Bone Marrow Transplantation, (1998) 21, 641–650  1998 Stockton Press All rights reserved 0268–3369/98 $12.00 Perspective Economic analyses of bone marrow and blood stem cell transplantation for leukemias and lymphoma: what do we know? TM Waters 1,2,3 , CL Bennett 1,2,4 , TS Pajeau 4 , KA Sobocinski 5 , JP Klein 5 , PA Rowlings 5 and MM Horowitz 5 1 Institute for Health Services Research and Policy Studies, Northwestern University, Evanston and Chicago, IL; 2 Lurie Cancer Center, Northwestern University, Chicago, IL; 3 Midwest Center for Health Services and Policy Research, Hines VA Hospital, Hines, IL; 4 Chicago VA Health System, Lakeside Division, Chicago, IL; USA, and 5 Statistical Center of the International Bone Marrow Transplant Registry/Autologous Blood and Marrow Transplant Registry, and the Health Policy Institute, Medical College of Wisconsin, Milwaukee, WI, USA Summary: The use of blood and/or bone marrow stem cell trans- plantation (SCT) grew extensively in the last decade as technological advances led to improved outcomes and wider availability. The first study of SCT costs, how- ever, was not published until 1989. This paper summar- izes current knowledge about costs and cost-effective- ness 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-effec- tive than chemotherapy. 2 In the past 3 years, with phys- icians facing increased economic pressures in the United Correspondence: Dr TM Waters, Institute for Health Services Research and Policy Studies, Northwestern University, 629 Noyes Street, Evanston, Illinois 60208, USA Received 29 January 1997; accepted 31 October 1997 States and abroad, many more studies have included econ- omic analyses. These studies report markedly lower costs of SCT than in 1989 and even more favorable cost-effec- tiveness profiles for persons with hematological malig- nancies. 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 stud- ies, highlighting the authors’ conclusions. Section 4 presents some additional issues to be considered in cost analyses of SCT. 1 A framework for comparing cost studies There is growing attention to the costs and cost-effective- ness of medical interventions. Review of the sizable body of literature devoted to the topic reveals conflicting defi- nitions of cost as well as discrepant methods for conducting and reporting studies. The work of the Panel on Cost-Effec- tiveness in Health and Medicine 5–8 represents a consensus of field leaders, convened by the US Public Health Service, on guidelines for conducting studies as well as a format for presenting results. The Panel’s recommendations were recently published in JAMA. 5–7 We used these guidelines and similar work by others to develop a framework for reviewing cost studies in SCT (Table 1). 3–8 While some of the standardization advocated in this and other papers may still be the subject of discussion, at a minimum, Table 1 provides a unifying framework with which one may compare studies. Once the salient aspects of a study are presented and/or compared, the reader can conduct his or her own evaluation of the weight of the findings.