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.
The purpose of this research study is to study the safety and effectiveness of a drug called bortezomib for the treatment of autoimmune cytopenia(s) (low blood cell counts) after bone marrow transplant that are not responding to standard treatments. Autoimmune cytopenias are low blood counts due to antibodies or proteins produced against an individual's own blood cells. Having a low red blood cell count (anemia) can make a person feel tired and require blood transfusions frequently. A low platelet count (blood cells that help blood to clot) can make a person bleed or bruise easily. A low neutrophil (white blood cell) count can make a person have infections. All of these things can be a serious complication after bone marrow transplant and can cause prolonged hospital stay. Bortezomib is being used in children with certain types of blood cancer, however, bortezomib has not been used in children with autoimmune cytopenia(s) and its use in this study is investigational.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
4
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Number of Participants With Response
For Autoimmune Hemolytic Anemia- At least 3 of 5 criteria should be met. 1. Stabilization of hemoglobin without transfusions by 2 weeks 2. Conversion of DAT from + to - by 6 weeks 3. Normalization of serum haptoglobin levels by 6 weeks 4. Normalization of indirect bilirubin levels by 6 weeks 5. Reduction in the frequency of transfusions by 50% by 4 weeks For Autoimmune Neutropenia- At least 2 of 3 criteria should be met. 1. Stabilization of absolute neutrophil count by 2 weeks 2. Undetectable antineutrophil antibodies by 6 weeks 3. Reduction in GCSF dose by 50% by 6 weeks For Autoimmune Thrombocytopenia- At least 2 of 3 criteria should be met. 1. Stabilization of platelet count without platelet transfusions by 2 weeks 2. Undetectable antiplatelet antibodies by 6 weeks 3. Reduction in the frequency of platelet transfusions by 50% from pre-bortezomib values by 6 weeks
Time frame: 6 weeks
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