Research ExplorerPublicationsAllogeneic hematopoietic cell transplantation in patients wi...

Allogeneic hematopoietic cell transplantation in patients with CALR-mutated myelofibrosis: A study of the Chronic Malignancies Working Party of EBMT

Standard

Allogeneic hematopoietic cell transplantation in patients with CALR-mutated myelofibrosis: A study of the Chronic Malignancies Working Party of EBMT. / Hernández-Boluda, Juan Carlos; Eikema, Diderik-Jan; Koster, Linda; Kröger, Nicolaus; Robin, Marie; de Witte, Moniek; Finke, Jürgen; Finazzi, Maria Chiara; Broers, Annoek; Raida, Ludek; Schaap, Nicolaas; Chiusolo, Patrizia; Verbeek, Mareike; Hazenberg, Carin L E; Halaburda, Kazimierz; Kulagin, Aleksandr; Labussière-Wallet, Hélène; Gedde-Dahl, Tobias; Rabitsch, Werner; Raj, Kavita; Drozd-Sokolowska, Joanna; Battipaglia, Giorgia; Polverelli, Nicola; Czerw, Tomasz; Yakoub-Agha, Ibrahim; McLornan, Donal P.

In: BONE MARROW TRANSPL, Vol. 58, No. 12, 12.2023, p. 1357-1367.

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

Harvard

Hernández-Boluda, JC, Eikema, D-J, Koster, L, Kröger, N, Robin, M, de Witte, M, Finke, J, Finazzi, MC, Broers, A, Raida, L, Schaap, N, Chiusolo, P, Verbeek, M, Hazenberg, CLE, Halaburda, K, Kulagin, A, Labussière-Wallet, H, Gedde-Dahl, T, Rabitsch, W, Raj, K, Drozd-Sokolowska, J, Battipaglia, G, Polverelli, N, Czerw, T, Yakoub-Agha, I & McLornan, DP 2023, 'Allogeneic hematopoietic cell transplantation in patients with CALR-mutated myelofibrosis: A study of the Chronic Malignancies Working Party of EBMT', BONE MARROW TRANSPL, vol. 58, no. 12, pp. 1357-1367. https://doi.org/10.1038/s41409-023-02094-1

APA

Hernández-Boluda, J. C., Eikema, D-J., Koster, L., Kröger, N., Robin, M., de Witte, M., Finke, J., Finazzi, M. C., Broers, A., Raida, L., Schaap, N., Chiusolo, P., Verbeek, M., Hazenberg, C. L. E., Halaburda, K., Kulagin, A., Labussière-Wallet, H., Gedde-Dahl, T., Rabitsch, W., ... McLornan, D. P. (2023). Allogeneic hematopoietic cell transplantation in patients with CALR-mutated myelofibrosis: A study of the Chronic Malignancies Working Party of EBMT. BONE MARROW TRANSPL, 58(12), 1357-1367. https://doi.org/10.1038/s41409-023-02094-1

Vancouver

Hernández-Boluda JC, Eikema D-J, Koster L, Kröger N, Robin M, de Witte M et al. Allogeneic hematopoietic cell transplantation in patients with CALR-mutated myelofibrosis: A study of the Chronic Malignancies Working Party of EBMT. BONE MARROW TRANSPL. 2023 Dec;58(12):1357-1367. https://doi.org/10.1038/s41409-023-02094-1

Bibtex

@article{b13d2dc7a0314f07a2ebdecbd2715e22,
title = "Allogeneic hematopoietic cell transplantation in patients with CALR-mutated myelofibrosis: A study of the Chronic Malignancies Working Party of EBMT",
abstract = "Allogeneic hematopoietic cell transplantation (allo-HCT) is curative for myelofibrosis (MF) but assessing risk-benefit in individual patients is challenging. This complexity is amplified in CALR-mutated MF patients, as they live longer with conventional treatments compared to other molecular subtypes. We analyzed outcomes of 346 CALR-mutated MF patients who underwent allo-HCT in 123 EBMT centers between 2005 and 2019. After a median follow-up of 40 months, the estimated overall survival (OS) rates at 1, 3, and 5 years were 81%, 71%, and 63%, respectively. Patients receiving busulfan-containing regimens achieved a 5-year OS rate of 71%. Non-relapse mortality (NRM) at 1, 3, and 5 years was 16%, 22%, and 26%, respectively, while the incidence of relapse/progression was 11%, 15%, and 17%, respectively. Multivariate analysis showed that older age correlated with worse OS, while primary MF and HLA mismatched transplants had a near-to-significant trend to decreased OS. Comparative analysis between CALR- and JAK2-mutated MF patients adjusting for confounding factors revealed better OS, lower NRM, lower relapse, and improved graft-versus-host disease-free and relapse-free survival (GRFS) in CALR-mutated patients. These findings confirm the improved prognosis associated with CALR mutation in allo-HCT and support molecular profiling in prognostic scoring systems to predict OS after transplantation in MF.",
keywords = "Humans, Primary Myelofibrosis/genetics, Transplantation, Homologous/adverse effects, Retrospective Studies, Hematopoietic Stem Cell Transplantation/adverse effects, Neoplasms/complications, Chronic Disease, Recurrence, Transplantation Conditioning/adverse effects, Graft vs Host Disease/etiology",
author = "Hern{\'a}ndez-Boluda, {Juan Carlos} and Diderik-Jan Eikema and Linda Koster and Nicolaus Kr{\"o}ger and Marie Robin and {de Witte}, Moniek and J{\"u}rgen Finke and Finazzi, {Maria Chiara} and Annoek Broers and Ludek Raida and Nicolaas Schaap and Patrizia Chiusolo and Mareike Verbeek and Hazenberg, {Carin L E} and Kazimierz Halaburda and Aleksandr Kulagin and H{\'e}l{\`e}ne Labussi{\`e}re-Wallet and Tobias Gedde-Dahl and Werner Rabitsch and Kavita Raj and Joanna Drozd-Sokolowska and Giorgia Battipaglia and Nicola Polverelli and Tomasz Czerw and Ibrahim Yakoub-Agha and McLornan, {Donal P}",
note = "{\textcopyright} 2023. The Author(s), under exclusive licence to Springer Nature Limited.",
year = "2023",
month = dec,
doi = "10.1038/s41409-023-02094-1",
language = "English",
volume = "58",
pages = "1357--1367",
journal = "BONE MARROW TRANSPL",
issn = "0268-3369",
publisher = "NATURE PUBLISHING GROUP",
number = "12",

}

RIS

TY - JOUR

T1 - Allogeneic hematopoietic cell transplantation in patients with CALR-mutated myelofibrosis: A study of the Chronic Malignancies Working Party of EBMT

AU - Hernández-Boluda, Juan Carlos

AU - Eikema, Diderik-Jan

AU - Koster, Linda

AU - Kröger, Nicolaus

AU - Robin, Marie

AU - de Witte, Moniek

AU - Finke, Jürgen

AU - Finazzi, Maria Chiara

AU - Broers, Annoek

AU - Raida, Ludek

AU - Schaap, Nicolaas

AU - Chiusolo, Patrizia

AU - Verbeek, Mareike

AU - Hazenberg, Carin L E

AU - Halaburda, Kazimierz

AU - Kulagin, Aleksandr

AU - Labussière-Wallet, Hélène

AU - Gedde-Dahl, Tobias

AU - Rabitsch, Werner

AU - Raj, Kavita

AU - Drozd-Sokolowska, Joanna

AU - Battipaglia, Giorgia

AU - Polverelli, Nicola

AU - Czerw, Tomasz

AU - Yakoub-Agha, Ibrahim

AU - McLornan, Donal P

N1 - © 2023. The Author(s), under exclusive licence to Springer Nature Limited.

PY - 2023/12

Y1 - 2023/12

N2 - Allogeneic hematopoietic cell transplantation (allo-HCT) is curative for myelofibrosis (MF) but assessing risk-benefit in individual patients is challenging. This complexity is amplified in CALR-mutated MF patients, as they live longer with conventional treatments compared to other molecular subtypes. We analyzed outcomes of 346 CALR-mutated MF patients who underwent allo-HCT in 123 EBMT centers between 2005 and 2019. After a median follow-up of 40 months, the estimated overall survival (OS) rates at 1, 3, and 5 years were 81%, 71%, and 63%, respectively. Patients receiving busulfan-containing regimens achieved a 5-year OS rate of 71%. Non-relapse mortality (NRM) at 1, 3, and 5 years was 16%, 22%, and 26%, respectively, while the incidence of relapse/progression was 11%, 15%, and 17%, respectively. Multivariate analysis showed that older age correlated with worse OS, while primary MF and HLA mismatched transplants had a near-to-significant trend to decreased OS. Comparative analysis between CALR- and JAK2-mutated MF patients adjusting for confounding factors revealed better OS, lower NRM, lower relapse, and improved graft-versus-host disease-free and relapse-free survival (GRFS) in CALR-mutated patients. These findings confirm the improved prognosis associated with CALR mutation in allo-HCT and support molecular profiling in prognostic scoring systems to predict OS after transplantation in MF.

AB - Allogeneic hematopoietic cell transplantation (allo-HCT) is curative for myelofibrosis (MF) but assessing risk-benefit in individual patients is challenging. This complexity is amplified in CALR-mutated MF patients, as they live longer with conventional treatments compared to other molecular subtypes. We analyzed outcomes of 346 CALR-mutated MF patients who underwent allo-HCT in 123 EBMT centers between 2005 and 2019. After a median follow-up of 40 months, the estimated overall survival (OS) rates at 1, 3, and 5 years were 81%, 71%, and 63%, respectively. Patients receiving busulfan-containing regimens achieved a 5-year OS rate of 71%. Non-relapse mortality (NRM) at 1, 3, and 5 years was 16%, 22%, and 26%, respectively, while the incidence of relapse/progression was 11%, 15%, and 17%, respectively. Multivariate analysis showed that older age correlated with worse OS, while primary MF and HLA mismatched transplants had a near-to-significant trend to decreased OS. Comparative analysis between CALR- and JAK2-mutated MF patients adjusting for confounding factors revealed better OS, lower NRM, lower relapse, and improved graft-versus-host disease-free and relapse-free survival (GRFS) in CALR-mutated patients. These findings confirm the improved prognosis associated with CALR mutation in allo-HCT and support molecular profiling in prognostic scoring systems to predict OS after transplantation in MF.

KW - Humans

KW - Primary Myelofibrosis/genetics

KW - Transplantation, Homologous/adverse effects

KW - Retrospective Studies

KW - Hematopoietic Stem Cell Transplantation/adverse effects

KW - Neoplasms/complications

KW - Chronic Disease

KW - Recurrence

KW - Transplantation Conditioning/adverse effects

KW - Graft vs Host Disease/etiology

U2 - 10.1038/s41409-023-02094-1

DO - 10.1038/s41409-023-02094-1

M3 - SCORING: Journal article

C2 - 37679647

VL - 58

SP - 1357

EP - 1367

JO - BONE MARROW TRANSPL

JF - BONE MARROW TRANSPL

SN - 0268-3369

IS - 12

ER -