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Published on: December 8, 2023
Costs and cost-effectiveness of allogeneic stem cell transplantation in children are predictable
Susanne Matthes-Martin1, Ulrike Pötschger, Ronald Barr
1Department of Pediatrics, St Anna Children's Hospital, Medical University of Vienna, Vienna, Austria. susanne.matthes@stanna.at
Insights
The total cost of pediatric stem cell transplantation (SCT) varies widely, influenced by patient age, disease status, and donor type. Despite high costs, SCT remains cost-effective for life-threatening conditions.
Area of Science:
- Pediatric Hematology/Oncology
- Health Economics
- Transplantation Medicine
Background:
- Pediatric stem cell transplantation (SCT) is a critical treatment for various life-threatening conditions.
- Understanding the economic implications of SCT is essential for resource allocation and healthcare policy.
- Previous cost analyses for pediatric SCT are limited, particularly regarding comprehensive cost-effectiveness evaluations.
Purpose of the Study:
- To determine the overall costs associated with pediatric stem cell transplantation (SCT), including donor search and first-year post-transplant expenses.
- To analyze the correlation between SCT costs and patient/transplantation characteristics, as well as a risk score for transplantation-related mortality.
- To evaluate the cost-effectiveness of pediatric SCT, calculating costs per surviving patient and per life-year gained.
Main Methods:
- A cohort of 141 pediatric patients undergoing SCT at a single institution was analyzed.
- Costs were systematically collected, encompassing donor search and the first year post-SCT.
- Statistical analyses correlated costs with patient demographics, disease status, donor type, and a validated transplantation-related mortality risk score.
Main Results:
- The median overall cost for pediatric SCT was €136,382, with significant variability (€26,897 to €601,348).
- Increased costs were associated with older patient age, use of non-matched sibling donors, and advanced disease.
- Costs strongly correlated with the transplantation-related mortality risk score, rising from €89,550 (score 0) to €274,915 (score 3).
- Cost-effectiveness decreased with higher risk scores, with costs per survivor ranging from €93,209 to €1,216,348.
Conclusions:
- Pediatric SCT costs exhibit substantial variation, predictable by patient age, disease, and donor type.
- The cost-effectiveness of pediatric SCT is within acceptable ranges for severe hemato-oncologic diseases, even for high-risk patients.
- These findings provide valuable economic data for healthcare providers and policymakers involved in pediatric transplantation.
Abstract:
The overall costs of pediatric stem cell transplantation (SCT), including donor search and costs during the first year post-SCT, were calculated in a cohort of 141 consecutive children undergoing SCT in a single institution. Costs were correlated with patient and transplantation characteristics and with a risk score for transplantation-related mortality. Cost-effectiveness was calculated based on the overall cost per surviving patient. Life-years gained were extrapolated from overall survival, and the costs per expected life-year gained were calculated. The overall median cost was €136,382 (175,815$), with a wide range, of €26,897 (34,679$) to €601,348 (775,343$). Increased costs were significantly associated with age, use of donors other than matched siblings, and advanced disease. There was a strong correlation of costs with a simple transplantation-related mortality risk score; median total costs were €89,550 (115,463$) for a score of 0, €127,349 (164,179$) for a score of 1, €156,578 (201,861$) for a score of 2, and €274,915 (354,499$) for a score of 3 (P < .001). Cost-effectiveness decreased with increasing transplantation-related mortality risk score; costs per survivor increased from €93,209 (120,200$) for a score of 0 to a maximum of €1,216,348 (1,568,579$) for a score of 3. Costs associated with pediatric SCT vary substantially; however, the combination of variables such as age, disease, and donor type is predictive of costs and cost-effectiveness. Costs per life-year gained are within the broadly accepted range in life-threatening hemato-oncologic diseases, even in the most cost-intensive patient cohort.
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