Ex vivo T-lymphopoiesis assays assisting corrective treatment choice for genetically undefined T-lymphocytopenia
Jazyk angličtina Země Spojené státy americké Médium print-electronic
Typ dokumentu časopisecké články
PubMed
39965724
DOI
10.1016/j.clim.2025.110453
PII: S1521-6616(25)00028-2
Knihovny.cz E-zdroje
- Klíčová slova
- Artificial thymic organoids (ATO), Congenital athymia, Diagnostic assays, Ex vivo T-lymphocyte differentiation, Hematopoietic cell transplantation (HCT), Newborn screening, Severe combined immunodeficiency (SCID), T-lymphocytopenia, Thymus transplantation,
- MeSH
- buněčná diferenciace MeSH
- dítě MeSH
- imunofenotypizace MeSH
- kojenec MeSH
- lidé MeSH
- lymfopenie * imunologie MeSH
- lymfopoéza * genetika MeSH
- mladiství MeSH
- předškolní dítě MeSH
- primární imunodeficience terapie genetika imunologie MeSH
- T-lymfocyty * imunologie MeSH
- thymus imunologie MeSH
- transplantace hematopoetických kmenových buněk * metody MeSH
- Check Tag
- dítě MeSH
- kojenec MeSH
- lidé MeSH
- mladiství MeSH
- mužské pohlaví MeSH
- předškolní dítě MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
Persistent selective T-lymphocytopenia is found both in SCID and congenital athymia. Without molecular diagnosis, it is challenging to determine whether HCT or thymus transplantation ought to be performed. Ex vivo T-lymphopoiesis assays have been proposed to assist clinical decision-making for genetically undefined patients. We investigated 20 T-lymphocytopenic patients, including 13 patients awaiting first-line treatment and 7 patients with failed immune reconstitution after previous HCT or thymus transplantation. Whilst developmental blocks in ex vivo T-lymphopoiesis indicated hematopoietic cell-intrinsic defects, successful T-lymphocyte differentiation required careful interpretation, in conjunction with clinical status, immunophenotyping, and genetic investigations. Of the 20 patients, 13 proceeded to treatment, with successful immune reconstitution observed in 4 of the 6 patients post-HCT and 4 of the 7 patients after thymus transplantation, the latter including two patients who had previously undergone HCT. Whilst further validation and standardization are required, we conclude that assessing ex vivo T-lymphopoiesis during the diagnostic pathway for genetically undefined T-lymphocytopenia improves patient outcomes by facilitating corrective treatment choice.
Allergy and Immunology Department The Royal Children's Hospital Melbourne Melbourne Australia
Center for Translational Immunology University Medical Center Utrecht; Utrecht Netherlands
Paediatric Immunology Department University Hospitals of Birmingham Birmingham United Kingdom
Paediatric Onco Haematology Unit Geneva University Hospital; Geneva Switzerland
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