Busulfan in Hematopoietic Stem Cell Transplantation

Abstract

The development of intravenous busulfan and its incorporation in the preparative regimens for allogeneic stem cell transplantation has changed transplantation for myeloid maligancies. Bypassing the oral route to achieve 100% bioavailability translated into improved control over drug administration, with increased safety and reliability of generating therapeutic busulfan levels, maximizing antileukemic efficacy. Busulfan-nucleoside analog-based conditioning chemotherapy, thus far represented by fludarabine, is becoming the conditioning chemotherapy regimen of choice for patients with acute myelogenous leukemia at many transplant centers. The use of busulfan-based conditioning is extending rapidly also to hematopoietic stem cell transplantation for lymphoid malignancies, genetic diseases and umbilical cord blood transplantation.

Busulfan–melphalan in high-risk neuroblastoma: the 30-year experience of a single institution

“…Patient characteristics are described in Table 1. Before HDC, patients received a median number of six courses of conventional chemotherapy (range [4][5][6][7][8][9][10][11][12][13][14], over a median duration of 6 months (range 3-23). Good and poor responses after the first line of conventional chemotherapy were obtained in 73 (34%) and 142 (66%) patients, respectively.…”

A pilot study of reduced toxicity conditioning with BU, fludarabine and alemtuzumab before the allogeneic hematopoietic SCT in children and adolescents

“…BU-based conditioning is extending rapidly also to HSCT for lymphoid malignancies, genetic diseases and umbilical cord blood transplantation (UCBT). 1 RTC HSCT in children may be associated with reduction in TRM and long-term late complications, as well as length of hospitalization, use of blood products, and risk of infections. [3][4][5]7 In the malignant setting, RTC HSCT eradicates malignant cells through a graft vs malignancy effect provided by alloreactive donor T-lymphocytes and/ or natural killer cells.…”

“…[1][2][3] RTC is defined as a regimen associated with myeloablation but associated with reduced toxicity secondary to conditioning. We and others have demonstrated RTC HSCT regimens to be safe and efficacious in a wide range of hematologic malignancies.…”

Immunological Considerations for Inducing Skin Graft Tolerance

“…Several investigators have reported the induction of allogeneic bone marrow chimerism without myeloablation (Sykes, 1996, Pan et al, 2003, Fuchimoto et al, 2000, Shapira et al, 2003, Matthews et al, 2004, Ciurea and Andersson, 2009, Przepiorka et al, 1999. Myeloablation refers to the massive radiation induced destruction of the host's bone marrow cells, especially the T cell lineages, and the use of allogeneic bone marrow to replace the destroyed cells.…”

“…Fludarabine has been reported to minimize the development of neoplasias as well as to suppress metabolism (Matthews et al, 2004). Busulfan has been described as a myeloablation agonist that has been used to engraft bone marrow and stem cells (Ciurea andAndersson, 2009, Przepiorka et al, 1999). Eight percent of the patients developed full chimerism as evidence by donor origin hematopoietic cells.…”