Experience with cultured thymus tissue in 105 children.

ABSTRACT:

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 op

SUBMITTER: Markert ML 

PROVIDER: S-EPMC8810898 | biostudies-literature | 2022 Feb

REPOSITORIES: biostudies-literature