De Bernardi B, Pastore G, Carli M, Ceci A, Paolucci G, Madon E, Mancini A, Tamaro P, Rebuffi L, Colella R, Cozzutto C
Cancer. 1982 Jul 1;50(1):10-7. doi: 10.1002/1097-0142(19820701)50:1<10::aid-cncr2820500104>3.0.co;2-9.
PTC, a mixture of oligopeptides of m-L-sarcholysin, acting primarily as an alkylating agent, was utilized as initial therapy following diagnosis in 80 children with nonlocalized neuroblastoma. Of the 67 evaluable patients (21 Stage III, 41 Stage IV and five Stage IV-S), 51 had measurable lesions allowing to evaluate PTC activity; objective tumor responses to the drug were recorded in 45 of these 51 cases (88.2%); 5/5 Stage III, 37/41 Stage IV, 3/5 Stage IV-S. Complete responses were obtained in seven patients (13.7%), partial responses in 32 (62.7%), objective improvement in six (11.8%). Four patients (7.8%) had either no tumor change, or tumor progression. There have been two early drug-related deaths (3.9%). Stage III and IV patients responding to PTC were then treated by irradiation + VCR, followed by cycles of a combination of ADriamycin, vincristine, and cyclophosphamide. Stage IV-S patients received no further therapy. Thirteen of 21 Stage III (61.9%), five of 41 Stage IV (12.2%) and four of five Stage IV-S (80%) are presently alive from 19-48 months (median, 27 months). PTC is an effective agent in advanced neuroblastoma. However, the results of this report do not indicate that its addition to a "standard" treatment, at least in the schedule adopted in this protocol, has improved the final outcome of children with nonlocalized disease.
PTC是一种由m-L-沙可洛辛的寡肽混合物组成的药物,主要作为一种烷基化剂,在80例非局限性神经母细胞瘤患儿确诊后被用作初始治疗。在67例可评估的患者中(21例III期、41例IV期和5例IV-S期),51例有可测量的病灶,从而能够评估PTC的活性;在这51例患者中有45例(88.2%)记录到了药物对肿瘤的客观反应;III期5/5例、IV期37/41例、IV-S期3/5例。7例患者(13.7%)获得完全缓解,32例(62.7%)部分缓解,6例(11.8%)有客观改善。4例患者(7.8%)肿瘤无变化或进展。有2例与药物相关的早期死亡(3.9%)。对PTC有反应的III期和IV期患者随后接受放疗 + 长春新碱治疗,之后进行阿霉素、长春新碱和环磷酰胺联合化疗周期。IV-S期患者未接受进一步治疗。21例III期患者中有13例(61.9%)、41例IV期患者中有5例(12.2%)以及5例IV-S期患者中有4例(80%)目前存活,存活时间为19至48个月(中位时间为27个月)。PTC是晚期神经母细胞瘤的一种有效药物。然而,本报告的结果并未表明,至少在本方案所采用的治疗方案中,将其添加到“标准”治疗中能改善非局限性疾病患儿的最终治疗结果。