Department of Endocrinology and Reproductive Medicine, Centre de Maladies Endocriniennes Rares de la Croissance et du Développement, Hôpital Universitaire Pitié Salpêtrière-Charles Foix, Sorbonne Université, Faculté de médecine, Paris Cedex 13, France.
Department of Internal Medicine, CHU Souro Sanou, Intitut Supérieur des Sciences de la Santé (IN.S.SA), Université Nazi Boni, Bobo-Dioulasso, Burkina Faso.
Eur J Endocrinol. 2022 Jul 21;187(3):373-381. doi: 10.1530/EJE-22-0160. Print 2022 Sep 1.
To analyze the effectiveness and safety of growth hormone (GH) replacement treatment in adult patients with Langerhans cell histiocytosis (LCH) and GH deficiency (GHD) enrolled in KIMS (Pfizer International Metabolic Database).
Patients with LCH and GHD were studied at baseline and some of them after 1 year of GH treatment. The effectiveness of GH is presented as change after 1 year of treatment (mean, 95% CI). The LCH population was compared to two other groups of patients enrolled in KIMS, granulomatous and lymphocytic hypophysitis.
At baseline, 81 adults with LCH (27 with childhood onset, 56% females), mean age at GHD onset of 29 (15) years were studied. Diabetes insipidus was diagnosed in 86% of patients. Analysis of 1 year of GH treatment was possible in 37 patients. One-year cross-sectional values for the GH dose were 0.39 (s.d.± 0.21) mg and -0.5 (-1.2 to 0.2) for insulin-like growth factor-1 s.d. Total cholesterol decreased 0.9 (-1.5 to -0.3 (mmol/L); P < 0.05); AGHDA-QoL-score (n = 20) was improved by 2.8 points (-5.6 to 0.0; P < 0.05), while mean BMI increased 0.6 ± 3 kg/m2 (95% CI: -0.2 to 1.4). All these effects did not differ from the two other groups after adjusting for age, gender, and baseline values. In 20 of 77 patients included in the safety analysis, 36 serious adverse events were reported during 435 patient-years (82.8/1000); no new safety signals were reported.
After 1 year of GH treatment in patients with LCH, metabolic variables and quality of life improved, with no new safety signals.
分析纳入 KIMS（辉瑞国际代谢数据库）的朗格汉斯细胞组织细胞增生症（LCH）伴生长激素缺乏（GHD）的成年患者使用生长激素（GH）替代治疗的有效性和安全性。
在基线时对 LCH 伴 GHD 的患者进行研究，其中一些患者在接受 GH 治疗 1 年后进行研究。GH 的有效性表现为治疗 1 年后的变化（平均值，95%CI）。将 LCH 人群与 KIMS 中纳入的另外两组患者（肉芽肿性和淋巴细胞性垂体炎）进行比较。
在基线时，研究了 81 名 LCH 成年患者（儿童发病 27 例，女性占 56%），GHD 发病时的平均年龄为 29（15）岁。86%的患者诊断为尿崩症。37 名患者可进行 1 年 GH 治疗的分析。1 年 GH 剂量的横断面值分别为 0.39（标准差±0.21）mg 和 -0.5（-1.2 至 0.2）胰岛素样生长因子-1 标准差。总胆固醇降低 0.9（-1.5 至-0.3mmol/L）；P<0.05）；AGHDA-QoL 评分（n=20）改善了 2.8 分（-5.6 至 0.0；P<0.05），而平均 BMI 增加了 0.6±3kg/m2（95%CI：-0.2 至 1.4）。调整年龄、性别和基线值后，这些影响在两组之间没有差异。在纳入安全性分析的 77 名患者中的 20 名中，在 435 名患者-年（82.8/1000）中报告了 36 例严重不良事件；未报告新的安全性信号。
在 LCH 患者接受 GH 治疗 1 年后，代谢变量和生活质量得到改善，且未出现新的安全性信号。
