Giusti Francesca, Cianferotti Luisella, Gronchi Giorgio, Cioppi Federica, Masi Laura, Faggiano Antongiulio, Colao Annamaria, Ferolla Piero, Brandi Maria Luisa
Department of Surgery and Translational Medicine, University of Florence, Largo Palagi 1, 50139, Florence, Italy.
Department of Clinical Medicine and Surgery, Federico II University, Naples, Italy.
Endocrine. 2016 Jun;52(3):495-506. doi: 10.1007/s12020-015-0696-5. Epub 2015 Jul 30.
Primary hyperparathyroidism is the main endocrinopathy associated with Multiple Endocrine Neoplasia type 1 syndrome. Cinacalcet is a calcimimetic agent licensed for the treatment of secondary hyperparathyroidism in patients with end-stage renal disease, and for the reduction of marked hypercalcemia in patients with parathyroid carcinoma and sporadic hyperparathyroidism requiring surgery but for whom parathyroidectomy is contraindicated. It may provide a medical alternative for the management of primary hyperparathyroidism in subjects affected by Multiple Endocrine Neoplasia type 1. In this longitudinal, intervention study, 33 MEN1 patients had been enrolled, 10 males and 23 females with a mean age of 40 ± 11.9 years, range 20-63. Primary hyperparathyroidism was the first clinical manifestation in 12 patients. All subjects commenced with Cinacalcet 30 mg/day, 22 patients starting therapy with calcimimetics as an alternative to surgery, and 11 patients opting for the medication after the onset of persistent post-surgical primary hyperparathyroidism. Duration of follow-up was 12 months. The results of this study show significant reductions in serum calcium. The changes in hormonal secretions of pituitary and gastroenteropancreatic glands were not significant, demonstrating the overall safety of this drug in this disease. Cinacalcet has been well tolerated by 28 patients, whereas five individuals complained of heartburn and grade 1 nausea, which did not prevent the completion of the study. In conclusion, Cinacalcet has resulted to be well tolerated and safe in patients with MEN1 syndrome and the calcium homeostasis was stabilized.
原发性甲状旁腺功能亢进是与1型多发性内分泌腺瘤综合征相关的主要内分泌疾病。西那卡塞是一种拟钙剂，被批准用于治疗终末期肾病患者的继发性甲状旁腺功能亢进，以及降低甲状旁腺癌和散发性甲状旁腺功能亢进且需要手术但甲状旁腺切除术禁忌的患者的明显高钙血症。它可能为1型多发性内分泌腺瘤患者原发性甲状旁腺功能亢进的管理提供一种药物替代方案。在这项纵向干预研究中，招募了33例1型多发性内分泌腺瘤患者，其中10例男性和23例女性，平均年龄为40±11.9岁，年龄范围为20 - 63岁。12例患者原发性甲状旁腺功能亢进是首发临床表现。所有受试者开始服用西那卡塞30毫克/天，22例患者开始使用拟钙剂治疗作为手术的替代方案，11例患者在持续性术后原发性甲状旁腺功能亢进发作后选择该药物。随访时间为12个月。这项研究的结果显示血清钙显著降低。垂体和胃肠胰腺腺体激素分泌的变化不显著，证明了该药物在这种疾病中的总体安全性。28例患者对西那卡塞耐受性良好，而5例患者抱怨有烧心和1级恶心，但这并未妨碍研究的完成。总之，西那卡塞在1型多发性内分泌腺瘤综合征患者中耐受性良好且安全，钙稳态得以稳定。
