High-dose chemotherapy followed by stem cell transplant (HDCT) is potentially curative for patients with refractory germ cell tumors (rGCT). There is scarce real-world data supporting its implementation in low- and middle-income countries. We described the experience of our tertiary cancer center in Sao Paulo, Brazil. We identified male patients ≥18 years-old with rGCT referred to HDCT after board discussion. Clinical data, including delays in HDCT protocol, were extracted from medical records, and survival outcomes were estimated using the Kaplan-Meier method. The log-rank test and Cox proportional hazard were used to determine effects on overall survival (OS). From January 2013 to January 2023, 34 patients were referred and considered eligible to receive 2 cycles of HDCT. Most patients had primary testicular tumors (82%), nonseminomatous histology (88%), and poor International Germ Cell Collaborative Group (IGCCCG) (79%). Twenty-three patients received HDCT (1 cycle, n = 8; 2 cycles, n = 15). Main reasons for not receiving any HDCT were death due to progressive disease (n = 1), performance deterioration (n = 7), and failure of stem cell mobilization (n = 3). OS at 2 years was 36.7% for the eligible population, 56.1% for patients who underwent at least 1 HDCT, and 77.1% for those who had ≥2 cycles. The 2-year OS rate for patients not given HDCT was 0%. All patients had delays in protocol, and poor-risk patients had longer intervals from referral to protocol initiation (0.7 vs. 1.8 month, < .01). Outcomes of patients who received ≥1 HDCT were encouraging; however, only 15 from 34 eligible patients were able to receive the planned 2 cycles of HDCT. Further strategies to minimize treatment delays in low- and middle-income countries are needed.

中文翻译：

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高剂量化疗联合自体干细胞移植治疗晚期生殖细胞肿瘤患者：来自巴西三级癌症中心的真实世界证据

高剂量化疗后进行干细胞移植（HDCT）对于难治性生殖细胞肿瘤（rGCT）患者有潜在疗效。很少有现实世界数据支持其在低收入和中等收入国家的实施。我们描述了巴西圣保罗三级癌症中心的经验。我们确定了 18 岁以上患有 rGCT 的男性患者，经董事会讨论后转至 HDCT。从病历中提取临床数据，包括 HDCT 方案的延迟，并使用 Kaplan-Meier 方法估计生存结果。使用对数秩检验和 Cox 比例风险来确定对总生存 (OS) 的影响。从2013年1月到2023年1月，34名患者被转诊并被认为有资格接受2个周期的HDCT。大多数患者患有原发性睾丸肿瘤（82%）、非精原细胞组织学（88%）和国际生殖细胞协作组（IGCCCG）较差（79%）。 23 名患者接受了 HDCT（1 个周期，n = 8；2 个周期，n = 15）。未接受任何 HDCT 的主要原因是疾病进展导致的死亡 (n = 1)、性能恶化 (n = 7) 和干细胞动员失败 (n = 3)。符合条件的人群的 2 年 OS 为 36.7%，接受至少 1 次 HDCT 的患者为 56.1%，接受 ≥ 2 个周期的患者为 77.1%。未接受 HDCT 的患者的 2 年 OS 率为 0%。所有患者均出现方案延迟，低风险患者从转诊到方案启动的时间间隔较长（0.7 个月与 1.8 个月，< .01）。接受≥1次HDCT的患者的结果令人鼓舞；然而，34 名符合条件的患者中只有 15 名能够接受计划的 2 个周期的 HDCT。低收入和中等收入国家需要采取进一步的策略来最大限度地减少治疗延误。