Messinger Yoav, Boklan Jessica, Goldberg John, DuBois Steven G, Oesterheld Javier, Abla Oussama, Martin Alissa, Weinstein Joanna, Hijiya Nobuko
a Children's Hospitals and Clinics of Minnesota , Minneapolis , MN , USA.
b Phoenix Children's Hospital , Phoenix , AZ , USA.
Pediatr Hematol Oncol. 2017 May;34(4):187-198. doi: 10.1080/08880018.2017.1360970. Epub 2017 Oct 17.
Relapsed/refractory acute myeloid leukemia (AML) has an extremely poor prognosis. We describe 17 children and adolescents with relapsed/refractory AML who received clofarabine, cyclophosphamide, and etoposide. Seven patients (41%) responded: 4 with a complete response (CR); 1 with CR with incomplete platelet recovery; and 2 with a partial response. Additionally, 4 developed hypocellular marrow without evidence of leukemia; 5 patients had resistant disease; and 1 suffered early toxic death. After further therapy including transplantation, 4 patients (24%) are alive without evidence of disease at a median of 60 months. This anthracycline-free regimen may be studied for relapsed or refractory AML, but due to the high risk of marrow aplasia reduced doses of clofarabine and cyclophosphamide should be used.
复发/难治性急性髓系白血病（AML）的预后极差。我们描述了17例接受氯法拉滨、环磷酰胺和依托泊苷治疗的复发/难治性AML儿童和青少年。7例患者（41%）有反应：4例完全缓解（CR）；1例CR但血小板恢复不完全；2例部分缓解。此外，4例出现骨髓细胞减少但无白血病证据；5例患者疾病耐药；1例早期死于毒性反应。经过包括移植在内的进一步治疗后，4例患者（24%）存活，中位时间60个月，无疾病证据。这种不含蒽环类药物的方案可用于复发或难治性AML的研究，但由于骨髓再生障碍风险高，应使用降低剂量的氯法拉滨和环磷酰胺。
