title: Feasibility of treating post-transplantation minimal residual disease in children with acute leukemia.
abstract: Outcomes are poor for patients with hematologic malignancies who experience overt relapse after allogeneic hematopoietic stem cell transplantation (HCT). Data on outcomes of post-transplantation minimal residual disease (MRD) are limited. In this single-institution, retrospective cohort analysis of children with acute leukemia and myelodysplastic syndrome, we document the pattern of relapse with a primary focus on outcomes of post-transplantation MRD. Forty of 93 patients (43%) who underwent a first allogeneic HCT and had systematic pretransplantation and post-transplantation MRD evaluations at +30, +60, +90, +180 days and +1 and +2 years post-transplantation experienced relapse. The median time to relapse was 4.8 months post-transplantation, with a median survival of 4 months post-relapse. Despite frequent, systematic, routine post-HCT disease restaging evaluation, 31 patients (78%) presented with overt disease at the time of relapse. Seven patients with acute leukemia who had post-transplantation MRD presented at a median of 1 month post-transplantation. Owing to rapid disease progression or treatment-related mortality, there was no improvement in survival in those patients whose leukemia was detected in a state of MRD post-transplantation. Our results suggest that early intervention strategies targeting post-transplantation MRD for relapse prevention in acute leukemia may not be feasible.
authors.affiliation: Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland; Pediatric Oncology, Johns Hopkins University, Baltimore, Maryland. Electronic address: Nirali.Shah@nih.gov.
authors.forename: Nirali N
authors.initials: NN
authors.lastname: Shah
authors.affiliation: Department of Pathology, Johns Hopkins University, Baltimore, Maryland.
authors.forename: Michael J
authors.initials: MJ
authors.lastname: Borowitz
authors.affiliation: Pediatric Oncology, Johns Hopkins University, Baltimore, Maryland.
authors.forename: Nancy C
authors.initials: NC
authors.lastname: Robey
authors.affiliation: Pediatric Oncology, Johns Hopkins University, Baltimore, Maryland.
authors.forename: Christopher J
authors.initials: CJ
authors.lastname: Gamper
authors.affiliation: Pediatric Oncology, Johns Hopkins University, Baltimore, Maryland.
authors.forename: Heather J
authors.initials: HJ
authors.lastname: Symons
authors.affiliation: Pediatric Oncology, Johns Hopkins University, Baltimore, Maryland.
authors.forename: David M
authors.initials: DM
authors.lastname: Loeb
authors.affiliation: Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland; Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, California.
authors.forename: Alan S
authors.initials: AS
authors.lastname: Wayne
authors.affiliation: Pediatric Oncology, Johns Hopkins University, Baltimore, Maryland.
authors.forename: Allen R
authors.initials: AR
authors.lastname: Chen
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D005240:Feasibility Studies; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007938:Leukemia / Q000473:pathology / Q000628:therapy*; D008297:Male; D018365:Neoplasm, Residual; D012189:Retrospective Studies; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult
keywords: Allogeneic hematopoietic cell transplantation; Leukemia; Minimal residual disease; Pediatrics; Relapse