Role of allo-HCT in “nonclassical” MPNs and MDS/MPNs: recommendations from the PH&G Committee and the CMWP of the EBMT

N Nicola Polverelli (1Unit of Bone Marrow Transplantation and Cellular Therapies - Fondazione IRCCS Policlinico San Matteo, Pavia, Italy) J Juan Carlos Hernández-Boluda (8Hospital Clínico Universitario, Instituto de Investigación Sanitaria, University of Valencia, Valencia, Spain) F Francesco Onida (22ASST Fatebenefratelli-Sacco, University Milan, Milan, Italy) C Carmelo Gurnari (1Translational Hematology & Oncology Research, Cleveland Clinic, Cleveland, OH) K Kavita Raj (20University College London Hospitals NHS Trust, London, United Kingdom) T Tomasz Czerw (24Hematology Department, Maria Skłodowska-Curie National Research Institute of Oncology, Gliwice, Poland) M Michelle Kenyon (1King's College Hospital NHS Foundation Trust, Haematology, London, United Kingdom) M Marie Robin (9Hôpital Saint Louis (APHP), Service d'hématologie, Paris, France) K Katja Sockel (4Department of Internal Medicine I, University Hospital Dresden, Dresden University of Technology, Dresden, Germany) A Annalisa Ruggeri (8San Raffaele Scientific Institute, Milan, Italy) I Isabel Sanchez-Ortega (6EBMT Executive Office, Barcelona, Spain) D Daniel A. Arber (The University of Chicago Medicine) L Luca Arcaini (Fondazione IRCCS Policlinico San Matteo, Pavia, Italy) F Fernando Barroso Duarte (16Hospital Universitário Walter Cantídio, Fortaleza, Brazil) G Giorgia Battipaglia (5Department of Clinical Medicine and Surgery, University Federico II, Hematology, Naples, Italy) Y Yves Chalandon (25Swiss Group for Clinical Cancer Research, Bern, Switzerland) F Fabio Ciceri (Vita-Salute San Raffaele University, Milan) N Nicholas C. P. Cross J Joanna Drozd-Sokolowska (1Medical University of Warsaw, Department of Hematology, Transplantation and Internal Medicine, Warsaw, Poland) V Vaneuza Funke (7Hospital das Clínicas - Universidade Federal do Paraná, Curitiba, Brazil) N Nico Gagelmann (From the Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.) N Naseema Gangat (4Mayo Clinic, Scottsdale, United States) J Jason Gotlib (15Division of Hematology, Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA) P Paola Guglielmelli (3Center for Research and Innovation of Myeloproliferative Neoplasms, AOU Careggi, University of Florence, Florence, Italy) C Claire Harrison (11Department of Haematology, Guy’s and St Thomas’ National Health Service Foundation Trust, London, United Kingdom) G Gabriela Hobbs (Massachusetts General Hospital, Boston, Massachusetts, United States) T Tania Jain (1Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, MD) J Joseph D. Khoury (3Department of Pathology, Microbiology, and Immunology, University of Nebraska Medical Center, Omaha, NE) J Jean Jacques Kiladjian (8Department of Haematology, King's College Hospital, London, United Kingdom) N Nicolaus Kröger (From the Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.) L Luca Malcovati M Massimo Martino (12Stem Cell Transplant and Cellular Therapies Unit, Great Metropolitan Hospital “Bianchi-Melacrino-Morelli”, Reggio Calabria, Italy) R Ruben Mesa (8Atrium Health Wake Forest, Winston, United States) A Attilio Orazi (9Texas Tech University Health Sciences Center, El Paso, TX, United States) E Eric Padron (Moffitt Cancer Cancer and Research Institute, Tampa, Florida, United States) F Francesca Palandri (2Seragnoli Hematology Institute, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy) F Francesco Passamonti (University of Milan, Milan) M Mrinal M. Patnaik (Division of Hematology, Department of Internal Medicine, Mayo Clinic) N Naveen Pemmaraju (The University of Texas MD Anderson Cancer Center, Houston, Texas, United States) D Deepti H. Radia (26Department of Haematology, Guy's & St Thomas' NHS Foundation Trust, London, United Kingdom) A Andreas Reiter D Domenico Russo (Institute of Endotypes in Oncology, Metabolism and Immunology “G. Salvatore,” National Research Council of Italy) C Christof Scheid (12Hospital Barmherzige Brueder Regensburg, Clinic for Oncology and Hematology, Regensburg, Germany) A Ayalew Tefferi (4Mayo Clinic, Scottsdale, United States) A Alessandro M. Vannucchi (3Center for Research and Innovation of Myeloproliferative Neoplasms, AOU Careggi, University of Florence, Florence, Italy) D Daniel H. Wiseman (7Cancer Research Division, Instituto de Investigación Sanitaria del Principado de Asturias, Oviedo, Spain) I Ibrahim Yakoub-Agha (9CHU de Lille, Univ Lille, INSERM U1286, Infinite, 59000 Lille, Lille, France) D Donal P. McLornan (14Department of Haematology and Stem Cell Transplantation, University College London Hospitals NHS Foundation Trust, London, United Kingdom)

Abstract

Abstract “Nonclassical” 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. To address this gap, the European Society for Blood and Marrow Transplantation (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 fourth edition of the (2016) World Health Organization 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, chronic eosinophilic leukemia (CEL)/CEL, not otherwise specified (CEL-NOS), myeloid/lymphoid neoplasm with eosinophilia and tyrosine kinase gene fusions with FGFR1, JAK2, ABL1, and FLT3 rearrangements, MDS/MPN with neutrophilia/atypical chronic myeloid leukemia, and MDS/MPN, NOS. For patients with MPN, NOS/MPN unclassifiable, standard recommendations for myelofibrosis should be applied. Similarly, in MDS/MPN with thrombocytosis, transplantation is recommended on the basis of 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.

Article Details

Journal Blood
Volume / Issue Vol. 145, Issue 22
Published May 29, 2025
Pages 2561-2573
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (48)

N

Nicola Polverelli

1Unit of Bone Marrow Transplantation and Cellular Therapies - Fondazione IRCCS Policlinico San Matteo, Pavia, Italy

J

Juan Carlos Hernández-Boluda

8Hospital Clínico Universitario, Instituto de Investigación Sanitaria, University of Valencia, Valencia, Spain

F

Francesco Onida

22ASST Fatebenefratelli-Sacco, University Milan, Milan, Italy

C

Carmelo Gurnari

1Translational Hematology & Oncology Research, Cleveland Clinic, Cleveland, OH

K

Kavita Raj

20University College London Hospitals NHS Trust, London, United Kingdom

T

Tomasz Czerw

24Hematology Department, Maria Skłodowska-Curie National Research Institute of Oncology, Gliwice, Poland

M

Michelle Kenyon

1King's College Hospital NHS Foundation Trust, Haematology, London, United Kingdom

M

Marie Robin

9Hôpital Saint Louis (APHP), Service d'hématologie, Paris, France

K

Katja Sockel

4Department of Internal Medicine I, University Hospital Dresden, Dresden University of Technology, Dresden, Germany

A

Annalisa Ruggeri

8San Raffaele Scientific Institute, Milan, Italy

I

Isabel Sanchez-Ortega

6EBMT Executive Office, Barcelona, Spain

D

Daniel A. Arber

The University of Chicago Medicine

L

Luca Arcaini

Fondazione IRCCS Policlinico San Matteo, Pavia, Italy

F

Fernando Barroso Duarte

16Hospital Universitário Walter Cantídio, Fortaleza, Brazil

G

Giorgia Battipaglia

5Department of Clinical Medicine and Surgery, University Federico II, Hematology, Naples, Italy

Y

Yves Chalandon

25Swiss Group for Clinical Cancer Research, Bern, Switzerland

F

Fabio Ciceri

Vita-Salute San Raffaele University, Milan

N

Nicholas C. P. Cross

J

Joanna Drozd-Sokolowska

1Medical University of Warsaw, Department of Hematology, Transplantation and Internal Medicine, Warsaw, Poland

V

Vaneuza Funke

7Hospital das Clínicas - Universidade Federal do Paraná, Curitiba, Brazil

N

Nico Gagelmann

From the Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.

N

Naseema Gangat

4Mayo Clinic, Scottsdale, United States

J

Jason Gotlib

15Division of Hematology, Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA

P

Paola Guglielmelli

3Center for Research and Innovation of Myeloproliferative Neoplasms, AOU Careggi, University of Florence, Florence, Italy

C

Claire Harrison

11Department of Haematology, Guy’s and St Thomas’ National Health Service Foundation Trust, London, United Kingdom

G

Gabriela Hobbs

Massachusetts General Hospital, Boston, Massachusetts, United States

T

Tania Jain

1Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, MD

J

Joseph D. Khoury

3Department of Pathology, Microbiology, and Immunology, University of Nebraska Medical Center, Omaha, NE

J

Jean Jacques Kiladjian

8Department of Haematology, King's College Hospital, London, United Kingdom

N

Nicolaus Kröger

From the Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.

L

Luca Malcovati

M

Massimo Martino

12Stem Cell Transplant and Cellular Therapies Unit, Great Metropolitan Hospital “Bianchi-Melacrino-Morelli”, Reggio Calabria, Italy

R

Ruben Mesa

8Atrium Health Wake Forest, Winston, United States

A

Attilio Orazi

9Texas Tech University Health Sciences Center, El Paso, TX, United States

E

Eric Padron

Moffitt Cancer Cancer and Research Institute, Tampa, Florida, United States

F

Francesca Palandri

2Seragnoli Hematology Institute, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy

F

Francesco Passamonti

University of Milan, Milan

M

Mrinal M. Patnaik

Division of Hematology, Department of Internal Medicine, Mayo Clinic

N

Naveen Pemmaraju

The University of Texas MD Anderson Cancer Center, Houston, Texas, United States

D

Deepti H. Radia

26Department of Haematology, Guy's & St Thomas' NHS Foundation Trust, London, United Kingdom

A

Andreas Reiter

D

Domenico Russo

Institute of Endotypes in Oncology, Metabolism and Immunology “G. Salvatore,” National Research Council of Italy

C

Christof Scheid

12Hospital Barmherzige Brueder Regensburg, Clinic for Oncology and Hematology, Regensburg, Germany

A

Ayalew Tefferi

4Mayo Clinic, Scottsdale, United States

A

Alessandro M. Vannucchi

3Center for Research and Innovation of Myeloproliferative Neoplasms, AOU Careggi, University of Florence, Florence, Italy

D

Daniel H. Wiseman

7Cancer Research Division, Instituto de Investigación Sanitaria del Principado de Asturias, Oviedo, Spain

I

Ibrahim Yakoub-Agha

9CHU de Lille, Univ Lille, INSERM U1286, Infinite, 59000 Lille, Lille, France

D

Donal P. McLornan

14Department of Haematology and Stem Cell Transplantation, University College London Hospitals NHS Foundation Trust, London, United Kingdom