Ex vivo T-lymphopoiesis assays assisting corrective treatment choice for genetically undefined T-lymphocytopenia
| Title: | Ex vivo T-lymphopoiesis assays assisting corrective treatment choice for genetically undefined T-lymphocytopenia |
|---|---|
| Authors: | Golwala ZM; Spiridou Goncalves H; Moirangthem RD; Evans G; Lizot S; de Koning C; Garrigue A; Corredera MM; Ocampo-Godinez JM; Howley E; Kricke S; Awuah A; Obiri-Yeboa I; Rai R; Sebire N; Bernard F; Bordon Cueto De Braem V; Boztug K; Cole T; Gennery AR; Hackett S; Hambleton S; Holm M; Kusters MA; Klocperk A; Marzollo A; Marcus N; Nademi Z; Pachlopnik Schmid J; Pichler H; Sellmer A; Soler-Palacin P; Soomann M; Torpiano P; van Montfrans J; Nierkens S; Adams S; Buckland M; Gilmour K; Worth A; Thrasher AJ; Davies EG; Andre I; Kreins AY |
| Source: | Clinical Immunology, May 2025 |
| Publisher Information: | Academic Press Inc. |
| Publication Year: | 2025 |
| Collection: | Newcastle University Library ePrints Service |
| Description: | © 2025 The AuthorsPersistent 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. |
| Document Type: | article in journal/newspaper |
| File Description: | application/pdf |
| Language: | unknown |
| Relation: | https://eprints.ncl.ac.uk/304866; https://eprints.ncl.ac.uk/fulltext.aspx?url=304866/6C68BF46-0278-4B1B-9505-7B35B6553D12.pdf&pub_id=304866 |
| Availability: | https://eprints.ncl.ac.uk/304866 |
| Rights: | https://creativecommons.org/licenses/by/4.0/ |
| Accession Number: | edsbas.AC6EF99C |
| Database: | BASE |