Hematology Briefings™: The Evolving Management Landscape for Patients With Sickle Cell Disease: Opportunities, Challenges, and Emerging Agents for Therapy

Released On
December 30, 2018

Expires On
December 30, 2019

Media Type

Completion Time
60 minutes


Sickle Cell

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This activity is provided by Physicians' Education Resource®.


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

Credit Available

  • Physicians — maximum of 1.0 AMA PRA Category 1 Credit(s)
  • Nurses — 1.0 contact hours.

Activity Overview

This activity, Hematology Briefings™: The Evolving Management Landscape for Patients With Sickle Cell Disease: Opportunities, Challenges, and Emerging Agents for Therapy, developed in Physicians’ Education Resource®, LLC, established Hematology Briefings™ legacy format, is an online interactive monograph that provides treatment strategies for patients with sickle cell disease (SCD). This presentation features a national thought leader, Laura DeCastro, MD, who provides key insights.

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 audio files/content until you finish the presentation.
  • At the end of the activity, educational content/audio files 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 continuing medical education (CME) activity is intended for hematologists and physicians including pediatricians, who manage patients with sickle cell disease. Nurse practitioners, nurses, physician assistants, nurses, and other healthcare professionals involved in the treatment and management of patients with sickle cell disease will be invited to participate.

Learning Objectives

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

  • Identify patients with SCD and vaso-occlusive (VOC) symptoms earlier to initiate prompt intervention and preventive therapy
  • Recognize the acute and chronic complications associated with VOC phenomena in SCD
  • Distinguish current and emerging treatments for management of SCD and VOC crises and best practices for their use in patients’ management
  • Better utilize shared-decision making as a regular part of patients’ management of SCD, including discussion of therapy options and choices

Faculty, Staff, and Planners' Disclosures

Laura M. De Castro, MD, MHSc

Clinical Director benign Hematology
Associate Professor
Division of Hematology and Oncology
Department of Internal Medicine
Pittsburgh, Pennsylvania

Disclosures: Grant/Research Support: Bayer, Global Blood Therapeutics, Ironwood Pharmaceuticals, Pfizer; Consultant: Pfizer

The staff of PER® have no relevant financial relationships with commercial interests to disclose.

Accreditation/Credit Designation

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

Physicians' Education Resource®, LLC, is approved by the California Board of Registered Nursing, Provider #16669, for 1.0 Contact Hours.

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 relative to diagnostic, treatment, or management 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®.

PER Pulse™ Recaps

1 of 3

Join renowned medical expert Laura DeCastro, MD, in a discussion of the management of sickle cell disease in the online continuing medical education activity Hematology Briefings™: The Evolving Management Landscape for Patients With Sickle Cell Disease: Opportunities, Challenges, and Emerging Agents for Therapy.

This first of 3 PER Pulse™ Recaps will focus on the epidemiology and diagnostic criteria of sickle cell disease.

Sickle cell disease (SCD) constitutes a global health concern affecting 1 in 2500 births. SCD affects approximately 100,000 individuals in the United States and an estimated 300,000 children across sub-Saharan Africa every year.1 Sickle cell disease is very common in areas with a high prevalence of malaria and especially among individuals whose ancestors came from sub-Saharan Africa; Spanish-speaking regions in the Western hemisphere (South America, the Caribbean, and Central America); Saudi Arabia; India; and Mediterranean countries such as Turkey, Greece, and Italy.1

This inherited hemoglobinopathy is confirmed by electrophoresis, demonstrating the presence of homozygous sickle cell hemoglobin after the patient has experienced their first acute pain crisis associated with SCD. Polymerization of deoxygenated sickle hemoglobin leads to decreased deformability of red blood cells.2

The major hallmarks of SCD are episodic painful crises, which account for the majority of hospital admissions in the population. An adult patient’s pain rate correlates with early death, with episodes increasing in frequency with age.


  1. Data and statistics on sickle cell disease (SCD). Centers for Disease Control and Prevention website. cdc.gov/ncbddd/sicklecell/data.html. Updated August 9, 2017. Accessed January 4, 2018.
  2. Schneider RG. Laboratory identification of hemoglobin variants in the newborn. In: Carter TP, Wiley AM, eds. Genetic Disease - Screening and Management. New York, NY: Liss; 1986:137.

2 of 3

As a follow-up to the online continuing medical education activity Hematology Briefings™: The Evolving Management Landscape for Patients With Sickle Cell Disease: Opportunities, Challenges, and Emerging Agents for Therapy, Laura DeCastro, MD, characterized the potentially practice-changing implications for the management of patients with sickle cell disease (SCD).

This second of 3 PER Pulse™ Recaps will focus on the application of emerging clinical trial data to the treatment paradigm of SCD.

Even with best practices in medical care, quality of life (QOL) is often reduced. Clinical outcomes have improved over the years, particularly those related to hydroxyurea therapy for pain crisis prevention and improvements in supportive care.1 Hydroxyurea has been shown to significantly reduce the rate of vaso-occlusive crises as well as the incidence of acute chest syndrome. Common adverse effects of hydroxyurea include low blood counts, gastrointestinal symptoms, and loss of appetite.2 More recently, a glutamine supplement powder was approved to reduce the frequency of acute pain and resultant hospitalizations in patients ≥5 years.3 In addition, data on the new monoclonal antibody crizanlizumab, which inhibits P-selectin, has shown promising efficacy in reducing the frequency of acute pain in adults with SCD.1,3 Crizanlizumab binds to P-selectin and blocks its interaction with P-selectin glycoprotein ligand 1.3 Overall, new therapies and pathways for management of SCD and its complications must still be elucidated to reduce morbidity and mortality associated with these disorders and improve patient outcomes and QOL.4

In addition to developing drugs aimed at reducing sickle cell pain crises and vaso-occlusive pain, clinicians must remain vigilant in providing preventive care and health maintenance for patients with SCD. This includes keeping patients up-to-date with immunizations and screening for renal, cardiac, and pulmonary disease, along with hypertension, stroke, and retinopathy. Moreover, clinicians must treat any concomitant infections promptly and thoroughly. All potential acute and chronic complications noted earlier must also be monitored continuously and managed promptly and aggressively.5


  1. Piel FB, Steinberg MH, Rees DC. Sickle cell disease. N Eng J Med. 2017;376(16):1561-1573. doi: 10.1056/NEJMra1510865.
  2. Brawley OW, Cornelius LJ, Edwards LR, et al. National Institutes of Health Consensus Development Conference statement: hydroxyurea treatment for sickle cell disease. Ann Intern Med. 2008;148(12):932-938. doi: 10.7326/0003-4819-148-12-200806170-00220.
  3. Niihara Y, Miller ST, Kanter J, et al. A phase 3 trial of l-glutamine in sickle cell disease. N Engl J Med. 2018;379(3):226-235. doi: 10.1056/NEJMoa1715971.
  4. Ataga KI, Kutlar A, Kanter J, et al. Crizanlizumab for the prevention of pain crises in sickle cell disease. N Engl J Med. 2017;376(5):429-439. doi: 10.1056/NEJMoa1611770.
  5. National Heart, Lung, and Blood Institute. Evidence-based management of sickle cell disease: expert panel report, 2014. nhlbi.nih.gov/health-topics/evidence-based-management-sickle-cell-disease. Published September 2014. Accessed January 4, 2019.

3 of 3

In this third of 3 PER Pulse™ Recaps, as a follow-up to the online continuing medical education activity Hematology Briefings™: The Evolving Management Landscape for Patients With Sickle Cell Disease: Opportunities, Challenges, and Emerging Agents for Therapy, Laura DeCastro, MD, focused on best practices, including shared decision making to improve management and patient quality of life.

The decision process for therapy in sickle cell disease (SCD) can be very complex for both patients and clinicians, and shared decision making can enhance this process. A study assessed physician responses on their perspective of patient values, preferences, and decisional needs regarding disease-modifying therapies in SCD and the narratives used in clinical practice for treatment decisions. Results observed in this study highlight that although all the physicians were advocates of patient education and active involvement, more than half of the physician narratives used with patients were promoting a particular therapeutic plan favored by the physician.1 The degree of physician involvement was influenced by perception of patient compliance, socioeconomic barriers, and the patient’s current clinical condition. Those physicians who advocated their own treatment option versus a more collaborative approach were more likely to discuss noncompliance and socioeconomic barriers as influencing their approach to patient management. The results suggest a stronger need for awareness and education about the shared decision-making model for both patients and physicians and the creation of decision-support systems regarding the use of disease-modifying therapies for SCD.1

  1. Bakshi N, Sinha C, Ross D. Shared decision making or physician advocate for a particular treatment option: a spectrum of approaches to decision making about disease modifying therapies in sickle cell disease. Blood. 2016;128(22):4757.