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Allogeneic hematopoietic stem cell transplantation with fludarabine, busulfan, and thiotepa conditioning is associated with favorable outcomes in myelofibrosis.
Bone Marrow Transplantation ( IF 4.5 ) Pub Date : 2019-08-28 , DOI: 10.1038/s41409-019-0653-7
Roni Shouval 1, 2 , Yakov Vega 1 , Joshua A Fein 1 , Ivetta Danylesko 1 , Noga Shem Tov 1 , Ronit Yerushalmi 1 , Marta Sobas 3 , Anna Czyż 3 , Arnon Nagler 1 , Avichai Shimoni 1
Bone Marrow Transplantation ( IF 4.5 ) Pub Date : 2019-08-28 , DOI: 10.1038/s41409-019-0653-7
Roni Shouval 1, 2 , Yakov Vega 1 , Joshua A Fein 1 , Ivetta Danylesko 1 , Noga Shem Tov 1 , Ronit Yerushalmi 1 , Marta Sobas 3 , Anna Czyż 3 , Arnon Nagler 1 , Avichai Shimoni 1
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Allogeneic stem cell transplantation is a curative therapy for myelofibrosis. The optimal conditioning regimen has not been well defined. We retrospectively compared transplantation outcomes in patients with myelofibrosis (n = 67) conditioned with myeloablative (MAC, 36%) and reduced-intensity (RIC, 46%) regimens, and more recently with the combination of thiotepa, busulfan, and fludarabine (TBF, 18%). Patients were transplanted from HLA-matched sibling (n = 26) or unrelated donors (n = 41) between the years 2003 and 2018. The median follow-up was 2.9 years for all patients but shorter in the TBF group (1.1 years). The probability of 3-year progression-free survival (PFS) was 43%. At 1 year, the rate of PFS was 80%, 54%, and 45% with TBF, MAC, and RIC, respectively (p = 0.031). In a multivariable model, there was a greater risk for death with MAC (hazard ratio [HR] 12.26, p = 0.026) and lower PFS with both MAC (hazard ratio [HR] 7.78, p = 0.017) and RIC (HR 5.43, p = 0.027) compared with TBF. Relapse was higher with RIC (HR 8.20, p = 0.043) while nonrelapse mortality was increased with MAC (HR 9.63 p = 0.049). Our results indicate that TBF is a promising preparative regimen in myelofibrosis patients transplanted from matched sibling or unrelated donors, and should be further explored.
中文翻译:
氟达拉滨,白消安和硫替泰条件的同种异体造血干细胞移植与骨髓纤维化的良好预后相关。
同种异体干细胞移植是一种治疗骨髓纤维化的方法。最佳的调理方案还没有很好的定义。我们回顾性地比较了采用清髓治疗(MAC,36%)和降低强度治疗(RIC,46%)的条件治疗的骨髓纤维化患者(n = 67)的移植结局,最近比较了噻替太帕,白消安和氟达拉滨(TBF)的组合,18%)。在2003年至2018年之间,从HLA匹配的同胞(n = 26)或无关的供体(n = 41)移植患者。所有患者的中位随访时间为2.9年,而TBF组的中位随访时间为1.1年。3年无进展生存期(PFS)的可能性为43%。1年时,TBF,MAC和RIC的PFS率分别为80%,54%和45%(p = 0.031)。在多变量模型中,与MAC(危险比[HR] 7.78,p = 0.017)和RIC(HR 5.43,p = 0.027)相比,MAC的死亡风险较高(危险比[HR] 12.26,p = 0.026),而PFS较低。与TBF。RIC的复发率更高(HR 8.20,p = 0.043),而MAC的复发率却更高(HR 9.63 p = 0.049)。我们的结果表明,TBF是从配对的同胞或无关亲戚供体移植的骨髓纤维化患者中一种有希望的制备方案,应进一步探索。
更新日期:2019-08-29
中文翻译:
![](https://scdn.x-mol.com/jcss/images/paperTranslation.png)
氟达拉滨,白消安和硫替泰条件的同种异体造血干细胞移植与骨髓纤维化的良好预后相关。
同种异体干细胞移植是一种治疗骨髓纤维化的方法。最佳的调理方案还没有很好的定义。我们回顾性地比较了采用清髓治疗(MAC,36%)和降低强度治疗(RIC,46%)的条件治疗的骨髓纤维化患者(n = 67)的移植结局,最近比较了噻替太帕,白消安和氟达拉滨(TBF)的组合,18%)。在2003年至2018年之间,从HLA匹配的同胞(n = 26)或无关的供体(n = 41)移植患者。所有患者的中位随访时间为2.9年,而TBF组的中位随访时间为1.1年。3年无进展生存期(PFS)的可能性为43%。1年时,TBF,MAC和RIC的PFS率分别为80%,54%和45%(p = 0.031)。在多变量模型中,与MAC(危险比[HR] 7.78,p = 0.017)和RIC(HR 5.43,p = 0.027)相比,MAC的死亡风险较高(危险比[HR] 12.26,p = 0.026),而PFS较低。与TBF。RIC的复发率更高(HR 8.20,p = 0.043),而MAC的复发率却更高(HR 9.63 p = 0.049)。我们的结果表明,TBF是从配对的同胞或无关亲戚供体移植的骨髓纤维化患者中一种有希望的制备方案,应进一步探索。