Cinacalcet is a calcimimetic approved in adults with
primary hyperparathyroidism (PHPT). Few cases reports described its use in pediatric HPT, with challenges related to the risk of
hypocalcemia, increased QT interval and drug interactions. In this study, we report the French experience in this setting.
Methods: We retrospectively analyzed data from 18 pediatric patients from 7 tertiary centers who received
cinacalcet for PHPT. The results are presented as median (interquartile range).
Results: At a median age of 10.8 (2.0-14.4) years, 18 patients received
cinacalcet for primary HPT (N = 13 inactive CASR mutation, N = 1 CDC73 mutation, N = 1
multiple endocrine neoplasia type 1, N=3 unknown etiology).
Cinacalcet was introduced at an estimated glomerular filtration rate (eGFR) of 120 (111-130) mL/min/1.73 m2, plasma
calcium of 3.04 (2.96-3.14) mmol/L, plasma
phosphate of 1.1 (1.0-1.3) mmol/L, age-standardized (z score)
phosphate of -3.0 (-3.5;-1.9), total ALP of 212 (164-245) UI/L, 25-OHD of 37 (20-46) ng/L, age-standardized (z score) ALP of -2.4 (-3.7;-1.4), PTH of 75 (59-123) ng/L corresponding to 1.2 (1.0-2.3)-time the upper limit for normal (ULN). The starting daily dose of
cinacalcet was 0.7 (0.6-1.0) mg/kg, with a maximum dose of 1.0 (0.9-1.4) mg/kg per day. With a follow-up of 2.2 (1.3-4.3) years on
cinacalcet therapy, PTH and
calcium significantly decreased to 37 (34-54) ng/L, corresponding to 0.8 (0.5-0.8) ULN (p = 0.01), and 2.66 (2.55-2.90) mmol/L (p = 0.002), respectively. In contrast, eGFR, 25-OHD, ALP and
phosphate and urinary
calcium levels remained stable.
Nephrocalcinosis was not reported but one patient displayed
nephrolithiasis.
Cinacalcet was progressively withdrawn in three patients; no side effects were reported.
Conclusions: