Ropustin for Refractory Aplastic Anaemia After Radiotherapy - a Single-centre, Prospective, Open-label, Single-arm Study

Inclusion criteria

• Age ≥18 years, male or female.
• 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 .
• 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.
• Baseline liver and renal function is less than two times the normal value.
• No active infection.
• Agreed to sign the consent form.
• 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%.
• Hematopoietic stem cell transplantation (HSCT) prior to enrolment.
• Prior treatment with ATG.
• Infection or bleeding 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.
• Previous 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.