh, Acharya Suchitra, Ahuja Sanjay, Chitlur Meera, Citla-Sridhar Divyaswathi, Fedewa Stacey Ann, Isaac Daniel, Kulkarni Roshni, Schieve Laura A, Sharathkumar Anjali, Soucie J Michael
Department of Pediatrics, Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora, CO.
Division of Blood Disorders and Public Health Genomics, Centers for Disease Control and Prevention, Atlanta, GA.
Blood Vessel Thromb Hemost. 2025 Jan 15;2(3):100047. doi: 10.1016/j.bvth.2025.100047. eCollection 2025 Aug.
Joint bleeding is the primary determinant of end-stage arthropathy in hemophilia; joint bleeding has greatly decreased with the use of prophylaxis and introduction of highly effective therapies. This study aimed to determine current risk factors for joint bleeding in persons with severe hemophilia A or B. Demographic, treatment, and bleeding outcome data from Community Counts, a US national surveillance project, were analyzed. Data were collected at annual visits between 2013 and 2022. Eligibility included factor VIII or IX of <1%, no inhibitor, age of 2 to 44 years, and on treatment with continuous prophylaxis. Annual joint bleeding rate (AJBR) differences across demographic and clinical subgroups were compared via rate ratios and 95% confidence intervals, and with multivariate methods accounting for multiple measurements over time. The analysis included 2527 males with hemophilia, 7211 observation years, and 10 046 joint bleeds. Lower AJBR in hemophilia A was most strongly associated with use of emicizumab. For both hemophilia A and B, patient-associated factors, younger age, fewer missed doses, and full employment were all independently associated with lower AJBR, as was treatment in the Northeast of the United States. The findings of this comprehensive analysis of a large, diverse sample drawn from hemophilia treatment centers across the United States, underscore the important contributions of patient, community, and treatment factors on joint outcomes in severe hemophilia. Use of emicizumab and few missed doses independently predicted low AJBR, highlighting the interplay of access and adherence to care.
关节出血是血友病终末期关节病的主要决定因素；随着预防措施的使用和高效疗法的引入，关节出血已大幅减少。本研究旨在确定重度甲型或乙型血友病患者当前关节出血的风险因素。对美国一项全国性监测项目“社区计数”中的人口统计学、治疗和出血结局数据进行了分析。数据收集于2013年至2022年的年度随访中。入选标准包括因子VIII或IX水平<1%、无抑制剂、年龄在2至44岁之间且接受持续预防治疗。通过率比和95%置信区间，并采用考虑了随时间多次测量的多变量方法，比较了不同人口统计学和临床亚组的年度关节出血率（AJBR）差异。分析纳入了2527名血友病男性患者，共7211个观察年和10046次关节出血事件。甲型血友病较低的AJBR与使用艾美赛珠单抗最为密切相关。对于甲型和乙型血友病，患者相关因素、年龄较小、漏服剂量较少以及全职工作均与较低的AJBR独立相关，美国东北部的治疗情况也是如此。这项对来自美国各地血友病治疗中心的大量多样样本进行的综合分析结果强调了患者、社区和治疗因素对重度血友病关节结局的重要贡献。使用艾美赛珠单抗和较少的漏服剂量独立预测了较低的AJBR，突出了获得治疗和坚持治疗之间的相互作用。
