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Experience with cultured thymus tissue in 105 children.

Publication ,  Journal Article
Markert, ML; Gupton, SE; McCarthy, EA
Published in: J Allergy Clin Immunol
February 2022

BACKGROUND: Currently, there are no approved therapies to treat congenital athymia, a condition of immune deficiency resulting in high early mortality due to infection and immune dysregulation. Multiple syndromic conditions, such as complete DiGeorge syndrome, 22q11.2 deletion syndrome, CHARGE (coloboma, heart defects, choanal atresia, growth or mental retardation, genital hypoplasia, and ear anomalies and/or deafness) syndrome, diabetic embryopathy, other genetic variants, and FOXN1 deficiency, are associated with congenital athymia. OBJECTIVE: Our aims were to study 105 patients treated with cultured thymus tissue (CTT), and in this report, to focus on the outcomes of 95 patients with treatment-naive congenital athymia. METHODS: A total of 10 prospective, single-arm open-label studies with patient enrollment from 1993 to 2020 form the basis of this data set. Patients were tested after administration of CTT for T-cell development; all adverse events and infections were recorded. RESULTS: A total of 105 patients were enrolled and received CTT (the full analysis set). Of those patients, 10 had diagnoses other than congenital athymia and/or received prior treatments. Of those 105 patients, 95 patients with treatment-naive congenital athymia were included in the efficacy analysis set (EAS). The Kaplan-Meier estimated survival rates at year 1 and year 2 after administration of CTT in the EAS were 77% (95% CI = 0.670-0.844) and 76% (95% CI = 0.657-0.834), respectively. In all, 21 patients died in the first year before developing naive T cells and 1 died in the second year after receipt of CTT; 3 subsequent deaths were not related to immunodeficiency. A few patients developed alopecia, autoimmune hepatitis, psoriasis, and psoriatic arthritis after year 1. The rates of infections, autologous graft-versus-host-disease manifestations, and autoimmune cytopenias all decreased approximately 1 year after administration of CTT. CONCLUSION: Treatment with CTT led to development of naive T cells with a 1-year survival rate of 77% and a median follow-up time of 7.6 years. Immune reconstitution sufficient to prevent infections and support survival typically develops 6 to12 months after administration of CTT.

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Published In

J Allergy Clin Immunol

DOI

EISSN

1097-6825

Publication Date

February 2022

Volume

149

Issue

2

Start / End Page

747 / 757

Location

United States

Related Subject Headings

  • Thymus Gland
  • T-Lymphocytes
  • Male
  • Infant
  • Humans
  • Forkhead Transcription Factors
  • Female
  • DiGeorge Syndrome
  • Child, Preschool
  • CHARGE Syndrome
 

Citation

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Markert, M. L., Gupton, S. E., & McCarthy, E. A. (2022). Experience with cultured thymus tissue in 105 children. J Allergy Clin Immunol, 149(2), 747–757. https://doi.org/10.1016/j.jaci.2021.06.028
Markert, M Louise, Stephanie E. Gupton, and Elizabeth A. McCarthy. “Experience with cultured thymus tissue in 105 children.J Allergy Clin Immunol 149, no. 2 (February 2022): 747–57. https://doi.org/10.1016/j.jaci.2021.06.028.
Markert ML, Gupton SE, McCarthy EA. Experience with cultured thymus tissue in 105 children. J Allergy Clin Immunol. 2022 Feb;149(2):747–57.
Markert, M. Louise, et al. “Experience with cultured thymus tissue in 105 children.J Allergy Clin Immunol, vol. 149, no. 2, Feb. 2022, pp. 747–57. Pubmed, doi:10.1016/j.jaci.2021.06.028.
Markert ML, Gupton SE, McCarthy EA. Experience with cultured thymus tissue in 105 children. J Allergy Clin Immunol. 2022 Feb;149(2):747–757.
Journal cover image

Published In

J Allergy Clin Immunol

DOI

EISSN

1097-6825

Publication Date

February 2022

Volume

149

Issue

2

Start / End Page

747 / 757

Location

United States

Related Subject Headings

  • Thymus Gland
  • T-Lymphocytes
  • Male
  • Infant
  • Humans
  • Forkhead Transcription Factors
  • Female
  • DiGeorge Syndrome
  • Child, Preschool
  • CHARGE Syndrome