Reference : Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients ...
Scientific journals : Article
Human health sciences : Hematology
http://hdl.handle.net/2268/8035
Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium.
English
Vermylen, C. mailto [> > > >]
Cornu, G. mailto [> > > >]
Ferster, Aline mailto [> > > >]
Brichard, B. [> > > >]
Ninane, J. [> > > >]
Ferrant, A. mailto [> > > >]
Zenebergh, A. [> > > >]
Maes, P. [> > > >]
Dhooge, C. [> > > >]
Benoit, Y. mailto [> > > >]
Beguin, Yves mailto [Centre Hospitalier Universitaire de Liège - CHU > > Hématologie clinique >]
Dresse, Marie-Françoise mailto [Centre Hospitalier Universitaire de Liège - CHU > > Pédiatrie CHR >]
Sariban, E. mailto [> > > >]
1998
Bone Marrow Transplantation
Nature Publishing Group
22
1
1-6
Yes (verified by ORBi)
International
0268-3369
London
United Kingdom
[en] Adolescent ; Adult ; Amenorrhea/etiology ; Anemia, Sickle Cell/therapy ; Belgium ; Child ; Child, Preschool ; Female ; Graft Rejection ; Graft Survival ; Graft vs Host Disease/etiology ; Hematopoietic Stem Cell Transplantation/adverse effects ; Humans ; Infant ; Male ; Seizures/etiology
[en] Fifty patients affected by sickle cell anaemia underwent transplantation of HLA-identical haematopoietic stem cells (bone marrow, 48; cord blood, 2). Two groups of patients were considered for transplantation. Group 1 included 36 permanent residents of a European country who, retrospectively, met the inclusion criteria accepted at a consensus conference held in Seattle in 1990, wherein children were selected because they already had evidence of a morbid course. Group 2 included 14 patients who were transplanted earlier, had not received more than three blood transfusions and were transplanted because they had decided to return to their country of origin. Kaplan-Meier estimates of overall survival, event-free survival and disease-free survival at 11 years of the whole grafted population are 93, 82 and 85%, respectively. In group 1, overall survival, EFS and DFS were 88, 76 and 80% and in group 2, 100, 93 and 93%, respectively. Clinical manifestations of the disease, as well as disease associated haemolytic anaemia, disappeared in all successfully treated patients. Recovery of spleen function was present in seven out of 10 evaluated patients. Adverse events (death, absence of engraftment, mixed chimerism and relapse) occurred more frequently in group 1 than in group 2 (25% vs 7%, P< 0.001). Acute graft-versus-host disease (GVHD) was present in 20 patients (grade I or II, 19; grade III, 1), chronic GVHD in 10 (limited, 7; extensive, 3). One patient developed an acute myeloid leukaemia. Gonadal dysfunction was present in all patients (six boys and eight girls) transplanted close to or after puberty, although transient in one adolescent girl.
http://hdl.handle.net/2268/8035
10.1038/sj.bmt.1701291

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