Clinical outcome of cord blood transplantation for nine children with juvenile myelomonocytic leukemia receiving fludarabine-busulfan-cyclophosphamide-based conditioning

Pediatr Transplant. 2022 Mar;26(2):e14181. doi: 10.1111/petr.14181. Epub 2021 Nov 8.

Abstract

Background: Juvenile myelomonocytic leukemia (JMML) is a rare hematological malignancy in young children and can only be cured through the allogeneic stem cell transplantation.

Procedure: We have retrospectively analyzed the outcomes of nine children with JMML after unrelated cord blood transplantation (UCBT).

Results: Eight patients who have received a myeloablative conditioning regimen of fludarabine (FLU), busulfan (BU), and cyclophosphamide (CY) have gotten engraftment. None of the nine patients has relapsed following initial UCBT. Six patients are still alive and in complete remission after UCBT with a median observation time of 43 months (range: 10-80 months).

Conclusions: This study shows that UCBT with FLU-BU-CY conditioning regimen can represent a suitable option for children with JMML.

Keywords: conditioning regimen; juvenile myelomonocytic leukemia; umbilical cord blood transplant.

MeSH terms

  • Antineoplastic Agents / administration & dosage
  • Busulfan / administration & dosage
  • Child
  • Child, Preschool
  • China
  • Cord Blood Stem Cell Transplantation*
  • Cyclophosphamide / administration & dosage
  • Humans
  • Infant
  • Leukemia, Myelomonocytic, Juvenile / therapy*
  • Male
  • Retrospective Studies
  • Transplantation Conditioning / methods*
  • Vidarabine / administration & dosage
  • Vidarabine / analogs & derivatives

Substances

  • Antineoplastic Agents
  • Cyclophosphamide
  • Vidarabine
  • Busulfan
  • fludarabine