Autoimmune Hemolytic Anemia (AIHA), Pure Red Blood Cell Aplasia (PRCA)
Conditions
Interventions
treatment group:Orelabrutinib 100 mg/day, administered orally once daily for 3 months. Efficacy will be assessed after 3 months
patients who respond will continue at the same dose for a total of 6 months, after which treatment will be discontinued. During the treatment period, the dose may be adjusted based on hemoglobin respo
Sponsors
West China Hospital of Sichuan University
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Test results from peripheral blood or bone marrow meet the diagnostic criteria for relapsed or refractory autoimmune hemolytic anemia or pure red cell aplasia (Relapsed: a persistent decline in hemoglobin levels following remission from AIHA/PRCA treatment; Refractory: a persistent decline in hemoglobin levels without recovery following second-line treatment for AIHA/PRCA). 2. Aged over 18 years; 3. Participants or their legally authorized representative will voluntarily sign the informed consent form, acknowledging that they understand the nature, purpose, and procedures of this trial.
Exclusion criteria
Exclusion criteria: 1. Myelofibrosis, Grade >= 2; 2. Previously treated with bispecific antibodies or CAR-T therapy; 3. Previously underwent allogeneic hematopoietic stem cell transplantation; 4. Previously received treatment with another BTK inhibitor for more than 4 weeks; 5. History of or current active malignant tumors; localized tumors that have been completely surgically removed for more than 1 year; 6. Clinically significant heart disease (NYHA Class III–IV); unstable angina; myocardial infarction within 6 months prior to enrollment; angioplasty or stent placement for heart disease within 6 months prior to enrollment; clinically significant arrhythmias; or uncontrolled hypertension; 7. A history of significant bleeding within 1 year prior to enrollment; 8. Positive for anti-HIV, HBV surface antigen, and HCV-RNA; 9. Anemia caused by other conditions, such as MDS, acute leukemia, and hereditary anemias like thalassemia and sickle cell anemia, etc. 10. Combined PNH with high disease activity: LDH > 1.5 times the upper limit of normal; 11. There is an uncontrolled active infection; 12. Combating Uncontrolled Diabetes; 13. Eastern Cooperative Oncology Group (ECOG) Performance Status score > 2; 14. Serum aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3 times the upper limit of normal; 15. Serum creatinine >= 2.5 times the upper limit of normal; 16. Women who are pregnant or breastfeeding; 17. Participants whom the research team deems ineligible for enrollment or who may be unable to comply with the trial for other reasons;
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 3-month Overall Response Rate;Adverse Event Frequency; | — |
Secondary
| Measure | Time frame |
|---|---|
| Rate of Transfusion Avoidance;Adverse Event Types;6-month Overall Response Rate;Event-free Survival;Time-to-Response;Overall Survival; | — |
Countries
China
Contacts
Public ContactQiang Zeng
West China Hospital of Sichuan University
Outcome results
None listed