Objectives: To describe safety and effectiveness of high (≥90-180 µg/kg) and low (<90-30 µg/kg) dose leuprorelin in treating central precocious puberty (CPP).
Methods: In this observational, retrospective study, effectiveness was evaluated based on regression or no progression of Tanner staging as the primary outcomes. LH, FSH, estradiol or testosterone suppression, and decrease in bone age to chronological age (BA/CA) ratio were secondary outcomes. Data from medical records were organized into a treatment phase in which subjects were treated with at least 9 continuous months of leuprorelin (Enantone) and a follow-up phase up to the day of last follow-up data for the subject.
Results: During the treatment phase, all subjects (104 females and 4 males) were treated with Enantone. The mean exposure time was 22.3 months and 97.2% subjects received the maximum dose of 3.75 mg. 69.4% subjects recorded AEs, the most common being upper respiratory tract infection. No SAEs were related to Enantone. One male had sufficient data for evaluation and showed progression of Tanner stage; 79.7% (63/79) females showed regression or no progression. LH and FSH peak levels were suppressed in 98.1% (52/53) and 100% (53/53) while suppression of estradiol occurred in 74.8% (77/103) of females and testosterone was suppressed in 100% (4/4) of males. 89.6% (86/96) of subjects had decrease from baseline in the BA/CA ratio at the end of the Enantone treatment phase. In the follow-up phase, 44 (62.9%) subjects were no longer on CPP treatment had a mean follow-up duration of 8.75 months; another 26 (37.1%) subjects were continuing their CPP treatment with another GnRH agonist. There were 10 AEs reported, none of which were related to Enantone. Of the 2 female subjects who had sufficient data for evaluations of Tanner staging, the percentage of regression or no progression was 50.0% (1/2) at month 9 and 0% (0/2) at month 12. There was insufficient data for this evaluation in male subjects. For subjects who were no longer receiving CPP treatment, only observations on estradiol suppression in 1 female 9 months post-treatment and BA/CA ratio in 3/4 females at 9 months post-treatment and in 2/3 females at 12 months after treatment were available.
Conclusions: The safety profile observed was consistent with the known profile of leuprorelin and no concerns were identified regarding long term administration. The majority of subjects received a maximum dose of 3.75 mg. Enantone appeared to have been effective in the treatment of CPP.
22 Sep 2021 - 26 Sep 2021