Sirolimus Treatment for Refractory PRCA

• Status: Completed
• Trial end date: 2021-05-01
• Target enrollment: 0
• Participant gender: All

Summary

Pure red cell aplasia (PRCA) is a kind of anemia characterized by severe reticulocytopenia and obvious bone marrow erythroblastic cells decreased. Cyclosporine and /or steroids are the first line therapy but some patients were refractory or intolerance to the treatment. The effects of the second line therapy are also not satisfactory and sometimes not available. The investigators aim to explore the efficacy and side-effect of sirolimus for refractory PRCA.

• Phase: Phase 4
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Bing Han
• Treatments: Sirolimus

Criteria

Inclusion Criteria:

1. Refractory pure red cell aplasia.

2. Excluding other diseases which might cause hematological abnormalities.

3. No response or intolerant to first and second line therapies.

4. 18-80 years old.

5. All subjects must: agree not to donate blood or be counseled about pregnancy precautions and risks of fetal exposure.

6. Written informed consent.

Exclusion Criteria:

1. NOT refractory pure red cell anemia.

2. Response and well tolerate to first or second line therapy.

3. Patients who are under 18-year-old or over 80-year-old.

4. Pregnant or lactating.

5. Patients unwilling to or unable to comply with the protocol.