The percent of patients who were unemployed due to PNH was similar to that reported in the International PNH Registry (17%). Over one-half of patients reported symptom severity as moderate to severe and some patients experienced certain symptoms with high frequency 52% of those with fatigue and 41% of those with headaches reported that their symptoms occurred frequently or almost constantly. In a study consisting of concept elicitation interviews with 29 patients with PNH, 97% of participants reported experiencing fatigue, 76% experienced headache, 66% experienced dyspnea, and 59% experienced abdominal pain. In addition, in a subset of 506 participants aged 18–59, 17.4% reported that their PNH was the reason they were not working or working part-time instead of full-time. Patients experiencing symptoms of PNH in the previous 6 months had significantly lower quality of life scores, as measured by the European Organisation for Research and Treatment of Cancer Quality of Life of Cancer Patients (EORTC QLQ-C30), than patients not experiencing symptoms. In the International PNH Registry, among the 856 patients for whom symptom data were available, the most commonly reported symptoms were dyspnea (64%), headache (63%), and fatigue (80%). Patients with PNH experience a high symptom burden that impacts and significantly reduces quality of life. The prevalence of PNH is estimated to be between 10 and 15 per million, with a mean age of diagnosis between 30 and 45 years. Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disease of the blood caused by a chronic dysregulation of the complement system and is characterized by hemolytic anemia, thrombosis, and impaired bone marrow function. The PNH-SQ is a content-valid questionnaire suitable for assessing daily symptom presence and severity in PNH clinical trials. The finalized PNH-SQ assesses the presence and severity of 10 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-over 24 h. Participants also suggested changes regarding item redundancy and relevance this feedback was used to finalize the instrument. The interviews demonstrated that participants understood the items of the PNH-SQ (90–100%) considered the symptoms relevant (> 50– > 90%) the recall period appropriate (> 80–100%) and the response options suitable (> 80–100%). Further, interviews confirmed the instrument captured the most common PNH symptoms, including fatigue (87%), abdominal pain (60%), and difficulty swallowing (47%), with fatigue ranked as the most bothersome symptom. Concept saturation was reached for all PNH symptoms. Participants mentioned 27 signs or symptoms of PNH spontaneously or after being probed 93% reported experiencing ≥ 1 PNH symptom. Patient interview data were also used to finalize the PNH conceptual model. We then interviewed 15 patients with PNH to assess comprehensiveness of symptom capture from the patient perspective and to cognitively debrief the PNH-SQ. Following a literature review and consultation with a PNH expert, we drafted the PNH Symptom Questionnaire (PNH-SQ) and a patient-centric conceptual model of PNH symptoms and impacts. Patient-reported outcome measures (PROs) used to measure symptoms of patients with paroxysmal nocturnal hemoglobinuria (PNH) in trials do not measure PNH symptoms comprehensively and do not assess daily fluctuations in symptoms.
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