Adult patients with non-severe aplastic anemia
Conditions
Interventions
Case series:All patients were treated with lutrombopag at a starting dose of 3 mg/day, with the dose adjusted every 2 weeks according to platelet count and safety, with a maximum dose of 12 mg/day and
Sponsors
Tianjin Medical University General Hospital
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Age>=18 years old (including upper and lower limits), male or female; 2. According to the Camitta criteria and the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition), clinical diagnosis of non-severe aplastic anemia (NSAA), including transfusion-dependent (TD-NSAA) and non-transfusion-dependent (NTD-NSAA) who have not been treated or have failed first-line treatment (relapsed or refractory) or have poor efficacy (CR has not been reached): a) Blood picture within 14 days prior to enrollment in at least two of the following three categories: hemoglobin (HGB) = 4 months (indication for component transfusion: HGB=4 component transfusions within 8 weeks prior to enrollment; 3. Eastern Cooperative Oncology Group (ECOG) score of 0-2; 4. PLT<=50×10^9/L during the screening period; 5. Subjects are not suitable or unwilling to undergo hematopoietic stem cell transplantation (HSCT) and plan to receive lutrombopag-based therapy according to the judgment of the investigator or the patient's wishes; 6. Understand the research procedures and voluntarily sign the informed consent form in writing.
Exclusion criteria
Exclusion criteria: 1. Patients with uncontrollable bleeding and/or infection after standardized treatment before enrollment; 2. Evidence of clonal hematologic bone marrow disease (MDS, AML) in the presence of cytogenetics; 3. Neutrophil PNH clone >=50% before enrollment; 4. For treatment-naïve NSAA subjects, the cumulative time of previous use of TPO receptor agonists (such as eltrombopag, hetrombopag, avatrombopag, romiplostim, recombinant human thrombopoietin, etc.) before screening is >5 days, or cumulative time <=5 days and less than 14 days from the washout time before the first dose; 5. For NSAA subjects who have relapsed and refractory after immunosuppressant treatment (such as CsA/TaC±ATG±TPO-RA, etc.), within 6 months from the last ATG treatment and/or within 3 months from the last TPO-RA at the time of screening (for immunosuppressants, if the drug is stable during the screening period and a stable dose is maintained during the planned study period, such as CSA/tacrolimus/etc., the stable dose can be enrolled in the form of concomitant medication with a stable dose, otherwise it should be eluted according to the exclusion criteria in Article 5); 6. HIV infection or hepatitis C antibody positive during the screening period (if hepatitis B surface antigen is positive, or hepatitis B surface antigen is negative but hepatitis B core antibody is positive, HBV-DNA testing is required, if it indicates viral replication, the subject should be excluded); 7. During the screening period, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) are greater than 2.5 times of the upper limit of normal, total bilirubin is greater than 1.5 times of the upper limit of normal, and serum creatinine is more than 1.5 times of the upper limit of normal; 8. History of liver cirrhosis or portal hypertension; 9. Subjects have had solid tumors of any organ system within 5 years prior to screening, regardless of whether they have been treated, metastasized, or recurred, except for local basal cell carcinoma of the skin; Subjects with hematologic tumors previously or found at screening; 10. Congestive heart failure, arrhythmia, peripheral arteriovenous thrombosis requiring drug treatment within 1 year before enrollment, or myocardial infarction or cerebral infarction within 3 months before enrollment; 11. Pregnant or lactating women (lactating female subjects are allowed to be enrolled if they stop breastfeeding before the first dose and stop breastfeeding within 5 days after the completion of treatment), or those who plan to receive/conceive in the past 6 months; 12. The female spouse of the patient or the male patient cannot take effective contraceptive measures; 13. Participated in clinical trials of other drugs within 1 month before enrollment or within 5 half-lives of other study drugs (whichever is longer); 14. Any other circumstance that, in the opinion of the investigator, may cause the subject to be unable to complete the study or bring obvious risks to the subject.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of subjects with a hematology response (at least one line response) at the Week 8 visit; | — |
Secondary
| Measure | Time frame |
|---|---|
| Time to obtain a hematologic response (at least one line of response).;Proportion of subjects who received a two-line response at the Week 8 visit;Proportion of subjects who received a three-line response at the week 8 visit;Median cumulative dose of lusutrombopag at the time of obtaining a hematologic response (at least one lineage).;Overall response rate (ORR) and complete response (CR) at Week 8; | — |
Countries
China
Contacts
Public ContactFu Rong
Tianjin Medical University General Hospital
Outcome results
None listed