United to Conquer Sickle Cell Disease

SCDC Update, August 2019

Trial Underway in U.S. Uses Gene-Editing Tool, CRISPR

Several dozen patients with sickle cell disease (SCD) will be among the first in the United States treated for a genetic disease with the experimental gene-editing technology CRISPR. This technology enables scientists to make very precise changes in DNA, raising hopes it will lead to new ways to prevent and treat many diseases. The study aims to recruit up to 45 adults with severe SCD. Read more about this exciting development in treating individuals living with SCD.

ASH Awarded ASAE Award for SCD Initiative 

The American Society of Hematology (ASH) has been recognized with the American Society of Association Executives (ASAE) Power of A Award in the Power to Prepare for the Future category for the Society’s sickle cell disease (SCD) initiative. These awards showcase how associations leverage their unique resources to solve problems, advance industry and professional performance, kickstart innovation, and improve world conditions. The ASH SCD initiative is a multifaceted, patient-centric effort that brings together stakeholders in the public and private sectors to improve outcomes for individuals living with SCD worldwide. This initiative includes the creation of the Sickle Cell Disease Coalition. Find out more here

The Latest in SCD Research, for the SCD Community

The Sickle Cell Disease Coalition Research & Clinical Trials Working Group is working on a project that focuses on distilling key sickle cell disease (SCD) information presented at national conferences and disseminating it to the SCD community. Each summary is written in plain language to ensure comprehension of topics covered.The first set of summaries comes from the American Society of Gene and Cell Therapy 2019 Annual Meeting: 

These summaries were developed by the National Marrow Donor Program/Be the Match

Results of 2018 GBT Acess to Care Summit

The Global Blood Therapeutics’ Sickle Cell Disease (SCD) Access-to-Care Summit, held on September 12, 2018, convened committed stakeholders from across the United States to explore ways of improving the lives of people living with SCD who face a number of challenges. The Summit’s Steering Committee has developed a report that addresses four areas that are important to broadening access to care for people living with SCD:

  • Expanding care within the institution: establishing a SCD clinic and/or day hospital for comprehensive management 
  • Utilizing existing care infrastructure: leveraging available institutional capacity by partnering with stakeholders outside the SCD arena 
  • Transitioning adolescent care: structuring transition between pediatric and adult care facilities 
  • Expanding care outside the institution: building outreach models to extend the range of care into the community and beyond

Click here to download the report. 

ASH Launches New Sickle Cell Disease Pain Management Webpage

The American Society of Hematology (ASH) recently launched a sickle cell disease (SCD) pain management webpage to help the SCD community and providers better address the ongoing challenges associated with managing acute and chronic pain. The webpage includes recent news and policy updates, as well as clinical resources to assist with pain management. One of the newest items on the webpage is an infographic on the Special Populations section of the U.S. Department of Health and Human Services (HHS) Pain Management Best Practices Inter-Agency Task Force Report. 

An Innovation in Provider Training Increases Access to Care for Those Living With SCD 

A shortage of expert providers, especially in rural and underserved areas, means that individuals with sickle cell disease (SCD) are often unable to access the comprehensive specialty care and treatments they need. Now, Project Extension for Community Healthcare Outcomes (ECHO®), an internationally recognized telementoring initiative is transforming provider training to increase access to care and improve the quality of life for SCD patients across the country. Project ECHO® uses telementoring to train and increase the number of clinicians knowledgeable about specialty disease care by developing hub-and-spoke knowledge-sharing networks, where an expert team (the hub) provides virtual learning sessions to community providers (the spokes). This continuous learning loop between specialists and community providers ultimately helps families in local communities access high-quality specialty care. In the past five years, Project ECHO telementoring hubs for SCD have spread across the United States, each led by a designated regional coordinating center (RCC), who are grantees of the Health Resources and Services Administration Sickle Cell Disease Treatment Demonstration Regional Collaborative Program. Learn more about their experiences and telemonitoring’s potential to improve access to SCD care here.

CDC’s Sickle Cell Data Collection (SCDC) Program Announcements 

In honor of September Sickle Cell Awareness Month, the Centers for Disease Control and Prevention (CDC) will share educational materials on transition from pediatric to adult care and back-to-school, diversity among the sickle cell disease (SCD) community, transfusions, and SCD surveillance. For more updates on these resources, follow @CDC_NCBDDD on Twitter. Make sure to also check out the following resources:

ASH Hydroxyurea Webinar 

The American Society of Hematology (ASH) “Hydroxyurea for Sickle Cell Disease Treatment” June webinar is now available on ASH on Demand. This webinar, moderated by Kim Smith-Whitley, MD (Children’s Hospital of Philadelphia) is geared towards those living with sickle cell disease and focuses on the use of hydroxyurea in adults and young adults. The webinar includes a story from an individual who has taken hydroxyurea, and an interactive Q&A session. Speakers include Sophie Lanzkron, MD (Johns Hopkins University) and sickle cell disease advocate Kyle Smith, MS. After watching the webinar, make sure to download ASH’s informational booklet on hydroxyurea, which is available in both English and Spanish. 

Upcoming SCD Events 


SCDAA Hosts Open House at New Office Location 

The Sickle Cell Disease Association of America is moving its national offices and will be hosting an Open House on Friday, August 23, 2019, from 2-4pm. Please make sure to update your contact information with the new address: 

Sickle Cell Disease Association of America, Inc.7240 Parkway Drive, Suite #180Hanover, MD 21076

If you are interested in attending, please RSVP by emailing lbrown@sicklecelldisease.org.

NHLBI Annual Sickle Cell Disease Research Meeting

The National Heart, Lung, and Blood Institute (NHLBI) will host its annual Sickle Cell Disease Research Meeting on August 26-28, 2019, in Bethesda, MD. This three-day meeting provides a yearly forum for investigators, practitioners, and interested health care providers to discuss the process of ongoing clinical trials, hear presentations about new developments in scientific and clinical aspects of sickle cell disease, and interact with other investigators and NHLBI program staff in an informal setting. This year’s program includes six symposia that cover a broad range of topics, including reproductive challenges of women with SCD and curative therapies in SCD. Additionally, National Institutes of Health program staff will present on the progress of two major initiatives: The NHLBI Sickle Cell Disease Implementation Consortium and the Cure Sickle Cell Disease Initiative. Onsite registration will be available. Click here to view the agenda. 

Annual Sickle Cell Adult Provider Network Symposium 

This year’s annual Sickle Cell Adult Provider Network (SCAPN) symposium will be held on Wednesday, August 28, 2019, during the upcoming National Heart Lung and Blood Institute Sickle Cell Disease Clinical Meeting in Bethesda, MD. This year’s symposium will focus on developing and improving adult sickle cell programs.  There will be four speakers representing both new and established programs of various sizes led by hematologists and a general internal medicine-pediatrics provider. The chairs of the symposium are Wally Smith, MD (Virginia Commonwealth University); John Roberts, MD (Yale New Have Health Care); and John Strouse, MD, PhD (Duke University School of Medicine). Speakers will include Sophie Lanzkron, MD, MHS (Johns Hopkins University); Payal Desai, MD (The Ohio State University); Biree Andemariam, MD (University of Connecticut); and Tiffany Pulcino, MD, MPH (University of Rochester).  For more information, contact Shirley Miller, SCAPN Coordinator at email shirleyrenee3@gmail.com.

Sickle Cell Science: Path to Progress Webinar Series 

The National Heart, Lung, and Blood Institute and key partner agencies and institutions will host a four-part webinar series in September to highlight Sickle Cell Awareness Month. Every Wednesday in September from 1-2pm ET, the webinar presenters will address some of the educational and informational needs expressed by patients, family members, researchers, health care providers, and others in the sickle cell disease community. The webinar series will cover implementation efforts in the United States and worldwide, and will address genetic therapies, bone marrow transplants and other therapies, and progress in pain management. 

  • September 4:Serving the Sickle Cell Disease Community Here and Abroad
  • September 11: Genetic Therapies in Sickle Cell Disease
  • September 18: Bone Marrow Transplants, Other Therapies, and Sickle Cell Disease
  • September 25: Sickle Cell Disease Care in the Emergency Department: Improvement Initiatives and Ongoing Research (CME available for this webinar)

Planning on tuning into the webinar series? Follow the conversation on Twitter using #ScienceofSCD.

Sickle Cell Disease Therapeutics Conference 

Global Blood Therapeutics (GBT) in partnership with the Sickle Cell Disease Association of America (SCDAA) will hold the 8th Annual Sickle Cell Disease Therapeutics Conference (SCDTC) on Tuesday, September 10, 2019 at The Westin City Center in Washington D.C. This annual forum convenes key community stakeholders including healthcare professionals, individuals living with sickle cell disease (SCD), advocates, investors, policy makers, and industry representatives to discuss relevant topics related to SCD along with the latest developments in treating SCD. This year’s event will be hosted by Beverley Francis-Gibson, SCDAA’s President and CEO.  Conference speakers include: Biree Andemariam, MD (SCDAA, University of Connecticut); Lakiea Bailey, PhD (Sickle Cell Community Consortium); Chuck Chesson, PhD (ASH Research Collaborative); Jeremie Estepp, MD (St. Jude Children’s Research Hospital); Andre Harris, CHW (Foundation for Sickle Cell Disease Research); Greg Kato, MD (University of Pittsburg); Allison King, MD, MPH, PhD (Washington University in St. Louis); and Hertz Nazaire (artist and SCD advocate). Can’t attend in person? Sickle Cell Warriors has partnered with GBT to livestream the conference via Facebook Live. For more information, visit the official SCDTC website: http://scdconference.com/.  

Sickle Cell Society UK Celebrates 40 Years 

The Sickle Cell Society is excited to be hosting its 40th Anniversary Gala Ball on Saturday, September 14 at the Royal National Hotel in Central London. The Gala Ball celebrates 40 years of working within the sickle cell community and will include a three course buffet, music, dancing, and a charity raffle. Find out more here: https://scs40galaball.eventbrite.com.

ASH RC Sickle Cell Disease Community Engagement Workshops

The ASH Research Collaborative (ASH RC) will host the final Sickle Cell Disease (SCD) Community Engagement Workshop on Saturday, September 14, 2019 in Washington, DC. The SCD Clinical Trials Network is committed to having individuals living with SCD involved early and in a meaningful way as the network develops strategies to improve enrollment, design, and execution of clinical trials. The goals of the workshop are to 1) understand barriers to clinical trial participation, as well as identify solutions to overcome challenges and; 2) prioritize research areas of interest to the community. 

Learnings from the workshops will help us develop a SCD Patient-Oriented Research Priority Report to be shared with SCD community stakeholders, establishing a common understanding of goals for community-focused SCD research. This report will be a living document that will be reevaluated as research, novel treatments, and therapeutics are developed further.  Please spread the word about the workshop within your SCD community.

SCDAA National Convention

Sickle Cell Disease Association of America’s 47th National Convention is scheduled for October 9-12, 2019, in Baltimore, MD. The National Convention is a four-day conference designed to address the multi-factorial aspects of sickle cell disease (SCD) and sickle cell trait. In an effort to advocate for improved quality of life for individuals and families affected with SCD and its associated morbidity and mortality, the conference 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, interactive panel discussions, and special events. This year’s keynote speaker is Dr. Amanda Brandow, from the Medical College of Wisconsin, who will address “The Study of The Microbiome in Sickle Cell Disease Pain.” Dr. Brandow’s research is focused on understanding the pathophysiology of acute and chronic pain in children and adults living with SCD. Click here to learn more and register today!

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