ESPE Abstracts (2022) 95 P1-363

ESPE2022 Poster Category 1 Pituitary, Neuroendocrinology and Puberty (77 abstracts)

Medium-and long-term follow-up observation of the efficacy and safety of GnRHa in the treatment of five cases of central precocious puberty caused by hypothalamic hamartoma

Siqi Huang , Zhe Meng , Zulin Liu , Lina Zhang , Lele Hou , Hui Ou & Liyang Liang

Pediatric department of Sun Yat-sen Memorial Hospital of Sun Yat-sen University, Guangzhou, China

Background: Hypothalamic hamartoma (HH) is the most common cause of organic central precocious puberty (CPP). Gonadotropin-releasing hormone analogue (GnRHa) is widely used in the treatment of HH combined with CPP, but there is no report on the medium-and long-term efficacy and safety of HH in China.

Objective: To investigate the medium-and long-term efficacy and safety of GnRHa in the treatment of children with CPP caused by HH.

Methods: Five children with CPP caused by HH were treated with GnRHa vary three to eight years. Tanner stage, sex hormone level, glucose and lipid metabolism, bone age and PAHtSDS were evaluated at baseline, during the follow-up period and after treatment.

Results: The initial age of breast enlargement or vaginal bleeding in girls was 0.25 to 5.67 years old, testicular enlargement in 1 male was 4.5 years old, whose Tanner stage was Ⅰto Ⅲ, age of first visit was 0.5 to 5.92 year-old, the level of sex hormone elevated at the onset. Besides, the younger the age of onset, the faster the progress of sexual development. After 3 months of GnRHa treatment, the breast development of 4 girls subsided, Tanner stage was B1 to B2, Two girls with vaginal bleeding did not experience vaginal bleeding after 2 months treatment. The testicular volume of the boy was smaller than before. Five patients were followed up regularly during the treatment. The bone age of four female was controllable during the treatment, △BA /△CA < 1, and the bone age of one man was poorly controlled during the treatment, △BA /△CA = 1-1.5. The PAHtSDS of five patients were improved. During the follow-up period, 5 children had no abnormal glucose and lipid metabolism, 1 female patient stopped GnRHa treatment at the bone age of 12. After stopping the drug, she began to develop breasts again. Menarche occurred one and a half years later, and her menstruation was regular. GV was 4-5.2 cm /y after stopping the drug.

Conclusion: The age of onset of CPP caused by HH is young, the level of sex hormone at onset elevated, and the bone age is significantly advanced. GnRHa treatment of precocious puberty caused by HH can improve PAHtSDS during the medium-and long-term follow-up period, there is no abnormality in glucose and lipid metabolism, the growth rate is normal after drug withdrawal, spontaneous menarche can occur, and the drug safety is good.

Key words: Hypothalamic Hamartoma, Central Precocious Puberty, GnRHa, Efficacy, Safety

Volume 95

60th Annual ESPE (ESPE 2022)

Rome, Italy
15 Sep 2022 - 17 Sep 2022

European Society for Paediatric Endocrinology 

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