February 28, 2019 is Rare Disease Day.
Rare Disease Day seeks to boost awareness about rare diseases among the general public, lawmakers, industry representatives, researchers, and health professionals. Discover how you can take part.
Innovations in Sickle Cell Disease Briefing
On Thursday, February 7, the Pediatric Hospital Sickle Cell Disease Collaborative, member of the SCD Coalition, hosted a briefing on Capitol Hill to highlight the innovations in sickle cell disease, including treatment advances and developing cures for SCD. Congresswoman Robin Kelly (D-IL) with the Congressional Black Caucus Health Braintrust, was the honorary co-host for the briefing, and she provided opening remarks. Drs. Andrew Campbell (Children’s National), Kim Smith-Whitely (Children’s Hospital of Philadelphia), and Beatrice Gee (Children’s Healthcare of Atlanta) provided attendees with information on what life is like for a patient with SCD, what treatments are available today, and what resources are needed for additional research.
Experimental Therapies Might Have Cured SCD Patients
Last month, The New York Times published an article about the success of gene therapy in sickle cell disease (SCD) patients. Success against SCD would be “the first genetic cure of a common genetic disease” and could free tens of thousands of Americans from agonizing pain. Read “These Patients Had Sickle Cell Disease. Experimental Therapies Might Have Cured Them” to learn more about the several people who have been cured of SCD by gene therapy.
ASGCT Patient Education on Gene Therapy Now Available!
The American Society of Gene and Cell Therapy launched a new patient education portal on their website in January. Resources include short animated videos, dynamic content and infographics. The section Gene Therapy 101 educates on the basics of gene therapy, the difference between gene therapy, cell therapy and gene editing, as well as the process of developing a treatment. Stay tuned throughout February as five disease specific topics will be launched, including one on blood disorders (which will include sickle cell disease). Wide distribution is encouraged to benefit the patient community, so please enjoy and share! Feel free to contact, Alison Kujawski, Program Manager, Outreach at email@example.com, with any comments or questions.
Public Feedback Needed for CMS SCD Indicator
The Centers for Medicare & Medicaid Services (CMS) is requesting public comment on a proposed algorithm for a sickle cell disease indicator in their Chronic Conditions Data Warehouse. The Chronic Condition Data Warehouse is a research database designed to: identify areas for improving the quality of care provided to chronically ill Medicare beneficiaries, identify ways to reduce program spending, and make current Medicare data more readily available to researchers studying chronic illness in the Medicare population. Additional information about the proposed SCD algorithm can be found here. Please send all comments to Jaclyn.Burton@GDIT.com by Friday, March 1, 2019.
Annual National Convention - Call for Abstracts
The Sickle Cell Disease Association of America (SCDAA) would like to highlight your work at the 47th Annual National Convention on October 9-12, 2019 in Baltimore, MD. This year’s theme is: Sickle Cell Community Embracing Change Together. Last year, the convention was attended by over 600 researchers, physicians, nurses, social workers and individuals living with sickle cell disease (SCD) and sickle cell trait. If you are a researcher, community-based member organization, physician, nurse, social worker or someone working on behalf of people with SCD and their families, submit an abstract by June 3, 2019. Click here to learn more about the Convention, abstract guidelines, and the submission process.
Sickle Cell Data Collection & New Funding Opportunity
The Centers for Disease Control and Prevention (CDC) has two announcements:
- The CDC’s Sickle Cell Data Collection program’s 2005 data reports for Georgia and California are now available online. The maps and figures on the webpages represent 2005 data on patient demographics, hospitalizations, and emergency department visits for SCD.
- New CDC funding opportunity pending: Characterizing the Complications Associated with Therapeutic Blood Transfusions for Hemoglobinopathies (DD19-1903) Activities will include education of patients, families, providers, and blood banks to decrease the complications from blood transfusions. Click “Subscribe” to receive updates about this funding opportunity. The opportunity status of this grant is currently listed as “forecasted,” which means that funds are not yet formally available and are pending budgetary and discretionary spending approvals and federal agency program decisions.
United Kingdom-based Raremark, the newest member of the Sickle Cell Disease Coalition, is here to support individuals living with sickle cell disease. They are proud to announce the launch of their patient-to-patient sickle cell community: https://raremark.com/sickle-cell-disease. If you are interested in learning more about how Raremark’s platform benefits individuals living with SCD, please email firstname.lastname@example.org.
Get Connected - Register Today
The Sickle Cell Disease Association of America’s Get Connected, is the first patient powered registry for sickle cell disease that allows for the secure storage of health information. This registry enables patients to better navigate the health care system and to be counted, showing significance of this public health issue. To register for Get Connected, visit www.GetConnectedSCD.org.
Future Africa Leaders Award
The President of Sickle Cell Youth Foundation (SCYF) in Tanzania, Yusuph Gesase has been awarded The Future Africa Leaders Award (FALA) in recognition of his work creating sickle cell disease (SCD) awareness campaigns, targeting youth in secondary schools and universities. SCYF focuses on creating awareness for SCD and provides free access to SCD screening tests to young people, to “Break the Sickle Cell Cycle,” the name given to Yusuph’s SCD secondary school awareness campaign in Tanzania. Tanzania is ranked as having the fifth highest burden of SCD in the world, with about 11,000 children born with SCD each year. Yusuph, who is also living with SCD, believes that providing education to the youth about SCD status early in their adolescence, may have an impact in reducing the prevalence of SCD in the country. For further information, please visit: sickleyouthfoundation.org.
Upcoming SCD Events
National Academies SCD Event
The National Academies of Sciences, Engineering, and Medicine is convening an ad hoc committee to develop a strategic plan and blueprint for addressing sickle cell disease in the United States. The first meeting of the committee is scheduled for Thursday, February 21, 2019. The public portion of the meeting is scheduled from 1:15 p.m. to 4:50 p.m. at the National Academy of Science Building in Washington, DC. If you would like to RSVP to attend in person or via webinar, you can register here. This project was commissioned by the Department of Health and Human Services’ Office of Minority Health and the group’s charge and roster are available here.
Warrior University Online Series
Sickle Cell Data Collection Program Webinar: Stepping Up Into Adult Care
The Centers for Disease Control and Prevention’s Sickle Cell Data Collection (SCDC) program will be hosting the webinar Stepping Up into Adult Care: The Sickle Cell Trevor Thompson Transition (ST3P-UP) Project, Thursday, February 28, 2019, 3:30- 5 PM ET. This project will compare the effectiveness of adding virtual peer mentoring to a structured education-based transition program for emerging adults with sickle cell disease. The outcomes studied will be decreasing the number of acute care visits per year, improving patient-reported outcomes, and reducing healthcare utilization among emerging adults with sickle cell disease. Principal Investigators Drs. Ifeyinwa (Ify) Osunkwo, from Carolinas HealthCare System, and Raymona Lawrence, from Georgia Southern University, will present. Click here to register.
Holistic Health and Sickle Cell Disease:A Focus on Mental and Behavioral Health Webinar
In recognition of Rare Disease Day, on Thursday, February 28, 2019, the Office of Minority Health (OMH) is creating awareness about SCD by continuing its webinar series focusing on the effects of the disease on patients. The Holistic Health and Sickle Cell Disease: A Focus on Mental and Behavioral Health webinar will take place February 28, 1-2:15PM ET and will discuss various aspects of mental and behavioral health in SCD patients, as well as a holistic approach to treatment. Presenters will share information on the mental health disparities that exist among SCD patients and ways healthcare providers can work to address them. Dr. Charles Jonassiant, University of Pittsburgh, Dr. Kairav Shah, University of Texas Health Science Center at Houston, and Dr. Carolyn Rowley, Cayenne Wellness Center, will present. Dr. Alexis Bakos, Senior Advisor to the Deputy Assistant Secretary for Minority Health will moderate the discussion. Click here to register.
Pathophysiology of Actue Vaso-occlusive Crisis in SCD Webinar
The Foundation for Women & Girls with Blood Disorders is holding a free educational webinar on Thursday, March 7, 2019, 12-1PM ET on the Role of Selectins and Adhesion in the Pathophysiology of Acute Vaso-occlusive Crisis in Sickle Cell Disease. Drs. Marilyn Telen and Andra James from Duke University will present. Click here to register.
SCDAA Advocacy Day - Save the Date
The Sickle Cell Disease Association of America (SCDAA) will host its 2019 Annual Advocacy Day event on April 8-9, 2019 on Capitol Hill in Washington, DC. SCDAA’s Advocacy Day is an opportunity for patients and caregivers affected by SCD to advocate for issues that are important to them. Individuals will meet with their members of Congress and share stories and key legislative issues impacting the SCD community. SCDAA’s 46 member organizations and affiliates from 35 states are invited to meet with legislators and staff to discuss key issues of interest to SCD patients and their loved ones. Information about registration will be made available soon on the SCDAA website.