Abstract：
BACKGROUND: Patients with relapsed primary mediastinal nonseminomatous germ cell tumor have low cure rates with salvage chemotherapy or surgery. The authors report survival outcomes of patients who received high-dose chemotherapy (HDCT) and peripheral blood stem cell transplantation (PBSCT) at Indiana University.
METHODS: The prospectively maintained Indiana University germ cell tumor database identified 32 patients with primary mediastinal nonseminomatous germ cell tumor who progressed after first-line cisplatin-based combination chemotherapy and received HDCT and PBSCT between 2006 and 2021. Therapy included two consecutive courses of HDCT consisting of 700 mg/m2 carboplatin and 750 mg/m2 etoposide, each for 3 consecutive days, and each followed by PBSCT. A second course was not given if the patient experienced progressive disease or prohibitive toxicity. Progression-free survival and overall survival were analyzed using the Kaplan-Meier method. Medians with 95% confidence intervals were also calculated along with 2-year probabilities.
RESULTS: The median age at HDCT was 30 years (range, 18-61 years). With a median follow-up of 4.7 years (range, 1-14 years), the 2-year progression-free survival rate was 31% (95% confidence interval, 16%-47%), and the 2-year overall survival rate was 35% (95% confidence interval, 19%-52%). At last follow-up, nine patients (28%) remained without evidence of disease, including two platinum-refractory patients and two patients who were receiving HDCT as third-line therapy. There were three treatment-related deaths.
CONCLUSIONS: Salvage HDCT and PBSCT is an active combination in patients who have relapsed primary mediastinal nonseminomatous germ cell tumor with curative potential and prolonged survival, including in platinum-refractory and third-line settings. The authors recommend this approach for initial salvage chemotherapy in this patient population.
METHODS: The prospectively maintained Indiana University germ cell tumor database identified 32 patients with primary mediastinal nonseminomatous germ cell tumor who progressed after first-line cisplatin-based combination chemotherapy and received HDCT and PBSCT between 2006 and 2021. Therapy included two consecutive courses of HDCT consisting of 700 mg/m2 carboplatin and 750 mg/m2 etoposide, each for 3 consecutive days, and each followed by PBSCT. A second course was not given if the patient experienced progressive disease or prohibitive toxicity. Progression-free survival and overall survival were analyzed using the Kaplan-Meier method. Medians with 95% confidence intervals were also calculated along with 2-year probabilities.
RESULTS: The median age at HDCT was 30 years (range, 18-61 years). With a median follow-up of 4.7 years (range, 1-14 years), the 2-year progression-free survival rate was 31% (95% confidence interval, 16%-47%), and the 2-year overall survival rate was 35% (95% confidence interval, 19%-52%). At last follow-up, nine patients (28%) remained without evidence of disease, including two platinum-refractory patients and two patients who were receiving HDCT as third-line therapy. There were three treatment-related deaths.
CONCLUSIONS: Salvage HDCT and PBSCT is an active combination in patients who have relapsed primary mediastinal nonseminomatous germ cell tumor with curative potential and prolonged survival, including in platinum-refractory and third-line settings. The authors recommend this approach for initial salvage chemotherapy in this patient population.
摘要：
背景：复发的原发性纵隔非精原细胞生殖细胞肿瘤患者在挽救性化疗或手术治疗中治愈率较低。作者报告了在印第安纳大学接受大剂量化疗（HDCT）和外周血干细胞移植（PBSCT）的患者的生存结果。
方法：前瞻性维护的印第安纳大学生殖细胞肿瘤数据库确定了32例原发性纵隔非精原细胞生殖细胞肿瘤患者，这些患者在以顺铂为基础的一线联合化疗后进展，并在2006年至2021年间接受了HDCT和PBSCT。治疗包括连续两个疗程的HDCT，包括700mg/m2卡铂和750mg/m2依托泊苷，每个连续3天，然后是PBSCT。如果患者出现进行性疾病或禁止性毒性，则不给予第二疗程。使用Kaplan-Meier方法分析无进展生存期和总生存期。还计算了95%确认间隔的中位数和2年概率。
结果：HDCT的中位年龄为30岁（范围，18-61岁）。中位随访时间为4.7年(范围，1-14年),2年无进展生存率为31%(95%置信区间,16%-47%），2年总生存率为35%(95%置信区间,19%-52%）。在最后的随访中，9名患者（28％）仍然没有疾病的证据，包括2例铂类难治性患者和2例接受HDCT三线治疗的患者.有3例与治疗相关的死亡。
结论：挽救性HDCT和PBSCT是复发性原发性纵隔非精原细胞生殖细胞肿瘤患者的积极组合，具有治愈潜力和延长生存期，包括铂金耐火材料和第三线设置。作者推荐这种方法用于该患者人群的初始挽救化疗。
方法：前瞻性维护的印第安纳大学生殖细胞肿瘤数据库确定了32例原发性纵隔非精原细胞生殖细胞肿瘤患者，这些患者在以顺铂为基础的一线联合化疗后进展，并在2006年至2021年间接受了HDCT和PBSCT。治疗包括连续两个疗程的HDCT，包括700mg/m2卡铂和750mg/m2依托泊苷，每个连续3天，然后是PBSCT。如果患者出现进行性疾病或禁止性毒性，则不给予第二疗程。使用Kaplan-Meier方法分析无进展生存期和总生存期。还计算了95%确认间隔的中位数和2年概率。
结果：HDCT的中位年龄为30岁（范围，18-61岁）。中位随访时间为4.7年(范围，1-14年),2年无进展生存率为31%(95%置信区间,16%-47%），2年总生存率为35%(95%置信区间,19%-52%）。在最后的随访中，9名患者（28％）仍然没有疾病的证据，包括2例铂类难治性患者和2例接受HDCT三线治疗的患者.有3例与治疗相关的死亡。
结论：挽救性HDCT和PBSCT是复发性原发性纵隔非精原细胞生殖细胞肿瘤患者的积极组合，具有治愈潜力和延长生存期，包括铂金耐火材料和第三线设置。作者推荐这种方法用于该患者人群的初始挽救化疗。
