Teriflunomide plus high-dose dexamethasone versus high-dose dexamethasone alone for adults with newly diagnosed ITP

Teriflunomide and High-dose Dexamethasone vs High-dose Dexamethasone Alone as First-line Treatment for Newly Diagnosed Adult Primary Immune Thrombocytopenia: A Prospective, Multicenter, Randomized Trial

This trial tests whether adding teriflunomide to high-dose dexamethasone helps adults with newly diagnosed immune thrombocytopenia raise platelet counts and control bleeding better than dexamethasone alone.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 132 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Peking University People's Hospital (Academic / other)
• Locations: 3 sites (Beijing, Beijing Municipality and 2 other locations)
• Trial ID: NCT07065968 on ClinicalTrials.gov

What this trial studies

This is a multicenter, open-label, randomized Phase 2 trial conducted in Beijing that enrolled 132 adults with newly diagnosed primary immune thrombocytopenia. Participants were randomized to receive either teriflunomide combined with high-dose dexamethasone or high-dose dexamethasone alone as first-line therapy. Platelet counts, bleeding symptoms, and other clinical measures were recorded before and after treatment, and adverse events were monitored throughout follow-up. The trial compares efficacy and safety outcomes between the combination and monotherapy groups.

Who should consider this trial

Good fit: Adults aged 18 or older with newly diagnosed, treatment-naïve primary ITP and a platelet count <30 x10^9/L or <50 x10^9/L with bleeding who can give informed consent are the intended participants.

Not a fit: Patients with secondary causes of thrombocytopenia, significant liver disease, severe organ insufficiency, pregnancy or lactation, active infection or malignancy, other autoimmune diseases, or prior ITP treatment are unlikely to qualify and may not benefit from this protocol.

Why it matters

Potential benefit: If successful, adding teriflunomide could improve platelet response rates and reduce bleeding compared with dexamethasone alone as first-line therapy.

How similar studies have performed: Other immunomodulatory and immunosuppressive approaches have shown benefit in ITP, but the use of teriflunomide in combination with high-dose dexamethasone for newly diagnosed ITP is relatively novel and not yet well established.

Eligibility criteria

Inclusion Criteria:

1. Patients aged ≥18 years;
2. Newly diagnosed, treatment naïve ITP patients;
3. Patients with a platelet count \<30 x10\^9/L or a platelet count \<50 x10\^9/L with bleeding manifestations at the enrollment;
4. Willing and able to sign written informed consent.

Exclusion Criteria:

1. Secondary ITP such as drug-related thrombocytopenia, viral infection (HIV, hepatitis B virus, or hepatitis C virus);
2. Pre-existing acute or chronic liver disease, or ALT/AST greater than 2 times the upper limit of normal (ULN);
3. Severe cardiac, renal, hepatic, or respiratory insufficiency;
4. Severe immunodeficiency;
5. Pregnancy or lactation;
6. Active or a history of malignancy;
7. Active infection requiring systemic therapy;
8. Myelodysplastic syndrome, aplastic anemia, or myelofibrosis;
9. A known diagnosis of other autoimmune diseases;
10. Patients who are deemed unsuitable for the study by the investigator.

Where this trial is running

Beijing, Beijing Municipality and 2 other locations

• Peking University First Hospital — Beijing, Beijing Municipality, China (Recruiting)
• Peking University Insititute of Hematology, Peking University People's Hospital — Beijing, Beijing Municipality, China (Recruiting)
• Peking University Third Hospital — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Principal investigator: Xiao-Hui Zhang, MD — Peking University Institute of Hematology, Peking University People's Hospital
• Study coordinator: Xiao-Hui Zhang, MD

Conditions Immune Thrombocytopenia