Growth Hormone (GH) Treatment in Achondroplasia*

This is the authors' abstract. We don't add key points for this paper.

Achondroplasia, a form of short stature with short limbs, is the most common disease among osteochondrodysplasia. The growth pattern of patients with achondroplasia is unique compared with the normal standard. At birth, patients' heights are within normal limits and height velocity is normal during the first year in most patients. Thereafter, it declines to the 3 percentile line and remains low until 8 years of age. The peak height velocity is much lower during puberty in these patients than in the normal and contributes to short stature in achondroplasts. Extremely short stature lowers quality of life, therefore, various medications have been tried to promote growth in achondroplasts but in vain. GH is now produced in sufficient quantity utilizing recombinant DNA technique. Since encouraging results have been obtained in the GH treatment of various kinds of short stature without GH deficiency, e.g., Turner's syndrome, intrauterine growth retardation, non‐endocrine short stature, etc., achondroplasia has been also considered as a candidate for GH treatment. There have been only a few studies on GH treatment in achondroplasia and the number of subjects studied is small. However, satisfactory growth‐promoting effects have been demonstrated in these studies although observation periods were short and side effects have not been reported. Further studies are necessary to confirm the optimal dosage and administration method on final height and side effects in long‐term use of GH.

Short-term GH may promote growth in achondroplasia but should not change practice; leaves open need for long-term RCTs on final height and safety.