Long-term safety and efficacy of Omnitrope®, a somatropin biosimilar, in children requiring growth hormone treatment: Italian interim analysis of the PATRO Children study.
BACKGROUND
PATRO Children is an ongoing observational, longitudinal, non-interventional, global post-marketing surveillance study, which is investigating the long-term safety and effectiveness of Omnitrope®, a somatropin biosimilar to Genotropin®, in children with growth disturbances. The primary endpoint of PATRO Children is long-term safety and the secondary endpoint is effectiveness, which is assessed by analysing auxological data such as height (HSDS) and height velocity (HVSDS) standard deviation scores. Here, we report the data from the Italian interim analysis of PATRO Children data up to August 2015.


METHODS
PATRO Children is enrolling children who are diagnosed with conditions of short stature requiring GH treatment and are receiving Omnitrope®. Adverse events (AEs) are assessed in all Omnitrope®-treated patients. Height is evaluated yearly to near-adult (final) height, and is herein reported as HSDS; height velocity is also assessed and reported as a standard deviation score (HVSDS).


RESULTS
Up to August 2015, a total of 186 patients (mean age 10.2 years, 57.5 % males) were enrolled :156 [84 %] had growth hormone deficiency, 12 [6.5 %] were born small for gestational age, seven [3.8 %] had Prader-Willi syndrome, one [0.5 %] had Turner syndrome and one [0.5 %] had chronic renal insufficiency; seven [3.8 %] patients had other indication profiles. The mean treatment duration with Omnitrope® was 28.1 ± 19.1 months. AEs were reported in 35.6 % of patients and included headache, pyrexia, arthralgia, abdominal pain, leg and/or arm pain and increased blood creatine phosphokinase. Two serious AEs in two patients were thought to be drug-related; one patient experienced a minimal increase in a known residual craniopharyngioma, and another a gait disturbance with worsening of walking difficulties. Similar to investigational studies, Omnitrope® treatment was associated with improvements in both HSDS and HVSDS.


CONCLUSIONS
Omnitrope® appears to be well tolerated and effective for the treatment of a wide range of paediatric indications, which is consistent with the outcomes from controlled clinical trials. These results need to be interpreted with caution until the data from the global PATRO Children study are available.
Pediatric Unit, Department of Medical and Surgical Sciences for Mother, Children and Adults, University of Modena and Reggio Emilia, Via del Pozzo, 41124, Modena, Italy. lorenzo.iughetti@unimore.it.
Lorenzo
L
Iughetti
Institute for Maternal and Child Health IRCCS Burlo Garofolo, Trieste, Italy.
Gianluca
G
Tornese
Department of Paediatrics- Arcispedale S. Maria Nuova-IRCCS, Reggio Emilia, Italy.
Maria Elisabeth
ME
Street
Pediatric Unit, Istituto Giannina Gaslini, Genova, Italy.
Flavia
F
Napoli
Endocrinology and Metabolic Diseases Unit, Department of Clinical Sciences and Community Health, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Milano, Italy.
Claudia
C
Giavoli
Pediatric Unit, Policlinico Giambattista Rossi, University of Verona, Verona, Italy.
Franco
F
Antoniazzi
Health Science Department, Anna Meyer Children's University Hospital, University of Florence, Firenze, Italy.
Stefano
S
Stagi
Department of Woman, Child and General and Specialized Surgery, Seconda Università degli Studi di Napoli, Napoli, Italy.
Caterina
C
Luongo
Pediatric Endocrinology Unit, Department of Woman's and Child's Health, University Hospital of Padua, Padova, Italy.
Sara
S
Azzolini
UOC Pediatria, I.R.C.S.S. Oasi Maria SS, Troina, Italy.
Letizia
L
Ragusa
Division of Paediatrics, Azienda Ospedaliero-Universitaria Maggiore della Carità, Novara, Italy.
Gianni
G
Bona
Department of Sciences and Pediatric Surgery, University of Bari "A. Moro", Bari, Italy.
Clara
C
Zecchino
Department of Human Pathology of Adulthood and Childhood, University of Messina, Messina, Italy.
Tommaso
T
Aversa
Department of Clinical Sciences and Community Health, University of Milan, Milano, Italy.|Division of Endocrine and Metabolic Diseases, IRCCS Istituto Auxologico Italiano, Milano, Italy.
Luca
L
Persani
Department of Paediatrics, Paediatric Endocrinology Unit, University of Milan, Milan, Italy.
Laura
L
Guazzarotti
Sandoz S.p.A, Milan, Italy.
Emiliano
E
Zecchi
Hexal AG, Holzkirchen, Germany.
Alberto
A
Pietropoli
Unit of Pediatric Endocrinology, S.Orsola-Malpighi Hospital, Bologna, Italy.
Stefano
S
Zucchini
D059451:Biosimilar Pharmaceuticals; D002648:Child; D023381:Endpoint Determination; D005260:Female; D006130:Growth Disorders / Q000188:drug therapy*; D019382:Human Growth Hormone / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D011358:Product Surveillance, Postmarketing; D016896:Treatment Outcome
D059451:Biosimilar Pharmaceuticals; D019382:Human Growth Hormone
Adolescents; Children; Infants; Omnitrope®; Paediatric; Recombinant human growth hormone