Trial Outcomes & Findings for Bortezomib to Treat Significant Complication of HSCT (NCT NCT01929980)

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

6 weeks

Results posted on

2016-10-24

Participant Flow

Participant milestones

Participant milestones
Measure	Bortezomib Four doses of bortezomib, 1.3mg/m2, will be given intravenously (through a needle in a vein) or subcutaneously (under the skin) on Days 1, 4, 8, 11. The format of receiving medications is- Therapy Dose and Route Frequency Rituximab 375 mg/m2 intravenously Once on day 1. Plasmapheresis 2 hours prior to Bortezomib Day 1,4, 8 and 11 Bortezomib 1.3 mg/m2 intravenously Day 1,4,8 and 11 Bortezomib
Overall Study STARTED	4
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Bortezomib Four doses of bortezomib, 1.3mg/m2, will be given intravenously (through a needle in a vein) or subcutaneously (under the skin) on Days 1, 4, 8, 11. The format of receiving medications is- Therapy Dose and Route Frequency Rituximab 375 mg/m2 intravenously Once on day 1. Plasmapheresis 2 hours prior to Bortezomib Day 1,4, 8 and 11 Bortezomib 1.3 mg/m2 intravenously Day 1,4,8 and 11 Bortezomib
Overall Study Physician Decision	1

Baseline Characteristics

Bortezomib to Treat Significant Complication of HSCT

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Bortezomib n=4 Participants Four doses of bortezomib, 1.3mg/m2, will be given intravenously (through a needle in a vein) or subcutaneously (under the skin) on Days 1, 4, 8, 11. The format of receiving medications is- Therapy Dose and Route Frequency Rituximab 375 mg/m2 intravenously Once on day 1. Plasmapheresis 2 hours prior to Bortezomib Day 1,4, 8 and 11 Bortezomib 1.3 mg/m2 intravenously Day 1,4,8 and 11 Bortezomib
Age, Categorical <=18 years	4 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=93 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants
Sex: Female, Male Female	1 Participants n=93 Participants
Sex: Female, Male Male	3 Participants n=93 Participants

PRIMARY outcome

Timeframe: 6 weeks

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

Outcome measures

Outcome measures
Measure	Bortezomib n=4 Participants Four doses of bortezomib, 1.3mg/m2, will be given intravenously (through a needle in a vein) or subcutaneously (under the skin) on Days 1, 4, 8, 11. The format of receiving medications is- Therapy Dose and Route Frequency Rituximab 375 mg/m2 intravenously Once on day 1. Plasmapheresis 2 hours prior to Bortezomib Day 1,4, 8 and 11 Bortezomib 1.3 mg/m2 intravenously Day 1,4,8 and 11 Bortezomib
Number of Participants With Response Neutropenia (n=3)	3 participants
Number of Participants With Response Thrombocytopenia (n=2)	2 participants
Number of Participants With Response Hemolytic Anemia (n=1)	1 participants

Adverse Events

Bortezomib

Serious events: 2 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Bortezomib n=4 participants at risk Four doses of bortezomib, 1.3mg/m2, will be given intravenously (through a needle in a vein) or subcutaneously (under the skin) on Days 1, 4, 8, 11. The format of receiving medications is- Therapy Dose and Route Frequency Rituximab 375 mg/m2 intravenously Once on day 1. Plasmapheresis 2 hours prior to Bortezomib Day 1,4, 8 and 11 Bortezomib 1.3 mg/m2 intravenously Day 1,4,8 and 11 Bortezomib
Gastrointestinal disorders Pneumatosis Coli	25.0% 1/4 • Number of events 1
Blood and lymphatic system disorders Thrombocytopenia	25.0% 1/4 • Number of events 1
Blood and lymphatic system disorders Neutropenia	25.0% 1/4 • Number of events 1
Infections and infestations C diff colitis	25.0% 1/4 • Number of events 1

Other adverse events

Other adverse events
Measure	Bortezomib n=4 participants at risk Four doses of bortezomib, 1.3mg/m2, will be given intravenously (through a needle in a vein) or subcutaneously (under the skin) on Days 1, 4, 8, 11. The format of receiving medications is- Therapy Dose and Route Frequency Rituximab 375 mg/m2 intravenously Once on day 1. Plasmapheresis 2 hours prior to Bortezomib Day 1,4, 8 and 11 Bortezomib 1.3 mg/m2 intravenously Day 1,4,8 and 11 Bortezomib
Gastrointestinal disorders Nausea	25.0% 1/4 • Number of events 1
Skin and subcutaneous tissue disorders Cellulitis	25.0% 1/4 • Number of events 1

Additional Information

Pooja Khandelwal, MD

Cincinnati Children's Hospital Medical Center

Phone: 513-803-9063

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place