Twenty years of unrelated donor hematopoietic cell transplantation for adult recipients facilitated by the National Marrow Donor Program.
ABSTRACT For more than 20 years the National Marrow Donor Program has facilitated unrelated donor hematopoietic cell transplants for adult recipients. In this time period, the volunteer donor pool has expanded to nearly 12 million adult donors worldwide, improvements have occurred in the understanding and technology of HLA matching, there have been many changes in clinical practice and supportive care, and the more common graft source has shifted from bone marrow (BM) to peripheral blood stem cells (PBSCs). The percentage of older patients who are receiving unrelated donor transplants is increasing; currently over 1 in 10 adult transplant recipients is over the age of 60 years. Chronic myelogenous leukemia (CML) was previously the most common diagnosis for unrelated donor transplantation, but it now comprises less than 10% of transplants for adult recipients. Transplants for acute myelogenous leukemia (AML), acute lymphoblastic leukemia (ALL), non-Hodgkin lymphoma (NHL), and myelodysplastic syndromes (MDS) all outnumber CML. Treatment-related mortality (TRM) has declined significantly over the years, particularly in association with myeloablative transplant preparative regimens. Correspondingly, survival within each disease category has improved. Particularly gratifying are the results in severe aplastic anemia (AA) where 2-year survival has doubled in just 10 years.
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ABSTRACT: Myelodysplastic syndromes (MDS) are heterogeneous malignant bone marrow disorders diagnosed most often in elderly white persons. MDS have significant clinical consequences, including cytopenias leading to infection, bleeding, and death; and approximately one-third of cases progress to acute myeloid leukemia (AML). Only one potentially curative therapy exists-allogeneic hematopoietic stem cell transplant (HSCT)-but this therapy is not widely used due to associated morbidity and mortality in elderly patients. Recent research suggests MDS occurs more frequently than previously thought and may be responsible for a substantial proportion of unexplained anemias in elderly persons. Incidence of MDS is expected to increase with increases in life expectancy. Therefore, we offer this comprehensive narrative update of MDS to inform the medical community treating the population at risk for MDS, with a focus on MDS epidemiology and clinical management in the United States. This review includes a brief historical background of MDS, provides an overview of the population burden of disease, discusses the molecular pathology of MDS, describes the clinical features and management of MDS, and discusses future directions in MDS research. Our objective is to inform general medicine practitioners and call attention to the need for translational research in MDS.Annals of Medicine 04/2014; · 4.73 Impact Factor
- Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation 09/2013; · 3.15 Impact Factor
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ABSTRACT: Mobilized peripheral blood has become the predominant stem cell source for allogeneic hematopoietic cell transplantation. In this retrospective single-center study of 442 patients with hematologic malignancies, we analyzed prognostic factors for long-term survival after peripheral blood stem cell transplantation from HLA-matched related or unrelated donors. To account for disease/status heterogeneity, patients were risk-stratified according to the Disease Risk Index. Five-year overall survival was similar after transplants with matched related and unrelated donors (45% and 46%, respectively, P=.49). Because donor age ≥ 60 years impacted outcome during model building, we further considered 3 groups of donors: matched unrelated (aged < 60 years by definition), matched related aged < 60 years and matched related aged ≥ 60 years. In multivariate analysis, the donor type/age group and the graft CD34+ and CD3+ cell doses impacted long-term survival. Compared with matched unrelated donor transplant, transplant from matched related donor < 60 years resulted in similar long-term survival (P=.67) while transplant from matched related donor ≥ 60 years was associated with higher risks for late mortality (hazard ratio: 4.41, P=.006) and treatment failure (hazard ratio: 6.33, P=.009). Lower mortality risks were observed after transplant with CD34+ cell dose > 4.5 x 106/kg (hazard ratio: 0.56, P=.002) and CD3+ cell dose > 3 x 108/kg (hazard ratio: 0.61, P=.01). The Disease Risk Index failed to predict survival. We built an Adapted Disease Risk Index by modifying risks for myeloproliferative neoplasms and multiple myeloma, that improved stratification ability for progression-free survival (P=.04) but not for overall survival (P=.82).Haematologica 11/2013; · 5.94 Impact Factor