Grants Awarded

Research Infrastructure for Clinical and Translational Research in Langerhans Cell Histiocytosis (LCH)

Principal Investigator
Carlos Rodriguez-Galindo MD
Dana-Farber Cancer Institute – Boston, Massachusetts USA

Date of Award
December 2012

Amount of Award

Layperson Summary
Langerhans Cell Histiocytosis (LCH) is a poorly understood disease with variable presentation. Studies
conducted by the Histiocyte Society have helped to identify prognostic factors, risk stratification principles, and
the best chemotherapeutic regimens. However, many challenges remain, including improving survival for
patients with more severe disease, decreasing reactivation, and preventing and treating associated
neurological deterioration. The LCH-IV trial will provide a comprehensive approach to study the spectrum of
clinical presentations while seeking to improve cure rates and duration of front line treatment, decrease
reactivation rates, improve treatment of recurrent disease, and prevent long-term effects. The LCH-IV protocol
includes 7 separate strata, which will function as individual protocols to which patients are eligible during the
time course of their disease. The LCH data center in Vienna will assume centralized data management and
statistical support and will play the role of coordinating center for European institutions. There is no
coordinating center in North America; yet strong participation from North American institutions is needed for
successful completion of the trial. The LCH-IV trial will require rigorous oversight and meticulous attention to
detail to ensure that all institutions comply with federal regulations and that data is collected and managed
properly. The resources requested in this proposal would help to develop necessary research infrastructure to
launch the LCH-IV in North America, facilitate trial activation, train data managers in the use of the web-based
data management tools, and coordinate the regulatory aspects. This proposal also aims to enhance clinical
and translational research by facilitating the creation of an LCH Consortium with the research infrastructure
necessary to develop and implement effective clinical and translational studies and biology research in LCH.
The consortium will focus on further development of biologically targeted approaches to treat patients with LCH
in order to improve outcomes, including prevention of treatment and disease-associated toxicities.

Twelve Month Report