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Titolo:
Neonatal bone marrow transplantation for severe combined immunodeficiency
Autore:
Kane, L; Gennery, AR; Crooks, BNA; Flood, TJ; Abinun, M; Cant, AJ;
Indirizzi:
Newcastle Gen Hosp, Dept Paediat Immunol, Newcastle Upon Tyne NE4 6BE, Tyne & Wear, England Newcastle Gen Hosp Newcastle Upon Tyne Tyne & Wear England NE4 6BE ngland
Titolo Testata:
ARCHIVES OF DISEASE IN CHILDHOOD
fascicolo: 2, volume: 85, anno: 2001,
pagine: F110 - F113
SICI:
0003-9888(200109)85:2<F110:NBMTFS>2.0.ZU;2-7
Fonte:
ISI
Lingua:
ENG
Soggetto:
IN-UTERO TRANSPLANTATION; CELLS; DEFICIENCY; MUTATIONS;
Keywords:
severe combined immunodeficiency; bone marrow transplantation; in utero transplantation;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Clinical Medicine
Life Sciences
Citazioni:
14
Recensione:
Indirizzi per estratti:
Indirizzo: Gennery, AR Newcastle Gen Hosp, Dept Paediat Immunol, Westgate Rd, Newcastle Upon TyneNE4 6BE, Tyne & Wear, England Newcastle Gen Hosp Westgate Rd Newcastle Upon Tyne Tyne & Wear England NE4 6BE
Citazione:
L. Kane et al., "Neonatal bone marrow transplantation for severe combined immunodeficiency", ARCH DIS CH, 85(2), 2001, pp. F110-F113

Abstract

Aims-To evaluate outcome following neonatal bone marrow transplantation (BMT) for severe combined immunodeficiency (SCID) when there is a family history of a previously affected sibling, and to compare results with those published for in utero BMT. Methods-A retrospective review of cases referred and transplanted between 1987 and 1999, focusing on infectious and graft versus host disease (GvHD) complications after BMT, and T and B lymphocyte function. Thirteen patientsreceived 18 stem cell transplants: four whole marrow, one cord blood, 10 parental T cell depleted (TCD) haplo-identical, and three TCD unrelated donor BMT. Nine were conditioned with busulphan and cyclophosphamide. Results-All are alive and well (six months to 11.5 years after BMT). Six had grade I-II acute GvHD and two chronic GvHD (now resolved). Three had a top up BMT for poor T cell function, one had a third BMT for graft failure and chronic GvHD, and one had a third BMT for graft failure. Twelve have good in vitro proliferation to T cell mitogens, and all have normal serum IgA levels. Three receive intravenous immunoglobulin; for one of these, it is less than one year since BMT. Nine are above the 2nd centile, and 10 of 12 old enough to be assessed have normal neurodevelopment. Conclusion-These results are better than those published for in utero BMT for SCID. Early postnatal BMT should be the preferred option in neonatal SCID.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 27/11/20 alle ore 22:27:59