Disease Status and Interval between HCTs Predict Outcome of Pediatric Patients who Undergo a Subsequent HCT for Relapsed Hematologic Malignancy.

BACKGROUND

Patients with hematologic malignancies who relapse after allogeneic hematopoietic cell transplant (HCT) have a poor prognosis. While proceeding to subsequent HCT can provide potential for long-term survival, there is limited data to guide which patients are most likely to benefit and which HCT strategies are best in this heavily pre-treated population.

OBJECTIVE

The goals of this study were to: i) describe the clinical outcomes of a subsequent HCT in pediatric patients with relapsed hematologic malignancies in a cohort enriched for haploidentical donors, and ii) evaluate the association of patient-, disease-, and treatment-related factors with survival.

STUDY DESIGN

We retrospectively evaluated patients who underwent a subsequent HCT for management of post-HCT relapse at a single institution between 2000-2021.

RESULTS

Among 106 patients who received a second allogeneic HCT, one-year event-free survival (EFS) was 34% and one-year overall survival (OS) was 46%, with five-year EFS of 26% and five-year OS of 31%. Only disease-related factors were associated with outcome after second HCT, specifically the interval between HCTs and presence or absence of active disease at the time of HCT. In this cohort, patient- and treatment-related factors were not associated with differences in EFS or OS. Patients receiving a third or fourth HCT (n=13) had comparable survival outcomes to those receiving a second HCT.

CONCLUSIONS

Our experience highlights that a subsequent HCT has curative potential for a subset of patients who relapse after HCT, including those who receive a subsequent HCT from a haploidentical donor. While relapse and treatment-related toxicities remain major challenges, our study indicates that achieving complete remission prior to subsequent HCTs has the potential to further improve outcomes.

Informations de copyright

Copyright © 2024. Published by Elsevier Inc.

Déclaration de conflit d'intérêts

Declaration of competing interest This study was supported by the National Institutes of Health (NIH)/National Cancer Institute grant P30CA021765, and the American Lebanese Syrian Associated Charities. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH. RE is supported by an American Society for Transplantation and Cellular Therapy New Investigator Award. SG is a co-inventor on patent applications in the fields of cell or gene therapy for cancer, a member of the Scientific Advisory Board of Be Biopharma and CARGO, and the Data and Safety Monitoring Board (DSMB) of Immatics and has received honoraria from TESSA Therapeutics within the last year. ASh has received consultant fee from Spotlight Therapeutics, Medexus Inc., Vertex Pharmaceuticals, Sangamo Therapeutics and Editas Medicine. He is a medical monitor for RCI BMT CSIDE and has received research funding from CRISPR Therapeutics and honoraria from Vindico Medical Education. The remaining authors declare no non-financial competing interests.