Aplastic Anemia
Conditions
Brief summary
Efficacy and safety of Romiplostim in the treatment of refractory transfusion-dependent NSAA.
Interventions
DRUGRomiplostim
Enrolled patients will be given Romiplostim (20 µg/kg subcutaneously once a week) for a minimum of 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
Yes
Inclusion criteria
1. Age ≥18 years, male or female. 2. Diagnosis consistent with refractory transfusion dependence NSAA defines refractory as patients who have failed to respond to at least 6 months of prior first-line treatment with adequate doses of cyclosporine (3-5 mg/kg) and who have been treated with an adequate dose of at least one povidone for 3 months. Definition of transfusion dependence: at least 1 component transfusion on average every 8 weeks and duration of transfusion dependence ≥ 4 months. 3. Satisfy at least one of the following conditions at the time of enrolment: haemoglobin \<90 g/L, platelets \<30×10\^9/L, neutrophils \<1.0×10\^9/L. 4. Agree to sign the consent form. 5. An Eastern Cooperative Oncology Group (ECOG) score of 0-2.
Exclusion criteria
1. Other causes of whole blood cytopenia, such as myelodysplastic syndromes (MDS). 2. Presence of cytogenetic evidence of clonal haematological bone marrow disorders (MDS, AML). 3. PNH clones ≥50%. 4. Haematopoietic stem cell transplantation (HSCT) prior to enrolment. 5. Prior treatment with ATG. 6. Infection or haemorrhage uncontrolled by standard therapy. 7. Allergy to roprostin. 8. Active HIV, HCV or HBV infection or cirrhosis or portal hypertension. 9. Any concomitant malignancy, localised basal cell carcinoma of the skin within 5 years. 10. Liver and renal function at baseline that is more than two times normal. 11. Active infection. 12. Past history of thromboembolic events, heart attack or stroke (including antiphospholipid antibody syndrome) and current use of anticoagulants. 13. Pregnant or lactating (breastfeeding) women. 14. Participation in another clinical trial within 3 months.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall response rate (ORR) | 3 months | Overall response rate (ORR) was defined as the ratio of complete response (CR) + partial response (PR).CR was defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients who did not receive a transfusion.PR was defined as not being transfusion-dependent (if previously transfusion-dependent), or doubling or normalisation of at least one cell line or baseline increase, or initial ANC \<0.5 × 10\^9/L increased by at least 0.5 × 10\^9/L after treatment, or initial PLT \<20 × 10\^9/L increased by at least 20 × 10\^9/L after treatment. |
| CRR | 3 months | CR was defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients who did not receive a transfusion. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| safety events | 3 months | Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 |
Countries
China
Outcome results
None listed