Bernardor Julie, Flammier Sacha, Cabet Sara, Lemoine Sandrine, Chapurlat Roland, Molin Arnaud, Bertholet-Thomas Aurélia, Bacchetta Justine
Centre de Référence des Maladies Rares du Calcium et du Phosphore, Centre de Référence des Maladies Rénales Rares, Filières de Santé Maladies Rares OSCAR, ORKID et ERKNet, Service de Néphrologie Rhumatologie et Dermatologie Pédiatriques, Hôpital Femme Mère Enfant, Bron, France.
INSERM UMR S1033 Research Unit, Lyon, France.
Front Pediatr. 2021 Nov 8;9:764040. doi: 10.3389/fped.2021.764040. eCollection 2021.
The use of teriparatide has been reported in children with hypoparathyroidism as an investigational physiologic replacement therapy. We aimed to retrospectively report our pediatric experience of bi-daily sub-cutaneous teriparatide. Results are presented as median (25th-75th quartile). As part of the routine follow-up of these patients with hypoparathyroidism, total calcium at H0 (i.e., just before injection) and H4 (i.e., 4 h after teriparatide injection) and other biomarker parameters were regularly assessed. At a median age of 10.7 (8.1-12.6) years, an estimated glomerular filtration rate (eGFR) of 110 (95-118) mL/min/1.73 m, calcium levels of 1.87 (1.81-1.96) mmol/L and an age-standardized phosphate of 3.8 (2.5-4.9) SDS, teriparatide therapy was introduced in 10 patients at the dose of 1.1 (0.7-1.5) μg/kg/day (20 μg twice daily), with further adjustment depending on calcium levels. Six patients already displayed nephrocalcinosis. Severe side effects were reported in one child: two episodes of symptomatic hypocalcemia and one of iatrogenic hypercalcemia; one teenager displayed dysgueusia. Calcium levels at H0 did not significantly increase whilst calcium at H4 and phosphate levels significantly increased and decreased, respectively. After 12 months, eGFR, calcium and age-standardized phosphate levels were 108 (90-122) mL/min/1.73 m, 2.36 (2.23-2.48) mmol/L, 0.5 (-0.1 to 1.5), and 68 (63-74) nmol/L, respectively, with a significant decrease in phosphate levels ( = 0.01). Urinary calcium and calcium/creatinine ratio remained stable; no nephrolithiasis was observed but two moderate nephrocalcinosis appeared. Intermittent teriparatide therapy significantly improves calcium and phosphate control, without increasing calciuria. It appears to be safe and well-tolerated in children.
已有报道在甲状旁腺功能减退症患儿中使用特立帕肽作为一种试验性生理替代疗法。我们旨在回顾性报告我们在儿童中每日两次皮下注射特立帕肽的经验。结果以中位数(第25-75四分位数)表示。作为这些甲状旁腺功能减退症患者常规随访的一部分,定期评估H0(即注射前)和H4(即特立帕肽注射后4小时)时的总钙及其他生物标志物参数。在中位年龄为10.7(8.1-12.6)岁、估计肾小球滤过率(eGFR)为110(95-118)mL/min/1.73 m²、钙水平为1.87(1.81-1.96)mmol/L以及年龄标准化磷酸盐为3.8(2.5-4.9)SDS的情况下,10名患者开始接受特立帕肽治疗,剂量为1.1(0.7-1.5)μg/kg/天(每日两次,每次20μg),并根据钙水平进一步调整。6名患者已出现肾钙质沉着症。1名儿童报告有严重副作用:2次症状性低钙血症发作和1次医源性高钙血症发作;1名青少年出现味觉障碍。H0时的钙水平没有显著升高,而H4时的钙水平显著升高,磷酸盐水平则显著下降。12个月后,eGFR、钙和年龄标准化磷酸盐水平分别为108(90-122)mL/min/1.73 m²、2.36(2.23-2.48)mmol/L、0.5(-0.1至1.5)SDS和68(63-74)nmol/L,磷酸盐水平显著下降(P = 0.01)。尿钙和钙/肌酐比值保持稳定;未观察到肾结石,但出现了2例中度肾钙质沉着症。间歇性特立帕肽治疗可显著改善钙和磷酸盐的控制,且不增加尿钙排泄。在儿童中似乎是安全且耐受性良好的。