Accreditation/Credit Designation

Physicians' Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Physicians' Education Resource®, LLC, designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Acknowledgment of Commercial Support

This activity is supported by an educational grant from Novartis Pharmaceuticals Corporation.

Advances in™ Sickle Cell Disease: Assessing the Impact of Emerging Agents

Release Date: September 30, 2019
Expiration Date: September 30, 2020
Media: Internet - based

Activity Overview

Sickle cell disease (SCD) is a continually increasing health problem, with approximately 300,000 individuals born annually worldwide with this genetic disease. A defect in the HBB gene on chromosome 11 results in the production of a form of hemoglobin that is less soluble than normal fetal or adult hemoglobin. Lifelong complications resulting from this mutation include anemia, infections, stroke, tissue damage, organ failure, intense painful episodes, and a shorter life expectancy. The intensive treatment needs and unpredictable, debilitating symptoms of people with SCD impose limitations on education, careers, and overall quality of life. Although hematopoietic stem cell transplantation can cure SCD, it is not feasible for most patients. However, new therapies in clinical trials offer targeted, pathophysiologic approaches to improve the quality and duration of life for people with SCD.

Advances in™ Sickle Cell Disease: Assessing the Impact of Emerging Agents will help clinicians apply a guideline-based approach for management of acute and chronic pain as well as the complications of SCD through a series of video interviews with 2 experts in SCD. The essentials of preventive care, multidisciplinary communication of treatment plans, importance of patient education, and efficacy and safety of emerging agents for treatment of SCD will be discussed.

Benefits of Participating

  • Increase knowledge and competence regarding preventive health maintenance and pain treatment care plans for people with SCD
  • Learn strategies to effectively manage acute pain episodes in the emergency department
  • Build confidence in your knowledge of how to optimally treat various complications of SCD
  • Understand the pathophysiologic approaches of emerging and investigative agents

Acknowledgement of Commercial Support

This activity is supported by an educational grant from Novartis Pharmaceuticals Corporation.

Instructions for This Activity and Receiving Credit

  • You will need to log in to participate in the activity.
  • Each presentation may contain an interactive question(s). You may move forward through the presentation; however, you may not go back to change answers or review videos/content until you finish the presentation.
  • At the end of the activity, “Educational Content/Video” will be available for your reference.
  • In order to receive a CME Certificate, you must complete the activity.
  • Complete the Posttest and pass with a score of 70% or higher, complete the Evaluation, and then click on “Request for Credit.” You may immediately download a CME Certificate upon completion of these steps.

Target Audience

This online educational activity is directed toward emergency medicine physicians, hospitalists, hematologists, nurse practitioners, physician assistants, nurses, and other health care professionals who would be involved in the diagnosis and management of SCD.

Learning Objectives

Upon successful completion of this educational activity, you should be better prepared to:

  • Summarize current guidelines for management of pain and complications due to SCD in affected patients.
  • Evaluate the efficacy and tolerability of agents for SCD being investigated in clinical trials.

Faculty, Staff, and Planners’ Disclosures

Faculty

Jason W. Wilson
Jason W. Wilson, MD, MA, FACEP, FAAEM
Medical Director, Clinical Decision Unit
Associate Medical Director, Adult Education
Research Director, Emergency Medicine
Associate Professor, Division of Emergency Medicine
Department of Internal Medicine
Morsani College of Medicine at the University of South Florida
Tampa General Hospital
Tampa, FL

Disclosures: Grant Research Support: Gilead, Boehringer Ingelheim, Portola; Consultant: Janssen, Pfizer, Gilead, Portola; Speakers Bureau: Janssen, Pfizer, Gilead, Portola.

Matthew M. Heeney
Matthew M. Heeney, MD
Associate Chief, Hematology
Director, Sickle Cell Program
Boston Children’s Hospital
Assistant Professor, Pediatrics
Harvard Medical School
Boston, MA

Disclosures: Grant Research Support: AstraZeneca, Sancilio/Micelle BioPharma, Pfizer, Novartis, Ironwood/Cyclerion, Intrinsic LifeSciences; Consultant: AstraZeneca, Sancilio/Micelle Biopharma, Novartis; Other: Advisory Board: AstraZeneca, Sancilio/Micella Biopharma, Novartis; Data Safety Monitoring Board: Vertex/CRISPR Therapeutics.

PER uses a blinded peer review process. The peer reviewer discloses the following: Peer reviewer has no relevant financial relationships with commercial interests to disclose.

The staff of Physicians' Education Resource®, LLC (PER®), have no relevant financial relationships with commercial interests to disclose.

Disclosure Policy and Resolution of Conflicts of Interest (COI)

As a sponsor accredited by the ACCME, it is the policy of PER® to ensure fair balance, independence, objectivity, and scientific rigor in all of its CME activities. In compliance with ACCME guidelines, PER® requires everyone who is in a position to control the content of a CME activity to disclose all relevant financial relationships with commercial interests. The ACCME defines “relevant financial relationships” as financial relationships in any amount occurring within the past 12 months that creates a COI.

Additionally, PER® is required by ACCME to resolve all COI. PER® has identified and resolved all COI prior to the start of this activity by using a multistep process.

Off-Label Disclosure and Disclaimer

This CME activity may or may not discuss investigational, unapproved, or off-label use of drugs. Participants are advised to consult prescribing information for any products discussed. The information provided in this CME activity is for continuing medical and nursing education purposes only and is not meant to substitute for the independent clinical judgment of a physician and nurse relative to diagnostic or treatment options for a specific patient’s medical condition.

The opinions expressed in the content are solely those of the individual faculty members, and do not reflect those of PER® or the company that provided commercial support for this program.

PER Pulse™ Recaps

1 of 3

Advances in™ Sickle Cell Disease: Assessing the Impact of Emerging Agents. In this certified continuing medical education activity, Matthew M. Heeney, MD, and Jason W. Wilson, MD, MA, FACEP, FAAEM, discuss the newest evidence-based clinical strategies and emerging treatments for children and adults with sickle cell disease (SCD).

To access this activity, go to https://www.gotoper.com/online-cme-activities/ai/ai-scd19?#registration.

This first of 3 PER Pulse™ Recaps focuses on proven strategies for best care of children with SCD. Some key points are below.

  • SCD is usually identified during standard newborn screening. It is the most common inherited blood disorder.
  • Symptoms commonly include severe anemia, infection, stroke, organ failure, and vaso-occlusive crises resulting in tissue damage and intense pain.
  • Severe episodes can begin in childhood, increasing the risk of multisystem acute and chronic conditions.
  • Up-to-date SCD guidelines recommend1 the following:
    • Newborn referral to pediatric hematology
    • Proactive multispecialty care (primary care physicians/pediatricians, hematology specialists, pharmacists, emergency departments, laboratories, and hospitals) to promote best care and patient outcomes
    • A comprehensive approach to care, including patient and family education, scheduled health maintenance visits, periodic comprehensive evaluations, genetic counseling, and psychosocial care to help with coping
    • Appropriate treatment of even minor acute illness because complications can develop rapidly
    • Infection prophylaxis and vaccination to reduce infection risk
    • Transcranial Doppler ultrasound to evaluate central nervous system (CNS) blood flow rates
      • Stroke prevention in children with high CNS blood flow rates by monthly transfusions to reduce sickle hemoglobin levels
    • Individualized pain management regimens by hematologists, including home care for mild to moderate pain. Severe pain is most successfully treated by patient-controlled analgesia and may require hospitalization.2
    • Care transition plans between pediatric and adult care providers as children mature, for care continuity and best outcomes

“Among the most important preventive interventions [for children with SCD] are those that reduce risk for stroke and severe infection.”—Matthew M. Heeney, MD

To access this activity, go to https://www.gotoper.com/online-cme-activities/ai/ai-scd19?#registration.

References

  1. Yawn BP, Buchanan GR, Afenyi-Annan AN, et al. Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members. JAMA. 2014;312(10):1033-1048. doi: 10.1001/jama.2014.10517.
  2. Uwaezuoke SN, Ayuk AC, Ndu IK, Eneh CI, Mbanefo NR, Ezenwosu OU. Vaso-occlusive crisis in sickle cell disease: current paradigm on pain management. J Pain Res. 2018;11:3141-3150. doi: 10.2147/JPR.S185582.

2 of 3

Advances in™ Sickle Cell Disease: Assessing the Impact of Emerging Agents. In this certified continuing medical education activity, Matthew M. Heeney, MD, and Jason W. Wilson, MD, MA, FACEP, FAAEM, discuss the newest evidence-based clinical strategies and emerging treatments for children and adults with sickle cell disease (SCD).

To access this activity, go to https://www.gotoper.com/online-cme-activities/ai/ai-scd19?#registration.

This second of 3 PER Pulse™ Recaps summarizes care principles proven to reduce lifetime morbidity risk in SCD. Some key points are below.

  • SCD is a common inherited blood disorder. Symptoms often include severe anemia, infection, stroke, organ failure, and vaso-occlusive crises resulting in tissue damage and intense pain.
  • Widespread vascular effects of SCD place patients at high risk of multisystem acute and chronic conditions and decreased life expectancy.
  • Up-to-date clinical evidence and guideline recommendations include the following:
    • Hydroxyurea decreases crisis severity in patients with severe SCD.1,2
    • Proactive multispecialty care (eg, primary care physicians, hematology specialists, pharmacists, emergency departments, laboratories, and hospitals) promotes best care and patient outcomes, decreasing morbidity.2
    • Appropriate treatment of acute illness is critical because life-threatening complications can develop rapidly.2
    • Comprehensive care requires ongoing patient and family education, scheduled health maintenance visits, periodic comprehensive evaluations, psychosocial care to promote coping and self-care, and genetic counseling.2
    • Individualized pain management regimens by hematologists include home care for mild to moderate pain. Severe pain is most successfully treated by patient-controlled analgesia and may require hospitalization.3

“The right multidisciplinary team [promotes] best patient outcomes.”—Jason W. Wilson, MD, MA, FACEP, FAAEM

To access this activity, go to https://www.gotoper.com/online-cme-activities/ai/ai-scd19?#registration.

References

  1. Platt OS. Hydroxyurea for the treatment of sickle cell anemia. N Engl J Med. 2008;358(13):1362-1369. doi: 10.1056/NEJMct0708272.
  2. Yawn BP, Buchanan GR, Afenyi-Annan AN, et al. Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members. JAMA. 2014;312(10):1033-1048. doi: 10.1001/jama.2014.10517.
  3. Uwaezuoke SN, Ayuk AC, Ndu IK, Eneh CI, Mbanefo NR, Ezenwosu OU. Vaso-occlusive crisis in sickle cell disease: current paradigm on pain management. J Pain Res. 2018;11:3141-3150. doi: 10.2147/JPR.S185582.

3 of 3

Advances in™ Sickle Cell Disease: Assessing the Impact of Emerging Agents. In this certified continuing medical education activity, Matthew M. Heeney, MD, and Jason W. Wilson, MD, MA, FACEP, FAAEM, discuss the newest evidence-based clinical strategies and emerging treatments for children and adults with sickle cell disease (SCD).

To access this activity, go to https://www.gotoper.com/online-cme-activities/ai/ai-scd19?#registration.

This third of 3 PER Pulse™ Recaps focuses on emerging treatments for SCD currently in preclinical or clinical trials. Some key points are below.

As new therapies become available, the most effective care for SCD may combine current treatments (eg, hydroxyurea and comprehensive pain management) with 1 or more new therapeutic agents. New treatment approaches include:

  • Disrupting cell aggregation. Several new agents interfere with proteins promoting intracellular cell adhesion during an SCD crisis. One such agent, the monoclonal antibody crizanlizumab, slowed pain onset and decreased pain intensity in phase II trials and may enter phase III.1
  • Inflammation inhibitors. Canakinumab, a monoclonal antibody to inflammation mediator interleukin-1, is in clinical trials exploring whether it can reduce pain and other symptoms by inhibiting inflammation in SCD.2
  • Influencing action of cyclic guanosine monophosphate (GMP). Phosphodiesterase 9A inhibitors and other agents reduce inflammation and intracellular adhesion by acting on cyclic GMP. These agents may lower the risk of vaso-occlusive crisis.3
  • Disrupting activation of platelets and other blood components. P2Y12 pathway inhibitors decrease function of platelets that have become hyperactive when they are damaged during SCD crisis, contributing to vaso-occlusion.
    • P2Y12 pathway inhibitors prasugrel and ticagrelor are currently in clinical trials investigating whether they can reduce vaso-occlusion and pain in children.4-5
    • Hematopoietic stem cell transplants can cure SCD but are often unfeasible for patients without human leukocyte antigen–matched donors. Gene addition and gene editing therapies in clinical trials increase production of fetal hemoglobin (Hgb) and other Hgb less likely to sickle, potentially creating curative stem cell transplant options for patients with SCD who do not have matched donors.

“Because pathophysiology of SCD is complex, patients may benefit most from combinations of treatments, including new agents, with different mechanisms of action.”—Matthew M. Heeney, MD

To access this activity, go to https://www.gotoper.com/online-cme-activities/ai/ai-scd19?#registration.

References

  1. Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients. clinicaltrials.gov/ct2/show/NCT03474965?term=crizanlizumab&cond=Sickle+Cell+Disease&draw=2&rank=1. Updated August 26, 2019. Accessed October 25, 2109.
  2. Study of Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia. clinicaltrials.gov/ct2/show/NCT02961218?term=Canakinumab&cond=Sickle+Cell+Disease&draw=2&rank=1. Updated October 24, 2019. Accessed October 25, 2019.
  3. Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Study of PF-04447943, Co-Administered With and Without Hydroxyurea, in Subjects With Stable Sickle Cell Disease. clinicaltrials.gov/ct2/show/NCT02114203?term=PF-04447943&draw=2&rank=8. Updated December 14, 2017. Accessed October 25, 2019.
  4. A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease. clinicaltrials.gov/ct2/show/NCT01476696?term=prasugrel&cond=Sickle+Cell+Disease&draw=2&rank=1. Updated February 13, 2014. Accessed October 25, 2019.
  5. A Study to Assess the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell Disease (Hestia2). clinicaltrials.gov/ct2/show/NCT02482298?term=ticagrelor&cond=Sickle+Cell+Disease&draw=2&rank=3. Updated December 18, 2018. Accessed October 25, 2016.

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