ESPE2023 Poster Category 1 GH and IGFs (48 abstracts)
Clinical predictors of good/poor response to growth hormone treatment (GHT) in children with idiopathic short stature (ISS)
Andrew Dauber 1 , Moshe Phillip 2,3 , Jean-Marc Ferran 4 , Nicky Kelepouris 5 , Navid Nedjatian 6 , Anne Helene Olsen 7 & Alexander Jorge 8
1Children’s National Hospital, Washington, USA. 2Schneider Children's Medical Center of Israel, Petah Tikva, Israel. 3Sackler Faculty of Medicine, Tel-Aviv University, Tel-Aviv, Israel. 4Qualiance ApS, Copenhagen, Denmark. 5US Medical Affairs, Rare Endocrine Disorders, Novo Nordisk Inc, Plainsboro, USA. 6Global Medical Affairs, Rare Endocrine Disorders, Novo Nordisk Health Care AG, Zurich, Switzerland. 7Epidemiology, Novo Nordisk A/S, Søborg, Denmark. 8Endocrinology Division/Genetic Endocrinology Unit, University of São Paulo School of Medicine, São Paulo, Brazil
Children with ISS vary in their response to GHT. We conducted a post hoc analysis to identify clinical characteristics associated with very good or poor response during year 1 of GHT in a subset of 1550 GH naïve children with ISS from NordiNet® IOS (NCT00960128) and the ANSWER Program (NCT01009905). We included patients aged 3–11 years (males) or 3–10 years (females) at treatment start, prepubertal throughout year 1 of treatment, with height SDS (HSDS) ≤–2 measured <2 months prior to GHT start. Children with gestational age <32 weeks, receiving any investigational medicinal product or with concomitant illness that could influence response to GHT were excluded, except children with ADHD. Response analysis set comprised 213 patients. Good/poor responders were defined as patients with a change in HSDS >1.0/<0.4, corresponding to >85th/<15th percentile, respectively, in the first year of GHT (n=31, for each group). Bivariate logistic analyses (Table) suggested age at treatment start, target HSDS and baseline HSDS corrected for target HSDS were the strongest predictors between good and poor responders. In a separate backward stepwise multivariate logistic regression, sex, age at treatment start and target HSDS were shown to be independent predictors of response to GHT (data not shown). By analysing clinical variables in prepubertal children with ISS who have had good/poor response during the first year of GHT, we identified predictors of response to GHT. Future studies of genetic variants associated with good/poor response to GHT could help identify genetic pathways involved in ISS and would assist in treatment individualisation.
| Variable | Poor responders n=31 | Good responders n=31 | Odds ratio [95% confidence interval] P-value |
| Sex (female) | 25.81% | 16.13% | 0.55 [0.16;1.93] P=0.35291 |
| Age at treatment start (years) | 7.7 (1.8) | 6.3 (2.2) | 0.70 [0.54;0.92] P=0.00964 |
| Baseline dose (µg/kg/day) | 46.2 (19.9) | 47.0 (21.6) | 1.00 [0.98;1.03] P=0.87056 |
| Gestational age (weeks) | 38.8 (2.1) | 38.5 (2.2) | 0.92 [0.72;1.17] P=0.48822 |
| Target HSDS | −1.10 (0.7) | −0.56 (0.7) | 2.94 [1.30;6.66] P=0.00972 |
| Target HSDS – baseline HSDS | 1.77 (1.1) | 2.33 (0.9) | 1.78 [1.01;3.12] P=0.04491 |
| Birth weight SDS | −0.45 (1.0) | −0.48 (0.7) | 0.95 [0.53;1.73] P=0.87617 |
| Birth length SDS | −0.29 (1.3) | −0.17 (1.3) | 1.08 [0.65;1.78] P=0.77387 |
| Baseline weight SDS | −2.01 (1.4) | −2.25 (1.6) | 0.89 [0.62;1.29] P=0.55228 |
| Baseline body mass index SDS | −0.13 (1.2) | −0.14 (1.2) | 0.99 [0.65;1.52] P=0.96486 |
| Baseline HSDS | −2.87 (0.7) | −2.95 (0.6) | 0.82 [0.37;1.83] P=0.62960 |
| Mean (SD) except percentage. For some items, data were not available for all patients. | |||
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