An update to the American Society for Blood and Marrow Transplantation (ASBMT) 2005 evidence-based review on the role of hematopoietic cell transplantation (HCT) in the treatment of children with acute lymphoblastic leukemia (ALL). Based on newly published data, an ASBMT panel of experts has provided recommendations on: allogeneic and autologous transplantation vs. non-transplant therapy; conditioning regimens for allogeneic HCT; and related vs. unrelated donor allogeneic HCT. The expert panel also made recommendations pertaining to various transplantation techniques and identified areas of needed research in the treatment of pediatric ALL.
HCT vs. immunosuppressive therapy in aplastic anemia
Five-year survival is higher in pediatric patients with idiopathic severe aplastic anemia undergoing hematopoietic cell transplantation (HCT) than in those receiving immunosuppressive therapy (IST), according to a retrospective study from the United Kingdom. The study compared 43 children receiving IST (antithymocyte globulin/cyclosporine) to 44 patients who received a 10/10 HLA-matched (HLA-A, -B, -C, -DRB1, -DQB1) unrelated donor transplant using a non-TBI regimen. Five-year estimated failure-free survival was 13% and 95% in the IST and HCT cohorts, respectively.
Age, conditioning regimen not prognostic in HCT for older patients
An analysis of 71 patients (median age 57 years; range 50-63) has shown that conditioning regimen and age are not predictors of hematopoietic cell transplantation (HCT) outcome. In a multivariate analysis of 53 patients with acute leukemia and 18 patients with myelodysplastic syndromes, only high-risk disease (Hazard ratio (HR): 3.50, 95% CI 1.43-8.56, p=0.006), an HLA-mismatched unrelated donor (HR: 4.03, 95% CI 1.4611.10, p=0.007), and HCT comorbidity index (HCT-CI) score ≥3 (HR: 4.41, 95% CI 1.31-14.77, p=0.016) were significant predictors of worse overall survival (OS). Conditioning regimen (myeloablative (n=35) or reduced-intensity (n=36)) and age did not predict OS.
Pre-transplant azacitidine (AZA) can benefit transplant recipients, according to a retrospective study examining AZA therapy and induction chemotherapy (IC) in 68 patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia transformed from MDS. Pre-HCT AZA led to a 66% lower hazard of relapse compared to IC (p=0.004), but after adjusting for cytogenetic risk, IPSS, and donor type, this was no longer statistically significant. Although patients in the AZA cohort were significantly older than IC-treated patients (median 60 vs. 47 years, respectively), non-relapse mortality was comparable in the two cohorts. The authors conclude that “therapy with azacitidine is associated with less toxicity than IC and may allow for similar post-HCT outcomes.”
A review of mesenchymal stromal cells (MSCs), multipotent cells with the ability to regulate hematopoiesis through production of growth factors and secretion of matrix proteins. The authors evaluate the latest research suggesting that post-transplant MSC infusions can facilitate engraftment and accelerate post-transplant hematopoietic recovery. They note that while current research shows that MSC infusion is safe and may play a role in preventing graft failure, much less is known about their capacity to accelerate post-transplant hematopoietic reconstitution.
Access NMDP webinar of PBSC vs. marrow study results
View a recording of the PBSC vs. Marrow Study Results webinar discussing the initial results of the BMT CTN Protocol (0201) Phase III PBSC versus Marrow Study that were presented during the 2011 American Society of Hematology Annual Meeting. Access the NMDP-sponsored webinar »
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