HEMATOPOIETIC STEM-CELL TRANSPLANTATION FOR SICKLE-CELL-ANEMIA - THE FIRST 50 PATIENTS TRANSPLANTED IN BELGIUM

Fifty patients affected by sickle cell anaemia underwent transplantati on of HLA-identical haematopoietic stem cells (bone marrow, 48; cord b lood, 2), Two groups of patients were considered for transplantation. Group 1 included 36 permanent residents of a European country who, ret rospectively, met the inclusion criteria accepted at a consensus confe rence held in Seattle in 1990, wherein children were selected because they already had evidence of a morbid course. Group 2 included 14 pati ents who were transplanted earlier, had not received more than three b lood transfusions and were transplanted because they had decided to re turn to their country of origin, Kaplan-Meier estimates of overall sur vival, event-free survival and disease-free survival at 11 years of th e 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, 1 00, 93 and 93%, respectively, Clinical manifestations of the disease, as well as disease associated haemolytic anaemia, disappeared in all s uccessfully 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-ho st 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 d eveloped an acute myeloid leukaemia. Gonadal dysfunction was present i n all patients (six boys and eight girls) transplanted close to or aft er puberty, although transient in one adolescent girl.