Hi everyone, I am searching for solid scientific references for using Growth hormone treatment in patients with Achondroplasia and Hypocondroplasia, because as you know the only approved FDA indications for Growth hormone treatment include: GH deficiency, chronic kidney disease, Turner syndrome, small-for-gestational age with failure to catch up to the normal height percentiles, Prader-Willi syndrome, idiopathic short stature, SHOX gene haploinsufficiency and Noonan syndrome.
There is no solid evidence. With novel drug therapy (i.e. CNP analoga) now firmly on the horizon, it is time to consider a new era in the management of disorders of chondrogenesis (growth).
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7098936/
Nice ,great thanks Prof.Wolfgang, you brought our attention to this treatment option ,Vosoritide(CNP-analogue) acting by inhibiting the FGFR3-mediated MAPK signaling pathway...by your respectful opinion is this drug available in near future in arab countries?? so i can give a hope to those patients in my country?
Dear Dr Wasnaa Hadi Abdullah
To my knowledge rhGH is still not recommended in Achondroplasia by most if not all of endocrine societies due to lack of evidence or sufficient trials. However, It seems there are promising results from Dr Mª Angeles Zorrilla Lopez-Perea
studies.
Growth hormone therapy is licensed for treatment of achondroplasia in Japan only. It was reported that it resulted in an increase in final adult height by 3.5-8.0 cm in males and 2.8-4.2 cm in females compared to untreated subjects. However, no randomized controlled trials have been conducted yet. So, there is no solid evidence for its use in the treatment of achondroplasia.
Reference: Yorifuji T, Higuchi S, Kawakita R. Growth Hormone Treatment for Achondroplasia. Pediatr Endocrinol Rev. 2018 Sep;16(Suppl 1):123-128. doi: 10.17458/per.vol16.2018.yhk.ghachondroplasia. PMID: 30378790.
- Badges
- Science method