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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.
No other members of the planning committee, speakers, presenters, authors, content reviewers and/or anyone else in a position to control the content of this education activity have relevant financial relationships with any companies whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients.

This activity is approved for AMA PRA Category 1 Credit™

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