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Outcome of haematopoietic stem cell transplantation in dyskeratosis congenita
F. Fioredda, S. Iacobelli, ET. Korthof, C. Knol, A. van Biezen, D. Bresters, P. Veys, A. Yoshimi, F. Fagioli, B. Mats, M. Zecca, M. Faraci, M. Miano, L. Arcuri, M. Maschan, T. O'Brien, MA. Diaz, J. Sevilla, O. Smith, R. Peffault de Latour, J. de...
Jazyk angličtina Země Anglie, Velká Británie
Typ dokumentu časopisecké články
PubMed
29984823
DOI
10.1111/bjh.15495
Knihovny.cz E-zdroje
- MeSH
- analýza přežití MeSH
- dárci tkání MeSH
- dospělí MeSH
- dyskeratosis congenita komplikace mortalita terapie MeSH
- lidé MeSH
- mladý dospělý MeSH
- nemoc štěpu proti hostiteli etiologie MeSH
- nemoci kostní dřeně etiologie MeSH
- plicní fibróza etiologie MeSH
- příprava pacienta k transplantaci metody MeSH
- transplantace hematopoetických kmenových buněk škodlivé účinky metody MeSH
- věkové faktory MeSH
- výsledek terapie MeSH
- Check Tag
- dospělí MeSH
- lidé MeSH
- mladý dospělý MeSH
- mužské pohlaví MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
Dyskeratosis congenita (DC) is a genetic multisystem disorder with frequent involvement of the bone marrow. Haematopoietic stem cell transplantation (HSCT) is the only definitive cure to restore haematopoiesis, even though it cannot correct other organ dysfunctions. We collected data on the outcome of HSCT in the largest cohort of DC (n = 94) patients ever studied. Overall survival (OS) and event-free survival (EFS) at 3 years after HSCT were 66% and 62%, respectively. Multivariate analysis showed better outcomes in patients aged less than 20 years and in patients transplanted from a matched, rather than a mismatched, donor. OS and EFS curves tended to decline over time. Early lethal events were infections, whereas organ damage and secondary malignancies appeared afterwards, even a decade after HSCT. A non-myeloablative conditioning regimen appeared to be most advisable. Organ impairment present before HSCT seemed to favour the development of chronic graft-versus-host disease and T-B immune deficiency appeared to enhance pulmonary fibrosis. According to the present data, HSCT in DC is indicated in cases of progressive marrow failure, whereas in patients with pre-existing organ damage, this should be carefully evaluated. Further efforts to investigate treatment alternatives to HSCT should be encouraged.
Bone Marrow Transplantation Department Great Ormond Street Hospital London United Kingdom
Bone Marrow Transplantation Department Russian Children's Hospital Moscow Russia
Bone Marrow Transplantation Unit Istituto Giannina Gaslini Genoa Italy
Department of Biochemistry and Medical Biotechnologies Federico 2 University Naples Italy
Department of Biology Università TorVergata Rome Italy
Department of Haematology Leiden University Hospital Leiden The Netherlands
Department of Haematology Oncology and Stem Cell Therapy King Faisal's Hospital Riyadh Saudi Arabia
Department of Haematology Oncology Hospital Infantil Universitario Nino Jesus Madrid Spain
Department of Haematology Ospedale San Martino Genoa Genova Italy
Department of Haematology St Louis Hospital Paris France
Department of Haematology St Mary's Hospital London United Kingdom
Department of Immunology Necker's Hospital Paris France
Department of Paediatric Haematology and Oncology Charles University Prague Czech Republic
Department of Paediatric Haematology Our Lady's Children's Hospital Dublin Ireland
Federal Research Centre of Paediatric Haematology Oncology and Immunology Moscow Russia
Haemato Immunology Department Robert Debre Hospital and Paris Diderot University Paris France
Haematology Oncology and Stem Cell Transplantation Research Centre Shariati Hospital Teheran Iran
Haematology Unit Istituto Giannina Gaslini Genoa Italy
Haematology Unit Sahlgrenska University Göteborg Sweden
Kids Cancer Centre Sydney Children's Hospital Sydney Australia
Paediatric Haematology and Oncology Hospital Vall d'Hebron Barcelona Spain
Paediatric Haematology Oncology Fondazione IRCCS Policlinico San Matteo Pavia Italy
Paediatric Stem Cell Transplantation Leiden University Medical Centre Leiden The Netherlands
Citace poskytuje Crossref.org
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