Wilton P, Wallström A
Department of Clinical Research, Kabi Pharmacia Peptide Hormones, Stockholm, Sweden.
Acta Paediatr Scand Suppl. 1991;379:93-8; discussion 99. doi: 10.1111/j.1651-2227.1991.tb12053.x.
This paper provides an overview of the diagnoses of patients entered in the Kabi Pharmacia International Growth Study (KIGS). By May 1991, data from a total of 5377 children treated with growth hormone (GH) were included in the main database. Of these children, 2691 were classified as having idiopathic GH deficiency (GHD), 866 as having GHD of known origin, and 1820 as having other causes of short stature. The majority of patients with idiopathic GHD have no history of perinatal trauma. In the patients with GHD of known origin, 137 were congenital cases and 729 acquired GHD. The largest number of congenital cases (114) belonged to the group of central malformations (e.g. septo-optic dysplasia and empty sella syndrome). Of the cases with acquired GHD, 73% were associated with tumours or leukaemia. Other causes of short stature include 12 groups of diagnoses, with more than 150 cases in four of them (idiopathic short stature, 635; defined syndromes with chromosomal aberrations, 337, of which 304 were Turner's syndrome; defined syndromes without chromosomal aberrations, 157; intrauterine growth retardation without stigmata, 366). Analysis of the KIGS data allows modern GH therapy for GHD to be compared with older treatment modalities. The study offers the advantage of larger numbers of cases than can be achieved in individual trials and allows assessment of the use of GH therapy for GHD of comparatively uncommon causes.
本文概述了卡比 Pharmacia 国际生长研究(KIGS)中纳入的患者诊断情况。截至1991年5月,共有5377名接受生长激素(GH)治疗的儿童数据被纳入主数据库。在这些儿童中,2691名被归类为特发性生长激素缺乏症(GHD),866名有已知病因的GHD,1820名有其他导致身材矮小的原因。大多数特发性GHD患者无围产期创伤史。在有已知病因的GHD患者中,137例为先天性病例,729例为获得性GHD。先天性病例中数量最多的一组(114例)属于中枢性畸形(如视隔发育不良和空蝶鞍综合征)。在获得性GHD病例中,73%与肿瘤或白血病有关。其他导致身材矮小的原因包括12组诊断,其中4组有超过150例(特发性身材矮小,635例;有染色体畸变的明确综合征,337例,其中304例为特纳综合征;无染色体畸变的明确综合征,157例;无体征的宫内生长迟缓,366例)。对KIGS数据的分析可以将现代GHD的生长激素治疗与旧的治疗方式进行比较。该研究的优势在于病例数量比单个试验更多,并且可以评估生长激素治疗在相对罕见病因的GHD中的应用。