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INTRODUCTION

ABIM MOC Activity ID

6/12/2018 - Medical Grand Rounds: Bench to Bedside: Mechanism-based treatments for hemoglobinopathies

QUIZ

EVALUATION

CERTIFICATE

INTRODUCTION

Credit Hours: CME 1.00

Target Audience:

Faculty, residents, fellows, and community physicians in General Internal Medicine and subspecialties.

Educational Objectives:

Upon completion of this activity, participants should be able to:

  • Increase patient outcomes through knowledge of current standard of care therapies for sickle cell disease.
  • Improve diagnostic skills for identifying patients at high risk for sickle cell disease morbidity and mortality.
  • Increase patient outcomes through knowledge of current and future potential to refer patients for gene therapy clinical research.
  • List three major mechanisms in the pathophysiology of sickle cell disease
  • List two approaches by which genome editing can ameliorate the pathophysiology of sickle cell disease
  • List two major mechanisms in the pathophysiology of beta thalassemia
  • Explain how protein quality control mechanisms impact the severity of beta thalassemia and how these mechanisms can be manipulated therapeutically.

Suggested Additional Reading:

  1. Traxler EA, Yao Y, Wang YD, Woodard KJ, Kurita R, Nakamura Y, Hughes JR, Hardison RC, Blobel GA, Li C, Weiss MJ. A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition. Nat Med. 2016 Sep;22(9):987-90. doi: 10.1038/nm.4170. Epub 2016 Aug 15. PubMed PMID: 27525524; PubMed Central PMCID: PMC5706766.
  2. Kato GJ, Piel FB, Reid CD, Gaston MH, Ohene-Frempong K, Krishnamurti L, Smith WR, Panepinto JA, Weatherall DJ, Costa FF, Vichinsky EP. Sickle cell disease. Nat Rev Dis Primers. 2018 Mar 15;4:18010. doi: 10.1038/nrdp.2018.10. Review. PubMed PMID: 29542687.
  3. Jayavaradhan R, Malik P. Genetic Therapies for Sickle Cell Disease. Pediatr Clin North Am. 2018 Jun;65(3):465-480. doi: 10.1016/j.pcl.2018.01.008. Review. PubMed PMID: 29803277.

Authors:
Gregory Kato, MD — Professor of Medicine Director, Adult Sickle Cell Center of Excellence Department of Medicine, Division of Hematology-Oncology Heart, Lung, Blood and Vascular Medicine Institute University of Pittsburgh School of Medicine
Dr. Kato receives grant support from Bayer. He is a consultant with Bioverativ, Novartis, Global Blood Therapeutics.
Mitchell J. Weiss, MD, PhD — Chairman, Department of Hematology Arthur Nienhuis Endowed Chair in Hematology St. Jude ChildrenÂ’s Research Hospital
Dr. Weiss receives grant/research support National Institutes of Health, Doris Duke Foundation, St. Jude Children's Research Hospital. He is a consultant with Glaxo SmithKline, Novartis, Beam Therapeutics. Dr. Weiss is a stockholder with Beam Therapeutics, Rubius Inc.
Authors disclosure of relevant financial relationships with any entity producing, marketing, re-selling, or distributing health care goods or services, used on, or consumed by, patients is listed above. No other planners, members of the planning committee, content reviewers and/or anyone else in a position to control the content of this education activity have relevant financial relationships to disclose.

The University of Pittsburgh School of Medicine is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

The University of Pittsburgh School of Medicine designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credits. Each physician should only claim credit commensurate with the extent of their participation in the activity.

The University of Pittsburgh is an affirmative action, equal opportunity institution.