ESPE Yearbook of Paediatric Endocrinology

To read the full abstract: J Clin Endocrinol Metab. 2017 Jul ;102 (7):2356-2363

[Comments on 6.6, 6.7 and 6.8] These 3 papers deal with endocine pharmacological treatment of patients with GD.

The first article by Chew et al. (6.6), from Australia, systematically reviews different pharmacological treatments in adolescents with GD, including GnRH analogs, hormonal receptor blockers and estrogen and androgen administration. The aim was to identify evidence on physical, psychosocial, and cognitive effects of the endocrine treatments. Only a small number of published papers fulfilled the high quality inclusion criteria and they were all observational. Pediatric endocrinologists active in this field form a solid experience base to support the current therapies. However, there is yet only low-quality evidence suggesting that the endocrine treatments for GD adolescents can achieve their wanted physical effects. Further, there is yet no evidence concerning the psychosocial and cognitive effects of these treatments in GD patients.

The second paper by Mahfouda et al. (6.7) reviews puberty suppression treatment in children with GD with respect to published evidence for physical, psychological and cognitive effects. Again, the authors found only a few published papers meeting evidence forming quality criteria. It seems clear that treatment is reasonably, safe but its efficacy is unproven. No quality data on psychological effects and other aspects of the treatment was available.

The third paper by Hannema et al. (6.8) from the Netherlands focuses specifically on estrogen treatment in adolescent transgirls (biologically male with female gender identity). A relatively small number (n=28) were treated for 1 year or more with 17-beta estradiol 2 mg daily to induce puberty. A median serum concentration was 100 pM estradiol, although some patients had much lower levels. Efficacy was proven by recording breast development; >80% reached Tanner 4-5 after 3 years of treatment. An important message from this study is that the treatment was safe. Accordingly, the authors argue that there is no need to measure metabolic and organ specific blood chemistry parameters.

Taken together, and as stated repeatedly in this chapter, it is clear from these and other publications (e.g., Tack L et al.) that the evidence base for endocrine treatments used in GD is weak. The approaches taken in the 3 papers above are important to improve this situation.

Tack L, Heyse R, Craen M, Dhondt K, Bossche HV, Laridaen J, Cools M. Consecutive Cyproterone Acetate and Estradiol Treatment in Late-Pubertal Transgender Female Adolescents. J Sex Med. 2017;14(5):747-757.