Treatment Options

Although there are several types of histiocytic disorders, we usually treat them the same way. The current standard treatment uses chemotherapy. However, for patients whose disease cells have certain genetic mutations, Cincinnati Children's uses an oral medicine called a gene inhibitor instead of chemotherapy.

To diagnose a histiocytic disorder, we’ll take a biopsy of the skin, bone or tissue lesion. Our pathology team tests and examines the biopsy to determine the diagnosis and if there are any genetic mutations.

In some cases, Langerhans cell histiocytosis (LCH) and other histiocytic disorders don't need treatment. If the disease is limited to just the skin or one bone lesion, it will likely go away on its own. Patients can continue regular check-ups to monitor the disease in case it spreads and needs treatment.

We will treat a histiocytic disorder if it involves more than one part of the body. For example, you or your child will need treatment if we find the disease on the skin and the liver, or if we find multiple bone lesions.

Standard Treatment: Chemotherapy

If the histiocytic condition affects multiple parts of the body, the standard treatment is chemotherapy. The treatment uses a combination of two or three drugs. Chemotherapy is given through the veins (intravenously) and is usually done at an outpatient clinic.

While many children can tolerate the side effects of chemotherapy, the treatment can cause fatigue, mild nausea, low blood counts and weight changes.

Gene Inhibitors

For some patients, Cincinnati Children's offers an alternative frontline treatment for histiocytic disorders. The treatment is an oral drug that blocks certain gene mutations that cause histiocytosis.

In recent years, scientists have discovered that LCH and other histiocytic disorders are caused by the same gene mutations as melanoma. Based on this discovery, Ashish Kumar, MD, PhD, a physician-scientist at Cincinnati Children's, started treating histiocytic disorders with a melanoma treatment that targets those gene mutations.

Since starting this treatment, every one of Kumar's patients has responded positively and none has relapsed while on the treatment. The side effects are also minimal compared to the standard chemotherapy treatment.