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The first beta-thalassemia patient to be treated and who did not receive backup cells was an 18-year-old E / 0 adult with clinical beta-thalassemia major who had been transfusion-dependent and on parenteral iron chelation therapy since early childhood and had undergone splenectomy at the age of six years, and for whom no HLA-MSD was available [169], [170], [174]

138 The dose-response trial also lacked a placebo control group and reported at eight months no differences in proportions of patients experiencing clinically significant improvements (300 mg daily 50%