Sirolimus in the Treatment of Refractory/Relapsed wAIHA
ConditionWarm Autoimmune Hemolytic Anemia
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age18-90
SponsorPeking Union Medical College Hospital
About this trial
Autoimmune hemolytic anemia (AIHA) is a rare and heterogeneous disorder characterized by the destruction of red blood cells through warm or cold antibodies. Glucocorticoid (combined with rituximab) is the first-line treatment. However, the recurrence rate is very high and some patients may not respond to steroids. Second-line therapies include cyclosporine A (CsA), cyclophosphamide, rituximab, azathioprine, and even splenectomy. Our previous study of sirolimus in refractory/relapsed AIHA and ES found an effective rate of 80%. Therefore, the investigators plan to explore the efficacy and safety of sirolimus in the treatment of refractory/relapsed wAIHA.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age ≥18 years old.
Diagnosed as primary warm autoimmune hemolytic anemia or Evans syndrome (primary or secondary). There is no treatment indication of other systemic involvement in the original disease if secondary.
No response to glucocorticoid therapy or recurrence.
Baseline liver (ALT, AST) was less than 2 times the normal value.
Disqualifiers
Patients with connective tissue disease or other organs involvement
Infection or bleeding that cannot be controlled by standard treatment.
Active HIV, HCV or HBV infection or cirrhosis or portal hypertension.
Progressed uncontrolled malignant tumors and lymphoma
Trial design
Design model
Single group
Treatments tested in this trial
Sirolimus
DrugOral administration, 1-3 mg/d, sirolimus plasma concentration: 4-15 ng/mL
Treatment groups
22 Participants
are divided into 1 treatment groupGroup A: Sirolimus on refractory/relapsed wAIHAExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Overall response rate (ORR) and complete response rate (CRR)
ORR defined as the proportion of patients who met the criteria of either complete response (CR) or partial response (PR).
Time frame
3,6,12 monthsSecondary outcomes
1
Number of participants with treatment-related adverse events and the number of relapses
Number of participants with treatment-related adverse events and the number of relapses
Time frame
12 months, end of the follow-up2
Adverse events
Safety analyses include assessments of the incidence and severity of adverse events; all adverse events that occurred or worsened during the treatment period will be reported, as well as adverse events that occurred later but are considered by the investigator to be related to the trial drug.
Time frame
3,6,12 months3
Relapse rate
Relapse rate defined as the proportion of patients whose response shift from PR or CR to no response (NR).
Time frame
3,6,12 monthsSponsors and contacts
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Source ClinicalTrials.gov