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Licensed Unlicensed Requires Authentication Published by De Gruyter February 17, 2016

Efficacy and safety of triptorelin 6-month formulation in patients with central precocious puberty

  • Karen Klein EMAIL logo , Joshua Yang , Javier Aisenberg , Nancy Wright , Paul Kaplowitz , Najiba Lahlou , Jeannete Linares , Eija Lundström , Daniela Purcea and Fernando Cassorla

Abstract

Background:

Triptorelin is an established treatment for central precocious puberty (CPP) as 1- and 3-month formulations. The current triptorelin 22.5 mg 6-month formulation is approved for prostate cancer therapy. This is the first study in patients with CPP.

Methods:

The efficacy and safety of the triptorelin 6-month formulation in CPP were investigated. The primary objective was to evaluate the efficacy in achieving luteinizing hormone (LH) suppression to pre-pubertal levels at month 6. This was an international, non-comparative phase III study over 48 weeks. Eighteen medical centers in the US, Chile and Mexico participated. Forty-four treatment naïve patients (39 girls and five boys) aged at treatment start 2–8 years for girls and 2–9 years for boys with an advancement of bone age over chronological age ≥1 year were to be included. Triptorelin was administered im twice at an interval of 24 weeks. LH, follicle stimulating hormone (FSH) (basal and stimulated), estradiol (girls), testosterone (boys), auxological parameters, clinical signs of puberty and safety were assessed.

Results:

Forty-one patients (93.2%) showed pre-pubertal LH levels (stimulated LH ≤5 IU/L) at month 6 and maintained LH suppression through month 12. The percentage of patients with LH suppression exceeded 93% at each time point and reached 97.7% at month 12. No unexpected drug-related adverse events were reported.

Conclusions:

The triptorelin 6-month formulation was safe and effective in suppressing the pituitary-gonadal axis in children with CPP. The extended injection interval may improve compliance and increase comfort in the management of CPP.


Corresponding author: Karen Klein, MD, University of California and Rady Children’s Hospital, 3020 Children’s Way, MC 5103 San Diego, CA 92123, USA, Phone: +858-966-4032

Acknowledgments

The authors thank the children and their families for participation in the study and the study coordinators for the conduct of the study. The authors also thank the following investigators for their participation: Tala Dajani, MD, Oskar Flores-Caloca, MD, Gad Kletter, MD, Susan Rose, MD, Kathleen Bethin, MD, David Domek, MD, Diane Merritt, MD, Quentin Van Meter, MD, Heidi Shea, MD, Lawrence Silverman, MD, Opada Alzohaili, MD, and Ricardo Gomez, MD.

  1. Author contributions: All the authors have accepted responsibility for the entire content of this submitted manuscript and approved submission.

  2. Research funding: Clinical Trial Registration Number: NCT01467882.

  3. Employment or leadership: JL, FC, NW, PK have nothing to declare. KK is a consultant for and serves on the speaker bureau for Abbvie Pharmaceuticals. JA serves on a speaker bureau for Abbvie, and Novo Nordisk. EL and DP are employees of Debiopharm International S.A.

  4. Honorarium: None declared.

  5. Competing interests: The funding organization(s) played a role in the study design; in the collection, analysis, and interpretation of data; in the writing of the report; or in the decision to submit the report for publication.

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Received: 2015-9-16
Accepted: 2015-12-14
Published Online: 2016-2-17
Published in Print: 2016-11-1

©2016 Walter de Gruyter GmbH, Berlin/Boston

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