Ospedale Infantile Regina Margherita

Sant'Ambrogio di Torino, Italy

Ospedale Infantile Regina Margherita

Sant'Ambrogio di Torino, Italy
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de Girolamo L.,Applicate | Lucarelli E.,IRCCS Instituto Ortopedico Rizzoli | Alessandri G.,Fondazione IRCCS Instituto Neurologico Carlo Besta | Avanzini M.A.,Laboratori Immunologia e Trapianti | And 13 more authors.
Current Pharmaceutical Design | Year: 2013

Mesenchymal stem cells (MSCs) were first isolated more than 50 years ago from the bone marrow. Currently MSCs may also be isolated from several alternative sources and they have been used in more than a hundred clinical trials worldwide to treat a wide variety of diseases. The MSCs mechanism of action is undefined and currently under investigation. For in vivo purposes MSCs must be produced in compliance with good manufacturing practices and this has stimulated research on MSCs characterization and safety. The objective of this review is to describe recent developments regarding MSCs properties, physiological effects, delivery, clinical applications and possible side effects. © 2013 Bentham Science Publishers.

Marini D.,Ospedale Infantile Regina Margherita | Kenny D.,Rush University Medical Center
EuroIntervention | Year: 2012

Aims: The aim of this study was to assess the midterm results of percutaneous closure of very large atrial septal defects (ASD) in children with transthoracic echocardiography (TTE) and multislice computed tomography (MSCT). Methods and results: Among 142 children who underwent percutaneous ASD closure with the AMPLATZER® Septal Occluder (ASO) (AGA Medical Corporation, Plymouth, MN, USA) during an eight year period, 51 patients with very large defects, were evaluated by TTE and MSCT after a period of at least two years following ASD closure. Median age at ASD closure was six years (range 4-10), with mean ASD size 20.9±2.9 mm. Median device size was 20 mm (range 15-26) and median device:septal length ratio 0.95 (range 0.8-1). Early complications included one transient complete atrioventricular block and one device embolisation. At a median follow-up of 55 months (range 25-92) all patients were clinically asymptomatic and had a normal ECG. TTE did not demonstrate device protrusion across the lumen of either the systemic or pulmonary veins. The mean device:septal length ratio had decreased from 0.96±0.05 to 0.8±0.02 (p<0.001). There was good correlation between the measure of atrial septum length by TTE and MSCT (r: 0.79, p<0.001). MSCT identified moderate dynamic device protrusion into the lumen of systemic or pulmonary veins in five patients and partial device malpositioning in two patients. Conclusions: Occlusion of very large ASD in children can be performed with low complications rate. MSCT provides detailed information regarding the location of the device with respect to surrounding anatomic structures and reveals anomalies not evident by TTE. © Europa Edition 2012. All rights reserved.

PubMed | Ospedale SantAntonio Abate, Ospedale di Bolzano., Ospedale Salesi, Ospedale Garibaldi and 47 more.
Type: Journal Article | Journal: Journal of pediatric surgery | Year: 2015

Our study aims at disclosing epidemiology and most relevant clinical features of esophageal atresia (EA) pointing to a model of multicentre collaboration.A detailed questionnaire was sent to all Italian Units of pediatric surgery in order to collect data of patients born with EA between January and December 2012. The results were crosschecked by matching date and place of birth of the patients with those of diagnosis-related group provided by the Italian Ministry of Health (MOH).A total of 146 questionnaires were returned plus a further 32 patients reported in the MOH database. Basing on a total of 178 patients with EA born in Italy in 2012, the incidence of EA was calculated in 3.33 per 10,000 live births. Antenatal diagnosis was suspected in 29.5% patients. 55.5% showed associated anomalies. The most common type of EA was Gross type C (89%). Postoperative complications occurred in 37% of type C EA and 100% of type A EA. A 9.5% mortality rate was reported.This is the first Italian cross-sectional nationwide survey on EA. We can now develop shared guidelines and provide more reliable prognostic expectations for our patients.

Conter V.,University of Milan Bicocca | Bartram C.R.,University of Heidelberg | Valsecchi M.G.,University of Milan Bicocca | Schrauder A.,University of Kiel | And 23 more authors.
Blood | Year: 2010

The Associazione Italiana di Ematologia Oncologia Pediatrica and the Berlin-Frankfurt-Münster Acute Lymphoblastic Leukemia (AIEOP-BFM ALL 2000) study has for the first time introduced standardized quantitative assessment of minimal residual disease (MRD) based on immunoglobulin and T-cell receptor gene rearrangements as polymerase chain reaction targets (PCR-MRD), at 2 time points (TPs), to stratify patients in a large prospective study. Patients with precursor B (pB) ALL (n = 3184) were considered MRD standard risk (MRD-SR) if MRD was already negative at day 33 (analyzed by 2 markers, with a sensitivity of at least 10-4); MRD high risk (MRD-HR) if 10-3 or more at day 78 and MRD intermediate risk (MRD-IR): others. MRD-SR patients were 42% (1348): 5-year event-free survival (EFS, standard error) is 92.3% (0.9). Fifty-two percent (1647) were MRD-IR: EFS 77.6% (1.3). Six percent of patients (189) were MRD-HR: EFS 50.1% (4.1; P < .001). PCR-MRD discriminated prognosis even on top of white blood cell count, age, early response to prednisone, and genotype. MRD response detected by sensitive quantitative PCR at 2 predefined TPs is highly predictive for relapse in childhood pB-ALL. The study is registered at http://clinicaltrials.gov: NCT00430118 for BFM and NCT00613457 for AIEOP. (Blood. 2010;115(16):3206-3214) © 2010 by The American Society of Hematology.

PubMed | Ospedale A.Segni, Territorial Unit of Nephrology and Dialysis, Ospedale SS. Trinita, Ospedale N.S. della Mercede and 12 more.
Type: Journal Article | Journal: BMC nephrology | Year: 2016

Hemodiafiltration with on-line endogenous reinfusion (HFR) is an extracorporeal dialytic method that combines diffusion, convection and adsorption. HFR-Supra (HFR-S) is a second-generation system with increased convective permeability and adsorption capability. Previous studies suggested that HFR reduces oxidative stress compared to standard haemodialysis. The principal aim of the present study was to compare antioxidant vitamins behavior and oxidative status of hemodialysis patients treated with HFR and HFR-S.The study was designed as a multicenter, randomized, crossover trial. Forty-one patients were recruited from 19 dialysis centers and after a 4-month washout stabilization period in on-line hemodiafiltration (ol-HDF), each patient was randomized to a sequence of treatments (HFR-S followed by HFR or viceversa) with each treatment applied over 6months. Plasma levels of Advanced Oxidation Protein Products, Total Antioxidant Status, vitamins C, A and E and their ligands (Retinol Binding Protein and total lipids) were measured at baseline and at the end of each treatment period.Results show that the higher convective permeability of HFR-S with respect to HFR did not produce additional beneficial effects on the patients oxidative status, a slight decrease of both Vitamin A and Retinol Binding Protein being the only difference registered in the long-term. However, as compared to ol-HDF, both the re-infusive techniques allowed to reduce the intradialytic loss of Vitamin C and, in the long-term, improve the patients oxidative status and increase Retinol Binding Protein plasma values. No significant differences were found between the Vitamin C concentration of pre- and post cartridge UF neither in HFR-S nor in HFR showing that the sorbent resin does not adsorb Vitamin C.HFR-S and HFR are almost equivalent in term of impact on antioxidant vitamins and oxidative status of hemodialysis patients. Nonetheless, as compared to ol-HDF, both treatments produced a sensible sparing of Vitamin C and may represent a new approach for reducing oxidative stress and related complications in dialysis patients. Long-term effects of re-infusive treatments on patients cardiovascular morbidity and mortality need to be evaluated.ClinicalTrials.gov Identifier NCT01492491 , retrospectively registered in 10 December 2011.

Grignani G.,Institute for Cancer Research and Treatment | Palmerini E.,Chemotherapy Unit | Dileo P.,Fondazione IRCCS Instituto Nazionale dei Tumori | Asaftei S.D.,Ospedale Infantile Regina Margherita | And 8 more authors.
Annals of Oncology | Year: 2012

Purpose: After standard multimodal therapy, the prognosis of relapsed and unresectable high-grade osteosarcoma is dismal and unchanged over the last decades. Recently, mitogen-activated protein kinases were shown to be activated in osteosarcoma specimens, suggesting, therefore, they are suitable targets for the multikinase inhibitor sorafenib. Thus, we explored sorafenib activity in patients with relapsed and unresectable osteosarcoma. Experimental design: Patients >14 years, progressing after standard treatment, were eligible to receive 400 mg of sorafenib twice daily until progression or unacceptable toxicity. The primary end point was progression-free survival (PFS) at 4 months. Secondary objectives were PFS, overall survival (OS), clinical benefit rate (CBR), defined as no progression at 6 months and safety. This nonrandomized phase II study used a Simon two-stage design. PFS and OS at 95% confidence intervals (95% CIs) were calculated by the Kaplan-Meier method. All tests were two sided. Results: Thirty-five patients were enrolled. PFS at 4 months was 46% (95% CI 28% to 63%). Median PFS and OS were 4 (95% CI 2-5) and 7 (95% CI 7-8) months, respectively. The CBR was 29% (95% CI 13% to 44%). We observed 3 (8%) partial responses (PRs), 2 (6%) minor responses (<30% tumor shrinkage) and 12 (34%) stable diseases (SDs). For six patients (17%), PR/SD lasted ≥6 months. Noteworthy, tumor density reduction and [ 18F]2-fluoro-2-deoxy-D-glucose-positron emission tomography responses were observed among SD patients. Sorafenib was reduced or briefly interrupted in 16 (46%) patients and permanently discontinued in one (3%) case due to toxicity. Conclusions: Sorafenib demonstrated activity as a second- or third-line treatment in terms of PFS at 4 months with some unprecedented long-lasting responses. Sorafenib, the first targeted therapy showing activity in osteosarcoma patients, deserves further investigations. © The Author 2011. Published by Oxford University Press on behalf of the European Society for Medical Oncology. All rights reserved.

Savino F.,Ospedale Infantile Regina Margherita | Roana J.,University of Turin | Mandras N.,University of Turin | Tarasco V.,Ospedale Infantile Regina Margherita | And 2 more authors.
Acta Paediatrica, International Journal of Paediatrics | Year: 2011

Aim: To evaluate modifications of gut microbiota after antibiotic therapy in breast-fed infants. Study design: Twenty-six exclusively breast-fed infants younger than 5 months hospitalized for pneumonia treated with ceftriaxone (50 mg per kilo per day administered intramuscularly) were recruited. Intestinal microbiota at day 0 - before starting antibiotic administration - at the end of the therapy (5 days after) and after 15 days after the stop was analysed. Stool samples were collected and immediately diluted and cultured on selective media to detect total bacteria, Enterobacteriaceae, enterococci and lactobacilli. Statistical analysis was performed by using Wilcoxon test. Results: After 5 days of antibiotic therapy, we observed a significant reduction in total faecal bacterial count (p = 0.003) in Enterobacteriaceae (p = 0.001) and enterococci (p < 0.001), in comparison with day 0. After 5 days of therapy, lactobacilli are no longer detected. Conversely, bacterial count values for all bacteria detected after 15 days from the end of therapy are significantly increased and similar to day 0. Conclusion: Our findings showed that gut microbiota was significantly modified after 5 days of antibiotic therapy; exclusively, breast-feeding may be relevant in promoting the re-establishment of gut microbiota composition in early infancy. © 2010 The Author(s)/Acta Pædiatrica © 2010 Foundation Acta Pædiatrica.

PubMed | University of Padua, Ospedale G.B. Morgagni L. Pierantoni, Treviso Hospital, San Giuseppe Moscati National Hospital AORN and 12 more.
Type: Journal Article | Journal: PloS one | Year: 2016

The use of antiemetics for vomiting in acute gastroenteritis in children is still a matter of debate. We conducted a double-blind randomized trial to evaluate whether a single oral dose of ondansetron vs domperidone or placebo improves outcomes in children with gastroenteritis. After failure of initial oral rehydration administration, children aged 1-6 years admitted for gastroenteritis to the pediatric emergency departments of 15 hospitals in Italy were randomized to receive one oral dose of ondansetron (0.15 mg/kg) or domperidone (0.5 mg/kg) or placebo. The primary outcome was the percentage of children receiving nasogastric or intravenous rehydration. A p value of 0.014 was used to indicate statistical significance (and 98.6% CI were calculated) as a result of having carried out two interim analyses. 1,313 children were eligible for the first attempt with oral rehydration solution, which was successful for 832 (63.4%); 356 underwent randomization (the parents of 125 children did not give consent): 118 to placebo, 119 to domperidone, and 119 to ondansetron. Fourteen (11.8%) needed intravenous rehydration in the ondansetron group vs 30 (25.2%) and 34 (28.8%) in the domperidone and placebo groups, respectively. Ondansetron reduced the risk of intravenous rehydration by over 50%, both vs placebo (RR 0.41, 98.6% CI 0.20-0.83) and domperidone (RR 0.47, 98.6% CI 0.23-0.97). No differences for adverse events were seen among groups. In a context of emergency care, 6 out of 10 children aged 1-6 years with vomiting due to gastroenteritis and without severe dehydration can be managed effectively with administration of oral rehydration solution alone. In children who fail oral rehydration, a single oral dose of ondansetron reduces the need for intravenous rehydration and the percentage of children who continue to vomit, thereby facilitating the success of oral rehydration. Domperidone was not effective for the symptomatic treatment of vomiting during acute gastroenteritis.

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