Burden of illness of trigeminal neuralgia among patients managed in a specialist center in England.

BACKGROUND: Trigeminal neuralgia (TN) causes severe episodic, unilateral facial pain and is initially treated with antiepileptic medications. For patients not responding or intolerant to medications, surgery is an option. METHODS: In order to expand understanding of the pain-related burden of illness associated with TN, a cross-sectional survey was conducted of patients at a specialist center that utilizes a multidisciplinary care pathway. Participants provided information regarding their pain experience and treatment history, and completed several patient-reported outcome (PRO) measures. RESULTS: Of 129 respondents, 69/128 (54%; 1 missing) reported no pain in the past 4 weeks. However, 84 (65%) respondents were on medications, including 49 (38%) on monotherapy and 35 (27%) on polytherapy. A proportion of patients had discontinued at least one medication in the past, mostly due to lack of efficacy (n = 62, 48%) and side effects (n = 51, 40%). A total of 52 (40%) patients had undergone surgery, of whom 30 had microvascular decompression (MVD). Although surgery, especially MVD, provided satisfactory pain control in many patients, 29% of post-surgical patients reported complications, 19% had pain worsen or stay the same, 48% were still taking pain medications for TN, and 33% reported new and different facial pain. CONCLUSIONS: In most PRO measures, respondents with current pain interference had poorer scores than those without pain interference. In the Patient Global Impression of Change, 79% expressed improvement since beginning of treatment at this clinic. These results indicate that while the multidisciplinary approach can substantially alleviate the impact of TN, there remains an unmet medical need for additional treatment options.

Concept confirmation of the Treatment Administration Satisfaction Questionnaire (TASQ) in rare paroxysmal nocturnal hemoglobinuria.

BACKGROUND: This study was conducted to evaluate content validity of the IntraVenous and SubCutaneous Treatment Administration Satisfaction Questionnaires (TASQ-IV and TASQ-SC), for use in a clinical trial population of participants with paroxysmal nocturnal hemoglobinuria (PNH) undergoing eculizumab treatment. METHODS: Participants underwent semi-structured combined brief introduction to disease history and full cognitive debriefing interviews to establish symptoms and key impacts of PNH and to explore the clarity and relevance of both sets of instructions (TASQ-IV and TASQ-SC). The clarity, relevance, response options, and recall period of the TASQ-IV items were also explored. RESULTS: Ten participants with PNH were recruited. Fatigue was the most commonly reported symptom (n = 7); the most commonly reported impact of PNH was on physical activity (n = 4). Nine participants indicated understanding and relevance of the TASQ-IV instructions; three participants suggested changes. Of the 20 TASQ-IV items, ≥ 15 were considered understandable, relevant and to have suitable response options (n ≥ 8). The TASQ-SC instructions were understood by all participants; seven participants indicated relevance. While a few participants suggested minor changes for the items, these reflected the one-off completion of the measure in an interview setting and were thus not considered sufficient to justify modification of the measure for clinical trial completion. CONCLUSIONS: Most participants understood the TASQ-IV and TASQ-SC instructions (n = 9 and 10, respectively) 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.