Acquired Pure Red Cell Aplasia
Conditions
Keywords
Acquired Pure Red Cell Aplasia, PRCA, Failure or Relapse after First-line Treatment
Brief summary
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.
Interventions
Enrolled patients would receive the combination therapy of bortezomib and dexamethasone.
Sponsors
Institute of Hematology & Blood Diseases Hospital, China
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Overall response rate | within 12 weeks |
Secondary
| Measure | Time frame |
|---|---|
| Frequency and severity of adverse events and severe adverse events | within 12 weeks |
| Relapse free survival | within 24 and 48 weeks |
Countries
China
Contacts
PRINCIPAL_INVESTIGATORJun Shi, PhD
Institute of Hematology & Blood Diseases Hospital, China
Outcome results
None listed