Encouraging real-world CV safety profile for rhGH in Noonan syndrome

medwireNews: Treatment with recombinant human growth hormone (rhGH) is not associated with cardiovascular (CV) adverse events in clinical practice, show data from two large, multicentre cohorts.

The researchers identified 412 patients with Noonan syndrome who were participants in the NordiNet® IOS or the ANSWER Program – two studies evaluating the long-term effectiveness and safety of rhGH.

These children were an average age of 9.3 years at baseline, had an average height standard deviation score of –2.65 and 70.9% were male. The average rhGH dose used was 0.047 mg/kg per day.

No CV safety events were reported during treatment, which Juan Pablo Kaski (Great Ormond Street Hospital & UCL Institute of Cardiovascular Science, London, UK) and co-researchers say “is encouraging, particularly given the pre-existing CV comorbidities in some patients.”

At baseline, 11.7% had a recorded CV morbidity, which the team observes is low given previous reports of cardiac anomalies in up to 80% of patients with Noonan syndrome.

“This contrast in prevalence suggests that patients with pre-existing CV disease may have been under-represented owing to a selection bias in GH prescribing practices, and/or the conditions may have been under-reported at baseline”, they write in Endocrine Connections.

“Thus, as active screening of participants was not required prior to enrolment in the study, the five CV comorbidities reported after GH treatment initiation may have also been pre-existing.”

These comorbidities comprised one case of pulmonary valve stenosis (PVS), one ruptured abdominal aortic aneurysm and three cases of unspecified CV disease.

At baseline, 18 patients had PVS, four had atrial septal defects and three had hypertrophic cardiomyopathy. No worsening of CV conditions was reported during rhGH treatment.

The researchers say the CV safety results “need to be interpreted with caution”, due to the low rate of pre-existing CV disease.

“However, the low prevalence of CV comorbidities in our patient population highlights the importance of baseline cardiovascular assessments before initiating GH therapy, particularly in patients with genotypes associated with a higher CV risk”, they say.

In total, there were 34 adverse events in 22 patients during treatment, with the most common being headache and arthralgia. Ten of these events were classed as serious, and included two instances of brain neoplasm, one with metastasis to the spine, and a case of Moyamoya disease.

By Eleanor McDermid

medwireNews is an independent medical news service provided by Springer Healthcare Ltd. © 2022 Springer Healthcare Ltd, part of the Springer Nature Group

Endocr Connect 2021; doi:10.1530/EC-21-0549
Martin Savage
Programme Director
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