Sickle Cell Disease Research Network

Facilitating Sickle Cell Disease Research to Improve Clinical Outcomes

Sickle Cell Disease Research

When you make decisions about how to manage your life with sickle cell disease (SCD), you are most likely relying on knowledge gained through clinical research. Clinical research is designed to answer medical questions.

There are different types of clinical research. One approach to clinical research is a clinical trial, which tests the safety and effectiveness of new medications and treatments or looks at new ways of using existing medications. Other types of clinical research studies look at the most effective ways to identify or prevent complications of a disease.

Clinical research helps lead to improvements in care and better health and quality of life for those living with SCD.

Promising new therapies and treatments are on the horizon, and there has never been more excitement for SCD research.

But clinical research cannot deliver the solutions the community needs without the involvement of the individuals who participate in the research studies.

The ASH Research Collaborative® (ASH RC) is committed to making sure the community has a central role in helping design research that fits the needs of the SCD community. Together, we will improve the health and quality of life for all affected by SCD through research.

Research for Better Health

You are probably familiar with the following treatments for SCD. But did you know about the role of clinical research in making them available?

Hydroxyurea

Hydroxyurea was the first medication for the treatment of adults and children with sickle cell disease to be approved by the U.S. Food and Drug Administration (FDA). This approval by the FDA was based on information learned from clinical research studies.

In one of the early research studies, The Multicenter Study of Hydroxyurea in Sickle Cell Anemia, adults with SCD volunteered to receive either hydroxyurea or a placebo (a pill that has no medicine) under the close watch of their doctors. The research participants who took hydroxyurea had fewer pain crises, fewer episodes of acute chest syndrome, needed fewer blood transfusions, and had fewer hospital stays. The study also showed that there were very few side effects caused by the medicine. The information learned from this study was published in 1995.

Capsules

Since then, there have been many more research studies of hydroxyurea in both adults and children that have shown it to be a safe and helpful medicine for the treatment of SCD. Individuals with sickle cell disease and their doctors rely on the information learned through clinical research when talking about and making decisions about starting hydroxyurea treatment.

Learn more: Hydroxyurea for SCD

Penicillin

Penicillin was discovered in 1929, and in the years that followed, doctors learned it could prevent and treat a variety of infections.

In 1986, an important research study, the Penicillin Prophylaxis in Sickle Cell Disease (PROPS) study, showed that infants with SCD who received daily penicillin had an 84% reduction in life-threatening blood infection compared to children with SCD who did not receive penicillin.

This study led to an increase in newborn screening to identify infants with SCD so doctors could make sure that babies with SCD could receive and benefit from this life-saving treatment.

Today, infants with SCD are started on Penicillin in the first few months of life. This has greatly reduced the number of deaths from life-threatening infections in children with SCD who are under 5 years old.

Learn More About Approved Medications

Each of the medications that is currently approved by the FDA to treat SCD was developed and then tested for safety and effectiveness through research.

To learn more about Crizanlizumab (Adakveo®), L-Glutamine (Endari®) and Voxelotor (Oxbryta®) the research that led to their approval, consult the Sickle Cell Disease Coalition SCD Therapy Fact Sheets.

There are more improvements to be made, but together we can ensure there are more success stories in the treatment of SCD!

Advancing Clinical Research to Improve Care

The ASH RC has two programs that are focused on improving outcomes for individuals living with SCD: The SCD Research Network and the Data Hub Sickle Cell Disease Program.

The SCD Research Network’s (SCD Network) mission is to advance research in SCD with a focus on research that holds the most promise for improving the health and quality of life for those living with SCD.

To ensure the voice of the SCD community is guiding all aspects of the SCD Network, local and National Community Advisory Boards (CABs) have been established. The ASH RC’s secure Data Hub is a central location that collects health information about individuals living with SCD.

Local CABs are affiliated with the SCD Network sites and serve as the connection between the community and the research team. They include individuals living with SCD, loved ones, or other advocates who help make sure research is sensitive to local needs and cultural issues. They also connect the research team with individuals affected by SCD in the community who may want to participate in studies.

Two members from each local CAB serve on the National CAB, which focuses on issues of importance to the whole network. They give feedback on research plans and connect the local CABs to one other and the ASH RC.

Interested in getting involved? Learn more about what our CABs are doing.

The Data Hub

The ASH RC’s secure Data Hub is a central location that collects health information about individuals living with SCD. When information from many people is combined, it can help health providers and researchers better understand the experience of those living with SCD and find ways to improve care through research and actions to improve quality of care.

Learn More

The ASH RC’s vision is to ensure you can:

Learn about opportunities to participate in clinical research.
Access better information to help you decide whether to participate in research.
Have a voice in how clinical research is set up and run.
Learn about the results of research studies and progress toward the development of new treatments.
Trust the research sites that are part of the SCD network.

Do you have questions about clinical research for SCD?

View the ASH RC guide for a more in-depth overview of what it is, how to participate, and resources that can further your understanding.

View the ASH RC Guide

SCD Community Engagment

To lay the foundation for the Sickle Cell Disease (SCD) Research Network, the ASH Research Collaborative® (ASH RC) organized community engagement workshops across the U.S. We extend heartfelt thanks to individuals living with SCD and their loved ones who generously shared their insights during these sessions.

The ASH RC recognized the critical need for community-focused research that truly reflects the voices and concerns of the SCD community and took on the charge of creating a dedicated community engagement program to support this effort. Insights gathered from these workshops were published in Blood Advances. The report highlights key barriers to clinical trial participation and offers solutions to overcome these challenges, ensuring that research priorities align with what matters most to the SCD community.

Read the full report here.

Contact Us

If you have any questions about the ASH RC’s work with the SCD community and how you can get involved, contact us!