Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study.

HCT SCID bone marrow transplantation late effects survivorship

Journal

The Journal of allergy and clinical immunology
ISSN: 1097-6825
Titre abrégé: J Allergy Clin Immunol
Pays: United States
ID NLM: 1275002

Informations de publication

Date de publication:
02 Oct 2023
Historique:
received: 30 03 2023
revised: 01 09 2023
accepted: 06 09 2023
pubmed: 5 10 2023
medline: 5 10 2023
entrez: 4 10 2023
Statut: aheadofprint

Résumé

The Primary Immune Deficiency Treatment Consortium (PIDTC) enrolled children in the United States and Canada onto a retrospective multicenter natural history study of hematopoietic cell transplantation (HCT). We investigated outcomes of HCT for severe combined immunodeficiency (SCID). We evaluated the chronic and late effects (CLE) after HCT for SCID in 399 patients transplanted from 1982 to 2012 at 32 PIDTC centers. Eligibility criteria included survival to at least 2 years after HCT without need for subsequent cellular therapy. CLE were defined as either conditions present at any time before 2 years from HCT that remained unresolved (chronic), or new conditions that developed beyond 2 years after HCT (late). The cumulative incidence of CLE was 25% in those alive at 2 years, increasing to 41% at 15 years after HCT. CLE were most prevalent in the neurologic (9%), neurodevelopmental (8%), and dental (8%) categories. Chemotherapy-based conditioning was associated with decreased-height z score at 2 to 5 years after HCT (P < .001), and with endocrine (P < .001) and dental (P = .05) CLE. CD4 count of ≤500 cells/μL and/or continued need for immunoglobulin replacement therapy >2 years after transplantation were associated with lower-height z scores. Continued survival from 2 to 15 years after HCT was 90%. The presence of any CLE was associated with increased risk of late death (hazard ratio, 7.21; 95% confidence interval, 2.71-19.18; P < .001). Late morbidity after HCT for SCID was substantial, with an adverse impact on overall survival. This study provides evidence for development of survivorship guidelines based on disease characteristics and treatment exposure for patients after HCT for SCID.

Sections du résumé

BACKGROUND BACKGROUND
The Primary Immune Deficiency Treatment Consortium (PIDTC) enrolled children in the United States and Canada onto a retrospective multicenter natural history study of hematopoietic cell transplantation (HCT).
OBJECTIVE OBJECTIVE
We investigated outcomes of HCT for severe combined immunodeficiency (SCID).
METHODS METHODS
We evaluated the chronic and late effects (CLE) after HCT for SCID in 399 patients transplanted from 1982 to 2012 at 32 PIDTC centers. Eligibility criteria included survival to at least 2 years after HCT without need for subsequent cellular therapy. CLE were defined as either conditions present at any time before 2 years from HCT that remained unresolved (chronic), or new conditions that developed beyond 2 years after HCT (late).
RESULTS RESULTS
The cumulative incidence of CLE was 25% in those alive at 2 years, increasing to 41% at 15 years after HCT. CLE were most prevalent in the neurologic (9%), neurodevelopmental (8%), and dental (8%) categories. Chemotherapy-based conditioning was associated with decreased-height z score at 2 to 5 years after HCT (P < .001), and with endocrine (P < .001) and dental (P = .05) CLE. CD4 count of ≤500 cells/μL and/or continued need for immunoglobulin replacement therapy >2 years after transplantation were associated with lower-height z scores. Continued survival from 2 to 15 years after HCT was 90%. The presence of any CLE was associated with increased risk of late death (hazard ratio, 7.21; 95% confidence interval, 2.71-19.18; P < .001).
CONCLUSION CONCLUSIONS
Late morbidity after HCT for SCID was substantial, with an adverse impact on overall survival. This study provides evidence for development of survivorship guidelines based on disease characteristics and treatment exposure for patients after HCT for SCID.

Identifiants

DOI: 10.1016/j.jaci.2023.09.027 PMID: 37793572
pubmed: 37793572
pii: S0091-6749(23)01208-3
doi: 10.1016/j.jaci.2023.09.027
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Informations de copyright

Copyright © 2023. Published by Elsevier Inc.

Auteurs

Hesham Eissa (H)

Division of Pediatric Hematology-Oncology-BMT, University of Colorado, Aurora, Wash. Electronic address: Hesham.eissa@cuanschutz.edu.

Monica S Thakar (MS)

Fred Hutchinson Cancer Center, Seattle, Wash; Department of Pediatrics, University of Washington, Seattle, Wash.

Ami J Shah (AJ)

Pediatrics [Hematology/Oncology/Stem Cell Transplantation and Regenerative Medicine], Stanford University/Lucille Packard Children's Hospital, Palo Alto, Calif.

Brent R Logan (BR)

Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wis.

Linda M Griffith (LM)

Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, Md.

Huaying Dong (H)

Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wis.

Roberta E Parrott (RE)

Duke University Medical Center, Durham, NC.

Richard J O'Reilly (RJ)

Department of Pediatrics, Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY.

Jasmeen Dara (J)

Division of Allergy, Immunology and Blood and Marrow Transplantation, Department of Pediatrics, University of California San Francisco School of Medicine and UCSF Benioff Children's Hospital, San Francisco, Calif.

Neena Kapoor (N)

Division of Hematology, Oncology and Blood and Marrow Transplant, Children's Hospital Los Angeles, Los Angeles, Calif.

Lisa Forbes Satter (L)

Immunology, Allergy, and Rheumatology, Baylor College of Medicine, Texas Children's Hospital, Houston, Tex.

Sharat Chandra (S)

Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; Department of Pediatrics, University of Cincinnati School of Medicine, Cincinnati, Ohio.

Malika Kapadia (M)

Boston Children's Hospital, Dana-Farber Cancer Institute, Boston, Mass.

Shanmuganathan Chandrakasan (S)

Bone Marrow Transplantation and Immune Deficiency, Children's Healthcare of Atlanta, Atlanta, Ga.

Alan Knutsen (A)

St Louis University, Cardinal Glennon Children's Hospital, St Louis, Mo.

Soma C Jyonouchi (SC)

Division of Allergy and Immunology, Children's Hospital of Philadelphia, Philadelphia, Pa; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pa.

Lyndsay Molinari (L)

Methodist Children's Hospital of South Texas, San Antonio, Tex.

Ahmad Rayes (A)

Division of Hematology, Oncology, Transplantation, and Immunology, Primary Children's Hospital, Huntsman Cancer Institute, Spense Fox Eccles School of Medicine at the University of Utah, Salt Lake City, Utah.

Christen L Ebens (CL)

Division of Pediatric Blood and Marrow Transplant and Cellular Therapy, University of Minnesota Masonic Children's Hospital, Minneapolis, Minn.

Pierre Teira (P)

Paediatric Haematology Oncology, Ste-Justine Hospital, Montreal, Canada.

Blachy J Dávila Saldaña (BJ)

Children's National Hospital, Washington, DC.

Lauri M Burroughs (LM)

Fred Hutchinson Cancer Center, Seattle, Wash; Department of Pediatrics, University of Washington, Seattle, Wash.

Sonali Chaudhury (S)

Hematology, Oncology, Neuro-oncology & Stem Cell Transplantation Division, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Ill.

Deepak Chellapandian (D)

Center for Cell and Gene Therapy for Non-malignant Conditions, Johns Hopkins All Children's Hospital, St Petersburg, Fla.

Alfred P Gillio (AP)

Children's Cancer Institute, Hackensack University Medical Center, Hackensack, NJ.

Fredrick Goldman (F)

Division of Pediatric Hematology and Oncology and Bone Marrow Transplant, University of Alabama at Birmingham, Birmingham, Ala.

Harry L Malech (HL)

Genetic Immunotherapy Section, NIAID, NIH, Bethesda, Md.

Kenneth DeSantes (K)

Division of Pediatric Hematology-Oncology & Bone Marrow Transplant, University of Wisconsin, American Family Children's Hospital, Madison, Wis.

Geoff D E Cuvelier (GDE)

Manitoba Blood and Marrow Transplant Program, CancerCare Manitoba, Winnipeg, Canada.

Jacob Rozmus (J)

Children's & Women's Health Centre of British Columbia, Vancouver, Canada.

Ralph Quinones (R)

Division of Pediatric Hematology-Oncology-BMT, University of Colorado, Aurora, Wash.

Lolie C Yu (LC)

Division of Heme-Onc/HSCT, Children's Hospital/LSUHSC, New Orleans, La.

Larisa Broglie (L)

Department of Pediatrics, Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, Medical College of Wisconsin, Milwaukee, Wis.

Victor Aquino (V)

Division of Pediatric Hematology and Oncology, The University of Texas Southwestern Medical Center, Dallas, Tex.

Evan Shereck (E)

Division of Pediatric Hematology/Oncology, Oregon Health and Science University, Portland, Ore.

Theodore B Moore (TB)

Department of Pediatric Hematology-Oncology, Mattel Children's Hospital, University of California, Los Angeles, Calif.

Mark T Vander Lugt (MT)

Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, Mich.

Talal I Mousallem (TI)

Duke University Medical Center, Durham, NC.

Joeseph H Oved (JH)

Department of Pediatrics, Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY.

Morna Dorsey (M)

Division of Allergy, Immunology and Blood and Marrow Transplantation, Department of Pediatrics, University of California San Francisco School of Medicine and UCSF Benioff Children's Hospital, San Francisco, Calif.

Hisham Abdel-Azim (H)

Division of Hematology, Oncology and Blood and Marrow Transplant, Children's Hospital Los Angeles, Los Angeles, Calif; Loma Linda University School of Medicine, Cancer Center, Children Hospital and Medical Center, Loma Linda, Calif.

Caridad Martinez (C)

Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, Tex.

Jacob H Bleesing (JH)

Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; Department of Pediatrics, University of Cincinnati School of Medicine, Cincinnati, Ohio.

Susan Prockop (S)

Boston Children's Hospital, Dana-Farber Cancer Institute, Boston, Mass.

Donald B Kohn (DB)

University of California, Los Angeles, Calif.

Jeffrey J Bednarski (JJ)

Department of Pediatrics, Washington University School of Medicine, St Louis, Mo.

Jennifer Leiding (J)

Orlando Health Arnold Palmer Hospital for Children, Orlando, Fla.

Rebecca A Marsh (RA)

Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; Department of Pediatrics, University of Cincinnati School of Medicine, Cincinnati, Ohio.

Troy Torgerson (T)

Allen Institute for Immunology, Seattle, Wash.

Luigi D Notarangelo (LD)

Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, Md.

Sung-Yun Pai (SY)

Center for Cancer Research, National Cancer Institute, NIH, Bethesda, Md.

Michael A Pulsipher (MA)

Division of Hematology, Oncology, Transplantation, and Immunology, Primary Children's Hospital, Huntsman Cancer Institute, Spense Fox Eccles School of Medicine at the University of Utah, Salt Lake City, Utah.

Jennifer M Puck (JM)

Division of Allergy, Immunology and Blood and Marrow Transplantation, Department of Pediatrics, University of California San Francisco School of Medicine and UCSF Benioff Children's Hospital, San Francisco, Calif.

Christopher C Dvorak (CC)

Division of Allergy, Immunology and Blood and Marrow Transplantation, Department of Pediatrics, University of California San Francisco School of Medicine and UCSF Benioff Children's Hospital, San Francisco, Calif.

Elie Haddad (E)

Department of Pediatrics and the Department of Microbiology, Immunology, and Infectious Diseases, University of Montreal, CHU Sainte-Justine, Montreal, Canada.

Rebecca H Buckley (RH)

Duke University Medical Center, Durham, NC.

Morton J Cowan (MJ)

Division of Allergy, Immunology and Blood and Marrow Transplantation, Department of Pediatrics, University of California San Francisco School of Medicine and UCSF Benioff Children's Hospital, San Francisco, Calif.

Jennifer Heimall (J)

Division of Allergy and Immunology, Children's Hospital of Philadelphia, Philadelphia, Pa; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pa.

Classifications MeSH