CLINICAL AND ECONOMIC BURDEN IN PEDIATRIC PATIENTS WITH TRANSFUSION-DEPENDENT BETA THALASSEMIA IN THE UNITED STATES
Author(s)
JIAHE li, MPH1, Arie Barlev, MSc, PharmD1, Siddharth Jain, MS, PharmD1, Kristin Evans, PhD2, Michelle Jerry, MS2, Duncan Brown, BSc, PhD1;
1Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 2Merative, Ann Arbor, MI, USA
1Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 2Merative, Ann Arbor, MI, USA
OBJECTIVES: Beta thalassemia is a rare, inherited blood disorder characterized by chronic hemolytic anemia. Patients with the most serious form, transfusion-dependent beta thalassemia (TDT), require regular, lifelong red blood cell transfusions (RBCTs) to survive. This study described clinical and economic outcomes for pediatric patients aged 5-11 years with TDT in the United States (US).
METHODS: This retrospective cohort study used administrative claims from US MarketScan Commercial, Medicare, and Multi-State Medicaid Databases to identify patients with >1 inpatient or >2 outpatient claims for beta thalassemia or Hemoglobin E-beta thalassemia between January 1, 2014 - December 31, 2024. Patients were considered transfusion dependent if they had at least 8 RBCTs in any 12-month period on or after the date of the earliest qualifying beta thalassemia claim. The index date was specified as the date of the first recorded RBCT. Patients were required to have >12 months of post-index continuous enrollment with medical and pharmacy benefits, be between 5-11 years at the index date, with no evidence of a hematopoietic stem cell transplant or sickle cell disease claims. Baseline demographics, RBCTs, complications and total costs (2024 USD) during the follow-up period were summarized.
RESULTS: Fifty-eight pediatric patients with TDT were identified. Mean patient age was 8.1 (SD: 2.0) years and 51.7% were female; 63.8% of patients were in the Commercial database and 36.2% in the Medicaid database. Mean follow-up time was 4.5 (SD: 2.6) years. Patients received a mean of 14.3 (SD: 4.2) RBCTs per patient per year (PPPY). The most common complications were iron overload/hemochromatosis (94.8%) and infections (37.9%). Mean total cost was $145,684 (SD: $83,937) PPPY.
CONCLUSIONS: From a young age, TDT imposes significant clinical and economic burden, highlighting the need for therapies that reduce healthcare burden and improve pediatric patient outcomes.
METHODS: This retrospective cohort study used administrative claims from US MarketScan Commercial, Medicare, and Multi-State Medicaid Databases to identify patients with >1 inpatient or >2 outpatient claims for beta thalassemia or Hemoglobin E-beta thalassemia between January 1, 2014 - December 31, 2024. Patients were considered transfusion dependent if they had at least 8 RBCTs in any 12-month period on or after the date of the earliest qualifying beta thalassemia claim. The index date was specified as the date of the first recorded RBCT. Patients were required to have >12 months of post-index continuous enrollment with medical and pharmacy benefits, be between 5-11 years at the index date, with no evidence of a hematopoietic stem cell transplant or sickle cell disease claims. Baseline demographics, RBCTs, complications and total costs (2024 USD) during the follow-up period were summarized.
RESULTS: Fifty-eight pediatric patients with TDT were identified. Mean patient age was 8.1 (SD: 2.0) years and 51.7% were female; 63.8% of patients were in the Commercial database and 36.2% in the Medicaid database. Mean follow-up time was 4.5 (SD: 2.6) years. Patients received a mean of 14.3 (SD: 4.2) RBCTs per patient per year (PPPY). The most common complications were iron overload/hemochromatosis (94.8%) and infections (37.9%). Mean total cost was $145,684 (SD: $83,937) PPPY.
CONCLUSIONS: From a young age, TDT imposes significant clinical and economic burden, highlighting the need for therapies that reduce healthcare burden and improve pediatric patient outcomes.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE295
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
SDC: Rare & Orphan Diseases, SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)