Allogeneic hematopoietic cell transplantation in patients with myeloid/lymphoid neoplasm with FGFR1-rearrangement: a study of the Chronic Malignancies Working Party of EBMT.
Hernández-Boluda, Juan-Carlos Pereira, Arturo Zinger, Nienke Gras, Luuk Martino, Rodrigo Nikolousis, Emmanouil Finke, Jürgen Chinea, Anabelle Rambaldi, Alessandro Robin, Marie
Hernández-Boluda, Juan-Carlos
Pereira, Arturo
Zinger, Nienke
Gras, Luuk
Martino, Rodrigo
Nikolousis, Emmanouil
Finke, Jürgen
Chinea, Anabelle
Rambaldi, Alessandro
Robin, Marie
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2022-01-23
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Abstract
llogeneic 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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Hernández-Boluda JC, Pereira A, Zinger N, Gras L, Martino R, Nikolousis E, Finke J, Chinea A, Rambaldi A, Robin M, Saccardi R, Natale A, Snowden JA, Tsirigotis P, Vallejo C, Wulf G, Xicoy B, Russo D, Maertens J, Daguindau E, Lenhoff S, Hayden P, Czerw T, McLornan DP, Yakoub-Agha I. 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. 2022 Mar;57(3):416-422. doi: 10.1038/s41409-021-01553-x. Epub 2022 Jan 23. Erratum in: Bone Marrow Transplant. 2022 Mar 31
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Corrigendum