학술논문
Real‐world complication burden and disease management paradigms in transfusion‐related β‐thalassaemia in Greece: Results from ULYSSES, an epidemiological, multicentre, retrospective cross‐sectional study
Document Type
Clinical report
Author
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
Source
ejHaem. August 2023, Vol. 4 Issue 3, p569, 13 p.
Subject
Language
English
Abstract
INTRODUCTION Beta (β)‐thalassaemia is a hereditary blood disorder characterised by a deficiency of functional β‐globin chains [1‐4]. Based on severity, β‐thalassaemia has been traditionally classified as major, intermedia or minor, [...]
: 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.
: 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.