Bortezomib to Treat Significant Complication of HSCT

Completed · Phase 2

Conditions studied: Allogeneic Stem Cell Transplantation, Refractory Autoimmune Cytopenia(s)

In brief

The purpose of this trial is to study the safety and effectiveness of a drug called Bortezomib for the treatment of low blood cell counts after bone marrow transplant.

Key facts

Study ID

NCT01929980

Run by

Children's Hospital Medical Center, Cincinnati

People needed

4

Starts

2012-07-01

Expected to finish

2014-05-01

Last updated by the study team

2016-10-24

Who can join

Age: 0 and older, up to 29. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• All patients, having undergone allogeneic stem cell transplantation at our center.
• Should have failed at least 2 standard treatments for autoimmune cytopenias. Standard treatments include corticosteroids, rituximab, IVIG, plasmapheresis, withdrawal of cyclosporine, cyclophosphamide and MMF. Definition of "failed" treatment will be no response of cytopenia after 2 weeks of continued treatment OR requirement of daily GCSF at 10 mcgs/kg/day for autoimmune neutropenia despite 2 weeks of treatment, transfusions of packed red blood cells or platelets 3 times weekly for 2weeks despite continued treatment OR 5days/week plasmapheresis for 2 weeks and inability to wean the duration.
• Definition of autoimmune hemolytic anemia- development of anemia, where there is a hemoglobin drop of >2 g/dL/48 hours or an absolute value of hemoglobin < 8 g/dL, and evidence of hemolysis by positive direct Coombs test with compatible peripheral blood cell morphology, reticulocyte count and bilirubin level.
• Definition of autoimmune neutropenia - absolute neutrophil counts < 500 for 2 weeks and presence of anti-neutrophil antibodies.
• Definition of autoimmune thrombocytopenia- Platelet counts < 20,000 cells/uL for 2 weeks and presence of anti-platelet antibodies.

You may not qualify if…

• Ongoing life threatening infections
• Documented anaphylaxis to bortezomib
• Failed engraftment
• Relapse of primary malignancy
• ≥6/8 matched or haploidentical transplants

Where it is running

• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States

Full record on ClinicalTrials.gov

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