Background: Despite robust advances in understanding of sickle cell disease (SCD) pathophysiology, therapeutic options for SCD patients largely remain limited to hydroxyurea and chronic red blood cell transfusions. And while survival of patients with SCD has improved with these offerings, patients continue to face the impacts of ongoing hemolysis, vasculopathy and vaso-occlusive events leading to national medical care costs exceeding $1 billion annually by some estimates. Allogeneic stem cell transplantation has been shown to cure SCD and is a comparatively cost-efficient alternative to one-time expensive treatments such as gene therapy. While costs associated with allogeneic transplant are roughly $200,000-$400,000 depending upon regimens used and institutional costs incurred, there have been few studies that describe the direct differences in healthcare utilization costs prior to and after transplantation in this population.

Methods: In this study, we performed a single-center evaluation of the pre, and post healthcare utilization costs associated with non-myeloablative haploidentical allogeneic stem cell transplantation (HSCT) in adult patients with SCD at our institution.

We examined the billing records for 22 adult patients that received standard of care and subsequently underwent HSCT for SCD at Johns Hopkins Medical Center between 2018-2025. Lifelong billing data for all care received at our institution was extracted for each patient, and charges were separated into SCD-related and non-SCD related charges. Data such as length of stay per hospitalization, and costs per outpatient and inpatient visits was extracted for each patient both prior to and after HSCT. Pre-transplant costs were defined as all SCD-related healthcare expenses incurred prior to transplant day -30, while post-transplant costs were defined as all SCD-related healthcare expense incurred after transplant day +90. Key utilization metrics were identified based on billing data characteristics, including CPT codes, billing departments of service, and revenue codes.

Results: The median age of transplantation was 26 years. Patients on average had 662 days of available pre-transplant data and 857 days of available post-transplant data. The median total pre-transplant charges were $136,079 per patient equating to a median of $265 per patient per day (standard deviation of $542 per day) compared to median total post-transplant charges of $70,000 per patient or $140 per patient per day (standard deviation of $394). Total pre-transplant inpatient admissions were 1.26 per 100 exposure days compared to total post-transplant admissions of .50 per 100 exposure days. Total pre-transplant emergency room (ER) visits were .78 per 100 exposure days compared to total post-transplant ER visits of .52 per 100 exposure days. Total pre-transplant vaso-occlusive crises (VOC) were 1.85 per 100 exposure days compared to total post-transplant VOC of .58 per 100 exposure days.

Conclusions: Non-myeloablative haploidentical allogeneic stem cell transplantation reduces healthcare utilization costs for patients with sickle cell disease. Despite the risk of complications of the haploidentical approach such as graft versus host disease or delayed engraftment; on average, healthcare utilization trends lower after HSCT, including decreased inpatient admissions, decreased ER visits, and decreased admissions for VOC. The results obtained from our analysis of healthcare utilization costs can be used for further exploration of cost effectiveness of the haploidentical approach to transplant when compared to other curative approaches for adults such as matched related donor transplants and gene therapy. Further analysis remains to be done to characterize what is driving healthcare utilization after transplant, and if the costs incurred after curative therapy are necessary and impactful to the post-transplant course.

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2025
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