Leading the Way in HLH Research Studies

Innovative research leads to medical breakthroughs that improve outcomes for children with HLH. Cincinnati Children’s helps lead research studies that could change how we diagnose and care for patients with HLH.

Our physician-researchers focus on innovative basic and translational research with the ultimate goal of improving curative treatments for HLH. They maintain strong collaborations with the National Institutes of Health (NIH) and other HLH centers around the globe, including in China, Japan, Vietnam, Canada and Sweden. Our goal is to further extend our scientific collaborations to new centers worldwide.

We are also working to increase research funding for HLH through industry grants, additional NIH funding and philanthropic support.

Antibody Therapy Offers a Unique Treatment for HLH

Emapalumab is the first Food & Drug Administration (FDA)-approved therapy for primary HLH. The FDA approved it in 2018 after an international clinical study showed that it was safe and effective for treating HLH. Michael Jordan, MD, a pediatric hematologist at Cincinnati Children’s, was the principal investigator in the United States for the international clinical study that was the basis of the FDA’s approval of emapalumab.

In clinical trials 63% of patients experienced a response to the drug, and 70% were able to proceed to stem cell transplant. Unlike chemotherapy, emapalumab does not suppress the bone marrow, and for many patients that could be the difference in surviving to transplant.

The INTO-HLH Registry

Cincinnati Children's leads the INTO-HLH (Insight into the Natural history and Treatment Outcomes of Hemophagocytic Lymphohistiocytosis) Registry, which is aimed at advancing what is known about HLH. The insights obtained from this Registry will help doctors to better diagnose and manage HLH and improve treatment outcomes for future patients. This Registry is open to all individuals, both living and deceased, with clinically suspected or confirmed diagnosis of HLH or related disorders  (e.g., MAS, CRS.) Please visit the INTO-HLH website to learn more or to enroll in the study.