SCDC Update, September 2018
September is Sickle Cell Awareness Month!
SCD Partner Groups Release State of Sickle Cell Disease 2018 Report Card
On September 20, over 20 Coalition member groups released the State of Sickle Cell Disease 2018 Report Card. In 2016, the American Society of Hematology released a report card examining the state of sickle cell disease (SCD) and goals for the future in each of the following four areas: access to care in the U.S., provider training and education, research and clinical trials, and global issues. The new report card was developed to assess how these areas have improved and how much work still needs to be done. To develop the 2018 report card, more than 100 stakeholders in the SCD community were asked to rate the state of SCD in each of the four established priority areas. The results of the poll indicate that initiatives like nationwide training programs for providers and recent legislation to enhance access to quality care have moved the needle in the right direction. While progress is being made, the report card also serves as a reminder that more can be done across many priority areas for individuals with SCD. To view the 2018 report card, click here.
Sickle Cell Disease Coalition Meets In Person for the First Time
On Thursday, September 6, the Sickle Cell Disease Coalition (SCDC) met for the first time in Washington, DC at the American Society of Hematology (ASH) headquarters. Sixty representatives from the Coalition’s member organizations including public health, research and provider organizations, patient groups, federal agencies, industry, and private sector groups were in attendance. The meeting covered the current state of sickle cell disease (SCD) since the Coalition was founded two years ago, big picture sickle cell initiatives planned for the next few years, and strategies about how the Coalition and its member organizations can work together to advance these exciting efforts. U.S. Department of Health and Human Services (HHS) Assistant Secretary for Health Admiral Brett Giroir, MD, presented the keynote address, which focused on his vision for conquering the burden of SCD and concluded with a discussion about how the Coalition could help advance this priority. The meeting also featured a patient panel with representatives from the Sickle Cell Disease Association of America and Sickle Cell Community Consortium. Another session on “Looking Forward in SCD,” included representatives from ASH, the Emergency Department Sickle Cell Care Collaborative, Hematology and Oncology Society of Africa, and the National Heart, Lung, and Blood Institute. Make sure to follow the Coalition on Twitter to stay up to date on SCDC activities.
ASH Announces Plan to Build SCD Clinical Trials Network
As part of its broader effort to identify new treatment strategies and secure a pathway to curing sickle cell disease (SCD) globally, the American Society of Hematology (ASH) is in the process of establishing a clinical trials network (CTN) to identify high-priority research questions and support efforts to address these questions. In June 2018, ASH brought on Dr. Charles Chesson to serve on the ASH staff as Director of the Sickle Cell Disease Clinical Trials Network, where he will be responsible for the network’s creation, implementation, and effective operation. Under his purview, the network will focus on infrastructure, programs, and services to help overcome the challenges of conducting clinical trials in SCD, including a comprehensive patient engagement strategy to increase understanding of both the disease and clinical trials overall. Find out more about Dr. Chesson’s role and direction of the network in this Q&A.
NHLBI Introduces the Cure Sickle Cell Initiative
On September 13 the National Heart, Lung, and Blood Institute (NHLBI), announced the new Cure Sickle Cell Initiative, a collaborative research effort to accelerate the development of promising genetic therapies to cure sickle cell disease. The initiative fosters a patient-focused research environment that will identify and support the most promising and innovative genetic therapies. This effort builds on the NHLBI’s legacy of sickle cell research, which is helping many patients live longer, fuller lives. To help promote this exciting new initiative, please share this message and the images below with your social networks.
For additional resources and information, please visit https://sicklecell.nhlbi.nih.gov.
U.S. Department of HHS and Office of Minority Health Introduce #SickleCellStories
To celebrate Sickle Cell Awareness Month, the U.S. Department of Health and Human Services’ Office of Minority Health will be releasing, #SickleCellStories on social media. This video series includes personal stories from patients, advocates, and family members, including a conversation between Assistant Secretary for Health ADM Brett Giroir, MD, and Surgeon General VADM Jerome Adams, MD, MPH. Anne Alfa, an individual living with sickle cell disease, is the first story featured. Anne says, “I want to be a hematologist, and it is because I’ve been dealing with sickle cell disease my whole life. Being a hematologist is just my purpose now.” Find out more about Anne by watching her #sicklecellstory.
Two New ASH Guidelines on SCD Available for Public Comment
The American Society of Hematology will be seeking comments on two draft clinical practice guidelines: sickle cell disease-related cardiopulmonary and kidney disease, and sickle cell disease-related cerebrovascular disease. Materials will be made available from September 24 - November 5 and can be found at www.hematology.org/Guidelines-Public-Comment. These guidelines are the second and third of five forthcoming guidelines on SCD. The first guideline on SCD-related transfusion support is still open for public comment and will close on October 1. Anyone is welcome to review the draft recommendations and submit comments, including ASH members, non-member physicians and researchers, allied professionals, representatives of medical societies, industry and insurance companies, patients, caregivers, and members of the public. Feedback received will be provided to the guideline panels for review. Comments will also be considered for implementation and dissemination efforts following publication, and future revision efforts. If you use social media, you may create your own tweet about the guidelines using #ASHGuidelines.
CDC’s Sickle Cell Awareness Month Activities
To celebrate Sickle Cell Awareness month, the Centers for Disease Control and Prevention (CDC) will be sending out a digital newsletter each week highlighting information, helpful tips, and various resources to help raise awareness about SCD:
- Week 1: CDC is sharing 5 important facts about SCD, along with other resources, to help you learn more about how CDC is working to improve the lives of people with SCD.
- Week 2: CDC will focus on resources for a healthy transition from pediatric to adult care for people with SCD, including “Stepping Up: A 2-Part Video Series for Teenagers & Young Adults with SCD.”
- Week 3: CDC will be sharing SCD resources for the Spanish-speaking community, including a sickle cell trait (SCT) toolkit that has factsheets and infographics on various SCD-related health topics so people with SCT can learn more about how to stay healthy.
- Week 4: CDC will share resources for healthcare providers about reducing SCD-related transfusion complications, including a provider training video series. Educational resources on minority blood donation will also be shared.
ASH and SCDAA Host Sickle Cell Trait Briefing on Capitol Hill
On Wednesday, September 5, the American Society of Hematology (ASH) and the Sickle Cell Disease Association of America (SCDAA) hosted a briefing on Capitol Hill to educate Members of Congress and their staff on sickle cell trait (SCT). The briefing presented a general overview about SCT and highlighted the importance of knowing what it means to have trait, the current state of SCT research, and a personal story of one family’s journey. The briefing featured presentations by ASH President Dr. Alexis Thompson, Dr. Rakhi Prakash Naik, and Mr. and Mrs. Derek Robertson. A recording of the briefing can be viewed online. Dr. Thompson also highlighted the CDC, ASH, and SCDAA developed Sickle Cell Trait Toolkit which includes a colllection of resources to increase understanding of sickle cell trait.
Join SCDAA for a Social Media Party
This week, the Sickle Cell Disease Association of America, Inc. (SCDAA) has been hosting a Social Media Party in honor of National Sickle Cell Awareness Month. Join SCDAA and other advocacy organizations, corporate and federal partners, as they share victories and other exciting information about all of the latest happenings in the sickle cell community. Each day has been centered around a particular theme. There is still time to participate- join the party this Friday on Instagram as groups share Pharma Initiatives & Advances in Finding a Universal Cure. For more information, click here.
Ironwood Pharmaceuticals #FightSickleCell Video Series
Ironwood Pharmaceuticals is proud to recognize Sickle Cell Awareness Month. Ironwood has joined forces with Sickle Cell 101 and A Year of Septembers Productions by sponsoring a series of videos highlighting the responses of patients and caregivers to the prompt, “You know you have sickle cell when…” The goal of the videos is to demonstrate the daily impact of living with sickle cell disease and help increase awareness and empathy among the public who may not understand what it is like to live with this condition. Follow #FightSickleCell to view the videos as they are released throughout the month. The trailer for the first video can be found here.
New Video Addresses the Importance of Managing Iron Toxicity from Transfusions
Increasing awareness of iron toxicity and the importance of chelation therapy specifically for younger patients is an ongoing and important goal in caring for transfusion dependent patients. Overall, medication compliance rates for children and teens can vary widely as they deal with moving toward independence, acceptance of their peers, and other unique challenges. With this in mind, ApoPharma Medical affairs has created a non-branded video using age-appropriate language and graphics. The video addresses some of the challenges around educating this age group about iron toxicity and how adhering to their medication is critical. This video is part of a larger awareness campaign initiated by ApoPharma for people living with thalassemia, in particular children and teens, so that they can feel connected and realize they are not alone. To view the video, please click here: https://ironwarriors.com/resources/.
Upcoming SCD Events & Webinars
The SCDAA 46th Annual National Convention
The Sickle Cell Disease Association of America’s (SCDAA) 46th Annual National Convention will take place October 13-18, 2018 in Baltimore, MD. This year’s theme is Celebrating Diversity Within the Sickle Cell Community: Commitment, Innovation, Practice. The SCDAA Annual National Convention is a four-day conference designed to address the multi-factorial aspects of sickle cell disease (SCD). The conference fosters the exchange of the latest scientific and clinical information related to the disease which is achieved 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. The convention is aimed at individuals living with SCD and sickle cell trait, their family members, physicians, researchers, nurses, social workers, psychologists, and community health workers. Click here to find out more, and to register.
FDA-ASH Sickle Cell Disease Clinical Endpoints Workshop
The U.S. Food and Drug Administration (FDA) and American Society of Hematology (ASH) will be co-hosting the Sickle Cell Disease (SCD) Clinical Endpoints Workshop on October 17-18, 2018, a one-time event that aims to identify opportunities to bring uniformity and standards to existing SCD endpoints, identify gaps, and propose development of new endpoints as a focus for future research.
The workshop will include seven panels on the following topics:
- Patient reported outcomes (PROs)
- Pain (non-PROs)
- End organ considerations
- Endpoints related to curative therapies
- Considerations of endpoints for low-resource settings
For more information about the workshop, and to register, click here. For those unable to attend, ASH will provide a live webcast of this Workshop. Further information regarding the webcast will be made available closer to the workshop date.This workshop is made possible through generous support from the Doris Duke Charitable Foundation, as well as support from numerous donors who contributed to the ASH Foundation’s Sickle Cell Disease Initiative Fund.