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6. Lentivirus-mediated gene therapy for Fabry disease

Author

Khan, Aneal, Barber, Dwayne L., Huang, Ju, Rupar, C. Anthony, Rip, Jack W., Auray-Blais, Christiane, Boutin, Michel, O’Hoski, Pamela, Gargulak, Kristy, McKillop, William M., Fraser, Graeme, Wasim, Syed, LeMoine, Kaye, Jelinski, Shelly, Chaudhry, Ahsan, Prokopishyn, Nicole, Morel, Chantal F., Couban, Stephen, Duggan, Peter R., Fowler, Daniel H., Keating, Armand, West, Michael L., Foley, Ronan, and Medin, Jeffrey A.

Published
2021
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11. Uncertain Diagnosis of Fabry Disease in Patients with Neuropathic Pain, Angiokeratoma or Cornea Verticillata: Consensus on the Approach to Diagnosis and Follow-Up

Author

van der Tol, L., primary, Cassiman, David, additional, Houge, Gunnar, additional, Janssen, Mirian C., additional, Lachmann, Robin H, additional, Linthorst, Gabor E, additional, Ramaswami, Uma, additional, Sommer, Claudia, additional, Tøndel, Camilla, additional, West, Michael L, additional, Weidemann, Frank, additional, Wijburg, Frits A, additional, Svarstad, Einar, additional, Hollak, Carla EM, additional, and Biegstraaten, Marieke, additional

Published
2014
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13. Cardiomyopathy and kidney function in agalsidase beta-treated female Fabry patients: a pre-treatment vs. post-treatment analysis

Author

Wanner, Christoph, Feldt-Rasmussen, Ulla, Jovanovic, Ana, Linhart, Aleš, Yang, Meng, Ponce, Elvira, Brand, Eva, Germain, Dominique P, Hughes, Derralynn A, Jefferies, John L, Martins, Ana Maria, Nowak, Albina, Vujkovac, Bojan, Weidemann, Frank, West, Michael L, Ortiz, Alberto, University Hospital of Würzburg, Rigshospitalet [Copenhagen], Copenhagen University Hospital, Sanofi Genzyme, University Hospital Münster - Universitaetsklinikum Muenster [Germany] (UKM), Handicap neuromusculaire : Physiopathologie, Biothérapie et Pharmacologies appliquées (END-ICAP), Université de Versailles Saint-Quentin-en-Yvelines (UVSQ)-Institut National de la Santé et de la Recherche Médicale (INSERM), University College of London [London] (UCL), The University of Tennessee Health Science Center [Memphis] (UTHSC), Federal University of Sao Paulo (Unifesp), University hospital of Zurich [Zurich], Dalhousie University [Halifax], Sanofi Genzyme, Genzyme Corp., This work was supported by Sanofi Genzyme, the sponsor of the Fabry Registry, including the editorial support of Tom Rouwette from Excerpta Medica. Sanofi Genzyme had a role in the design and conduct of the research and in the collection, management, analysis, interpretation of the data, and preparation and courtesy review of the manuscript. The authors are responsible for the content of this manuscript and the decision to submit the manuscript for publication., University of Zurich, and Wanner, Christoph

Subjects
Male, lcsh:Diseases of the circulatory (Cardiovascular) system, Adolescent, Cardiomyopathy, 10265 Clinic for Endocrinology and Diabetology, Agalsidase beta, 610 Medicine & health, Kidney, 2705 Cardiology and Cardiovascular Medicine, Kidney function, Original Research Articles, Humans, ddc:610, Original Research Article, Language, Fabry disease, Myocardium, Female patients, Isoenzymes, lcsh:RC666-701, alpha-Galactosidase, Enzyme replacement therapy, Female, [SDV.IB]Life Sciences [q-bio]/Bioengineering, Glycolipids, Cardiomyopathies
Abstract

Aims: Long-term treatment effect studies in large female Fabry patient groups are challenging to design because of phenotype heterogeneity and lack of appropriate comparison groups, and have not been reported. We compared long-term cardiomyopathy and kidney function outcomes after agalsidase beta treatment with preceding treatment-naive outcomes. Methods and results: Self-controlled pretreatment and post-treatment comparison (piecewise mixed linear modelling) included Fabry female patients ≥18 years at treatment initiation who received agalsidase beta (0.9–1.1 mg/kg every other week) for ≥2 years, with ≥2 pretreatment and ≥2 post-treatment outcome measurements during 10-year follow-up. Left ventricular posterior wall thickness (LVPWT)/interventricular septal thickness (IVST) and estimated glomerular filtration rate (eGFR, Chronic Kidney Disease Epidemiology Collaboration creatinine equation) analyses included 42 and 86 patients, respectively, aged 50.0 and 46.3 years at treatment initiation, respectively. LVPWT and IVST increased pretreatment (follow-up 3.5 years) but stabilized during 3.6 years of treatment (LVPWT: n = 38, slope difference [95% confidence interval (CI)] = −0.41 [−0.68, −0.15] mm/year, Ppre–post difference pre–post difference = 0.07). These findings were not modified by renal involvement or antiproteinuric agent use. Compared with the treatment-naive period (follow-up 3.6 years), eGFR decline remained modest and stabilized within normal ranges during 4.1 years of treatment (slope difference, 95% CI: −0.13 [−1.15, 0.89] mL/min/1.73m2/year, Ppre–post difference = 0.80). Conclusions: Cardiac hypertrophy, progressing during pretreatment follow-up, appeared to stabilize during sustained agalsidase beta treatment. eGFR decline remained within normal ranges. This suggests that treatment may prevent further Fabry-related progression of cardiomyopathy in female patients and maintain normal kidney function.

Published
2020

14. Early indicators of disease progression in Fabry disease that may indicate the need for disease-specific treatment initiation: findings from the opinion-based PREDICT-FD modified Delphi consensus initiative

Author

Hughes, Derralynn A, primary, Aguiar, Patricio, additional, Deegan, Patrick B, additional, Ezgu, Fatih, additional, Frustaci, Andrea, additional, Lidove, Olivier, additional, Linhart, Aleš, additional, Lubanda, Jean-Claude, additional, Moon, James C, additional, Nicholls, Kathleen, additional, Niu, Dau-Ming, additional, Nowak, Albina, additional, Ramaswami, Uma, additional, Reisin, Ricardo, additional, Rozenfeld, Paula, additional, Schiffmann, Raphael, additional, Svarstad, Einar, additional, Thomas, Mark, additional, Torra, Roser, additional, Vujkovac, Bojan, additional, Warnock, David G, additional, West, Michael L, additional, Johnson, Jack, additional, Rolfe, Mark J, additional, and Feriozzi, Sandro, additional

Published
2020
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18. The Safety of Agalsidase Alfa Enzyme Replacement Therapy in Canadian Patients with Fabry Disease Following Implementation of a Bioreactor Process.

Author

Khan, Aneal, Sirrs, Sandra M., Bichet, Daniel G., Morel, Chantal F., Tocoian, Adina, Lan, Lan, and West, Michael L.

Subjects
ENZYME replacement therapy, ANGIOKERATOMA corporis diffusum, SYMPTOMS, CARDIOVASCULAR diseases, LYSOSOMAL storage diseases, CHILD patients
Abstract

Background and Objective: Fabry disease, an X-linked lysosomal storage disorder characterized by absent or reduced alpha-galactosidase activity, is a lifelong disease that impairs patients' quality of life. Patients with Fabry disease have a considerably shortened lifespan, with mortality being mainly due to renal failure, cardiovascular disease, or cerebrovascular disease. Enzyme replacement therapy with agalsidase alfa has been shown to attenuate the renal, cardiovascular, and neuropathic disease progression associated with Fabry disease. The objective of this study was to investigate the safety of a new animal component-free version of agalsidase alfa. Methods: A phase III/IV, open-label, single-arm, multicenter safety study was conducted in Canadian patients with Fabry disease between August 2011 and September 2017 as a regulatory requirement to assess the safety of agalsidase alfa produced using an animal component-free bioreactor process. Eligible patients had a documented diagnosis of Fabry disease and satisfied current Canadian guidelines for receiving enzyme replacement therapy for Fabry disease. Following treatment with animal component-free bioreactor-processed agalsidase alfa, treatment-emergent adverse events were monitored, and post hoc analyses of infusion-related reactions by antidrug antibody and neutralizing antibody statuses were conducted. The data were analyzed using descriptive statistics. Results: A total of 167 patients (mean [standard deviation] age, 48.9 [14.8] years), including six pediatric patients (< 18 years of age), received at least one full or partial infusion of agalsidase alfa animal component-free. Fewer than 5% of treatment-emergent adverse events (212/4446) observed in 40 patients were reported as infusion-related reactions. Antidrug antibody and neutralizing antibody status did not affect the proportion of patients with infusion-related reactions. No clinically significant changes in vital signs were observed in patients over the course of the study. Conclusions: Long-term treatment with bioreactor-produced agalsidase alfa animal component-free did not reveal new safety signals in this population of Canadian patients with Fabry disease. The treatment-emergent adverse event profile was consistent with the clinical manifestations of the disease and the known safety profile of roller bottle-produced agalsidase alfa. Clinical Trial Registration: ClinicalTrials.gov identifier NCT01298141. [ABSTRACT FROM AUTHOR]

Published
2021
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19. Cardio-renal outcomes with long-term alfa enzyme replacement therapy : a 10-year Fabry outcome survey (FOS) analysis

Author

Ramaswami, Uma, Beck, Michael, Hughes, Derralynn A., Kampmann, Christoph, Botha, Jaco, Pintos-Morell, G., West, Michael L., Niu, Dauming, Nicholls, Kathy, and Giugliani, Roberto

Subjects
Cardio-renal outcomes, Fabry disease, Resultado do tratamento, Enzyme replacement therapy, Terapia de reposição de enzimas, Doença de Fabry, Agalsidase alfa
Abstract

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 analyzed. Estimated glomerular filtration rate (eGFR) and left ventricular mass indexed to height (LVMI) were analyzed after treatment start (baseline) for patients with ≥3 measurements, including baseline and year 10. Results: Median (range) age (years) of the evaluable treated renal cohort at treatment start was 48.8 (17.9–67.3) for females (n=62), 34.4 (18.0–66.8) for males (n=90). With eGFR ≥60 mL/min/1.73 m2 at baseline, mean (95% CI) rate of eGFR change (eGFR/year) over 10 years was relatively stable in females (n=52; −0.55 [−1.12, +0.01]) and slightly declined in males (n=79; −1.99 [−2.45, −1.54]). With impaired kidney function (eGFR 48 g/m2.7 in females, >50 g/m2.7 in males) at baseline, mean (95% CI) LVMI/year slightly increased over 10 years in females (n=18; +1.51 [+0.91, +2.12]) and males (n=14; +0.87 (+0.19, +1.55). Without LVH at baseline, mean (95% CI) LVMI/year was stable in females (n=16; +0.52 [−0.13, +1.17]) and males (n=21; +0.57 [+0.02, +1.13]) over 10 years. Conclusion: Agalsidase alfa-treated patients with 10-year FOS data and preserved kidney function and/or normal LVMI at baseline remained largely stable; those with decreased kidney function or LVH at baseline experienced modest declines in renal function and/or increases in LVMI.

Published
2019

23. Long-term outcomes with agalsidase alfa enzyme replacement therapy : analysis using deconstructed composite events

Author

Beck, Michael, Hughes, Derralynn, Kampmann, Christoph, Pintos-Morell, Guillem, Ramaswami, Uma, West, Michael L., Giugliani, Roberto, and Universitat Autònoma de Barcelona

Subjects
medicine.medical_specialty, Pediatrics, Younger age, Short Communication, Endocrinology, Diabetes and Metabolism, 030232 urology & nephrology, Terapia de reposição de enzimas, Renal function, Left ventricular hypertrophy, Biochemistry, Doença de Fabry, LVH, left ventricular hypertrophy, Long-term effectiveness, 03 medical and health sciences, 0302 clinical medicine, Endocrinology, Internal medicine, medicine, Retrospective analysis, Long term outcomes, Genetics, lcsh:QH301-705.5, Molecular Biology, Agalsidase alfa, lcsh:R5-920, Fabry disease, Egfr, estimated glomerular filtration rate, business.industry, eGFR, estimated glomerular filtration rate, Enzyme replacement therapy, NE, not estimable, medicine.disease, ERT, enzyme replacement therapy, lcsh:Biology (General), lcsh:Medicine (General), business, FOS, Fabry Outcome Survey, 030217 neurology & neurosurgery
Abstract

Altres ajuts: This work was supported by Shire International GmbH. This is a retrospective analysis of Fabry Outcome Survey data from children/adults (n = 677) receiving agalsidase alfa enzyme replacement therapy for a median of 3 years, examining cerebrovascular, cardiac, and renal morbidity endpoints separately. Cardiac events occurred at younger ages than cerebrovascular or renal events, cerebrovascular events were more frequent in females than males, and males were more likely to experience cardiac and renal events at a younger age than females.

Published
2017

25. Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: an international cohort study

Author

Arends, Maarten, primary, Biegstraaten, Marieke, additional, Wanner, Christoph, additional, Sirrs, Sandra, additional, Mehta, Atul, additional, Elliott, Perry M, additional, Oder, Daniel, additional, Watkinson, Oliver T, additional, Bichet, Daniel G, additional, Khan, Aneal, additional, Iwanochko, Mark, additional, Vaz, Frédéric M, additional, van Kuilenburg, André B P, additional, West, Michael L, additional, Hughes, Derralynn A, additional, and Hollak, Carla E M, additional

Published
2018
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26. Prevalence of Fabry Disease and Outcomes in Young Canadian Patients With Cryptogenic Ischemic Cerebrovascular Events

Author

Lanthier, Sylvain, primary, Saposnik, Gustavo, additional, Lebovic, Gerald, additional, Pope, Karen, additional, Selchen, Daniel, additional, Moore, David F., additional, Selchen, Dan, additional, Boulanger, Jean-Martin, additional, Buck, Brian, additional, Butcher, Ken, additional, del Campo, Martin, additional, Gosselin, Sylvie, additional, Hachinski, Vladimir, additional, Hill, Michael D., additional, Mackey, Ariane, additional, Mehdiratta, Manu, additional, Spence, J. David, additional, Stotts, Grant, additional, Swartz, Rick, additional, West, Michael L., additional, and Yegappan, Chidam, additional

Published
2017
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27. Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34 + Cells for Correction of Fabry Disease

Author

Huang, Ju, primary, Khan, Aneal, additional, Au, Bryan C., additional, Barber, Dwayne L., additional, López-Vásquez, Lucía, additional, Prokopishyn, Nicole L., additional, Boutin, Michel, additional, Rothe, Michael, additional, Rip, Jack W., additional, Abaoui, Mona, additional, Nagree, Murtaza S., additional, Dworski, Shaalee, additional, Schambach, Axel, additional, Keating, Armand, additional, West, Michael L., additional, Klassen, John, additional, Turner, Patricia V., additional, Sirrs, Sandra, additional, Rupar, C. Anthony, additional, Auray-Blais, Christiane, additional, Foley, Ronan, additional, and Medin, Jeffrey A., additional

Published
2017
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28. Screening, diagnosis, and management of patients with Fabry disease: conclusions from a “Kidney Disease: Improving Global Outcomes” (KDIGO) Controversies Conference

Author

Schiffmann, Raphael, primary, Hughes, Derralynn A., additional, Linthorst, Gabor E., additional, Ortiz, Alberto, additional, Svarstad, Einar, additional, Warnock, David G., additional, West, Michael L., additional, Wanner, Christoph, additional, Bichet, Daniel G., additional, Christensen, Erik Ilsø, additional, Correa-Rotter, Ricardo, additional, Elliott, Perry M., additional, Feriozzi, Sandro, additional, Fogo, Agnes B., additional, Germain, Dominique P., additional, Hollak, Carla E.M., additional, Hopkin, Robert J., additional, Johnson, Jack, additional, Kantola, Ilkka, additional, Kopp, Jeffrey B., additional, Kröner, Jürgen, additional, Linhart, Aleš, additional, Martins, Ana Maria, additional, Matern, Dietrich, additional, Mehta, Atul B., additional, Mignani, Renzo, additional, Najafian, Behzad, additional, Narita, Ichiei, additional, Nicholls, Kathy, additional, Obrador, Greg T., additional, Oliveira, João Paulo, additional, Pisani, Antonio, additional, Politei, Juan, additional, Ramaswami, Uma, additional, Ries, Markus, additional, Terryn, Wim, additional, Tøndel, Camilla, additional, Torra, Roser, additional, Vujkovac, Bojan, additional, Waldek, Stephen, additional, and Walter, Jerry, additional

Published
2017
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30. Total synthesis of (pm)-calanolide A, a non-nucleoside inhibitor of HIV-1 reverse transcriptase

Author

Chenera, Balan, West, Michael L., Finkelstein, Joseph A., and Dreyer, Geoffrey B.

Subjects
Reverse transcriptase -- Analysis, Enzyme inhibitors -- Research, HIV (Viruses) -- Analysis, Biological sciences, Chemistry
Abstract

A phloroglucinol center is surrounded by coumarin, chromane and chromene ring systems, which are used to determine the properties of the dipyranocoumarins. The genus Calophyllum is the origin of numerous tropical plants that yield the dipyranocoumarins. Human immunodeficiency virus-1 reverse transcriptase (HIV-1 RT) is powerfully hindered by calanolide A and inophyllum B, two dipyranocoumarins revealed by screening assays. The RT mutants which can overcome calanolide A and inophyllum B have been identified, along with other nonnucleoside HIV-1 RT hindering factors such as TIBO and nevirapine.

Published
1993

31. Cardiovascular Events in Patients With Fabry Disease

Author

Patel, Manesh R., primary, Cecchi, Franco, additional, Cizmarik, Marta, additional, Kantola, Ilkka, additional, Linhart, Ales, additional, Nicholls, Kathy, additional, Strotmann, Jörg, additional, Tallaj, Jose, additional, Tran, Thi Chien, additional, West, Michael L., additional, Beitner-Johnson, Dana, additional, and Abiose, Ademola, additional

Published
2011
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32. Therapeutic goals in the treatment of Fabry disease

Author

Mehta, Atul, primary, West, Michael L., additional, Pintos-Morell, Guillem, additional, Reisin, Ricardo, additional, Nicholls, Kathy, additional, Figuera, Luis E., additional, Parini, Rossella, additional, Carvalho, Luiz R., additional, Kampmann, Christoph, additional, Pastores, Gregory M., additional, and Lidove, Olivier, additional

Published
2010
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37. Cardiovascular Events in Patients With Fabry Disease Natural History Data From the Fabry Registry

Author

Patel, Manesh R., Cecchi, Franco, Cizmarik, Marta, Kantola, Ilkka, Linhart, Ales, Nicholls, Kathy, Strotmann, Jörg, Tallaj, Jose, Tran, Thi Chien, West, Michael L., Beitner-Johnson, Dana, and Abiose, Ademola

Subjects
Fabry disease, hypertension, lysosomal storage diseases, cardiomyopathy, left ventricular hypertrophy
Abstract

ObjectivesThese analyses were designed to determine the incidence of major cardiovascular (CV) events and the natural history of CV complications in patients with Fabry disease.BackgroundFabry disease, a genetic disorder caused by deficiency of alpha-galactosidase A activity, is associated with CV dysfunction.MethodsMajor CV events (myocardial infarction, heart failure, or cardiac-related death) were analyzed in 2,869 Fabry Registry patients during the natural history period (i.e., before enzyme replacement therapy or among patients who never received therapy). Multivariate logistic regression analyses were performed to identify significant predictors of CV events.ResultsEighty-three of 1,424 men (5.8%) and 54 of 1,445 women (3.7%) experienced CV events at mean ages of 45 and 54 years, respectively. Heart failure was the most common first CV event, reported by 50 men (3.5%) and 33 women (2.3%). Hypertension and left ventricular hypertrophy were the risk factors most strongly associated with CV events. When these parameters were used as covariates in logistic regression analyses, the odds ratio (OR) for hypertension in men was 7.8 (95% confidence interval [CI]: 2.1 to 28.6, p = 0.0019), and the OR for hypertension in women was 4.5 (95% CI: 1.6 to 12.3, p = 0.0037). The OR for left ventricular hypertrophy was 4.8 in men (95% CI: 1.03 to 22.2, p = 0.0463) and 8.2 in women (95% CI: 2.6 to 26.0, p = 0.0003).ConclusionsMajor CV events occurred in approximately 5% of Fabry Registry patients during the natural history period. All patients with Fabry disease should be monitored for possible CV risk factors, particularly hypertension and left ventricular hypertrophy.

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39. Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events.

Author

Beck M, Hughes D, Kampmann C, Pintos-Morell G, Ramaswami U, West ML, and Giugliani R

Abstract

This is a retrospective analysis of Fabry Outcome Survey data from children/adults ( n  = 677) receiving agalsidase alfa enzyme replacement therapy for a median of 3 years, examining cerebrovascular, cardiac, and renal morbidity endpoints separately. Cardiac events occurred at younger ages than cerebrovascular or renal events, cerebrovascular events were more frequent in females than males, and males were more likely to experience cardiac and renal events at a younger age than females.

Published
2017
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