Whither the bone marrow transplant?

Abstract

Bone marrow transplantation (BMT) has become an accepted and important medical intervention which has become a routine part of medical practice. Its utility has, however, been questioned recently in a number of diseases in which its role has been clearly established on the basis that there are better non-transplant therapeutic options. The suspicion that these moves to eradicate BMT as an option may not stem from purely scientific reasons has prompted the preparation of these personal reflections. I will focus this discussion only on two diseases in which BMT has been shown to be useful: chronic myelogenous leukemia (CML) and multiple myeloma (MM).

Quantitative and qualitative differences in use and trends of hematopoietic stem cell transplantation: a Global Observational Study

“…They point to the different requirements within high or low income countries, hence different cost-effectiveness considerations. 20,21,[26][27][28] Unrelated donor transplant rates were associated with GNI/capita, the presence of an unrelated donor registry and the number of registered donors. The association is likely reciprocal; high income countries perform more HSCT in general and are more likely to invest in an unrelated donor registry.…”

“…17,[22][23][24] Studies on macro-economic aspects or on cost-effectiveness in individual countries have gained broader acceptance only recently. 11,21,22,25 They were triggered in part by some rapid changes in the use of HSCT, such as for breast cancer or chronic myeloid leukemia 18,26 and by the rising awareness of the disturbing gap between unlimited requests and limited resources in any health care system. 27,28 Availability of resources, governmental support and access to therapy were identified as factors associated with use; availability of resources, evidence, external regulations and positive or negative expectations of transplant physicians as factors associated with diffusion.…”

“…There is no need for intensive high cost pre-treatment as is the case for patients with acute leukemia and, the search for a matched family donor requires minimal resources. [17][18][19][20][21] The economic aspects of HSCT with its patient-centered approach have traditionally concentrated on costs of the individual procedure for an individual patient. 17,[22][23][24] Studies on macro-economic aspects or on cost-effectiveness in individual countries have gained broader acceptance only recently.…”

Stem Cell Transplantation Procedures Are Becoming Affordable for Individuals Living in Developing (Middle-Income) Countries

“…As examples, it should be mentioned that in some middle-income countries, in the case of chronic myelogenous leukemia, it is more affordable to conduct a simplified allo-SCT than to use tyrosine kinase inhibitors continuously, with similar long-term results [9,10], and that in the case of multiple myeloma conducting a simplified auto-SCT is more affordable than the prolonged use of novel antimyeloma drugs [4,9]. These observations, which may be irrelevant in high-income countries, are critical in underprivileged circumstances of economic restrictions [2,9] and should be used to define health care policies.…”

Whither the Bone Marrow Transplantation in Multiple Myeloma?

“…However, its utility in a number of diseases has been questioned recently on the basis that there are better nontransplant therapeutic options. The question of whether these moves to eradicate bone marrow transplantation as an option stem from purely scientific reasons has been raised [1]. Front-line treatment of high-dose melphalan and autologous stem cell transplantation (ASCT) has improved the prognosis of patients with multiple myeloma (MM).…”