BACKGROUND: β-thalassemia is a potentially life-threatening, hereditary blood disorder characterized by ineffective erythropoiesis and peripheral hemolysis, leading to profound anemia and iron avidity. It is estimated to affect approximately 15,000 people in the U.S. and European Union. Patients with the most severe form of the disease, transfusion-dependent β-thalassemia (TDT), must adhere to a lifelong regimen of frequent red blood cell (RBC) transfusions for survival. While regular transfusions effectively manage the symptoms of TDT, they also substantially accelerate iron overload. As a result, patients with TDT must also adhere to lifelong chelation therapy regimens to reduce iron-associated morbidity and mortality. A variety of chelation therapies are available in the U.S., and treatment regimens should be optimized for the specific needs of individual patients. While the burden of chronic treatment on patients is clear, there are limited data available on economic costs of RBC transfusions and chelation therapy in the treatment of TDT in the U.S.

OBJECTIVES: Objectives of this study were to: 1) develop an algorithm to identify patients with TDT in a U.S. administrative claims database; 2) evaluate demographic characteristics of these patients; 3) examine treatment patterns with RBC transfusions and chelation therapy; and 4) estimate the annual reimbursed costs associated with the use of RBC transfusions and chelation therapy in these patients.

METHODS: This retrospective analysis used the U.S. MarketScan Commercial and Medicaid Multi-State Database, which together represent the administrative inpatient and outpatient medical, and outpatient pharmacy experience of approximately 30 million (in 2016) commercially-insured employees and their dependents, and Medicaid beneficiaries. These individuals are covered under a variety of fee-for-service and managed care health plans.

Patients with TDT included in this study were required to have ≥ 8 RBC transfusions in a given 12-month period (first transfusion date = index date) between October 1, 2011 and September 30, 2016, one or more non-diagnostic inpatient or outpatient medical claims with a β-thalassemia or hemoglobin E β-thalassemia diagnosis between 3 months before and 15 months after the index date, and no evidence of allogeneic stem cell transplantation between the first and eighth transfusions. Demographic characteristics of the TDT patients were summarized. The annual number of RBC transfusions and prescriptions for chelation therapy were summarized on a per-patient basis, as well as the annual costs (i.e. reimbursed amounts) for these therapies. Additional costs associated with management of patients with TDT (e.g. ongoing monitoring of iron overload with cardiac T2* MRI and liver iron tests) are being estimated in the study but are not included in this abstract.

RESULTS: A total of 8,480 patients with a diagnosis of β-thalassemia were identified, of which 253 patients (3.0%) met the selection criteria for inclusion in this analysis. Mean patient age was 22.8 years (median 21 years) and 53.4% were female; 33.2% lived in the Northeast, 25.7% in the Midwest, 24.9% in the South, and 16.2% in the West. Patients with TDT in this analysis had a median of 14 blood transfusions annually (range 8-56), with an average cost of $1,478 per transfusion. The mean annual cost of transfusions per patient was$22,478. The majority of patients (84.1%) had ≥ 1 prescription for chelation therapy, with 73.8% receiving an oral chelation therapy regimen only, 7.8% IV only, and 18.4% combination therapy. On average, chelation therapy cost $52,718 per patient, per year. CONCLUSION: In the U.S., the average annual economic cost of TDT conventional chronic symptom management with RBC transfusions and iron chelation therapy is approximately$75,000. Treatment with both transfusion and chelation are life-long requirements for most patients with TDT; thus the cumulative economic burden for basic management of TDT is substantial. Furthermore, these costs represent only a portion of the total economic burden of TDT, which also includes the costs of ongoing monitoring of cardiac and other organ iron overload, costs associated with morbidities related to TDT and iron overload, and the significant time burden for patients (e.g. to meet multi-disciplinary clinical teams including endocrinologists, cardiologists, etc.; and transfusion appointments).

Disclosures

Paramore: bluebird bio: Employment, Equity Ownership. Vlahiotis: Truven Health Analytics, an IBM Watson Health company: Employment. Moynihan: Truven Health Analytics, an IBM Watson Health company: Employment. Cappell: Truven Health Analytics, an IBM Watson Health company: Employment. Ramirez-Santiago: bluebird bio: Employment, Equity Ownership.

## Author notes

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Asterisk with author names denotes non-ASH members.