Howell S J, Wilton P, Lindberg A, Shalet S M
Department of Endocrinology, Christie Hospital NHS Trust, Withington, Manchester, United Kingdom.
J Pediatr. 1998 Aug;133(2):201-5. doi: 10.1016/s0022-3476(98)70245-8.
To assess the efficacy and safety of growth hormone (GH) therapy in children with GH deficiency in association with neurofibromatosis.
Retrospective analysis of data from the Pharmacia and Upjohn International Growth Database (KIGS) in a total of 102 GH-deficient children with neurofibromatosis treated with recombinant GH.
Median pretreatment height velocity was 4.2 cm/yr (1.7 to 6.4 cm/yr), increased to 7.1 cm/yr (4.6 to 10.0 cm/yr) in the first year of GH therapy, and remained significantly greater than pretreatment at 5.7 cm/yr (2.9 to 8.3 cm/yr) and 5.7 cm/yr (2.6 to 7.9 cm/yr) in the second and third years, respectively. The median height SD score increased from -2.4 to -1.8 by the end of 3 years of treatment. Five patients had either a recurrence of an intracranial tumor or a second intracranial tumor; this incidence of tumor occurrence is comparable to that reported previously in similar patients with neurofibromatosis. Other adverse events were relatively minor and unlikely to be attributable to GH therapy
The data indicate that GH replacement therapy, per se, for patients with neurofibromatosis and GH deficiency is likely to be beneficial and unassociated with excessive malignant risk.
评估生长激素（GH）治疗神经纤维瘤病合并生长激素缺乏症儿童的疗效和安全性。
对辉瑞国际生长数据库（KIGS）中102例接受重组生长激素治疗的神经纤维瘤病合并生长激素缺乏症儿童的数据进行回顾性分析。
治疗前身高增长速度中位数为4.2厘米/年（1.7至6.4厘米/年），生长激素治疗第一年增至7.1厘米/年（4.6至10.0厘米/年），在第二年和第三年分别仍显著高于治疗前，为5.7厘米/年（2.9至8.3厘米/年）和5.7厘米/年（2.6至7.9厘米/年）。治疗3年末，身高标准差评分中位数从-2.4增至-1.8。5例患者出现颅内肿瘤复发或发生第二个颅内肿瘤；这种肿瘤发生率与先前报道的类似神经纤维瘤病患者相当。其他不良事件相对较轻，不太可能归因于生长激素治疗。
数据表明，对于神经纤维瘤病合并生长激素缺乏症患者，生长激素替代治疗本身可能有益，且与过度恶性风险无关。
