Study finds one treatment stands above others for adults with Langerhans cell histiocytosis

A study by a Baylor College of Medicine physician-researcher has shed light on the most effective treatment for adults with Langerhans cell histiocytosis (LCH) in bones. LCH is a disease that can affect the skin, mouth, ears, bones, brain, gastrointestinal system, liver, spleen, or bone marrow.

In the study, appearing in the current issue of , researchers reviewed for 58 adults with the disease and compared the effectiveness of three chemotherapy treatments – vinblastine/prednisone, 2-Chlorodeoxyadenosine, and cytosine arabinoside.

"Cytarabine (cytosine arabinoside) is clearly the winner here – it's the most effective and least toxic," said Dr. Ken McClain, professor of pediatrics – hematology/oncology at BCM and director of the Histiocytosis Program at Texas Children's Cancer Center.

The study is important because LCH is rare in adults and there is very little research on the disease. There have been no studies in the literature up to now that compare different treatments, according to McClain.

"The No. 1 thing that I hope comes out of this study is that physicians stop using vinblastine/prednisone, which is the least effective and most toxic chemotherapy treatment for this disease," he said.

LCH occurs when white blood cells called histiocytes and lymphocytes gather together and attack the skin, bones, brain, , major organs, mouth and ears. It affects children and adults and can range from a single skin lesion to multi-organ involvement. It can be chronic and debilitating and, in some cases, fatal.

The study reviewed patients who were seen from 2001 to 2011 at BCM. It offers data that could be useful for future clinical trials but, more importantly, it provides literature for physicians who do not know how to treat patients with this rare disease.

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