Specialists at Cincinnati Children’s have developed a new, highly effective therapy for patients with Langerhans cell histiocytosis (LCH). It is an alternative to chemotherapy, which can cause short- and long-term side effects. In the first three years, 100 percent of our patients have responded positively, and none has relapsed.

The LCH Center cares for patients from our local area, across the nation and around the world. Our multidisciplinary team includes pediatric hematologists / oncologists, a care manager, nurse practitioners and a psychologist. They collaborate closely with other specialists at Cincinnati Children’s to ensure that patients receive comprehensive, personalized care.

We care for children, adolescents, young adults and – in some cases – older patients with LCH and non-LCH forms of the disease.

Innovative Treatment

For many years, no one knew what caused Langerhans cell histiocytosis. But doctors did know that it was a “cancer-like” condition. They treated it with chemotherapy, which is effective for many patients with LCH but can have significant side effects. Even when chemotherapy works for patients with LCH, the relapse rate at the one-year mark is as high as 40 percent. And chemotherapy does not work for 15 percent to 20 percent of patients with LCH.

Ashish Kumar, MD, PhD, a physician-scientist at Cincinnati Children’s, began exploring a new treatment approach for Langerhans cell histiocytosis that uses the drugs dabrafenib and trametinib.

Dr. Kumar

Scientists discovered that LCH is caused by the same gene mutations as melanoma (a type of skin cancer). In 2016, Ashish Kumar, MD, PhD, a physician-scientist at Cincinnati Children’s, began exploring a new treatment approach based on this discovery. He thought that certain oral drugs used to treat melanoma (dabrafenib and trametinib) could be effective in treating LCH.

Kumar used one of the drugs on two patients whose LCH was not responding to chemotherapy. Both patients made a full recovery. Kumar began offering the new treatment as a first-line therapy for patients with LCH. In the first three years of using this therapy at Cincinnati Children’s, every patient has responded positively to the treatment, and none has relapsed. The children who received this therapy have not experienced any side effects, and are growing and developing normally.

Not all patients with LCH need treatment. If a patient’s symptoms are limited to an isolated bone lesion with no sign of disease elsewhere, it is likely that the disease will go away on its own. When a patient does need treatment, our team offers the choice of the new therapy or chemotherapy.

LCH Research Efforts

Kumar and colleagues at the LCH Center are preparing a new clinical research study to further evaluate the safety and effectiveness of using dabrafenib and trametinib to treat LCH.