Higham Christine S, Steinberg Seth M, Dombi Eva, Perry Arie, Helman Lee J, Schuetze Scott M, Ludwig Joseph A, Staddon Arthur, Milhem Mohammed M, Rushing Daniel, Jones Robin L, Livingston Michael, Goldman Stewart, Moertel Christopher, Wagner Lars, Janhofer David, Annunziata Christina M, Reinke Denise, Long Lauren, Viskochil David, Baker Larry, Widemann Brigitte C
Pediatric Oncology Branch, NCI, CCR, Bethesda, MD, USA.
Biostatistics and Data Management Section, NCI, Bethesda, MD, USA.
Sarcoma. 2017;2017:8685638. doi: 10.1155/2017/8685638. Epub 2017 Sep 12.
Worse chemotherapy response for neurofibromatosis type 1- (NF1-) associated compared to sporadic malignant peripheral nerve sheath tumors (MPNST) has been reported.
We evaluated the objective response (OR) rate of patients with AJCC Stage III/IV chemotherapy-naive NF1 MPNST versus sporadic MPNST after 4 cycles of neoadjuvant chemotherapy, 2 cycles of ifosfamide/doxorubicin, and 2 cycles of ifosfamide/etoposide. A Simon optimal two-stage design was used (target response rate 40%).
34 NF1 (median age 33 years) and 14 sporadic (median age 40 years) MPNST patients enrolled. Five of 28 (17.9%) evaluable NF1 MPNST patients had a partial response (PR), as did 4 of 9 (44.4%) patients with sporadic MPNST. Stable disease (SD) was achieved in 22 NF1 and 4 sporadic MPNST patients. In both strata, results in the initial stages met criteria for expansion of enrollment. Only 1 additional PR was observed in the expanded NF1 stratum. Enrollment was slower than expected and the trial closed before full accrual.
This trial was not powered to detect differences in response rates between NF1 and sporadic MPNST. While the OR rate was lower in NF1 compared to sporadic MPNST, qualitative responses were similar, and disease stabilization was achieved in most patients.
据报道，与散发性恶性外周神经鞘瘤（MPNST）相比，1型神经纤维瘤病（NF1）相关的MPNST化疗反应更差。
我们评估了AJCC III/IV期初治NF1 MPNST患者与散发性MPNST患者在接受4个周期新辅助化疗（2个周期异环磷酰胺/阿霉素和2个周期异环磷酰胺/依托泊苷）后的客观缓解（OR）率。采用西蒙最优两阶段设计（目标缓解率40%）。
34例NF1（中位年龄33岁）和14例散发性（中位年龄40岁）MPNST患者入组。28例可评估的NF1 MPNST患者中有5例（17.9%）获得部分缓解（PR），9例散发性MPNST患者中有4例（44.4%）获得部分缓解。22例NF1和4例散发性MPNST患者病情稳定（SD）。在两个分层中，初始阶段的结果均符合扩大入组标准。在扩大的NF1分层中仅观察到1例额外的PR。入组速度慢于预期，试验在完全入组前结束。
该试验无足够效力检测NF1和散发性MPNST之间缓解率的差异。虽然NF1的OR率低于散发性MPNST，但定性反应相似，大多数患者病情得到稳定。
