Immune thrombocytopenia
Conditions
Interventions
Rituximab group (RTX positive control cohort):None
Daratumumab group (DARA observational cohort):None
Sponsors
Beijing Children's Hospital, Capital Medical University
Eligibility
Sex/Gender
All
Age
1 Years to 17 Years
Inclusion criteria
Inclusion criteria: 1. Aged 1 to 17 years, regardless of gender; 2. Clinically diagnosed with primary immune thrombocytopenia (referring to the "Adapted Guideline for the Diagnosis and Treatment of Primary Immune Thrombocytopenia for Chinese Children (2021 Edition)"), regardless of disease duration; 3. Previous failure (no response, inability to maintain efficacy, or relapse) after first-line standard therapy for ITP (glucocorticoids and/or intravenous immunoglobulin) as specified by guidelines; 4. Platelet count <10×10^9/L in two consecutive tests at least 24 hours apart during screening period, with bleeding symptoms requiring treatment; 5. Guardians understand the study procedures and sign informed consent form, and children aged 8 years and above sign the pediatric version of informed consent form simultaneously.
Exclusion criteria
Exclusion criteria: 1. Various secondary ITP, including but not limited to systemic lupus erythematosus, antiphospholipid syndrome, immunodeficiency diseases, chronic active infections, lymphoproliferative diseases, or drug-related thrombocytopenia; 2. Previous treatment with anti-CD38 monoclonal antibody, anti-CD20 monoclonal antibody, or other biological targeted agents; 3. Uncontrolled active viral or bacterial infections, including hepatitis B, hepatitis C, cytomegalovirus, Epstein-Barr virus, syphilis, HIV positive, etc.; 4. Significant hypogammaglobulinemia, known primary immunodeficiency, or requiring long-term immunoglobulin replacement therapy; 5. Uncontrolled primary diseases of vital organs, such as malignant tumors, liver failure, heart failure, renal failure, etc.; 6. Live vaccination within 4 weeks before screening; 7. Pregnancy in adolescent females or positive pregnancy test; 8. Mental disorders that prevent proper informed consent and study participation and follow-up; 9. Toxic symptoms from pre-study treatment have not resolved; 10. Other serious or other circumstances that the researcher deems unsuitable for participation in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Early and Sustained Overall Response Rate (ES-ORR); | — |
Secondary
| Measure | Time frame |
|---|---|
| Time to Response (TTR);Overall Response Rate (ORR) and Complete Response Rate (CRR) at Week 1, Week 4, and Week 8;Sustained Response Rate (SRR) at Week 12 and Week 24;Relapse Rate;Change in Bleeding Score from Baseline;Rescue Treatment Rate (RTR);Hospitalization Duration and Readmission Rate; | — |
Countries
China
Contacts
Public ContactMa Jingyao
Beijing Children's Hospital, Capital Medical University
Outcome results
None listed