Adjusting for age and sex, the 10-year mortality rate for patients with transfusion-dependent β-thalassemia (TDT) is much higher compared with the general population, according to research published in the British Journal of Hematology. In addition, the comorbidity rate is also higher among this patient population.
β-thalassemia has varying clinical severity, depending on a number of genetic factors. Severity determines whether patients have TDT, which is associated with both worse disease-related comorbidities and adverse events linked with regular red blood cell transfusion.
While survival has greatly increased over the past half-century in this population, from a life expectancy of 17 years in 1970 to more than 40 years among 80% of patients after 2000, TDT is linked to numerous comorbidities and clinical complications.
For this study, researchers designed a retrospective analysis “to explore the 10-year mortality and prevalence of [comorbidities] in patients with TDT, using Hospital Episode Statistics (HES) data from the National Health Service (NHS) in England.” All evaluated data were obtained between 2009 and 2018.
Of more than 5000 patients identified with β-thalassemia, 612 patients with TDT in England were included in the analysis. Overall, 76% of patients had at least 1 comorbidity, 54% of patients had at least 2, and 37% had at least 3. Endocrine disorders, excluding diabetes, and osteoporosis were the most common comorbidities, affecting 40% of patients each, followed by diabetes (34%).
A total of 18% of patients had cardiac disease, notably atrial fibrillation (11%) and heart failure (9%).
Adjusting for age and sex, the 10-year mortality rate in the TDT population was found to be 6.2%, compared with 1.2% in the general population (P <.001).
The investigators noted several study limitations associated with the use of HES data, which was primarily obtained from NHS hospitals. Some of the limitations included the lack of clinical severity of the recorded comorbidities; the unsuitability of data for analysis of activity outside of the hospital setting, such as chelation therapy protocols; the possibility that the quality of data may be compromised due to clinical coding; and HES data not including adverse psychological events of health-related quality of life events associated with TDT.
“Using these data as a baseline it may also be possible to evaluate the impact of new therapies for [thalassemia], including transfusion-sparing agents and curative treatments,” the investigators noted.
“New treatment options that aim to reduce the burden of the disease by obviating the need for chronic transfusion and chelation therapy are needed to lessen the disease burden on patients and reduce the impact on NHS services,” concluded the authors.
Disclosures: Some authors have declared affiliations with or received funding from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.
Jobanputra M, Paramore C, Laird SG, McGahan M, Telfer P. Co-morbidities and mortality associated with transfusion-dependent beta-thalassaemia in patients in England: a 10-year retrospective cohort analysis. Br J Haematol. Published online October 23, 2020. doi:10.1111/bjh.17091