The Clinical Manifestations and Natural History of Hepatocellular Carcinoma

Abstract Hemorrhagic stroke (HS) is a major cause of death and disability worldwide, despite being less common, it presents more aggressively and leads to more severe sequelae than ischemic stroke. There are two types of HS: Intracerebral Hemorrhage (ICH) and Subarachnoid Hemorrhage (SAH), differing not only in the site of bleeding, but also in the mechanisms responsible for acute and subacute symptoms. This is a systematic review of databases in search of works of the last five years relating to the comprehension of both kinds of HS. Sixty two articles composed the direct findings of the recent literature and were further characterized to construct the pathophysiology in the order of events. The road to the understanding of the spontaneous HS pathophysiology is far from complete. Our findings show specific and individual results relating to the natural history of the disease of ICH and SAH, presenting common and different risk factors, distinct and similar clinical manifestations at onset or later days to weeks, and possible complications for both.

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CLINICAL MANIFESTATIONS AND NATURAL HISTORY OF GENITAL HUMAN PAPILLOMAVIRUS INFECTIONS

Obstetrics and Gynecology Clinics of North America ◽

10.1016/s0889-8545(05)70277-1 ◽

1996 ◽

Vol 23 (4) ◽

pp. 783-809 ◽

Cited By ~ 4

Author(s):

Michael J. Campion ◽

Mitchell D. Greenberg ◽

Tarek I.G. Kazamel

Keyword(s):

Human Papillomavirus ◽

Natural History ◽

Clinical Manifestations ◽

Papillomavirus Infections ◽

History Of

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Clinical Manifestations and Natural History of Egg Allergy in Infants and Children

Journal of Allergy and Clinical Immunology ◽

10.1016/j.jaci.2006.11.311 ◽

2007 ◽

Vol 119 (1) ◽

pp. S73

Author(s):

R.G. Heine ◽

N. Laske ◽

D.J. Hill

Keyword(s):

Natural History ◽

Clinical Manifestations ◽

Infants And Children ◽

Egg Allergy ◽

History Of ◽

In Infants And Children

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

10.21203/rs.2.19376/v2 ◽

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.

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

Orphanet Journal of Rare Diseases ◽

10.1186/s13023-020-01549-8 ◽

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.

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