In a retrospective study, we evaluated the cost and cost-effectiveness of

In a retrospective study, we evaluated the cost and cost-effectiveness of allogeneic peripheral blood stem cell transplantation (PBSCT) (n=30) compared with bone marrow transplantation (BMT) (n=110) in children with acute leukemia at 1 year of follow up. the treatment success rate was 18.8% for PBSCT patients and 23.5% for BMT (P=NS). The cumulative average cost for patients in the BMT group was $457,078 compared to $377,316 for PBSCT (P=NS). Point estimates of the incremental cost-effectiveness ratio (ICER) show that allogeneic transplantation of bone marrow grafts is usually dominant over PBSCT for its lower costs and higher effectiveness in patients with standard risk disease (ICER = ?$687,108; 95% CI = BIBR 953 distributor $2.4 million to dominated). For patients with high risk disease, BMT was more effective and more costly and the ICER was $1.69 million (95% CI = $29.7 million to dominated) per additional treatment success. The comparative economic evaluation provides support for BMT for standard risk patients, but a BIBR 953 distributor great degree of uncertainty limits the obvious advantage for either treatment option in patients with high risk disease. Further research using larger and randomized controlled trials will be required to confirm the long term cost-effectiveness of each procedure. strong class=”kwd-title” Keywords: children, acute leukemia, allogeneic stem cell transplantation INTRODUCTION Improvements in stem cell transplantation (SCT) technology have improved the outcome and increased the availability of the technique, encouraging its use as a front-line treatment for many severe malignant and non-malignant disorders. Although bone marrow (BM) was initially, the exclusive source of stem cells, peripheral blood stem cells (PBSC) have become an increasingly favored alternative. Indeed, they have now largely superseded bone marrow as the source of cells in autologous transplantation, due to preferable clinical outcomes, such as faster engraftment kinetics, and shorter hospitalization [1C3]. While allogeneic Rabbit polyclonal to HHIPL2 PBSC produce comparable benefits in the allogeneic setting, these are partially offset by the association of this source of stem cells with an increased risk of graft-versus-host disease (GvHD), which occurs even after recent improvements in GvHD prophylaxis and in molecular techniques for establishing donor-recipient histocompatibility [4C10]. Consequently it is uncertain whether PBSC are preferable to BM cells overall for use as allografts. In practice, bone marrow continues to be the main stem cell source for matched sibling donor and matched unrelated donor transplantations, while PBSC is usually more widely used for haploidential transplantation, since this permits an increased dose of stem cells to be given, with apparently beneficial effects on engraftment [11C13]. Umbilical cord blood is a third source of stem cells for allogeneic SCT and may reduce the incidence of severe GvHD compared to the alternatives [14]. The limitations of umbilical cord blood are, however, significant, including greatly delayed time to hematological recovery and increased risk of contamination, both of which lead to higher overall costs [15]. Although allogeneic SCT with blood or marrow stem cells offers the prospect of a permanent remedy, few studies have addressed the overall costs and cost-effectiveness of the procedure or compared the cost effectiveness of these stem cell sources. Previous adult studies have compared the cost-effectiveness of each source, but little is known about cost comparisons in the pediatric populace [16C20]. This is a significant deficiency, since the disease mix in the pediatric versus the adult populace (e.g. ALL AML), the biology of the diseases, and the risks of severe GvHD are all strikingly different from adult cohorts. This may result in different predisposition to post transplant complications and different overall outcomes and hence cost effectiveness assessments. We now compare the costs and cost-effectiveness of allogeneic peripheral blood stem cell transplantation (PBSCT) and bone marrow transplantation (BMT) BIBR 953 distributor in pediatric patients with acute leukemia. We examined the one-year post transplant economic implications of PBSCT versus BMT treatment by stratifying patients disease status in a retrospective study derived from a single institution in which patients received their main transplant from 2001 to 2006. PATIENTS AND METHODS Patients We studied children (range 0 years C 18 years) with acute leukemia, who received allogeneic PBSCT or BMT between January 1st, 2001 and September 30th, 2006 in the Stem Cell Transplant.