Economic analyses of bone marrow and blood stem cell transplantation for leukemias and lymphoma: what do we know?

Abstract

Summary:The use of blood and/or bone marrow stem cell transplantation (SCT) grew extensively in the last decade as technological advances led to improved outcomes and wider availability. The first study of SCT costs, however, was not published until 1989. This paper summarizes current knowledge about costs and cost-effectiveness of allogeneic and autologous SCT for leukemias and lymphoma. Methodological issues in cost studies such as types of costs, methods of data collection, and time horizons are discussed, and studies are evaluated with regard to these issues. Considerations specific to economic analyses of SCT are considered, including the potential impact of technological changes, learning curve effects, and inter-institutional differences. Keywords: cost-effectiveness, economics, stem cell transplantation The field of blood and bone marrow transplantation (stem cell transplantation, SCT) has changed dramatically over the past 10 years. Major advances in SCT technology has led to improved outcomes and wider availability. Numbers of SCT have increased from less than 5000 to more than 40 000 annually, and hundreds of papers related to SCT have been published.1 Yet relatively few studies have addressed the costs and cost-effectiveness of this complex technology, although perceived high costs contribute to the controversy over its use.The first comprehensive study of the costs of SCT was reported in 1989 and indicated that, while allogeneic bone marrow transplantation for patients with acute myelogenous leukemia (AML) was very costly, it was more cost-effective than chemotherapy.2 In the past 3 years, with physicians facing increased economic pressures in the United States and abroad, many more studies have included economic analyses. These studies report markedly lower costs of SCT than in 1989 and even more favorable cost-effectiveness profiles for persons with hematological malignancies. In this paper, we provide an overview of economic analyses in general and subsequently summarize studies of costs of SCTs for leukemia and lymphoma.Because the studies we review were conducted utilizing various methodologies and make numerous, differing assumptions regarding cost and clinical parameters, we begin our paper with a proposed framework for reviewing cost analyses (Section 1). We then review some of the 'issues' that our framework highlights, based on the work of others in the field (Section 2).3-8 Section 3 begins with a table summarizing the current literature on costs of SCT using the framework developed in Section 1. Section 3 also discusses the more current, well-designed/presented studies, highlighting the authors' conclusions. Section 4 presents some additional issues to be considered in cost analyses of SCT. A framework for comparing cost studiesThere is growing attention to the costs and cost-effectiveness of medical interventions. Review of the sizable body of literature devoted to the topic reveals conflicting definitions of cost as well as discrepant methods for conducting and reporting studies. The w...

Cost-effectiveness and clinical outcomes of double versus single cord blood transplantation in adults with acute leukemia in France

“…The cost of HSCT has been previously evaluated mainly in the setting of transplants from HLA identical siblings 5 and matched unrelated donors. 6 The reported costs varied greatly, from US$ 60,000 to 200,000.…”

“…In the first published studies, the estimated costs for HSCT varied greatly (Online Supplementary Table S1) depending on the country in which they were performed, type of donor, transplant center procedures and year of transplantation. 5,7,[9][10][11] A more recent comparative study of autologous and allogeneic HSCT for patients transplanted for hematologic malignancies in the USA estimated a 100-day total cost of US$ 203,026 for allogeneic HSCT. 12 Unrelated donor cord blood transplantation (UCBT) has become a widely accepted transplant modality in the absence of an HLA-matched donor.…”

“…It is, however, a complex, resource-intense and costly procedure. [1][2][3][4] The cost of HSCT has been previously evaluated, mainly in the setting of HLA identical sibling 5 and matched unrelated donor transplants.…”

Use of concurrent G-CSF + GM-CSF vs G-CSF alone for mobilization of peripheral blood stem cells in children with malignant disease

“…Although our overall cost for PBPCT is lower than in other reports on the subject, [27][28][29][30] it is very difficult to establish comparisons because of differences in institutions, protocols, diseases and salaries for medical and paramedical personnel. The lower cost may be due to the following factors: (1) the mean weights of our patients were 34 and 36 kg for group I and group II, respectively; (2) we performed single apheresis in most of the patients; and (3) we did not evaluate indirect cost.…”

Why aren't we performing more allografts for aggressive non-Hodgkin's lymphoma?

“…40 While formal cost-utility comparisons are not available, there is no doubt that autoSCT is cheaper than alloSCT, so the use of alloSCT should be restricted to indications where the outcome following autoSCT is likely to be inferior.…”