New approach for bone marrow transplantation in patients with class 3 thalassemia aged younger than 17 years

被引:144
作者
Sodani, P
Gaziev, D
Polchi, P
Erer, B
Giardini, C
Angelucci, E
Baronciani, D
Andreani, M
Manna, M
Nesci, S
Lucarelli, B
Clift, RA
Lucarelli, G [1 ]
机构
[1] Mediterranean Inst Hematol, I-00152 Rome, Italy
[2] Fred Hutchinson Canc Res Ctr, Clin Div, Seattle, WA 98104 USA
[3] Azienda Osped S Salvatore Pesaro, Unita Operat Ematol, Pesaro, Italy
[4] Azienda Osped S Salvatore Pesaro, Ctr Trapianto Midollo Osseo Muraglia, Pesaro, Italy
关键词
D O I
10.1182/blood-2003-08-2800
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
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 in-sufficient 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%. (C) 2004 by The American Society of Hematology.
引用
收藏
页码:1201 / 1203
页数:3
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