土耳其 B 型血友病回顾性分析：确定主要特征和治疗方案

IF 3.4 3区医学 Q2 HEMATOLOGY

Research and Practice in Thrombosis and Haemostasis Pub Date : 2024-10-01 DOI:10.1016/j.rpth.2024.102588

Bülent Zülfikar , Başak Koç , Fahri Şahin , Hatice İlgen Şaşmaz , Kaan Kavaklı , Can Balkan , Ali Bülent Antmen , Sinan Akbayram , Birol Güvenç , Vahap Okan , Emine Türkkan , Canan Albayrak , Davut Albayrak , Nazan Sarper , Tülin Tiraje Celkan , Orhan Ayyıldız , Salih Aksu , Türkan Patıroğlu , Zafer Şalcıoğlu , Adalet Meral Güneş , Muhlis Cem Ar

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引用次数: 0

摘要

背景B型血友病（HB）是一种X连锁隐性遗传性出血性疾病，在男性中发病率较高。目标首次在土耳其全国范围内对B型血友病患者进行队列研究，以确定其人口统计学、临床特征和治疗模式。方法这项多中心回顾性研究纳入了1961年至2018年间在35个中心登记的433名存活的B型血友病患者。按年龄亚组（0-17 岁、18-64 岁和≥65 岁）、疾病严重程度按因子水平（重度，<1 IU/dL；中度，1-5 IU/dL；轻度，>5 IU/dL）进行分析。此外，还根据患者在相关研究中心开始随访的年份进行了分层，形成了两个时期：1993-2006年（简称A时期）和2007-2018年（简称B时期）。结果男性居多（98.6%），数据输入时的中位年龄为22.1岁（n = 429）。大多数患者（49.0%）患有中度 HB，其次是重度（30.0%）和轻度（15.7%）。在 377 名有完整治疗资料的患者中，209 人（55.4%）从诊断开始就接受了预防性治疗，79 人（21.0%）只接受了按需治疗。此外，89 名患者（23.6%）最初接受了按需治疗，后来转为预防性治疗。膝关节是主要出血部位，也是最常干预的关节。大多数大手术（47.5%）和小手术（53.3%）都是在重度 HB 患者身上进行的，而一半的放射性滑膜切除术是在中度 HB 患者身上进行的。

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Retrospective analysis of hemophilia B in Turkey: identifying main characteristics and treatment options

Background

Hemophilia B (HB), an X-linked recessive inherited bleeding disorder, exhibits a high prevalence among males.

Objectives

To present the first national cohort of persons with HB to define the demographics, clinical characteristics, and treatment patterns in Turkey.

Methods

This multicenter, retrospective study included 433 alive persons with HB registered in 35 centers between 1961 and 2018. Analyses were performed by age subgroups (0-17 years, 18-64 years, and ≥65 years), disease severity by factor levels (severe, <1 IU/dL; moderate, 1-5 IU/dL; mild, >5 IU/dL). Additionally, patients were stratified based on the initiation year of follow-up at the relevant study center, creating 2 periods: 1993-2006 (referred to as period A) and 2007-2018 (referred to as period B).

Results

Predominantly male (98.6%), the median age at data entry was 22.1 years (n = 429). The majority (49.0%) had moderate HB, followed by severe (30.0%) and mild (15.7%) disease. Of the 377 patients with complete treatment details, 209 (55.4%) were under prophylaxis from their diagnosis onwards, while 79 patients (21.0%) only received on-demand treatment. Additionally, 89 patients (23.6%) initially underwent on-demand treatment and later were switched to prophylaxis. Knees were the primary site of bleeding and the most frequently intervened joints. Most of the major (47.5%) and minor (53.3%) orthopedic procedures were carried out in persons with severe HB, while half of radioactive synovectomy procedures were performed on persons with moderate HB.

Conclusion

This paper describes the demographics, clinical characteristics, and treatments patterns of a large cohort of alive persons with HB on a national scale.

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