Department of Stem Cell Transplantation, The University of Texas M.D. Anderson Cancer Center, Houston, Texas 77030-4009, USA.
Biol Blood Marrow Transplant. 2011 Dec;17(12):1874-7. doi: 10.1016/j.bbmt.2011.07.011. Epub 2011 Jul 20.
Patients with acute myeloid leukemia (AML) and internal tandem duplication of FMS-like tyrosine kinase receptor-3 gene (FLT3-ITD) mutation have poor prognoses and are often treated with allogeneic hematopoietic stem cell transplantation (HSCT). Sorafenib, an inhibitor of multiple kinases including FLT3, has shown promising activity in FLT3-ITD-positive AML. We treated 16 patients with FLT3-ITD-positive AML who relapsed after HSCT with sorafenib alone (n = 8) or in combination with cytotoxic chemotherapy (n = 8). The number of circulating blasts decreased in 80% of cases, but none of the patients achieved complete remission (CR); 3 achieved partial remission. Two patients were bridged to a second transplantation but both relapsed within 3 months of the transplantation. Median overall survival (OS) was 83 days, with none surviving more than a year. Sorafenib is not effective in the treatment of FLT3-ITD-positive AML relapsing after HSCT. Preventive strategies after HSCT may be more suitable for these high-risk patients.
FLT3-ITD 阳性 AML 患者经 HSCT 后复发，采用索拉非尼单药或联合细胞毒化疗治疗
患有急性髓系白血病（AML）和 FMS 样酪氨酸激酶受体-3 基因内部串联重复突变（FLT3-ITD）的患者预后较差，常采用异基因造血干细胞移植（HSCT）治疗。索拉非尼是一种多激酶抑制剂，包括 FLT3，在 FLT3-ITD 阳性 AML 中显示出良好的活性。我们采用索拉非尼单药（n = 8）或联合细胞毒化疗（n = 8）治疗 16 例 HSCT 后复发的 FLT3-ITD 阳性 AML 患者。80%的患者循环中 blast 减少，但无完全缓解（CR）病例，3 例部分缓解。2 例患者桥接至二次移植，但均在移植后 3 个月内复发。中位总生存期（OS）为 83 天，无患者存活超过 1 年。索拉非尼对 HSCT 后复发的 FLT3-ITD 阳性 AML 无效。HSCT 后预防策略可能更适合这些高危患者。
