Dataset Information

Predicting First-Year Growth in Response to Growth Hormone Treatment in Prepubertal Korean Children with Idiopathic Growth Hormone Deficiency: Analysis of Data from the LG Growth Study Database.

ABSTRACT: BACKGROUND:The first-year growth in response to growth hormone (GH) treatment seems to be the most important factor in determining the overall success of GH treatment. METHODS:Data from children (n = 345) who were in the LG Growth Study Database were used to develop a model. All subjects had been diagnosed with idiopathic growth hormone deficiency (GHD) and presented in a prepubertal state during the first year of GH treatment. RESULTS:The ?height standard deviation score (SDS) during 1st year of GH treatment was correlated positively with weight-SDS (? = 0.304, P < 0.001), body mass index (BMI)-SDS (? = 0.443, P < 0.001), paternal height-SDS (? = 0.296, P = 0.001), MPH-SDS (? = 0.421, P < 0.001) and MPH SDS minus baseline height SDS (? = 0.099, P < 0.001) but negatively with chronological age (? = -0.294, P < 0.001), bone age (? = -0.249, P < 0.001). A prediction model of 1st year growth in response to GH treatment in prepubertal Korean children with idiopathic GHD is as follows: ?height SDS during 1st year of GH treatment = 1.06 - 0.05 × age + 0.09 × (MPH SDS minus baseline height SDS) + 0.05 × BMI SDS. This model explained 19.6% of the variability in the response, with a standard error of 0.31. CONCLUSION:The present model to predict first-year response to GH treatment might allow more tailored and personalized GH treatment in Korean prepubertal children with idiopathic GHD. TRIAL REGISTRATION:ClinicalTrials.gov Identifier: NCT01604395.

SUBMITTER: Cho WK

PROVIDER: S-EPMC7234860 | biostudies-literature | 2020 May

REPOSITORIES: biostudies-literature

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Predicting First-Year Growth in Response to Growth Hormone Treatment in Prepubertal Korean Children with Idiopathic Growth Hormone Deficiency: Analysis of Data from the LG Growth Study Database.

Cho Won Kyoung WK Ahn Moon Bae MB Kim Eun Young EY Cho Kyoung Soon KS Jung Min Ho MH Suh Byung Kyu BK

Journal of Korean medical science 20200518 19

<h4>Background</h4>The first-year growth in response to growth hormone (GH) treatment seems to be the most important factor in determining the overall success of GH treatment.<h4>Methods</h4>Data from children (n = 345) who were in the LG Growth Study Database were used to develop a model. All subjects had been diagnosed with idiopathic growth hormone deficiency (GHD) and presented in a prepubertal state during the first year of GH treatment.<h4>Results</h4>The Δheight standard deviation score ( ...[more]

PMID: 32419399

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Project description:BackgroundQuantifying the association between adherence and the growth response to growth hormone (GH) treatment is hampered by suboptimal methods of measuring adherence, confounders associated with the growth response, and restriction of the outcome parameters to yearly growth velocities.AimTo investigate the effect of adherence on the two-year growth response to GH treatment in prepubertal children with idiopathic isolated growth hormone deficiency (GHD) participating in the easypod connect observational study (ECOS), a 5-year, Phase IV open-label study to continuously assess real-world adherence via the easypod electronic drug-delivery device.Patients and methodsOutcome measures were change in height standard deviation score (?HSDS), index of responsiveness (IoR), and parameters of two catch-up growth (CUG) curve functions (monomolecular growth curve and second degree polynomial) with adj-HSDS (HSDS minus Target height (TH) SDS) as dependent variable. Inclusion criteria were GHD, naïve to GH treatment, known TH, age <10y in girls and <12y in boys, ?3 measurements, HSDS <-2 at start, complete data on growth and adherence in the first and second year. Linear regression analyses were performed to test the association between adherence (continuous and high vs. low) and the outcome measures, also adjusted for potential clinical confounders (age at start, adj-HSDS at start, birth weight SDS, gestational age (<37 weeks vs ?37 weeks), GH dose, GH max (n = 58)). The formula of IoR already adjusts for confounders.ResultsIn total, 95 patients complied with the inclusion criteria. The strongest associations were found between high adherence in the second year (?91% as cut-off value) and IoR 2y (+0.62), and average adherence and high adherence (?78%) in the first two years and ?HSDS 0-2y (+0.11 SD per 1 injection/week, and +0.34 SD for high vs. low adherence).ConclusionSuboptimal adherence negatively affected the growth response in the first two years of GH treatment.

Project description:Background:Treatment of children with growth hormone deficiency (GHD) or idiopathic short stature (ISS) using GH is only effective for bone growth prior to epiphyseal fusion. Aromatase inhibitor therapy (AIT) blocks estrogen production, thereby delaying epiphyseal fusion. The current study analyzed baseline characteristics and longitudinal data of male patients with GHD or ISS who were treated with GH and concomitant AIT. Methods:Data were obtained from the observational American Norditropin® Studies: Web-Enabled Research (ANSWER) Program, which collected efficacy and safety data of patients treated with Norditropin®. A longitudinal cohort approach compared patient characteristics, including chronologic age, bone age, and height standard deviation score (HSDS), in GH-treated males before and after AIT initiation. Results:A total of 142 GH-naïve patients with GHD (n?=?115) or ISS (n?=?27) with mean (± SD) baseline chronological ages of 12.10?±?3.00 and 10.76?±?3.07?years, respectively, were analyzed. The majority were classified at advanced Tanner stages II to V. Patients with GHD had mean HSDS of -?1.97?±?0.78 at baseline and?-?0.99?±?0.88 prior to AIT initiation, while corresponding values for patients with ISS were?-?2.15?±?0.72 and?-?1.04?±?0.79, respectively. In patients evaluated after 2?years of concomitant AIT, mean HSDS had decreased to -?0.40?±?1.16 and?-?0.65?±?0.52 for patients with GHD and ISS, respectively. Patients with GHD had a mean bone age/chronological age ratio (BA/CA) of 0.91?±?0.11 at baseline and 0.97?±?0.10 prior to AIT initiation, while corresponding values for patients with ISS were 0.85?±?0.16 and 0.99?±?0.10, respectively. In patients evaluated after 2?years of concomitant AIT, mean BA/CA values were 0.95?±?0.10 and 0.96?±?0.06 for patients with GHD and ISS, respectively. Conclusions:In this real-world analysis, use of AIT with GH in males appeared to be associated with ongoing growth over 2?years, and AIT likely augmented growth potential as indicated by continued HSDS increase with decreased BA/CA after AIT initiation. Trial registration:This trial was sponsored by Novo Nordisk and is registered with ClinicalTrials.gov (NCT01009905). Registered November 11, 2009; retrospectively registered.

Project description:Abstract Growth hormone (GH) replacement therapy currently requires daily injections. Somapacitan is a long-acting GH-derivative being developed for once-weekly (OW) use in children and adults with GH deficiency (GHD). A phase 2, multinational, randomized, open-label, controlled trial (ClinicalTrials.gov: NCT02616562) investigated the efficacy and safety of OW somapacitan compared with daily GH (Norditropin® FlexPro®). GH-treatment-naïve prepubertal children with GH deficiency received 0.04 (n=16), 0.08 (n=15) or 0.16 mg/kg/wk (n=14) subcutaneous (sc) OW somapacitan, or sc GH 0.034 mg/kg/day (0.24 mg/kg/wk; n=14) for 52 wks, followed by a 104-wk safety extension with all patients on somapacitan receiving 0.16 mg/kg/wk while GH dose was unaltered. Safety endpoints included frequency of adverse events (AEs), including injection-site reactions, and occurrence of anti-somapacitan/anti-human growth hormone (hGH) antibodies. The 52-wk efficacy and safety results have been reported. We report here safety results at 104 wks’ total treatment. Number of AEs (% of patients) was as follows: OW somapacitan 0.04/0.16 mg/kg/wk, 51 (75%); 0.08/0.16 mg/kg/wk, 89 (80%); 0.16/0.16 mg/kg/wk, 89 (100%); and for daily GH, 82 (100%). Nasopharyngitis, influenza, allergic rhinitis and gastroenteritis were the most common AEs across all treatment groups. Pyrexia was more common in the OW somapacitan 0.04/0.16 mg/kg/wk (43.8%) and 0.08/0.16 mg/kg/wk (26.7%) groups vs the higher-dose OW somapacitan and daily GH groups (7.1% each). Most AEs were mild to moderate and unlikely related to treatment. Ten serious AEs were reported in five (8.5%) children and unlikely related to treatment, except two AEs of moderate severity during the first 26 wks: generalized edema and vomiting in one child on 0.16 mg/kg/wk OW somapacitan, rated as probably related to treatment although she was also given intravenous antibiotics for suspected infection. Injection-site reactions were reported in the 0.04/0.16 mg/kg/wk (n=2) and 0.16/0.16 mg/kg/wk (n=1) OW somapacitan groups and in the daily GH group (n=1). Four children (0.04/0.16 mg/kg/wk) and one child (0.16/0.16 mg/kg/wk) had transient anti-somapacitan antibodies; one child (0.16/0.16 mg/kg/wk) had low-titer anti-somapacitan antibodies at two consecutive visits; in one child (daily GH group) with persistent low-titer anti-hGH antibodies, treatment was discontinued at wk 52. All antibodies were non-neutralizing. In conclusion, OW somapacitan was well tolerated at all doses, with no new safety or tolerability issues identified after up to 104 wks of treatment. The frequency, severity and type of AEs were similar in the OW somapacitan and daily GH treatment groups except for pyrexia, which was unlikely related to treatment and more frequently reported in the lowest dose somapacitan group.

Dataset Information

Predicting First-Year Growth in Response to Growth Hormone Treatment in Prepubertal Korean Children with Idiopathic Growth Hormone Deficiency: Analysis of Data from the LG Growth Study Database.

Publications

Predicting First-Year Growth in Response to Growth Hormone Treatment in Prepubertal Korean Children with Idiopathic Growth Hormone Deficiency: Analysis of Data from the LG Growth Study Database.

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