SCDC Update

FDA Approves First New Drug for Sickle Cell Disease in 20 Years

On July 7, 2017 the U.S. Food and Drug Administration (FDA) approved L-glutamine powder (Endari™, Emmaus Medical, Inc.) for oral administration to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients 5 years and older. This is the first drug approved for SCD in more than 20 years. This therapy has been found to reduce both the complications associated with SCD, as well as the frequency and length of hospital visits. For the official press release from the FDA, click here.

4th Annual Sickle Cell Patient & Caregiver Educational Symposium

The 4th annual Sickle Cell Patient & Caregiver Educational Symposium, hosted by the Sickle Cell Community Consortium, Hope for SCD, Supporters of Families with Sickle Cell, and Bold Lips for Sickle Cell, was held last week in Atlanta, GA. The Symposium, which was organized entirely by individuals with sickle cell disease, addressed an array of topics including the science of SCD, mental health, women’s health, clinical trial participation, and caring for a child with SCD. These topics were covered under three different tracks; for adults; teens and young adults; and children and caregivers.

Dr. Lakiea Bailey, Executive Director of the Sickle Cell Community Consortium, set the tone for the meeting with an opening message of strength, unity, and perseverance to the sickle cell community: “We are sickle cell warriors, we are they that overcame.” The Symposium ended with a Bold Lips and Bow Ties Awards Gala and Prom as a way for those who were unable to attend their prom due to sickness, to have the chance to dress up and experience a prom for the first time. The meeting has grown significantly in the past four years and attendees included individuals with SCD, their loved ones, and representatives from a number of SCDC member organizations, including the Sickle Cell Disease Association of America, American Society of Hematology, National Marrow Donor Program/Be the Match, Pfizer, and Global Blood Therapeutics.

ASH to Develop Clinical Guidelines to Improve Care for People with Sickle Cell Disease

As part of the American Society of Hematology’s (ASH) multifaceted initiative to address the global burden of sickle cell disease (SCD), the Society is developing new clinical practice guidelines on SCD. These state-of-the-art guidelines will be produced and maintained by five panels of hematologists, other clinicians, people living with SCD, and experts in evidence-based guideline production. The panels will address the care and management of acute and chronic complications of SCD, including (1) pain caused by the disease; (2) stroke prevention and treatment; (3) prevention and treatment of cardiopulmonary and kidney diseases; (4) blood transfusions to improve red blood cell count and mitigate complications of SCD; and (5) improve the understanding of stem cell transplantation. Publication of the guidelines is expected in 2019. Learn more.

CDC Releases Tools to Promote Better Emergency Care for Individuals

People with sickle cell disease (SCD) often require care in the emergency department (ED) of hospitals and clinics for health issues, such as severe SCD-related pain. The Centers for Disease Control and Prevention released new materials about barriers to care and ways to improve care in the ED for patients with SCD:

• Sickle Cell Disease in the Emergency Department: Confronting Barriers to Care – A blog article that highlights the story of Constance Benson, an individual with Sickle Cell Disease, who experienced firsthand the barriers to care in the Emergency Department.
• Do You Use the Emergency Department for Care of Sickle Cell Disease? What to Know Before You Go – A fact sheet that provides tips for patients with Sickle Cell Disease about receiving better care in the Emergency Department, such as creating a pain management plan.
• 3 Tips About Sickle Cell Disease Every Emergency Provider Needs to Know – A fact sheet that gives tips to Emergency Department health providers about care for patients with Sickle Cell Department such as promptly treating pain experienced by patients with SCD by giving appropriate fluids and pain medicine.

September is Sickle Cell Awareness Month

It’s not too early to start thinking about Sickle Cell Awareness Month. Help us #ConquerSCD this September by sharing the Sickle Cell Disease Coalition’s sample social media posts to spread the word about SCD! These sample posts convey clear information on a variety of SCD topics, including:

• Access to care
• Training and educations
• Research and clinical trials
• How SCD affects people globally

These messages can be shared with images and videos, which can be accessed here. Don’t forget to add the hashtag #ConquerSCD throughout the month of September.

Upcoming SCD Events

NHLBI’s Annual Sickle Cell Disease Clinical Research Meetings
Register for the National Heart, Lung, and Blood Institute’s 2017 Annual Sickle Cell Disease (SCD) Clinical Research Meetings, scheduled for August 14-16, 2017, at the Natcher Conference Center on the main campus of the National Institutes of Health in Bethesda, MD. The meeting serves as a yearly forum for investigators and health care providers to discuss new developments in scientific and clinical aspects of SCD in an informal setting. For more information and to register, please click here.

Registration closes Wednesday, July 26.

6th Annual Sickle Cell Disease Therapeutics Conference
The 6th Annual Sickle Cell Disease Conference, a forum to discover the latest advancements and future trends for sickle cell disease and drug development is scheduled for September 14, 2017, in New York, NY. Conference attendees will hear from innovative industry leaders, patients, physicians, and clinical-stage companies. To register for the conference, please visit www.SCDconference.com.

11th Annual Sickle Cell Disease and Thalassemia Conference
The 2017 Annual Scientific Conference on Sickle Cell and Thalassemia is a three-day conference aimed at all those with a common interest in sickle cell disease and thalassemia. It will be held in London on October 11-13, 2017. There will also be sessions on genetics and genomic progress, curative therapies and emerging services, as well as abstract and poster presentations. For more information and to register click here.

SCDAA’s 45th Annual National Convention
The Sickle Cell Disease Association of America’s conference is scheduled for October 25-28, 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.