Peripheral precocious puberty: clinical, diagnostic and therapeutical principles

Peripheral precocious puberty (PPP) is the result of the presence of precocious puberty due to the increase of sex steroids with no evidence of activation of the hypothalamic-pituitary-gonadal axis. It is much less common than central precocious puberty (CPP) and it is secondary to either genetic disorders or very heterogeneous acquired diseases. In recent years, molecular advances have made remarkable progress in understanding the pathophysiology of some of these disorders, most notably in McCune-Albright syndrome and testotoxicosis. In addition, new imaging techniques and better hormone assays have improved the early diagnosis of acquired disorders, particularly tumour disease causing PPP. Unfortunately, medical advances in the diagnosis of these disorders have not been reflected in the medical treatment of McCune-Albright syndrome and testotoxicosis. Despite having tried various treatment options in both disorders, the results are very disappointing, especially in patients with McCune-Albright syndrome. To our knowledge, this failure is based on the absence of well-designed clinical trials that include an adequate number of patients followed up until the end of their growth.