[Major beta-thalassemia and bone marrow allograft. Two cases and a study of the literature].
A 5 year 9 month-old boy has received a bone marrow allograft for beta-thalassaemia major. Conditioning included busulfan: 16 mg/kg, cyclophosphamide 200 mg/kg and a (6 Gy) thoracoabdominal irradiation. After a 16 months follow-up, the child is currently in complete remission without treatment with all the markers of his donor. His 9 year-old sister has been allografted for beta-thalassaemia major, with the same conditioning regimen. After engraftment, rejection occurred at day 85 with severe aplastic anaemia. A second graft was performed with the same donor without engraftment and the patient died at day 18 of pneumonitis. A review of the literature is proposed and the ethical choices are discussed.
Frappaz
D
D
Service de pédiatrie, Hôpital Nord, Saint-Priest-en Jarez, France.
Freycon
F
F
Richard
O
O
Galacteros
F
F
Bertheas
M F
MF
Dutou
L
L
Bouteille
M
M
D016026:Bone Marrow Transplantation; D002066:Busulfan / Q000627:therapeutic use; D003520:Cyclophosphamide / Q000627:therapeutic use; D006801:Humans; D007223:Infant; D008297:Male; D011183:Postoperative Complications; D013789:Thalassemia / Q000628:therapy*; D013909:Thorax / Q000528:radiation effects; D014184:Transplantation, Homologous
D003520:Cyclophosphamide; D002066:Busulfan
Bêta-thalassémie majeure et allogreffe médullaire. Deux observations et une étude de la littérature.