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Transfusion-Dependent Beta-Thalassemia (TDT) Significantly Impacts 
Quality of Life of 
Patients and Caregivers1,2

Ongoing Management and its Complications can be a Burden on Caregivers and Patients Living with Beta-Thalassemia

Patient and caregiver quality of life are significantly affected by ongoing transfusions, iron chelation therapy, and disease complications.1,2

Beta-thalassemia blood transfusion

People with transfusion-dependent beta-thalassemia (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.5 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

Living with transfusion-dependent beta-thalassemia

As hemoglobin levels wane between transfusions, patients with TDT may experience symptoms of anemia.3 Moreover, the life-long need for transfusion and chelation places a significant burden on their quality of life. The process of receiving a transfusion can:5

  • Take up a patient's entire day
  • Require time off work or school
  • Require transport to and from the transfusion center

Complications Of Their Disease and Chronic Treatment Are Associated With Lowered Quality of Life Scores In TDT Patients

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

  • Adolescent and adult patients in the Thalassemia Longitudinal Cohort (TLC) had significantly worse HRQOL on 5 of the 8 subscales (physical functioning, role-physical, general health, social functioning, and role-emotional; p<0.05), and on both summary scales (physical component summary and mental component summary)
  • Lower HRQOL was also reported in women, older patients, and those who had more disease complications and chelation-related side effects

Quality of Life: Conventional Therapy vs. Allogeneic HSCT7

A study of 109 ex-thalassemia 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 thalassemia 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

Quality of life scores for transplanted beta-thalassemia patients vs conventionally treated patients
Differences in quality of life scores between transplanted and conventionally treated thalassemia patients were seen for role-physical, bodily pain and role emotional.

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):2262-2270.

Mortality Risk: Conventional Therapy vs. Allogeneic HSCT8

A study of 516 pediatric 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 overall survival (OS) between the two cohorts.

  • In patients who received HSCT, 30‑year overall survival (OS) was 82.6 ± 2.7% and thalassemia-free survival (TFS) was 77.8 ± 2.9%
  • In patients receiving conventional transfusion/chelation therapy, 30‑year OS was 85.3 ± 2.7%

Clinical Outcomes In Conventionally Treated vs. Transplanted Thalassemia Patients8

Clinical outcomes for transplanted beta-thalassemia patients vs conventionally treated patients

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;92:1303-1310.

family family

The vast majority (94.2%) of patients who survived HSCT were thalassemia-free. The study confirmed that the Pesaro risk score was significantly associated with higher overall survival and thalassemia-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

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Consider Asking Your Patients:

"If you could change a few things about how your treatment affects you, such as in your relationships or personal goals, what would they be? Can you tell me an instance of how treatment has impacted your life?"

woman walking

Actor portrayals throughout. Not real patients.

Take the Beta-Thalassemia Challenge

In a longitudinal cohort of 264 thalassemia patients over age 14 (of which 85.6% had beta-thalassemia), patients reported having lower health‑related quality of life than the normal population in which health domains?


According to a longitudinal cohort of 264 thalassemia patients over age 14 (of which 85.6% had regularly transfused beta-thalassemia), patients reported having significantly lower HRQOL than the normal population in 5 of the 8 SF‑36 health domains.1*