Bone Marrow Failure Disorders

Researchers used a literature review and consultation with an expert in paroxysmal nocturnal hemoglobinuria (PNH) to develop a PNH Symptom Questionnaire (PNH-SQ) and patient-centric conceptual model of PNH symptoms and their impacts. Data from interviews of 15 patients with PNH were used to finalize the conceptual model. The PNH-SQ assesses over a 24-hour period the presence and severity of 10 PNH symptoms: abdominal pain, chest discomfort, difficulty sleeping, difficulty swallowing, difficulty thinking clearly, fatigue, headache, muscle weakness, pain in the legs or back, and shortness of breath.