Hemophagocytic lymphohistiocytosis (HLH) was once considered incurable, but that’s not the case anymore. Today, many promising therapies offer new hope to patients and families. At Cincinnati Children’s, our experts are at the forefront of HLH care and research. They provide rapid, accurate diagnoses and personalized therapies for children with HLH and are helping providers from other institutions care for their patients as well. Their research is leading to higher survival rates and more lasting cures.

Families who have faced this disease will tell you that battling HLH is a long and difficult journey. At the HLH Center of Excellence, we provide expert medical care, emotional support and other resources a family needs to meet the challenges ahead.

Why Choose Cincinnati Children’s

Expert Team

Cincinnati Children’s is the most experienced hospital in the nation in treating HLH. Our HLH team includes researchers and physicians who have studied the disease extensively. They care for between 10 and 20 children with HLH a year, a large number considering how rare HLH is.

HLH affects the immune system, causing uncontrolled inflammation that damages organs and can rapidly lead to death. Unfortunately, many physicians don’t have the experience to recognize the symptoms. As a result, HLH is often misdiagnosed or diagnosed too late. Most patients without access to effective treatments such as hematopoietic stem cell transplantation will die of their disease. A cure is possible, but an accurate and quick diagnosis is essential.

Advanced Testing Through Two Specialty Labs

The Diagnostic Immunology Laboratory and Molecular Genetics Laboratory at Cincinnati Children’s offer sophisticated tests with rapid results—essential for patients with HLH. These renowned labs have been offering HLH testing for more than 20 years, and the staff provides expert interpretation of test results. All of our patients undergo advanced genetic testing to see if a genetic mutation (change) has caused the disease. Our experienced genetics counselors work with physicians to determine how test results should guide the care plan.

State-of-the-Art Care

The standard of care for HLH is to use a combination of chemotherapy and corticosteroids to cool down and control abnormal inflammation in the body. This is often effective, allowing children to continue to the next phase of treatment, which is hematopoietic stem cell transplant (HSCT).

The goal of HSCT is to cure HLH by replacing the body’s immune system. The Bone Marrow Transplantation Program at Cincinnati Children’s has performed more than 2,200 HSCTs since the program was founded in 1986. Currently, the program averages 100 transplants each year, making it one of the largest programs in the U.S. Physicians and researchers in the Bone Marrow Transplantation Program have created exciting new HSCT approaches for many rare diseases, including HLH.

Research Opportunities and Breakthroughs

Our physician-researchers participate and often lead national studies to find better treatments for HLH. Sometimes they can offer emerging therapies to children with HLH—long before these therapies are available elsewhere.

Our researchers recently helped pioneer a new treatment for HLH that is less toxic and potentially more effective than chemotherapy. This treatment uses the drug emapalumab to block a harmful inflammatory molecule called interferon gamma. Emapalumab is approved by the Food and Drug Administration (FDA) for use after other therapies have not worked well. Our researchers are studying whether it can be offered as a first-line treatment instead of chemotherapy.