Villafuerte Beatriz, Barrio Raquel, Martín-Frías María, Alonso Milagros, Roldán Belén
Unidad de Endocrinología y Diabetes Pediátrica, Servicio de Pediatría, Hospital Universitario Ramón y Cajal, Universidad de Alcalá, Madrid, España.
Unidad de Endocrinología y Diabetes Pediátrica, Servicio de Pediatría, Hospital Universitario Ramón y Cajal, Universidad de Alcalá, Madrid, España.
Endocrinol Diabetes Nutr (Engl Ed). 2019 Jun-Jul;66(6):368-375. doi: 10.1016/j.endinu.2018.11.007. Epub 2019 Feb 14.
Treatment with recombinant human growth hormone (rhGH) has been shown to improve adult height in pediatric patients with GH deficiency (GHD). However, reassessment of patients after they reach their final height shows some of them have permanent GH deficiency (PGHD), while others had a transient deficiency (TGHD). The study objective was to assess, in a cohort of pediatric patients with GHD, potential differences in response to treatment with rhGH depending on whether deficiency is permanent or transient.
A retrospective study of 89 patients with GHD, who were monitored from diagnosis to adult height. Clinical, auxological, radiographic and laboratory variables were collected at diagnosis, after the first year of treatment, and when they had reached their adult height.
PGHD was found in 28% of patients. Initial height was -2.46 ± 0.86 SD and -2.24 ± 0.68 SD in subjects with PGHD and TGHD respectively. Peak GH level at diagnosis was 4.26 ± 2.78 and 6.20 ± 2.01 ng/mL (p < 0.01) in the PGHD and TGHD groups respectively. After the first year of treatment, increase in growth velocity was greater in the PGHD group: 4.33 ± 3.53 SD vs. 2.95 ± 2.54 SD in the PGHD group (p = 0.043). Final height was -0.81 ± 0.87 SD in the PGHD and -0.95 ± 0.83 SD in the TGHD group (p = 0.47).
Patients with PGHD had a better short- and long-term response to rhGH. They also showed lower GH levels in stimulation tests at diagnosis, as previously reported.
重组人生长激素（rhGH）治疗已被证明可提高生长激素缺乏症（GHD）儿科患者的成人身高。然而，患者达到最终身高后的重新评估显示，其中一些人存在永久性生长激素缺乏（PGHD），而另一些人则存在暂时性缺乏（TGHD）。本研究的目的是评估一组GHD儿科患者中，根据缺乏是永久性还是暂时性，对rhGH治疗反应的潜在差异。
对89例GHD患者进行回顾性研究，从诊断到成人身高进行监测。在诊断时、治疗第一年结束时以及达到成人身高时收集临床、体格测量、影像学和实验室变量。
28%的患者被发现存在PGHD。PGHD和TGHD患者的初始身高分别为-2.46±0.86标准差和-2.24±0.68标准差。PGHD组和TGHD组诊断时的生长激素峰值水平分别为4.26±2.78和6.20±2.01 ng/mL（p<0.01）。治疗第一年结束后，PGHD组的生长速度增加更大：PGHD组为4.33±3.53标准差，而TGHD组为2.95±2.54标准差（p=0.043）。PGHD组的最终身高为-0.81±0.87标准差，TGHD组为-0.95±0.83标准差（p=0.47）。
PGHD患者对rhGH的短期和长期反应更好。如先前报道的那样，他们在诊断时的刺激试验中生长激素水平也较低。
