Genome-wide analysis defines genetic determinants of MPN subtypes and identifies a sex-specific association at <i>CDH22</i> / <i>CD40</i>

W William J. Tapper (1Faculty of Medicine, University of Southampton, Southampton, United Kingdom) A Ahmed A. Z. Dawoud (1Faculty of Medicine, University of Southampton, Southampton, United Kingdom) J Joannah Score (1Faculty of Medicine, University of Southampton, Southampton, United Kingdom) A Andrew J. Chase (1Faculty of Medicine, University of Southampton, Southampton, United Kingdom) E E. Joanna Baxter J Joanne Ewing (3Department of Haematology, Heartlands Hospital, Birmingham, United Kingdom) L Louise Wallis (4Department of Haematology, Royal Bournemouth Hospital, Bournemouth, United Kingdom) P Paola Guglielmelli (3Center for Research and Innovation of Myeloproliferative Neoplasms, AOU Careggi, University of Florence, Florence, Italy) D Dolors Colomer B Beatriz Bellosillo (7Hospital del Mar, Hospital del Mar Research Institute, Barcelona, Spain) M Montse Gomez (8Hospital Clínico Universitario, Instituto de Investigación Sanitaria, University of Valencia, Valencia, Spain) J Juan Carlos Hernández-Boluda (8Hospital Clínico Universitario, Instituto de Investigación Sanitaria, University of Valencia, Valencia, Spain) C Carlos Besses (7Hospital del Mar, Hospital del Mar Research Institute, Barcelona, Spain) F Francisco Cervantes (10Hematology Department, Hospital Clinic, IDIBAPS, University of Barcelona, Barcelona, Spain) S Steffen Koschmieder A Anthony R. Green A Andreas Reiter A Alessandro Vannucchi (5Center Research and Innovation of Myeloproliferative Neoplasms, Dipartimento di Medicina Sperimentale e Clinica, Azienda Ospedaliero Universitaria 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) N Nicholas C. P. Cross

Abstract

Abstract To identify genetic variants that influence myeloproliferative neoplasm (MPN) phenotypes, we undertook a 2-stage patient-only genome-wide association study. MPN subtypes (essential thrombocythemia [ET]; polycythemia vera [PV]) were compared with each other to healthy controls and stratified analyses was performed for chromosome 9p aberrations, JAK2 V617F mutation burden, and sex. The ET vs PV analysis identified known associations: (1) at HBS1L-MYB that increased ET risk (Pmeta = 7.93 × 10–6, odds ratio [OR] = 1.28) and reduced PV risk (Pmeta = 9.43 × 10–5, OR = 0.81) and (2) at GFI1B-GTF3C5 that predisposed to PV only (Pmeta = 1.43 × 10–9, OR = 1.38). Two further linked intronic variants, rs2425786 and rs2425788, at CDH22/CD40 were significant in females only (Pmeta = 2.67 × 10–8), with predisposition to PV (Pmeta = .0006, OR = 1.3) and reduction of ET risk (Pmeta = 7.82 × 10–5, OR = 0.75). A polygenic risk score consisting of 48 variants from 31 loci demonstrated moderate discriminative performance for ET and PV (area under the curve [AUC] = 0.718) and was improved by optimization for disease subtype (AUCET = 0.724 and AUCPV = 0.755). Overall, our results reveal that multiple germline variants influence MPN phenotype, with HBS1L-MYB and a novel sex-specific association with CDH22/CD40 being the strongest determinants.

Article Details

Journal Blood
Volume / Issue Vol. 146, Issue 26
Published December 25, 2025
Pages 3228-3233
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (20)

W

William J. Tapper

1Faculty of Medicine, University of Southampton, Southampton, United Kingdom

A

Ahmed A. Z. Dawoud

1Faculty of Medicine, University of Southampton, Southampton, United Kingdom

J

Joannah Score

1Faculty of Medicine, University of Southampton, Southampton, United Kingdom

A

Andrew J. Chase

1Faculty of Medicine, University of Southampton, Southampton, United Kingdom

E

E. Joanna Baxter

J

Joanne Ewing

3Department of Haematology, Heartlands Hospital, Birmingham, United Kingdom

L

Louise Wallis

4Department of Haematology, Royal Bournemouth Hospital, Bournemouth, United Kingdom

P

Paola Guglielmelli

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

D

Dolors Colomer

B

Beatriz Bellosillo

7Hospital del Mar, Hospital del Mar Research Institute, Barcelona, Spain

M

Montse Gomez

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

J

Juan Carlos Hernández-Boluda

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

C

Carlos Besses

7Hospital del Mar, Hospital del Mar Research Institute, Barcelona, Spain

F

Francisco Cervantes

10Hematology Department, Hospital Clinic, IDIBAPS, University of Barcelona, Barcelona, Spain

S

Steffen Koschmieder

A

Anthony R. Green

A

Andreas Reiter

A

Alessandro Vannucchi

5Center Research and Innovation of Myeloproliferative Neoplasms, Dipartimento di Medicina Sperimentale e Clinica, Azienda Ospedaliero Universitaria 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

N

Nicholas C. P. Cross