Background: In the last decade, options for treatment of sickle cell disease (SCD) have grown remarkably with the development of new disease modifying agents, expanding use of hematopoietic cell transplantation (HCT) and the recent development of gene therapy. Previous reports have evaluated cost effectiveness of various disease modifying agents and have compared costs of HCT and gene therapy to medical management of SCD. These studies have demonstrated a significant upfront cost that varies depending on the stem cell source and conditioning regimen applied, but significant reduction in subsequent cost and healthcare utilization occurs. However, these studies have largely been performed in the United States (US), while the limited studies of HCT costs in other countries report much lower cost for HCT.

The aim of the current study was to compare the costs of transplant for SCD between centers in the US that have a primarily private insurance and utilise myeloablative or reduced toxicity conditioning with a center in Canada that has a universal healthcare plan and employ a non-myeloablative regimen.

Methods Three centers participated in the study, two from the US (Children's Healthcare of Atlanta and St. Jude Children's Research Hospital) and one from Canada (Alberta Children's Hospital, Calgary). Any child or young adult who received an HCT for SCD from a matched sibling donor and consented to data sharing was eligible to take part. Data was collected retrospectively from hospital-based financial datasets. Additionally, chart review of the Canadian participants was performed manually for validation of that dataset. Individuals transplanted from 2013-2022 had encounter level data abstracted to include the 6 weeks prior to HCT and two years post. Data was categorised into groups: clinical care: relating to inpatient and outpatient care visits as well as provider fees; investigation expenses including laboratory and diagnostic imaging costs; and pharmaceutical costs. The overall cost of HCT as well as for each group were compared between countries. Outlier data underwent chart review to determine accuracy. Canadian costs were adjusted to US dollars based on the exchange rate in 2022 at the time of initial analysis.

Results Seventy-seven children were included in the study, 54 from the US and 23 subjects from Canada. Overall, the median cost per patient of HCT for the US sites was $130,604 (IQR $111,667-$2,231,007) while that in Calgary was $107, 925 (IQR $96,447-$131,294) and the difference was not statistically significant (p= 0.15). Comparison of cost categories revealed significant differences in clinical (US: $9,052 [IQR $6,116-$221,383]; Canada: $55,675 [IQR $47,010-$68,707]; p=<0.05), imaging (US: $806 [IQR $275-$14,646]; Canada: $495 [IQR $45-$752]; p=<0.05), and pharmacy costs (US: $39,967 [IQR $32,366-$306,959]; Canada: $6,622 [IQR $5,861-$7,722]; p= <0.05). Laboratory costs were not statistically significant (US $26,320 [IQR $18,945-$1,697,889]; Canada: $31,310 [IQR $28,224-$44,332]).

Conclusions The total cost of HCT for SCD in the US is similar to that in Canada. This suggests that current HCT approaches and/or supportive care in the US may provide a cost reduction that mitigates prior published differences in HCT costs between the two countries. However significant differences in categories of cost remain suggesting opportunities to identify potential inefficiencies and reduce costs further in both countries. In particular, while the conditioning intensity may explain some of the differences, a substantial difference in pharmaceutical and investigative cost was demonstrated. The difference in pharmaceutical expenses may owe to diverse health financing frameworks and pricing systems. Given these updated costs of HCT a more comprehensive present era cost benefit analysis of SCD treatment options is warranted.

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2025
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