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Successful Treatment of an Adolescent Male With Severe Refractory Evans Syndrome Using Bortezomib-based Therapy

Knight, Tristan MD*,†; Ravindranath, Yaddanapudi MB, BS*,†; Callaghan, Michael U. MD*,†

Journal of Pediatric Hematology/Oncology: October 05, 2018 - Volume Publish Ahead of Print - Issue - p
doi: 10.1097/MPH.0000000000001325
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Evans syndrome is defined by bilineal autoimmune cytopenia, typically coombs positive hemolytic anemia and thrombocytopenia. Corticosteroids are the mainstay of treatment, with rituximab and/or mycophenolate mofetil often used in steroid-refractory cases. However, no treatment methodology has ever evaluated by a randomized clinical trial. We present a 15-year-old boy with Evans syndrome and common variable immunodeficiency who experienced a severe, refractory flare 16 months postsplenectomy. After failing to respond to multiple other agents, he achieved a durable response to a bortezomib-based regimen. Bortezomib may be a reasonable second or third line option, especially before high-morbidity therapies such as splenectomy or stem cell transplantation.

*Department of Pediatrics, Division of Pediatric Hematology and Oncology, Children’s Hospital of Michigan

Wayne State University School of Medicine, Detroit, MI

Non-FDA approved use of medications is discussed.

Y.R. receives financial support from the Georgie Ginopolis endowed chair at Wayne State University School of Medicine.

M.U.C.—Sancillio: Site PI; Octapharma: Membership on an entity’s Board of Directors or advisory committees; CSL Behring: Membership on an entity’s Board of Directors or advisory committees; Pfizer Inc.: Membership on an entity’s Board of Directors or advisory committees, Site PI, Research Funding; Roche/Genentech: Membership on an entity’s Board of Directors or advisory committees, Site PI, Speakers Bureau; Global Blood Therapeutics: Site PI; Roche; Shire: Speakers Bureau; Grifols: Membership on an entity’s Board of Directors or advisory committees; Bayer HealthCare; Pfizer Inc.; Roche; Shire: Consultancy; Alnylam Pharmaceuticals Inc.: Owns stock, stock options, or bonds; Bayer: Membership on an entity’s Board of Directors or advisory committees; Baxalta: Membership on an entity’s Board of Directors or advisory committees, Speakers Bureau; Novo Nordisk: Speakers Bureau; Shire: Membership on an entity’s Board of Directors or advisory committees, Speakers Bureau; Biogen: Membership on an entity’s Board of Directors or advisory committees. The remaining authors declare no conflict of interest.

Reprints: Michael U. Callaghan, MD, Children’s Hospital of Michigan, 3901 Beaubien St, Detroit, MI 48201 (e-mail: mcallagh@med.wayne.edu).

Received June 13, 2018

Accepted August 31, 2018

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