Treatment of acute lymphoblastic leukemia (ALL) is still a challenge despite years of researching, especially for those of poor prognosis. Zhang and his team recently proved that FLT3 gene mutation was identified in ~5% of ALL and the mutation spectrum is different from AML. Recently, chimeric antigen receptor T cells (CART) therapy presents great efficacy in treating refractory leukemia. We report a case of a refractory ALL patient with FLT3-ITD mutations and unfavorable karyotypes, who failed to respond to chemotherapy and small molecule tyrosine kinase inhibitors, successfully treated by CART therapy. FLT3-ITD mutations were downregulated dramatically into 14.1% positive 3 days after the infusion and remained negative until now. MRD has stayed to be negative from the 10th day. This case suggests that CART-cell therapy might be effective in treating FLT3-ITD positive refractory ALL, implying the possibility to overcome the traditional prognosis scoring system for leukemia and providing a new chance for other leukemia patients with inferior prognosis factors.

尽管经过多年的研究，急性淋巴细胞白血病 (ALL) 的治疗仍然是一个挑战，尤其是对于那些预后不良的患者。Zhang 和他的团队最近证明 FLT3 基因突变在约 5% 的 ALL 中被发现，并且突变谱与 AML 不同。最近，嵌合抗原受体 T 细胞 (CART) 疗法在治疗难治性白血病方面表现出极大的疗效。我们报告了一例具有 FLT3-ITD 突变和不利核型的难治性 ALL 患者，他们对化疗和小分子酪氨酸激酶抑制剂没有反应，通过 CART 治疗成功。FLT3-ITD 突变在输注 3 天后显着下调至 14.1% 阳性，并且直到现在仍保持阴性。从第 10 天开始，MRD 一直为阴性。