Childhood Histiocytosis Program

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Histiocytosis is a general name for a group of rare disorders characterized by increased numbers of blood cells called histiocytes in the blood and tissues. These cells usually help fight infection and destroy certain foreign substances in the body. When a child has histiocytosis, these cells begin to attack the body, targeting bodily organs including the bone marrow, liver, spleen, lungs, skin, bone and brain. Histiocytosis usually occurs in children, often during infancy, but also in adults.

There are two major types of histiocytosis:

Other related diseases include:

  • Juvenile xanthogranuloma
  • Rosai Dorfman disease

Dana-Farber/Boston Children's Cancer and Blood Disorders Center provides expert treatment for newly diagnosed or recurrent histiocytosis, which may involve treatment with chemotherapy. Our doctors provide unparalleled expertise in the diagnosis and treatment of the major types of histiocytosis — LCH and HLH — and treat its related disorders as well.

How We Diagnose and Treat Histiocytosis

Our team uses a variety of laboratory and diagnostic imaging studies to determine if a child has LCH, HLH, or another form of the disease. This may include blood tests, urinalysis, a bone marrow biopsy, liver or pulmonary function tests, or imaging studies like X-rays or CT, MRI, or PET scans.

We treat our patients in accordance with protocols established by the International Histiocyte Society. Treatment depends on the individual patient and is planned after doctors determine the type and extent of the disease. In some cases, minimal treatment with steroids may be sufficient. Systemic chemotherapy may be needed in patients with more extensive disease. If a child has HLH and needs a stem cell transplant (also called a bone marrow transplant), Dana-Farber/Boston Children's is one of the oldest, largest, and most experienced pediatric stem cell transplant centers in the country.

Our Histiocytosis Treatment Specialists

Our patients have access to the broadest set of pediatric, hematologic, and oncologic expertise available. The breadth of our expertise allows us to assemble a team of experts to meet the specific needs of each patient.

At the core of the treatment team are hematologists and oncologists who specialize in treating children with histiocytosis. Because many organ and body systems may be involved with the disease, a patient’s treatment team may also include dermatologists, pulmonologists, endocrinologists, gastroenterologists, neurologists, dentists, orthopedic surgeons, neurosurgeons, radiologists, and radiation oncologists.

We round out the team with experts that help the child prepare for life during and after treatment, including psychiatrists and psychologists, child life specialists, social workers, nutritionists, and school specialists.

All senior medical staff members of the histiocytosis treatment program participate in clinical research activities, so our patients have access to the very best and up-to-date treatments available.

See a complete list of the specialists in our Childhood Hematologic Malignancy Center

Histiocytosis Research and Clinical Trials

The main objective of our histiocytosis research program is to improve our understanding and treatment of LCH and HLH. Our research program offers unique access to clinical trials in which children can receive the newest treatments, including trials sponsored by the Histiocyte Society. A major focus of our research is the rapid translation of scientific discoveries to the bedside to benefit patients. In order to determine the effectiveness of treatments, we have established a clinical database to identify patterns of disease in patients with LCH. Our scientists are also working to develop reduced-intensity treatment strategies that have fewer short-term and long-term side effects.