Safety Outcomes and Near-Adult Height Gain of Growth Hormone-Treated Children with SHOX Deficiency: Data from an Observational Study and a Clinical Trial

Benabbad, Imane
Rosilio, Myriam
Child, Christopher J.
Carel, Jean-Claude
Ross, Judith L.
Deal, Cheri L.
Drop, Stenvert L.S.
Zimmermann, Alan G.
Jia, Nan
Quigley, Charmian A.
Blum, Werner F.

^aEndocrinology and Diabetes Unit, Eli Lilly, Neuilly-sur-Seine, France; ^bLilly Research Laboratories, Windlesham, UK; ^cDepartment of Pediatric Endocrinology and Diabetology, and INSERM U690, University Paris 7, Denis Diderot, Hôpital Robert Debré, Paris, France; ^dDepartment of Pediatrics, Thomas Jefferson University, Philadelphia, PA, and ^eDuPont Hospital for Children, Wilmington, DE, USA; ^fUniversity of Montreal and CHU Ste-Justine, Montreal, QC, Canada; ^gDepartment of Pediatrics, Erasmus Medical Centre Sophia, Rotterdam, The Netherlands; ^hGlobal Statistical Sciences, Eli Lilly and Company, and ⁱIndiana University School of Medicine, Indianapolis, IN, USA; ^jUniversity Children's Hospital, University of Giessen, Giessen, Germany

^*Imane Benabbad, Endocrinology and Diabetes Unit, Eli Lilly, 24, Boulevard Vital Bouhot, FR-92521 Neuilly-sur-Seine (France), E-Mail [email protected]

Received: September 5, 2016; Accepted: October 27, 2016; Published online: December 22, 2016

Hormone Research in Paediatrics 87(1):p 42-50, February 2017. | DOI: 10.1159/000452973

Abstract

Background/Aims:

To assess auxological and safety data for growth hormone (GH)-treated children with SHOX deficiency.

Methods:

Data were examined for GH-treated SHOX-deficient children (n = 521) from the observational Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS). For patients with near-adult height information, GeNeSIS results (n = 90) were compared with a clinical trial (n = 28) of SHOX-deficient patients. Near-adult height was expressed as standard deviation score (SDS) for chronological age, potentially increasing the observed effect of treatment.

Results:

Most SHOX-deficient patients in GeNeSIS had diagnoses of Leri-Weill syndrome (n = 292) or non-syndromic short stature (n = 228). For GeNeSIS patients with near-adult height data, mean age at GH treatment start was 11.0 years, treatment duration 4.4 years, and height SDS gain 0.83 (95% confidence interval 0.49-1.17). Respective ages, GH treatment durations and height SDS gains for GeNeSIS patients prepubertal at baseline (n = 42) were 9.2 years, 6.0 years and 1.19 (0.76-1.62), and for the clinical trial cohort they were 9.2 years, 6.0 years and 1.25 (0.92-1.58). No new GH-related safety concerns were identified.

Conclusion:

Patients with SHOX deficiency who had started GH treatment before puberty in routine clinical practice had a similar height gain to that of patients in the clinical trial on which approval for the indication was based, with no new safety concerns.