Grants Awarded

Date of Award
December 2015

Amount of Award
$50,000

Layperson Summary
Improvements in outcomes for patients with LCH and advances in our understanding of LCH biology have been limited by the rarity of the disease and the lack of coordinated efforts through cooperative groups or consortia. True advances have not occurred until multi-institutional studies have been developed. Studies conducted by the Histiocyte Society have defined the prognostic factors, the risk stratification principles, andthe best chemotherapeutic regimens. However, many challenges remain, including improving survival for patients with organ dysfunction, decreasing reactivation rates, and preventing and treating LCH neurodegeneration. The LCH-IV study has been designed to provide a framework in which all these questions can be addressed. Seven separate strata have been included, each one functioning as individual protocols to which patients are eligible during the time course of their disease. The LCH data center in Vienna will assume the centralized data management and statistical support and will also play the role of coordinating center for European institutions. St. Jude Children’s Research Hospital (St. Jude) will serve as the coordinating center for LCH-IV in North America. The Operations Center of the North American Consortium for Histiocytosis (NACHO), located at St. Jude, will be responsible for facilitating and overseeing the LCH-IV to ensure that all institutions participating in North America comply with applicable regulations and that data is collected and managed properly. The resources requested in this proposal will be used for per-case reimbursement to North American institutions enrolling patients on Stratum I of the LCH-IV ($1,000 per patient).

Twelve Month Report
Improvements in outcomes for patients with LCH and advances in our understanding of LCH biology have been limited by the rarity of the disease and the lack of coordinated efforts through cooperative groups or consortia. True advances have not occurred until multi-institutional studies have been developed. Studies conducted by the Histiocyte Society have defined the prognostic factors, the risk stratification principles, and the best chemotherapeutic regimens. However, many challenges remain, including improving survival for patients with organ dysfunction, decreasing reactivation rates, and preventing and treating LCH neurodegeneration. The LCH-IV study has been designed to provide a framework in which all these questions can be addressed. Seven separate strata have been included, each one functioning as individual protocols to which patients are eligible during the time course of their disease. The LCH data center in Vienna serves as the centralized data management and statistical support and will also play the role of coordinating center for European institutions. St. Jude Children’s Research Hospital (St. Jude) serves as the coordinating center for LCH-IV in North America. The Operations Center of the North American Consortium for Histiocytosis (NACHO), located at St. Jude, is responsible for facilitating and overseeing the LCH-IV to ensure that all institutions participating in North America comply with applicable regulations and that data is collected and managed properly. The support of this grant is being used for per-case reimbursement to North American institutions enrolling patients on Stratum I of the LCH-IV ($1,000 per patient). In the past year, the first site has received full approval of the study and has enrolled the first patient on the study. Nine additional sites across the U.S. are in the process of getting the study approved at their local institutions to allow them to begin enrolling patients on the study.