Patientswith newly diagnosed transformed Fanconi anemia (FA) have poor outcomes andshould achieve complete remission (CR) prior to allogeneic hematopoietic stemcell transplantation (alloHSCT), according to results from a study published inthe American Journal of Hematology.

Stefano Giardino, MD, of the hematopoietic stem cell transplantation unit at the Istituto Giannina Gaslini in Italy, and colleagues retrospectively analyzed outcomes of 74 patients with transformed FA (36 male; median age, 14 years); 35 patients had myelodysplastic syndrome, 35 had acute leukemia, and 4 patients had high-risk cytogenetic abnormality. All patients underwent alloHSCT from 1999 to 2016.

The primary end points were overall survival (OS) and event-free survival (EFS). Secondary endpoints included the incidence of grade 2 to 4 acute graft-vs-host disease (AGVHD) and chronic graft-vs-host disease (CGVHD), non-relapse mortality (NRM), and incidence of relapse. To identify potential factors that may influence outcomes, the researchers assessed the type of diagnosis, preHSCT cytoreductive therapies and related toxicities, disease status prior to HSCT, donor type, and conditioning regimen.

Ata median follow-up of 7 years, 5-year OS and EFS were 42% and 39%,respectively; 5-year cumulative incidence relapse and NRM rates were 21% and40%, respectively. Patients in CR during transplant had better OS than those whostill had active disease (OS, 71% vs 37%, respectively; P =.04). No other factors had a significant effecton patient outcomes.

Of22 patients who received cytoreductive therapy prior to HSCT, 40.9% experienceda grade 3 to grade 4 toxicity event; this did not appear to effect survivalafter HSCT (3 year OS, toxicity preHSCT 48% vs no toxicity 51%; P =.98).At 100 days, the cumulative incidence of grade 2 to 4 AGVHD was 38%, and the cumulativeincidence of 5-year CGVHD was 40%.

At5 years, NRM was 40%, while incidence of relapse was 21%. Transplant-relatedevents were the cause of mortality in 81% of patients (34 of 42 deaths).

Limitationsof the study included the retrospective design and incomplete data for somevariables. The authors highlighted the large number of patients for this raredisorder as the primary strength.

Inorder to optimize the chances of the only curative option for FA in malignanttransformation, a sequential cytoreductive therapy followed by HSCT appears areasonable approach in FA patients with AL, if a previously identified donor israpidly available, wrote the authors. However, since the risk oftreatment-related complications is high, these patients should be managed inhighly specialized centers and transplant approaches aimed at reducing theoccurrence of [GVHD] and transplant-related complications should be prioritized.

Reference

Giardino S, Latour RP, Aljurf M, et al. Outcome of patients with Fanconi anemia developing myelodysplasia and acute leukemia who received allogeneic hematopoietic stem cell transplantation: a retrospective analysis on behalf of EBMT group [published online April 8, 2020]. Am J Hematol. doi: 10.1002/ajh.25810

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Outcomes of Stem Cell Transplantation in Patients With Newly Diagnosed Transformed Fanconi Anemia - Hematology Advisor