Cargando…

Cardio- Renal Outcomes With Long- Term Agalsidase Alfa Enzyme Replacement Therapy: A 10- Year Fabry Outcome Survey (FOS) Analysis

PURPOSE: Following the publication of 5-year agalsidase alfa enzyme replacement therapy (ERT) outcomes data from the Fabry Outcome Survey (FOS), 10-year data were analyzed. PATIENTS AND METHODS: FOS (ClinicalTrials.gov identifier: NCT03289065) data (April 2001 to August 2018) were retrospectively an...

Descripción completa

Detalles Bibliográficos
Autores principales:	Ramaswami, Uma, Beck, Michael, Hughes, Derralynn, Kampmann, Christoph, Botha, Jaco, Pintos-Morell, Guillem, West, Michael L, Niu, Dau-Ming, Nicholls, Kathy, Giugliani, Roberto
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Dove 2019
Materias:	Original Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6819672/ https://www.ncbi.nlm.nih.gov/pubmed/31749608 http://dx.doi.org/10.2147/DDDT.S207856

Ejemplares similares

Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease: A Fabry Outcome Survey analysis
por: Beck, Michael, et al.
Publicado: (2015)

Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events
por: Beck, Michael, et al.
Publicado: (2017)

Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registry
por: Beck, Michael, et al.
Publicado: (2022)

Update on role of agalsidase alfa in management of Fabry disease
por: Ramaswami, Uma
Publicado: (2011)

Analysis of Renal and Cardiac Outcomes in Male Participants in the Fabry Outcome Survey Starting Agalsidase Alfa Enzyme Replacement Therapy Before and After 18 Years of Age
por: Parini, Rossella, et al.
Publicado: (2020)

Renoprotective Effect of Agalsidase Alfa: A Long-Term Follow-Up of Patients with Fabry Disease
por: Cybulla, Markus, et al.
Publicado: (2022)

Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: an international cohort study
por: Arends, Maarten, et al.
Publicado: (2018)

Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years’ treatment
por: Kampmann, Christoph, et al.
Publicado: (2015)

Erratum to: Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years’ treatment
por: Kampmann, Christoph, et al.
Publicado: (2016)

Prompt Agalsidase Alfa Therapy Initiation is Associated with Improved Renal and Cardiovascular Outcomes in a Fabry Outcome Survey Analysis
por: Hughes, Derralynn, et al.
Publicado: (2021)

Agalsidase alfa and agalsidase beta in the treatment of Fabry disease: does the dose really matter?
por: Pisani, Antonio, et al.
Publicado: (2015)

Agalsidase alfa (Replagal(™)) in the treatment of Anderson-Fabry disease
por: Pastores, Gregory M
Publicado: (2007)

Clinical observation of patients with Fabry disease after switching from agalsidase beta (Fabrazyme) to agalsidase alfa (Replagal)
por: Tsuboi, Kazuya, et al.
Publicado: (2012)

Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study
por: Linhart, Aleš, et al.
Publicado: (2023)

CORRIGENDUM: Clinical observation of patients with Fabry disease after switching from agalsidase beta (Fabrazyme) to agalsidase alfa (Replagal)
Publicado: (2012)

Gender Differences in the Application of Spanish Criteria for Initiation of Enzyme Replacement Therapy for Fabry Disease in the Fabry Outcome Survey
por: Barba-Romero, Miguel-Ángel, et al.
Publicado: (2016)

Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease
por: Goláň, Lubor, et al.
Publicado: (2015)

Treatment of Fabry Disease: Outcome of a Comparative Trial with Agalsidase Alfa or Beta at a Dose of 0.2 mg/kg
por: Vedder, Anouk C., et al.
Publicado: (2007)

Paediatric Fabry disease: prognostic significance of ocular changes for disease severity
por: Kalkum, Gisela, et al.
Publicado: (2016)

Fabry disease in children and the effects of enzyme replacement treatment
por: Pintos-Morell, Guillem, et al.
Publicado: (2009)

Efficacy and safety of enzyme-replacement-therapy with agalsidase alfa in 36 treatment-naïve Fabry disease patients
por: Tsuboi, Kazuya, et al.
Publicado: (2017)

Measuring patient experiences in Fabry disease: validation of the Fabry-specific Pediatric Health and Pain Questionnaire (FPHPQ)
por: Ramaswami, Uma, et al.
Publicado: (2012)

The Safety of Agalsidase Alfa Enzyme Replacement Therapy in Canadian Patients with Fabry Disease Following Implementation of a Bioreactor Process
por: Khan, Aneal, et al.
Publicado: (2021)

Agalsidase alfa in pediatric patients with Fabry disease: a 6.5-year open-label follow-up study
por: Schiffmann, Raphael, et al.
Publicado: (2014)

Fabry disease and incidence of cancer
por: Bird, Sarah, et al.
Publicado: (2017)

Clinical course of patients with Fabry disease who were switched from agalsidase-β to agalsidase-α
por: Tsuboi, Kazuya, et al.
Publicado: (2014)

Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease
por: Germain, Dominique P, et al.
Publicado: (2015)

Renal outcomes of agalsidase beta treatment for Fabry disease: role of proteinuria and timing of treatment initiation
por: Warnock, David G., et al.
Publicado: (2012)

An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy
por: Goker-Alpan, Ozlem, et al.
Publicado: (2016)

Time to treatment benefit for adult patients with Fabry disease receiving agalsidase β: data from the Fabry Registry
por: Ortiz, Alberto, et al.
Publicado: (2016)

Cardiomyopathy and kidney function in agalsidase beta‐treated female Fabry patients: a pre‐treatment vs. post‐treatment analysis
por: Wanner, Christoph, et al.
Publicado: (2020)

Development of a Biosimilar of Agalsidase Beta for the Treatment of Fabry Disease: Preclinical Evaluation
por: van Kuilenburg, André B. P., et al.
Publicado: (2023)

Consequences of a global enzyme shortage of agalsidase beta in adult Dutch Fabry patients
por: Smid, Bouwien E, et al.
Publicado: (2011)

Improvement of gastrointestinal symptoms in a significant proportion of male patients with classic Fabry disease treated with agalsidase beta: A Fabry Registry analysis stratified by phenotype
por: Hopkin, Robert J., et al.
Publicado: (2020)

Spanish Fabry and Gaucher disease patients show striking differences in Beliefs about Medicines (BMQ) and Brief Illness Perception (BIPQ) questionnaires
por: González-Lamuño, Domingo, et al.
Publicado: (2022)

Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy was underpowered
por: Ortiz, Alberto, et al.
Publicado: (2015)

Letter concerning “Enzyme replacement therapy in a patient with Fabry disease and the development of IgE antibodies against agalsidase beta but not agalsidase alpha”, by Tanaka et al.
por: Linthorst, Gabor E., et al.
Publicado: (2010)

Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS)
por: Muenzer, Joseph, et al.
Publicado: (2021)

Antiproteinuric therapy and Fabry nephropathy: factors associated with preserved kidney function during agalsidase-beta therapy
por: Warnock, David G, et al.
Publicado: (2015)

Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes
por: Hughes, Derralynn A, et al.
Publicado: (2023)

Cannot write session to /tmp/vufind_sessions/sess_prmokabp71fs0c3au063nhbm49