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CME

Key Advances in Rare Blood Diseases in 2024: A Year in Review

Clinical Thought

Read this commentary covering some of the most impactful findings from 2024 on the care of patients with rare blood disorders including hemophilia, immune thrombocytopenia, and sickle cell disease.

Physicians: Maximum of 0.25 AMA PRA Category 1 Credit

Released: January 30, 2025

Expiration: January 29, 2026

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Provided by Clinical Care Options, LLC

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Supporters

Supported by an educational grant from Novo Nordisk.

Novo Nordisk

Target Audience

This activity is intended for hematologists and other healthcare professionals who diagnose and treat patients with rare blood diseases.

Program Learning Goal

The goal of this activity is to improve the knowledge and competence of learners in the integration of current and emerging data into practice to improve clinical outcomes in patients with rare blood diseases.

Learning Objectives

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

  • Integrate current and emerging data on rare blood diseases (including but not limited to: hemophilia, aTTP/iTTP, beta thalassemia, CAD, HUS, ITP, PNH, and SCD) into clinical practice

  • Evaluate the impact of data presented at national and global conferences and congresses in 2024 on treatment algorithms for patients with rare nonmalignant hematologic diseases

Disclosure

Clinical Care Options, LLC (CCO) requires instructors, planners, managers, and other individuals who are in a position to control the content of this activity to disclose all financial conflicts of interest (COI) they may have with ineligible companies. All relevant COI are thoroughly vetted and mitigated according to CCO policy. CCO is committed to providing its learners with high-quality CME/CE activities and related materials that promote improvements or quality in healthcare and not a specific proprietary business interest of an ineligible company.

The faculty reported the following relevant financial relationships or relationships to products or devices they have with ineligible companies related to the content of this educational activity:

Primary Author

Hanny Al-Samkari, MD

Classical Hematologist and Clinical Investigator
The Peggy S. Blitz Endowed Chair in Hematology/Oncology
Co-Director, Hereditary Hemorrhagic Telangiectasia Center
Massachusetts General Hospital
Associate Professor of Medicine
Harvard Medical School
Boston, Massachusetts

Hanny Al-Samkari, MD: consultant/advisor/speaker: Agios, Alnylam, Alpine, Amgen, Argenx, Novartis, Pharmacosmos, Sanofi, Sobi; researcher (paid to institution): Agios, Amgen, Novartis, Sobi, Vaderis.

Catherine M. Broome, MD

Professor of Medicine
Director of Cellular Apheresis
Lombardi Cancer Center
MedStar Georgetown University
Washington, DC

Catherine M. Broome, MD: consultant/advisor/speaker: Alexion, Argenx, Sanofi; researcher: Alpine.

Guy Young, MD

Director, Hemostasis and Thrombosis Center
Director, Clinical Coagulation Laboratory
Cancer and Blood Disorders Institute
Children's Hospital Los Angeles
Professor of Pediatrics
Division of Hematology/Oncology
Department of Pediatrics
University of Southern California Keck School of Medicine
Los Angeles, California

Guy Young, MD: consultant/advisor/speaker: ASC Biotherapeutics, Biomarin, CSL Behring, Genentech/Roche, Hema Biologics, Novo Nordisk, Octapharma, Pfizer, Sanofi Genzyme, Spark, Takeda.

The planners and content peer reviewers from Clinical Care Options, LLC do not have any relevant financial relationships to disclose.

Instructions for Credit

Participation in this self-study activity should be completed in approximately 0.25 hours. To successfully complete this activity and receive credit, participants must follow these steps during the period from January 30, 2025, through January 29, 2026:

  1. Login or Sign Up for an account by clicking at the top of this page.

  2. Read the target audience, learning objectives, and faculty disclosures.

  3. View and study the content in its entirety.

  4. Submit answers to the posttest questions and evaluation questions online.

You must receive a test score of at least 65 and respond to all evaluation questions to receive a certificate. After submitting the evaluation, you may access your online certificate by selecting the certificate link on the confirmation page. Records of all CME/CE activities completed can be found on the "My Certificates" page. There are no costs/fees for this activity.

Format

This program has been made available online.

Disclosure of Unlabeled Use

This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do not recommend the use of any agent outside of the labeled indications.

The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.

Disclaimer

Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications and/or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.

Accreditation

Joint Accreditation Statement

In support of improving patient care, Clinical Care Options, LLC (CCO) is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

Physician Continuing Medical Education

CCO designates this enduring material for a maximum of 0.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

CME Passport

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The CME credits that physicians earn from this activity will be submitted to ACCME's CME Passport, a free, centralized web application where you can create a personalized account to view, track, and generate transcripts of your reported CME credit. Visit www.cmepassport.org to create your account.