Barnabas Rohit, Jaiswal Sanjeet Kumar, Memon Saba Samad, Sarathi Vijaya, Malhotra Gaurav, Verma Priyanka, Patil Virendra A, Lila Anurag R, Shah Nalini S, Bandgar Tushar R
Department of Endocrinology, Seth G.S Medical College and KEM Hospital, Mumbai, Maharashtra, India.
Department of Endocrinology, Vydehi Institute of Medical Sciences and Research Center, Bengaluru, Karnataka, India.
Indian J Endocrinol Metab. 2021 Mar-Apr;25(2):148-159. doi: 10.4103/ijem.IJEM_52_21. Epub 2021 Sep 8.
Radionuclide therapy is a promising treatment modality in metastatic pheochromocytoma/paraganglioma (PPGL). There is scarce data on I-metaiodobenzyl guanidine (I-MIBG) therapy from the Indian subcontinent. Hence, we aim to study the safety and effectiveness of low-dose, low-specific activity (LSA) I-MIBG therapy in patients with symptomatic, metastatic PPGL.
Clinical, hormonal, and radiological response parameters and side effects of LSA I-MIBG therapy in patients with symptomatic, metastatic PPGL were retrospectively reviewed. World health organizations' (WHO) symptomatic, hormonal, and tumor response, and response evaluation criteria in solid tumors (RECIST1.1) criteria were used to assess the response.
Seventeen (PCC: 11, sympathetic PGL: 06) patients (15 with disease progression) received low-dose LSA I-MIBG therapy. Complete remission (CR), partial remission (PR), stable disease (SD), and progressive disease (PD) were 18% (3/17), 24% (4/17), 18% (3/17), and 41% (7/17), respectively, for WHO symptomatic response; 20% (2/10), 10% (1/10), 30% (3/10), and 40% (4/10), respectively, for WHO hormonal response; and 19% (3/16), 6% (1/16), 31% (5/16), and 44% (7/16), respectively for tumor response based on RECIST1.1. All patients with symptomatic PD and 50% (2/4) with hormonal PD had progression as per RECIST1.1 criteria. Side effects included thrombocytopenia, acute myeloid leukemia, mucoepidermoid carcinoma, and azoospermia in 6% (1/17) each.
Our study reaffirms the modest efficacy and safety of low-dose, LSA I-MIBG therapy in patients with symptomatic, metastatic PPGL. Symptomatic, but not hormonal, progression after I-MIBG therapy correlates well with tumor progression and should be further evaluated with imaging. In resource-limited settings, anatomic imaging alone may be used to assess tumor response to I-MIBG therapy.
放射性核素治疗是转移性嗜铬细胞瘤/副神经节瘤（PPGL）一种很有前景的治疗方式。来自印度次大陆的关于碘-间位碘代苄胍（I-MIBG）治疗的数据很少。因此，我们旨在研究低剂量、低比活度（LSA）I-MIBG治疗有症状的转移性PPGL患者的安全性和有效性。
回顾性分析有症状的转移性PPGL患者接受LSA I-MIBG治疗的临床、激素和影像学反应参数以及副作用。采用世界卫生组织（WHO）的症状、激素和肿瘤反应标准以及实体瘤疗效评价标准（RECIST1.1）来评估反应。
17例患者（嗜铬细胞瘤11例，交感神经副神经节瘤6例）（15例病情进展）接受了低剂量LSA I-MIBG治疗。WHO症状反应的完全缓解（CR）、部分缓解（PR）、病情稳定（SD）和疾病进展（PD）分别为18%（3/17）、24%（4/17）、18%（3/17）和41%（7/17）；WHO激素反应分别为20%（2/10）、10%（1/10）、30%（3/10）和40%（4/10）；基于RECIST1.1的肿瘤反应分别为19%（3/16）、6%（1/16）、31%（5/16）和44%（7/16）。所有有症状PD的患者以及50%（2/4）有激素PD的患者根据RECIST1.1标准均有病情进展。副作用包括血小板减少、急性髓系白血病、黏液表皮样癌和无精子症，各占6%（1/17）。
我们的研究再次证实低剂量、LSA I-MIBG治疗有症状的转移性PPGL患者的疗效和安全性一般。I-MIBG治疗后有症状但非激素性的病情进展与肿瘤进展密切相关，应通过影像学进一步评估。在资源有限的情况下，仅解剖学成像可用于评估肿瘤对I-MIBG治疗的反应。
