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

Abstract

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.

Addressing Thalassaemia Management from Patients’ Perspectives: An International Collaborative Assessment

“…Overall, the majority of thalassaemia patients around the world are receiving good-quality care including timely blood transfusions [22], chelation therapy [23], adequate disease-specific medical monitoring, and appropriate imaging, and about half of them report being satisfied or very satisfied with the medical management provided [24]. Nevertheless, based on our findings, a sizeable percentage of the study participants lack comprehensive services, face difficulties in accessing guideline-based recommended care, face barriers in receiving timely care due to financial difficulties, and are not satisfied with the level of healthcare services they receive.…”

Addressing Thalassaemia Management from a Patients’ Perspective: an International Collaborative Assessment

“…Overall, the majority of Thalassaemia patients around the world is receiving good quality care including timely blood transfusions [21], chelation therapy [22], adequate disease-specific medical monitoring, appropriate imaging and about half of them report being satisfied or very satisfied with medical management [23]. Nevertheless, based on our findings, a sizeable percentage of the study participants, are lacking comprehensive services, are facing difficulties in accessing guideline-based recommended care, face barriers to receive timely care due to financial difficulties and are not satisfied with the level of healthcare services they enjoy.…”