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People with transfusion‑dependent β‑thalassaemia (TDT) must adhere to a lifelong regimen of red blood cell (RBC) transfusions to survive and concomitant iron chelation to manage the iron overload caused by chronic transfusions.3 A lifetime of transfusions compounded by treatment‑related complications can be physically and psychologically demanding for TDT patients and their caregivers, significantly impacting health-related quality of life (HRQOL) for both.1,2
As haemoglobin levels wane between transfusions, patients with TDT may experience symptoms of anaemia.4 Moreover, the ongoing need for transfusion and chelation places a significant burden on their quality of life. The process of receiving a transfusion can take up a patient’s entire day when travel, blood tests and results, blood transport to the transfusion centre, and the transfusion itself are considered.1,5
According to a longitudinal cohort study looking at health-related quality of life (HRQOL) in 264 patients over age 14 in North America (n=229) and the United Kingdom (n=35), patients reported having a lower HRQOL than the general population.1
A study of 109 ex-thalassaemia patients (median follow-up age 22.8 years) conducted more than 20 years after HSCT showed that long-term HRQOL scores (SF-36) in these patients were similar to the general population. However, clinically meaningful differences were seen for the General Health scale (p=0.005) due to patients who experienced acute or chronic GvHD, resulting in limitations of physical performance.
The study also showed that patients who received HSCT for thalassaemia reported improved HRQOL when compared to patients on conventional medical therapy.
Age- And Gender-Adjusted Mean Differences of SF-36 Scale Scores In Transplanted Patients (n=109) And Conventionally Treated Patients (n=124)7
Adapted from La Nasa G, Caocci G, Efficace F, et al. Long-term health-related quality of life evaluated more than 20 years after hematopoietic stem cell transplantation for thalassemia. Blood. 2013;122(13):2262-70.
A study of 516 paediatric and adult patients looked at survival rates in age/sex-matched patients who received conventional therapy with transfusion and chelation (n=258) vs. those who received allogeneic HSCT (n=258). Two-thirds of the patients who received HSCT had matched related donors, and a third had matched unrelated donors.
There was no statistically significant difference found in 30‑year OS between the two cohorts.
Clinical Outcomes In Conventionally Treated vs. Transplanted Thalassaemia Patients8
Adapted from Caocci G, Orofino MG, Vacca A, et al. Long‐term survival of beta thalassemia major patients treated with hematopoietic stem cell transplantation compared with survival with conventional treatment. Am J Hematol. 2017;92:1303–10.
The vast majority (94.2%) of patients who survived HSCT were thalassaemia-free. The study confirmed that the Pesaro risk score was significantly associated with higher overall survival and thalassaemia-free survival. However, mortality in the first ten years following HSCT was significantly higher compared to conventional treatment, with nearly 50% of the mortality being driven by acute GvHD.8 Unfortunately, the incidence of acute GvHD following transplantation has not significantly improved over the decades, and lowering the incidence of mortality due to acute GvHD remains an unmet need.8
Take the β-Thalassaemia Challenge
In a longitudinal cohort of 264 thalassaemia patients over age 14 (of which 85.6% had regularly transfused beta‑thalassaemia), patients reported having lower HRQOL than the normal population in which health domains?
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