Background: Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder. Corticosteroids are first-line therapy, but about one-third of patients relapse or are refractory. Eltrombopag (a TPO-RA) promotes platelet production, yet some patients show no response or relapse upon discontinuation. Telitacicept, a TACI-Fc fusion protein, dual-targets BAFF and APRIL, inhibiting B cell and plasma cell function and reducing autoantibody production, potentially providing synergistic immunomodulatory benefit. Objective: To evaluate the sustained response rate of eltrombopag plus telitacicept vs. eltrombopag alone in patients with steroid-refractory/relapsed ITP. Design: Randomized, open-label, controlled, exploratory Phase II study. 40 patients planned (20 combination, 20 monotherapy). Combination group: eltrombopag + telitacicept . Monotherapy group: eltrombopag alone for 12 weeks. Monotherapy patients with no response after 4 weeks may cross over to the combination group. After 12 weeks, treatment is stopped and patients are followed until Week 24. Primary endpoint: Proportion of patients maintaining platelet count ≥30×10⁹/L with no bleeding at 24 weeks post-treatment. Secondary endpoints include platelet response rates during 12 weeks, safety, bleeding events, etc. Expected results: The combination group is expected to have a significantly higher proportion of patients achieving the primary endpoint without increased adverse events. Population: Age ≥18, diagnosed ITP ≥3 months, baseline platelets \<30×10⁹/L, prior corticosteroid failure. Safety: Monitoring for bleeding, infection, thrombosis, cytopenia, etc., with dose adjustment/cessation as per protocol. Conclusion: This study explores whether dual-targeting (platelet production + autoimmune suppression) with eltrombopag and telitacicept can provide more durable remission for steroid-refractory/relapsed ITP patients.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
40
eltrombopag plus telitacicept vs. eltrombopag alone
Ethics Committee of Blood disease hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, China
Sustained Response Comparison
To compare the sustained response rate of eltrombopag combined with telitacicept versus eltrombopag monotherapy in patients with immune thrombocytopenia who are refractory or relapsed to prior corticosteroid therapy.
Time frame: Within 24weeks of first dose
Platelet Response Within 12 Weeks
Percentage of subjects achieving at least one platelet count ≥30×10⁹/L with an increase of ≥2-fold from baseline within 12 weeks of first dose, without receiving rescue therapy.
Time frame: Within 12 weeks of first dose
Platelet ≥50×10⁹/L at Week 12
Proportion of patients with platelet count ≥50×10⁹/L at Week 12 of treatment.
Time frame: Within 12 weeks of first dose
Platelet ≥100×10⁹/L at Week 12
Proportion of patients with platelet count ≥100×10⁹/L at Week 12 of treatment.
Time frame: Within 12 weeks of first dose
Time to First Platelet Response
Time to first platelet count ≥30×10⁹/L with an increase of ≥2-fold from baseline.
Time frame: Within 12 weeks of first dose
Proportion of Days with Platelet ≥30×10⁹/L
Proportion of days with platelet count ≥30×10⁹/L within the 12-week period.
Time frame: Within 12 weeks of first dose
Platelet Response After Crossover (Monotherapy Group)
Percentage of subjects in the monotherapy group achieving platelet count ≥30×10⁹/L with an increase of ≥2-fold from baseline within 12 weeks after crossover.
Time frame: With in 24 weeks of first dose
Safety and Tolerability
Safety and tolerability endpoints: incidence and severity of adverse events and serious adverse events
Time frame: Within 24 weeks of first dose
Time to Rescue Therapy or Platelet Decline
Time from treatment discontinuation to first need for rescue therapy or platelet count \<30×10⁹/L.
Time frame: Within 24 weeks of first dose
Proportion Requiring Rescue Therapy
Proportion of patients requiring rescue therapy during the follow-up period.
Time frame: Within 24 weeks of first dose
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