Project EVOLVE: an international analysis of postimmunotherapy lineage switch, an emergent form of relapse in leukemia

S Sara K. Silbert A Alexander W. Rankin (1Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD) C Chloe N. Hoang (1Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD) A Alexandra Semchenkova (2Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia) R Regina M. Myers E Elena Zerkalenkova (4Laboratory of Cytogenetics and Molecular Genetics, Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia) H Hao-Wei Wang A Alexandra E. Kovach (6Department of Pathology and Laboratory Medicine, Children's Hospital Los Angeles, Los Angeles, CA) C Constance M. Yuan (2Laboratory of Pathology, National Cancer Institute, National Institutes of Health, Bethesda, MD) D Dana Delgado Colon (2National Institutes of Health, Laboratory of Pathology, NCI, Bethesda, United States) L Loïc Vasseur (2Hôpital St Louis, Adolescents and young adults department, Paris, France) A Alex Bataller (2Division of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX) S Samuel John K Kaylyn Utley Lyons (10Children's Hospital Colorado, University of Colorado School of Medicine, Denver, CO) B Barbara Friedes (3Department of Pediatrics and the Center for Childhood Cancer Research, Children’s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA) A Anna Alonso-Saladrigues (11Leukemia and Lymphoma Department, Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, Barcelona, Spain) H Hisham Abdel-Azim (1Division of Transplant/Cell Therapy and Hematological Malignancies, Cancer Center, Departments of Pediatrics and Medicine, Loma Linda University School of Medicine, Children Hospital and Medical Center, Loma Linda, United States) E Estelle Balducci (Université Paris Cité) A Ahmed Assim Aljudi (16Department of Pathology, Children's Healthcare of Atlanta, Atlanta, GA) M Marie Balsat (9Service d’Hématologie, Hospices Civils de Lyon, Hôpital Lyon Sud, Pierre-Bénite, France) D D. Nathan Biery (1Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD) A Aghiad Chamdin (18Pediatric Hematology & Oncology, Children's Hospital of Michigan/Central Michigan University, Mount Pleasant, MI) B Bill H. Chang (19Division of Pediatric Hematology and Oncology, Doernbecher Children's Hospital at Oregon Health & Science University, Portland, OR) R Raymund S. Cuevo (20Inova Schar Cancer Institute, Fairfax, VA) B Barbara De Moerloose D David S. Dickens (19Division of Pediatric Hematology/Oncology, University of Iowa Stead Family Children’s Hospital, Iowa City, IA) U Ulrich Duffner (2Section of Pediatric Bone Marrow Transplantation and Cellular Therapy, Helen DeVos Children's Hospital, Grand Rapids, United States) N Nicolas Duployez (Unité Mixte de Recherche (UMR) 9020-UMR-S 1277-Canther-Cancer Heterogeneity, Plasticity and Resistance to Therapies, Institut de Recherche contre le Cancer de Lille, University of Lille, CNRS, Inserm, Centre Hospitalier Universitaire Lille, Lille, France) F Firas El Chaer M Michelle Ann Elliott (26Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, MN) G Gabriele Escherich S Sneha Fernandes (2Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom) M Mandi R. Fitzjohn (10Children's Hospital Colorado, University of Colorado School of Medicine, Denver, CO) Z Zhubin Gahvari (3University of Wisconsin-Madison - Carbone Cancer Center, Division of Hematology, Oncology, Palliative Care, Madison, United States) S Stephan A. Grupp R Rui Rochelle He (30Department of Pathology, Inova Fairfax Medical Campus, Inova Health System, Falls Church, VA) C Cynthia Harrison (1Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD) C Christopher B. Hergott (1Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA) E Emily M. Hsieh (33Division of Hematology/Oncology/Transplantation and Cellular Therapy, Cancer and Blood Disease Institute, Children’s Hospital Los Angeles, Norris Comprehensive Cancer Center, Keck School of Medicine, University of Southern California, Los Angeles, CA) A Annette S. Kim (1Department of Pathology, University of Michigan, Ann Arbor, MI) D Dennis J. Kuo (35Division of Pediatric Hematology-Oncology, University of California San Diego/Rady Children's Hospital San Diego, San Diego, CA) D Daniel P. Larson (36Division of Hematopathology, Mayo Clinic, Rochester, MN) B Benjamin J. Lee (37Department of Pharmacy, Chao Family Comprehensive Cancer Center, University of California Irvine Health, Orange, CA) T Thibaut Leguay R R. Coleman Lindsley (Dana-Farber Cancer Institute, Boston, Massachusetts, United States) A Abhishek A. Mangaonkar (26Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, MN) K Kerstin Mezger (40Clinic of Pediatric Hematology and Oncology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany) H Holly L. Pacenta (41Division of Hematology and Oncology, Cook Children's Medical Center, Fort Worth, TX) J Jing Pan M Marlie Provost (43HCA HealthONE, Rocky Mountain Hospital for Children, Denver, CO) L Latika Puri (44Department of Hematology, Loma Linda University Children’s Hospital, Loma Linda, CA) S Sunil S. Raikar A Armando Martinez (5Flow Cytometry Unit, Laboratory of Pathology, National Cancer Institute, National Institutes of Health, Bethesda, MD) I Isabella Bristol (5Flow Cytometry Unit, Laboratory of Pathology, National Cancer Institute, National Institutes of Health, Bethesda, MD) K Kyle Murphy (5Flow Cytometry Unit, Laboratory of Pathology, National Cancer Institute, National Institutes of Health, Bethesda, MD) L Lauren Reiman (70Keck School of Medicine, University of Southern California, Los Angeles, CA) M Michele Redell (3Baylor College of Medicine, Pediatrics, Houston, United States) K Kelly Reed (47University of Virginia School of Nursing, Charlottesville, VA) G Gabrielle Roth-Guepin (2Fi LMC, Lyon, France) J Jeremy Rubinstein (33Cincinnati Children's Hospital Medical Center, Division of Oncology, Cincinnati, United States) S Süreyya Savaşan (32Department of Pediatric Hematology and Oncology, Children’s Hospital of Michigan, Detroit, MI) K Kristian Schafernak (52Division of Pathology and Laboratory Medicine, Phoenix Children's Hospital, Phoenix, AZ) A Alexandra Stevens (1Texas Children's Hospital, Baylor College of Medicine, Department of Pediatrics, Section of Hematology and Oncology, Houston, United States) A Aimee Talleur N Naomi Torres Carapia (55Children’s Hospital Los Angeles, Los Angeles, CA) J Jacques Vargaftig A Anant Vatsayan (7Children's Hospital, Washington, United States) M Matthias Wölfl (58Pediatric Oncology, Children's Hospital, University Hospital Würzburg, Würzburg, Germany) L Liping Zhao (School of Life Science and Medicine) S Susana Rives (12Institut de Recerca Sant Joan de Déu, Barcelona, Spain) V Vanessa A. Fabrizio (61Pediatric Bone Marrow Transplant/Cellular Therapies, University of Colorado Anschutz/Children's Hospital Colorado, Denver, CO) K Koji Sasaki (1The University of Texas MD Anderson Cancer Center, Houston, TX) I Ibrahim Aldoss N Nicolas Boissel S Susan R. Rheingold K Kara L. Davis S Sara Ghorashian E Elad Jacoby (1Gray Faculty of Medical and Health Sciences, Tel Aviv University, Tel Aviv, Israel) A Alexander Popov A Adam J. Lamble N Nirali N. Shah

Abstract

Abstract Lineage switch (LS), defined as the immunophenotypic transformation of acute leukemia, has emerged as a mechanism of relapse after antigen-targeted immunotherapy, which is associated with dismal outcomes. Through an international collaborative effort, we identified cases of LS after a host of antigen-targeted therapies (eg, CD19, CD22, CD38, and CD7), described how LS was diagnosed, reviewed treatment approaches, and analyzed overall outcomes for this form of postimmunotherapy relapse. Collectively, 75 cases of LS were evaluated, including 53 (70.7%) cases of B-cell acute lymphoblastic leukemia (B-ALL) transforming to acute myeloid leukemia (AML), 17 (22.7%) cases of B-ALL transforming to mixed phenotypic acute leukemia (MPAL)/acute leukemias of ambiguous lineage (ALAL), and 5 (6.7%) cases of rare LS presentation (ie, T-cell ALL to AML). An additional 10 cases with incomplete changes in immunophenotype, referred to as “lineage drift” were also described. With a primary focus on the 70 cases of LS from B-ALL to AML or MPAL/ALAL, LS emerged at a median of 1.5 months (range, 0-36.5) after immunotherapy, with 81.4% presenting with LS within the first 6 months from the most proximal immunotherapy. Although most involved KMT2A rearrangements (n = 45, 64.3%), other rare cytogenetic and/or molecular alterations were uniquely observed. Treatment outcomes were generally poor, with remission rates of <40%. The median overall survival after LS diagnosis was 4.8 months. Outcomes were similarly poor for those with rare immunophenotypes of LS or lineage drift. This global initiative robustly categorizes lineage changes after immunotherapy and, through enhanced understanding, establishes a foundation for improving outcomes of LS.

Article Details

Journal Blood
Volume / Issue Vol. 146, Issue 4
Published July 24, 2025
Pages 437-455
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (81)

S

Sara K. Silbert

A

Alexander W. Rankin

1Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD

C

Chloe N. Hoang

1Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD

A

Alexandra Semchenkova

2Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia

R

Regina M. Myers

E

Elena Zerkalenkova

4Laboratory of Cytogenetics and Molecular Genetics, Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia

H

Hao-Wei Wang

A

Alexandra E. Kovach

6Department of Pathology and Laboratory Medicine, Children's Hospital Los Angeles, Los Angeles, CA

C

Constance M. Yuan

2Laboratory of Pathology, National Cancer Institute, National Institutes of Health, Bethesda, MD

D

Dana Delgado Colon

2National Institutes of Health, Laboratory of Pathology, NCI, Bethesda, United States

L

Loïc Vasseur

2Hôpital St Louis, Adolescents and young adults department, Paris, France

A

Alex Bataller

2Division of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX

S

Samuel John

K

Kaylyn Utley Lyons

10Children's Hospital Colorado, University of Colorado School of Medicine, Denver, CO

B

Barbara Friedes

3Department of Pediatrics and the Center for Childhood Cancer Research, Children’s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA

A

Anna Alonso-Saladrigues

11Leukemia and Lymphoma Department, Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, Barcelona, Spain

H

Hisham Abdel-Azim

1Division of Transplant/Cell Therapy and Hematological Malignancies, Cancer Center, Departments of Pediatrics and Medicine, Loma Linda University School of Medicine, Children Hospital and Medical Center, Loma Linda, United States

E

Estelle Balducci

Université Paris Cité

A

Ahmed Assim Aljudi

16Department of Pathology, Children's Healthcare of Atlanta, Atlanta, GA

M

Marie Balsat

9Service d’Hématologie, Hospices Civils de Lyon, Hôpital Lyon Sud, Pierre-Bénite, France

D

D. Nathan Biery

1Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD

A

Aghiad Chamdin

18Pediatric Hematology & Oncology, Children's Hospital of Michigan/Central Michigan University, Mount Pleasant, MI

B

Bill H. Chang

19Division of Pediatric Hematology and Oncology, Doernbecher Children's Hospital at Oregon Health & Science University, Portland, OR

R

Raymund S. Cuevo

20Inova Schar Cancer Institute, Fairfax, VA

B

Barbara De Moerloose

D

David S. Dickens

19Division of Pediatric Hematology/Oncology, University of Iowa Stead Family Children’s Hospital, Iowa City, IA

U

Ulrich Duffner

2Section of Pediatric Bone Marrow Transplantation and Cellular Therapy, Helen DeVos Children's Hospital, Grand Rapids, United States

N

Nicolas Duployez

Unité Mixte de Recherche (UMR) 9020-UMR-S 1277-Canther-Cancer Heterogeneity, Plasticity and Resistance to Therapies, Institut de Recherche contre le Cancer de Lille, University of Lille, CNRS, Inserm, Centre Hospitalier Universitaire Lille, Lille, France

F

Firas El Chaer

M

Michelle Ann Elliott

26Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, MN

G

Gabriele Escherich

S

Sneha Fernandes

2Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom

M

Mandi R. Fitzjohn

10Children's Hospital Colorado, University of Colorado School of Medicine, Denver, CO

Z

Zhubin Gahvari

3University of Wisconsin-Madison - Carbone Cancer Center, Division of Hematology, Oncology, Palliative Care, Madison, United States

S

Stephan A. Grupp

R

Rui Rochelle He

30Department of Pathology, Inova Fairfax Medical Campus, Inova Health System, Falls Church, VA

C

Cynthia Harrison

1Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD

C

Christopher B. Hergott

1Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA

E

Emily M. Hsieh

33Division of Hematology/Oncology/Transplantation and Cellular Therapy, Cancer and Blood Disease Institute, Children’s Hospital Los Angeles, Norris Comprehensive Cancer Center, Keck School of Medicine, University of Southern California, Los Angeles, CA

A

Annette S. Kim

1Department of Pathology, University of Michigan, Ann Arbor, MI

D

Dennis J. Kuo

35Division of Pediatric Hematology-Oncology, University of California San Diego/Rady Children's Hospital San Diego, San Diego, CA

D

Daniel P. Larson

36Division of Hematopathology, Mayo Clinic, Rochester, MN

B

Benjamin J. Lee

37Department of Pharmacy, Chao Family Comprehensive Cancer Center, University of California Irvine Health, Orange, CA

T

Thibaut Leguay

R

R. Coleman Lindsley

Dana-Farber Cancer Institute, Boston, Massachusetts, United States

A

Abhishek A. Mangaonkar

26Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, MN

K

Kerstin Mezger

40Clinic of Pediatric Hematology and Oncology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany

H

Holly L. Pacenta

41Division of Hematology and Oncology, Cook Children's Medical Center, Fort Worth, TX

J

Jing Pan

M

Marlie Provost

43HCA HealthONE, Rocky Mountain Hospital for Children, Denver, CO

L

Latika Puri

44Department of Hematology, Loma Linda University Children’s Hospital, Loma Linda, CA

S

Sunil S. Raikar

A

Armando Martinez

5Flow Cytometry Unit, Laboratory of Pathology, National Cancer Institute, National Institutes of Health, Bethesda, MD

I

Isabella Bristol

5Flow Cytometry Unit, Laboratory of Pathology, National Cancer Institute, National Institutes of Health, Bethesda, MD

K

Kyle Murphy

5Flow Cytometry Unit, Laboratory of Pathology, National Cancer Institute, National Institutes of Health, Bethesda, MD

L

Lauren Reiman

70Keck School of Medicine, University of Southern California, Los Angeles, CA

M

Michele Redell

3Baylor College of Medicine, Pediatrics, Houston, United States

K

Kelly Reed

47University of Virginia School of Nursing, Charlottesville, VA

G

Gabrielle Roth-Guepin

2Fi LMC, Lyon, France

J

Jeremy Rubinstein

33Cincinnati Children's Hospital Medical Center, Division of Oncology, Cincinnati, United States

S

Süreyya Savaşan

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

K

Kristian Schafernak

52Division of Pathology and Laboratory Medicine, Phoenix Children's Hospital, Phoenix, AZ

A

Alexandra Stevens

1Texas Children's Hospital, Baylor College of Medicine, Department of Pediatrics, Section of Hematology and Oncology, Houston, United States

A

Aimee Talleur

N

Naomi Torres Carapia

55Children’s Hospital Los Angeles, Los Angeles, CA

J

Jacques Vargaftig

A

Anant Vatsayan

7Children's Hospital, Washington, United States

M

Matthias Wölfl

58Pediatric Oncology, Children's Hospital, University Hospital Würzburg, Würzburg, Germany

L

Liping Zhao

School of Life Science and Medicine

S

Susana Rives

12Institut de Recerca Sant Joan de Déu, Barcelona, Spain

V

Vanessa A. Fabrizio

61Pediatric Bone Marrow Transplant/Cellular Therapies, University of Colorado Anschutz/Children's Hospital Colorado, Denver, CO

K

Koji Sasaki

1The University of Texas MD Anderson Cancer Center, Houston, TX

I

Ibrahim Aldoss

N

Nicolas Boissel

S

Susan R. Rheingold

K

Kara L. Davis

S

Sara Ghorashian

E

Elad Jacoby

1Gray Faculty of Medical and Health Sciences, Tel Aviv University, Tel Aviv, Israel

A

Alexander Popov

A

Adam J. Lamble

N

Nirali N. Shah