Bone Marrow Failure Disorders

A team of investigators from the US and UK has confirmed content validity of the IntraVenous and SubCutaneous Treatment Administration Satisfaction Questionnaires (TASQ-IV and TASQ-SC) for use in clinical trials of patients with paroxysmal nocturnal hemoglobinuria (PNH) undergoing treatment with eculizumab. “Most participants understood the TASQ-IV and TASQ-SC instructions (n=9 and n=10, respectively),” they wrote, “and the TASQ-IV items were considered clear, relevant, and to have suitable response options, demonstrating face and content validity of the instruments for the clinical trial setting.”