expert roundtables

Modernizing the Approach to Vaso-Occlusive Crisis in Sickle Cell Disease

by Stella T. Chou, MD; Michael Rutledge DeBaun, MD, MPH; and Julie Kanter, MD

Overview

Hydroxyurea reduces the frequency of vaso-occlusive crisis (VOC), but some patients with sickle cell disease (SCD) still experience recurrent crises. Innovative approaches are thus sought, which might draw on new and emerging therapeutic advancements in conjunction with improving access to high-quality collaborative care.

Q:

What are the unmet needs in the current approach to VOC in the United States? 

Julie Kanter, MD

Associate Professor, Division of Hematology and Oncology
Director, Adult Sickle Cell Program
Codirector, Lifespan Comprehensive Sickle Cell Center
University of Alabama at Birmingham
Birmingham, AL

“I take more of an adult-focused perspective on SCD management, and from that viewpoint, I think that we must optimize outpatient therapies for VOC. We need more preventive medications. Hydroxyurea is effective but insufficient, so we need more options.”

Julie Kanter, MD

Patient access to care is among the unmet needs, and we need to improve access to high-quality SCD care. Many providers in the United States are not familiar with the guidelines for managing SCD, while others are affiliated with hospitals that have SCD centers, so there is a wide range of practice settings to consider. I support implementation of a hub and spokes model of care (ie, where more experienced SCD practices and centers can offer an avenue for partnership with more remote hospitals, ensuring continuity of services in those regions).

One of our biggest challenges involves pediatric patients with SCD who present to an adult-focused Emergency Department. Pediatric patients with SCD may not receive the care they need in this setting, owing to a variety of barriers. For instance, the provider may be unclear or unfamiliar with the recommendations, or they may be reluctant to administer a given dose of opiates to a child. So, limited knowledge of SCD treatment guidelines among health care professionals continues to pose a barrier to effective care and to strong patient-provider relationships.

I take more of an adult-focused perspective on SCD management, and from that viewpoint, I also think that we must optimize outpatient therapies for VOC. We need more preventive medications. Hydroxyurea is effective but insufficient, so we need more options. There are newer treatments that may help in this regard. For instance, crizanlizumab is a humanized anti–P-selectin monoclonal antibody that decreases VOC up to 45% in recent studies. There are also other medications in the pipeline that have shown promise, such as modifiers of cGMP. However, along with any treatment advancements, we also really need to optimize access to care to improve care and to ensure that all individuals with SCD have access to an SCD specialist.

Stella T. Chou, MD

Associate Professor of Pediatrics
Chief, Division of Transfusion Medicine
Children’s Hospital of Philadelphia
Perelman School of Medicine at the University of Pennsylvania
Philadelphia, PA

“Improving access to outpatient care for VOC is key, and this can help avoid unnecessary admissions and lengthy hospital stays. Behavioral and psychosocial support systems are important as well, especially for patients with SCD who have recurrent VOC episodes.”

Stella T. Chou, MD

Acute painful crisis is a leading reason for people with SCD to go to the hospital or the Emergency Department. Improving access to outpatient care for VOC is key, and this can help avoid unnecessary admissions and lengthy hospital stays. Outpatient access can be improved by having dedicated pain medicine units or short-stay units. At our institution, we have a 24-hour unit that is overseen by nurses and physicians who are solely dedicated to taking care of individuals with SCD and managing pain episodes. Thus, dedicated staff can be very responsive to a patient’s VOC from the earliest point in its development, and our experience has been that this may reduce hospitalizations.

Behavioral and psychosocial support systems are important as well, especially for those with SCD who have recurrent VOC episodes. When compared with patients with other chronic diseases, those with SCD may lack access to adequate psychosocial support resources, and I think that if we could improve access to psychosocial support, we might be able to improve long-term outcomes as well.

Michael Rutledge DeBaun, MD, MPH

Professor of Pediatrics and Medicine
Vice Chair for Clinical and Translational Research
J.C. Peterson Chair in Pediatrics
Director, Vanderbilt-Meharry Center for Excellence in Sickle Cell Disease
Vanderbilt University Medical Center
Nashville, TN

In my experience, children and adults with SCD have benefitted from a collaborative approach that mirrors the Chronic Care Model developed by Edward Wagner, MD, MPH, and colleagues.”

Michael Rutledge DeBaun, MD, MPH

Part of the solution to some of these challenges in the transition from pediatric to adult SCD and primary care providers draws on innovative collaborations aimed at improving care, communication, and expectations. There are established models for the clinical management of children and adults with chronic diseases. Edward Wagner, MD, MPH, and his team developed the Chronic Care Model, an evidence-based framework for health care that delivers safe, effective, and collaborative care to patients. If you go to a pediatric clinic for hemophilia, cystic fibrosis, or diabetes, for instance, you will find that practitioners have adopted some form of this model. In our Center for Excellence, over the last decade, children and adults with SCD have benefitted from this type of approach to their medical care. We have been able to embrace and implement a Chronic Care Model for SCD in our pediatric and adult SCD centers. In this approach, the nurse case manager is the cornerstone of the team, working with the family to educate them about the disease with written and online materials and to develop a plan to manage acute pain episodes at home and preventive strategies to encourage good health. Our SCD team has embraced a comprehensive approach centered around empowering the family to understand SCD and the optimal treatment for management. A large part of the success of the Chronic Care Model relates to the development of a partnership between the providers and the family, which allows for shared decision making and fosters evidence-based care.  

References

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

Bodenheimer T, Wagner EH, Grumbach K. Improving primary care for patients with chronic illness: the chronic care model, part 2. JAMA. 2002;288(15):1909-1914. doi:10.1001/jama.288.15.1909

Brandow AM, DeBaun MR. Key components of pain management for children and adults with sickle cell disease. Hematol Oncol Clin North Am. 2018;32(3):535-550. doi:10.1016/j.hoc.2018.01.014

Hoppe CC, Styles L, Heath LE, et al. Design of the DOVE (Determining Effects of Platelet Inhibition on Vaso-Occlusive Events) trial: a global phase 3 double-blind, randomized, placebo-controlled, multicenter study of the efficacy and safety of prasugrel in pediatric patients with sickle cell anemia utilizing a dose titration strategy. Pediatr Blood Cancer. 2016;63(2):299-305. doi:10.1002/pbc.25771

Improving Chronic Illness Care. The Chronic Care Model. Accessed July 21, 2020. http://www.improvingchroniccare.org/index.php?p=The_Chronic_CareModel&s=2

Kutlar A, Kanter J, Liles DK, et al. Effect of crizanlizumab on pain crises in subgroups of patients with sickle cell disease: a SUSTAIN study analysis. Am J Hematol. 2019;94(1):55-61. doi:10.1002/ajh.25308

Mayo-Gamble TL, Murry VM, Cunningham-Evres J, et al. Engaging individuals with sickle cell disease in patient-centered outcomes research: a community health ambassador training model. J Health Care Poor Underserved. 2020;31(1):353-369. doi:10.1353/hpu.2020.0027

Sil S, Lai K, Lee JL, et al. Preliminary evaluation of the clinical implementation of cognitive-behavioral therapy for chronic pain management in pediatric sickle cell disease. Complement Ther Med. 2020;49:102348. doi:10.1016/j.ctim.2020.102348

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