Hunold Andrea, Weddeling Nicole, Paulussen Michael, Ranft Andreas, Liebscher Caren, Jürgens Herbert
Department of Pediatric Hematology and Oncology, University Children's Hospital, Muenster, Germany.
Pediatr Blood Cancer. 2006 Nov;47(6):795-800. doi: 10.1002/pbc.20719.
The prognosis of Ewing tumor (ET) patients has significantly improved to cure rates approximating 70%. The prognosis in relapse, however, is poor. Promising response rates have recently been reported for the combination of topotecan (TOPO) and cyclophosphamide (CYC) encouraging wider application of this combination in patients with relapsed ETs. This report summarizes the experience of patients treated with TOPO/CYC for recurrent or refractory disease within the German ET trials.
Fifty-four patients aged 3.2-49.5 (median: 17.4) years received TOPO (0.75 mg/m2/day, days 1-5) and CYC (250 mg/m2/day, days 1-5) following first (40) or second (6) relapse or progression under first-line therapy (8).
A median of 3 (range: 1-11) TOPO/CYC courses were given. Sixteen patients (32.6%) showed partial response (PR), 13/49 (26.5%) had stable disease (SD), 14/49 (28.6%) progressed, 2/49 (4.1%) showed a mixed response (MR). In 4 patients response was not documented, 5/54 patients with complete initial resection at the diagnosis of relapse were excluded from the response analysis. At completion of relapse therapy, 24/54 patients had entered complete (19) or partial (5) remission, 2 had SD, 26 showed progression, information was unavailable in 2 patients. Of the 19 relapse patients achieving complete response (CR), 10 maintained remission (52.6%). At the time of evaluation, after a median follow-up for survivors of 23.1 (range: 7.8-59.8) months from the event prompting TOPO/CYC treatment, 14/54 patients (25.9%) were in continuous complete (13) or partial (1) remission. Overall survival (OAS) after 1 year was 0.61 (95%-CI 0.47-0.74).
TOPO/CYC is active in relapsed ETs and warrants further evaluation.
尤因肉瘤（ET）患者的预后已显著改善，治愈率接近70%。然而，复发后的预后较差。最近有报道称，拓扑替康（TOPO）与环磷酰胺（CYC）联合使用的缓解率令人鼓舞，这促使该联合方案在复发ET患者中得到更广泛应用。本报告总结了在德国ET试验中接受TOPO/CYC治疗复发性或难治性疾病患者的经验。
54例年龄在3.2 - 49.5岁（中位数：17.4岁）的患者在首次（40例）或第二次（6例）复发或一线治疗（8例）进展后接受TOPO（0.75 mg/m²/天，第1 - 5天）和CYC（250 mg/m²/天，第1 - 5天）治疗。
中位给予3（范围：1 - 11）个TOPO/CYC疗程。16例患者（32.6%）显示部分缓解（PR），13/49例（26.5%）疾病稳定（SD），14/49例（28.6%）进展，2/49例（4.1%）显示混合反应（MR）。4例患者的反应未记录，5/54例在复发诊断时初始完全切除的患者被排除在反应分析之外。复发治疗结束时，24/54例患者进入完全（19例）或部分（5例）缓解，2例疾病稳定，26例进展，2例患者信息不可用。在19例达到完全缓解（CR）的复发患者中，10例维持缓解（52.6%）。在评估时，从启动TOPO/CYC治疗事件起，幸存者的中位随访时间为23.1（范围：7.8 - 59.8）个月，14/54例患者（25.9%）处于持续完全（13例）或部分（例）缓解状态。1年后总生存率（OAS）为0.61（95%可信区间0.47 - 0.74）。
TOPO/CYC对复发ET有效，值得进一步评估。
