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52. 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

54. 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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55. Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: An international cohort study

Author

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

Published
2018
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56. 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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57. 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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58. 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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62. Chronic kidney disease and an uncertain diagnosis of Fabry disease: Approach to a correct diagnosis

Author

van der Tol, Linda, primary, Svarstad, Einar, additional, Ortiz, Alberto, additional, Tøndel, Camilla, additional, Oliveira, João Paulo, additional, Vogt, Liffert, additional, Waldek, Stephen, additional, Hughes, Derralynn A., additional, Lachmann, Robin H., additional, Terryn, Wim, additional, Hollak, Carla E., additional, Florquin, Sandrine, additional, van den Bergh Weerman, Marius A., additional, Wanner, Christoph, additional, West, Michael L., additional, Biegstraaten, Marieke, additional, and Linthorst, Gabor E., additional

Published
2015
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63. Urinary biomarker investigation in children with Fabry disease using tandem mass spectrometry

Author

Auray-Blais, Christiane, primary, Blais, Catherine-Marie, additional, Ramaswami, Uma, additional, Boutin, Michel, additional, Germain, Dominique P., additional, Dyack, Sarah, additional, Bodamer, Olaf, additional, Pintos-Morell, Guillem, additional, Clarke, Joe T.R., additional, Bichet, Daniel G., additional, Warnock, David G., additional, Echevarria, Lucia, additional, West, Michael L., additional, and Lavoie, Pamela, additional

Published
2015
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65. Consensus recommendation on Fabry disease diagnosis in adult patients with kidney disease

Author

van der Tol, Linda, primary, Biegstraaten, Marieke, additional, Florquin, Sandrine, additional, Vogt, Liffert, additional, van den Bergh Weerman, Marius A., additional, Hollak, Carla E.M., additional, Hughes, Derralynn A., additional, Lachmann, Robin H., additional, Oliveira, João P., additional, Ortiz, Alberto A., additional, Svarstad, Einar, additional, Terryn, Wim, additional, Tøndel, Camilla, additional, Waldek, Stephen, additional, Wanner, Christoph, additional, West, Michael L., additional, and Linthorst, Gabor E., additional

Published
2014
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67. 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

68. 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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69. How well does urinary lyso-Gb3 function as a biomarker in Fabry disease?

Author

Auray-Blais, Christiane, primary, Ntwari, Aimé, additional, Clarke, Joe T.R., additional, Warnock, David G., additional, Oliveira, João Paulo, additional, Young, Sarah P., additional, Millington, David S., additional, Bichet, Daniel G., additional, Sirrs, Sandra, additional, West, Michael L., additional, Casey, Robin, additional, Hwu, Wuh-Liang, additional, Keutzer, Joan M., additional, Zhang, X. Kate, additional, and Gagnon, René, additional

Published
2010
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70. 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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73. Orthogonally Protected Monosaccharide Building Blocks for Solid Phase Production of Diversity Oriented Libraries

Author

Rajaratnam, Premraj, primary, Gupta, Praveer, additional, Katavic, Peter, additional, Kuipers, Krystle, additional, Huyh, Ngoc, additional, Ryan, Sarah, additional, Falzun, Tania, additional, Tometzki, Gerrald B., additional, Bornaghi, Laurent, additional, Le Thanh, Giang, additional, Abbenante, Giovanni, additional, Liu, Ligong, additional, Meutermans, Wim, additional, Wimmer, Norbert, additional, and West, Michael L., additional

Published
2010
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74. Urinary globotriaosylceramide excretion correlates with the genotype in children and adults with Fabry disease

Author

Auray-Blais, Christiane, primary, Cyr, Denis, additional, Ntwari, Aimé, additional, West, Michael L., additional, Cox-Brinkman, Josanne, additional, Bichet, Daniel G., additional, Germain, Dominique P., additional, Laframboise, Rachel, additional, Melançon, Serge B., additional, Stockley, Tracy, additional, Clarke, Joe T.R., additional, and Drouin, Régen, additional

Published
2008
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79. Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34+ Cells for Correction of Fabry Disease

Author

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

Subjects
lysosomal storage disease, α-galactosidase A, hematopoietic stem/progenitor cell, xenotransplantation, GMP production
Abstract

Fabry disease is a rare lysosomal storage disorder (LSD). We designed multiple recombinant lentivirus vectors (LVs) and tested their ability to engineer expression of human α-galactosidase A (α-gal A) in transduced Fabry patient CD34+ hematopoietic cells. We further investigated the safety and efficacy of a clinically directed vector, LV/AGA, in both ex vivo cell culture studies and animal models. Fabry mice transplanted with LV/AGA-transduced hematopoietic cells demonstrated α-gal A activity increases and lipid reductions in multiple tissues at 6 months after transplantation. Next we found that LV/AGA-transduced Fabry patient CD34+ hematopoietic cells produced even higher levels of α-gal A activity than normal CD34+ hematopoietic cells. We successfully transduced Fabry patient CD34+ hematopoietic cells with “near-clinical grade” LV/AGA in small-scale cultures and then validated a clinically directed scale-up transduction process in a GMP-compliant cell processing facility. LV-transduced Fabry patient CD34+ hematopoietic cells were subsequently infused into NOD/SCID/Fabry (NSF) mice; α-gal A activity corrections and lipid reductions were observed in several tissues 12 weeks after the xenotransplantation. Additional toxicology studies employing NSF mice xenotransplanted with the therapeutic cell product demonstrated minimal untoward effects. These data supported our successful clinical trial application (CTA) to Health Canada and opening of a “first-in-the-world” gene therapy trial for Fabry disease.

Published
2017
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93. 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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96. How well does urinary lyso-Gb3 function as a biomarker in Fabry disease?

Author

Auray-Blais, Christiane, Ntwari, Aimé, Clarke, Joe T.R., Warnock, David G., Oliveira, João Paulo, Young, Sarah P., Millington, David S., Bichet, Daniel G., Sirrs, Sandra, West, Michael L., Casey, Robin, Hwu, Wuh-Liang, Keutzer, Joan M., Zhang, X. Kate, and Gagnon, René

Subjects
*LYSOSOMAL storage diseases, *BIOMARKERS, *URINALYSIS, *GLYCOSPHINGOLIPIDS, *MASS spectrometry, *LIQUID chromatography
Abstract

Abstract: Background: Fabry disease is characterized by accumulation of glycosphingolipids, such as globotriaosylceramide (Gb3), in many tissues and body fluids. A novel plasma biomarker, globotriaosylsphingosine (lyso-Gb3), is increased in patients with the disease. Until now, lyso-Gb3 was not detectable in urine, possibly because of the presence of interfering compounds. Methods: We undertook to: 1) characterize lyso-Gb3 in urine; 2) develop a method to quantitate urinary lyso-Gb3 by mass spectrometry; 3) evaluate urinary lyso-Gb3 as a potential biomarker for Fabry disease; and 4) determine whether lyso-Gb3 is an inhibitor of α-galactosidase A activity. We analyzed urinary lyso-Gb3 from 83 Fabry patients and 77 healthy age-matched controls. Results: The intraday and interday bias and precision of the method were <15%. Increases in lyso-Gb3/creatinine correlated with the concentrations of Gb3 (r 2 =0.43), type of mutations (p =0.0006), gender (p <0.0001) and enzyme replacement therapy status (p =0.0012). Urine from healthy controls contained no detectable lyso-Gb3. Lyso-Gb3 did not inhibit GLA activity in dried blood spots. Increased urinary excretion of lyso-Gb3 of Fabry patients correlated well with a number of indicators of disease severity. Conclusion: Lyso-Gb3 is a reliable independent biomarker for clinically important characteristics of Fabry disease. [ABSTRACT FROM AUTHOR]

Published
2010
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97. Renal and multisystem effectiveness of 3.9 years of migalastat in a global real-world cohort: Results from the followME Fabry Pathfinders registry.

Author

Hughes DA, Sunder-Plassmann G, Jovanovic A, Brand E, West ML, Bichet DG, Pisani A, Nowak A, Torra R, Khan A, Azevedo O, Lehman A, Linhart A, Rutecki J, Giuliano JD, Krusinska E, and Nordbeck P

Abstract

Fabry disease is a progressive, X-linked lysosomal disorder caused by reduced or absent α-galactosidase A activity due to GLA variants. The effects of migalastat were examined in a cohort of 125 Fabry patients with migalastat-amenable GLA variants in the followME Pathfinders registry (EUPAS20599), an ongoing, prospective, patient-focused registry evaluating outcomes for current Fabry disease treatments. We report annualised estimated glomerular filtration rate (eGFR) and Fabry-associated clinical events (FACEs) in a cohort of patients who had received ≥3 years of migalastat treatment in a real-world setting. As of August 2022, 125 patients (60% male) had a mean migalastat exposure of 3.9 years. At enrolment, median age was 58 years (males, 57; females, 60) with a mean eGFR of 83.7 mL/min/1.73 m 2 (n = 122; males, 83.7; females, 83.8) and a median left ventricular mass index of 115.1 g/m 2 (n = 61; males, 131.2; females, 98.0). Mean (95% confidence interval) eGFR annualised rate of change in the overall cohort (n = 116) was -0.9 (-10.8, 9.9) mL/min/1.73 m 2 /year with a similar rate of change observed across patients with varying levels of kidney function at enrolment. Despite population age and baseline morbidity, 80% of patients did not experience a FACE during the mean 3.9 years of migalastat exposure. The incidence of renal, cardiac, and cerebrovascular events was 2.0, 83.2, and 4.1 events per 1000 patient-years, respectively. These data support a role of migalastat in preserving renal function and multisystem effectiveness during ≥3 years of migalastat treatment in this real-world Fabry population., (© 2024 The Author(s). Journal of Inherited Metabolic Disease published by John Wiley & Sons Ltd on behalf of SSIEM.)

Published
2024
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98. Use of denosumab to treat refractory hypercalcemia in a peritoneal dialysis patient with immobilization and tertiary hyperparathyroidism.

Author

Pratt RM, West ML, and Tennankore KK

Subjects
Aged, Humans, Hypercalcemia complications, Hyperparathyroidism, Secondary therapy, Kidney Failure, Chronic therapy, Male, Mobility Limitation, Bone Density Conservation Agents therapeutic use, Denosumab therapeutic use, Hypercalcemia drug therapy, Hyperparathyroidism, Secondary complications, Kidney Failure, Chronic complications, Peritoneal Dialysis
Abstract

Hypercalcemia due to excess parathyroid hormone (PTH) production is a common condition among patients with end-stage renal disease (ESRD), often referred to as tertiary hyperparathyroidism. There are limited effective medical treatment options currently available for such patients. Denosumab is a monoclonal antibody that inhibits osteoclast activation, thereby reducing calcium release from bones. Denosumab has been used to treat medically-refractory hypercalcemia in non-ESRD patients with hyperparathyroidism. Denosumab has also been used to treat non-PTH-mediated hypercalcemia in patients with advanced chronic kidney disease and ESRD. In this case report, we describe the use of denosumab to successfully treat a case of medically refractory hypercalcemia due to immobilization in a patient on peritoneal dialysis with severe underlying tertiary hyperparathyroidism. In spite of persistently elevated PTH, hypercalcemia quickly resolved after a single dose of denosumab. The patient subsequently developed temporary hypocalcemia requiring medical intervention. Our case report, which is the first described use of denosumab for treatment of hypercalcemia in the setting of tertiary hyperparathyroidism in a peritoneal dialysis patient, adds to the body of literature suggesting denosumab is a useful therapeutic agent in patients with ESRD. Issues with post-treatment electrolyte management and other therapeutic considerations are also discussed.

Published
2020
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99. 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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