-
Je něco špatně v tomto záznamu ?
Ex vivo T-lymphopoiesis assays assisting corrective treatment choice for genetically undefined T-lymphocytopenia
ZM. Golwala, H. Spiridou Goncalves, RD. Moirangthem, G. Evans, S. Lizot, C. de Koning, A. Garrigue, MM. Corredera, JM. Ocampo-Godinez, E. Howley, S. Kricke, A. Awuah, I. Obiri-Yeboa, R. Rai, N. Sebire, F. Bernard, V. Bordon Cueto De Braem, K....
Clinical immunology.
2025 ;
274 (-) :
110453.
[pub] 20250216
Jazyk angličtina Země Spojené státy americké
Typ dokumentu časopisecké články
Perzistentní odkaz
https://www.medvik.cz/link/bmc25009294
- 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
CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences Vienna Austria
Center for Translational Immunology University Medical Center Utrecht
Department of Histopathology Great Ormond Street Hospital for Children NHS Foundation Trust
Human Lymphohematopoiesis Laboratory Imagine Institute INSERM UMR 1163 Université Paris Cité
Medical University of Vienna Department of Pediatrics and Adolescent Medicine Vienna Austria
Paediatric Immunology Department University Hospitals of Birmingham Birmingham United Kingdom
Paediatric Onco Haematology Unit Geneva University Hospital
Pediatric Immunology University of Zurich Zurich Switzerland
Princess Maxima Center for Pediatric Oncology Utrecht Netherlands
Sackler Faculty of Medicine Tel Aviv University Tel Aviv Israel
SIHMDS Haematology Great Ormond Street Hospital for Children NHS Foundation Trust
Citace poskytuje Crossref.org
- 000
- 00000naa a2200000 a 4500
- 001
- bmc25009294
- 003
- CZ-PrNML
- 005
- 20250429134846.0
- 007
- ta
- 008
- 250415e20250216xxu f 000 0|eng||
- 009
- AR
- 024 7_
- $a 10.1016/j.clim.2025.110453 $2 doi
- 035 __
- $a (PubMed)39965724
- 040 __
- $a ABA008 $b cze $d ABA008 $e AACR2
- 041 0_
- $a eng
- 044 __
- $a xxu
- 100 1_
- $a Golwala, Zainab M $u Infection, Immunity and Inflammation Research & Teaching Department, Great Ormond Street Institute of Child Health, University College London; London, United Kingdom; Department of Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Foundation Trust; London, United Kingdom
- 245 10
- $a Ex vivo T-lymphopoiesis assays assisting corrective treatment choice for genetically undefined T-lymphocytopenia / $c ZM. Golwala, H. Spiridou Goncalves, RD. Moirangthem, G. Evans, S. Lizot, C. de Koning, A. Garrigue, MM. Corredera, JM. Ocampo-Godinez, E. Howley, S. Kricke, A. Awuah, I. Obiri-Yeboa, R. Rai, N. Sebire, F. Bernard, V. Bordon Cueto De Braem, K. Boztug, T. Cole, AR. Gennery, S. Hackett, S. Hambleton, M. Holm, MA. Kusters, A. Klocperk, A. Marzollo, N. Marcus, Z. Nademi, J. Pachlopnik Schmid, H. Pichler, A. Sellmer, P. Soler-Palacin, M. Soomann, P. Torpiano, J. van Montfrans, S. Nierkens, S. Adams, M. Buckland, K. Gilmour, A. Worth, AJ. Thrasher, EG. Davies, I. André, AY. Kreins
- 520 9_
- $a 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.
- 650 _2
- $a lidé $7 D006801
- 650 12
- $a lymfopenie $x imunologie $7 D008231
- 650 12
- $a lymfopoéza $x genetika $7 D038041
- 650 12
- $a transplantace hematopoetických kmenových buněk $x metody $7 D018380
- 650 12
- $a T-lymfocyty $x imunologie $7 D013601
- 650 _2
- $a mužské pohlaví $7 D008297
- 650 _2
- $a ženské pohlaví $7 D005260
- 650 _2
- $a kojenec $7 D007223
- 650 _2
- $a předškolní dítě $7 D002675
- 650 _2
- $a dítě $7 D002648
- 650 _2
- $a imunofenotypizace $7 D016130
- 650 _2
- $a thymus $x imunologie $7 D013950
- 650 _2
- $a primární imunodeficience $x terapie $x genetika $x imunologie $7 D000081207
- 650 _2
- $a buněčná diferenciace $7 D002454
- 650 _2
- $a mladiství $7 D000293
- 655 _2
- $a časopisecké články $7 D016428
- 700 1_
- $a Spiridou Goncalves, Helena $u Infection, Immunity and Inflammation Research & Teaching Department, Great Ormond Street Institute of Child Health, University College London; London, United Kingdom
- 700 1_
- $a Moirangthem, Ranjita Devi $u Human Lymphohematopoiesis Laboratory, Imagine Institute, INSERM UMR 1163, Université Paris Cité; Paris, France; Smart Immune; Paris, France
- 700 1_
- $a Evans, Grace $u Infection, Immunity and Inflammation Research & Teaching Department, Great Ormond Street Institute of Child Health, University College London; London, United Kingdom
- 700 1_
- $a Lizot, Sabrina $u Human Lymphohematopoiesis Laboratory, Imagine Institute, INSERM UMR 1163, Université Paris Cité; Paris, France
- 700 1_
- $a de Koning, Coco $u Center for Translational Immunology (CTI), University Medical Center Utrecht; Utrecht, Netherlands
- 700 1_
- $a Garrigue, Alexandrine $u Human Lymphohematopoiesis Laboratory, Imagine Institute, INSERM UMR 1163, Université Paris Cité; Paris, France
- 700 1_
- $a Corredera, Marta Martin $u Human Lymphohematopoiesis Laboratory, Imagine Institute, INSERM UMR 1163, Université Paris Cité; Paris, France; Smart Immune; Paris, France
- 700 1_
- $a Ocampo-Godinez, Juan Moises $u Infection, Immunity and Inflammation Research & Teaching Department, Great Ormond Street Institute of Child Health, University College London; London, United Kingdom
- 700 1_
- $a Howley, Evey $u Department of Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Foundation Trust; London, United Kingdom
- 700 1_
- $a Kricke, Susanne $u SIHMDS-Haematology, Great Ormond Street Hospital for Children NHS Foundation Trust; London, United Kingdom
- 700 1_
- $a Awuah, Arnold $u Department of Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Foundation Trust; London, United Kingdom
- 700 1_
- $a Obiri-Yeboa, Irene $u Department of Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Foundation Trust; London, United Kingdom
- 700 1_
- $a Rai, Rajeev $u Infection, Immunity and Inflammation Research & Teaching Department, Great Ormond Street Institute of Child Health, University College London; London, United Kingdom
- 700 1_
- $a Sebire, Neil $u Department of Histopathology, Great Ormond Street Hospital for Children NHS Foundation Trust; London, United Kingdom
- 700 1_
- $a Bernard, Fanette $u Paediatric Onco-Haematology Unit, Geneva University Hospital; Geneva, Switzerland
- 700 1_
- $a Bordon Cueto De Braem, Victoria $u Department of Pediatric Hemato-Oncology and Hematopoietic Stem cell Transplantation, Ghent University Hospital, Ghent, Belgium
- 700 1_
- $a Boztug, Kaan $u St. Anna Children's Cancer Research Institute, Vienna, Austria; Medical University of Vienna, Department of Pediatrics and Adolescent Medicine, Vienna, Austria; CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria; St. Anna Children's Hospital, Medical University of Vienna, Department of Pediatrics and Adolescent Medicine, Vienna, Austria
- 700 1_
- $a Cole, Theresa $u Allergy and Immunology Department, The Royal Children's Hospital Melbourne, Melbourne, Australia
- 700 1_
- $a Gennery, Andrew R $u Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom; Paediatric Immunology and Haematopoietic Stem Cell Transplantation, Great North Children's Hospital, Newcastle upon Tyne, United Kingdom
- 700 1_
- $a Hackett, Scott $u Paediatric Immunology Department, University Hospitals of Birmingham, Birmingham, United Kingdom
- 700 1_
- $a Hambleton, Sophie $u Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom; Paediatric Immunology and Haematopoietic Stem Cell Transplantation, Great North Children's Hospital, Newcastle upon Tyne, United Kingdom
- 700 1_
- $a Holm, Mette $u Department of Paediatrics and Adolescent Medicine, Infections and Immunology, Aarhus University Hospital, Aarhus, Denmark
- 700 1_
- $a Kusters, Maaike A $u Department of Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Foundation Trust; London, United Kingdom
- 700 1_
- $a Klocperk, Adam $u Department of Immunology, Second Faculty of Medicine, Charles University and University Hospital in Motol, Prague, Czechia
- 700 1_
- $a Marzollo, Antonio $u Pediatric Hematology, Oncology and Stem Cell Transplant Division, Padua University Hospital, Padua, Italy
- 700 1_
- $a Marcus, Nufar $u Allergy and Immunology Unit, Schneider Children's Medical Center of Israel, Kipper Institute of Immunology, Petach Tikva, Israel; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel
- 700 1_
- $a Nademi, Zohreh $u Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom; Paediatric Immunology and Haematopoietic Stem Cell Transplantation, Great North Children's Hospital, Newcastle upon Tyne, United Kingdom
- 700 1_
- $a Pachlopnik Schmid, Jana $u Division of Immunology and the Children's Research Centre, University Children's Hospital Zurich, Zurich, Switzerland; Pediatric Immunology, University of Zurich, Zurich, Switzerland
- 700 1_
- $a Pichler, Herbert $u St. Anna Children's Cancer Research Institute, Vienna, Austria; St. Anna Children's Hospital, Medical University of Vienna, Department of Pediatrics and Adolescent Medicine, Vienna, Austria
- 700 1_
- $a Sellmer, Anna $u Department of Paediatrics and Adolescent Medicine, Infections and Immunology, Aarhus University Hospital, Aarhus, Denmark
- 700 1_
- $a Soler-Palacin, Pere $u Pediatric Infectious Diseases and Immunodeficiencies Unit, Department of Pediatrics, Hospital Infantil Vall d'Hebron, Vall d'Hebron Barcelona Hospital Campus, Barcelona, Spain; Department of Pediatrics, Obstetrics and Gynecology, Preventive Medicine and Public Health, Faculty of Medicine, Universitat Autònoma de Barcelona, Barcelona, Spain; Infection in Immunocompromised Pediatric Patients, Vall d'Hebron Research Institute, Vall d'Hebron Barcelona Hospital Campus, Barcelona, Spain
- 700 1_
- $a Soomann, Maarja $u Division of Immunology and the Children's Research Centre, University Children's Hospital Zurich, Zurich, Switzerland
- 700 1_
- $a Torpiano, Paul $u Department of Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Foundation Trust; London, United Kingdom
- 700 1_
- $a van Montfrans, Joris $u Department of Pediatric Immunology and Infectious Diseases, Wilhelmina Children's Hospital, University Medical Centre Utrecht; Utrecht, Netherlands
- 700 1_
- $a Nierkens, Stefan $u Center for Translational Immunology (CTI), University Medical Center Utrecht; Utrecht, Netherlands; Princess Maxima Center for Pediatric Oncology, Utrecht, Netherlands
- 700 1_
- $a Adams, Stuart $u SIHMDS-Haematology, Great Ormond Street Hospital for Children NHS Foundation Trust; London, United Kingdom
- 700 1_
- $a Buckland, Matthew $u Department of Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Foundation Trust; London, United Kingdom
- 700 1_
- $a Gilmour, Kimberly $u Department of Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Foundation Trust; London, United Kingdom
- 700 1_
- $a Worth, Austen $u Department of Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Foundation Trust; London, United Kingdom
- 700 1_
- $a Thrasher, Adrian J $u Infection, Immunity and Inflammation Research & Teaching Department, Great Ormond Street Institute of Child Health, University College London; London, United Kingdom
- 700 1_
- $a Davies, E Graham $u Infection, Immunity and Inflammation Research & Teaching Department, Great Ormond Street Institute of Child Health, University College London; London, United Kingdom; Department of Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Foundation Trust; London, United Kingdom
- 700 1_
- $a André, Isabelle $u Human Lymphohematopoiesis Laboratory, Imagine Institute, INSERM UMR 1163, Université Paris Cité; Paris, France
- 700 1_
- $a Kreins, Alexandra Y $u Infection, Immunity and Inflammation Research & Teaching Department, Great Ormond Street Institute of Child Health, University College London; London, United Kingdom; Department of Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Foundation Trust; London, United Kingdom. Electronic address: a.kreins@ucl.ac.uk
- 773 0_
- $w MED00005218 $t Clinical immunology $x 1521-7035 $g Roč. 274 (20250216), s. 110453
- 856 41
- $u https://pubmed.ncbi.nlm.nih.gov/39965724 $y Pubmed
- 910 __
- $a ABA008 $b sig $c sign $y - $z 0
- 990 __
- $a 20250415 $b ABA008
- 991 __
- $a 20250429134842 $b ABA008
- 999 __
- $a ok $b bmc $g 2310959 $s 1246375
- BAS __
- $a 3
- BAS __
- $a PreBMC-MEDLINE
- BMC __
- $a 2025 $b 274 $c - $d 110453 $e 20250216 $i 1521-7035 $m Clinical immunology $n Clin Immunol $x MED00005218
- LZP __
- $a Pubmed-20250415
