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

Publication ,  Journal Article
Eissa, H; Thakar, MS; Shah, AJ; Logan, BR; Griffith, LM; Dong, H; Parrott, RE; O'Reilly, RJ; Dara, J; Kapoor, N; Forbes Satter, L; Chandra, S ...
Published in: J Allergy Clin Immunol
January 2024

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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Published In

J Allergy Clin Immunol

DOI

EISSN

1097-6825

Publication Date

January 2024

Volume

153

Issue

1

Start / End Page

287 / 296

Location

United States

Related Subject Headings

  • Transplantation Conditioning
  • Severe Combined Immunodeficiency
  • Retrospective Studies
  • Incidence
  • Humans
  • Hematopoietic Stem Cell Transplantation
  • Child
  • Canada
  • Allergy
  • 3204 Immunology
 

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Chicago
ICMJE
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Eissa, H., Thakar, M. S., Shah, A. J., Logan, B. R., Griffith, L. M., Dong, H., … Heimall, J. (2024). Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study. J Allergy Clin Immunol, 153(1), 287–296. https://doi.org/10.1016/j.jaci.2023.09.027
Eissa, Hesham, Monica S. Thakar, Ami J. Shah, Brent R. Logan, Linda M. Griffith, Huaying Dong, Roberta E. Parrott, et al. “Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study.J Allergy Clin Immunol 153, no. 1 (January 2024): 287–96. https://doi.org/10.1016/j.jaci.2023.09.027.
Eissa H, Thakar MS, Shah AJ, Logan BR, Griffith LM, Dong H, et al. Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study. J Allergy Clin Immunol. 2024 Jan;153(1):287–96.
Eissa, Hesham, et al. “Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study.J Allergy Clin Immunol, vol. 153, no. 1, Jan. 2024, pp. 287–96. Pubmed, doi:10.1016/j.jaci.2023.09.027.
Eissa H, Thakar MS, Shah AJ, Logan BR, Griffith LM, Dong H, Parrott RE, O’Reilly RJ, Dara J, Kapoor N, Forbes Satter L, Chandra S, Kapadia M, Chandrakasan S, Knutsen A, Jyonouchi SC, Molinari L, Rayes A, Ebens CL, Teira P, Dávila Saldaña BJ, Burroughs LM, Chaudhury S, Chellapandian D, Gillio AP, Goldman F, Malech HL, DeSantes K, Cuvelier GDE, Rozmus J, Quinones R, Yu LC, Broglie L, Aquino V, Shereck E, Moore TB, Vander Lugt MT, Mousallem TI, Oved JH, Dorsey M, Abdel-Azim H, Martinez C, Bleesing JH, Prockop S, Kohn DB, Bednarski JJ, Leiding J, Marsh RA, Torgerson T, Notarangelo LD, Pai S-Y, Pulsipher MA, Puck JM, Dvorak CC, Haddad E, Buckley RH, Cowan MJ, Heimall J. Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study. J Allergy Clin Immunol. 2024 Jan;153(1):287–296.
Journal cover image

Published In

J Allergy Clin Immunol

DOI

EISSN

1097-6825

Publication Date

January 2024

Volume

153

Issue

1

Start / End Page

287 / 296

Location

United States

Related Subject Headings

  • Transplantation Conditioning
  • Severe Combined Immunodeficiency
  • Retrospective Studies
  • Incidence
  • Humans
  • Hematopoietic Stem Cell Transplantation
  • Child
  • Canada
  • Allergy
  • 3204 Immunology