Aplastic Anemia
Conditions
Brief summary
This study aimed to explore the efficacy and safety of romiplostim N01 in the treatment of chemoradiotherapy-induced aplastic anemia (AA)
Interventions
DRUGRomiplostim N01
Romiplostim N01 20 µg/kg subcutaneously once a week for at least 3 months
Sponsors
Peking Union Medical College Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Age ≥ 18 years old; 2. Diagnosed with relapsed/refractory chemoradiotherapy-induced AA. Relapsed/ refractory was defined as patients who had no response to at least 3 months of cyclosporine or thrombopoietin receptor agonists (TPO-RAs) such as eltrombopag; 3. At least one of the following conditions must be met: hemoglobin \< 90 g/ L, platelet count \< 30×109/L, neutrophil count \< 1.0×109/L; 4. With baseline liver and kidney functions \<2 upper limit of normal (ULN); 5. Without active infection that cannot be controlled by standard treatment; 6. Signed the informed consent; 7. ECOG score ≤ 2;
Exclusion criteria
1. Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.; 2. With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics; 3. Paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50%; 4. Received hematopoietic stem cell transplantation before enrollment; 5. Received ATG treatment before enrollment; 6. Severe bleeding or infection that cannot be controlled by standard treatment; 7. Allergic or intolerant to romiplostim N01; 8. Active infections of HIV, HCV, or HBV, liver cirrhosis, or portal hypertension 9. History of malignant tumors within 5 years; 10. History of arterial or venous thrombosis; 11. Pregnant or lactating patients; 12. Participated in other clinical trials within 3 months.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| ORR (Overall Response Rate) | 3-month, 6-month | ORR=PRR (Partial Response Rate)+CRR (Complete Response Rate) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| CRR | 3-month, 6-month | Proportion of patients who achieved CR |
| Adverse event (AE) rate | through study completion, an average of 1 year | Proportion of patients with AEs, according to CTCAE |
| Predictive factors for ORR or CRR | 3-month | Calculated by single-/multivariate regression |
| Clonal evolution rate | through study completion, an average of 1 year | Proportion of patients progressed to myelodysplastic syndromes (MDS)/ acute myeloid leukemia (AML) |
Contacts
CONTACTBing Han
Outcome results
None listed