10 research outputs found
Long-term safety and efficacy of Omnitrope\uae, 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\uae, a somatropin biosimilar to Genotropin\uae, 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\uae. Adverse events (AEs) are assessed in all Omnitrope\uae-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\uae was 28.1 \ub1 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\uae treatment was associated with improvements in both HSDS and HVSDS. Conclusions: Omnitrope\uae 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
Permanent Neonatal Diabetes Mellitus (PNDM) in a patient with R201H/KCNJ11 gene mutation: Unusual clinical course
KCNJ11 gene encodes Kir6.2, subunit of the β-cell ATP-dependent K-channel
(made up of 4 Kir6.2 and 4 sulfonylurea receptor 1 subunits); its heterozygous
activating mutation is a frequent cause of PNDM. Our 20y old pt was
the only child born from healthy unrelated parents (BW 2.3Kg, L 48cm). High
plasma glucose (PG) (10-16.7mmol/l) and glycosuria (no ketonuria) appeared
at 2d of life reaching 33.3mmol/l at 12d. Despite the progressive PG increase
and low fasting C-peptide (Cp) (149pmol/l at 12d, nv 178-680), she showed
good health conditions and regular weight gain until 35d old. Insulin (Ins) (1
IU/Kg/d) was started at 37d of life, because of ketonuria and failure to thrive
(PG 32mmol/l). Ins/Kg/d, progressively reduced, was stopped at 2.5y because
of good metabolic control and frequent hypoglycaemic events. Cp decreased
from 149pmol/l (at 12d) to undetectable value (8.5m old) with a subsequent
increase (119pmol/l, at 20m). At diagnosis anti-Ins and anti-β-cell antibodies
(AA) were negative. OGTT at 2.9y: normal fasting PG (4.5mmol/l) and Ins
(47.3pmol/l), impaired GT (8.4mmol/l at 120‘), blunted Ins peak (87.5pmol/
l). HbA1c constantly <7%. At 4.1y and 7.5y: PG at 120‘ 7.2 and 11.7mmol/l,
respectively. At 8y, Ins therapy was restored (0.8 U/kg/d) having high HbA1c
values (10.2-11.5%) and anti-gliadin AA were detected; asymptomatic celiac
disease was confirmed by biopsy. At 19.7y, IVGTT showed low basal
(231pmol/l, nv 281-1317pmol/l) and peak (265pmol/l) Cp. A transition G->A
at position 602, with a His->Arg at codon 201 (R201H), was identified in the KCNJ11 gene (mutation absent in parents). Ins was progressively reduced and
stopped within 11d and glibenclamide (Gl) administered (increasing dose:
after 3m 0.39mg/Kg/d, no HbA1c increase). After 4w on Gl, Cp raised to
1158pmol/l. This mutation is the most frequent genetic defect associated with
PNDM: no one of 9 described pts currently older than 3y experienced honey
moon, 8/9 being constantly Ins-treated (1 adult pt on tolbutamide since childhood).
Our data indicate that this mutation can confer a broad range of clinical
presentation and prompts the KCNJ11 gene analysis in all cases of ND
Low Bone Mineral Density is Associated to Poor Glycemic Control and Increased Dickkopf-1 (DKK-1) Serum Levels in Children and Adolescents with Type 1 Diabetes
Raccomandazioni per la gestione dell’esercizio fisico nel diabete tipo 1 in età pediatrica - Gruppo di Studio di Diabetologia Pediatrica S.I.E.D.P.
Low prevalence of HNF1A mutations after molecular screening of multiple MODY genes in 58 Italian families recruited in the pediatric or adult diabetes clinic from a single Italian hospital
Si può “Valere” di più: un progetto italiano studia i disturbi alimentari negli adolescenti con diabete tipo 1. Dati preliminari
High frequency of diabetic ketoacidosis at diagnosis of type 1 diabetes in Italian children: A nationwide longitudinal study, 2004-2013
This longitudinal population-based study analyses the frequency of diabetic ketoacidosis (DKA) at type 1 diabetes diagnosis in Italian children under 15 years of age, during 2004–2013. DKA was defined
as absent (pH ≥ 7.30), mild/moderate (7.1 ≤ pH < 7.30) and severe (pH < 7.1). Two multiple logistic regression models were used to evaluate the time trend of DKA frequency considered as present versus
absent and severe versus absent, adjusted for gender, age group and geographical area of residence at diagnosis. Overall, 9,040 cases were ascertained. DKA frequency was 40.3% (95%CI: 39.3–41.4%), with
29.1% and 11.2% for mild/moderate and severe DKA, respectively. Severe DKA increased significantly during the period (OR = 1.03, 95%CI: 1.003–1.05). Younger-age children and children living in Southern
Italy compared to Central Italy were at significantly higher risk of DKA and severe DKA. Family history of type 1 diabetes and residence in Sardinia compared to Central Italy were significantly associated with a
lower probability of DKA and severe DKA. The high frequency of ketoacidosis in Italy over time and high variability among age groups and geographical area of residence, strongly suggests a continuing need
for nationwide healthcare strategies to increase awareness of early detection of diabetes
Emerging effects of early environmental factors over genetic background for type 1 diabetes susceptibility: Evidence from a nationwide Italian twin study
Identification of candidate children for maturity-onset diabetes of the young type 2 (MODY2) gene testing: A seven-item clinical flowchart (7-iF)
Recommendations for self-monitoring in pediatric diabetes: A consensus statement by the ISPED
none165noScaramuzza, Andrea; Cherubini, Valentino; Tumini, Stefano; Bonfanti, Riccardo; Buono, Pietro; Cardella, Francesca; D’Annunzio, Giuseppe; Frongia, Anna Paola; Lombardo, Fortunato; Monciotti, Anna Carla Maria; Rabbone, Ivana; Schiaffini, Riccardo; Toni, Sonia; Zucchini, Stefano; Frontino, Giulio; Iafusco, Dario; Arnaldi, Claudia; Banin, Patrizia; Barbetti, Fabrizio; Beccaria, Luciano; Benelli, Marzia; Berardi, Rosario; Biagioni, Martina; Bianchi, Giuliana; Bizzarri, Carla; Blasetti, Annalisa; Bobbio, Adriana; Boccato, Stefano; Bontempi, Franco; Bruzzese, Mariella; Cadario, Francesco; Calcaterra, Valeria; Cannatà, Alessandra; Cappa, Marco; Cardani, Roberta; Cardinale, Giuliana Marcella; Carloni, Ines; Castaldo, Vincenzo; Cauvin, Vittoria; Cerutti, Franco; Cester, Anna Maria; Chessa, Margherita; Chiarelli, Francesco; Chiari, Giovanni; Chiumello, Giuseppe; Cicchetti, Mario; Cirillo, Dante; Citriniti, Felice; Citro, Giuseppe; Coccioli, Maria Susanna; Cotellessa, Mario; Crinò, Antonino; De Berardinis, Fiorella; De Filippo, Gianpaolo; De Giorgi, Giovanni; De Luca, Filippo; De Marco, Rosaria; Delvecchio, Maurizio; Faleschini, Elena; Federico, Giovanni; Fifi, Anna Rita; Fontana, Franco; Franzese, Adriana; Frezza, Elda; Frongia, Annapaola; Gaiero, Alberto; Galderisi, Alfonso; Gallo, Francesco; Gargantini, Luigi; Ghione, Silvia; Giorgetti, Chiara; Gualtieri, Antonella; Guasti, Monica; Guerraggio, Lucia; Iannilli, Antonio; Ingletto, Dario; Iossa, Carmine; Iovene, Brunella; Iughetti, Lorenzo; Kaufmann, Peter; La Loggia, Alfonso; Lazzaro, Nicola; Lenzi, Lorenzo; Lera, Riccardo; Lia, Rosanna; Lo Presti, Donatella; Lorini, Renata; Lucchesi, Sonia; Luceri, Sergio; Madeo, Simona Filomena; Maffeis, Claudio; Mainetti, Benedetta; Mammi, Francesco; Manca Bitti, Maria Luisa; Marigliano, Marco; Marinari, Alessandra; Marinaro, Anna Maria; Meloni, Gianfranco; Marsciani, Alberto; Mastrangelo, Lisa; Mastrangelo, Costanzo; Meschi, Franco; Minasi, Domenico; Minenna, Adelaide; Minuto, Nicola; Monciotti, Carlamaria; Morganti, Gianfranco; Mozzillo, Enza; Nugnes, Rosa; Paradiso, Emanuela; Pardi, Daniela; Pasquino, Bruno; Patrizia Patera, Ippolita; Pennati, Cristina; Pepe, Rossella; Piccini, Barbara; Perrotta, Angelo; Piccinno, Elvira; Pinelli, Leonardo; Piredda, Gavina; Pocecco, Mauro; Ponzi, Giuseppe; Prandi, Elena; Predieri, Barbara; Prisco, Francesco; Quinci, Maria; Ricciardi, Maria Rossella; Rigamonti, Andrea; Ripoli, Carlo; Sabbion, Alberto; Salardi, Silvana; Salvatoni, Alessandro; Salvo, Caterina; Salzano, Giuseppina; Saporiti, Anna; Sardi, Rita; Schieven, Eleonardo; Scipione, Mirella; Soci, Cristina; Soro, Miriam; Spallino, Luisa; Stamati, Filomena; Suprani, Tosca; Savastio, Silvia; Taccardi, Rosa Anna; Tarchini, Luis; Tomaselli, Letizia; Tonini, Giorgio; Torelli, Cataldo; Tornese, Gianluca; Trada, Michela; Valerio, Giuliana; Vanelli, Maurizio; Vanini, Roberto; Vascotto, Marina; Vergerio, Amedeo; Viscardi, Matteo; Zaffani, Silvana; Zampolli, Maria; Zanatta, Manuela; Zanette, Giorgio; Zanfardino, Angela; Zecchino, Clara; Zedda, Maria Antonietta; Zuccotti, Gian VincenzoScaramuzza, Andrea; Cherubini, Valentino; Tumini, Stefano; Bonfanti, Riccardo; Buono, Pietro; Cardella, Francesca; D’Annunzio, Giuseppe; Frongia, Anna Paola; Lombardo, Fortunato; Monciotti, Anna Carla Maria; Rabbone, Ivana; Schiaffini, Riccardo; Toni, Sonia; Zucchini, Stefano; Frontino, Giulio; Iafusco, Dario; Arnaldi, Claudia; Banin, Patrizia; Barbetti, Fabrizio; Beccaria, Luciano; Benelli, Marzia; Berardi, Rossana; Biagioni, Martina; Bianchi, Giuliana; Bizzarri, Carla; Blasetti, Annalisa; Bobbio, Adriana; Boccato, Stefano; Bontempi, Franco; Bruzzese, Mariella; Cadario, Francesco; Calcaterra, Valeria; Cannatà, Alessandra; Cappa, Marco; Cardani, Roberta; Cardinale, Giuliana Marcella; Carloni, Ines; Castaldo, Vincenzo; Cauvin, Vittoria; Cerutti, Franco; Cester, Anna Maria; Chessa, Margherita; Chiarelli, Francesco; Chiari, Giovanni; Chiumello, Giuseppe; Cicchetti, Mario; Cirillo, Dante; Citriniti, Felice; Citro, Giuseppe; Coccioli, Maria Susanna; Cotellessa, Mario; Crinò, Antonino; De Berardinis, Fiorella; De Filippo, Gianpaolo; De Giorgi, Giovanni; De Luca, Filippo; De Marco, Rosaria; Delvecchio, Maurizio; Faleschini, Elena; Federico, Giovanni; Fifi, Anna Rita; Fontana, Franco; Franzese, Adriana; Frezza, Elda; Frongia, Annapaola; Gaiero, Alberto; Galderisi, Alfonso; Gallo, Francesco; Gargantini, Luigi; Ghione, Silvia; Giorgetti, Chiara; Gualtieri, Antonella; Guasti, Monica; Guerraggio, Lucia; Iannilli, Antonio; Ingletto, Dario; Iossa, Carmine; Iovene, Brunella; Iughetti, Lorenzo; Kaufmann, Peter; La Loggia, Alfonso; Lazzaro, Nicola; Lenzi, Lorenzo; Lera, Riccardo; Lia, Rosanna; Lo Presti, Donatella; Lorini, Renata; Lucchesi, Sonia; Luceri, Sergio; Madeo, Simona Filomena; Maffeis, Claudio; Mainetti, Benedetta; Mammi, Francesco; Manca Bitti, Maria Luisa; Marigliano, Marco; Marinari, Alessandra; Marinaro, Anna Maria; Meloni, Gianfranco; Marsciani, Alberto; Mastrangelo, Lisa; Mastrangelo, Costanzo; Meschi, Franco; Minasi, Domenico; Minenna, Adelaide; Minuto, Nicola; Monciotti, Carlamaria; Morganti, Gianfranco; Mozzillo, Enza; Nugnes, Rosa; Paradiso, Emanuela; Pardi, Daniela; Pasquino, Bruno; Patrizia Patera, Ippolita; Pennati, Cristina; Pepe, Rossella; Piccini, Barbara; Perrotta, Angelo; Piccinno, Elvira; Pinelli, Leonardo; Piredda, Gavina; Pocecco, Mauro; Ponzi, Giuseppe; Prandi, Elena; Predieri, Barbara; Prisco, Francesco; Quinci, Maria; Ricciardi, Maria Rossella; Rigamonti, Andrea; Ripoli, Carlo; Sabbion, Alberto; Salardi, Silvana; Salvatoni, Alessandro; Salvo, Caterina; Salzano, Giuseppina; Saporiti, Anna; Sardi, Rita; Schieven, Eleonardo; Scipione, Mirella; Soci, Cristina; Soro, Miriam; Spallino, Luisa; Stamati, Filomena; Suprani, Tosca; Savastio, Silvia; Taccardi, Rosa Anna; Tarchini, Luis; Tomaselli, Letizia; Tonini, Giorgio; Torelli, Cataldo; Tornese, Gianluca; Trada, Michela; Valerio, Giuliana; Vanelli, Maurizio; Vanini, Roberto; Vascotto, Marina; Vergerio, Amedeo; Viscardi, Matteo; Zaffani, Silvana; Zampolli, Maria; Zanatta, Manuela; Zanette, Giorgio; Zanfardino, Angela; Zecchino, Clara; Zedda, Maria Antonietta; Zuccotti, Gian Vincenz