The efficacy and safety of IGF-1 therapy in patients with severe primary IGF-I deficiency has been evaluated for more than two decades. Most of the therapeutic experience comes from treating the more severe IGF-I deficient patients, who usually present with a phenotype characteristic of growth hormone receptor deficiency or Laron syndrome. Although most of these patients do not experience enough catchup growth to bring their height into normal range, many individuals achieve an adult height significantly greater than what would have been predicted in the absence of IGF-1 therapy. In the last couple of years a few reports on the benefit of IGF-1 therapy for patients with milder types of IGF-I deficiency have also been published, with variable height outcomes. More short children with prior diagnosis of idiopathic short stature are now being diagnosed with specific molecular defects of the growth hormone/IGF-I axis. Because of this, the clinical spectrum of primary IGF-I deficiency is widening to include many patients with such a milder phenotype, creating a need for well-designed long-term clinical studies evaluating the growth response to growth promoting agents such as rhIGF-1 in these individuals.

已经评估了IGF-1疗法在严重原发性IGF-I缺乏症患者中的疗效和安全性已有超过二十年的历史。大多数治疗经验来自治疗较严重的IGF-I缺乏症的患者，这些患者通常表现出生长激素受体缺乏症或Laron综合征的表型特征。尽管这些患者中的大多数患者没有经历足够的追赶性增长，无法使其身高达到正常范围，但许多人的成年身高明显高于没有IGF-1治疗时的身高。在过去的几年中，关于轻度IGF-I缺乏类型患者的IGF-1治疗的益处的一些报道也已发表，但其身高结果却有所不同。现在正在诊断更多具有先天性矮小身材的矮小儿童，他们患有生长激素/ IGF-I轴的特定分子缺陷。因此，原发性IGF-I缺乏症的临床范围正在扩大，以包括许多具有这种较轻表型的患者，因此需要进行精心设计的长期临床研究，以评估对生长促进剂（如rhIGF-1）的生长反应在这些人中。