Nelson Hamerschlak

Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study.

Patients with refractory severe aplastic anemia (SAA) who lack a matched sibling or unrelated donor need new therapeutic approaches. Hematopoietic SCT (HSCT) using mismatched or haploidentical related donors has been used in the past, but was associated with a significant risk of GVHD and mortality. Recently, the use of post-transplant cyclophosphamide (Cy) has been shown to be an effective strategy to prevent GVHD in recipients of haploidentical HSCT, but the majority of reports have focused on patients with hematology malignancies. We describe the outcome of 16 patients who underwent haploidentical transplantation using a reduced-intensity conditioning regimen with post-transplant Cy. Stem cell sources were BM (N=13) or PBSCs (N=3). The rate of neutrophil engraftment was 94% and of platelet engraftment was 75%. Two patients had secondary graft failure and were successfully salvaged with another transplant. Three patients developed acute GVHD being grades 2–4 in two. Five patients have died and the 1-year OS was 67.1% (95% confidence interval: 36.5–86.4%). In our small series, the use of a reduced-intensity conditioning with post-transplant Cy in haploidentical BMT was associated with high rates of engraftment and low risk of GVHD in patients with relapsed/refractory SAA.

Identification of genes located at breakpoints of uncharacterized chromosomal translocations by the use of chromosomal microdissection and next generation DNA sequencing

Background Cancer results from accumulation of pathogenic genetic changes in a somatic cell. Chromosomal translocations (CT) are among the most common molecular changes associated with the development of cancer. The mechanism by which CT causes cancer is through the juxtaposition of two genes, generating a hybrid protein with oncogenic function or through the placing of a protooncogene under the control of a promoter region active in the cell of origin. While most genes located at the breakpoints of common CT have been identified, several, less frequent, CT remain uncharacterized.

Stem cell transplantation for acute myeloid leukemia (AML): The Brazilian experience

6725 Background: Data from the International Bone Marrow Transplant Registry (IBMTR) contribute for improvement of Bone Marrow Transplant (BMT) worldwide. We studied the Brazilian experience in BMT for AML to compare with international data. Methods: We performed a retrospective study by sending questionnaires to 14 BMT centers regarding clinical and treatment variables. Statistical analyses concerning autollogous BMT (autoBMT) and allogeneic BMT (alloBMT) were performed using the Kaplan-Meier method and the log-rank test. All p values were two-tailed. Results: We collected data from 726 pts (205 autoBMT and 521 alloBMT). Median overall survival (OS) for autoBMT pts was longer than alloBMT pts (1035 vs 466 days, p=0,0012). AlloBMT stem cell source (SCS): 73% bone marrow stem cell (BMSC), 23% peripheral blood stem cells (PBSC) and 4% umbilical cord blood. Among the autoBMT pts, the SCS was 63% PBSC, 22% BMSC and 15% both. The SCS did not impact on OS. There was no difference in OS between different FAB cla...