Introduction: Achondroplasia is a rare skeletal dysplasia characterized by severe disproportionate short stature. Vosoritide is currently the only approved therapy. The CrescNet registry is a network of primary and specialized pediatric tertiary centers that aims to improve early detection of growth disorders in Europe. In 2021, an achondroplasia-specific data collection module was set up within CrescNet to enhance data collection among children with achondroplasia and assess the impact of interventions. Here, we describe the module setup and report preliminary real-world outcomes of vosoritide treatment over 3 years.Methods: The module was established in 10 of 11 countries participating in CrescNet. Achondroplasia-specific data were collected, including developmental milestones, interventions (such as limb-lengthening surgery, treatment with vosoritide and growth hormone), complications, and health-related quality of life, alongside standard anthropometric measurements (eg height, weight, etc). Pseudonymized data were sent to the CrescNet central database, Leipzig University Hospital, Germany, for analysis by age and treatment status.Results: As of May 2025, 486 participants from 32 tertiary centers were enrolled. Data from 73 untreated and 186 vosoritide-treated individuals with genetically documented achondroplasia were analyzed. In vosoritide-treated individuals, mean height standard deviation score, referenced to an untreated European achondroplasia population, significantly increased from baseline at 1, 2, and 3 years after vosoritide initiation (P≤0.0001).Conclusions: The module facilitates the collection of real-world data to improve understanding of the natural history of achondroplasia and outcomes associated with interventions. Growth data from vosoritide-treated individuals were consistent with clinical trial findings and published real-world data. Longer-term follow-up is ongoing.

Introduction: Achondroplasia is a rare skeletal dysplasia characterized by severe disproportionate short stature. Vosoritide is currently the only approved therapy. The CrescNet registry is a network of primary and specialized pediatric tertiary centers that aims to improve early detection of growth disorders in Europe. In 2021, an achondroplasia-specific data collection module was set up within CrescNet to enhance data collection among children with achondroplasia and assess the impact of interventions. Here, we describe the module setup and report preliminary real-world outcomes of vosoritide treatment over 3 years.Methods: The module was established in 10 of 11 countries participating in CrescNet. Achondroplasia-specific data were collected, including developmental milestones, interventions (such as limb-lengthening surgery, treatment with vosoritide and growth hormone), complications, and health-related quality of life, alongside standard anthropometric measurements (eg height, weight, etc). Pseudonymized data were sent to the CrescNet central database, Leipzig University Hospital, Germany, for analysis by age and treatment status.Results: As of May 2025, 486 participants from 32 tertiary centers were enrolled. Data from 73 untreated and 186 vosoritide-treated individuals with genetically documented achondroplasia were analyzed. In vosoritide-treated individuals, mean height standard deviation score, referenced to an untreated European achondroplasia population, significantly increased from baseline at 1, 2, and 3 years after vosoritide initiation (P≤0.0001).Conclusions: The module facilitates the collection of real-world data to improve understanding of the natural history of achondroplasia and outcomes associated with interventions. Growth data from vosoritide-treated individuals were consistent with clinical trial findings and published real-world data. Longer-term follow-up is ongoing.

AbstractIntroductionResearch on severe obesity (SO) is scarce and often contradictory. As higher weight status persists into adulthood, we aimed to analyze long-term trends in the prevalence of SO in children and adolescents in Germany using a large real-world data set. Furthermore, we analyzed subgroup differences and assessed how the COVID-19 pandemic affected weight status.MethodsWe analyzed data from the CrescNet auxological network including 1,495,401 clinical visits by 4-16-year-old children (2002-2023). Weight trends were examined with a focus on SO using logistic regression, stratified by sex and age. Effects are reported as odds ratio per 5 years (OR5) pre-pandemic and as OR for consecutive years during the pandemic. Quantile regression assessed trends of the 50th, 90th, 97th, and 99th percentiles of excess weight.ResultsPre-pandemic, SO remained stable or declined in children under 12 until 2010: OR5: 0.8-1, p<0.001) but rose significantly afterward (OR5: 1.1-1.2, p<0.001). Children between 12-16 years of age showed a continuous increase, especially boys (boys12-16: OR5: 1.3, girls12-16: OR5: 1.1, p<0.001). During the pandemic, SO peaked in 2021 across all groups (OR21vs19: 1.3-1.7, p<0.001). By 2023, younger children returned to pre-pandemic levels, while older children, particularly 8-16-year-old girls, remained at higher weights (OR23vs19: 1.2-1.5, p<0.001-0.002). Weight gain was most prominent in SO groups but could also be seen in pre-pandemic and pandemic overweight and obesity subgroups, generally decreasing toward the end of the pandemic. ConclusionSO has increased over the last two decades, with the COVID-19 pandemic accelerating this trend, particularly in adolescents. While younger children recovered by 2023, excess weight in older children, especially girls, continues to escalate.

AbstractIntroductionResearch on severe obesity (SO) is scarce and often contradictory. As higher weight status persists into adulthood, we aimed to analyze long-term trends in the prevalence of SO in children and adolescents in Germany using a large real-world data set. Furthermore, we analyzed subgroup differences and assessed how the COVID-19 pandemic affected weight status.MethodsWe analyzed data from the CrescNet auxological network including 1,495,401 clinical visits by 4-16-year-old children (2002-2023). Weight trends were examined with a focus on SO using logistic regression, stratified by sex and age. Effects are reported as odds ratio per 5 years (OR5) pre-pandemic and as OR for consecutive years during the pandemic. Quantile regression assessed trends of the 50th, 90th, 97th, and 99th percentiles of excess weight.ResultsPre-pandemic, SO remained stable or declined in children under 12 until 2010: OR5: 0.8-1, p<0.001) but rose significantly afterward (OR5: 1.1-1.2, p<0.001). Children between 12-16 years of age showed a continuous increase, especially boys (boys12-16: OR5: 1.3, girls12-16: OR5: 1.1, p<0.001). During the pandemic, SO peaked in 2021 across all groups (OR21vs19: 1.3-1.7, p<0.001). By 2023, younger children returned to pre-pandemic levels, while older children, particularly 8-16-year-old girls, remained at higher weights (OR23vs19: 1.2-1.5, p<0.001-0.002). Weight gain was most prominent in SO groups but could also be seen in pre-pandemic and pandemic overweight and obesity subgroups, generally decreasing toward the end of the pandemic. ConclusionSO has increased over the last two decades, with the COVID-19 pandemic accelerating this trend, particularly in adolescents. While younger children recovered by 2023, excess weight in older children, especially girls, continues to escalate.

Introduction: Several studies have analyzed the association between the maximal growth hormone serum level obtained during a growth hormone stimulation test (GH-Max) and the body mass index standard deviation score (BMI-SDS). However, as sample sizes were quite small, our study aimed to analyze the association between GH-Max and BMI-SDS within a large cohort of 991 children. Further, we investigated other influencing factors, like test type, age, sex, puberty and preterm birth. Methods: Children with short stature (height < 10th percentile) received growth hormone stimulation tests with arginine or glucagon at the Department of Paediatric Endocrinology of the University of Leipzig Medical Center. The study population included a total of 1,438 tests (633 tests on girls, 805 tests on boys), with the majority consisting of prepubertal children (tests = 1,138). The mean age at testing was 7.74 years. Analyses were carried out on the entire cohort as well as stratified by test types. We performed univariate and multivariate analyses using linear mixed effect models to assess the effects on GH-Max. Results: GH-Max and BMI-SDS were significantly negatively associated with an effect size of ß = -1.10 (p < 0.001), independent from the test type. The GH-Max values were significantly (p < 0.001) higher for glucagon (mean value: 9.65 ng/ml) than those for arginine tests (mean value: 8.50 ng/ml). Age, sex, premature birth, and puberty were not significantly related to GH-Max values. Conclusion: We confirmed the negative association between GH-Max and weight status of short children found in previous studies. Therefore, considering BMI-SDS may be helpful in the assessment of growth hormone stimulation tests in short-statured children, but it should not be the determining factor for a treatment decision.

Introduction: Several studies have analyzed the association between the maximal growth hormone serum level obtained during a growth hormone stimulation test (GH-Max) and the body mass index standard deviation score (BMI-SDS). However, as sample sizes were quite small, our study aimed to analyze the association between GH-Max and BMI-SDS within a large cohort of 991 children. Further, we investigated other influencing factors, like test type, age, sex, puberty and preterm birth. Methods: Children with short stature (height < 10th percentile) received growth hormone stimulation tests with arginine or glucagon at the Department of Paediatric Endocrinology of the University of Leipzig Medical Center. The study population included a total of 1,438 tests (633 tests on girls, 805 tests on boys), with the majority consisting of prepubertal children (tests = 1,138). The mean age at testing was 7.74 years. Analyses were carried out on the entire cohort as well as stratified by test types. We performed univariate and multivariate analyses using linear mixed effect models to assess the effects on GH-Max. Results: GH-Max and BMI-SDS were significantly negatively associated with an effect size of ß = -1.10 (p < 0.001), independent from the test type. The GH-Max values were significantly (p < 0.001) higher for glucagon (mean value: 9.65 ng/ml) than those for arginine tests (mean value: 8.50 ng/ml). Age, sex, premature birth, and puberty were not significantly related to GH-Max values. Conclusion: We confirmed the negative association between GH-Max and weight status of short children found in previous studies. Therefore, considering BMI-SDS may be helpful in the assessment of growth hormone stimulation tests in short-statured children, but it should not be the determining factor for a treatment decision.