Skip Navigation

Adult Histiocyte Disorders Center Collaborations and Research

  • Studies lead to innovation

    Collaborative research and care among specialists with vast expertise in the management of histiocyte diseases have the strong potential to result in better outcomes for patients. Collaborations tend to yield:

    • More effective treatment strategies
    • Better management of side effects
    • Minimization of those side effects

    Working together

    In a field whose research has been fragmented in the past, the Adult Histiocyte Disorders Center has led the effort to unite leading national and international organizations in collaborative research:

    • We are currently partnering with the North American Consortium for Histiocytosis (NACHO), the American Histiocyte Society, and the Erdheim-Chester Disease Global Alliance. Together, we are now very effectively conducting several scientific and clinical studies of these diseases.
    • In association with the University of Toronto, we are collaborating with Sick Kids Hospital to compile a database of rare histiocyte disorders in adults and children. This database will include clinical and pathology information that will serve as a valuable repository for such data going forward.
    • Working with Dana-Farber colleagues, we are researching the use of patient-derived xenografts (PDX) – an exciting technology that is having a significant impact on the development and testing of new therapies for histiocyte disorders. Using this technology, we are creating cell lines that allow us to rapidly screen the effectiveness of therapeutic drugs.

    Clinical trials and stem cell transplant

    As world leaders in clinical trials for adult histiocyte disorders, we have several studies in progress, including:

    • A study of the drug Lenalidomide for adult histiocyte disorders: This research study is investigating the chemotherapy drug Lenalidomide as a possible treatment for one of three histiocyte disorders – Langerhans cell histiocytosis (LCH), Erdheim-Chester disease (ECD), or histiocytic sarcoma (HS).
    • A phase II study of the drug clofarabine in patients with recurrent or refractory Langerhans cell histiocytosis: This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans cell histiocytosis.
    • As world leaders in therapeutic stem cell (bone marrow) transplant, members of our  Stem Cell Transplantation Program are studying the efficacy of transplant in the treatment of certain histiocyte disorders.

    Genetic testing

    At our Center, we are searching for an inherited tendency towards these diseases. Using our advanced gene sequencing platform,  OncoPanel, we conduct, without charge, genetic profiling of the disease for every patient for whom tissue is available.

    We are also collaborating with Baylor University/Texas Children's Hospital in a study of genetic links in histiocyte disorders.


  • Dr. Eric Jacobsen on Rosia-Dorfman disease treatment and research
    In this video, Eric Jacobsen, MD, explains Rosai-Dorfman, a rare form of histiocytosis. He discusses recent research identifying important mutations present in this disease that may be treated with targeted therapy. These findings offer hope to patients with this condition and may lead to further treatment advances.