Curative potential of fludarabine, melphalan, and non-myeloablative dosage of busulfan in elderly patients with myeloid malignancy
Curative potential of fludarabine, melphalan, and non-myeloablative dosage of busulfan in elderly patients with myeloid malignancy
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氟达拉滨、美法仑和非清髓剂量白消安对老年骨髓恶性肿瘤患者的治疗潜力
DOI：
10.1007/s12185-019-02763-2
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发表时间：
2020
期刊：
影响因子：
2.1
通讯作者：
Yasunori Ueda
中科院分区：
文献类型：
--
作者：
Tomoaki Ueda;Tomoyasu Jo;Kazuya Okada;Yasuyuki Arai;Takayuki Sato;Takeshi Maeda;Tatsuhito Onishi;Yasunori Ueda
虽然氟达拉滨和大剂量美法仑（FLU/MEL）的组合已被广泛用于异基因干细胞移植，但大剂量MEL会导致危及生命的不良事件，尤其是在老年患者中。为了降低MEL的毒性而不丧失其抗白血病作用，我们制定了一个方案，包括FLU（125 mg/m2）、MEL（100 mg/m2）和非清髓性白消安剂量[4 mg/kg口服（口服）或3.2 mg/kg静脉内（iv）; FLU/MEL/BU]。我们回顾性分析了32例在我院接受FLU/MEL/BU治疗的髓系恶性肿瘤患者。中位年龄为59岁，allo-SCT后的中位观察期为8.2年。大多数患者（97%）在移植时的疾病状态得到控制。中性粒细胞植入率为93.3%。5年总生存率（OS）、无病生存率（DFS）、无复发死亡率（NRM）和复发率（RR）分别为68.5%、62.1%、22.0%和15.9%。值得注意的是，FLU/MEL/iv BU的结局非常好，5年OS和DFS分别为75.6%和70.8%，5年NRM和RR分别为19.3%和9.8%。总之，FLU/MEL/BU，特别是FLU/MEL/iv BU，对控制的骨髓恶性肿瘤具有治愈潜力。
Although the combination of fludarabine and high-dose melphalan (FLU/MEL) has been widely used in allogeneic stem cell transplantation, high-dose MEL causes life-threatening adverse events, especially in elderly patients. To reduce the toxicity of MEL without losing its antileukemic effect, we formulated a regimen comprising FLU (125 mg/m2), MEL (100 mg/m2), and a non-myeloablative busulfan dosage [4 mg/kg orally (oral) or 3.2 mg/kg intravenously (iv); FLU/MEL/BU]. We retrospectively analyzed 32 patients with myeloid malignancies who received FLU/MEL/BU at our institute. Median age was 59 years and the median observation period after allo-SCT was 8.2 years. The disease status of most of the patients (97%) at transplantation was controlled. The rate of neutrophil engraftment was 93.3%. The 5-year overall survival (OS), disease-free survival (DFS), non-relapse mortality (NRM), and relapse rate (RR) were 68.5%, 62.1%, 22.0%, and 15.9%, respectively, in all patients. Notably, the outcome of FLU/MEL/iv BU was excellent, with the 5-year OS and DFS being 75.6% and 70.8%, respectively, accompanied by a reduced 5-year NRM and RR of 19.3% and 9.8%, respectively. In conclusion, FLU/MEL/BU, particularly FLU/MEL/iv BU, has curative potential for controlled myeloid malignancies.