Kattamis Antonis, Voskaridou Ersi, Delicou Sophia, Klironomos Evangelos, Lafiatis Ioannis, Petropoulou Foteini, Diamantidis Michael D, Lafioniatis Stylianos, Evliati Loukia, Kapsali Eleni, Karvounis-Marolachakis Kiki, Timotheatou Despoina, Deligianni Chrysoula, Viktoratos Panagiotis, Kourakli Alexandra
First Department of Pediatrics Thalassemia Unit National and Kapodistrian University of Athens Athens Greece.
Expertise Center in Rare Haematological Diseases-Haemoglobinopathies General Hospital of Athens "Laikon" Athens Greece.
EJHaem. 2023 May 23;4(3):569-581. doi: 10.1002/jha2.695. eCollection 2023 Aug.
Patients with transfusion-dependent beta (β)-thalassaemia experience a broad range of complications. ULYSSES, an epidemiological, multicentre, retrospective cross-sectional study, aimed to assess the prevalence and severity of treatment and disease complications, capture disease management and identify predictors of complications in patients with transfusion-dependent β-thalassaemia, treated in routine settings in Greece. Eligible patients were adults diagnosed with β-thalassaemia ≥12 months before enrolment and having received ≥6 red blood cell (RBC) units (excluding elective surgery) with no transfusion-free period ≥35 days in the 24 weeks before enrolment. Primary data were collected at a single visit and through chart review. Between Oct 21, 2019, and Jun 15, 2020, 201 eligible patients [median (interquartile range, IQR) age 45.7 (40.2-50.5) years; 75.6% > 40 years old; 64.2% female] were enrolled, a mean (standard deviation) of 42.9 (7.8) years after diagnosis. Median (IQR) age at diagnosis and RBC transfusion initiation were 0.8 (0.4-2.8) and 1.3 (1.0-5.0) years, respectively. From diagnosis to enrolment, patients had developed a median of six (range: 1-55) complications; 19.6% were grade ≥3. The most represented complications were endocrine/metabolic/nutrition disorders (91.5%), surgical/medical procedures (67.7%) and blood/lymphatic system disorders (64.7%). Real-world data generated by ULYSSES underscore the substantial complication burden of transfusion-dependent β-thalassaemia patients, routinely managed in Greece.
依赖输血的β地中海贫血患者会出现各种各样的并发症。“尤利西斯”（ULYSSES）是一项流行病学、多中心、回顾性横断面研究，旨在评估在希腊常规治疗环境中接受治疗的依赖输血的β地中海贫血患者的治疗及疾病并发症的患病率和严重程度，了解疾病管理情况并确定并发症的预测因素。符合条件的患者为在入组前≥12个月被诊断为β地中海贫血且在入组前24周内接受了≥6个红细胞（RBC）单位（不包括择期手术）且无≥35天无输血期的成年人。主要数据通过单次就诊和病历审查收集。在2019年10月21日至2020年6月15日期间，纳入了201例符合条件的患者[年龄中位数（四分位间距，IQR）为45.7（40.2 - 50.5）岁；75.6%＞40岁；64.2%为女性]，诊断后平均（标准差）42.9（7.8）年。诊断和开始红细胞输血时的年龄中位数（IQR）分别为0.8（0.4 - 2.8）岁和1.3（1.0 - 5.0）岁。从诊断到入组，患者出现的并发症中位数为6种（范围：1 - 55种）；19.6%为≥3级。最常见的并发症是内分泌/代谢/营养障碍（91.5%）、外科/医疗程序（67.7%）和血液/淋巴系统障碍（64.7%）。“尤利西斯”（ULYSSES）生成的真实世界数据凸显了在希腊接受常规管理的依赖输血的β地中海贫血患者的巨大并发症负担。
