Cinacalcet effectively treats infants and young children with refractory hyperparathyroidism (SHPT) from advanced chronic kidney disease (CKD), researchers reported in Pediatric Nephrology.

Carolyn L. Abitbol, MD, of Holtz Children’s Hospital and her team studied the medical records of 10 pediatric patients aged 6 months to 7½ years with intact parathyroid hormone (iPTH) levels at or above 500 pg/mL, despite treatment with active vitamin D and phosphate binders. Cinacalcet, a calcimimetic, was administered at a mean starting dose of 0.7 mg/kg/day and increased by 50% every 2 to 4 weeks as needed until iPTH decreased to 150 to 300 pg/mL. At the start of cinacalcet therapy, the patients had a median age of 18 months.

Results showed that adjuvant cinacalcet therapy decreased iPTH by a median 82% over 6 months to goal, while also supporting children’s growth. The median effective dose was 2.8 mg/kg/day with efficacy observed by 112 days. Children’s mean height increased significantly from -0.62 standard deviations before therapy to 0.91 afterward. According to multiple regression analysis, the most significant factors for growth were concurrent treatment with growth hormone and being younger than 2 years. Quicker response to cinacalcet treatment also was associated with better growth. 

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Most patients experienced transient oversuppression of iPTH during treatment. Four patients also had biochemical asymptomatic hypocalcemia. 

“Cinacalcet may be used effectively and safely in infants and small children with refractory sHPT in advanced CKD using a cautious dosing regimen,” Dr Abitbol and colleagues concluded. “Cinacalcet successfully brings iPTH to target level and supports growth when other treatments have been ineffective.” 

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Arenas Morales AJ, DeFreitas MJ, Katsoufis CP, et al. Cinacalcet as rescue therapy for refractory hyperparathyroidismin young children with advanced chronic kidney disease. Ped Nephrol. DOI:10.1007/s00467-018-4055-7