Background: PATRO Children is an ongoing observational, longitudinal, non-interventional, global post-marketing\nsurveillance study, which is investigating the long-term safety and effectiveness of Omnitrope�®, a somatropin\nbiosimilar to Genotropin�®, in children with growth disturbances. The primary endpoint of PATRO Children is long-term\nsafety and the secondary endpoint is effectiveness, which is assessed by analysing auxological data such as height\n(HSDS) and height velocity (HVSDS) standard deviation scores. Here, we report the data from the Italian interim analysis\nof PATRO Children data up to August 2015.\nMethods: PATRO Children is enrolling children who are diagnosed with conditions of short stature requiring GH\ntreatment and are receiving Omnitrope�®. Adverse events (AEs) are assessed in all Omnitrope�®-treated patients.\nHeight is evaluated yearly to near-adult (final) height, and is herein reported as HSDS; height velocity is also\nassessed and reported as a standard deviation score (HVSDS).\nResults: Up to August 2015, a total of 186 patients (mean age 10.2 years, 57.5 % males) were enrolled :156 [84 %]\nhad growth hormone deficiency, 12 [6.5 %] were born small for gestational age, seven [3.8 %] had Prader-Willi\nsyndrome, one [0.5 %] had Turner syndrome and one [0.5 %] had chronic renal insufficiency; seven [3.8 %] patients had\nother indication profiles. The mean treatment duration with Omnitrope�® was 28.1 �± 19.1 months. AEs were reported in\n35.6 % of patients and included headache, pyrexia, arthralgia, abdominal pain, leg and/or arm pain and increased\nblood creatine phosphokinase. Two serious AEs in two patients were thought to be drug-related; one patient\nexperienced a minimal increase in a known residual craniopharyngioma, and another a gait disturbance with\nworsening of walking difficulties. Similar to investigational studies, Omnitrope�® treatment was associated with\nimprovements in both HSDS and HVSDS.\nConclusions: Omnitrope�® appears to be well tolerated and effective for the treatment of a wide range of paediatric\nindications, which is consistent with the outcomes from controlled clinical trials. These results need to be interpreted\nwith caution until the data from the global PATRO Children study are available.
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