small subset of patients with transfusion-dependent beta-thalassemia (TDT) and patients with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) underwent hematopoietic stem cell transplant (HSCT); however, clinical complications were common, resulting in morbidity and high economic burden, according to results of a retrospective study presented at the 2022 ASH Annual Meeting.

“HSCT is a curative option for some patients with TDT or some patients with SCD with recurrent VOCs, but there’s limited research on the outcomes,” Chuka Udeze, PharmD, of Vertex Pharmaceuticals Inc. in Boston, said when presenting the study.

This retrospective study evaluated data from 3734 patients with TDT and 44 with SCD from the Merative MarketScan Commercial, Medicare Supplemental, and Multistate Medicaid Databases. Patients with TDT had 8 unique red blood cell transfusions during a 1-year period. All patients with SCD experienced a minimum of 2 VOCs per year for 2 consecutive years.


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There were 7.7% of patients with TDT and 1.3% with SCD who underwent HSCT. Of these patients, the mean age was 12.6 years (range, 0-45) and 47% of patients were female. There were 74.2% and 25.8% of patients with Medicaid or commercial insurance, respectively. The mean follow-up was 2.9 years.

Clinical complications of HSCT were common. The rate of unspecified complications was 53.2% and transplant rejection/failure occurred among 9.7% of patients. Graft vs host disease developed among 59.7% of patients, of which, 38.7% of cases were acute and 32.3% were chronic.

Healthcare resource utilization was high, with a mean cost of $442,752 within the first 100 days after transplant. The majority of this cost accrued during the inpatient stay. Costs continued to accumulate after 100 days, with a mean of $623,301 by 1 year post transplant. Of this, $552,899 was due to inpatient stays, $50,641 due to outpatient visits, and $19,762 due to outpatient pharmacy.

Dr Udeze concluded that “many patients who receive HSCTs have complications and there is substantial economic burden, and this exemplifies the unmet need for additional curative therapeutic options.”

Disclosures: Some of the study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of disclosures.

Reference

Udeze C, Evans K, Yang Y, et al. Clinical and economic outcomes in patients with transfusion-dependent β-thalassemia and patients with sickle cell disease with recurrent vaso-occlusive crises receiving hematopoietic stem cell transplants in the United States. Presented at ASH 2022. December 10-13, 2022. Abstract 4820.