Case Report: Luspatercept for transfusion-dependent anemia of unknown etiology

Anemia is a common clinical problem with diverse etiologies. Transfusion-dependent anemia (TDA), defined by the need for frequent red blood cell transfusions, can arise from various hematologic disorders. However, in rare instances, despite extensive diagnostic evaluation, the underlying cause may remain elusive. We present a case of a young woman with severe, transfusion-dependent anemia of unknown etiology who failed multiple therapeutic strategies but achieved transfusion independence following treatment with luspatercept. This case highlights the potential for off-label use of luspatercept beyond currently approved indications.