Safety and Effectiveness of Omnitrope® (Somatropin) in PATRO Children: A Multi-Center, Post-Marketing Surveillance Study Comparison of United States and Rest of World Data
Omnitrope® appears to improve height in children with growth hormone deficiency, with a majority experiencing mild to moderate adverse events.
Where it sits
this study against the rest of the hgh (somatropin) corpusSummary and findings
This study evaluated the safety and effectiveness of Omnitrope® (somatropin) in children with growth hormone deficiency across the US and other countries. A total of 294 patients were enrolled in the US and 6206 internationally, with 66.0% and 48.0% experiencing adverse events, respectively. The improvement in mean height SD score after 3 years was +1.25 in US patients and +1.35 in international patients.
Abstract
PAtients TReated with Omnitrope ® (PATRO) Children is an international, non-interventional study with Omnitrope ® (somatropin, Sandoz Inc.), conducted across 14 different countries. Here we present a comparison of safety and effectiveness data from patients in the United States (US) and in other countries. All visits and assessments are carried out according to routine clinical practice, and doses of Omnitrope ® are given according to country-specific prescribing information. By September 2018, 294 patients had been enrolled in the US (53.1% recombinant human growth hormone [rhGH]-naïve) and 6206 patients had been enrolled across 13 other countries (international group; 86.2% rhGH-naïve). The most common indication in both groups was growth hormone deficiency (GHD). Overall, 194 US patients (66.0%) and 2977 international patients (48.0%) experienced adverse events (AEs; 886 and 11,716 events, respectively), most of which were of mild or moderate intensity. The AEs were suspected to be treatment-related in five US patients (1.7%) and 452 international patients (7.3%). All reported neoplasms were benign, non-serious, and considered unrelated to rhGH therapy. No cases of diabetes mellitus or hyperglycemia were reported. In rhGH-naïve GHD patients, after 3 years of rhGH therapy, the improvement in mean height SD score from baseline was +1.25 and +1.35 in US and international patients, respectively. <h4>Conclusion: </h4> Omnitrope ® treatment appears to be well tolerated and effective in US patients and those from other countries. Across the pediatric indications included, there was no evidence of an increased risk of developing uncommon or unexpected AEs with rhGH. Trial registration: NA
Background
This paper addresses the safety and effectiveness of Omnitrope® (somatropin) in children with growth hormone deficiency (GHD). Prior studies have indicated that rhGH can improve growth outcomes, but comprehensive safety data across diverse populations are limited. Understanding the safety profile and effectiveness in both the US and international contexts is crucial for informed clinical practice.
Methods
This is a multi-center, non-interventional study that included 294 patients in the US and 6206 patients in 13 other countries. Patients were treated with Omnitrope® according to country-specific prescribing information. The primary outcome measured was the change in mean height SD score after 3 years of treatment, with secondary outcomes including the incidence of adverse events.
Results
The primary endpoint showed a mean height SD score improvement of +1.25 in US patients and +1.35 in international patients after 3 years. A total of 194 US patients (66.0%) and 2977 international patients (48.0%) experienced adverse events, with 886 and 11,716 events reported, respectively. Treatment-related adverse events were reported in 1.7% of US patients and 7.3% of international patients.
Interpretation
The findings suggest that Omnitrope® treatment is associated with improvements in height in both US and international pediatric populations. While the results are statistically significant, the clinical significance of the height improvement may vary among individual patients. Limitations include the non-interventional nature of the study and the absence of a control group, which makes it difficult to draw definitive conclusions about the treatment's effectiveness and safety.
Key findings
- 294 patients enrolled in the US, 6206 patients internationally.
- 66.0% of US patients and 48.0% of international patients experienced adverse events.
- 886 adverse events reported in the US and 11,716 in the international group.
- 1.7% of US patients and 7.3% of international patients had treatment-related adverse events.
- Mean height SD score improvement of +1.25 in US patients and +1.35 in international patients after 3 years.
Limitations
- Non-interventional study design limits causal inference.
- No control group for comparison.
- Potential for reporting bias in adverse event data.
- Short duration of follow-up (3 years).
- Data primarily from self-reported assessments.