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


Hesham Eissa, University of Colorado Anschutz Medical Campus
Monica S. Thakar, Fred Hutchinson Cancer Center
Ami J. Shah, Lucile Packard Children's Hospital Stanford
Brent R. Logan, Medical College of Wisconsin
Linda M. Griffith, National Institute of Allergy and Infectious Diseases (NIAID)
Huaying Dong, Medical College of Wisconsin
Roberta E. Parrott, Duke University Medical Center
Richard J. O'Reilly, Memorial Sloan-Kettering Cancer Center
Jasmeen Dara, UCSF School of Medicine
Neena Kapoor, Children's Hospital Los Angeles
Lisa Forbes Satter, Texas Children's Hospital
Sharat Chandra, Cincinnati Children's Hospital Medical Center
Malika Kapadia, Boston Children's Hospital
Shanmuganathan Chandrakasan, Children's Healthcare of Atlanta
Alan Knutsen, Saint Louis University
Soma C. Jyonouchi, The Children's Hospital of Philadelphia
Lyndsay Molinari, Methodist Children's Hospital of South Texas
Ahmad Rayes, Huntsman Cancer Institute
Christen L. Ebens, University of Minnesota Twin Cities
Pierre Teira, CHU Sainte-Justine - Le Centre Hospitalier Universitaire Mère-Enfant
Blachy J. Dávila Saldaña, Childrens National Health System
Lauri M. Burroughs, Fred Hutchinson Cancer Center
Sonali Chaudhury, Children's Memorial Hospital
Deepak Chellapandian, All Children's Hospital St. Petersburg
Alfred P. Gillio, Hackensack University Medical Center
Fredrick Goldman, The University of Alabama at Birmingham
Harry L. Malech, National Institute of Allergy and Infectious Diseases (NIAID)
Lolie C. Yu, LSU Health Sciences Center - New OrleansFollow

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Journal of Allergy and Clinical Immunology


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: We investigated outcomes of HCT for severe combined immunodeficiency (SCID). 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: 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: 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.

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