Non-severe aplastic anemia
Conditions
Interventions
Immune Deficiency Mutation Group:Discontinue Kanglelong
administer Jili Shengxue Fang and Rapamycin.
MPL-associated mutation group:Discontinue, Administer + high dose
Erythropoiesis is still acceptable / group with iron overload.:Discontinue Prednisolone, Qili Shengxue Formula + Roxadustat.
Sponsors
Zhejiang Provincial Traditional Chinese Medicine Hospital
Eligibility
Sex/Gender
All
Age
3 Years to 88 Years
Inclusion criteria
Inclusion criteria: 1. Meets the diagnostic criteria of NSAA 2. Meets any of the following in terms of treatment: 1)After 3 months of treatment with plus, there was no significant improvement in all three lineages (no sustained, clinically meaningful upward trend relative to baseline); 2) After 6 months of treatment with plus, PR was not achieved; 3) During treatment with plus, disease progression occurred or adverse reactions of grade 3 or higher according to CTCAE v5.0 were observed at any time point; 4) Meets the "molecular background related to efficacy differences" or clinical characteristics summarized in the first stage and currently at this center, and is considered unsuitable for treatment as a newly diagnosed patient.
Exclusion criteria
Exclusion criteria: 1. Congenital or hereditary aplastic anemia (AA) (e.g., Fanconi anemia, telomeropathies, etc.) or clear evidence of hereditary bone marrow failure syndromes; 2. Coexisting myelodysplastic syndromes (MDS), clinical overt phase of paroxysmal nocturnal hemoglobinuria (PNH), leukemia, or other hematological disorders; 3. History of hematopoietic stem cell transplantation (HSCT); 4. Coexisting severe infections or dysfunction of major organs (e.g., decompensated cirrhosis, severe renal insufficiency) or other conditions significantly affecting efficacy assessment; 5. Contraindications or high risk for androgen therapy, including active liver disease, previous androgen-related liver damage, hepatoadenoma, cholestasis, uncontrolled dyslipidemia, or severe prostate disease; 6. Pregnancy, lactation, or recent plans for pregnancy without reliable contraception; 7. Poor adherence, unable to complete treatment and follow-up assessments as per the study protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Overall Response Rate (ORR);Complete Remission Rate (CR);Partial Remission Rate (PR); | — |
Secondary
| Measure | Time frame |
|---|---|
| Hemoglobin Improvement Level;Platelet improvement degree;Absolute Neutrophil Count Improvement Degree;Blood Transfusion-Dependent Outcome;Functional Assessment of Cancer Therapy - Anemia Scale Scoring;Adverse reaction incidence rate; | — |
Countries
China
Contacts
Public ContactWuDijing
Medical University(Zhejiang Provincial Hospital of Chinese Medicine)
Outcome results
None listed