A Prospective Study on the Treatment of Recurrent/Refractory/Intolerable NSAA With Lusutrombopag
- Registration Number
- NCT06426043
- Lead Sponsor
- Peking Union Medical College Hospital
- Brief Summary
In a prospective, single-arm study, the efficacy and safety of Lusutrombopag in the treatment of relapsed/refractory/intolerable non-severe aplastic anemia (NSAA) were explored.
- Detailed Description
The enrolled patients: were given Lusutrombopag at 3mg/qd orally for 12 weeks (the starting dose of lusutrombopag was 3mg, taken once daily. After 2 weeks of continuous administration, the dose was increased by 3mg every 2 weeks based on the platelet count and safety of the subjects. The dose was gradually increased to 9mg/d over a total of 12 weeks). The treatment duration was at least 3 months. When the platelet increase was \<20×10\^9/L, the daily dose was increased by 3mg, up to a maximum of 9mg/day. When the platelet increase was ≥50×109/L and ≤200×10\^9/L, the dose was maintained at the previous level. When the platelet count was ≥200×10\^9/L and ≤400×10\^9/L, the daily dose was reduced by 3mg. When the platelet count was \>400×10\^9/L, the drug could be suspended, and the dose was reduced by 3mg when the platelet count decreased to \<200×10\^9/L. In this case, if the lowest dose of 3mg/day was used, the drug could be suspended. Responders continued treatment for 6 months. Other TPO-RA therapies were not allowed during the study period.
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 40
- Participants must be at least 18 years old, male or female.
- Participants must be diagnosed with NSAA and have a refractory/relapsed/intolerable response to standard-dose cyclosporine (CsA). The definition of refractory/relapsed is patients who have been treated with sufficient doses of cyclosporine (3-5mg/kg) for at least 6 months without response or relapse. The definition of intolerable is patients who cannot tolerate CsA and have stopped treatment due to significant side effects.
- Participants must meet the following criteria at enrollment: platelets <30×109/L.
- Baseline liver and kidney function must be within 2 times of normal range.
- No active infection; no pregnancy or breastfeeding.
- Participants must agree to sign the informed consent form.
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2.
- Other causes of pancytopenia, such as myelodysplastic syndrome (MDS).
- Evidence of clonal hematopoietic system bone marrow disease (MDS, AML) with cytogenetics.
- PNH clone ≥50%.
- Received hematopoietic stem cell transplant (HSCT) prior to enrollment.
- Received ATG treatment within 6 months prior to enrollment.
- Infection or bleeding that cannot be controlled with standard therapy.
- Allergic to ruxolitinib.
- Active HIV, HCV, or HBV infection, cirrhosis, or portal hypertension.
- Any malignant tumor within 5 years, or local basal cell carcinoma of the skin.
- History of thromboembolic events, myocardial infarction, or stroke (including antiphospholipid syndrome) and current use of anticoagulants.
- Pregnant or breastfeeding (lactating) women.
- Participated in another clinical trial within 3 months.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Lusutrombopag Lusutrombopag Administer lusutrombopag at 3mg/qd orally for 12 weeks (lusutrombopag starting dose is 3mg, once daily. After 2 weeks of continuous administration, the dose can be increased by 3mg every 2 weeks based on the platelet count and safety of the subject. The dose can be gradually increased to 9mg/d over a total of 12 weeks). The course should be at least 3 months. When the platelet increase is \<20×10\^9/L, the daily dose can be increased by 3mg up to a maximum of 9mg/day; when the platelet increase is ≥50×10\^9/L and ≤200×10\^9/L, the dose can be maintained; when the platelet count is ≥200×10\^9/L and ≤400×10\^9/L, the daily dose can be reduced by 3mg; when the platelet count is \>400×10\^9/L, the drug can be suspended and resumed when the platelet count decreases to \<200×10\^9/L, with the daily dose reduced by 3mg. In this case, if the lowest dose of 3mg/day is used, the drug can be suspended. Responders continue treatment until 6 months.
- Primary Outcome Measures
Name Time Method Overall response rate at 3 months 3 month Proportion of patients who achieved complete response, partial response and hematological response
Overall response rate at 6 months 6 month Proportion of patients who achieved complete response, partial response and hematological response
- Secondary Outcome Measures
Name Time Method adverse event rate at 6 months 6 month Proportion of patients with adverse events
adverse event rate at 3 months 3 month Proportion of patients with adverse eventsProportion of patients with adverse events
Trial Locations
- Locations (1)
Peking Union Medical College Hospital
🇨🇳Beijing, Beijing, China