Clinical Trial: Use of Bortezomib to Treat Refractory Autoimmune Cytopenia(s) in Allogeneic Stem Cell Transplantation

Study Status: Not yet recruiting
Recruit Status: Unknown status
Study Type: Interventional




Official Title: Use of Bortezomib to Treat Refractory Autoimmune Cytopenia(s) in Allogeneic Stem Cell Transplantation

Brief Summary:

Autoimmune cytopenias are a significant complication observed after hematopoietic cell transplant (HCT) and carries a substantial rate of morbidity and mortality. This entity is usually challenging to treat, resulting in prolonged hospitalization and predisposes patients to complications either directly due to the specific cytopenia or due to the immune suppression that is part of its therapeutic management. One of the mediators of refractory autoimmune cytopenias are antibodies produced by plasma cells, which are historically challenging to target. The purpose of this study is to use a proteasome inhibitor called Bortezomib for the treatment of refractory autoimmune cytopenias after HCT, and monitor the clinical response.

This is a pilot study which will enroll 5 patients over 2 years, prospectively. Our hypothesis is that bortezomib will effectively target plasma cells and successfully treat refractory autoimmune cytopenias. This will be a relevant study as it will be the first step in establishing a novel therapeutic modality and potentially reducing length of hospitalization and degree of immune suppression for refractory autoimmune cytopenias.