Sickle cell disease (SCD) is one of the most common genetic disorders in the US. Pain is the most common cause of acute morbidity in children and adults with SCD. Pain episodes are periodic, self-limited but often excruciating events that can last hours to days, sometimes weeks if inadequately treated. Studies on SCD-related pain have primarily focused on adults, therefore, the National Heart, Lung and Blood Institute identified acute pain management and prevention as a high-priority area for research in children. Most pain episodes are managed at home for children with SCD, yet there are no controlled studies examining the management of pain in this setting. In addition, there is growing evidence that pain is frequently under-treated at home. For more severe pain episodes, patients with SCD often rely on care in the emergency department (ED). Despite published protocols recommending rapid assessment and treatment, children with SCD experience significant delays and variation in the treatment of their pain. From the PI's qualitative work with parents of children with SCD, parents are often overwhelmed by the prospect of managing their child's pain at home. They also report delays in the treatment of their child's SCD- related pain in the ED. Parents believe that the use of an individualized pain plan would improve the caregiver's ability to prevent and manage their child's SCD-related pain. Written, symptom-based, management plans have been proven effective in treating chronic diseases, such as asthma and cancer-related pain.
The specific aims of this project are to: 1) Design and conduct a national survey of directors of pediatric SCD or hematology programs and the division chiefs of pediatric emergency medicine to understand if, and how, pain protocols or other methods are used in the home and ED settings for the management of SCD- related pain in children;2) Convene an expert panel to develop quality indicators for the prevention and management of acute SCD-related pain in children in the home and ED settings using the Modified Delphi method, and to refine an individualized written pain plan for use in children with SCD;and 3) Perform a pilot randomized controlled trial of children with SCD aged 5 to 12 years to evaluate the feasibility and efficacy of using an individualized written pain plan in the home and ED settings. In addition to acquiring quantitative research skills through the proposed research plan, the candidate will gain knowledge in the management of children with SCD and pain;receive instruction on the cultural aspects of health care, advanced biostatistics, and study design through additional coursework;and learn about systems approaches to caring for children with chronic illness. Information and experiences gained from this project will be used to design a multicenter randomized controlled trial to examine the effect of an individualized pain plan on the health care utilization, physical function, and quality of life of children with SCD.

Public Health Relevance

Sickle cell disease (SCD) is one of the most common genetic disorders in the US. Pain episodes are the most common cause of acute morbidity in children with SCD, and typically increase in frequency and severity as they grow older. The public health implications of this proposal are that better management of SCD-related pain will result in improved health, educational, and quality of life outcomes in these children, which will allow them to lead more productive lives as adults.

National Institute of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
Mentored Patient-Oriented Research Career Development Award (K23)
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Special Emphasis Panel (ZHL1-CSR-X (M1))
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Werner, Ellen
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Boston Medical Center
United States
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