A concept elicitation study to understand the relationship between sleep and pain in rheumatoid arthritis and axial spondyloarthritis.

PURPOSE: This qualitative study (GSK study: 213635) was designed to better understand sleep disturbance as experienced by individuals with rheumatoid arthritis (RA) or axial spondyloarthritis (axSpA), and the relationship between sleep disturbance and pain and other aspects of the disease and disease activity. METHODS: Sixty-minute, one-on-one, concept elicitation interviews were conducted with 30 participants (15 with RA and 15 with axSpA) from the US. Interviews were audio-recorded and transcribed verbatim. Interview transcripts were coded and analyzed to explore themes related to pain and sleep disturbance, and relationships among those themes. RESULTS: Pain was a prominent driver of sleep disturbance; 12 participants with RA (80%) and 14 with axSpA (93%) reported that pain impacted their ability to fall asleep, while all 15 with RA (100%) and 14 with axSpA (93%) reported that pain impacted their ability to stay asleep. Two-thirds of participants with RA (67%) or axSpA (60%) described a bi-directional relationship, whereby pain worsened sleep disturbance and sleep disturbance further aggravated pain. Factors other than pain, such as fatigue and emotional health, were also reported as important contributors to sleep disturbance (RA: n = 12/15, 80%; axSpA: n = 14/15, 93%). Participants with RA or axSpA described complex interconnections between fatigue, emotional health, pain, and sleep, often labeling these relationships as "vicious cycles". Notably, half of all participants reported sleep disturbance occurring without pain or other understood causes. CONCLUSION: These perspectives collected from people with RA or axSpA suggest that reducing sleep disruption directly may offer clinically relevant benefits.

Content Validation of the Movement Disorder-Childhood Rating Scale (MD-CRS) for Dyskinetic Cerebral Palsy.

BACKGROUND: Dyskinetic cerebral palsy (DCP), a lifelong neurological disorder beginning in early childhood, manifests with hyperkinetic movements and dystonia. The Movement Disorder-Childhood Rating Scale (MD-CRS) is a clinician-reported outcome measure assessing the intensity of movement disorders and their effect on daily life in pediatric patients. Content validity of clinical outcome assessments is key to accurately capturing patient perspective. Evidence demonstrating content validity of the MD-CRS in patients with DCP is needed. This study captures input from patients with DCP and their caregivers regarding the content validity of the MD-CRS. METHODS: This qualitative, noninterventional, cross-sectional study included interviews with children/adolescents (aged six to 18 years) with DCP and caregivers of children with DCP. Participants were asked to describe body regions and daily functions affected by DCP. Caregivers also reviewed MD-CRS Part I to evaluate the relevance of the items and corresponding response options. Descriptions of DCP were coded and mapped to MD-CRS items and response options. Caregiver feedback on MD-CRS Part I was analyzed using inductive content analysis. RESULTS: Eight patients and 12 caregivers were interviewed. Participants confirmed that the body regions and activities listed in the MD-CRS were affected by DCP and that involuntary movements interfered with all motor, oral/verbal, self-care, and video protocol activities. Caregivers endorsed the response options for 12 of 15 items in MD-CRS Part I and suggested clarifications for others. CONCLUSIONS: Participants confirmed that affected body regions and activities listed in the MD-CRS were relevant to their experience with DCP, demonstrating the content validity of this tool in children/adolescents with DCP.

Understanding the Symptom Burden and Impact of Myasthenia Gravis from the Patient's Perspective: A Qualitative Study.

INTRODUCTION: Patients with generalized myasthenia gravis (gMG) experience functional impairment due to MG symptoms. This study aimed to assess, from the patient perspective, the symptoms, impacts, and treatment goals of individuals diagnosed with gMG. METHODS: Semi-structured, in-depth concept-elicitation interviews were conducted with 28 individuals diagnosed with gMG in the United States. RESULTS: Participants reported gMG symptoms that affected many body regions and functions, with an average of 16 symptoms per participant. The most frequently reported symptoms were eyelid drooping (93%), physical fatigue (89%), symptoms affecting the legs (82%), difficulty breathing (82%), and difficulty holding head up (82%). Nearly all participants (96%) reported fluctuations in symptoms and severity. Participants' most bothersome symptoms were blurry/double vision (43%), breathing difficulties (36%), all-over fatigue (36%), and swallowing problems (29%). Impacts on physical functioning included an inability to participate in hobbies/sports, need for increased planning, and difficulties performing activities of daily living. All participants reported emotional impacts and impacts on their work and finances. Their treatment goals included reduced fatigue and weakness, improved symptom stability, and minimization of the impact of symptoms, in particular the emotional impact. CONCLUSIONS: The fluctuating and unpredictable nature of gMG symptoms was found to have a substantial impact on patients' emotional, social, and economic well-being. Participants' goals for symptom management suggest that greater focus is needed to help them quickly resume a normal lifestyle by achieving symptom stability. Impacts of fluctuating and unpredictable symptoms are difficult to measure, but it is important to consider symptom fluctuation as well as ongoing symptomatology when making treatment decisions, and to recognize the impact of uncontrolled symptoms on patients, their partners/caregivers, and family/friends. These factors are often not reflected in burden/cost-of-illness studies.

The aim of this study was to understand­from the patient's point of view­the range of generalized myasthenia gravis (gMG) symptoms that they experience, which symptoms bother them most, and which symptoms have the greatest impact on everyday life, as well as patients' treatment goals. Researchers asked these questions in one-on-one interviews with 28 people in the US who have gMG. Study participants reported living with symptoms that come and go, and are sometimes severe, making it difficult to lead a normal life. The most frequently reported symptoms were eyelid drooping (reported by 93% of study participants), physical fatigue (89%), symptoms affecting the legs (82%), difficulty breathing (82%), and difficulty holding head up (82%). The symptoms that bothered patients most were difficulties with vision (43%), problems breathing (36%), all-over fatigue (36%), and trouble swallowing (29%). Participants reported that gMG symptoms affected physical functioning, making it hard to participate in hobbies/sports, increasing the amount of planning needed to conduct their daily lives, and hindering their ability to do day-to-day activities (like driving, eating, and bathing). All participants reported that they were affected emotionally, and that the symptoms of gMG impacted their ability to work and their financial well-being. Participants' treatment goals included reducing fatigue and weakness, making symptoms more stable, and reducing the impact of symptoms, particularly emotional impacts. These responses reveal the extensive effects of gMG symptoms on patients' daily lives and highlight that symptom stability is especially important to people with gMG.

Qualitative research with patients and caregivers of patients with PIK3CA related overgrowth spectrum: content validity of clinical outcome assessments.

BACKGROUND: PIK3CA-Related Overgrowth Spectrum (PROS) are rare syndromes caused by a mutation in the PIK3CA gene, including fibroadipose hyperplasia or overgrowth; congenital lipomatous overgrowth, vascular malformations, epidermal nevi, scoliosis/skeletal and spinal (CLOVES); megalencephaly-capillary malformation (MCAP or M-CM); fibro-adipose vascular anomaly (FAVA); Klippel-Trenaunay syndrome (KT; also known as, Klippel-Trenaunay-Weber syndrome); capillary, lymphatic, and venous malformations (CLVM); and lymphatic malformation (LM). Characterized by malformations and tissue overgrowth, PROS manifests at birth or in early childhood. Pain and functional limitations associated with these conditions may greatly impact the health-related quality of life (HRQoL) of persons with PROS including physical functioning, work/school, social functioning, and emotional well-being. RESULTS: Selected clinical outcome assessments (COAs), identified during a literature review, were tested with adults with PROS, and children with PROS and their caregivers to determine comprehensibility, relevance, and appropriateness for measuring symptom severity and HRQoL. Tested were the Patient Global Impression of Symptom Severity (PGI-S), Brief Pain Inventory (BPI), Wong-Baker FACES, Patient-Reported Outcomes Measurement Information System (PROMIS) Profile, PROMIS Pediatric Short Form Sleep Disturbance, and PROMIS Dyspnea Severity. Qualitative interviews tested the self-report adult, self-report pediatric, and observer-report COAs with adults with PROS, and children with PROS and their caregivers. Ten adults (≥ 18 years old) with PROS, and 20 children (6-17 years old) with PROS and their caregivers, participated. All reported positive feedback on item relevance. Adults and children over the age of 12 comprehended and responded to self-reported items. Secondary objectives examined the age children could self-report their conditions using pediatric versions and assessed available observer-report versions of the COAs with caregivers. Some participants under the age of 12 had trouble understanding some terminology. Further, adults and children with cognitive impairment associated with MCAP/M-CM sometimes had difficulty with self-report. Caregivers were able to report their child's symptoms and impacts using observer-report COAs. Participant feedback prompted further consideration of the measurement of pain in this population, including variability of pain over time, location of pain, and type. CONCLUSIONS: This study provided valuable information from patients about PROS, supporting the content validity of the COAs, with recommended revisions. COAs are easily understood by persons with PROS and caregivers and are appropriate for measuring symptoms and disease-related impacts across diverse PROS syndromes in clinical trials.