United to Conquer Sickle Cell Disease

SCDC Update

FDA Approves Hydroxyurea for Treatment of Pediatric Patients With Sickle Cell  

Last month, the U.S. Food and Drug Administration (FDA) granted approval to hydroxyurea (Siklos®, Addmedica) to reduce the frequency of painful crises and the need for blood transfusions in pediatric patients from 2 years of age and older with sickle cell anemia with recurrent moderate to severe painful crises. This is the first FDA approval of hydroxyurea for use in pediatric patients with sickle cell disease. Congratulations to Sickle Cell Disease Coalition member Addmedica for getting this drug approved for pediatric patients. Read the exciting announcement in the American Society of Hematology/FDA Drug Update

Sickle Cell Patients, Families and Doctors Face A ‘Fight for Everything’ 

On December 27, The Washington Post featured an article about sickle cell disease and the uphill battle for better treatment, care, and research. Several representatives from Sickle Cell Disease Coalition member organizations, including Dr. Mary Hulihan, from the Centers for Disease Control and Prevention, and Dr. Patricia Kavanaugh, from the Emergency Department Sickle Cell Care Collaborative, were both interviewed for the story. While the article discusses the challenges faced as a sickle cell patient and the struggles those living with this devastating disease endure, it ends on a positive note. “Things are happening that have never happened before. I’ve been around sickle cell for a while, and never before have you seen this kind of buzz,” said Derek Robertson, president of the Maryland Sickle Cell Disease Association. Read the full story here

June 19th, National Sickle Cell Awareness Day in Canada  

The Sickle Cell Disease Association of Canada/Association d’Anémie Falciforme du Canada (SCDAC/AAFC) is proud to announce that June 19th is officially recognized as the National Awareness Day for Sickle Cell Disease in Canada. On December 8th, 2015, due to the advocacy efforts of the SCDAC/AAFC, Senator Jane Cordy introduced in the Senate Bill S-211, an Act recognizing June 19th as National Awareness Day for Sickle Cell Disease in Canada. Between 2015 and 2017, SCDAC/AAFC held two education and advocacy events on Parliament Hill to ensure that every Senator and Member of Parliament (MP) is exposed to and educated on Sickle Cell Disease (SCD). The passing of bill S-211 has put Canada on the world map as the first country to pass this legislation in line with the recommendations from the World Health Organization and the United Nations who designated the 19th day of June as World Sickle Cell Day. In Canada, bill S-211 will increase SCD awareness and education which will hopefully translate to more knowledgeable care providers and increased access to standard care. For more information about SCDAC/AAFC, visit www.sicklecelldisease.ca.

GBT Receives FDA Breakthrough Therapy Designation for Voxelotor for Treatment of SCD  

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation (BTD) to voxelotor, a disease modifying oral therapy from Global Blood Therapeutics (GBT). “The FDA’s decision to grant voxelotor the first Breakthrough Therapy designation for the treatment of sickle cell disease(SCD) reflects a recognition of the promising efficacy and safety data we have collected to date for this investigational drug, as well as an acknowledgement of the overwhelming need for major advances over available therapies in the treatment of SCD patients,” said Ted W. Love, president and chief executive officer of GBT. The FDA selectively grants BTD to expedite the development and review of drugs that have demonstrated preliminary clinical evidence indicating the potential for substantial improvement over available therapy. For more information, read the press release

Sickle Cell Disease Community Health Worker Certification Training

This year, the Sickle Cell Disease Association of America will be hosting a Community Health Worker Training Certificate for sickle cell disease (SCD). Anyone with a passion for community health and those who are personally affected by SCD are encouraged to apply. Coursework is done online except for 80 hours of fieldwork (40 hours in a community organization and 40 hours in a medical institution). At the conclusion of the course, students must pass a final exam. The cost of this course is approximately $1,200, including the exam, shipping, printing, plus the cost of the textbook. The course begins February 21st. To apply visit www.sicklecelldisease.org

Date Course Title
February 21st Orientation
February 26th Primary Health Care and Human Services 1.1
February 28th Primary Health Care and Human Services 1.2
March 5th  Communication
March 7th Patient Advocacy
March 12th Community Health Education
March 14th Capacity Building
March 19th Services Skills and Responsibilities 1.1
March 21st Services Skills and Responsibilities 1.2
March 26th SCD 101
March 28th SCD 102
April 2nd Study Session
April 9th Final Exam 

Join the New SCD Digital Community: oneSCDVoice 

oneSCDvoice, an online digital platform for the sickle cell disease community, was created with the vision of empowering those impacted by SCD through facilitating access to information and support from the SCD community. This innovative digital education platform was developed in collaboration with the SCD community, including more than 25 sickle cell disease patients, advocates, and medical professionals, as well as members from the Sickle Cell Disease Association of America, Pfizer Rare Disease and rareLife solutions. For more information visit oneSCDvoice.

Learn About the Sickle Cell Outcome REsearch (SCORE) Consortium

SCDC member, the Sickle Cell Outcome REsearch (SCORE) consortium of the Netherlands, was founded in 2016 and is composed of pediatric and adult hematologists from seven comprehensive sickle-cell centers (Amsterdam, Rotterdam, Leiden, The Hague, Utrecht, Nijmegen and Groningen). SCORE is designed as a multicenter, prospective cohort study which aims to include the majority of SCD patients in the Netherlands. The goal of the collaboration is to identify factors and biomarkers contributing to the morbidity and mortality of SCD by uniformly collecting data. The research focuses on combining doctor-reported outcomes measures (measures assessed by physicians, also called clinical data) with outcome measures from the patients’ perspective, also called PROMs (patient reported outcome measures) and PREMs (patient reported experience measures). By incorporating the patients and families’ values, beliefs and cultural norms, the impact of treatment and care can be fully evaluated.


Upcoming SCD Events 

NICHQ SCD Webinar - January 24th 

Join the National Institute for Children’s Health Quality (NICHQ) for a webinar: Strategies and Next Steps for Improving Sickle Cell Disease. Come hear lessons learned and recommendations stemming from the Sickle Cell Disease Treatment Demonstration Program, funded by the Health Resources and Services Administration Maternal and Child Health Bureau (HRSA MCHB). As the National Coordinating Center, NICHQ provided backbone and technical support for this national program focused on improving the quality and access to care for patients with sickle cell disease. Hear grantees share their on-the-ground efforts to create systems change and make a difference in the lives of children and adults with sickle cell disease. Register to save your virtual seat.

2018 Foundation for Sickle Cell Disease Research and Educational Symposium 

The Foundation for Sickle Cell Disease Research (FSCDR) is committed to supporting innovative research in sickle cell disease (SCD) to help maximize quality of life and improve survival for the generations of people affected by SCD. The next annual FSCDR symposium will take place June 15-June17, 2018 in Washington, DC. Registration is now open

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The status quo is unacceptable & we are setting out to change it. Join us to #ConquerSCD! www.scdcoalition.org

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