Peer-reviewed article
Long-term effectiveness and safety of daily growth hormone therapy in Japanese children with Noonan syndrome: a post-marketing surveillance study.
Koji Muroya, Masanobu Kawai, Hiroyuki Yamagishi, Takaaki Endo, Alberto Pietropoli et al. ·
Summary
Written by AI, not yet AICS's view · claude-haiku-4-5-20251001This study tracked 70 Japanese children with Noonan syndrome who received growth hormone therapy over several years to check if it was safe and effective. Most children showed improvement in height, with few serious side effects reported. The researchers found that the treatment worked well, though they recommend careful monitoring for children who also have heart problems.
Key points
- 70 children received daily growth hormone therapy; 35 were new patients and 35 had been in a previous 4-year trial
- New patients showed height improvement of 1.01 SDS (Japanese standard) and existing patients 1.01 SDS, indicating effective treatment
- Only 5 adverse drug reactions were reported in new patients and 5 in existing patients across the entire study period
- One death occurred in a patient with pre-existing cardiomyopathy, but causality to the drug was judged unlikely
- No new safety issues were identified; treatment was well tolerated overall
Limitations
This was a non-interventional observational study without a control group, so we cannot be certain that height improvements were due solely to the treatment rather than other factors. The study is specific to Japanese children with Noonan syndrome and may not generalise to other populations.
Abstract
Reports on long-term safety and effectiveness of daily GH replacement therapy in Japanese children with short stature due to Noonan syndrome (NS) are limited. This post-marketing, prospective, non-interventional study (ClinicalTrials.gov NCT03435627) evaluated the long‑term safety and effectiveness of daily GH therapy in this patient population. The study took place at 22 sites in Japan during November 2017-January 2022. Seventy participants were enrolled and received Norditropin® at least once during the study as per routine clinical practice: new patients (n = 35) received Norditropin® (somatropin) after study initiation and existing patients (n = 35) were previously enrolled in a 4-year trial of Norditropin® for NS (ClinicalTrials.gov NCT01927861). The main outcome measures were adverse drug reactions (ADRs) and serious adverse events (SAEs). Improvements in height were also measured. In total, four new patients experienced five ADRs and one new patient experienced one SAE. Five existing patients experienced one ADR each and three existing patients experienced one SAE each. One existing patient with pre-existing cardiomyopathy who experienced an SAE (arrhythmia) died during the study; Norditropin® causality was judged 'unlikely.' Change from baseline in mean (SD) height standard deviation score (SDS), according to Japanese and NS standards, was 1.01 (0.5) and 0.92 (0.3) for new patients and 1.01 (1.0) and 1.31 (0.8) for existing patients, respectively. Our results show that Norditropin® effectively improved height outcomes in Japanese children with NS and was well tolerated with no new safety issues identified. For patients with NS and cardiomyopathy receiving GH therapy, careful monitoring is advised.
Findings the Hub has extracted
Each claim below is labelled by its type of design and links back to this report.
- Intervention
- Daily growth hormone therapy (Norditropin®, somatropin)
- Population
- 70 Japanese children with Noonan syndrome; 35 new patients and 35 existing patients from a prior trial
Key finding
New patients improved by mean 1.01 SDS in height (Japanese standard) and 0.92 SDS (Noonan syndrome standard)
Muroya et al. · PubMed (US National Library of Medicine) · 22 Nov 2025