ASH Brings Sickle Cell Disease Awareness to Capitol Hill

ASH Brings Sickle Cell Disease Awareness to Capitol Hill

The National Heart, Lung, and Blood In-stitute’s (NHLBI) recently released expert panel report on the management of sickle cell disease (SCD) is an important and timely step toward raising the standard of care for patients afflicted with this disorder. ASH has endorsed the evidence-based report based on its overall merit and its potential to improve care for the nearly 100,000 Americans with SCD.

To discuss the report’s findings and highlight the need for state-of-the-art clinical care for SCD patients, on September 16, ASH co-hosted a congressional briefing on Capitol Hill with the Sickle Cell Disease Association of America (SCDAA), featuring remarks from:

  • NHLBI Director Gary Gibbons, MD
  • Patient advocate Carlton Haywood Jr., PhD, MA, from The Johns Hopkins School of Medicine and Berman Institute of Bioethics
  • ASH Councillor Alexis Thompson, MD, from Ann & Robert H. Lurie Children’s Hospital of Chicago and Feinberg School of Medicine, Northwestern University
  • SCDAA Chief Medical Officer Kim Smith-Whitley, MD, of The Children’s Hospital of Philadelphia

Speakers were also joined by Representatives Danny Davis and Charles Rangel, who urged their congressional colleagues to join the newly formed bicameral and bipartisan Congressional Sickle Cell Disease Caucus.

Dr. Thompson, representing ASH, began the briefing with an overview of the biology of the disease and its far-reaching implications for a patient’s quality of life. “We need to understand how complex this single-gene, single-cell problem is — it affects multiple organs; it involves many different pathways,” Dr. Thompson said. “But, in our minds, it also presents multiple opportunities for research.”

Speaking on behalf of the NHLBI, Dr. Gibbons noted that the guideline development process identified clinical research gaps and will be useful for the NHLBI as a research agency. “NHLBI is committed to a robust research investment that will change the natural history of SCD and continue to develop an evidence base that can improve health outcomes and help SCD patients live fuller lives.”

“Of course,” he added, “that doesn’t stop in the laboratory — the discoveries need to be successfully implemented in the real world.”

ASH is also actively involved in defining top research and training priorities for the next five years, including identifying predictors of disease severity and developing novel therapies. “Without ongoing and greater investment we are likely to lose some of the progress we have already made,” Dr. Thompson concluded. “So, we urge Congress to recognize the value of biomedical research … and propose widespread adoption and implementation of the new NHLBI guidelines to provide a framework for optimizing care across the lifespan.”

ASH to Host a Stakeholders Meeting Early Next Year

ASH plans to convene a meeting with the key stakeholders to discuss dissemination strategies of the recently released expert panel report on the management of sickle cell disease. ASH staff plans to schedule the meeting in early 2015 once all of the stakeholders have a chance to thoroughly review the guidelines and consider potential dissemination activities. Federal representatives and representatives from various medical specialty societies (e.g., American College of Emergency Physicians, American Academy of Pediatrics) will be invited. The main objectives of the meeting will be to identify key messages stemming from the guidelines and the target audience, the best mechanisms for disseminating those messages (e.g., clinical tools, webinars, patient-focused resources, etc.), and delegating which stakeholders will be responsible for each high-priority dissemination approach and how stakeholders can collaborate on implementing them.

Pocket Guides for Easy Reference
To help busy clinicians translate the NHLBI Expert Panel’s recommendations for managing sickle cell disease into practice, ASH has condensed this comprehensive report into three free pocket guides. These guides will debut at the 2014 annual meeting and will be available for ordering in the ASH Store ( at that time.

Hydroxyurea and Transfusion Therapy for the Treatment of Sickle Cell Disease Authors: Susan E. Creary, MD; John J. Strouse, MD, PhD
This guide will summarize the evidence-based and consensus recommendations for the use of the two FDA-approved therapies for SCD: hydroxyurea (to treat sickle cell anemia in adults) and red cell transfusion (to treat acute complications and prevent chronic complications).

Management of Acute Complications of Sickle Cell Disease
Authors: Timothy McCavit, MD, MSCS; Payal Desai, MD
This guide will address the many acute complications associated with SCD, including pain from vaso-occlusive crisis, acute chest syndrome, multi-system organ failure, priapism, and stroke. Recommendations for both evaluation and management will be provided. Indications for and against transfusion will be highlighted.

Management of Chronic Complications of Sickle Cell Disease
Authors: Suzie A. Noronha, MD; Sophie Lanzkron, MD
Individuals with SCD are at high risk for developing serious multi-organ system chronic conditions. This guide will outline screening and follow-up procedures for several common SCD complications and will summarize recommendations for the long-term use of opioids to manage chronic pain.