scholarly journals Natural History of HumanCalicivirusInfection: A Prospective Cohort Study

2002 ◽  
Vol 35 (3) ◽  
pp. 246-253 ◽  
Author(s):  
Barry Rockx ◽  
Matty de Wit ◽  
Harry Vennema ◽  
Jan Vinjé ◽  
Erwin de Bruin ◽  
...  
Keyword(s):  
Cohort Study ◽  
Natural History ◽  
Prospective Cohort Study ◽  
Prospective Cohort ◽  
History Of

scholarly journals The SPARKLE registry: protocol for an international prospective cohort study in patients with alpha-mannosidosis

2020 ◽  
Author(s):  
Julia Hennermann ◽  
Nathalie Guffon ◽  
Federica Cattaneo ◽  
Ferdinando Ceravolo ◽  
Line Borgwardt ◽  
...  
Keyword(s):  
Cohort Study ◽  
Natural History ◽  
Prospective Cohort Study ◽  
Prospective Cohort ◽  
Clinical Care ◽  
Clinical Manifestations ◽  
Routine Clinical Care ◽  
Safety Outcomes ◽  
History Of

Abstract Background: Alpha-mannosidosis is a lysosomal storage disorder caused by reduced enzymatic activity of alpha-mannosidase. SPARKLE is an alpha-mannosidosis registry intended to obtain long-term safety and effectiveness data on the use of velmanase alfa during routine clinical care in patients with alpha-mannosidosis. It is a post-approval commitment to European marketing authorization for Velmanase alfa (Lamzede®), the first enzyme replacement therapy for the treatment of non-neurologic manifestations in patients with mild to moderate alpha-mannosidosis. In addition, SPARKLE will expand the current understanding of alpha-mannosidosis by collecting data on the clinical manifestations, progression, and natural history of the disease in treated and untreated patients, respectively.Results: The SPARKLE registry is designed as a multicenter, multinational, noninterventional, prospective cohort study of patients with alpha-mannosidosis, starting patient enrollment in 2020. Patients will be followed for up to 15 years. Safety and effectiveness as post-authorization outcomes under routine clinical care in patients with treatment will be evaluated. The primary safety outcomes are the rate of adverse events (anti-velmanase alfa-immunoglobulin G antibody development, infusion-related reactions, and hypersensitivity). Secondary safety outcomes include the evaluation of medical events, change in vital signs, laboratory tests, physical examination, and electrocardiogram results. The primary effectiveness outcome is a global treatment response rate, evaluated as the individual aggregate of single endpoints from pharmacodynamic, functional, and quality of life effectiveness outcomes; secondary effectiveness outcomes are to characterize the population of patients with alpha-mannosidosis with regard to clinical manifestation, progression, and natural history of the disease. Any patient in the European Union with a diagnosis of alpha-mannosidosis who is willing to participate will likely be eligible for inclusion in the registry. Publications to disseminate scientific insights from the registry are planned. Conclusion: This study will provide real-world data on the long-term safety and effectiveness of velmanase alfa in patients with alpha-mannosidosis during routine clinical care and increase the understanding of the natural course, clinical manifestations, and progression of this ultra-rare disease.

scholarly journals The SPARKLE registry: protocol for an international prospective cohort study in patients with alpha-mannosidosis

2020 ◽  
Vol 15 (1) ◽  
Author(s):  
Julia B. Hennermann ◽  
Nathalie Guffon ◽  
Federica Cattaneo ◽  
Ferdinando Ceravolo ◽  
Line Borgwardt ◽  
...  
Keyword(s):  
Cohort Study ◽  
Natural History ◽  
Prospective Cohort Study ◽  
Prospective Cohort ◽  
Clinical Care ◽  
Clinical Manifestations ◽  
Routine Clinical Care ◽  
Safety Outcomes ◽  
History Of

Abstract Background Alpha-mannosidosis is a lysosomal storage disorder caused by reduced enzymatic activity of alpha-mannosidase. SPARKLE is an alpha-mannosidosis registry intended to obtain long-term safety and effectiveness data on the use of velmanase alfa during routine clinical care in patients with alpha-mannosidosis. It is a post-approval commitment to European marketing authorization for Velmanase alfa (Lamzede®), the first enzyme replacement therapy for the treatment of non-neurologic manifestations in patients with mild to moderate alpha-mannosidosis. In addition, SPARKLE will expand the current understanding of alpha-mannosidosis by collecting data on the clinical manifestations, progression, and natural history of the disease in treated and untreated patients, respectively. Results The SPARKLE registry is designed as a multicenter, multinational, noninterventional, prospective cohort study of patients with alpha-mannosidosis, starting patient enrollment in 2020. Patients will be followed for up to 15 years. Safety and effectiveness as post-authorization outcomes under routine clinical care in patients with treatment will be evaluated. The primary safety outcomes are the rate of adverse events (anti-velmanase alfa-immunoglobulin G antibody development, infusion-related reactions, and hypersensitivity). Secondary safety outcomes include the evaluation of medical events, change in vital signs, laboratory tests, physical examination, and electrocardiogram results. The primary effectiveness outcome is a global treatment response rate, evaluated as the individual aggregate of single endpoints from pharmacodynamic, functional, and quality-of-life effectiveness outcomes; secondary effectiveness outcomes are to characterize the population of patients with alpha-mannosidosis with regard to clinical manifestation, progression, and natural history of the disease. Any patient in the European Union with a diagnosis of alpha-mannosidosis who is willing to participate will likely be eligible for inclusion in the registry. Publications to disseminate scientific insights from the registry are planned. Conclusion This study will provide real-world data on the long-term safety and effectiveness of velmanase alfa in patients with alpha-mannosidosis during routine clinical care and increase the understanding of the natural course, clinical manifestations, and progression of this ultra-rare disease.

Single-center prospective, cohort study of the natural history of acute pancreatitis

Pancreatology ◽  
2014 ◽  
Vol 14 (3) ◽  
pp. S98-S99
Author(s):  
Giulia Martina Cavestro ◽  
Leandro Gioacchino ◽  
Milena Di Leo ◽  
Raffaella Alessia Zuppardo ◽  
Olivia B. Morrow ◽  
...  
Keyword(s):  
Acute Pancreatitis ◽  
Cohort Study ◽  
Natural History ◽  
Prospective Cohort Study ◽  
Prospective Cohort ◽  
Single Center ◽  
History Of

Sa1411 The Natural History of Incidental Colonic Diverticulosis on Screening Colonoscopy: A Prospective Cohort Study

2016 ◽  
Vol 150 (4) ◽  
pp. S308 ◽  
Author(s):  
Rani Shayto ◽  
Krystelle Hanna ◽  
Nathalie Ziyade ◽  
Hamed Chehab ◽  
Jean M. Chalhoub ◽  
...  
Keyword(s):  
Cohort Study ◽  
Natural History ◽  
Prospective Cohort Study ◽  
Prospective Cohort ◽  
Screening Colonoscopy ◽  
Colonic Diverticulosis ◽  
History Of

179 A PROSPECTIVE COHORT STUDY INVESTIGATING THE NATURAL HISTORY OF ACUTE-ON-CHRONIC LIVER FAILURE

2010 ◽  
Vol 52 ◽  
pp. S77-S78
Author(s):  
F. Nevens ◽  
A. Katoonizadeh ◽  
C. Verslype ◽  
G. Maleux ◽  
T. Roskams ◽  
...  
Keyword(s):  
Cohort Study ◽  
Natural History ◽  
Liver Failure ◽  
Prospective Cohort Study ◽  
Prospective Cohort ◽  
Chronic Liver ◽  
Chronic Liver Failure ◽  
History Of

scholarly journals Natural history of gastroesophageal reflux: A prospective cohort study in a stratified, randomized population in Beijing

2019 ◽  
Vol 20 (10) ◽  
pp. 523-531
Author(s):  
Xiucai Fang ◽  
Liming Zhu ◽  
Dongsheng Wu ◽  
Zhifeng Wang ◽  
Guijun Fei ◽  
...  
Keyword(s):  
Cohort Study ◽  
Natural History ◽  
Gastroesophageal Reflux ◽  
Prospective Cohort Study ◽  
Prospective Cohort ◽  
History Of

scholarly journals Natural history of multiple system atrophy in the USA: a prospective cohort study

2015 ◽  
Vol 14 (7) ◽  
pp. 710-719 ◽  
Author(s):  
Phillip A Low ◽  
Stephen G Reich ◽  
Joseph Jankovic ◽  
Clifford W Shults ◽  
Matthew B Stern ◽  
...  
Keyword(s):  
Cohort Study ◽  
Natural History ◽  
Multiple System Atrophy ◽  
Prospective Cohort Study ◽  
Prospective Cohort ◽  
The Usa ◽  
History Of

The natural history of adolescent binge drinking: Findings from a 15-year prospective cohort study

2014 ◽  
Vol 140 ◽  
pp. e46
Author(s):  
Louisa Degenhardt ◽  
C. O’Loughlin ◽  
Wendy Swift ◽  
H. Romaniuk ◽  
J. Carlin ◽  
...  
Keyword(s):  
Cohort Study ◽  
Natural History ◽  
Binge Drinking ◽  
Prospective Cohort Study ◽  
Prospective Cohort ◽  
History Of
Sign in / Sign up

Export Citation Format

Share Document