Molecular assessment of pretransplant chemotherapy in the treatment of juvenile myelomonocytic leukemia.

ABSTRACT:

Background

Despite the intensity of hematopoietic stem cell transplantation (HCT), relapse remains the most common cause of death in juvenile myelomonocytic leukemia (JMML). In contrast to other leukemias where therapy is used to reduce leukemic burden prior to transplant, many patients with JMML proceed directly to HCT with active disease. The objective of this study was to elucidate whether pre-HCT therapy has an effect on the molecular burden of disease and how this affects outcome post-HCT.

Procedure

Twenty-one patients with JMML who received pre-HCT therapy and were transplanted at UCSF were analyzed in this study. The mutant allele frequency of the driver mutation was assessed before and after pre-HCT therapy, using custom amplicon next-generation sequencing.

Result

SUBMITTER: Hecht A 

PROVIDER: S-EPMC6754267 | biostudies-literature | 2019 Nov

REPOSITORIES: biostudies-literature