Time and Side-Effect Burden Associated with Red Blood Cell Transfusions in Patients Diagnosed with Transfusion-Dependent β-Thalassemia

Introduction: β-thalassemia is a genetic blood disorder marked by the inability to produce hemoglobin. Diagnosed primarily in childhood, transfusion-dependent β-thalassemia (TDT) is the most severe form of this disorder and results in chronic anemia, requiring frequent red blood cell (RBC) transfusions to maintain hemoglobin levels. While previous studies have shown that health-related quality of life (HRQoL) is adversely affected in patients with TDT, the issues associated with the transfusion process itself in these patients (eg, side effects and time requirements) have not always been examined. These issues are the focus of this study.

Methods: Following exploratory qualitative interviews with a small sample of patients identified by the Cooley's Anemia Foundation (CAF), a 30-minute web-based survey was developed to assess HRQoL variables in TDT. Subsequently, CAF identified patients in the US who were ≥ 18 years of age, had a self-reported physician diagnosis of β-thalassemia, and had received ≥ 1 RBC transfusion in the past 6 months, and invited them to participate in the study. Following informed consent, patients completed a number of validated and ad hoc questions about their treatment, side effects, psychological burden, time requirements for a transfusion, and caregiver information. Data were collected in May 2021. Descriptive results are presented as frequencies, means, and standard deviations (SD).

Results: A total of 100 patients were identified and recruited by CAF and completed the survey. Of these, 35 (35%) were male, with a mean age of 36.0 (SD = 10.4) years. Patients had been diagnosed with TDT for a mean of 34.4 years (SD = 10.3) and had received a mean of 9.6 (SD = 4.3) RBC transfusions in the previous 6 months. In addition to receiving RBC transfusions (100%), other treatments ever received for TDT included iron chelation therapy (94%), surgery (47%), and stem cell/bone marrow transplantation (5%). Regarding RBC transfusions, 66% of patients reported requiring them more than once per month, 31% each month, and 3% every other month. Common patient-reported side effects of RBC transfusions were iron overload (81%), rashes/hives (74%), fatigue (61%), and pain/bruising at the injection site (52%). Additionally, 86% of patients reported feeling fatigued leading up to RBC transfusions, 75% reported that treatments for TDT had become routine, and 64% accepted the routine. Time required for an RBC transfusion was estimated at a mean of 7.4 hours (SD = 11.3) including transfusion preparation time. Travel and wait times were estimated as requiring an additional 7.1 hours. Other time requirements included frequent outpatient visits and frequent laboratory tests, averaging 3.9 visits and 6.1 visits in a 6-month period, respectively. Overall, 13% of patients reported having a caregiver. Caregiver activities included travel to and from appointments (77%), help with household duties (77%), and assistance in treatment decisions (62%).

Conclusions: The results of this survey suggest there is a substantial time and side-effect burden associated with transfusion and treatment among patients diagnosed with TDT. This includes considerable time devoted to preparing for and receiving transfusions, transfusion-related side effects, frequent laboratory and outpatient appointments, and help from a caregiver. Medical treatments that would decrease the dependency on transfusions in TDT could decrease this burden and improve overall quality of life.