Phase 2
N=4
Bortezomib to Treat Significant Complication of HSCT
Allogeneic Stem Cell Transplantation · Refractory Autoimmune Cytopenia(s)
Bottom Line
View on ClinicalTrials.gov: NCT01929980 ↗Enrolled (actual)
4
Serious AEs
50.0%
Results posted
Oct 2016
Primary outcome: Primary: Number of Participants With Response — 3; 2; 1 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Bortezomib (Drug)
- Age
- Pediatric, Adult · 0+ yrs
- Sex
- All
- Sponsor
- Children's Hospital Medical Center, Cincinnati
- Primary completion
- May 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Response |
3; 2; 1 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- 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.
Exclusion Criteria
- Ongoing life threatening infections
- Documented anaphylaxis to bortezomib
- Failed engraftment
- Relapse of primary malignancy
- ≥6/8 matched or haploidentical transplants
Data sourced from ClinicalTrials.gov (NCT01929980). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.