Abstract
Allogeneic hematopoietic cell transplantation (allo-HCT) is the only curative treatment for patients with myeloid/lymphoid neoplasm (MLN) with FGFR1 rearrangement, but data on overall results are limited. We report on the largest series of patients (n = 22) with FGFR1-rearranged MLN undergoing allo-HCT. Distribution according to cytogenetic subtype was: t(8;13) in 11 cases, t(8;22) in 7 cases, t(6;8) in 2 cases, and other (n = 2). Over a third of patients displayed a chronic myeloproliferative (MPN) phenotype, another third showed MPN features with concomitant lymphoma or acute leukemia, and the remaining ones presented as acute leukemia. After a median follow-up of 4.1 years from transplant, the estimated 5-year survival rate, progression-free survival, non-relapse mortality and relapse incidence was 74%, 63%, 14% and 23%, respectively. Causes of death were relapse/progression (n = 4), graft-versus-host disease (n = 2) and organ toxicity (n = 1). Six patients experienced disease relapse at a median of 6.1 months (range: 2.3–119.6). Two of them achieved complete remission with ponatinib or pemigatinib and were alive at 34.5 and 37 months from relapse, respectively. These data highlight the significant curative potential of allo-HCT in this aggressive disease. Maintenance with tyrosine kinase inhibitors may be a promising approach, at least in cases with detectable residual disease after transplant.
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Change history
31 March 2022
A Correction to this paper has been published: https://doi.org/10.1038/s41409-022-01652-3
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Acknowledgements
JCHB, AP, LG, TC, DM and IYA were involved in study design, analysis and writing of the paper. NZ was the data manager. All other co-authors contributed data to the study, critically revised the paper and approved the submitted and final version.
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All authors declare no competing financial interests. This study was not economically funded. The data that support the findings of this study are available on request from the corresponding author. The data are not publicly available due to privacy or ethical restrictions. This retrospective study was approved by the Chronic Malignancies Working Party (CMWP) of EBMT. Informed consent for inclusion in the EBMT registry was obtained in all patients.
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The original online version of this article was revised: Two members from that institution collaborated in our study but we didn’t choose the preferred one for the final version of the manuscript. The suggested coauthor is Dr Emmanouil Nikolousis (manos.nikolousis@nhs.net) in place of Shankara Paneesha (shankara.paneesha@nhs.net). Both of them have agreed on this change.
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Hernández-Boluda, JC., Pereira, A., Zinger, N. et al. Allogeneic hematopoietic cell transplantation in patients with myeloid/lymphoid neoplasm with FGFR1-rearrangement: a study of the Chronic Malignancies Working Party of EBMT. Bone Marrow Transplant 57, 416–422 (2022). https://doi.org/10.1038/s41409-021-01553-x
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DOI: https://doi.org/10.1038/s41409-021-01553-x
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