OBJECTIVE The therapeutic options for metastatic pheochromocytomas/paragangliomas (mPPGLs) include chemotherapy with cyclophosphamide/vincristine/dacarbazine (CVD), temozolomide monotherapy, radionuclide therapies, and tyrosine kinase inhibitors such as sunitinib. The objective of this multicenter retrospective study was to evaluate and compare the responses of mPPGLs including those with pathogenic variants in succinate dehydrogenase subunit B (SDHB), to different systemic treatments. DESIGN This is a retrospective analysis of treatment responses of mPPGL patients (n = 74) to systemic therapies. METHODS Patients with mPPGLs treated at 6 specialized national centers were selected based on participation in the ENSAT registry. Survival until detected progression (SDP) and disease-control rates (DCRs) at 3 months were evaluated based on imaging reports. RESULTS For the group of patients with progressive disease at baseline (83.8% of 74 patients), the DCR with first-line CVD chemotherapy was 75.0% (n = 4, SDP 11 months; SDHB [n = 1]: DCR 100%, SDP 30 months), with somatostatin peptide receptor-based radionuclide therapy (PPRT) 85.7% (n = 21, SDP 17 months; SDHB [n = 10]: DCR 100%, SDP 14 months), with 131I-meta-iodobenzylguanidine (131I-MIBG) 82.6% (n = 23, SDP 43 months; SDHB [n = 4]: DCR 100%, SDP 24 months), with sunitinib 100% (n = 7, SDP 18 months; SDHB [n = 3]: DCR 100%, SDP 18 months), and with somatostatin analogs 100% (n = 4, SDP not reached). The DCR with temozolomide as second-line therapy was 60.0% (n = 5, SDP 10 months; SDHB [n = 4]: DCR 75%, SDP 10 months). CONCLUSIONS We demonstrate in a real-life clinical setting that all current therapies show reasonable efficacy in preventing disease progression, and this is equally true for patients with germline SDHB mutations.

中文翻译：

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转移性嗜铬细胞瘤/副神经节瘤对全身治疗的反应：一项回顾性多中心队列研究。

目的 转移性嗜铬细胞瘤/副神经节瘤 (mPPGL) 的治疗选择包括环磷酰胺/长春新碱/达卡巴嗪 (CVD) 化疗、替莫唑胺单一疗法、放射性核素疗法和酪氨酸激酶抑制剂（如舒尼替尼）。这项多中心回顾性研究的目的是评估和比较 mPPGL（包括琥珀酸脱氢酶 B 亚基 (SDHB) 致病性变异的 mPPGL）对不同全身治疗的反应。设计 这是对 mPPGL 患者 (n = 74) 对全身疗法的治疗反应的回顾性分析。方法 根据 ENSAT 登记的参与情况，选择在 6 个国家专门中心接受治疗的 mPPGL 患者。根据影像报告评估 3 个月时检测到进展之前的生存率 (SDP) 和疾病控制率 (DCR)。结果 对于基线时疾病进展的患者组（74 名患者中的 83.8%），一线 CVD 化疗的 DCR 为 75.0%（n = 4，SDP 11 个月；SDHB [n = 1]：DCR 100%， SDP 30 个月），采用基于生长抑素肽受体的放射性核素治疗 (PPRT) 85.7%（n = 21，SDP 17 个月；SDHB [n = 10]：DCR 100%，SDP 14 个月），采用 131I-间碘苄基胍（ 131I-MIBG) 82.6%（n = 23，SDP 43 个月；SDHB [n = 4]：DCR 100%，SDP 24 个月），联合舒尼替尼 100%（n = 7，SDP 18 个月；SDHB [n = 3] ：DCR 100%，SDP 18 个月），生长抑素类似物 100%（n = 4，SDP 未达到）。替莫唑胺作为二线治疗的 DCR 为 60.0%（n = 5，SDP 10 个月；SDHB [n = 4]：DCR 75%，SDP 10 个月）。结论 我们在现实临床环境中证明，当前所有疗法在预防疾病进展方面均显示出合理的功效，对于具有种系 SDHB 突变的患者同样如此。