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Adherence to treatment in children with growth hormone deficiency, small for gestational age and Turner syndrome in Mexico: results of the Easypod™ connect observational study (ECOS).

墨西哥生长激素缺乏症、小于胎龄儿和特纳综合征患儿的治疗依从性: Easypod 的结果™连接观察性研究 (ECOS)。

  • 影响因子:2.82
  • DOI:10.1007/s40618-020-01218-4
  • 作者列表:"Blanco-López A","Antillón-Ferreira C","Saavedra-Castillo E","Barrientos-Pérez M","Rivero-Escalante H","Flores-Caloca O","Calzada-León R","Rosas-Guerra CC","Koledova E","Chiquete E","Ayala-Estrada A
  • 发表时间:2020-04-01

BACKGROUND:Assessing adherence to growth hormone (GH) is challenging. The Easypod™ connect device delivers pre-set doses of recombinant human GH (r-hGH) and stores a digital record of adherence that can be shared with healthcare provider. We assessed adherence to r-hGH delivered with Easypod™ according to the approved pediatric indications for r-hGH: growth hormone deficiency (GHD), born small for gestational age (SGA) who failed to show catch-up growth and Turner syndrome (TS). METHODS:ECOS (NCT01555528) was a multicenter (24 countries), 5-year, longitudinal, observational study, which aimed to evaluate country-specific adherence to r-hGH therapy prescribed via the Easypod™ electronic injection device. The primary endpoint was yearly adherence. Secondary endpoints were height velocity, height velocity standard deviation scores (SDS), height, height SDS and IGF-1 concentrations. Clinical and auxological data were obtained from medical records and adherence from Easypod™ logs. RESULTS:This study included 147 Easypod™-naïve Mexican children assessed during 3 years (mean age: 9.96 ± 3.41 years, 56.8% boys, mean height SDS at baseline: - 2.17 ± 0.97): 118 with GHD, 24 SGA and 5 with TS. A total of 105 (71.4%) patients were GH naïve. Overall median adherence was > 90% over the first year of treatment and > 80% at 3 years. Adherence was not different by r-hGH indication or between GH-naïve or experienced patients. At 1-year follow-up, mean change in height SDS was 0.57 ± 0.34, whereas mean height velocity SDS was 2.85 ± 2.51. In all, 84.7% patients had normal IGF-1 concentrations at 1-year follow-up. Adherence was associated with change in height SDS (r = 0.239, p = 0.005) and height velocity SDS (r = 0.194, p = 0.027). CONCLUSION:Adherence rates with the Easypod™ device are high and maintained over time in GHD, SGA and TS Easypod™-naïve Mexican patients. High adherence is associated with better outcomes. Easypod™ assists physicians in monitoring adherence to r-hGH.


背景: 评估生长激素 (GH) 的依从性具有挑战性。Easypod™Connect device 提供预先设定剂量的重组人 GH (r-hGH),并存储可与医疗保健提供者共享的依从性数字记录。我们评估了与 Easypod 交付的 r-hGH 的依从性™根据 r-hGH 批准的儿科适应症: 生长激素缺乏症 (GHD),出生小于胎龄儿 (SGA) 谁未能表现出追赶性生长和特纳综合征 (TS)。 方法: ECOS (NCT01555528) 是一项多中心 (24 个国家) 、 5 年、纵向、观察性研究,旨在评估通过 Easypod 规定的 r-hGH 治疗的国家特异性依从性™电子注射装置。主要终点是年度依从性。次要终点为身高速度、身高速度标准差评分 (SDS) 、身高 SDS 和 IGF-1 浓度。临床和生长学数据来自医疗记录和 Easypod 的依从性。™日志。 结果: 本研究包括 147 Easypod™-3 岁期间评估的初始墨西哥儿童 (平均年龄: 9.96 ± 3.41 岁,56.8% 男孩,基线时平均身高 SDS:-2.17 ± 0.97): 118 患有 GHD, 24 SGA 和 5 与 TS。共有 105 例 (71.4%) 患者为 GH 初治。治疗第一年的总体中位依从性为> 90%,3 年时> 80%。R-hGH 适应症或 GH 初治或经验患者之间的依从性无差异。1 年随访时,身高 SDS 的平均变化为 0.57 ± 0.34,而平均身高速度 SDS 为 2.85 ± 2.51。总之,84.7% 例患者在 1 年随访时 IGF-1 浓度正常。依从性与身高 SDS (r = 0.239,p = 0.005) 和身高速度 SDS (r = 0.194,p = 0.027) 的变化相关。 结论: Easypod 的贴壁率™在 GHD 、 SGA 和 TS Easypod 中,设备很高并随着时间的推移而保持不变™-天真的墨西哥病人.高依从性与更好的结果相关。Easypod™协助医生监测 r-hGH 的依从性。



作者列表:["Galm, Brandon P.","Buckless, Colleen","Swearingen, Brooke","Torriani, Martin","Klibanski, Anne","Bredella, Miriam A.","Tritos, Nicholas A."]

METHODS:Purpose Given the paucity of reliable predictors of tumor recurrence, progression, or response to somatostatin receptor ligand (SRL) therapy in acromegaly, we attempted to determine whether preoperative MR image texture was predictive of these clinical outcomes. We also determined whether image texture could differentiate somatotroph adenomas from non-functioning pituitary adenomas (NFPAs). Methods We performed a retrospective study of patients with acromegaly due to a macroadenoma who underwent transsphenoidal surgery at our institution between 2007 and 2015. Clinical data were extracted from electronic medical records. MRI texture analysis was performed on preoperative non-enhanced T1-weighted images using ImageJ (NIH). Logistic and Cox models were used to determine if image texture parameters predicted outcomes. Results Eighty-nine patients had texture parameters measured, which were compared to that of NFPAs, while 64 of these patients had follow-up and were included in the remainder of analyses. Minimum pixel intensity, skewness, and kurtosis were significantly different in somatotroph adenomas versus NFPAs (area under the receiver operating characteristic curve, 0.7771, for kurtosis). Furthermore, those with a maximum pixel intensity above the median had an increased odds of IGF-I normalization on SRL therapy (OR 5.96, 95% CI 1.33–26.66), which persisted after adjusting for several potential predictors of response. Image texture did not predict tumor recurrence or progression. Conclusion Our data suggest that MRI texture analysis can distinguish NFPAs from somatotroph macroadenomas with good diagnostic accuracy and can predict normalization of IGF-I with SRL therapy.

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翻译标题与摘要 下载文献
作者列表:["Xiong Y","Tang Y","Fan F","Zeng Y","Li C","Zhou G","Hu Z","Zhang L","Liu Z"]

METHODS::Growth hormone-secreting pituitary adenoma (GHPA), a benign endocrine tumor located in the base of the skull, results in acromegaly. In addition to the mass effect of the tumor itself in the sellar region, GHPA can lead to the overgrowth of almost every organ. Previous findings indicated that the processes underlying acromegaly were partly attributable to hyperactivity of the growth hormone/insulin-like growth factor-1 (GH/IGF-1) axis. However, the mechanisms driving this syndrome remains largely unknown. Additionally, the roles of GHPA-derived exosomes, which contain functional microRNAs and proteins that manipulate target cell proliferation and differentiation in distal extremities, are also unknown. In this study, we demonstrated that GHPA exosomes promote bone formation in vitro and trabecula number in vivo. The mechanism of increased trabecula formation may be attributable to GHPA exosome-induced osteoblast proliferation via increased cell viability and DNA replication. We further discovered that exosomal hsa-miR-21-5p plays a distinct role from the GH/IGF-1 axis in these processes. Accordingly, the results of this study provide a novel mechanism whereby GHPA influences distal extremities and a new perspective for treating GHPA.

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翻译标题与摘要 下载文献
作者列表:["Arlien-Søborg MC","Grøndahl C","Bæk A","Dal J","Madsen M","Høgild ML","Pedersen SB","Bjerre M","Jørgensen JOL"]

METHODS:BACKGROUND:Fibroblast growth factor 21 (FGF21) is a circulating hormone with pleiotropic metabolic effects, which is inactivated by fibroblast activation protein (FAP). Data regarding interaction between FGF21, FAP, and growth hormone (GH) are limited, but it is noteworthy that collagens are also FAP substrates, since GH potently stimulates collagen turnover. AIM:To measure circulating FGF21 components, including FAP, in patients with acromegaly before and after disease control. METHODS:Eighteen patients with active acromegaly were studied at the time of diagnosis and ≥ 6 months after disease control by either surgery or medical treatment. Serum levels of total and active FGF21, β-klotho, FAP, and collagen turnover markers were measured by immunoassays. Expression of putative FGF21-dependent genes were measured in adipose tissue by reverse transcriptase-polymerase chain reaction, body composition assessed by dual-energy x-ray absorptiometry scan, and insulin sensitivity estimated with homeostatic model assessment of insulin resistance (HOMA-IR). RESULTS:Total FGF21, active FGF21 and β-klotho remained unchanged. Insulin sensitivity and body fat mass increased after disease control but neither correlated with active FGF21. Expression of FGF21-dependent genes did not change after treatment. FAP levels (µg/L) were markedly reduced after treatment [105.6 ± 29.4 vs 62.2 ± 32.4, P < 0.000]. Collagen turnover markers also declined significantly after treatment and ΔFAP correlated positively with ΔProcollagen Type I (P < 0.000) and Type III (P < 0.000). CONCLUSION:1) Circulating FGF21 and β-klotho do not change in response to acromegaly treatment, 2) FAP concentrations in serum decrease after disease control and correlate positively with collagen turnover markers, and 3) FAP is a hitherto unrecognized GH target linked to collagen turnover. CLINICAL TRIALS REGISTRATION:NCT00647179.