Inclusion and exclusion criteria
β-thalassemia syndrome, cytopenias, pulmonary deficits, liver and kidney function, hemoglobinopathies, thrombotic, transfusion-dependent, infection, NTDT, neurological problems
INFECTION, HEMOGLOBINOPATHIES, CYTOPENIAS, CARDIAC DISEASE
The study included diagnosed HU refractory TDT patients of either gender, aged ≥ 18 months. Patient/parent/guardian was educated regarding the course of thalidomide therapy, and then full informed consent was obtained. Patients who remained transfusion-dependent despite using HU for ≥ 6 months were considered HU refractory. TDT diagnosis was ascertained by obtaining a valid Hb electrophoresis and/or HPLC report performed pre-transfusion at an age of 6 months or older, suggestive of β-thalassemia syndrome. Alternatively genetic testing profile (comprising PCR or HBB gene sequencing) suggestive of β-thalassemia syndrome was also regarded a diagnostic strategy. Patients with NTDT, viable pregnancy or planning conception, prescribed erythropoiesis stimulating agents for ≤ 3 months before enrollment, hemoglobinopathies other than β-thalassemia, altered liver and kidney function, thrombotic predisposition, history of neurological problems, cardiac disease, pulmonary deficits, cytopenias and/or active infection were deferred/excluded from the study. Enrolled female patients were well informed that they should absolutely avoid pregnancy during the study period and until 6 months after thalidomide withdrawal.