Allogeneic hematopoietic cell transplantation in systemic mastocytosis: is there a high risk for veno-occlusive disease?

2016 ◽  
Vol 96 (6) ◽  
pp. 655-657 ◽  
Author(s):  
Celalettin Ustun ◽  
Angela Smith ◽  
Zuzan Cayci ◽  
Elizabeth L. Courville ◽  
Selim Corbacioglu ◽  
...  
Keyword(s):  
High Risk ◽  
Cell Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
Systemic Mastocytosis ◽  
Occlusive Disease ◽  
Hematopoietic Cell ◽  
Allogeneic Hematopoietic Cell Transplantation

Allogeneic hematopoietic cell transplantation for adults with acute myeloid leukemia: myths, controversies, and unknowns

Blood ◽  
2011 ◽  
Vol 117 (8) ◽  
pp. 2307-2318 ◽  
Author(s):  
Vikas Gupta ◽  
Martin S. Tallman ◽  
Daniel J. Weisdorf
Keyword(s):  
Acute Myeloid Leukemia ◽  
High Risk ◽  
Cell Transplantation ◽  
Myeloid Leukemia ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Allogeneic Hematopoietic Cell Transplantation ◽  
Reduced Intensity Conditioning ◽  
Reduced Intensity ◽  
Acute Myeloid

AbstractProgress in the last decade has improved the understanding of leukemia biology. Molecular markers in combinations with cytogenetics have improved the risk stratification of acute myeloid leukemia (AML) and informed decision-making. In parallel, several important advances in the transplant field, such as better supportive care, improved transplant technology, increased availability of alternative donors, and reduced-intensity conditioning have improved the safety as well as access of allogeneic hematopoietic cell transplantation (HCT) for a larger number of patients. In this review, the positioning of HCT in the management of patients with AML is evaluated in view of changing risk/benefit ratios associated with both conventional treatments and transplantation, and some of the controversies are addressed in light of emerging data. Increasing data demonstrate outcomes of alternative donor transplantation approaching HLA-identical sibling donors in high-risk AML supporting the inclusion of alternative donors in trials of prospective studies evaluating post remission strategies for high-risk AML. The use of reduced-intensity conditioning has expanded the eligibility of HCT to older patients with AML, and outcome data are encouraging. Continued study of HCT versus alternative therapies is required to optimize patients' outcomes in AML.

Comparison of 5-Azacytidine Versus Allogeneic Hematopoietic Cell Transplantation In Elderly (≥ 60 years) Patients with De Novo High-Risk MDS - a Retrospective Matched Pair Analysis

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 3500-3500
Author(s):  
Uwe Platzbecker ◽  
Johannes Schetelig ◽  
Juergen Finke ◽  
Rudolf Trenschel ◽  
Bart Lee Scott ◽  
...  
Keyword(s):  
High Risk ◽  
Board Of Directors ◽  
Cell Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
De Novo ◽  
Hematopoietic Cell ◽  
Allogeneic Hematopoietic Cell Transplantation ◽  
Disease Stage ◽  
Advisory Committees ◽  
Allogeneic Hct

Abstract Abstract 3500 Background: Myelodysplastic syndromes (MDS) are a disease of the elderly in whom until recently, allogeneic hematopoietic cell transplantation (HCT) has not been considered a reasonable therapeutic option. Best supportive care (BSC), only including transfusion support, has been the standard of care for the majority of these patients (pts). However, innovations in transplant protocols have changed that approach resulting in an increased frequency of allogeneic HCT. Further, several innovative non-transplant therapeutic modalities for MDS pts have been developed. In fact, treatment with DNA-methyltransferase inhibitors (MTI) such as 5-azacytidine (5-aza) has changed the natural course of the disease and prolonged median survival by an average of 9 months compared to BSC. However, allogeneic HCT is currently still the only modality with proven curative potential, although there has been no true randomized trial comparison of HCT to 5-aza therapy only. Methods: Consequently, we performed a retrospective analysis in 126 pts, 60 –77, (median 64) years of age with de novo high-risk MDS according to FAB classification (RAEB n=88, RAEB-t n=20, CMML n=18) undergoing allogeneic HCT at a median of 9 months from initial MDS diagnosis. Cytogenetics were available in 108 pts (good: n=63, intermediate: n=18, poor: n=27), resulting in IPSS classification (in 107 pts) of INT-1 (n=28), INT-2 (n=45) or HIGH (n=34). The ECOG was available in 125 pts, and was either 0 (19%), 1 (66%) or >1 (15%). Prior to HCT, most pts had progressed to a higher stage than present at diagnosis (RAEB n=45, RAEB-t n=10, CMML n=10, AML n=61), the median blast count in the marrow being 12%. Patients were prepared for HCT with one of several reduced (RIC, n=78) or more conventional intensity (n=48) conditioning regimens followed by stem cells from either related (n=50) or unrelated (n=76) donors. The outcome of this high-risk MDS cohort was compared to patients from the French (GFM) 5-aza patient registry matched for age, gender, prior induction chemotherapy, time from diagnosis to therapy (either HCT or 5-aza), disease stage and cytogenetics at diagnosis as well as at start of therapy. The groups could be well matched except for age (median age 5-aza: 67 vs. HCT: 64; p=0.001). Further, the HCT group included more pts with a higher ECOG score (ECOG>0: 81% vs. 59%; p=0.05) prior to start of therapy. Results: With a median follow-up of 20 months for surviving pts from the start of therapy the estimated 3-year overall survival (OS) was 39% (95% CI, 30% to 48%) for HCT and 7% (95% CI, 0 to 16%) for 5-aza, respectively. In multivariate Cox regression analysis, ECOG score (p<0.001), disease stage p=0.021), cytogenetic risk group (p=0.002) and type of treatment (5-aza vs. HCT HR: 1.8; p=0.003) were associated with survival. Conclusion: These retrospective data suggest that allogeneic HCT from related or unrelated donors, using various conditioning approaches, might offer a meaningful survival benefit compared to 5-aza in patients with high-risk MDS, even in the 7th decade of life. There is a need for prospective clinical trials in order to determine the place of this approach within the growing therapeutic opportunities for pts with MDS. Disclosures: Platzbecker: Celgene: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding. Ades:Celgene: Honoraria, Membership on an entity's Board of Directors or advisory committees.

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