Efficacy and safety of Romiplostim in the treatment of refractory transfusion-dependent NSAA.
Age ≥18 years, male or female.
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.
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.
Agree to sign the consent form.
Other causes of whole blood cytopenia, such as myelodysplastic syndromes (MDS).
Presence of cytogenetic evidence of clonal haematological bone marrow disorders (MDS, AML).
PNH clones ≥50%.
Haematopoietic stem cell transplantation (HSCT) prior to enrolment.
Single group
Romiplostim
DrugEnrolled patients will be given Romiplostim (20 µg/kg subcutaneously once a week) for a minimum of 3 months.
Overall response rate (ORR)
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.
Overall response rate (ORR)
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
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.
CRR
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.
safety events
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
safety events
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
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