Bortezomib Plus Dexamethasone for Acquired Pure Red Cell Aplasia Failure or Relapse After First-line Treatment

Study Overview
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Brief Summary
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This is an open-label, single-arm study to evaluate the safety and efficacy of bortezomib plus dexamethasone for acquired pure red cell aplasia failure or relapse after first-line treatment.

Official Title
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Safety and Efficacy of Bortezomib Plus Dexamethasone for Acquired Pure Red Cell Aplasia Failure or Relapse After First-line Treatment: A Prospective Phase II Trial

Conditions
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Acquired Pure Red Cell Aplasia

Intervention / Treatment
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* Drug: bortezomib/dexamethasone


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: ECOG ≤ 2. Age from 18 to 70. Diagnosed with acquired pure red cell aplasia. Meets the criteria of first-line treatment failure or relapse. Organs in good function. Signed informed consent. Exclusion Criteria: Nursing woman. Active bacterial, virus, fungal or parasitic infection, including HIV infection, HBsAg or HBV DNA positive, HCV DNA positive, etc. Secondary PRCA caused by lymphoproliferative disorders, including large granular lymphocytic leukemia, waldenström macroglobulinemia, small lymphocytic lymphoma/chronic lymphocytic leukemia, etc. Secondary PRCA caused by either smoldering multiple myeloma or symptomatic multiple myeloma. Secondary PRCA caused by either ABO major mismatched stem cell transplantation or organ transplantation. Secondary PRCA caused by solid tumors except for thymoma. Secondary PRCA caused by drugs or pregnancy. Secondary PRCA caused by the B19 virus. Have contraindications for glucocorticoids, or unable to tolerate glucocorticoids for comorbidities. Previously received treatment in other trials within 4 weeks before enrollment. Previously treated with the proteasome inhibitor. Experience active hemorrhage condition, including gastrointestinal bleeding, respiratory tract bleeding and central nervous system bleeding within 2 months before enrollment or during bortezomib/dexamethasone treatment. Have a history of malignant tumors. Have a history of mental illness. Inability to understand or to follow study procedures.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: bortezomib/dexamethasone<br>Enrolled patients will receive the combination therapy of bortezomib and dexamethasone. | Drug: bortezomib/dexamethasone<br>* Enrolled patients would receive the combination therapy of bortezomib and dexamethasone.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall response rate |  | within 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency and severity of adverse events and severe adverse events |  | within 12 weeks | 
| Relapse free survival |  | within 24 and 48 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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PRCA, Acquired Pure Red Cell Aplasia, Failure or Relapse after First-line Treatment