Dataset Information

Advances in unrelated and alternative donor hematopoietic cell transplantation for nonmalignant disorders.

ABSTRACT: The role of hematopoietic cell transplantation in non-malignant disorders has increased exponentially with the recognition that multiple diseases can be controlled or cured if engrafted with donor-derived cells. This review provides an overview of advances made in alternative donor transplants for nonmalignant disorders.Stem cell sources, novel transplant methods, and sophisticated supportive care have simultaneously made giant strides toward improving the safety and efficacy of hematopoietic cell transplantation. This has led to the utilization of marrow, cord, peripheral blood stem cell and haploidentical stem cell sources, and novel reduced toxicity or reduced intensity conditioning regimens to transplant non-malignant disorders such as immune dysfunctions, marrow failure syndromes, metabolic disorders and hemoglobinopathies. Transplant complications such as graft rejection, infections, and graft versus host disease are better combated in this modern era of medicine, achieving better survival with decreased late effects. These aspects of transplant for non-malignant disorders are discussed.This review presents the progress made in the realm of hematopoietic cell transplantation for non-malignant disorders. It advocates the consideration of alternative donor transplants in the absence of human leukocyte antigen matched siblings when indicated by disease severity. The ultimate goal is to provide curative transplant options for more patients that can benefit from this intervention, prior to detrimental outcomes.

SUBMITTER: Shenoy S

PROVIDER: S-EPMC4403859 | biostudies-literature | 2015 Feb

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Advances in unrelated and alternative donor hematopoietic cell transplantation for nonmalignant disorders.

Shenoy Shalini S Boelens Jaap J JJ

Current opinion in pediatrics 20150201 1

<h4>Purpose of review</h4>The role of hematopoietic cell transplantation in non-malignant disorders has increased exponentially with the recognition that multiple diseases can be controlled or cured if engrafted with donor-derived cells. This review provides an overview of advances made in alternative donor transplants for nonmalignant disorders.<h4>Recent findings</h4>Stem cell sources, novel transplant methods, and sophisticated supportive care have simultaneously made giant strides toward imp ...[more]

PMID: 25565572

Similar Datasets

Project description:BackgroundIdentification of non-human leukocyte antigen (HLA) genetic risk factors could improve survival after allogeneic blood or marrow transplant (BMT) through matching at additional loci or individualizing risk prediction. We hypothesized that non-HLA loci contributed significantly to 1-year overall survival (OS), disease related mortality (DRM) or transplant related mortality (TRM) after unrelated donor (URD)BMT.MethodsWe performed a genome-wide association study (GWAS) in 2,887 acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and acute lymphoblastic leukemia (ALL) patients and their ≥8/8 HLA-matched URDs comprising two independent cohorts treated from 2000-2011.FindingsUsing meta-analyses of both cohorts, genome-wide significant associations (p < 5 × 10-8) were identified in: recipient genomes with OS at MBNL1 (rs9990017, HR = 1.4, 95% CI 1.24-1.56, p = 3.3 × 10-8) and donor-recipient genotype mismatch with OS at LINC02774 (rs10927108, HR = 1.34, 95% CI 1.21-1.48, p = 2.0 × 10-8); donor genomes with DRM at PCNX4 (rs79076914, HR = 1.7, 95% CI 1.41-2.05, p = 3.15 × 10-8), LINC01194 (rs79498125, HR = 1.86, 95% CI 1.49-2.31, p = 2.84 × 10-8), ARID5B (rs2167710, HR = 1.5, 95% CI 1.31-1.73, p = 6.9 × 10-9) and CT49 (rs32250, HR = 1.44, 95% CI1.26-1.64, p = 2.6 × 10-8); recipient genomes at PILRB with TRM (rs141591562, HR = 2.33, 95% CI 1.74-3.12, p = 1.26 × 10-8) and donor-recipient genotype mismatch between EPGN and MTHF2DL with TRM (rs75868097, HR = 2.66, 95% CI 1.92-3.58, p = 4.6 × 10-9). Results publicly available at https://fuma.ctglab.nl/browse.InterpretationThese data provide the first evidence that non-HLA common genetic variation at novel loci with biochemical function significantly impacts 1-year URD-BMT survival. Our findings have implications for donor selection, could guide treatment strategies and provide individualized risk prediction after future validation and functional studies.FundingThis project was funded by grants from the National Institutes of Health, USA.

Dataset Information

Advances in unrelated and alternative donor hematopoietic cell transplantation for nonmalignant disorders.

Publications

Advances in unrelated and alternative donor hematopoietic cell transplantation for nonmalignant disorders.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets