Distinct biological subtypes of chronic GVHD after pediatric hematopoietic cell transplantation

B Bernard Ng (1Department of Statistics, Centre for Molecular Medicine and Therapeutics, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada) A Andrew C. Harris (2MSK Kids Stem Cell Transplantation and Cellular Therapies, Memorial Sloan Kettering Cancer Center, New York, NY) S Sayeh Abdossamadi (3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada) G Geraldine Aubert (4Repeat Diagnostics Inc, North Vancouver, BC, Canada) R Rajinder Bajwa (5Department of Pediatrics, Nationwide Children’s Hospital, Columbus, OH) M Monica Bhatia (6Pediatric Stem Cell Transplant Program, Morgan Stanley Children’s Hospital, Columbia University, New York, NY) H Henrique Bittencourt (7Pediatric Hematology-Oncology Division, Saint-Justine University Hospital Centre, Montreal, QC, Canada) N Nataliya P. Buxbaum (8Department of Pediatric Oncology, Roswell Park Comprehensive Cancer Center, Buffalo, NY) E Emi H. Caywood (9Nemours Children’s Health, Thomas Jefferson University, Wilmington, DE) S Sonali Chaudhury (11Department of Hematology, Oncology, Neuro-Oncology, and Stem Cell Transplantation, Ann & Robert H. Lurie Children’s Hospital, Northwestern University, Chicago, IL) J Joseph H. Chewning (12Division of Pediatric Hematology and Oncology, University of Alabama at Birmingham, Birmingham, AL) S Sung Won Choi (13Blood and Marrow Transplantation Program, University of Michigan, Ann Arbor, MI) A Ashley Chopek (14Manitoba Blood and Marrow Transplant Program, CancerCare Manitoba, University of Manitoba, Winnipeg, MB, Canada) J Julia Chu D Donald Coulter (16Division of Pediatric Hematology-Oncology, University of Nebraska Medical Center, Omaha, NE) S Shahinaz M. Gadalla R Richard T. Hogg (3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada) D David A. Jacobsohn (18Division of Blood and Marrow Transplantation, Children’s National Hospital, George Washington University School of Medicine and Health Sciences, Washington, DC) A Amanda K. Johnson (19Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, University of Utah/Primary Children’s Hospital, Salt Lake City, UT) M Michael Joyce (20Department of Pediatrics, Nemours Children’s Health, Jacksonville, FL) K Kimberly A. Kasow (21Department of Pediatric Bone Marrow Transplant, University of North Carolina at Chapel Hill, Chapel Hill, NC) M Michael Kent (22Center for Cancer and Blood Disorders, Atrium Health/Levine Children’s Hospital, Charlotte, NC) C Carrie L. Kitko (23Pediatric Stem Cell Transplant Program, Vanderbilt University Medical Center, Nashville, TN) D Donna Lau (3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada) A Anita Lawitschka (24Stem Cell Transplant Unit, St. Anna Children’s Hospital, Medical University of Vienna, Vienna, Austria) V Victor A. Lewis (25Department of Pediatric Oncology and Transplant, Alberta Children’s Hospital, University of Calgary, Calgary, AB, Canada) A Amanda M. Li (British Columbia Children’s Hospital, University of British Columbia, Vancouver) L Laura McLaughlin (26Department of Bone Marrow Transplantation and Cellular Therapeutics, Children’s Hospital Colorado, Denver, CO) D David Mitchell E Eneida R. Nemecek (28Department of Pediatric Blood and Marrow Transplantation, Doernbecher Children’s Hospital, Oregon Health & Science University, Portland, OR) V Vaishnavi Parthasarathy A Anna B. Pawlowska (30Department of Pediatric Hematology, Oncology and Hematopoietic Stem Cell Transplant, City of Hope, Duarte, CA) F Filip Pirsl (18Division of Blood and Marrow Transplantation, Children’s National Hospital, George Washington University School of Medicine and Health Sciences, Washington, DC) M Michael A. Pulsipher M Muna Qayed J Jacob Rozmus (3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada) S Süreyya Savaşan (32Department of Pediatric Hematology and Oncology, Children’s Hospital of Michigan, Detroit, MI) T Tal Schechter (33Department of Pediatric Hematology/Oncology, Children’s Hospital of Michigan, Central Michigan University, Mount Pleasant, Michigan) S Shalini Shenoy (34Department of Pediatric Hematology-Oncology, Saint Louis Children’s Hospital–Washington University School of Medicine, St. Louis, MO) A Alima Suleimenova (3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada) D Dong Jun Zheng (3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada) E Elena Ostroumov (3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada) A Andrew Gilman (35Medical Monitoring Support Center, ICON Biotech, Center for Cell and Gene Therapy, Charlotte, NC) R Ramon I. Klein Geltink (36Department of Pathology and Laboratory Medicine, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada) D Daniel Wolff G Geoffrey D. E. Cuvelier (25Department of Pediatric Oncology and Transplant, Alberta Children’s Hospital, University of Calgary, Calgary, AB, Canada) K Kirk R. Schultz (3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada)

Abstract

Abstract Chronic graft-versus-host-disease (cGVHD) is the primary nonrelapse limitation to a successful hematopoietic cell transplantation and is largely treated as a single biological entity. We hypothesized that there exist different biological subtypes of cGVHD. Using the Applied Biomarkers of Late Effects of Childhood Cancer (ABLE) network database, which is derived from the largest pediatric cGVHD cohort worldwide, we applied clustering analysis to subtype patients with cGVHD from the ABLE1.0 and 2.0 studies (51 patients with cGVHD and 158 with non-cGVHD). We found 3 distinct cGVHD subtypes: cGVHD-1 was characterized by an effector memory T-cell, cytotoxic natural killer cell, and early precursor B-cell predominant pattern; cGVHD-2 was phosphatidylcholine, cytokine, and plasma cell predominant; and cGVHD-3 had more naïve CD4+ T cells and naïve regulatory T cells, had later onset, and was the only subtype with measurable T-cell receptor excision circles. We partially replicated these subtypes using metabolomic data from a separate pediatric cohort of the Children’s Oncology Group trial ASCT0031 (33 patients with cGVHD and 39 with non-cGVHD). Furthermore, cGVHD-1 was associated with serotherapy (predominantly antithymocyte globulin) exposure, and cGVHD-3 was associated with receiving peripheral blood stem cells from donors, total body irradiation, and no previous acute GVHD. cGVHD-2 was associated with liver involvement and cGVHD-2 and -3 with de novo cGVHD. Overall, none of the subtypes were closely associated with organ involvement. Contrasting each subtype against patients with non-cGVHD, the 3 subtypes shared common markers, all of which were used in our previous cGVHD diagnostic classifier. These findings suggest the presence of distinct biological subtypes of cGVHD that may help guide therapeutic strategies.

Article Details

Journal Blood
Volume / Issue Vol. 147, Issue 3
Published January 15, 2026
Pages 241-253
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (47)

B

Bernard Ng

1Department of Statistics, Centre for Molecular Medicine and Therapeutics, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada

A

Andrew C. Harris

2MSK Kids Stem Cell Transplantation and Cellular Therapies, Memorial Sloan Kettering Cancer Center, New York, NY

S

Sayeh Abdossamadi

3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada

G

Geraldine Aubert

4Repeat Diagnostics Inc, North Vancouver, BC, Canada

R

Rajinder Bajwa

5Department of Pediatrics, Nationwide Children’s Hospital, Columbus, OH

M

Monica Bhatia

6Pediatric Stem Cell Transplant Program, Morgan Stanley Children’s Hospital, Columbia University, New York, NY

H

Henrique Bittencourt

7Pediatric Hematology-Oncology Division, Saint-Justine University Hospital Centre, Montreal, QC, Canada

N

Nataliya P. Buxbaum

8Department of Pediatric Oncology, Roswell Park Comprehensive Cancer Center, Buffalo, NY

E

Emi H. Caywood

9Nemours Children’s Health, Thomas Jefferson University, Wilmington, DE

S

Sonali Chaudhury

11Department of Hematology, Oncology, Neuro-Oncology, and Stem Cell Transplantation, Ann & Robert H. Lurie Children’s Hospital, Northwestern University, Chicago, IL

J

Joseph H. Chewning

12Division of Pediatric Hematology and Oncology, University of Alabama at Birmingham, Birmingham, AL

S

Sung Won Choi

13Blood and Marrow Transplantation Program, University of Michigan, Ann Arbor, MI

A

Ashley Chopek

14Manitoba Blood and Marrow Transplant Program, CancerCare Manitoba, University of Manitoba, Winnipeg, MB, Canada

J

Julia Chu

D

Donald Coulter

16Division of Pediatric Hematology-Oncology, University of Nebraska Medical Center, Omaha, NE

S

Shahinaz M. Gadalla

R

Richard T. Hogg

3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada

D

David A. Jacobsohn

18Division of Blood and Marrow Transplantation, Children’s National Hospital, George Washington University School of Medicine and Health Sciences, Washington, DC

A

Amanda K. Johnson

19Division of Pediatric Hematology, Oncology, and Bone Marrow Transplant, University of Utah/Primary Children’s Hospital, Salt Lake City, UT

M

Michael Joyce

20Department of Pediatrics, Nemours Children’s Health, Jacksonville, FL

K

Kimberly A. Kasow

21Department of Pediatric Bone Marrow Transplant, University of North Carolina at Chapel Hill, Chapel Hill, NC

M

Michael Kent

22Center for Cancer and Blood Disorders, Atrium Health/Levine Children’s Hospital, Charlotte, NC

C

Carrie L. Kitko

23Pediatric Stem Cell Transplant Program, Vanderbilt University Medical Center, Nashville, TN

D

Donna Lau

3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada

A

Anita Lawitschka

24Stem Cell Transplant Unit, St. Anna Children’s Hospital, Medical University of Vienna, Vienna, Austria

V

Victor A. Lewis

25Department of Pediatric Oncology and Transplant, Alberta Children’s Hospital, University of Calgary, Calgary, AB, Canada

A

Amanda M. Li

British Columbia Children’s Hospital, University of British Columbia, Vancouver

L

Laura McLaughlin

26Department of Bone Marrow Transplantation and Cellular Therapeutics, Children’s Hospital Colorado, Denver, CO

D

David Mitchell

E

Eneida R. Nemecek

28Department of Pediatric Blood and Marrow Transplantation, Doernbecher Children’s Hospital, Oregon Health & Science University, Portland, OR

V

Vaishnavi Parthasarathy

A

Anna B. Pawlowska

30Department of Pediatric Hematology, Oncology and Hematopoietic Stem Cell Transplant, City of Hope, Duarte, CA

F

Filip Pirsl

18Division of Blood and Marrow Transplantation, Children’s National Hospital, George Washington University School of Medicine and Health Sciences, Washington, DC

M

Michael A. Pulsipher

M

Muna Qayed

J

Jacob Rozmus

3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada

S

Süreyya Savaşan

32Department of Pediatric Hematology and Oncology, Children’s Hospital of Michigan, Detroit, MI

T

Tal Schechter

33Department of Pediatric Hematology/Oncology, Children’s Hospital of Michigan, Central Michigan University, Mount Pleasant, Michigan

S

Shalini Shenoy

34Department of Pediatric Hematology-Oncology, Saint Louis Children’s Hospital–Washington University School of Medicine, St. Louis, MO

A

Alima Suleimenova

3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada

D

Dong Jun Zheng

3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada

E

Elena Ostroumov

3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada

A

Andrew Gilman

35Medical Monitoring Support Center, ICON Biotech, Center for Cell and Gene Therapy, Charlotte, NC

R

Ramon I. Klein Geltink

36Department of Pathology and Laboratory Medicine, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada

D

Daniel Wolff

G

Geoffrey D. E. Cuvelier

25Department of Pediatric Oncology and Transplant, Alberta Children’s Hospital, University of Calgary, Calgary, AB, Canada

K

Kirk R. Schultz

3Michael Cuccione Childhood Cancer Research Program, British Columbia Children’s Hospital, University of British Columbia, Vancouver, BC, Canada