hrp0092lb-1 | Late Breaking Posters | ESPE2019

A Trial Investigating the Long-Term Efficacy and Safety of Two Doses of Norditropin® (Somatropin; Recombinant Human Growth Hormone) in Japanese Children with Short Stature Due to Noonan Syndrome Over Four Years of Treatment

Horikawa Reiko , Ogata Tsutomu , Matsubara Yoichi , Yokoya Susumu , Ogawa Yoshihisa , Nishijima Keiji , Endo Takaaki , Ozono Keiichi

Objectives: This trial (NCT01927861) evaluated the growth-promoting effect and safety of Norditropin® (somatropin; recombinant human growth hormone) in Japanese children with short stature due to Noonan syndrome over four years of treatment.Methods: Pre-pubertal children diagnosed with Noonan syndrome were randomized 1:1 to receive Norditropin® 0.033 mg/kg/day or 0.066 mg/kg/day. Change in...

hrp0097p1-289 | GH and IGFs | ESPE2023

Long-term effectiveness and safety of growth hormone therapy in Japanese children with short stature due to Noonan syndrome (NS): real-world data

Muroya Koji , Kawai Masanobu , Yamagishi Hiroyuki , Endo Takaaki , Pietropoli Alberto , Horikawa Reiko

Norditropin® was approved for children with short stature due to NS in Japan in 2017. The aim of this post-marketing surveillance study was to evaluate long-term safety and effectiveness of Norditropin® for the approved indication. This real-world non-interventional study (NCT03435627) was conducted February 2018–January 2022. Seventy patients enrolled: 35 received Norditropin® after study initiation (new patients); 35 were previously ra...