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.

Keywords: complications; epidemiological; real‐world; transfusion‐related β‐thalassaemia.

FIGURE 1

Genotypic profile of the study patient population. (A) Distribution of patients by β‐thalassemia genotype. (B) Frequency of β‐globin gene mutations in the study population.

FIGURE 2

History of complications in the study population. (A) Number of disease and treatment complications between diagnosis and enrolment in the overall population. (B) Percentage of patients with disease and treatment complications between diagnosis and enrolment overall and by age group. Patients who had complications attributed to more than one factor have been included in all applicable categories. (C) Severity of disease and treatment complications overall and by age group. ICT, iron chelation therapy.

FIGURE 3

Most common β‐thalassemia disease‐ and treatment‐related complications in the overall population. (A) Most common complications by System Organ Class in the overall study population, and in the subgroups of 18–40 and > 40 years of age. (B) Complications in ≥5.0% of the overall study population.

FIGURE 4

Most common grade ≥3 complications in the overall study population.

FIGURE 5

Association of patient and disease characteristics with the presence of β‐thalassemia disease‐ and treatment‐related complications at the study visit. (A) Multivariable logistic regression analysis for the association of patient and disease characteristics with the presence of disease‐ and/or treatment‐related complications. (B) Multivariable logistic regression analysis for the association of patient and disease characteristics with the presence of disease‐related complications. (C) Multivariable logistic regression analysis for the association of patient and disease characteristics with the presence of treatment‐related complications. Values in bold indicate statistical significance, that is, p < 0.05. ^aIn the 48 weeks before enrolment; ^bIncludes surgical and comorbidity history. CI, confidence interval; ICT, iron chelation therapy; OR, odds ratio.