eligibility_summary
Eligible: 6–17 y, ITP platelets <20×10^9/L (6–11 chronic, 12–17 persistent/chronic), prior therapy failure/relapse, concomitant meds stable (steroids ≥14 d, immunosuppressants ≥3 mo, stop TPO/TPO‑RA ≥1 mo), no infection/fever ×1 mo, PT/aPTT ≤20% above normal, WBC/ANC/Hb normal (allow steroid leukocytosis, bleeding‑related anemia), age‑appropriate consent. Exclude: thrombosis/risk, failed all TPO‑RAs, anti‑CD20 <3 mo, antiplatelet/anticoagulant >3 d in last 2 wks, other trial <3 mo, severe organ/psych disease, HIV, HBV/HCV/active hepatitis, ALT/AST/ALP >1.5×ULN, Cr/bili >1.2×ULN, albumin <LLN−10%, MF≥2, abnormal platelet aggregation, or investigator decision.
trial_source
clinical_trials.gov from Dec 2, 2025
annotation_status
ai
ai_summary
NCT05718856: Phase 4, randomized, open-label trial in Chinese children with persistent/chronic ITP after steroid failure. Interventions: (1) TPO receptor agonists (eltrombopag, hetrombopag, avatrombopag)—oral small-molecule TPO-RAs, (2) TPO-RAs combined with an anti-CD20 monoclonal antibody (rituximab or ortuzumab). Mechanisms: TPO-RAs activate the thrombopoietin receptor (c-Mpl) on megakaryocyte progenitors, engaging JAK/STAT and MAPK signaling to boost megakaryopoiesis and platelet production. Anti-CD20 mAbs bind CD20 on pre-B and mature B cells, depleting them via ADCC, complement, and apoptosis, thereby lowering antiplatelet autoantibody production and immune-mediated platelet clearance. Targets: c-Mpl on megakaryocyte lineage (to increase platelets) and CD20+ B lymphocytes (to reduce autoimmunity). Monotherapy nonresponders may receive rescue anti-CD20 at week 12.