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.