Costs and Cost-Effectiveness of Allogeneic Stem Cell Transplantation in Children Are Predictable

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.

Total Body Irradiation–Based Myeloablative Haploidentical Stem Cell Transplantation Is a Safe and Effective Alternative to Unrelated Donor Transplantation in Patients Without Matched Sibling Donors

“…Total costs, including costs for donor search and graft acquisition, have been reported to be 50% to 60% higher after MUD versus sibling donor transplantations [40,45]. In the study by van Agthoven et al [40], average 2-year costs, including donor identification expenses per patient undergoing transplantation (in Euros), were V98,334 (sibling bone marrow transplantation), V98,977 (sibling PBSC transplantation), and V151,754 (MUD), representing a 53% increase in costs for unrelated donor versus sibling donor transplantation.…”

Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel

“…These data compare to the costs of HSCT from sibling donors for non-malignant diseases of US$ 112,000-150,000 US, which would translate into US$ 1900 US/expected life year in case of HSCT in infancy. 101 In conclusion, for both TM and SCD, HSCT is cost-effective in comparison to life-long supportive therapy.…”

Challenges to the Sustainability of the U.S. Public Cord Blood System

“…As expected, patients at more-advanced stages of disease tend to have higher treatment costs, but whether this is due to some aspect of the cord blood transplant or simply due to the fact that sicker patients may require more post-transplant care is unclear. 92,99,100 Among pediatric patients, one study found that older children (above age ten) had higher costs, but overall, pediatric patients also tend to be more costly. 100,101 Costs also varied by the condition for which the patient was being treated, but all these factors are likely to affect decisions about conditioning, graft type, and HSC source, as well as health care setting.…”

“…92,99,100 Among pediatric patients, one study found that older children (above age ten) had higher costs, but overall, pediatric patients also tend to be more costly. 100,101 Costs also varied by the condition for which the patient was being treated, but all these factors are likely to affect decisions about conditioning, graft type, and HSC source, as well as health care setting. Moreover, HSC transplants in inpatient settings will typically be reimbursed based on the episode, which may include a risk adjustment for patientlevel factors.…”