Burden of Illness Among Patients With Transfusion Dependent Beta-Thalassemia in Spain: A Registry-Based Study
Author(s)
RAJESHWARI NAIR, MPH, PhD, Other1, Nanxin Li, MBA, PhD1, Jose Manuel Marco Sanchez, MD2, Sara Alonso, PhD1, Nieves Dorado, MD1, Qing Liu, MD, PhD3, Hongbo Yang, BA, MA, PhD3, Elena Cela, MD2.
1Vertex Pharmaceuticals, Inc., Boston, MA, USA, 2Hospital General Universitario Gregorio Marañón, Madrid, Spain, 3Analysis Group, Boston, MA, USA.
1Vertex Pharmaceuticals, Inc., Boston, MA, USA, 2Hospital General Universitario Gregorio Marañón, Madrid, Spain, 3Analysis Group, Boston, MA, USA.
OBJECTIVES: Patients with transfusion-dependent β-thalassemia (TDT) require lifelong care, including frequent red blood cell transfusions (RBCTs) that lead to excess tissue iron, end organ damage, and the need for regular iron chelation therapy (ICT) for survival. A registry-based study was conducted to assess the burden of illness among patients with TDT in Spain.
METHODS: A retrospective cohort study was conducted using data from the Spanish Registry of Hemoglobinopathies and Rare Anemias of the Spanish Society of Hematology Pediatric Oncology-Spanish Hematology and Hemotherapy Society (SEHOP-SEHH). Patients with a diagnosis of TDT were identified between January 1, 2014 and December 31, 2021. Patients were required to be 12 years or older at study inclusion, with at least 1 year of follow-up data. Patients with hereditary persistence of fetal hemoglobin, α-thalassemia, sickle cell disease, or hematopoietic stem cell transplant were excluded. Patients were followed until the earliest occurrence of death, loss to follow-up, or the end of the study period (December 31, 2022). Demographics, acute and chronic complications, and treatment use were descriptively summarized.
RESULTS: A total of 44 patients with TDT met the study eligibility criteria. The mean age at study inclusion was 14.0 years (standard deviation [SD]: 2.0), and 55.0% were male. The mean duration of follow-up was 26.0 years (SD: 2.4). Patients received an average of 21.7 RBCTs per-patient-per-year (SD: 6.0) during follow-up. Most patients (95.0%) received ICT to manage their iron overload. The most frequent chronic complications were transfusion-related iron overload (70.5%), hepatobiliary complications (70.5%), bone and joint complications (43.0%), endocrine complications (25.0%), and gallstones (22.5%).
CONCLUSIONS: Patients with TDT in Spain experience substantial burden from serious chronic complications on standard of care with frequent RBCTs and ICT for TDT management, highlighting the need for novel therapies for these patients.
METHODS: A retrospective cohort study was conducted using data from the Spanish Registry of Hemoglobinopathies and Rare Anemias of the Spanish Society of Hematology Pediatric Oncology-Spanish Hematology and Hemotherapy Society (SEHOP-SEHH). Patients with a diagnosis of TDT were identified between January 1, 2014 and December 31, 2021. Patients were required to be 12 years or older at study inclusion, with at least 1 year of follow-up data. Patients with hereditary persistence of fetal hemoglobin, α-thalassemia, sickle cell disease, or hematopoietic stem cell transplant were excluded. Patients were followed until the earliest occurrence of death, loss to follow-up, or the end of the study period (December 31, 2022). Demographics, acute and chronic complications, and treatment use were descriptively summarized.
RESULTS: A total of 44 patients with TDT met the study eligibility criteria. The mean age at study inclusion was 14.0 years (standard deviation [SD]: 2.0), and 55.0% were male. The mean duration of follow-up was 26.0 years (SD: 2.4). Patients received an average of 21.7 RBCTs per-patient-per-year (SD: 6.0) during follow-up. Most patients (95.0%) received ICT to manage their iron overload. The most frequent chronic complications were transfusion-related iron overload (70.5%), hepatobiliary complications (70.5%), bone and joint complications (43.0%), endocrine complications (25.0%), and gallstones (22.5%).
CONCLUSIONS: Patients with TDT in Spain experience substantial burden from serious chronic complications on standard of care with frequent RBCTs and ICT for TDT management, highlighting the need for novel therapies for these patients.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO35
Topic
Clinical Outcomes, Epidemiology & Public Health
Disease
Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)