Ben-Ari Tal, Chodick Gabriel, Shalev Varda, Goldstein Dalit, Gomez Roy, Landau Zohar
MaccabiTech, Maccabi Healthcare Services, Tel Aviv, Israel.
Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
Front Pediatr. 2021 Aug 20;9:711979. doi: 10.3389/fped.2021.711979. eCollection 2021.
To assess a decade of growth hormone (GH) treatment patterns and outcomes in a real-world setting in Israel using a state-of-the-art computerized database. This large retrospective database study included 2,379 children initiating GH treatment in Maccabi Healthcare Services (between January 2004 and December 2014). Good adherence with therapy (proportion of days covered >80%) was assessed during follow-up. At GH treatment initiation: 62.1% were boys; height standard deviation score (SDS) was -2.36 ± 0.65 (mean ± SD); age was 9.8 ± 3.1 years; and time from short stature diagnosis to first GH purchase was 4.8 ± 3.3 years. Mean treatment period was 3.5 ± 0.95 years; 79.4% of children were treated for more than 3 years. The two main indications for GH therapy were idiopathic short stature (ISS) ( = 1,615, 67.9%) and GH deficiency (GHD) ( = 611, 25.7%). Children in the highest socio-economic-status (SES) tertile comprised 61.3% of ISS and 59.7% of GHD. After 3 years, mean height gain SDS was 1.09 ± 0.91 for GHD and 0.96 ± 0.57 for ISS ( = 0.0004). Adult height (age 15 for girls and 17 for boys) was recorded for 624 patients (26.2%) with better outcomes for GHD than ISS (-1.0±0.82 vs. -1.28±0.93, respectively; = 0.0002). Good adherence was achieved in 78.2% of the cohort during the first year and declined thereafter to 68.1% during the third year of the treatment. Children who initiate GH therapy are predominantly male, belong mainly to the upper SES, commence treatment a long period after initial recognition of short stature, and have suboptimal adherence. Appropriate referral, diagnosis, and follow-up care may result in better treatment outcomes with GH therapy.
利用一个先进的计算机化数据库评估以色列现实环境中十年的生长激素（GH）治疗模式及结果。这项大型回顾性数据库研究纳入了2379名在马卡比医疗服务机构开始接受GH治疗的儿童（2004年1月至2014年12月期间）。在随访期间评估治疗依从性良好（覆盖天数比例>80%）的情况。在开始GH治疗时：62.1%为男孩；身高标准差评分（SDS）为-2.36±0.65（均值±标准差）；年龄为9.8±3.1岁；从矮小症诊断到首次购买GH的时间为4.8±3.3年。平均治疗期为3.5±0.95年；79.4%的儿童接受治疗超过3年。GH治疗的两个主要适应症是特发性矮小症（ISS）（n = 1615，67.9%）和生长激素缺乏症（GHD）（n = 611，25.7%）。社会经济地位（SES）最高三分位数的儿童占ISS的61.3%和GHD的59.7%。3年后，GHD的平均身高增长SDS为1.09±0.91，ISS为0.96±0.57（p = 0.0004）。记录了624例患者（26.2%）的成人身高（女孩15岁，男孩17岁），GHD的结果优于ISS（分别为-1.0±0.82和-1.28±0.93；p = 0.0002）。队列中78.2%的患者在第一年实现了良好的依从性，此后在治疗的第三年降至68.1%。开始接受GH治疗的儿童以男性为主，主要属于较高的SES，在最初确认矮小症后很长一段时间才开始治疗，并且依从性欠佳。适当的转诊、诊断和后续护理可能会使GH治疗产生更好的治疗效果。
