Jais JP, Knebelmann B, Giatras I, et al. X-linked Alport syndrome: natural history in 195 families and genotype- phenotype correlations in males. J Am Soc Nephrol. 2000;11:649-657.
Kashtan CE. Alport syndrome. An inherited disorder of renal, ocular, and cochlear basement membranes. Medicine (Baltimore). 1999;78:338-360.
Storey H, Savige J, Sivakumar V, Abbs S, Flinter FA. COL4A3/COL4A4 mutations and features in individuals with autosomal recessive Alport syndrome. J Am Soc Nephrol. 2013;24:1945-1954.
Pescucci C, Mari F, Longo I, et al. Autosomal-dominant Alport syndrome: natural history of a disease due to COL4A3 or COL4A4 gene. Kidney Int. 2004;65:1598-1603.
Fallerini C, Dosa L, Tita R, et al. Unbiased next generation sequencing analysis confirms the existence of autosomal dominant Alport syndrome in a relevant fraction of cases. Clin Genet. 2014;86:252-257.
van der Loop FT, Heidet L, Timmer ED, et al. Autosomal dominant Alport syndrome caused by a COL4A3 splice site mutation. Kidney Int. 2000;58:1870-1875.
Alport syndrome foundation. http://alportsyndrome.org/what-is-alport-syndrome/
Plevová P, Gut J, Janda J. Familial hematuria: A review. Medicina (Kaunas). 2017;53:1-10.
Lager DJ, Abrahams N. Practical renal pathology, a diagnostic approach: a volume in the pattern recognition series, expert consult: online and print, 1e. January 18, 2013. Pages 113-119.
Bernstein J. The glomerular basement membrane abnormality in Alport's syndrome. Am J Kidney Dis. 1987;10:222-229.
Gubler MC. Inherited diseases of the glomerular basement membrane. Nat Clin Pract Nephrol. 2008;4:24-37.
van der Loop FT, Monnens LA, Schröder CH, et al. Identification of COL4A5 defects in Alport's syndrome by immunohistochemistry of skin. Kidney Int. 1999;55:1217-1224.
Kashtan CE, Ding J, Gregory M, et al. Clinical practice recommendations for the treatment of Alport syndrome: a statement of the Alport Syndrome Research Collaborative. Pediatr Nephrol. 2013;28:5-11.
Kashtan CE. Clinical manifestations, diagnosis, and treatment of Alport syndrome (hereditary nephritis). Available at: https://www.uptodate.com/contents/clinical-manifestations-diagnosis-and-treatment-of-alport-syndrome-hereditary-nephritis?csi=ab26f8cb-7804-4fa4-bcb2-e122693c47f6&source=contentShare Accessed on January 16, 2018.
Chau BN, Xin C, Hartner J, et al. MicroRNA-21 promotes fibrosis of the kidney by silencing metabolic pathways. Sci Transl Med. 2012;4:121ra18.
Gomez IG, MacKenna DA, Johnson BG, et al. Anti-microRNA-21 oligonucleotides prevent Alport nephropathy progression by stimulating metabolic pathways. J Clin Invest. 2015;125:141-156.
Gomez IG, Grafals M, Portilla D, Duffield JS. MicroRNAs as potential therapeutic targets in kidney disease. J Formos Med Assoc. 2013;112:237-243.
Rangan G, Wang Y, Harris D. NF-kappaB signalling in chronic kidney disease. Front Biosci (Landmark Ed). 2009;14:3496-522.
Pedruzzi LM, Stockler-Pinto MB, Leite M Jr, Mafra D. Nrf2-keap1 system versus NF-κB: the good and the evil in chronic kidney disease? Biochimie. 2012;94:2461-2466.
Kim HJ, Vaziri ND. Contribution of impaired Nrf2-Keap1 pathway to oxidative stress and inflammation in chronic renal failure. Am J Physiol Renal Physiol. 2010;298:F662-F671.
Tapia E, Zatarain-Barron ZL, Hernandez-Pando R, et al. Curcumin reverses glomerular hemodynamic alterations and oxidant stress in 5/6 nephrectomized rats. Phytomedicine. 2013;20:359-366.
Shelton LM, Lister A, Walsh J, et al. Integrated transcriptomic and proteomic analyses uncover regulatory roles of Nrf2 in the kidney. Kidney Int. 2015;88:1261-1273.
Suzuki T, Motohashi H, Yamamoto M. Toward clinical application of the Keap1-Nrf2 pathway. Trends Pharmacol Sci. 2013;34:340-346.
Pergola PE, Raskin P, Toto RD, et al. Bardoxolone methyl and kidney function in CKD with type 2 diabetes. N Engl J Med. 2011;365:327-336.
de Zeeuw D, Akizawa T, Audhya P, et al. Bardoxolone methyl in type 2 diabetes and stage 4 chronic kidney disease. N Engl J Med. 2013;369:2492-2503.
Chin MP, Reisman SA, Bakris GL, et al. Mechanisms contributing to adverse cardiovascular events in patients with type 2 diabetes mellitus and stage 4 chronic kidney disease treated with bardoxolone methyl. Am J Nephrol. 2014;39:499-508.
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Moderator

Matthew R. Weir, MD

Professor of Medicine; Director, Division of Nephrology, University of Maryland School of Medicine, Baltimore

Disclosures

Disclosure: Matthew R. Weir, MD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: Akebia Therapeutics; AstraZeneca Pharmaceuticals LP; Janssen Pharmaceuticals; Merck Sharp & Dohme Corp.; Relypsa, Inc.; Sanofi

Dr Weir does not intend to discuss off-label uses of drugs, mechanical devices, biologics, or diagnostics approved by the FDA for use in the United States.

Dr Weir does intend to discuss investigational drugs, mechanical devices, biologics, or diagnostics not approved by the FDA for use in the United States.

Panelists

Daniel W. Coyne, MD

Professor of Medicine; Medical Director, Chromalloy American Kidney Center; Director, Medicine Multispecialty Outpatient Center, Barnes-Jewish Hospital, Washington University School of Medicine, St. Louis, Missouri

Disclosures

Disclosure: Daniel W. Coyne, MD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: Fresenius Medical Care North America; GlaxoSmithKline; MediBeacon; Novadiol; Reata Pharmaceuticals, Inc.
Served as a speaker or a member of a speakers bureau for: Fresenius Medical Care North America
Received grants for clinical research from: FibroGen, Inc.; GlaxoSmithKline; Janssen Pharmaceuticals; MediBeacon

Dr Coyne does not intend to discuss off-label uses of drugs, mechanical devices, biologics, or diagnostics approved by the FDA for use in the United States.

Dr Coyne does intend to discuss investigational drugs, mechanical devices, biologics, or diagnostics not approved by the FDA for use in the United States.

Pablo E. Pergola, MD, PhD

Professor of Medicine, UT Health San Antonio; Research Director, Renal Associates PA and Clinical Associate, San Antonio, Texas

Disclosures

Disclosure: Pablo E. Pergola, MD, PhD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: AbbVie Inc.; Akebia Therapeutics; Gilead Sciences, Inc.; Keryx Biopharmaceuticals, Inc.; Vifor Pharma
Served as a speaker or a member of a speakers bureau for: Keryx Biopharmaceuticals, Inc.

Dr Pergola does not intend to discuss off-label uses of drugs, mechanical devices, biologics, or diagnostics approved by the FDA for use in the United States.

Dr Pergola does intend to discuss investigational drugs, mechanical devices, biologics, or diagnostics not approved by the FDA for use in the United States.

Editor

Anne G. Le, PharmD

Senior Scientific Director, Medscape, LLC

Disclosures

Disclosure: Anne G. Le, PharmD, has disclosed no relevant financial relationships.

CME Reviewer

Nafeez Zawahir, MD

CME Clinical Director, Medscape, LLC

Disclosures

Disclosure: Nafeez Zawahir, MD, has disclosed no relevant financial relationships.

CME / ABIM MOC

Advances in CKD: Focus on Alport Syndrome

Authors: Matthew R. Weir, MD; Daniel W. Coyne, MD; Pablo E. Pergola, MD, PhD
CME / ABIM MOC Released: 2/28/2018
THIS ACTIVITY HAS EXPIRED FOR CREDIT
Valid for credit through: 2/28/2019

Start Activity

Target Audience and Goal Statement

This activity is intended for nephrologists, internal medicine physicians, primary care physicians, nurse practitioners, physician assistants, nurses, and other clinicians involved in the management of nephrology disorders such as Alport syndrome.

The goal of this activity is to increase awareness of current and emerging clinical data and therapeutic strategies that have the potential to change current treatment paradigms for patients with Alport syndrome.

Upon completion of this activity, participants will:

Have increased knowledge regarding the
- Current management strategies used in Alport syndrome
- Latest data from clinical trials of emerging therapies for Alport syndrome treatment
Demonstrate greater confidence in their ability to
- Manage patients with Alport syndrome

Disclosures

Moderator

Matthew R. Weir, MD

Professor of Medicine; Director, Division of Nephrology, University of Maryland School of Medicine, Baltimore

Disclosures

Disclosure: Matthew R. Weir, MD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: Akebia Therapeutics; AstraZeneca Pharmaceuticals LP; Janssen Pharmaceuticals; Merck Sharp & Dohme Corp.; Relypsa, Inc.; Sanofi

Dr Weir does not intend to discuss off-label uses of drugs, mechanical devices, biologics, or diagnostics approved by the FDA for use in the United States.

Dr Weir does intend to discuss investigational drugs, mechanical devices, biologics, or diagnostics not approved by the FDA for use in the United States.

Panelists

Daniel W. Coyne, MD

Professor of Medicine; Medical Director, Chromalloy American Kidney Center; Director, Medicine Multispecialty Outpatient Center, Barnes-Jewish Hospital, Washington University School of Medicine, St. Louis, Missouri

Disclosures

Disclosure: Daniel W. Coyne, MD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: Fresenius Medical Care North America; GlaxoSmithKline; MediBeacon; Novadiol; Reata Pharmaceuticals, Inc.
Served as a speaker or a member of a speakers bureau for: Fresenius Medical Care North America
Received grants for clinical research from: FibroGen, Inc.; GlaxoSmithKline; Janssen Pharmaceuticals; MediBeacon

Dr Coyne does not intend to discuss off-label uses of drugs, mechanical devices, biologics, or diagnostics approved by the FDA for use in the United States.

Dr Coyne does intend to discuss investigational drugs, mechanical devices, biologics, or diagnostics not approved by the FDA for use in the United States.

Pablo E. Pergola, MD, PhD

Professor of Medicine, UT Health San Antonio; Research Director, Renal Associates PA and Clinical Associate, San Antonio, Texas

Disclosures

Disclosure: Pablo E. Pergola, MD, PhD, has disclosed the following relevant financial relationships:
Served as an advisor or consultant for: AbbVie Inc.; Akebia Therapeutics; Gilead Sciences, Inc.; Keryx Biopharmaceuticals, Inc.; Vifor Pharma
Served as a speaker or a member of a speakers bureau for: Keryx Biopharmaceuticals, Inc.

Dr Pergola does not intend to discuss off-label uses of drugs, mechanical devices, biologics, or diagnostics approved by the FDA for use in the United States.

Dr Pergola does intend to discuss investigational drugs, mechanical devices, biologics, or diagnostics not approved by the FDA for use in the United States.

Editor

Anne G. Le, PharmD

Senior Scientific Director, Medscape, LLC

Disclosures

Disclosure: Anne G. Le, PharmD, has disclosed no relevant financial relationships.

CME Reviewer

Nafeez Zawahir, MD

CME Clinical Director, Medscape, LLC

Disclosures

Disclosure: Nafeez Zawahir, MD, has disclosed no relevant financial relationships.

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From Medscape Education Nephrology

CME / ABIM MOC

Advances in CKD: Focus on Alport Syndrome

Authors: Matthew R. Weir, MD; Daniel W. Coyne, MD; Pablo E. Pergola, MD, PhDFaculty and Disclosures

THIS ACTIVITY HAS EXPIRED FOR CREDIT

CME / ABIM MOC Released: 2/28/2018

Valid for credit through: 2/28/2019

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The educational activity presented above may involve simulated case-based scenarios. The patients depicted in these scenarios are fictitious and no association with any actual patient is intended or should be inferred.

The material presented here does not necessarily reflect the views of Medscape, LLC, or companies that support educational programming on medscape.org. These materials may discuss therapeutic products that have not been approved by the US Food and Drug Administration and off-label uses of approved products. A qualified healthcare professional should be consulted before using any therapeutic product discussed. Readers should verify all information and data before treating patients or employing any therapies described in this educational activity.

References

Faculty and Disclosures

Moderator

Matthew R. Weir, MD

Panelists

Daniel W. Coyne, MD

Pablo E. Pergola, MD, PhD

Editor

Anne G. Le, PharmD

CME Reviewer

Nafeez Zawahir, MD

CME / ABIM MOC

Advances in CKD: Focus on Alport Syndrome

Target Audience and Goal Statement

Disclosures

Moderator

Matthew R. Weir, MD

Panelists

Daniel W. Coyne, MD

Pablo E. Pergola, MD, PhD

Editor

Anne G. Le, PharmD

CME Reviewer

Nafeez Zawahir, MD

Accreditation Statements

For Physicians

Instructions for Participation and Credit

Advances in CKD: Focus on Alport Syndrome