scholarly journals Characterization of Adult Patients With SMA Treated in US Hospital Settings: A Natural History Study in the Premier Healthcare Database

2021 ◽  
pp. 1-10
Author(s):  
Nicole B. Johnson ◽  
Crystal Proud ◽  
Christina L. Wassel ◽  
Jill Dreyfus ◽  
Thos Cochrane ◽  
...  

Background: Spinal muscular atrophy (SMA) is a rare genetic disease characterized by progressive muscular weakness and atrophy resulting from motor neuron degeneration. Limited information is available on disease progression among older SMA patients, particularly adults. Objective: This study sought to characterize the natural history of SMA among adult patients in US hospital settings through the assessment of symptoms, complications, costs, and healthcare resource utilization (HRU) over 3 years before the availability of disease-modifying therapies. Methods: The study population included adult (≥18 years) patients with inpatient and/or hospital-based outpatient discharge records and≥2 primary or secondary SMA ICD-9 codes≥30 days apart in the Premier Healthcare Database during the main study period (2007– 2014). Index date was the date of the first SMA ICD-9 code. The frequency of SMA-related symptoms and complications was assessed 1 year preindex through 2 years postindex to characterize disease progression. Costs and HRU were also assessed across the study period. Results: A total of 446 adult patients from 337 US hospitals met inclusion criteria for these analyses. All evaluated SMA-related symptoms and complications increased steadily over time, from 1 year preindex to 2 years postindex both overall and in each age group. Adult patients with SMA had increasing total costs and HRU over the 3-year study period: total costs were $1759 preindex and $12,308 by 2 years postindex. Conclusions: Findings are consistent with increasing disease burden over time and support the progressive nature of SMA for adult patients with hospital interactions.

2021 ◽  
Vol 10 (6) ◽  
pp. 1161
Author(s):  
Raluca Pais ◽  
Thomas Maurel

The epidemiology and the current burden of chronic liver disease are changing globally, with non-alcoholic fatty liver disease (NAFLD) becoming the most frequent cause of liver disease in close relationship with the global epidemics of obesity, type 2 diabetes and metabolic syndrome. The clinical phenotypes of NAFLD are very heterogeneous in relationship with multiple pathways involved in the disease progression. In the absence of a specific treatment for non-alcoholic steatohepatitis (NASH), it is important to understand the natural history of the disease, to identify and to optimize the control of factors that are involved in disease progression. In this paper we propose a critical analysis of factors that are involved in the progression of the liver damage and the occurrence of extra-hepatic complications (cardiovascular diseases, extra hepatic cancer) in patients with NAFLD. We also briefly discuss the impact of the heterogeneity of the clinical phenotype of NAFLD on the clinical practice globally and at the individual level.


Author(s):  
Mélanie Annoussamy ◽  
Andreea M. Seferian ◽  
Aurore Daron ◽  
Yann Péréon ◽  
Claude Cances ◽  
...  

2003 ◽  
Vol 149 (1) ◽  
pp. 53-59 ◽  
Author(s):  
F Lombardo ◽  
F De Luca ◽  
M Rosano ◽  
C Sferlazzas ◽  
C Lucanto ◽  
...  

OBJECTIVE: The loss of pancreatic beta-cells is thought to be one of the principal causes of diabetes mellitus (DM) in cystic fibrosis (CF), but the role of peripheral insulin resistance (IR) in the pathogenesis of DM in CF remains unclear. The aim of this study was to evaluate whether eventual changes of glucose tolerance (GT) over time were associated with modifications of insulin secretion or sensitivity. METHODS: Plasma glucose and insulin responses to an oral GT test (OGTT) were investigated and reinvestigated 13 Years later in 14 CF patients with initial and persistent fasting euglycemia and no history of insulin treatment. Insulin sensitivity (IS) at both tests was assessed on the basis of insulin and glucose levels both in the fasting state and during OGTTs. RESULTS: From the 1st to the 2nd OGTT: (a) the prevalence of DM responses significantly increased; (b) the areas beneath the respective glucose and insulin curves significantly increased and decreased respectively; (c) IR and IS indices decreased and increased respectively, even in the patients who developed DM; (d) pulmonary function significantly worsened in the entire series, especially in the patients who developed DM. CONCLUSIONS: (i) the natural history of glyco-metabolic status in CF is characterized by deteriorating GT over time; (ii) insulinopenia plays a prominent role in the pathogenesis of GT worsening; (iii) IR does not play any significant part in the pathogenesis of DM development; (iv) deterioration of lung function tests is more severe in the subjects who develop DM over time.


2018 ◽  
Vol 2018 ◽  
pp. 1-4 ◽  
Author(s):  
Ala I. Sharara ◽  
Nathalie Ziade ◽  
Rani H. Shayto ◽  
Luma Basma O. Rustom ◽  
Hamed Chehab ◽  
...  

Background. The natural history of colonic diverticulosis is unclear.Methods. Patients with incidental diverticulosis identified in a previous prospective cross-sectional screening colonoscopy study were evaluated retrospectively for clinic or hospital visit(s) for diverticular disease (DD= acute diverticulitis or diverticular bleeding) using review of electronic health records and patient phone interview.Results. 826 patients were included in the screening colonoscopy study. Three were excluded for prior DD. In all, 224 patients (27.2%; mean age 62.3 ± 8.2) had incidental diverticulosis distributed in the left colon (67.4%), right colon (5.8%), or both (22.8%). Up-to-date information was available on 194 patients. Of those, 144 (74.2%) could be reached for detailed interview and constituted the study population. Over a mean follow-up of 7.0 ± 1.7 years, DD developed in 6 out of 144 patients (4.2%) (4 acute cases of diverticulitis, 1 probable case of diverticular bleeding, and 1 acute case of diverticulitis and diverticular bleeding). Two patients were hospitalized, and none required surgery. The time to event was 5.1 ± 1.6 years and the incidence rate was 5.9 per 1000 patient-years. On multivariate analysis, none of the variables collected at baseline colonoscopy including age, gender, obesity, exercise, fiber intake, alcohol use, constipation, or use of NSAIDs were associated with DD.Conclusion. The natural history of incidental diverticulosis on screening colonoscopy was highly favorable in this well-defined prospectively identified cohort. The common scenario of incidental diverticulosis at screening colonoscopy makes this information clinically relevant and valuable to physicians and patients alike.


Author(s):  
F.C.T. Moore

In his youth, Bonnet made a meticulous and creative study of insects, which won him international fame for his discoveries, as well as his methods. He turned to psychology and offered a detailed, but speculative, account of the physiology of mental states. His empirical work was overtaken by speculative ambition. In later life, he developed (from elements already present in his early studies) a comprehensive view of the universe, of its history and its natural history, of theology and of moral philosophy. Christianity was proved, the great chain of being was mapped over time towards an ultimate perfection, and human morality, based on self-love, formed part of the Creator’s scheme. The Creator, at the moment of creation, brought into being all the elements from which this vast unfolding would occur, without further intervention.


2002 ◽  
Vol 181 (2) ◽  
pp. 135-137 ◽  
Author(s):  
R. G. McCreadie ◽  
R. Padmavati ◽  
R. Thara ◽  
T. N. Srinivasan

BackgroundSpontaneous dyskinesia and parkinsonism have been reported in never-medicated patients with schizophrenia but there has been no previous study of the natural history of these conditions.AimsTo determine the prevalence of spontaneous dyskinesia and parkinsonism in a group of never-medicated, chronically ill patients with schizophrenia on two occasions separated by an 18-month interval.MethodDyskinesia was assessed by the Abnormal Involuntary Movements Scale using Schooler and Kane criteria for its presence; parkinsonism by the Simpson and Angus scale; and mental state by the Positive and Negative Syndrome Scale for schizophrenia.ResultsThirty-seven patients were examined on two occasions. Nine (24%) had dyskinesia on both occasions, 12 (33%) on one occasion and 16 (43%) on neither occasion. Twenty-one (57%) had dyskinesia on at least one occasion. Thirteen patients (35%) had parkinsonism on at least one occasion.ConclusionsSpontaneous dyskinesia and parkinsonism fluctuate over time. The former was found on at least one occasion in the majority of patients. It is an integral part ofthe schizophrenic disease process.


2019 ◽  
Vol 29 ◽  
pp. S132
Author(s):  
J. Exposito ◽  
D. Natera-de Benito ◽  
L. Carrera ◽  
A. Frongia ◽  
M. Alarcón ◽  
...  

Sign in / Sign up

Export Citation Format

Share Document