Search results for "Natural History"
showing 10 items of 124 documents
MARITAL STATUS IS A PROGNOSTIC FACTOR IN AMYOTROPHIC LATERAL SCLEROSIS
2017
Background and objectives Several variables have been linked to a shorter survival in patients with amyotrophic lateral sclerosis (ALS), for example, female sex, older age, site of disease onset, rapid disease progression, and a relatively short diagnostic delay. With regard to marital status, previous studies suggested that living with a partner might be associated to a longer survival and a higher likelihood to proceed to tracheostomy. Therefore, to further strengthen this hypothesis, we investigated the role of marital status as a prognostic variable in a cohort of ALS patients. Methods We performed a retrospective analysis on 501 consecutive ALS patients for which a complete disease's n…
Infantile hemangiomas with minimal or arrested growth: A retrospective case series
2018
Background/objectives Infantile hemangiomas (IH) with minimal or arrested growth (MAG) constitute a distinctive subset of IH based on their clinical characteristics and natural history. They are often confused with capillary malformations. Methods A retrospective observational study has been carried out in which clinical and perinatal characteristics have been evaluated in all IH-MAG evaluated in our Dermatology Department in a 5-year period (January 2013-December 2017). Results A total of 14 IH-MAG affecting 13 patients were identified (10 girls and 3 boys). All were born full term, with a mean birth weight of 3448 g for girls vs 3540 g for boys, corresponding to the 72nd percentile for bo…
Comparing Natural History of Early and Late Onset Pediatric Multiple Sclerosis
2022
Objective: This study was undertaken to describe and compare disease course and prognosis of early (ie, disease onset before age 11 years) and late (ie, disease onset after age 11 years) onset pediatric multiple sclerosis. Methods: Prospectively collected clinical information from Italian Multiple Sclerosis Register of 1993 pediatric multiple sclerosis patients, of whom 172 had early onset, was analyzed. Cox models adjusted for sex, baseline Expanded Disability Status Scale score, and disease-modifying treatments and stratified for diagnostic criteria adopted (Poser vs McDonald) were used to assess the risk of reaching irreversible Expanded Disability Status Scale scores of 3, 4, and 6, and…
Characteristics and management of primary and other immune thrombocytopenias: Spanish registry study
2017
The natural history and its modulation by treatments administered for immune thrombocytopenia (ITP) in the clinical practice remains unknown. In addition, little information is available on the characteristics and management of ITP in Spain. We conducted an observational, multicenter, registry in 70 Hematology Services from Spain between 2009 and 2011, which included children from 2 months of age and adults with primary ITP or another ITP diagnosed within the last 6 months (platelet count [PC] 484 patients were included (median [Q1, Q3] age 52 [29,74] years, 87.6% adults), 56% women, 10.5% with secondary ITP. Median (Q1, Q3) PC at diagnosis was 12 × 109/l (4, 32); 72% of patients had bleed…
Importance of surgical history in diagnosing mucopolysaccharidosis type II (Hunter syndrome): data from the Hunter Outcome Survey.
2010
Purpose: To characterize surgical histories typical of patients with mucopolysaccharidosis type II, thereby broadening understanding of the natural history of these patients and helping physicians recognize the disease. Methods: Data on surgical interventions from the Hunter Outcome Survey—a multinational, observational database of patients with mucopolysaccharidosis type II—were analyzed. The study population comprised 527 patients for whom surgical data were reported on/before July 23, 2009. Results: Surgical interventions were performed in 83.7% of the study population. Patients underwent their first operation at a median age of 2.6 years. Tympanostomies, repairs of inguinal hernias, and…
Changes in the requirement for early surgery in inflammatory bowel disease in the era of biological agents.
2020
This is the peer reviewed version of the following article: Changes in the requirement for early surgery in inflammatory bowel disease in the era of biological agents. Journal of Gastroenterology and Hepatology (2020): 29 April, which has been published in final form at https://doi.org/10.1111/jgh.15084. This article may be used for non-commercial purposes in accordance with Wiley Terms and Conditions for Use of Self-Archived Versions
Management of chronic hepatitis C in childhood: The impact of therapy in the clinical practice during the first 2 decades
2011
Background and aim: Treatment of chronic hepatitis C in children is controversial and its role in the clinical practice is unknown. We retrospectively investigated the impact of treatment in a large cohort of children with chronic hepatitis C over the past 20years. Methods: 376 hepatitis C virus RNApositive children were recruited consecutively in five Italian centres since 1990and followed for1–17years. Results: 86 (23%)subjects were treated: 73 with recombinant interferon alone and 13 with pegylated-interferon and ribavirin. Sustained clearance of hepatitis C virus RNA was observed in 25%of the former, in 92%of the latter and in 9% of untreated cases(p < 0.001). Loss of viraemia was re…
Incidence of curvature progression in idiopathic scoliosis patients treated with scoliosis in-patient rehabilitation (SIR): an age- and sex-matched c…
2003
The goal of this study is to test the hypothesis that physiotherapy-based intervention can reduce incidence of progression in children with IS. Two independent patient groups matched by age and sex at diagnosis were analysed using the outcome parameter, incidence of progression (or =5 degrees ). One group was untreated and the other received scoliosis in-patient rehabilitation (SIR). Incidence of progression in groups of untreated patients ranged from 1.5-fold (71.2% vs 46.7%) to 2.9-fold (55.8% vs 19.2%) higher than in groups of patients treated with SIR, even when SIR-treated groups included patients with more severe curvatures. Statistically, the differences were highly significant. Effo…
Epidemiology of pancreatic neuroendocrine neoplasms. a gender perspective
2020
Purpose: Pancreatic neuroendocrine neoplasms (PNENs) are a group of clinically rare and heterogeneous tumors of the pancreas. Currently there are no studies investigating the gender difference in PNEN susceptibility. Thus, the purpose of this study was aimed at examining how gender shapes risk factors, clinicopathological features, and comorbidities in PNENs. Methods: The study design consisted of an Italian multicenter, retrospective study. The study included all consecutive patients with PNENs followed at the participating centers. Two hundred and twenty-nine patients (105 males,124 females, age 54 ± 0.98 years) with PNENs were enrolled at the participating centers. The clinicopathologica…
Fabry disease in children and response to enzyme replacement therapy: results from the Fabry Outcome Survey
2011
The Fabry Outcome Survey (FOS) was established to extend the knowledge of the natural history of Fabry disease and to assess the effects of enzyme replacement therapy (ERT) with agalsidase alfa. As of March 2009, 64 boys and 34 girls with Fabry disease had enrolled in the FOS and been treated with agalsidase alfa for at least 6 months. The prevalence of symptoms tended to be reduced after 12 and 24 months of ERT in patients who experienced symptoms at baseline. In the entire population, non-significant decreases in the prevalence of gastrointestinal problems in boys and pain crises in girls were observed after 12-24 months. Kidney function and left ventricular mass indexed to height remaine…