Aplastic Anemia
Conditions
Keywords
After radiotherapy and chemotherapy, Romiplostim
Brief summary
To investigate the efficacy and safety of roprostin in the treatment of refractory AA after radiotherapy.
Interventions
DRUGRomiplostim
Enrolled patients were given roprostin (20 µg/kg subcutaneously once weekly) for at least 3 months, with discontinuation of roprostin for platelet counts ≥50 x 10\^9/L and continuation of roprostin for platelet counts \<50 x 10\^9/L. Responders were continued to 6 months. Responders continue to use the drug until 6 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, male or female. 2. Diagnosis consistent with refractory AA after radiotherapy. refractory is defined as patients who have failed to respond to at least an adequate amount of supportive therapy, cyclosporine or povidone TPO-RA for 3 months . 3. At least one of the following conditions was met at enrolment: haemoglobin \<90 g/L. Platelets \<30 x 10\^9/L, neutrophils \<1.0 x 10\^9/L. 4. Baseline liver and renal function is less than two times the normal value. 5. No active infection. 6. Agreed to sign the consent form. 7. 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. Hematopoietic stem cell transplantation (HSCT) prior to enrolment. 5. Prior treatment with ATG. 6. Infection or bleeding 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. Previous history of thromboembolic events, heart attack or stroke (including antiphospholipid antibody syndrome) and current use of anticoagulants. 11. Pregnant or lactating (breastfeeding) women. 12. Participation in another clinical trial within 3 months.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| ORR | 3 months, 6 months | Overall response rate (ORR) is defined as the ratio of complete response (CR) + partial response (PR).CR is defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients not receiving transfusion.PR is defined as not transfusion dependent (if previously dependent), or at least one cell lineage doubled or normalised or increased at baseline, or initial ANC \< 0.5 x 10\^9/L increased by at least 0.5 x 10\^9/L after treatment, or initial PLT \< 20 x 10\^9/L increased by at least 20 x 10\^9/L after treatment. |
| CRR | 3 months, 6months | Overall response rate (ORR) is defined as the ratio of complete response (CR) + partial response (PR).CR is defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients not receiving transfusion.PR is defined as not transfusion dependent (if previously dependent), or at least one cell lineage doubled or normalised or increased at baseline, or initial ANC \< 0.5 x 10\^9/L increased by at least 0.5 x 10\^9/L after treatment, or initial PLT \< 20 x 10\^9/L increased by at least 20 x 10\^9/L after treatment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| safety events | 3 months，6months | Proportion and severity of adverse events in patients during the study period |
Countries
China
Contacts
Primary ContactBing Han, PhD
Backup ContactLeyu Wang
Outcome results
None listed