ESPE2024 Top 20 Posters Top 20 Posters (19 abstracts)
Real World Effectiveness of Vosoritide in 165 Children with Achondroplasia – Data from a Multicenter European Registry
Katja Wechsung 1 , Maesa Al-Halak 2 , Daniela Avdjieva-Tzavella 3 , Susanne Bechtold-Dalla Pozza 4 , Christoph Beger 5 , Ruth Gausche 5 , Corinna Grasemann 6 , Wolfgang Högler 7 , Matthias Hösl 8 , Heike Hoyer-Kuhn 9 , Angela Huebner 10 , Anja Ingwers 11 , Marco Janner 12 , Alexandra Keller 13 , Philip Kunkel 14 , Sean Nader 8 , Klaus Mohnike 15 , Nicole Muschol 16 , Katja Palm 15 , Simone Pötzsch 17 , Frank Rutsch 18 , Dirk Schnabel 1 , Ondrej Soucek 19 , Ioana Streata 20 , Björn Vogt 18 , Thomas MK Völkl 21 , Joachim Wölfle 22 , Jiri Zeman 23 & Roland Pfäffle 5
1Charité - Universitätsmedizin, Berlin, Germany. 2KBO Kinderzentrum, Munich, Germany. 3SBAL Children´s Hospital, Sofia, Bulgaria. 4Ludwig Maximilians University, Munich, Germany. 5Leipzig University, Leipzig, Germany. 6Center for rare diseases Ruhr, Bochum, Germany. 7Johannes Kepler University Linz, Linz, Austria. 8Schön Clinics, Vogtareuth, Germany. 9University Cologne, Cologne, Germany. 10Faculty of Medicine and University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany. 11Amedes Endocrinology, Berlin, Germany. 12Pediatric Endocrinology Bern, Bern, Switzerland. 13Kinderzentrum Leipzig, Leipzig, Germany. 14Mannheim University, Mannheim, Germany. 15Otto von Guericke University, Magdeburg, Germany. 16University Medical Center Hamburg-Eppendorf, Hamburg, Germany. 17Helios Clinic Plauen, Plauen, Germany. 18University Münster, Muenster, Germany. 19Motol University Hospital, Prague, Czech Republic. 20Regional Center of Medical Genetics Dolj – Emergency Clinical County Hospital Craiova University of Medicine and Pharmacy from Craiova, Craiova, Romania. 21KJF Klinikum Josefinum, Augsburg, Germany. 22Friedrich-Alexander University, Erlangen, Germany. 23General University Hospital, Prague, Czech Republic
Introduction: Vosoritide is licensed by EMA for the treatment of children with achondroplasia above 4 months of age in 2023 (initial approval for children above 2 years in 2021). The CNP analogue modulates enchondral bone growth and improves growth velocity. Response to therapy varies between individuals. Nineteen European centers enter auxological and diagnosis-specific data into the achondroplasia module of the CrescNet® registry to monitor treatment. We report changes in weight, height and sitting height after two years of treatment in children from the CrescNet® registry and analyze factors that modify growth response.
Methods: Changes in height and weight were analyzed in 165 children (80 females), and sitting height/height ratio in 78 children. SDS values were calculated using the LMS method with achondroplasia-specific reference data. We defined treatment response according to delta height SDS as: non-response (< 0.0), moderate (0.0 to 0.5) and good response (> 0.5). Linear models were derived for delta height SDS and change in sitting height ratio SDS since treatment start (baseline), with treatment length, baseline sitting height ratio SDS, baseline BMI SDS, baseline age, and sex as influencing factors.
Results: Mean age at baseline was 6.60 years (SD = 3.7; 4% below 2 years, 30% 2-4 years, 30% 4-8 years, 26% 8-12 years and 10% above 12 years). 9 children (7.6%, 7 female) were non-responders at 1 year and 2 children (4.2%, 1 female) at 2 years of vosoritide treatment. Moderate responders were 61 children (51.7%) at 1 year and 6 children (12.8%) at 2 years. Good response was seen in 48 children (40.7%) at 1 year and 39 children (83%) at 2 years. In the linear model, the overall change in height was +0.55 SDS per year of treatment, with influences from baseline age (-0.01, P = 0.014), sex (female -0.018, P < 0.001) and sitting height ratio SDS (0.029, P = 0.005). There was no significant change in SDS of sitting height/height ratio during treatment (-0.39, P = 0.13).
Conclusion: The majority of children treated with vosoritide responded well to treatment, comparable to clinical trial data – however some showing a delayed response. Young age at baseline (more pronounced in females), BMI-SDS and sitting height ratio SDS positively correlated with growth. There were few non-responders in our study. Adherence data were not available. Further data are needed to characterize non-responders to Vosoritide and to define the implications of good and moderate response in clinical practice.
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