For families weighing BMT for sickle cell disease, better information matters
For families weighing BMT for sickle cell disease, better information matters
Original published date: 6/23/2026
Dr. Noronha sees parents face the same hard decision again and again. The WeDecide study is built to help them.
Understanding how a treatment impacts a person’s whole life
Children, teens and young adults with sickle cell disease may follow different treatment paths. Some have a BMT—which is also known as a bone marrow transplant or blood stem cell transplant. Their blood stem cells for the transplant come from someone who is related to them, like a brother or sister, or an unrelated donor from the NMDP RegistrySM. Others may continue with non-transplant treatments, like medicines that help lower pain and complications.
The WeDecide study compares those who receive BMT using blood stem cells from a sibling to non-transplant treatments.
What makes this study different is that it's not just about collecting medical facts or data.
The study aims to help families get answers to questions like:
- Will a transplant improve quality of life?
- What are the risks and benefits?
- Which children are most likely to benefit from transplant?
We wanted to do this study to be able to really compare the lived experiences of both groups.
Suzie Noronha, MD, FAAP
Sickle cell disease physician at URMC | Youth Stakeholder Committee lead for WeDecide
Families need more than numbers
Traditional research often focuses on things that are easier to measure, like lab values, emergency room visits or test results.
Dr. Noronha—who leads the Youth Stakeholder Committee for the WeDecide study and is the local principal investigator at URMC—says those data only tell part of the story.
"The data we get from traditional studies are only the tip of the iceberg. It gives you a very, very small glimpse into the life of a person who lives with this disease. To really accurately judge the effectiveness of a treatment, we have to understand how it impacts their whole life, not just the numbers that are easy to measure and compare in statistics," she explained.
That's why patient-reported outcomes, or PROs, are such an important part of WeDecide.
PROs let patients share how they feel in their own words, without a doctor interpreting their answers. These surveys ask about things like pain, tiredness, mood, mental health, memory, daily life and overall well-being. They help researchers understand what treatment looks like in real life, not just on paper.
For families who are already taking part in the study, filling out the PRO survey matters. Each survey adds to what doctors and researchers can learn. Each answer can help future families make a more informed choice.
A study built with the sickle cell community
Something else that makes this study unique—it included the voices of teenage and adult patients, caregivers and health care providers in its design from the start.
"We really wanted to understand what patients and parents wanted to know from a study like this. WeDecide is a Patient-Centered Outcomes Research Institute (PCORI®) project. What that means is the patient is central to everything we do. We want their opinions and input into how we design, conduct and communicate about the study," Dr. Noronha shared.
Their feedback helped shape the study questions and the surveys themselves. The goal was to make sure the study asks questions that feel relevant, respectful and worth the participants’ time to answer. And, most important, that the information that comes out of the study is helpful to the sickle cell disease community.
Having useful information comparing the quality of life of the two groups is what they said is most important to help them make informed decisions.
Dr. Noronha
Dr. Noronha said that through how they’re designed and how the outcomes are shared, studies like WeDecide can help build trust by showing that researchers and physicians are working with—and for—the sickle cell community.
A gift to future families
Many families join the study because they want to help others, even if the study will not directly help their own child right away. They want future patients and families to have better information, better care and better lives, Dr. Noronha shared.
And for the families already participating in the study, she has a simple message: thank you.
Sharing a little aspect of their lives will provide such meaningful information to future patients and families who are embarking on one of the most important decisions of their child's life.
Dr. Noronha
Families who join WeDecide are doing more than participating in research. They're helping build a clearer path for the families who come next.
WeDecide study updates
WeDecide is enrolling children, teens and young adults ages 3 to 20 who have sickle cell disease.
- 160 patients will receive a transplant using bone marrow from a fully HLA-matched brother or sister
- 320 patients who haven't had a BMT before will receive non-transplant care
This work is supported through a PCORI Award (BPS-2023C1-31041).
WeDecide study sites in the United States 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
- University of Montreal, QC
- Sick Kids, Toronto
- BC Children's Hospital, Vancouver
WeDecide study milestones
- As of March 2026
- 94 patients enrolled receiving BMT
- 128 patients enrolled receiving non-BMT treatment
- Through 2027WeDecide study enrollment continues in the U.S. and Canada
- Through 2030PRO follow-up continues for three years after enrollment
- 2031WeDecide study results expected
Free sickle cell disease resources for patients and families
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