Winer Karen K, Fulton Kara A, Albert Paul S, Cutler Gordon B
Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD.
Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD.
J Pediatr. 2014 Sep;165(3):556-63.e1. doi: 10.1016/j.jpeds.2014.04.060. Epub 2014 Jun 16.
To compare the response with synthetic human parathyroid hormone (PTH) 1-34 delivered by twice-daily injection vs insulin pump in children with severe congenital hypoparathyroidism due to calcium receptor mutation or autoimmune polyglandular syndrome type 1.
Children and young adults aged 7-20 years with congenital hypoparathyroidism (N = 12) were randomized to receive PTH 1-34, delivered either by twice-daily subcutaneous injection or insulin pump for 13 weeks, followed by crossover to the opposite delivery method. The principal outcome measures were serum and urine calcium levels. Secondary outcomes included serum and urine magnesium and phosphate levels and bone turnover markers.
PTH 1-34 delivered via pump produced near normalization of mean serum calcium (2.02 ± 0.05 [pump] vs 1.88 ± 0.03 [injection] mmol/L, P < .05, normal 2.05-2.5 mmol/L), normalized mean urine calcium excretion (5.17 ± 1.10 [pump] vs 6.67 ± 0.76 mmol/24 h/1.73 m(2), P = .3), and significantly reduced markers of bone turnover (P < .02). Serum and urine calcium and magnesium showed a biphasic pattern during twice-daily injection vs minimal fluctuation during pump delivery. The PTH 1-34 dosage was markedly reduced during pump delivery (0.32 ± 0.04 vs 0.85 ± 0.11 μg/kg/d, P < .001), and magnesium supplements were also reduced (P < .001).
Compared with twice-daily delivery, pump delivery of PTH 1-34 provides more physiologic calcium homeostasis and bone turnover in children with severe congenital hypoparathyroidism.
比较每日两次注射合成人甲状旁腺激素（PTH）1-34与胰岛素泵给药对因钙受体突变或1型自身免疫性多内分泌腺综合征导致的严重先天性甲状旁腺功能减退症患儿的疗效。
将12名7-20岁的先天性甲状旁腺功能减退症患儿随机分为两组，分别接受每日两次皮下注射PTH 1-34或胰岛素泵给药，为期13周，之后交叉采用另一种给药方式。主要观察指标为血清和尿钙水平。次要观察指标包括血清和尿镁、磷水平以及骨转换标志物。
通过胰岛素泵给药的PTH 1-34使平均血清钙接近正常水平（泵给药组为2.02±0.05[泵]与注射组为1.88±0.03 mmol/L，P<0.05，正常范围为2.05-2.5 mmol/L），使平均尿钙排泄正常化（泵给药组为5.17±1.10[泵]与6.67±0.76 mmol/24 h/1.73 m²，P = 0.3），并显著降低了骨转换标志物（P<0.02）。与胰岛素泵给药期间波动极小相比，每日两次注射期间血清和尿钙及镁呈现双相模式。胰岛素泵给药期间PTH 1-34剂量显著降低（0.32±0.04与0.85±0.11 μg/kg/d，P<0.001），镁补充剂用量也减少（P<0.001）。
与每日两次给药相比，胰岛素泵给药的PTH 1-34能为严重先天性甲状旁腺功能减退症患儿提供更生理性的钙稳态和骨转换。
