This randomized, open-label study aim to compare the efficacy and safety of Daratumumab (anti-CD38 monoclonal antibody) with Rituximab in pediatric ITP patients.This study will be conducted in pediatric ITP patients who had not responded to or had relapsed after previous glucocorticoid treatment.
Age ≥6，<18 years, male or female.
Before enrollment, the subjects have been clinically diagnosed with primary immune thrombocytopenia for no less than three months according to the American Society of Hematology guidelines 2011 Evidence-Based Practice Guideline (Neunert et al. 2011) or the International Consensus Report for the Investigation and Management of Primary Immune Thrombocytopenia (Provan et al. 2010), as applicable locally.
Patients have failed glucocorticoid therapy (either due to inefficacy, efficacy could not be maintained, or relapse). Patients were required to have a response history (PLT≥50×10^9/L) to standard first-line treatment of ITP (glucocorticoid and/or intravenous immunoglobulin).
Subjects with a platelet count of <30×10^9/L within the 24 hours prior to the first dose of the study drug; The mean platelet count of at least two separate assessments (at least 1 week apart) <30×10^9/L during the screening visit, and no platelet count > 35×10^9/L.
Subjects with a history of using CD20 monoclonal antibody or CD38 monoclonal antibody.
Subjects who are diagnosed with autoimmune hemolytic anemia or various secondary thrombocytopenic disorders.
Subjects with history of any thrombotic or embolic events or extensive and severe bleeding, such as hemoptysis, major upper gastrointestinal bleeding, intracranial hemorrhage, or the presence of sepsis or other irregular bleeding within the 12 months preceding the initiation of the first dose of study drug.
Subjects who have participated in any other investigational drug studies (including vaccine studies) or been exposed to other investigational drugs within the first 4 weeks or 5 half-lives (whichever was longer) prior to the first dose of study drug.
Parallel
Rituximab
DrugAll subjects were randomly assigned to group A (active comparator) and group B (experimental). For subjects in group A (active comparator) , rituximab (375mg/m2) was given once.
Daratumumab
DrugAll subjects were randomly assigned to group A (active comparator) and group B (experimental). For subjects in group B (experimental), Daratumumab (anti-CD38 monoclonal antibody ) (16mg/kg) was given once a week for eight times.
Overall response rate at week 12
Overall response rate was defined as either partial response or complete response at week 12. Partial response was characterised by at least two consecutive (≥7 days apart) platelet counts of ≥30 to \<100×10\^9/L, with a minimum doubling from baseline and no bleeding. Complete response was characterised by at least two consecutive (≥7 days apart) platelet counts of ≥100×10\^9/L, with no bleeding.
Complete response rate at week 12
Complete response was characterised by at least two consecutive (≥7 days apart) platelet counts of ≥100×10\^9/L, with no bleeding at week 12.
Partial response rate at week 12
Partial response was characterised by at least two consecutive (≥7 days apart) platelet counts of ≥30 to \<100×10\^9/L, with a minimum doubling from baseline and no bleeding at week 12.
Overall response rate at week24
Overall response rate was defined as either partial response or complete response at week 24. Partial response was characterised by at least two consecutive (≥7 days apart) platelet counts of ≥30 to \<100×10\^9/L, with a minimum doubling from baseline and no bleeding. Complete response was characterised by at least two consecutive (≥7 days apart) platelet counts of ≥100×10\^9/L, with no bleeding.
Overall response rate at week52
Overall response rate was defined as either partial response or complete response at week 52. Partial response was characterised by at least two consecutive (≥7 days apart) platelet counts of ≥30 to \<100×10\^9/L, with a minimum doubling from baseline and no bleeding. Complete response was characterised by at least two consecutive (≥7 days apart) platelet counts of ≥100×10\^9/L, with no bleeding.
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