PCORI Board Approves $18 Million for Research on Sickle Cell Disease
Last month, the Patient Centered Outcomes Research Institute (PCORI) approved $18 million to fund two studies comparing different methods to manage the transition of people with sickle cell disease (SCD) from pediatric to adult care. These studies are fundamental to understanding how to improve care for adults living with SCD, because many adult providers are unfamiliar with treating SCD. These two projects join five other previously awarded projects that focus on SCD. Together they are a part of PCORI’s $80 million investment in research on rare diseases.
Seven Projects Pursuing Cures for Sickle Cell Disease Win $6M in Research Support
The Doris Duke Charitable Foundation announced last month that seven projects would be receiving approximately $6 million total through the inaugural Sickle Cell Disease/Advancing Cures awards competition. The researchers won support for the potential of their projects to address the underlying cause of sickle cell disease, attacking it at its core by restoring hemoglobin function through various approaches. These projects hold great promise to develop disease-modifying approaches to sickle cell disease and translate them into clinically feasible therapies, aiming to remove patients’ need to manage the disease’s acute, devastating and lifelong symptoms, and ultimately increasing their life expectancy. For more information, click here.
NICHQ Releases Congressional Report on its SCD Treatment Demonstration Program Findings
On September 29, The National Institute for Children’s Health Quality (NICHQ) released a congressional report on the outcomes from the Sickle Cell Disease Treatment Demonstration Program (SCDTDP) 2014-2017. Serving as the National Coordinating Center for the SCDTDP, NICHQ worked with four regional coordinating centers to: improve access to care by increasing the number of: providers treating patients with sickle cell disease (SCD); providers prescribing hydroxyurea; and patients with SCD that are receiving care from providers knowledgeable about treating SCD. A full copy of the congressional report—which includes the outcomes, lessons learned and recommendations stemming from this work—is available on the NICHQ website.
National Poll Provides Insight Into African American Perceptions’ About Sickle Cell Disease
In September, Pfizer in collaboration with the National Newspaper Publishers Association (NNPA), and Howard University, released results from a new national poll designed to deepen understanding and gauge perceptions around sickle cell disease (SCD) among African Americans. The poll, which included responses from adults in the US who self-identified as African American, revealed that while the majority of respondents were familiar with SCD and understood the disease in general, only one-third (36%) were aware that it disproportionately affects people of African descent, demonstrating a critical need for education and awareness. “With this knowledge from the dedicated research team at Howard University, and through our collaboration with Pfizer, we can spur meaningful conversation and assess the best ways to improve disease education for those impacted in our communities,” said Dr. Benjamin F. Chavis, Jr., President of NNPA, a trade association of 211 African American-owned community newspapers from around the US. For more on the poll results, click here.
SCDAA-HRSA Solicits RFPs for Newborn Screening Follow-up Program
The Sickle Cell Disease Association of America, Inc. (SCDAA) released a request for proposals (RFP) for Sickle Cell Disease Community Based Organizations (CBOs) to work with state collaborators to improve the care and reduce the number of individuals with sickle cell disease (SCD) who are “lost to follow-up” by supporting efforts focused on SCD education and service coordination. This program will ensure that individuals diagnosed with SCD receive appropriate follow-up services including counseling, education, access to a medical home, and other support services. This effort will be overseen by SCDAA, serving as the National Coordinating Center. SCDAA plans to support up to 17 CBOs and proposals are due by November 13, 2017. To view and download a full copy of the RFP and instructions please click here or log on to the SCDAA website at www.sicklecelldisease.org.
Making Advances Against Sickle Cell Disease
The U.S. Food and Drug Administration (FDA)’s Drs. Patricia Oneal and Rosanna Setse addressed how much progress has been made in sickle cell disease (SCD), and how much still needs to be done in the September 26th FDA Voice blog post. From the approval of the drug Endari (L-glutamine oral powder), to the number of clinical trials for SCD, this blog post summarizes how the FDA is hoping to improve outcomes for those living with the disease. You can read it here.
Sickle Cell Disease Highlighted on Capitol Hill
The U.S. Congress focused on SCD and sickle cell trait (SCT) throughout September to celebrate National Sickle Cell Awareness Month. The U.S. Senate unanimously passed Senate Resolution 273 to designate September as Sickle Cell Awareness Month and highlight the urgency of finding a widely accessible cure, identifying early detection methods, and ensuring those living with the disease are aware of available resources.The U.S. House of Representatives introduced House Resolution 539 to encourage SCT research, surveillance, and awareness. Additionally, the Congressional Black Caucus Health Braintrust, the authority on African American and minority health on Capitol Hill, featured SCD and the American Society of Hematology’s multifaceted SCD initiative in the Fall Edition of The Broadsheet, a mini-report on minority health.
Upcoming SCD Events
Conferences & Training
Research and Clinical Trials in Sickle Cell Disease - Family Engagement
Join the Sickle Cell Disease Association of Canada in collaboration with the Sickle Cell Disease Association of Nova Scotia on Thursday, October 19, 2017, 9 a.m. – 3 p.m. to learn about research and clinical trials in sickle cell disease.
Senator Jane Cordy who advanced “An Act Respecting Sickle Cell Awareness Day” will also be present. Meet healthcare professionals, researchers, and patient families.
- Meals and accommodations will be provided for patient families outside of HRM
- Child friendly space available
- Refreshments will be provided
- Bus tickets available if desired
Project ECHO Training on Sickle Cell Disease
A Project Extension for Community Health Care Outcomes (ECHO) training will be held on Monday, October 23-Tuesday, October 24, 2017 in Atlanta, GA. The training will be led by the American Academy of Pediatrics, a Project ECHO Superhub and will be designed specifically for organizations interested in using the ECHO model in their own settings to improve the health care of individuals living with sickle cell disease (SCD). The ECHO model increases access to specialty treatment in rural and undeserved areas by building capacity amongst front-line physicians to identify, treat, and manage the care of patients with complex conditions.
The training provides the following:
- Overview of the ECHO model and the importance of following the protocol in achieving success
- Overview of key skills and requirements needed to properly use model
- Framework needed to develop a teleECHO (virtual) clinic for patients with SCD
- Information about using the ECHO model to develop a state-wide surveillance (health monitoring system)
- Presentations from groups who have already used the ECHO model to improve care of patients with SCD
- Opportunity to network with the other training session attendees
Attendance is free, but space is limited. Please RSVP to Mandip Kuar at firstname.lastname@example.org and include a brief description of your organizations’ s proposed strategy for using Project ECHO to improve care for patients with SCD.
Please not that this event will take place during the two days preceding the Sickle Cell Disease Association of America’s 45th Annual National Convention. These two events are not connected.
SCDAA’s 45th Annual National Convention
The Sickle Cell Disease Association of America’s conference is scheduled for Wednesday, October 25- Saturday, October 28, 2017, in Atlanta, Georgia. This convention fosters the exchange of the latest scientific and clinical information related to the disease. This is done through the offering of innovative symposia, training seminars and interactive panel discussions. In addition, this year’s convention offers an array of exciting activities designed to educate and motivate the entire community to get involved in the fight against SCD. To learn more about the event and to register, please click here.
Keynote Speakers & Presentations
Friday, October 27- Patrick Hines, MD, PhD – Innovations in Blood Function Diagnostics
Saturday, October 28 – Clinton Joiner, MD, PhD – The Red Blood Cell Membrane as a Target for New SCD Therapies
Saturday, October 28 – Alexis Thompson, MD, MPH – Gene Therapy as a Cure for Sickle Cell Disease
ASH Meeting to Highlight SCD
The American Society of Hematology’s (ASH) 59th Annual Meeting and Exposition will take place December 9-12, 2017 in Atlanta, Georgia. The meeting will provide an invaluable educational experience and the opportunity to review thousands of scientific abstracts highlighting updates in the hottest topics in hematology. This year’s meeting will include sickle cell disease in a number of its scientific and educational sessions. More information about the schedule and program is available here. Also, if you missed the ASH Consultative Hematology Course in September, which highlighted nonmalignant hematology, you are in luck. A second course will take place on Monday December 11th at the Annual Meeting, with one session focusing on Sickle Cell Disease in Adults: What does the consultant hematologist need to know? Registration for this meeting is now open.