Abatacept Looks Promising in Chronic GVHD Treatment
Nahas MR, Soiffer RJ, Kim HT, et al. Phase I Clinical Trial Evaluating Abatacept in Patients with Steroid-Refractory Chronic Graft-Versus-Host Disease. Blood 2018; 131 (25): 2836-2845
Use of abatacept generated a clinical partial response per 2005 National Institutes of Health Consensus Criteria in 44% of patients with steroid-refractory chronic graft-versus-host disease (SR-cGVHD) following allogeneic stem cell transplantation, according to research published in Blood. The Phase I clinical trial with 16 evaluable participants concluded that the drug was well-tolerated with no dose-limiting toxicities. Notably, use of abatacept led to a 51.3% reduction in prednisone use among clinical responders. Clinical responders also had increased PD-1 expression on circulating CD4 and CD8 T cells. The findings suggest that costimulatory blockade with abatacept holds potential as a therapeutic agent for SR-cGVH.
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Allogeneic Transplant for Adults With Sickle Cell Disease
Ozdogu H, Boga C, Yeral M, et al. Allogenic Peripheral Stem Cell Transplantation From HLA-Matched Related Donors for Adult Sickle Cell Disease. Bone Marrow Transplantation 2018: 53: 880-890
Study results reported in Bone Marrow Transplantation point to a promising intervention for adult sickle cell disease (SCD) patients, who often experience complications from stem cell transplants. The research from the University of Baskent in Ankara, Turkey, involved 20 patients who underwent allogeneic peripheral blood stem cell transplantation from human leukocyte antigen-identical related donors. The conditioning regimen included fludarabine, busulfan, and anti-T-cell lymphocyte globulin plus very low dose total body irradiation. The procedure was followed by post-transplant cyclophosphamide and sirolimus as prevention against graft versus host disease (GVHD). The novel treatment approach achieved favorable outcomes with no mortality, graft rejection, or other major adverse events. Additionally, the incidence of GVHD was low, with no cases of grade III-IV acute GVHD or extensive chronic GVHD. While the findings of the small, single-site study are encouraging, the investigators concede that additional controlled studies are needed to compare transplant protocols and long-term results.
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Transplant for Secondary AML Outcomes
Sengsayadeth S, Labopin M, Boumendil A, et al. Transplant Outcomes for Secondary Acute Myeloid Leukemia: Acute Leukemia Working Party of the ESBBMT Study. Biology of Bone and Marrow Transplantation 2018; 24 (7): 1406-1414
Risk factors contributing to poor survival rates in patients with secondary acute myeloid leukemia (sAML) after stem cell transplant include active disease, adverse cytogenetics, and older age, according to a report in Biology of Bone and Marrow Transplantation. Researchers reached the conclusion following a systemic analysis of the European Society for Blood and Bone Marrow Transplantation registry. With a sample population of 4,997 sAML patients who underwent allogeneic hematopoietic stem cell transplantation (HCT) from 2000 to 2016, the two-year cumulative incidence of chronic graft-versus-host disease (GVHD) was 33.5%, while the rate of relapse was 33.7% and nonrelapse mortality was 27.5%. The rates of overall survival, leukemia-free survival and GVHD-free, relapse-free survival at two years were 44.5%, 38.8%, and 27.2%, respectively. The team also identified ex vivo T cell depletion, the presence of other malignant hematologic disease, patient cytomegalovirus seropositivity, and Karnofsky performance status as other predictors of low rates of overall survival and leukemia-free survival. These factors, they conclude, should be considered in sAML patients who require HCT.
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