Raciborska Anna, Bilska Katarzyna, Rodriguez-Galindo Carlos
Department of Oncology and Surgical Oncology for Children and Youth, Institute of Mother and Child, Warsaw, Poland.
Departments of Global Pediatric Medicine and Oncology, St. Jude Children's Research Hospital, Memphis, TN, US.
Dev Period Med. 2019;23(1):39-44. doi: 10.34763/devperiodmed.20192301.3944.
Background: Patients with Ewing sarcoma have a dismal outcome. Maintenance treatment with trofosfamide has been proposed as an effective regimen for some paediatric malignancies. Aim: We sought to evaluate the schedule of trofosfamide for patients with high-risk primary bone Ewing sarcoma.
Materials and methods: Fifteen patients with primary bone Ewing sarcoma received treatment with trofosfamide (150 mg/m2 p.o. days 1-10) every 28 days. All patients had standard tumour imaging and laboratory evaluation. All toxicities were documented.
Results: A total of 90 cycles (median 5 cycles/patient) were administered. A complete response was maintained in nine patients, while six patients had disease progression during treatment. Median time to progression was 1.9 months (range 1.8 to 4.6). Eleven patients (73.3%) are alive including nine with no evidence of disease with a median follow-up of 3.9 years (range 1.4 to 7.6). All patients with active disease at the start of the trofosfamide treatment died. There were no significant toxicities.
Conclusions: Treatment with trofosfamide is well-tolerated and could have a role to maintain response in patients with primary bone Ewing sarcoma. Further studies are needed to better define the use of this regimen in the upfront management of those patients.
背景：尤因肉瘤患者预后不佳。已提出用曲磷胺维持治疗作为某些儿童恶性肿瘤的有效方案。目的：我们试图评估曲磷胺用于高危原发性骨尤因肉瘤患者的给药方案。
材料与方法：15例原发性骨尤因肉瘤患者接受曲磷胺治疗（口服150mg/m²，第1 - 10天），每28天一个周期。所有患者均进行标准的肿瘤影像学检查和实验室评估。记录所有毒性反应。
结果：共给药90个周期（中位5个周期/患者）。9例患者维持完全缓解，6例患者在治疗期间病情进展。中位进展时间为1.9个月（范围1.8至4.6个月）。11例患者（73.3%）存活，其中9例无疾病证据，中位随访3.9年（范围1.4至7.6年）。曲磷胺治疗开始时所有有活动性疾病的患者均死亡。无明显毒性反应。
结论：曲磷胺治疗耐受性良好，可能在维持原发性骨尤因肉瘤患者缓解方面发挥作用。需要进一步研究以更好地确定该方案在这些患者初始治疗中的应用。
