Literatür Detay Bilgisi
Bortezomib for refractory autoimmunity in pediatrics;

Yazarlar : Khandelwal P, Davies S et al.;

Yayın : Biol Blood Marrow Transplant

Yayın Yılı : 2014

Pubmed Linki :

Konu : Diğer

Literatür İçeriği :  Therapy of refractory autoimmunity remains challenging. In this study, we evaluated the therapeutic effect of bortezomib, a proteasome inhibitor, by targeting plasma cells in seven patients, median age 9.9 years. Four doses of bortezomib were administered at a dose of 1 intravenously (n=6) or subcutaneously (n=1) every seventy-two hours. Bortezomib was administered at a median of 120 days from laboratory confirmation of autoantibodies. All patients had failed 2 or more standard therapies. Rituximab was administered on the first day if B cells were present, and all patients received plasmapheresis 2 hours prior to bortezomib administration. Six patients experienced resolution of cytopenias. Two of six patients experienced recurrence of cytopenias after initial response. Adverse effects include nausea (n=1), thrombocytopenia (n=2), Clostridium difficile colitis (n=1)), febrile neutropenia (n=1) and cellulitis at the subcutaneous injection site (n=1). Our experience suggests that bortezomib may be beneficial in the treatment of refractory autoimmunity in children.

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