Abstract
The discussion around the cost of allogeneic hematopoietic stem cell transplantation and follow-up of patients, methods of calculation and cost structure, despite the breadth of application of hematopoietic stem cell transplantation in oncohematological practice, remains an unresolved problem. The discussion is complicated using various modifications of therapeutic options regarding the preparation of the patient for surgery and the prevention of complications. The purpose of this article is a comparative assessment of the clinical and economic efficiency of the two most used technological platforms – TCRαβ/CD19 depletion and ‘’in vivo’’ depletion with high doses of cyclophosphamide, having studied the features of each of these methods in the real practice of one center. Materials and Methods. For evaluation, two “match paired” groups of children were selected, who underwent transplantation according to one of the methods for hemoblastosis in the period from May 2013 to January 2021 at the National Medical Research Center for Pediatric Hematology, oncology and immunology named after Dmitry Rogachev" of the Ministry of Health of Russia. Based on the obtained indicators of clinical effectiveness (significance of differences was made using the nonparametric Mann-Whitney test), pharmacoeconomic modeling was carried out on the basis of the Markov model, the cost structure was determined and the pharmacoeconomic analysis "cost-effectiveness" was performed, incremental analysis "cost-effectiveness" ”, as well as the calculation of indicators of the cost of the operation after 5 years, taking into account discounting. The results of overall and event-free survival, as well as survival without the development of graft-versus-host disease and relapse were used as endpoints. The costs are divided into 2 time periods: stage 1 – up to 30 days after HSCT and stage 2 – from 31 days after HSCT and up to 1 year. Costs are divided into direct and indirect. For indirect costs, the average figures of the available socio-economic data are taken. Results of the study: The total costs for the two stages were comparable for both platforms: TCRa/b/CD19 deletions – 6702.094 thousand rubles; PtCy – 7325.661 thousand rubles. The costs are unevenly distributed over the stages: the 1st stage is more expensive for the TCR platform, the second for PtCy. In cost-effectiveness analysis, each unit of efficiency (1% survival per 1 year of curation will cost more for the PtCy platform. Given the WHO recommended discount factor of 3% per year, over 5 years, the annual cost for the TCR method should decrease to 5933.316 thousand rubles, and for the PtСy platform up to 6,485.355 thousand rubles without considering macroeconomic indicators. Each additional unit of efficiency (1% survival rate) of TCR will cost: survival rate of 207 855.67 rubles, for survival without GVHD and recurrence of 51 963.92 rubles. Conclusions. Pharmacoeconomically less costly for a period of 1 year of curation is the preventive TCR platform compared to the depletion platform ''in vivo'' using high doses of cyclophosphamide, despite the higher cost of its use at the 1st stage of curation.
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