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FDA Approves New Treatment for Children and Young Adults with Chronic Graft versus Host Disease

This week marked another milestone for the treatment of graft versus host disease in children. The U.S. Food and Drug Administration (FDA) approved ibrutinib (Imbruvica®) for children 1 year or older with chronic graft-vs-host disease (cGVHD) that has not responded to earlier treatment. The drug was approved in 2017 to treat adults with cGVHD. Notably, a new oral formula of ibrutinib helps address challenges children may have with swallowing capsules or tablets.

GvHD can be a life-threatening serious complication of stem cell transplants, which are used to treat certain types of leukemia and lymphoma, as well as multiple myeloma. GvHD occurs when the donor’s cells (the graft) see the patient’s healthy cells (the host) as foreign and attacks them. Click here to learn more about diagnosis, symptoms and LLS support services available for cGVHD.
This approval was based on the iMAGINE trial, which included 47 patients, between the ages of 1 and 22 years who had moderate or severe cGVHD. Six in 10 (60%) of patients responded to ibrutinib treatment by week 25 and the median duration of response was more than 5 months. The most common adverse reactions were anemia, musculoskeletal pain, fever, diarrhea, pneumonia, abdominal pain, mouth inflammation or soreness, low platelet counts and headache.

“Every advance that improves quality of life and health outcomes for patients with blood cancer is an important step in our mission,” says LLS chief scientific officer Lee Greenberger. Nowhere is this more important than in children. LLS recently introduced the Dare to Dream Project, the bigger, bolder, next phase of the LLS Children’s Initiative to develop safer, less toxic, and more effective treatment for kids with blood cancers.

LLS helped advance ibrutinib for treatment of lymphoma, as well as additional therapies for GVHD

Ibrutinib targets the Bruton’s tyrosine kinase (BTK) enzyme, which plays a key role in B-cell cancers. Ibrutinib was originally approved by the FDA starting in 2013 to treat a variety of lymphomas: chronic lymphocytic leukemia (CLL), small lymphocytic leukemia (SLL), mantle cell lymphoma (MCL), marginal zone lymphoma (MZL), and Waldenström macroglobulinemia (WM). Ibrutinib has not only extended survival, but also improved quality of life for thousands of patients, and has transformed the treatment of CLL and WM.

LLS has supported clinical trials and laboratory studies of ibrutinib since 2010. Dr. John Byrd at Ohio State University (OSU) – a long-time LLS grant recipient and world-renowned CLL expert, was instrumental in advancing ibrutinib. LLS is also supporting Dr. Jennifer Woyach, also at OSU and Dr. Nitin Jain of MD Anderson Cancer Center, who are developing next-generation BTK inhibitors that show promise in patients who do not response to ibrutinib therapy. Other current LLS-funded researchers include Dr. Kirk Schultz of the University of British Columbia and Dr. Ivan Maillard of the University of Pennsylvania Medical Center.

Additional therapies have been approved recently to treat GVHD. One of them, ruxolitinib, which was supported by LLS and initially developed to treat a myeloproliferative disorder, was approved in 2019 to treat cGVHD in patients 12 and older. The new applications of ibrutinib and ruxolitinib underscore the value of LLS foundational investments, which have paved the way for new applications of existing therapies.