Section 3 | National Heart, Lung, and Blood Institute Recommendations and Guidelines The NHLBI Evidence-Based Management of Sickle Cell Disease (SCD) Expert Panel Report (National Heart, Lung, and Blood Institute, 2014) is recognized as a standard, primary source of information that outlines national guidelines for improving care — both recommendations and consensus panel guidance. This NHLBI report was developed by an expert panel comprised of healthcare professionals with expertise in family medicine, general internal medicine, adult and pediatric hematology, psychiatry, transfusion medicine, obstetrics and gynecology, emergency department nursing, and evidence-based medicine. The purpose of this guide was to provide clinicians, mainly primary care providers, with a digital resource of expert, evidence-based treatment recommendations to guide improvement work on a national scale. The report provides specific recommendations for: • Comprehensive health maintenance throughout the lifespan • Effective management of pain episodes and common complications • Appropriate use of HU and blood transfusions
Hydroxyurea As part of their review process, the NHLBI expert panel examined more than 400 studies conducted between 200710 and a prior published NIH Consensus Conference on Hydroxyurea document for studies conducted before that time. This includes the Multicenter Study of Hydroxyurea (MSH) in patients (1992-2008), which was a randomized, double blind, placebo-controlled trial involving 299 adults living with SCD who had experienced three or more vaso-occlusive crises (VOCs) in the previous year. This trial demonstrated that those on HU treatment had reduced frequency of painful episodes, ACS events, and the need for red blood cell transfusions and hospitalizations. The FDA used this study as the evidence to approve HU for treatment in patients with SCD unless otherwise advised by their doctors not to take it. HU NHLBI Recommendations: • Educate all patients with SCA and their family members about hydroxyurea therapy. (See consensus treatment protocol on page 145). (Consensus–Panel Expertise) • In adults with SCA who have three or more sickle cell-associated moderate to severe pain crises in a 12-month period, treat with hydroxyurea. (Strong Recommendation, High-Quality Evidence)
In the long-term, the use of these guidelines will assist in achieving the collective goal of improving health outcomes and quality of life for people living with SCD. However, according to the Office of Minority Health, only about 1 in 4 people living with SCD receive the standard of care outlined in current guidelines (Office of Minority Health, 2020).
•
This NHLBI report formed the foundation for many of the targeted quality measures for the Program and continues to be a vital reference for clinicians and sites that take care of people living with SCD. Below are select NHLBI guidelines and/or consensus panel guidance for areas of RCC work completed during this Program: • HU • Immunizations • TCD • Pain management
• In adults with SCA who have severe symptomatic chronic anemia that interferes with daily activities or quality of life, treat with hydroxyurea. (Strong Recommendation, Moderate-Quality Evidence)
The full report can be found at here. Sickle Cell Disease Treatment Demonstration Regional Collaboratives Program
In adults with SCA who have sickle cell-associated pain that interferes with daily activities and quality of life, treat with hydroxyurea. (Strong Recommendation, Moderate-Quality Evidence)
• In adults with SCA who have a history of severe/recurrent ACS, treat with hydroxyurea.* (Strong Recommendation, Moderate-Quality Evidence)
• In infants 9 months of age and older, children, and adolescents with SCA, offer treatment with hydroxyurea regardless of clinical severity to reduce SCD-related complications (e.g., pain, dactylitis, ACS, anemia). (Strong Recommendation, High-Quality Evidence for ages 9–42 months; Moderate Recommendation, Moderate-Quality Evidence for children >42 months and adolescents). Note: The panel intentionally used the term “offer” realizing that patients’ values and preferences may differ particularly considering treatment burden (e.g., laboratory monitoring,
64
