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Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase

Peter Bader1, Ulrike Pötschger2, Jean-Hugues Dalle3

  • 1Goethe University, University Hospital, Department of Pediatrics, Division for Stem Cell Transplantation, Immunology and Intensive Care Medicine, Frankfurt, Germany.

Blood Advances
|September 22, 2023
PubMed

Insights

This study compared two TBI-free conditioning regimens for pediatric acute lymphoblastic leukemia (ALL) patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). Both regimens were well-tolerated with low nonrelapse mortality, but relapse remained a significant challenge.

Area of Science:

  • Pediatric Hematology Oncology
  • Stem Cell Transplantation
  • Leukemia Research

Background:

  • Allogeneic hematopoietic stem cell transplantation (HSCT) is a vital treatment for high-risk or relapsed pediatric acute lymphoblastic leukemia (ALL).
  • Total body irradiation (TBI)-based conditioning regimens can cause severe late sequelae in young children.
  • TBI-free conditioning regimens offer a potentially safer alternative for this vulnerable population.

Purpose of the Study:

  • To evaluate the safety and efficacy of two TBI-free conditioning regimens in children under 4 years old with ALL undergoing HSCT.
  • To compare event-free survival (EFS) and overall survival (OS) between fludarabine/thiotepa/busulfan (Flu/Thio/Bu) and fludarabine/thiotepa/treosulfan (Flu/Thio/Treo) regimens.
  • To assess nonrelapse mortality (NRM), relapse rates, and graft-versus-host disease (GVHD) incidence.

Main Methods:

  • The FORUM study (NCT01949129) enrolled 191 children aged <4 years with ALL.
  • Patients received conditioning with fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) prior to HSCT.
  • Outcomes including OS, EFS, NRM, relapse, and GVHD were analyzed with a median follow-up of 3 years.

Main Results:

  • Three-year overall survival (OS) was 63% for Flu/Thio/Bu and 76% for Flu/Thio/Treo (P=.075).
  • Three-year event-free survival (EFS) was 52% for both regimens (P=.794).
  • Cumulative incidence of relapse at 3 years was 42% for Flu/Thio/Bu and 45% for Flu/Thio/Treo (P=.920).
  • Grade >1 acute GVHD was more frequent with Flu/Thio/Bu (29%) than Flu/Thio/Treo (17%) (P=.049).
  • Nonrelapse mortality (NRM) was low for both regimens (6% vs 3%).

Conclusions:

  • Both TBI-free conditioning regimens (Flu/Thio/Bu and Flu/Thio/Treo) demonstrated good tolerability and low NRM in young children with ALL undergoing HSCT.
  • Relapse emerged as the primary cause of treatment failure, highlighting the need for strategies to mitigate relapse risk.
  • The Flu/Thio/Treo regimen showed a trend towards improved OS and lower acute GVHD incidence, warranting further investigation.

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