H. Rubie et al., INTENSIFIED CONDITIONING REGIMEN WITH BUSULFAN FOLLOWED BY ALLOGENEICBMT IN CHILDREN WITH MYELODYSPLASTIC SYNDROMES, Bone marrow transplantation, 13(6), 1994, pp. 759-762
Four consecutive children with myelodysplastic syndromes (MDS) underwe
nt matched allogeneic bone marrow transplantation (BMT). Ages ranged f
rom 3.2 to 6.3 years. Diagnosis was assessed according to FAB classifi
cation: refractory anemia-RA (n = 1), RA with excess of blasts (RAEB)
(n = 1), and juvenile chronic myelogenous leukemia (JCML) (n = 2). Ini
tial treatment included transfusions for all of them, splenectomy (n =
2) and chemotherapy (n = 1). Patients were all prepared with busulfan
21 mg/kg (480 mg/m(2)), cytosine arabinoside 24 000 mg/m(2), melphala
n 140 mg/m(2). Graft-versus-host disease (GVHD) prophylaxis associated
cyclosporine-methotrexate. Engraftment was prompt and complete in all
children. Toxicity included severe mucositis (n = 3), moderate veno-o
cclusive disease (n = 2), acute GVHD (n = 3), chronic GVHD (n = 1). Se
quelae have not yet been seen. All patients are alive and disease-free
with a follow-up ranging from 7 to 35 months, with a Karnofsky score
of 90-100%. Combined busulphan conditioning can offer an alternative t
o total body irradiation-based regimens in order to avoid late side-ef
fects in children.