Initiating an improved intensity of chemotherapy in children with severe Langerhans cell histiocytosis (LCH) can lessen the mortality rate for this disorder by almost 20 percent when the patient displays a quick response to such treatment, according to a new international study.
The study is the second in a series of international randomized clinical trials for Langerhans cell histiocytosis coordinated by the Histiocyte Society.
'The incidence of this particular disorder is so rare, without the cooperation of researchers and participants across the globe, the trial could not have been conducted on a large enough scale to see such positive, and definitive outcomes,' noted senior author Stephan Ladisch, MD, Bosworth Chair for Cancer Biology and Director of the Center for Cancer and Immunology Research at Children's National Medical Center.
'The value of international collaborations such as this is incalculable to the research of so-called 'orphan diseases' that receive little government funding support, but still have a significant effect on the lives of children around the world,' he added.
The trial induced a rapid response to the application of chemotherapy—involving prednisone, vinblastine, and for some participants, the addition of etopisode. As a result, the research team observed a statistically significant improved prognosis for children with multi-system LCH.
The researchers observed this significant effect in both study groups of the second clinical trial.
'There is a strong indication that increasing treatment intensity was associated with higher rapid respond and survival rates. This suggests that therapy intensification...may be essential for successful treatment of high-risk disease,' the authors said.
The results were based on the findings in 193 pre-identified LCH patients identified as 'risk patients,' those with LCH affecting risk organs including the liver, lungs, hematopoeitic system, and spleen, or patients with disease onset younger than 2 years of age.
The study is published in the March 1 edition of Blood, the journal of the American Association of Hematology.