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UR Medicine Golisano Children’s Hospital received a multi-million-dollar grant to lead a first-of-its-kind study comparing the effectiveness of treatments for sickle cell disease. Funded by the (PCORI), the research aims to help families make more informed treatment decisions for children of different ages and at various disease stages.

The project will compare two treatment options:

The study aims to recruit 480 children, adolescents and young adult patients. Approximately 320 participants receiving disease modify therapies will be recruited by centers participating in the Globin Regional Data and Discovery (GRNDaD) registry, and 160 participants receiving HLA MRD BMT will be recruited by centers participating in the Sickle Cell Transplant Advocacy and Research Alliance (STAR). “This large sample size will allow us to thoroughly compare the two treatments and assess how they impact quality of life,” said John Horan, MD, MPH, the study’s lead investigator and a professor in the division of Hematology and Oncology.

Horan emphasizes that the findings will help families make choices based on the relative risks and benefits of each treatment. While an HLA-matched bone marrow transplant offers a potential cure, it requires a genetic match, most often from a sibling, and carries a very small risk of death. Additionally, transplant recipients can develop graft-versus-host disease (GVHD), where the donor’s immune cells attack the recipient's body, leading to complications such as rashes or organ damage.

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For children who lack an HLA matched brother or sister or choose not to undergo a transplant, disease-modifying therapies are the alternative. Treatments like blood transfusions and Hydroxyurea (a chemotherapy drug) help manage the symptoms of sickle cell disease, the most common of which is severe pain. Pain occurs when sickled blood cells block blood vessels, depriving tissues of oxygen, which can lead to tissue death. These episodes are often compared to the intense pain of a heart attack.

“This study will allow us to look at specific subgroups of patients,” Horan added. “A three-year-old who hasn’t experienced significant complications will have different treatment needs compared to a 19-year-old with multiple health issues. This research will help determine which treatment path makes the most sense based on a combination of factors.”

Horan is a co-founder of (Sickle Cell Transplant Advocacy & Research Alliance) a nonprofit organization formed by pediatric hematologists and stem cell transplant experts to improve the lives of children with sickle cell disease through blood and marrow transplants.

“We have 25 to 30 centers involved in this study,” said Horan. “We’re hopeful that it will provide critical answers to families managing sickle cell disease across the country.”