http://www.cnr.it/ontology/cnr/individuo/prodotto/ID27788

Long-term outcome and lineage-specific chimerism in 194 Wiskott-Aldrich Syndrome patients treated by hematopoietic cell transplantation between 1980-2009: an international collaborative study. (Articolo in rivista)

Type

Prodotto della ricerca (Classe)
Articolo in rivista (Classe)

Label

Long-term outcome and lineage-specific chimerism in 194 Wiskott-Aldrich Syndrome patients treated by hematopoietic cell transplantation between 1980-2009: an international collaborative study. (Articolo in rivista) (literal)

Anno

2011-01-01T00:00:00+01:00 (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#doi

10.1182/blood-2010-11-319376 (literal)

Alternative label

Moratto D; Giliani S; Bonfim C; Mazzolari E; Fischer A; Ochs HD; Cant AJ; Thrasher AJ; Cowan MJ; Albert MH; Small T; Pai SY; Haddad E; Lisa A; Hambleton S; Slatter M; Cavazzana-Calvo M; Mahlaoui N; Picard C; Torgerson TR; Burroughs L; Koliski A; Neto JZ; Porta F; Qasim W; Veys P; Kavanau K; Hönig M; Schulz A; Friedrich W; Notarangelo LD. (2011)
Long-term outcome and lineage-specific chimerism in 194 Wiskott-Aldrich Syndrome patients treated by hematopoietic cell transplantation between 1980-2009: an international collaborative study.
in Blood; WB Saunders Co.-Elsevier Inc., Philadelphia (Stati Uniti d'America)
(literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#autori

Moratto D; Giliani S; Bonfim C; Mazzolari E; Fischer A; Ochs HD; Cant AJ; Thrasher AJ; Cowan MJ; Albert MH; Small T; Pai SY; Haddad E; Lisa A; Hambleton S; Slatter M; Cavazzana-Calvo M; Mahlaoui N; Picard C; Torgerson TR; Burroughs L; Koliski A; Neto JZ; Porta F; Qasim W; Veys P; Kavanau K; Hönig M; Schulz A; Friedrich W; Notarangelo LD. (literal)

Pagina inizio

1675 (literal)

Pagina fine

1684 (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#url

http://bloodjournal.hematologylibrary.org/content/118/6/1675.long (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#numeroVolume

118 (literal)

Rivista

Blood (Rivista)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#pagineTotali

13 (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#numeroFascicolo

6 (literal)

Note

Scopu (literal)
PubMe (literal)
ISI Web of Science (WOS) (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#affiliazioni

\"A. Nocivelli\" Institute for Molecular Medicine, Pediatric Clinic, University of Brescia, and Laboratory of Genetic Disorders of Childhood, Spedali Civili, Brescia, Italy; Bone Marrow Transplantation Unit, Federal University of Parana, Curitiba, Brazil; Department of Haematology/Oncology, Spedali Civili, Brescia, Italy Necker Medical School and Paris Descartes University, France; Center for Immunity and Immunotherapies, Seattle Children's Research Institute, University of Washington, Seattle, USA; Children's Bone Marrow Transplant Unit, Great North Childrens Hospital, Newcastle, UK 7 Centre for Immunodeficiency, Institute of Child Health, London, UK; Division of Blood and Marrow Transplantation, UCSF Children's Hospital, San Francisco, USA; Department of Pediatric Haematology/Oncology, Dr. von Haunersches Kinderspital, Munich, Germany; Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, USA; Department of Pediatric Hematology-Oncology, Children's Hospital, Boston, USA; Division of Immunology, Department of Pediatrics, CHU Sainte-Justine, Universite de Montreal, Montreal, Canada ; Institute of Molecular Genetics - CNR - National Research Council of Italy, Pavia, Italy; Department of Pediatrics, University of Ulm, Ulm, Germany; Pediatric Hematology-Immunology Unit, Necker Hospital, Assistance Publique Hopitaux de Paris, Paris, France; Study Center of Primary Immunodeficiencies, Assistance Publique Hopitaux de Paris, Necker Hospital, Paris, France; Department of Bone Marrow Transplantation, Great Ormond Street Children's Hospital NHS Trust, London, United Kingdom, Division of Immunology and The Manton Center for Orphan Disease Research Childrens Hospital Boston Karp Building, Boston, Cancer Research Center and University of Washington, Seattle, USA; Division of Immunology and The Manton Center for Orphan Disease Research, Children's Hospital, Harvard Medical School, Boston, USA. (literal)

Titolo

Long-term outcome and lineage-specific chimerism in 194 Wiskott-Aldrich Syndrome patients treated by hematopoietic cell transplantation between 1980-2009: an international collaborative study. (literal)

Abstract

In this retrospective collaborative study, we have analyzed long-term outcome and donor cell engraftment in 194 patients with the Wiskott-Aldrich syndrome who have been treated by hematopoietic cell transplantation (HCT) in the period 1980-2009. Overall survival was 84.0%, and was even higher (89.1% 5-year survival) for those who received HCT since the year 2000, reflecting recent improvement of outcome following transplants from mismatched family donors and for patients who received HCT from an unrelated donor at more than 5 years of age. Patients who went to transplant in better clinical conditions had a lower rate of post-HCT complications. Retrospective analysis of lineage-specific donor cell engraftment showed that stable full donor chimerism was attained by 72.3% of the patients who survived for at least one year after HCT. Mixed chimerism was associated with an increased risk of incomplete reconstitution of lymphocyte count and post-HCT autoimmunity, and myeloid donor cell chimerism <50% was associated with persistent thrombocytopenia. These observations indicate continuous improvement of outcome after HCT for WAS, and may have important implications for the development of novel protocols aiming to obtain full correction of the disease and reduce post-HCT complications. (literal)

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