Treosulfan/fludarabine as an allogeneic hematopoietic stem cell transplant conditioning regimen for high-risk patients

被引:21
作者
Baronciani, Donatella [1 ,2 ]
Rambaldi, Alessandro [3 ]
Iori, Anna Paola [4 ]
Di Bartolomeo, Paolo [5 ]
Pilo, Federica [1 ,2 ]
Pettinau, Martina [1 ,2 ]
Depau, Cristina [1 ,2 ]
Mico, Caterina [3 ]
Santarone, Stella [5 ]
Angelucci, Emanuele [1 ,2 ]
机构
[1] Osped Oncol Armando Businco, Unita Operat Ematol, I-09121 Cagliari, Italy
[2] Osped Oncol Armando Businco, Ctr Trapianti Wilma Deplano, I-09121 Cagliari, Italy
[3] Osped Riuniti Bergamo, UO Ematol & Ctr Trapianti, Bergamo, Italy
[4] Univ Roma La Sapienza, Ist Ematol, Rome, Italy
[5] Osped Civile Pescara, UO Ematol & Ctr Trapianti, Pescara, Italy
关键词
D O I
10.1002/ajh.21240
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
In years, new conditioning regimens have been explored in patients not eligible for conventional transplant with the aim to reduce transplant-related mortality. In a phase II multicentric prospective trial, we investigated the safety and feasibility of the treosulfan-fludarabine combination prior to allogeneic hematopoietic stem cell transplant in patients with various hematological malignancies not eligible for conventional regimens because of previous intensive treatment, older age, and comorbidities. Forty-six consecutive patients, median age 48 years (range 17-69), were enrolled. Sixteen of them were in complete remission, and 20 had a HSCT comorbidity index >= 1. Forty-four patients had regular and sustained engraftment, and 39 out of 40 evaluable patients developed complete chimerism. Nonhematological toxicity was limited. Risk of transplant-related mortality was 9% (95% CI, 2-17%) at day +100 and plotted at 15% (95% CI, 7-22%) after 7 months. The estimated overall survival and progression-free survival with a median follow-up of 20 months were 51% and 38%, respectively. The estimated 30 months progression-free survival for patients transplanted in remission was 56%. The treosulfan-fludarabine combination is a reduced-toxicity but myeloablative regimen that can be proposed to patients not fitting criteria for conventional transplant regimens. Longer follow-up and further prospective studies are necessary to evaluate this regimen.
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收藏
页码:717 / 720
页数:4
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