Viktoria Blumenberg, Filippo Birocchi, Angela Shih, Giulia Escobar, Adele Mucci, Alexander Armstrong, Diana D. Cirstea, Deshea L Harris, David J. Bozym, Benjamin R. Puliafito, Richard A. Newcomb, Tejaswini M. Dhawale, Patrick C. Johnson, Areej El-Jawahri, Richard Jeffrey, Alexis Barselau, Alex Li, Estelle Emmanuel-Alejandro, Daniella Cook, Kevin A Lindell, Samantha O. Luk, Ryan Chaffee, Andrew J. Yee, Andrew R. Branagan, Noopur S. Raje, Charlotte Graham, Sarah P. Hammond, Frederic D. Bushman, Aoife M. Roche, Shantan Reddy, Alexander G. McFarland, Yi-Bin Chen, Bryan D. Choi, Christopher W. Mount, Michael Dougan, Valentina Nardi, Aliyah Sohani, Kathleen M.E. Gallagher, Marcela V. Maus, Matthew J. Frigault, Mark B. Leick
ABSTRACT: Intractable diarrhea is a recently described complication following B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor (CAR) T-cell therapy for multiple myeloma with reported mortality rates of 36% to 50%. The optimal clinical management is unknown. Here, we report a series of 5 patients who presented with severe diarrhea after BCMA CAR T-cell treatment. We hypothesized that the Janus kinase inhibitor, ruxolitinib, might be an effective therapy based on its success in graft-versus-host-disease after allogeneic bone marrow transplant and other immune-driven diarrhea syndromes. Three patients received ruxolitinib, all of whom experienced rapid clinical improvement. Among the 2 patients with matched pretreatment and posttreatment biopsies, both showed signs of histopathologic response, including 1 with CAR T-cell-associated indolent T-cell lymphoproliferative disease of the gastrointestinal tract.