A Study of Romiplostim for the Treatment of Refractory Transfusion-dependent NSAA

Inclusion criteria

• 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.
• An Eastern Cooperative Oncology Group (ECOG) score of 0-2.

Exclusion criteria

• 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.
• Prior treatment with ATG.
• Infection or haemorrhage uncontrolled by standard therapy.
• Allergy to roprostin.
• Active HIV, HCV or HBV infection or cirrhosis or portal hypertension.
• Any concomitant malignancy, localised basal cell carcinoma of the skin within 5 years.
• Liver and renal function at baseline that is more than two times normal.
• Active infection.
• Past history of thromboembolic events, heart attack or stroke (including antiphospholipid antibody syndrome) and current use of anticoagulants.
• Pregnant or lactating (breastfeeding) women.
• Participation in another clinical trial within 3 months.