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Role of allo-HCT in "non-classical" MPNs and MDS/MPNs: recommendations from the PH&G Committee and the CMWP of the EBMT

Role of allo-HCT in "non-classical" MPNs and MDS/MPNs: recommendations from the PH&G Committee and the CMWP of the EBMT
Role of allo-HCT in "non-classical" MPNs and MDS/MPNs: recommendations from the PH&G Committee and the CMWP of the EBMT
'Non-classical' Myeloproliferative Neoplasms (MPNs) and Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPNs) represent a heterogeneous group of malignancies characterized by a wide range of clinical manifestations. Unlike classical MPNs, there is no standardized management approach for these conditions, particularly concerning the indications for and management of allogeneic hematopoietic cell transplantation (HCT). To address this gap, the EBMT Practice Harmonization and Guidelines (PH&G) Committee and the Chronic Malignancies Working Party (CMWP) have collaborated to develop shared guidelines aimed at optimizing the selection and management of patients with these rare forms of neoplasms. A comprehensive review of the literature from the publication of the revised 4th edition of the (2016) WHO classification onward was conducted. A multidisciplinary group of experts in the field convened to produce this document, which was developed through multiple rounds of draft circulation. Key recommendations include the early identification of potential transplant candidates, particularly in cases of chronic neutrophilic leukemia (CNL), chronic eosinophilic leukemia (CEL) / CEL, not otherwise specified (CEL-NOS), myeloid/lymphoid neoplasm with eosinophilia and tyrosine kinase gene fusions (MLN-TK) with FGFR1, JAK2, ABL1, and FLT3 rearrangements, MDS/MPN with neutrophilia / atypical chronic myeloid leukemia (aCML), and MDS/MPN, NOS. For patients with MPN, NOS / MPN unclassifiable, standard recommendations for myelofibrosis (MF) should be applied. Similarly, in MDS/MPN with thrombocytosis, transplantation is recommended based on established MDS guidelines. Given the current lack of robust evidence, this document will serve as a valuable resource to guide future research activities, providing a framework for addressing critical unanswered questions and advancing the field.
0006-4971
2561-2573
Polverelli, Nicola
e8d70d09-d373-4115-8d09-b80c2589f284
Hernandez Boluda, Juan Carlos
ed9707fd-4e15-4903-8b35-4dc14c0ea735
Onida, Francesco
6e96d434-1c93-4baa-9c42-25f9dbdf3679
Gurnari, Carmelo
815cd21e-dc79-4fe8-a130-85500e09bdd5
Raj, Kavita
cbc578ee-05b1-4cd4-bf6d-3535de712dad
Czerw, Tomasz
4a9bf539-9ebd-4e9a-b751-f14f07946629
Kenyon, Michelle
52e6c237-7aec-4a91-b8d6-69fdb4318a75
Robin, Marie
50defd60-1c46-4931-b342-bf488eb0a4ae
Sockel, Katja
460b5ed9-1312-4e76-8139-8707dcc78f8f
Ruggeri, Annalisa
ecc9471e-ee4d-458e-ac7a-e24bd789fd9e
Cross, Nicholas C.P.
f87650da-b908-4a34-b31b-d62c5f186fe4
et al
Polverelli, Nicola
e8d70d09-d373-4115-8d09-b80c2589f284
Hernandez Boluda, Juan Carlos
ed9707fd-4e15-4903-8b35-4dc14c0ea735
Onida, Francesco
6e96d434-1c93-4baa-9c42-25f9dbdf3679
Gurnari, Carmelo
815cd21e-dc79-4fe8-a130-85500e09bdd5
Raj, Kavita
cbc578ee-05b1-4cd4-bf6d-3535de712dad
Czerw, Tomasz
4a9bf539-9ebd-4e9a-b751-f14f07946629
Kenyon, Michelle
52e6c237-7aec-4a91-b8d6-69fdb4318a75
Robin, Marie
50defd60-1c46-4931-b342-bf488eb0a4ae
Sockel, Katja
460b5ed9-1312-4e76-8139-8707dcc78f8f
Ruggeri, Annalisa
ecc9471e-ee4d-458e-ac7a-e24bd789fd9e
Cross, Nicholas C.P.
f87650da-b908-4a34-b31b-d62c5f186fe4

et al (2025) Role of allo-HCT in "non-classical" MPNs and MDS/MPNs: recommendations from the PH&G Committee and the CMWP of the EBMT. Blood, 145 (22), 2561-2573, [2024028000]. (doi:10.1182/blood.2024028000).

Record type: Review

Abstract

'Non-classical' Myeloproliferative Neoplasms (MPNs) and Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPNs) represent a heterogeneous group of malignancies characterized by a wide range of clinical manifestations. Unlike classical MPNs, there is no standardized management approach for these conditions, particularly concerning the indications for and management of allogeneic hematopoietic cell transplantation (HCT). To address this gap, the EBMT Practice Harmonization and Guidelines (PH&G) Committee and the Chronic Malignancies Working Party (CMWP) have collaborated to develop shared guidelines aimed at optimizing the selection and management of patients with these rare forms of neoplasms. A comprehensive review of the literature from the publication of the revised 4th edition of the (2016) WHO classification onward was conducted. A multidisciplinary group of experts in the field convened to produce this document, which was developed through multiple rounds of draft circulation. Key recommendations include the early identification of potential transplant candidates, particularly in cases of chronic neutrophilic leukemia (CNL), chronic eosinophilic leukemia (CEL) / CEL, not otherwise specified (CEL-NOS), myeloid/lymphoid neoplasm with eosinophilia and tyrosine kinase gene fusions (MLN-TK) with FGFR1, JAK2, ABL1, and FLT3 rearrangements, MDS/MPN with neutrophilia / atypical chronic myeloid leukemia (aCML), and MDS/MPN, NOS. For patients with MPN, NOS / MPN unclassifiable, standard recommendations for myelofibrosis (MF) should be applied. Similarly, in MDS/MPN with thrombocytosis, transplantation is recommended based on established MDS guidelines. Given the current lack of robust evidence, this document will serve as a valuable resource to guide future research activities, providing a framework for addressing critical unanswered questions and advancing the field.

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Role of allo-HCT in "non-classical" MPNs and MDS/MPNs: recommendations from the PH&G Committee and the CMWP of the EBMT - Accepted Manuscript
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Accepted/In Press date: 23 February 2025
e-pub ahead of print date: 19 March 2025
Published date: 29 May 2025

Identifiers

Local EPrints ID: 500720
URI: http://eprints.soton.ac.uk/id/eprint/500720
ISSN: 0006-4971
PURE UUID: 89aa2685-3d53-475b-a73c-b8b40dbb04bf
ORCID for Nicholas C.P. Cross: ORCID iD orcid.org/0000-0001-5481-2555

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Date deposited: 12 May 2025 16:33
Last modified: 28 Aug 2025 01:41

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Contributors

Author: Nicola Polverelli
Author: Juan Carlos Hernandez Boluda
Author: Francesco Onida
Author: Carmelo Gurnari
Author: Kavita Raj
Author: Tomasz Czerw
Author: Michelle Kenyon
Author: Marie Robin
Author: Katja Sockel
Author: Annalisa Ruggeri
Corporate Author: et al

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