Department of Internal Medicine and Hematology, AV3, ASUR Marche, Macerata, Italy.
Acute Leukemia Working Party - European Society for Blood and Marrow Transplantation, Paris, France.
J Hematol Oncol. 2019 Apr 25;12(1):44. doi: 10.1186/s13045-019-0727-4.
Limited data is available to guide the choice of the conditioning regimen for patients with acute myeloid leukemia (AML) undergoing transplant with persistent disease.
We retrospectively compared outcome of fludarabine-treosulfan (FT), thiotepa-busulfan-fludarabine (TBF), and sequential fludarabine, intermediate dose Ara-C, amsacrine, total body irradiation/busulfan, cyclophosphamide (FLAMSA) conditioning in patients with refractory or relapsed AML.
Complete remission rates at day 100 were 92%, 80%, and 88% for FT, TBF, and FLAMSA, respectively (p = 0.13). Non-relapse mortality, incidence of relapse, acute (a) and chronic (c) graft-versus-host disease (GVHD) rates did not differ between the three groups. Overall survival at 2 years was 37% for FT, 24% for TBF, and 34% for FLAMSA (p = 0.10). Independent prognostic factors for survival were Karnofsky performance score and patient CMV serology (p = 0.01; p = 0.02), while survival was not affected by age at transplant. The use of anti-thymocyte globulin (ATG) was associated with reduced risk of grade III-IV aGVHD (p = 0.02) and cGVHD (p = 0.006), with no influence on relapse.
In conclusion, FT, TBF, and FLAMSA regimens provided similar outcome in patients undergoing transplant with active AML. Survival was determined by patient characteristics as Karnofsky performance score and CMV serology, however was not affected by age at transplant. ATG appears able to reduce the incidence of acute and chronic GVHD without influencing relapse risk.
对于患有持续性疾病的急性髓系白血病（AML）患者进行移植时，可供选择的条件方案的数据有限。
我们回顾性比较了氟达拉滨-替伊莫单抗（FT）、噻替哌-白消安-氟达拉滨（TBF）和序贯氟达拉滨、中剂量阿糖胞苷、安吖啶、全身照射/白消安、环磷酰胺（FLAMSA）在难治性或复发性 AML 患者中的疗效。
FT、TBF 和 FLAMSA 组第 100 天的完全缓解率分别为 92%、80%和 88%（p=0.13）。三组之间非复发死亡率、复发率、急性（a）和慢性（c）移植物抗宿主病（GVHD）发生率无差异。FT、TBF 和 FLAMSA 的 2 年总生存率分别为 37%、24%和 34%（p=0.10）。生存的独立预后因素为卡诺夫斯基表现评分和患者 CMV 血清学（p=0.01；p=0.02），而移植时的年龄对生存没有影响。使用抗胸腺细胞球蛋白（ATG）与降低 III-IV 级 aGVHD（p=0.02）和 cGVHD（p=0.006）的风险相关，而对复发没有影响。
综上所述，FT、TBF 和 FLAMSA 方案在患有活动性 AML 的患者进行移植时提供了相似的疗效。生存由患者特征决定，如卡诺夫斯基表现评分和 CMV 血清学，但不受移植时年龄的影响。ATG 似乎能够降低急性和慢性 GVHD 的发生率，而不影响复发风险。
