Quality of life and subjective symptom impact in Japanese patients with myotonic dystrophy type 1.

BACKGROUND: Although functional impairment in patients with myotonic dystrophy is an important determinant of the quality of life (QoL), patients' subjective evaluation of their symptoms may also affect their QoL. The aim of this study was to investigate the association between subjective symptom impact and the QoL of patients with myotonic dystrophy, after controlling for functional impairment. METHODS: Eligible patients with myotonic dystrophy type 1 (DM1) were recruited from four hospitals in Japan. The subjective symptom impact of four symptoms (muscle weakness, fatigue, pain, and myotonia) and overall QoL were evaluated using the Individualized Neuromuscular Quality of Life (INQoL) questionnaire. Functional impairment was assessed using the modified Rankin Scale. RESULTS: Seventy-seven patients with DM1 were included in this study. Overall QoL was significantly associated with subjective symptom impact of muscular weakness, fatigue, pain, myotonia, swallowing difficulty, and droopy eyelids. In the regression models, disease duration (beta = 0.11) and moderate to severe functional impairment (beta = 0.33) explained a significant part of the overall QoL. Furthermore, muscular weakness, fatigue, and myotonia significantly explained additional variance of the overall QoL (beta = 0.17-0.43). CONCLUSIONS: Subjective symptom impact and functional impairment are independent features influencing the QoL of Japanese patients with DM1.

Development of Focal Segmental Glomerulosclerosis Patient-Reported Outcome Measures: Symptom Diary and Symptom Impact Questionnaire.

BACKGROUND: Focal segmental glomerulosclerosis (FSGS) is a kidney disease that affects patients' functioning and well-being. This study aimed to develop patient-reported outcome questionnaires to measure patient experiences related to FSGS. STUDY DESIGN: Qualitative patient interviews to identify important symptoms and concepts (concept elicitation) formed the basis for the development of 2 questionnaires, one on symptoms and one on their impact. Additional qualitative interviews were implemented to evaluate/refine the questionnaires (cognitive debriefing). Transcripts of concept elicitation and cognitive debriefing interviews, conducted by telephone, were analyzed for concepts of interest using qualitative text analysis. SETTING & PARTICIPANTS: Patients with FSGS (aged 18-65 years with estimated glomerular filtration rates ≥ 40mL/min/1.73m2) whose disease remained inadequately controlled after 2 or fewer courses of treatment. METHODOLOGY: Qualitative concept elicitation and cognitive debriefing interviews. ANALYTICAL APPROACH: Interview transcripts were analyzed using qualitative software, MAXQDA. RESULTS: 30 patients completed concept elicitation interviews; 9 patients completed cognitive debriefing interviews. Frequently mentioned symptoms included swelling from the waist down/legs/knees/feet/ankles (67%), fatigue (57%), stomach/abdomen swelling (43%), body pain/pressure (30%), and shortness of breath (20%), as well as impacts on physical (52%), emotional (68%), and social functioning (89%). Based on analyses of interview transcripts and clinical input, 2 questionnaires, one on symptoms and one on the impact of the symptom, were drafted. The 23-item FSGS Symptom Diary (assessing the frequency and severity of FSGS symptoms during the past 24 hours) and the FSGS Symptom Impact Questionnaire (17 items assessing interference with activities and emotions during the past 7 days) were iteratively revised based on cognitive debriefing interviews. LIMITATIONS: The study was restricted to English-speaking adults located in the United States, and the concept elicitation interview group had a low number of African Americans. CONCLUSIONS: The FSGS Symptom Diary and FSGS Symptom Impact Questionnaire are new FSGS-specific patient-reported outcomes measures designed to support a comprehensive assessment of symptoms and symptom impact in adults with FSGS. Future research is needed to evaluate their quantitative measurement properties.

The Adaptation of 12-Item Utah Photophobia Symptom Impact Scale (UPSIS-12) in Turkish, Internal Consistency, and Reproducibility.

Introduction: The 12-item Utah Photophobia Symptom Impact Scale (UPSIS-12) is a questionnaire to assess the impact of photophobia on daily living during and outside migraine episodes. We aimed to translate UPSIS-12 and assess its usability in the Turkish cohort by analyzing internal consistency, reliability, and reproducibility. Methods: In this multicenter prospective, descriptive study, 120 patients with migraine were recruited. The patients received the diagnosis of migraine without aura (MwoA) and migraine with aura (MwA) according to the International Classification of Headache Disorders 3rd edition. The scale was translated into Turkish, and the patients were evaluated by headache experts in two visits four weeks apart from each other. Results: The questionnaire showed highly correlated test-retest reliability (r=0.828). Cronbach alpha values of internal consistency revealed good to excellent results ranging from 0.78-0.82. There was a strong and positive correlation between self and physician-administered scales at both visits (r=0.964 and 0.985, respectively). The questionnaire was considered "well understood" by 95.6% of the patients. The average UPSIS-12 score of each visit was 25.8 and 26.9 (average standard deviation of 10.4). Subjects with MwoA (25.5±10.4) had remarkably lower mean scores compared to MwoA plus MwA (29.2±8.9) and MwA (32.9±9.4) (p=0.0167). Patients with high frequency migraine presented with a higher mean UPSIS-12 score than those with low frequency (31.3±9.5 and 24.7±9.8, respectively). It was noted that the mean UPSIS-12 scores of those with severe (29.4±9.4) and moderate ictal cutaneous allodynia (CA) (28.4±10.6) were higher than those without CA (22.0±9.3)(p=0.020). Conclusion: The Turkish version of the UPSIS-12 questionnaire is a reliable tool and well-understood by the Turkish population. Patients with MwA, frequent migraine headache days and moderate to severe CA presented with higher scores. Given the independent burden of photophobia in migraineurs, the use of UPSIS-12 scale should be encouraged in Turkish population.

Factors influencing alopecia areata treatment decisions: A qualitative assessment.

Background: Alopecia areata (AA) is a disease of hair loss in which patients may benefit from comprehensive understanding of AA's disease process and therapeutic options during treatment decision-making. Objective: Determine factors influencing patients' AA treatment decision-making. Methods: Qualitative interviews were conducted using semi-structured interview guides. Interviews were coded using inductive thematic analysis. Results: Twenty-one participants with AA were interviewed. Coding interrater reliability was κ = 0.87-0.91, indicating strong-almost perfect agreement. Participants faced multiple barriers, including lack of access to health care (n = 10, 47.6%) and lack of transparency about their condition and treatment options (n = 9, 42.9%). Information about AA was sought from primarily the internet (n = 15, 71.4%) and physician recommendation (n = 15, 71.4%). When choosing AA treatments, patients often considered treatment efficacy (n = 21, 100%), safety (n = 21, 100%), and convenience of use (n = 20, 95.2%). Limitations: Referral and regional biases may be present and limit generalizability. Conclusions: Patients with AA face various challenges including medical uncertainty and lack of information. Patients need trustworthy and accessible sources of information regarding their treatment that also take into consideration their preferences and values.

The STatin Adverse Treatment Experience Survey: Experience of patients reporting side effects of statin therapy.

BACKGROUND: It is important to understand patients' experiences of statin-associated adverse effects to potentially identify those at risk for stopping treatment. OBJECTIVE: The goal of the STatin Adverse Treatment Experience survey was to describe patients' experiences after reporting ≥1 recent statin-associated adverse event and identify opportunities to improve adherence and outcomes. METHODS: The survey was developed in 3 stages: qualitative item development, pilot evaluation of initial item performance, and quantitative evaluation using a large commercial sample. Respondents with self-reported high cholesterol who had taken a statin in the past 2 years and experienced ≥1 statin-associated symptom in the past 6 months were included (N = 1500). RESULTS: Mean age was 58 years, 40.3% were men, and 43.2% had tried ≥2 statins. Many had clinical comorbidities associated with increased risk for cardiovascular disease (atherosclerotic cardiovascular disease, 22.5%; diabetes, 25.8%; hypertension, 56.0%). The most important patient-reported reasons for continuing current statin therapy (n = 1168; 77.9%) were avoiding a heart attack or stroke, lowering cholesterol, and doctor recommendation. Being bothered by and not being able to tolerate side effects were the main reasons respondents discontinued statins (n = 332; 22.1%). Respondents who discontinued statins reported significantly higher mean Symptom Severity (10.6 vs 8.7, P < .001) and Impact Severity scores (11.8 vs 9.8, P < .001) compared with those who continued. CONCLUSION: The STatin Adverse Treatment Experience survey highlights the importance of patients' adverse experiences with statins and how symptom and impact scores affect decisions to continue or discontinue therapy. These data provide a foundation to increase providers' awareness of statin tolerability from the patient's perspective and encourage benefit-risk discussions.

A web-based intervention for carers of individuals with anorexia nervosa (We Can): Trial protocol of a randomised controlled trial investigating the effectiveness of different levels of support.

BACKGROUND: Anorexia nervosa (AN) is a life-threatening mental disorder that is associated with substantial caregiver burden. Carers of individuals with AN report high levels of distress and self-blame, and insufficient knowledge to help their loved ones. However, carers can have a very important role to play in aiding recovery from AN, and are often highly motivated to assist in the treatment process. This manuscript presents the protocol for a randomised controlled trial (RCT) of We Can, a web-based intervention for carers for people with AN. The study aims to investigate the effectiveness of We Can delivered with different intensities of support. METHODS: The study takes the form of a multi-site, two-country, three group RCT, comparing We Can (a) with clinician messaging support (We Can-Ind), (b) with moderated carer chatroom support (We Can-Chat) and (c) with online forum only (We Can-Forum). Participants will be 303 carers of individuals with AN as well as, where possible, the individuals with AN themselves. Recruitment will be via specialist eating disorder services and carer support services in the UK and Germany. Randomisation of carers to one of the three intervention conditions in a 1:1:1 ratio will be stratified by whether or not the individual with AN has (a) agreed to participate in the study and (b) is a current inpatient. The We Can intervention will be provided to carers online over a period of 12 weeks. Participants will complete self-report questionnaires at pre-intervention (T1), mid-intervention (mediators only; 4-weeks post-randomisation), post-intervention (T2; 3-months post randomisation), and 6 months (T3) and 12 months (T4) after randomisation. The primary outcome variables are carer symptoms of depression and anxiety. Secondary outcome variables will be measured in both carers and individuals with AN. Secondary carer outcome variables will include alcohol and drug use and quality of life, caregiving behaviour, and the acceptability and use of We Can and associated supports. Secondary outcomes measured in individuals with AN will include eating disorder symptoms, and symptoms of depression and anxiety. The study will also evaluate the cost-effectiveness of the three We Can conditions, and test for mediators and moderators of the effects of We Can. The trial is registered at the International Standard Randomisation Controlled Trial Number (ISRCTN) database, registration number: ISRCTN11399850. DISCUSSION: The study will provide insight into the effectiveness of We Can and its optimal method/s of delivery.

Evaluating Disease Severity in Chronic Pain Patients with and without Fibromyalgia: A Comparison of the Symptom Impact Questionnaire and the Polysymptomatic Distress Scale.

OBJECTIVE: To compare the relative effectiveness of the Polysymptomatic Distress Scale (PSD) with the Symptom Impact Questionnaire (SIQR), the disease-neutral revision of the updated Fibromyalgia Impact Questionnaire (FIQR), in their ability to assess disease activity in patients with rheumatic disorders both with and without fibromyalgia (FM). METHODS: The study included 321 patients from 8 clinical practices with some 16 different chronic pain disorders. Disease severity was assessed by the Medical Outcomes Study Short Form-36 (SF-36). Univariate analyses were used to assess the magnitude of PSD and SIQR correlations with SF-36 subscales. Hierarchical stepwise regression was used to evaluate the unique contribution of the PSD and SIQR to the SF-36. Random forest regression probed the relative importance of the SIQR and PSD components as predictors of SF-36. RESULTS: The correlations with the SF-36 subscales were significantly higher for the SIQR (0.48 to 0.78) than the PSD (0.29 to 0.56; p < 0.001). Stepwise regression revealed that the SIQR was contributing additional unique variance on SF-36 subscales, which was not the case for the PSD. Random forest regression showed SIQR Function, Symptoms, and Global Impact subscales were more important predictors of SF-36 than the PSD. The single SIQR pain item contributed 55% of SF-36 pain variance compared to 23% with the 19-point WPI (the Widespread Pain Index component of PSD). CONCLUSION: The SIQR, the disease-neutral revision of the updated FIQ, has several important advantages over the PSD in the evaluation of disease severity in chronic pain disorders.