Clonal Evolution of Myeloid Malignancies Treated With Microtransplantation: A Single-Centre Experience

Microtransplantation is a cellular therapy used in acute myeloid leukaemia and myelodysplastic syndromes as a maintenance therapy in patients ineligible for a regular allogeneic stem cell transplantation. We performed a monocentric retrospective study of acute myeloid leukaemia, myelodysplastic syndromes, and chronic myelomonocytic leukaemia patients who underwent microtransplantations at Nice University Hospital. We analysed the evolution of the disease mutational status after microtransplantation. We report 18 patients who underwent microtransplantation courses, with a total of 47 microtransplantations performed between February 2020 and June 2022. We observed long-term remissions even in high-risk patients. Founder mutations persisted throughout the follow-up, whereas it was more variable for other nonfounder mutations, with most of the nonfounder mutations variant allele frequency decreasing over time. Safety data were reassuring; no graft versus host disease was recorded, and cytokine release syndromes were manageable. Relapses or progressions were associated with the emergence or increase of a TP53 mutated clone. Microtransplantation is a promising therapy for patients ineligible for regular allogeneic stem transplantation. Further larger and randomised studies are required to establish its place as a maintenance therapy in myeloid malignancies.

Publication Details

Journal
Repository for Publications and Research Data (ETH Zurich)
Published
2025-03-01
DOI
https://doi.org/10.3929/ethz-b-000729242
Primary Topic
Evolution and Paleontology Studies
Type
article
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article

Clonal Evolution of Myeloid Malignancies Treated With Microtransplantation: A Single-Centre Experience

Repository for Publications and Research Data (ETH Zurich)
Evolution and Paleontology Studies
article

Clonal Evolution of Myeloid Malignancies Treated With Microtransplantation: A Single-Centre Experience

article en

Abstract

Microtransplantation is a cellular therapy used in acute myeloid leukaemia and myelodysplastic syndromes as a maintenance therapy in patients ineligible for a regular allogeneic stem cell transplantation. We performed a monocentric retrospective study of acute myeloid leukaemia, myelodysplastic syndromes, and chronic myelomonocytic leukaemia patients who underwent microtransplantations at Nice University Hospital. We analysed the evolution of the disease mutational status after microtransplantation. We report 18 patients who underwent microtransplantation courses, with a total of 47 microtransplantations performed between February 2020 and June 2022. We observed long-term remissions even in high-risk patients. Founder mutations persisted throughout the follow-up, whereas it was more variable for other nonfounder mutations, with most of the nonfounder mutations variant allele frequency decreasing over time. Safety data were reassuring; no graft versus host disease was recorded, and cytokine release syndromes were manageable. Relapses or progressions were associated with the emergence or increase of a TP53 mutated clone. Microtransplantation is a promising therapy for patients ineligible for regular allogeneic stem transplantation. Further larger and randomised studies are required to establish its place as a maintenance therapy in myeloid malignancies.

Repository for Publications and Research Data (ETH Zurich)
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