title: New approach for bone marrow transplantation in patients with class 3 thalassemia aged younger than 17 years.
abstract: When prepared for transplantation with busulfan (BU) 14 mg/kg and cyclophosphamide (CY) 120 to 160 mg/kg, patients with thalassemia in risk class 3, aged younger than 17 years, who receive transplants from HLA-identical donors, had a 30% incidence of transplant rejection with recurrence of thalassemia. This, relatively poor, outcome was ascribed to insufficient immune suppression or to inadequate eradication of the thalassemic marrow, or both. In an attempt to enhance both immune suppression and eradication of the thalassemic clones, hydroxyurea, azathioprine, and fludarabine were added to the BU and CY. This regimen, called protocol 26, was applied to 33 consecutive patients with class 3 thalassemia aged younger than 17 years and was well tolerated with 93% survival. The incidence of recurrent thalassemia after the transplantation decreased from 30% to 8%.
authors.affiliation: Mediterranean Institute of Hematology, 00152 Rome, Italy.
authors.forename: Pietro
authors.initials: P
authors.lastname: Sodani
authors.forename: David
authors.initials: D
authors.lastname: Gaziev
authors.forename: Paola
authors.initials: P
authors.lastname: Polchi
authors.forename: Buket
authors.initials: B
authors.lastname: Erer
authors.forename: Claudio
authors.initials: C
authors.lastname: Giardini
authors.forename: Emanuele
authors.initials: E
authors.lastname: Angelucci
authors.forename: Donatella
authors.initials: D
authors.lastname: Baronciani
authors.forename: Marco
authors.initials: M
authors.lastname: Andreani
authors.forename: Marisa
authors.initials: M
authors.lastname: Manna
authors.forename: Sonia
authors.initials: S
authors.lastname: Nesci
authors.forename: Barbarella
authors.initials: B
authors.lastname: Lucarelli
authors.forename: Reginald A
authors.initials: RA
authors.lastname: Clift
authors.forename: Guido
authors.initials: G
authors.lastname: Lucarelli
mesh_terms: D000293:Adolescent; D000367:Age Factors; D000970:Antineoplastic Agents / Q000008:administration & dosage; D016026:Bone Marrow Transplantation / Q000379:methods* / Q000401:mortality; D002614:Chelating Agents / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D002985:Clinical Protocols; D003676:Deferoxamine / Q000008:administration & dosage; D017707:Erythrocyte Transfusion; D005260:Female; D006133:Growth Substances / Q000008:administration & dosage; D006410:Hematopoiesis / Q000187:drug effects; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage; D008297:Male; D011379:Prognosis; D012008:Recurrence; D018570:Risk Assessment; D016019:Survival Analysis; D013789:Thalassemia / Q000401:mortality / Q000628:therapy*
chemical_list: D000970:Antineoplastic Agents; D002614:Chelating Agents; D006133:Growth Substances; D007166:Immunosuppressive Agents; D003676:Deferoxamine