New study helps families of children with sickle cell disease make informed treatment decisions
JUNE 1, 2025—Sickle cell disease is a serious blood disorder that's passed down from parents. It affects many people, especially those who are Black or Hispanic. It can cause severe pain, organ damage, reduced quality of life and shortened life expectancy. A cure for sickle cell disease exists—a blood or marrow transplant (BMT) using cells from a donor. However, BMT has risks and benefits that families and care teams need to consider when they're deciding what's best for each person.
NMDPSM is proud to support the WeDecide study, which compares BMT and non-BMT treatments for children, adolescents and young adults with sickle cell disease.
"Right now, BMT is the recommended curative treatment option for people with sickle cell disease. However, it's essential to weigh its risks and benefits compared to other treatments," explained Katie Schoeppner, MSW, LICSW, senior director of Patient Services at NMDP. "The WeDecide study is designed to help families and their health care teams do just that."
"By authentically engaging those most impacted by sickle cell disease, we generate the data and insights needed for families and doctors to make informed, personalized treatment decisions—together," says Raymona Lawrence, DrPH, MPH, Georgia Southern University.
WeDecide is a critical study that centers the voices of patients and families, ensuring they are true partners in care."
RAYMONA LAWRENCE, DrPH, MPH
Georgia Southern University
The study also lays the groundwork for future research involving gene therapy, which has been approved as a sickle cell disease treatment.
Who can join the WeDecide study
WeDecide will enroll 480 patients with sickle cell disease between the ages of 3 and 21 years old. Participants include:
- 160 patients receiving a transplant using bone marrow from a fully HLA-matched brother or sister through a Sickle Cell Transplant and Research Alliance (STAR) center
- 320 patients who haven't had a BMT before receiving non-transplant disease-modifying therapy (such as medicine to reduce pain crises) through a Globin Research Network for Data and Discovery (GRNDaD) registry center
Enrollment continues through 2027, with participants followed for three years. This will allow researchers to compare outcomes between the two groups.
Understanding the long-term benefits and risks of bone marrow transplantation compared to standard treatment will be a game-changer for sickle cell care."
DERECK DAVIS, MD
University of Mississippi
WeDecide study goals
The WeDecide study will:
- Compare how BMT from a matched sibling and non-transplant treatments affect quality of life and cognitive function (such as thinking, learning and memory)
- Describe risks and benefits of BMT from a sibling donor in children and adolescents with sickle cell disease
- Identify factors that may help determine which children are most likely to benefit from BMT
This study offers a thorough assessment that will inform and empower us in the decision-making process! I wholeheartedly welcome this initiative, which will provide much-needed clarity and guidance for our community."
BIBA TINGA
Executive Director of the Sickle Cell Association of Canada, Montreal
WeDecide study sites in the U.S. and Canada
U.S. study sites
- Children's Healthcare of Atlanta, Ga.
- University of Alabama at Birmingham, Ala.
- Children's Hospital of Alabama, Ala.
- Atrium Health, Charlotte, N.C.
- UNC Chapel Hill, N.C.
- UNC Charlotte, N.C.
- Nemours Children's Health, Wilmington, Del.
- Dana-Farber Cancer Institute, Boston, Mass.
- Boston Children's Hospital, Mass.
- Hackensack Meridian Health, N.J.
- The University of Chicago, Ill.
- Lurie Children's Chicago, Ill.
- Children's National Hospital, Washington, D.C.
- Children's Hospital of Philadelphia, Pa.
- St. Jude Children’s Research Hospital, Memphis, Tenn. (HQ)
- Indiana University-Purdue University Indianapolis, Ind.
- Indiana Riley Children Hospital, Indianapolis, Ind.
- University of Oklahoma, Oklahoma City, Okla.
- Montefiore Hospital, Bronx, N.Y.
- Columbia University Medical Center, New York, N.Y.
- Cohen’s Children’s Medical Center, Queens, N.Y.
- Roswell Park Comprehensive Cancer Center, Buffalo, N.Y.
- University of Rochester Medical Center, N.Y.
- Washington University in St. Louis, Mo.
- Nationwide Children’s Hospital, Columbus, Ohio
- UT Health San Antonio, Texas
- Methodist Hospital, San Antonio, Texas
- Texas Children’s Hospital/Baylor College of Medicine, Houston
Canada study sites
- Children's Hospital of Winnipeg, Manitoba
- Alberta Children's Hospital, Calgary, AB
- University of Montreal, QC
- Sick Kids, Toronto
- BC Children's Hospital, Vancouver
This work is supported through a Patient-Centered Outcomes Research Institute (PCORI®) Award (BPS-2023C1-31041).
NMDP resources for families
Explore resources
Find helpful tools if you're considering or getting ready for BMT for sickle cell disease—including a free resource kit and journal to guide your appointments.
Search for clinical trials
Learn how NMDP clinical trial navigators can guide you in searching for and joining trials for sickle cell disease. An online search tool is also available.
Get personalized support
Tap into an extra layer of support for your non-medical needs. Our sickle cell disease patient navigators will answer your questions and offer guidance tailored to you.