Clinical burden and healthcare resource utilization after allogeneic hematopoietic stem cell transplant for sickle cell disease or β-thalassemia

INTRODUCTION: Allogeneic hematopoietic stem cell transplant (allo-HSCT) is a potentially curative treatment for patients with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or β-thalassemia. Real-world data on post-allo HSCT outcomes are limited among these patients. METHODS: Patients with SCD with recurrent VOCs or β-thalassemia with allo-HSCT were identified in the Merative MarketScan Research Databases. Clinical outcomes, allo-HSCT complications, and healthcare resource utilization were summarized after allo-HSCT. RESULTS: Eighty-one patients with SCD with recurrent VOCs and 33 with β-thalassemia were included. About one-third of patients with SCD experienced a VOC and one-quarter of patients with β-thalassemia required a red blood cell transfusion, in the year after the first 100 days following allo-HSCT. Approximately 40.7% of patients with SCD and 42.4% of patients with β-thalassemia developed acute graft-versus-host disease (GvHD), while 32.1% and 27.3% developed chronic GvHD, respectively. Patients with SCD had a mean of 74.1 (59.6) outpatient visits per patient per year and patients with β-thalassemia had a mean (SD) of 76.1 (61.3). LIMITATIONS: Due to the lack of International Classification of Diseases, 10th Revision (ICD-10) codes to distinguish between different types of allo-HSCT, this study was unable to assess outcomes of patients with haploidentical hematopoietic stem cell transplantation (haplo-HSCT), which is a type of allo-HSCT. CONCLUSION: Many patients with SCD with recurrent VOCs or β-thalassemia endure potentially life-threatening transplant-related complications and require substantial healthcare utilization following allo-HSCT. These findings suggest limitations of allo-HSCT and underscore the need for alternative curative treatment options.