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This is a prospective, randomized, controlled trial. ITP patients who failed prior full-does TPO-RA monotheray for 14 days. Patients are randomly assigned at a 1:1 ratio to receive baricitinib plus TPO-RA or TPO-RA alone. Patients are randomly assigned at a 1:1 ratio to receive baricitinib plus TPO-RA or TPO-RA alone. The primary endpoint was the 14-day overall response rate without any rescue therapy.
This is a prospective, randomized, controlled trial.Eligible patients were at least 18 years old, had a diagnosis of primary ITP and did not respond after receiving TPO-RA (hetrombopag or eltrombopag) at the full dose (hetrombopag 7.5mg per day or eltrombopag 75 mg per day) for 14 days (platelet count below 30×10^9/L or a value less than a 2-fold increase from their baseline platelet count). Patients are randomly assigned at a 1:1 ratio to receive baricitinib plus TPO-RA or TPO-RA alone. Both groups will continue their prior full-dose TPO-RA therapy for 14 days (day 1-14), while baricitinib was given orally at a dose of 2 mg twice daily concomitantly in the combination group for 14 days (day 1-14). The primary endpoint was the 14-day overall response rate without bleeding and any rescue therapy. The secondary endpoints included the 28-day overall response rate, 14-day complete response rate, the 28-day complete response rate, time to response, WHO bleeding scores, health-related quality of life, and adverse events.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Combined therapy | Experimental | Oral baricitinib is given at a dose of 2 mg twice daily for 14 days (day 1-14); prior full-dose TPO-RA therapy (hetrombopag 7.5mg once daily or eltrombopag 75mg once daily) was continued for 14 days (day 1-14). |
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| Monotherapy | Active Comparator | Prior full-dose TPO-RA (hetrombopag 7.5mg once daily or eltrombopag 75mg once daily) was continued. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Baricitinib | Drug | Oral baricitinib is given at a dose of 2 mg twice daily for 14 days. |
| |
| Measure | Description | Time Frame |
|---|---|---|
| 14-day Overall response rate | Overall response was defined as platelet count over 30,000/μL and at least a 2-fold increase of the baseline count in the absence of bleeding and rescue therapy. | From enrollment to the end of treatment at 14 days |
| Measure | Description | Time Frame |
|---|---|---|
| 14-day Complete response (CR) rate | Complete response (CR) was defined as platelet count over 100,000/μL and absence of bleeding. | From enrollment to the end of treatment at 14 days |
| 28-day ovrall response rate |
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Inclusion Criteria:
Exclusion Criteria:
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| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| C000596027 | baricitinib |
| C000614661 | hetrombopag |
| C520809 | eltrombopag |
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| TPO-RA |
| Drug |
Hetrombopag is given at an initial dose of 7.5 mg once daily for 14 days; eltrombopag is given at an initial dose of 75 mg once daily for 14 days |
|
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Overall response was defined as platelet count over 30,000/μL and at least a 2-fold increase of the baseline count and absence of bleeding.
| From enrollment to the end of treatment at 28 days |
| 28-day CR rate | Complete response (CR) was defined as platelet count over 100,000/μL and absence of bleeding. | From enrollment to the end of treatment at 28 days |
| Time to response (TTR) | The time from treatment initiation to achieve a CR or a R. | From the start of study treatment (Day 1) up to day 14 |
| Bleeding events | Bleeding was assessed with the WHO bleeding scale (grade 0, no bleeding; grade 1, petechiae; grade 2, mild blood loss; grade 3, gross blood loss; grade 4, debilitating blood loss). | From the start of study treatment (Day 1) to the end of day 14 |
| Health-related quality of life (HRQoL) | ITP-patient assessment questionnaire was used to assess the HRQoL before and after treatment. | From the start of study treatment (Day 1) to the end of day 14 |
| AE | Adverse events | From enrollment to the end of treatment at 14 days |
| D006425 |
| Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |