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TPO-RAs
TPO-RAs (thrombopoietin receptor agonists) stimulate the thrombopoietin receptor to increase platelet production in the bone marrow.
TPO-RAs (thrombopoietin receptor agonists) stimulate the thrombopoietin receptor to increase platelet production in the bone marrow. Used for Immune thrombocytopenia (ITP), Thrombocytopenia associated with chronic liver disease.
At a glance
| Generic name | TPO-RAs |
|---|---|
| Also known as | TPO-R agonists |
| Sponsor | Institute of Hematology & Blood Diseases Hospital, China |
| Drug class | Thrombopoietin receptor agonist |
| Target | TPO receptor (MPLS) |
| Modality | Small molecule |
| Therapeutic area | Hematology |
| Phase | FDA-approved |
Mechanism of action
TPO-RAs bind to and activate the thrombopoietin (TPO) receptor on megakaryocyte progenitor cells, promoting their proliferation and differentiation into mature megakaryocytes that produce platelets. This mechanism is used to treat thrombocytopenia by boosting endogenous platelet generation without requiring platelet transfusions.
Approved indications
- Immune thrombocytopenia (ITP)
- Thrombocytopenia associated with chronic liver disease
Common side effects
- Headache
- Fatigue
- Diarrhea
- Thromboembolic events
- Bone marrow fibrosis (with long-term use)
Key clinical trials
- A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP) (PHASE2)
- Efficacy and Safety of Romiplostim N01 Combined With Rituximab in Patients With Oral TPO-RA-Refractory Primary Immune Thrombocytopenia (NA)
- The Use of Eltrombopag Post HSCT in BMFD (PHASE2, PHASE3)
- Rate of Prolonged Response After Stopping Thrombopoietin-receptor Agonists Treatment in ITP (PHASE4)
- Response Rate and Duration After Discontinuation Thrombopoietin Receptor Agonists Primary Immune Thrombocytopenia
- Validation of the EQ-5D-Y-3L and EQ-5D-Y-5L for Paediatric Patients in China
- TPO-RA in Primary Immune Thrombocytopenia (ITP) in Patients Older Than 14 Years (NA)
- TPO-RAs Combining Anti-CD 20 Monoclonal Antibody Versus TPO-RAs in the Management of Pediatric Primary Immune Thrombocytopenia (ITP) (PHASE4)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- TPO-RAs CI brief — competitive landscape report
- TPO-RAs updates RSS · CI watch RSS
- Institute of Hematology & Blood Diseases Hospital, China portfolio CI