Ferrara Alfonso M, Lombardi Giuseppe, Pambuku Ardi, Meringolo Domenico, Bertorelle Roberta, Nardin Margherita, Schiavi Francesca, Iacobone Maurizio, Opocher Giuseppe, Zagonel Vittorina, Zovato Stefania
Departments of Clinical and Experimental Oncology Familial Cancer Clinic and Oncoendocrinology.
Medical Oncology 1.
Anticancer Drugs. 2018 Jan;29(1):102-105. doi: 10.1097/CAD.0000000000000570.
Pheochromocytomas (PCCs) and paragangliomas (PGLs) are neuroendocrine tumors with a strong genetic background. The mainstay of treatment for PCC/PGLs is surgery. However, for unresectable lesions, no curative treatment is currently available. Temozolomide (TMZ) has been shown to determine radiological and biochemical response in malignant PCC/PGLs. We report two cases of PCC/PGLs treated with TMZ. Case 1 is a 51-year-old man with local and distant recurrence (liver and bone metastases) of right adrenal PCC. Case 2 is a 54-year-old woman with a PCC/PGL syndrome caused by a mutation in MAX gene (c.171+1G>A), operated on for bilateral adrenal PCC and presenting with a large unresectable abdominal PGL. Both patients presented hypertension due to catecholamine hypersecretion. TMZ determined radiological response according to RECIST criteria, reduction of urinary catecholamine levels, and controlled hypertension in both patients. Furthermore, the current study demonstrates, for the first time, that MAX-related PGLs are responsive to TMZ.
嗜铬细胞瘤（PCCs）和副神经节瘤（PGLs）是具有强大遗传背景的神经内分泌肿瘤。PCC/PGLs的主要治疗方法是手术。然而，对于不可切除的病变，目前尚无治愈性治疗方法。替莫唑胺（TMZ）已被证明可在恶性PCC/PGLs中产生放射学和生化反应。我们报告了两例接受TMZ治疗的PCC/PGLs病例。病例1是一名51岁男性，患有右肾上腺PCC的局部和远处复发（肝和骨转移）。病例2是一名54岁女性，因MAX基因（c.171+1G>A）突变导致PCC/PGL综合征，接受了双侧肾上腺PCC手术，出现一个不可切除的巨大腹部PGL。两名患者均因儿茶酚胺分泌过多而出现高血压。TMZ根据RECIST标准产生了放射学反应，降低了尿儿茶酚胺水平，并控制了两名患者的高血压。此外，本研究首次证明，与MAX相关的PGLs对TMZ有反应。
