A Qualitative Concept Elicitation Study to Understand Patient-Reported Symptoms and Impacts of Neisseria gonorrhoeae Infections in the United States.

BACKGROUND: Gonorrhea is the second most common bacterial sexually transmitted infection in the United States, with rising rates. Emerging antimicrobial resistance threatens public health, and vaccines are in development. This study documents patient-reported gonorrhea symptoms and health-related quality-of-life (HRQoL) impact in women who have sex with men (WSM), men who have sex with women (MSW), and men who have sex with men (MSM). METHODS: Semistructured qualitative interviews were conducted (April 2021-March 2022) among US adults with recent (≤6 months) confirmed gonorrhea. Concept saturation was achieved, confirming adequate participant numbers to meet objectives. Elicited symptom and HRQoL impacts were used to develop a conceptual disease model. Common patient-reported outcome measure (PROM) items were compared with elicited concepts. RESULTS: Thirty-two participants (15 WSM, 8 MSW, and 9 MSM) were included. Eight were asymptomatic, 6 had repeat infections, and 5 women and 4 men had complications (i.e., infertility or pelvic inflammatory disease, and urethritis, respectively).The most frequently reported symptoms were vaginal discharge (n = 11 of 12), dysuria (n = 10 of 12), and abdominal/vaginal pain (n = 9 of 12) for symptomatic WSM; dysuria (n = 5 of 6) and penile discharge (n = 4 of 6) for symptomatic MSW; and throat, testicular, or rectal pain (n = 4 of 6) for symptomatic MSM. All (symptomatic and asymptomatic) participants reported HRQoL impacts including negative emotional experiences (n = 31 of 32), and interference with sexual activity (n = 30 of 32), relationships (n = 24 of 32), and social life (n = 17 of 32). Concepts were poorly represented in existing PROMs. CONCLUSIONS: Participants reported a diverse range of symptoms and HRQoL impacts, to help inform the value of new treatment and prevention options. More research is needed to quantify patient burden and develop PROMs.

Efficacy and safety of single-agent belantamab mafodotin versus pomalidomide plus low-dose dexamethasone in patients with relapsed or refractory multiple myeloma (DREAMM-3): a phase 3, open-label, randomised study.

BACKGROUND: Multiple myeloma remains incurable, and heavily pretreated patients with relapsed or refractory disease have few good treatment options. Belantamab mafodotin showed promising results in a phase 2 study of patients with relapsed or refractory multiple myeloma at second or later relapse and a manageable adverse event profile. We aimed to assess the safety and efficacy of belantamab mafodotin in a phase 3 setting. METHODS: In the DREAMM-3 open-label phase 3 study, conducted at 108 sites across 18 countries, adult patients were enrolled who had confirmed multiple myeloma (International Myeloma Working Group criteria), ECOG performance status of 0-2, had received two or more previous lines of therapy, including two or more consecutive cycles of both lenalidomide and a proteasome inhibitor, and progressed on, or within, 60 days of completion of the previous treatment. Participants were randomly allocated using a central interactive response technology system (2:1) to receive belantamab mafodotin 2·5 mg/kg intravenously every 21 days, or oral pomalidomide 4·0 mg daily (days 1-21) and dexamethasone 40·0 mg (20·0 mg if >75 years) weekly in a 28-day cycle. Randomisation was stratified by previous anti-CD38 therapy, International Staging System stage, and number of previous therapies. The primary endpoint was progression-free survival in all patients who were randomly allocated. The safety population included all randomly allocated patients who received one or more doses of study treatment. This trial is registered with ClinicalTrials.gov, NCT04162210, and is ongoing. Data cutoff for this analysis was Sept 12, 2022. FINDINGS: Patients were recruited between April 2, 2020, and April 18, 2022. As of September, 2022, 325 patients were randomly allocated (218 to the belantamab mafodotin group and 107 to the pomalidomide-dexamethasone group); 184 (57%) of 325 were male and 141 (43%) of 325 were female, 246 (78%) of 316 were White. Median age was 68 years (IQR 60-74). Median follow-up was 11·5 months (5·5-17·6) for belantamab mafodotin and 10·8 months (5·6-17·1) for pomalidomide-dexamethasone. Median progression-free survival was 11·2 months (95% CI 6·4-14·5) for belantamab mafodotin and 7·0 months (4·6-10·6) for pomalidomide-dexamethasone (hazard ratio 1·03 [0·72-1·47]; p=0·56). Most common grade 3-4 adverse events were thrombocytopenia (49 [23%] of 217) and anaemia (35 [16%]) for belantamab mafodotin, and neutropenia (34 [33%] of 102) and anaemia (18[18%]) for pomalidomide-dexamethasone. Serious adverse events occurred in 94 (43%) of 217 and 40 (39%) of 102 patients, respectively. There were no treatment-related deaths in the belantamab mafodotin group and one (1%) in the pomalidomide-dexamethasone group due to sepsis. INTERPRETATION: Belantamab mafodotin was not associated with statistically improved progression-free survival compared with standard-of-care, but there were no new safety signals associated with its use. Belantamab mafodotin is being tested in combination regimens for relapsed or refractory multiple myeloma. FUNDING: GSK (study number 207495).

Understanding the patient experience of living with notalgia paresthetica: A qualitative interview study.

Background: Notalgia paresthetica (NP) is a chronic sensory neuropathy that causes intense pruritus, typically affecting the upper portion of the back and lasting for months to years. The impacts of pruritus and the full symptom experience are not well documented. Objective: To describe patients' NP symptom experience and the impacts of living with NP-related itch. Methods: Semistructured, one-to-one qualitative telephone interviews were conducted in adults living with NP. The worst itch severity during the previous 24 hours was assessed using an 11-point numerical rating scale. Results: Thirty participants (23 females; aged 41-80 years) completed interviews. Seventeen NP-related symptoms were described, with daily itch being the most common. Participants' itch ranged from severe to very severe (50%) and was experienced for a median of 2.8 years. Most (73%) participants were not receiving treatment for NP at the time of the study. Other reported symptoms included skin pain or sensitivity and secondary symptoms from itching (skin discoloration, lumps or bumps, bleeding or scabbing). NP-related itch was frequently reported to affect mood, interfere with sleep, and disrupt self-care. Limitations: Only English-speaking participants living in the United States were included. Conclusion: This study highlights the patient experience of living with NP and findings reveal that there remains an unmet need for effective therapeutic options to address NP-related itch.

Validation of patient-reported vaso-occlusive crisis day as an endpoint in sickle cell disease studies.

Individuals with sickle cell disease (SCD) experience vaso-occlusive crises (VOC). Historically, VOC episodes have been assessed through medical utilization, thereby excluding events managed at home. In order to validate a daily patient-reported outcome for patients with SCD to accurately report their VOC status and experience of a pain crisis, a SCD Diary was included in Evaluation of Longitudinal Pain Study in Sickle Cell Disease (ELIPSIS), a longitudinal, six-month, non-interventional study. The daily patient-completed diary included a description of SCD pain crisis, followed by questions on: pain crisis in the past 24 h (VOC Day question; respective response yes or no), worst pain, tiredness, and functioning. Thirty-five patients with SCD participated in ELIPSIS. Analyses were performed to validate the patient-reported VOC Day. Mean symptoms and functioning scores on the first or last VOC Day of a VOC Event were compared using t-tests with the mean of the three non-VOC Days before and after the event. Mean severity of symptoms and functioning scores on all VOC Days compared to all non-VOC Days were higher, with statistically significant mean differences between first/last VOC Days and respective three non-VOC Days (p's < .01). A subset of patients (n = 15) and caregivers (n = 9) were interviewed to evaluate their understanding of the SCD Diary questions. Nearly all confirmed that the pain crisis description accurately described the VOC experience, and participants expressed confidence differentiating SCD crisis pain from everyday pain. These results demonstrate patients can reliably report their experiences with VOC-related pain crises using the SCD Diary.

Clinical scoring algorithm for the prescription opioid misuse and abuse questionnaire (POMAQ).

OBJECTIVE: The Prescription Opioid Misuse and Abuse Questionnaire (POMAQ) was developed to identify prescription opioid misuse and abuse among patients with chronic pain. A clinical scoring algorithm was developed and refined to align with the patient experience. METHODS: This study utilized data from the POMAQ validation study (3033-4, NCT02660606) conducted on a sample of patients with chronic pain living in the United States. The study was carried out in two phases. Two purposefully enriched patient samples, one for each phase, were created based on patient responses to select POMAQ items and the availability of urine and hair samples. Two clinical experts (SHS, SFB) reviewed patient data to classify prescription opioid use behavior. Classification differences were adjudicated by a third clinical expert (JTF). Comparisons were made between the final clinical classification determined by the experts and the proposed classification based on the POMAQ algorithm. RESULTS: Sixty patients were included in Phase I (only POMAQ data) and 52 in Phase II (including POMAQ and ancillary sources [e.g. electronic medical records, urine toxicity screen]). Refinements were made to the POMAQ scoring algorithm following discussions with clinical experts to ensure it was clinically relevant. For both phases, classifications were reviewed and discussed to achieve maximal concordance of classifications across experts. The proposed scoring algorithm was then modified to maximize agreement with the expert interpretation of clinically relevant patient experiences. CONCLUSION: The clinical scoring algorithm for the POMAQ was developed and refined to reflect clinically relevant patient behaviors identified by expert review. Future testing is needed to determine the sensitivity and specificity of this measure.

Symptoms, impacts, and suitability of the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT™) questionnaire in patients with sarcoidosis-associated pulmonary hypertension (SAPH): a qualitative interview study.

BACKGROUND: Sarcoidosis-associated pulmonary hypertension (SAPH) is a prevalent and serious complication of sarcoidosis. No SAPH-specific self-report instruments for assessing SAPH symptoms and their impact on patients are available to date. This study sought to determine whether the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT™) questionnaire is suitable for use in patients with SAPH. METHODS: Patients diagnosed with SAPH participated in qualitative one-on-one telephone interviews to better understand SAPH symptoms and their impacts on patients' lives and to determine the appropriateness of the PAH-SYMPACT™ for use in patients with SAPH. The interviews comprised concept elicitation, completion of the PAH-SYMPACT™, and cognitive debriefing. Interview transcripts were analyzed by content analysis. RESULTS: Eleven patients with SAPH were interviewed between August 2019 and June 2020. In the concept elicitation, all 11 participants endorsed shortness of breath and nine participants (82%) rated it as their "most bothersome or severe" symptom. Impacts endorsed by all 11 participants were difficulty walking uphill or up stairs and difficulty in performing daily activities. Cognitive debriefing indicated that the PAH-SYMPACT™ items were relevant and understandable to most participants and reflected their experiences of SAPH. Participants indicated that no key symptoms or impacts of SAPH were missing. They also reported that the PAH-SYMPACT™ instructions and response options were clear, and that it would be feasible to complete the 11 symptom items and one oxygen use item as part of their daily schedule. CONCLUSIONS: This study suggests the PAH-SYMPACT™ is suitable for assessing symptoms and their impact in patients with SAPH. However, larger longitudinal studies are needed to confirm that it is fit for use in this patient population and that it can be used to reliably detect temporal changes in patients' symptom status. Trial registration Not applicable.

A Review of Interventional Trials in Youth-Onset Type 2 Diabetes: Challenges and Opportunities.

INTRODUCTION: In recent decades, the dramatic rise of obesity among youth in the US has been accompanied by a rise in the prevalence of type 2 diabetes (T2D) in this population. This alarming trend underscores the importance of conducting trials to evaluate new therapies in children with T2D. METHODS: A targeted review of peer-reviewed literature and trials registered on www.clinicaltrials.gov was conducted in January 2021 to identify pharmaceutical interventional studies in youth with T2D. Information regarding enrollment data, study design elements, subjects' baseline characteristics, and key treatment outcomes was documented. RESULTS: Among the 16 clinical studies included in this review, only five appeared to meet projected enrollment targets in < 4 years. Although three other studies met recruitment targets, two took approximately 5 years to complete and the third took nearly 10 years. CONCLUSIONS: Despite legislation requiring evaluation of pharmaceutical treatments in pediatric populations, surprisingly few interventional studies have been conducted in children with T2D. This review highlights that recruitment challenges may be impeding the conduct and completion of interventional studies. Consequently, few pharmaceutical treatments have been proven to be effective and approved for children with T2D. Metformin and liraglutide remain the only non-insulin treatments formally approved in the US for use in this population. More clinical research is needed to support regulatory decision-making as well as treatment decisions for children with T2D in clinical settings. Sponsors and investigators will need to implement strategies for improving trial enrollment as well as work with regulatory agencies to develop novel study designs that may require fewer patients.

Patient Perceptions of and Preferences Between Characteristics of Injectable Diabetes Treatments.

INTRODUCTION: The administration of medications targeting type 2 diabetes mellitus (T2D) has evolved over time. As injection delivery systems continue to evolve, it is necessary to understand patients' perceptions of currently available treatments. The objective of this study was to examine the patient perspective of injectable treatment for T2D and identify characteristics of these treatments that are most important to patients. METHODS: Data were collected via an online survey study with a sample of individuals in the UK and US who were treated for T2D with injectable medication. The survey was designed to elicit perceptions of the treatment process for injectable glucagon-like peptide 1 (GLP-1) receptor agonists and insulin. RESULTS: The sample included 504 participants (251 UK, 253 US). Approximately half (50.4%) were treated with a GLP-1 receptor agonist and half (49.6%) were treated with insulin. Respondents were presented with a list of 17 characteristics of injectable medication and asked to indicate which were most important to them. Respondents most frequently selected confidence in administering the correct dose (n = 300, 59.5%); ease of selecting the correct dose (n = 268, 53.2%); overall ease of using the injection device (n = 239, 47.4%); frequency of injections (n = 223, 44.2%); and ease of carrying the device when necessary to inject away from home (n = 190, 37.7%). Characteristics least frequently cited as important included dose escalation (n = 79, 15.7%); handling the needle (n = 74, 14.7%); connectivity to an electronic device (n = 70, 13.9%); and the time required to prepare and inject each dose (n = 62, 12.3%). CONCLUSION: Results of this survey suggest that patients prioritize some attributes of injectable treatments over others. These findings may have implications for clinical practice and development of injection devices.

Symptoms, impacts, and suitability of the Pulmonary Arterial Hypertension - Symptoms and Impact (PAH-SYMPACT™) questionnaire in patients with chronic thromboembolic pulmonary hypertension (CTEPH): a qualitative interview study.

BACKGROUND: Chronic thromboembolic pulmonary hypertension (CTEPH) is a rare form of pulmonary hypertension caused by blood clots and scar tissue in the blood vessels of the lungs. Health-related quality of life is often significantly impaired in patients with CTEPH. However, a better understanding of how CTEPH symptoms affect patients' lives is needed to optimally assess the impact of the disease and treatment. OBJECTIVES: This qualitative study aimed to better understand the symptoms of CTEPH and how they affect patients' lives, as well as to determine the appropriateness of the Pulmonary Arterial Hypertension - Symptoms and Impact (PAH-SYMPACT™) questionnaire for use in this patient population. METHODS: Adults diagnosed with CTEPH, recruited from two clinical sites in the US, participated in one-to-one qualitative telephone interviews. They described their experience of CTEPH symptoms and the impact these symptoms have on their lives. They also provided feedback on the comprehensibility and relevance of the PAH-SYMPACT™'s instructions, items, and response options. RESULTS: Participants (N = 12) had a mean age of 62.5 years. Two thirds were female and most (83%) had undergone pulmonary endarterectomy and/or balloon pulmonary angioplasty. The most frequently endorsed symptoms were shortness of breath (endorsed by all 12 participants), fatigue (11 participants), and lightheadedness (10 participants). All participants identified shortness of breath as an "extremely important" symptom, and seven participants rated fatigue as "extremely important." The most frequent impacts of CTEPH were on ability to walk quickly (endorsed by all 12 participants), ability to walk up inclines or stairs (11 participants), and ability to carry things (11 participants). The PAH-SYMPACT™ items were relevant to most participants and reflected their experience of CTEPH. All participants indicated that no important CTEPH symptoms were missing from the PAH-SYMPACT™. Overall, the instructions, items, and response options of the PAH-SYMPACT™ were clear and easy to understand. CONCLUSIONS: The symptoms and impacts experienced by patients with CTEPH align with items included in the PAH-SYMPACT™. The PAH-SYMPACT™ appears to be fit for purpose for assessing disease status in patients with CTEPH.

Construct validity and reproducibility of the Prescription Opioid Misuse And Abuse Questionnaire (POMAQ).

OBJECTIVE: The Prescription Opioid Misuse and Abuse Questionnaire was developed to identify prescription opioid abuse and misuse among patients with chronic pain, however, evidence of construct validity and reproducibility is needed. METHODS: Chronic pain patients were recruited from five Department of Defense Military Health System clinics across the United States. Construct validity was examined using subjective clinician-reported and patient-reported measures as well as objective information (e.g. hair/urine drug screens and electronic medical records). Test-retest reliability was assessed across 2 timepoints among a subgroup of patients with stable chronic pain. RESULTS: Of 3,263 screened patients, 938 (28.7%) met eligibility and were enrolled; 809 (86.2%) completed the Prescription Opioid Misuse and Abuse Questionnaire. Construct validity was supported by comparison to other validated questionnaires and hair and urine screens which yielded high agreements with patient reports on the Prescription Opioid Misuse and Abuse Questionnaire. Electronic medical record data supported patients' Prescription Opioid Misuse and Abuse Questionnaire responses regarding physician and emergency room visits and opioid refills. The Prescription Opioid Misuse and Abuse Questionnaire had excellent test-retest reliability; the percentage agreement between the two Prescription Opioid Misuse and Abuse Questionnaire administrations was high (>90%) for most questions. DISCUSSION: Results suggest that the Prescription Opioid Misuse and Abuse Questionnaire is a valid and reproducible tool that can be used to assess the presence of prescription opioid misuse and abuse among patients with chronic pain.

Insight into chronic pain in the United States: descriptive results from the Prescription Opioid Misuse and Abuse Questionnaire (POMAQ) validation study.

OBJECTIVE: A chronic pain patient sample living in the United States who participated in a cross-sectional study to evaluate the validity and reproducibility of the Prescription Opioid Misuse and Abuse Questionnaire is characterized. METHODS: Patients with chronic pain identified through electronic medical records as refilling at least one opioid prescription within the prior 3 months were recruited from five United States Department of Defense Military Health System clinics. Patients completed the Prescription Opioid Misuse and Abuse Questionnaire, Brief Pain Inventory-Short Form, Medical Outcomes Study: 36-item Short Form, and sociodemographic questions online. Clinical characteristics and electronic medical records for 1 year prior to consent were collected. RESULTS: 809 (86.2%) participants completed the Prescription Opioid Misuse and Abuse Questionnaire. Mean (± standard deviation) age was 55.4 ± 12.7 years; the majority female (55.5%) and white (74.8%). Mean duration of chronic pain was 14.7 ± 10.5 years; the most common pain conditions were lower back pain (76.6%), neck or shoulder pain (60.3%), and osteoarthritis (38.7%). The most commonly prescribed opioids were oxycodone (35.7%), tramadol (34.5%), and hydrocodone (26.9%); 54.8% took one opioid, 44.9% took 2 or more opioids. DISCUSSION: Participants' health status was poor; pain severity and interference were moderate. Electronic medical record data revealed high healthcare resource utilization. This chronic pain population was severely impacted by their pain condition(s).

Validity and analysis of the Diabetes Injection Device Preference Questionnaire (DID-PQ).

INTRODUCTION: The Diabetes Injection Device Preference Questionnaire (DID-PQ) was designed to assess patient preference between two non-insulin injection devices. In a recent crossover study, people with type 2 diabetes (T2D) completed the DID-PQ after performing mock injections with two non-insulin injection devices. The purpose of the current analysis was to use these data to assess construct validity of the DID-PQ and demonstrate one way to test whether there is a significant preference for one injection device over another. METHODS: Data were from an open-label, multicenter, randomized, crossover study assessing preference between the dulaglutide and semaglutide injection pens. In addition to the 10-item DID-PQ, people with T2D completed a global item assessing overall preference. DID-PQ responses were compared to the global preference item (percent agreement, Gwet's AC1, prevalence-adjusted and bias-adjusted Kappa [PABAK]). For each item of the DID-PQ, a two-sided binomial test assessed whether the difference in preference was statistically significant. RESULTS: The sample included 310 participants (48.4% female; mean age = 60.0). The DID-PQ had minimal missing data. There was strong concordance (percent agreement > 78%) between the global preference item and all DID-PQ items except item 6, which assesses preference related to needle size (59.7%). The Gwet AC1 and PABAK statistics also indicated strong agreement between the global preference item and all DID-PQ items except item 6. There was a statistically significant difference (p < 0.0001) in preference on every DID-PQ item, with more participants preferring the dulaglutide device. DISCUSSION: Patient preference has been recommended as a "major factor driving the choice of medication" in a consensus report by the American Diabetes Association and the European Association for the Study of Diabetes. Current findings suggest that the DID-PQ may be a useful tool for providing insight into preferences of people with T2D using non-insulin injectable medication.

Assessing patient PREFERence between the dulaglutide pen and the semaglutide pen: A crossover study (PREFER).

AIM: When selecting treatments for type 2 diabetes (T2D), it is important to consider not only efficacy and safety, but also other treatment attributes that have an impact on patient preference. The objective of this study was to examine preference between injection devices used for two weekly GLP-1 receptor agonists. MATERIALS AND METHODS: The PREFER study was an open-label, multicentre, randomized, crossover study assessing patient preference for dulaglutide and semaglutide injection devices among injection-naïve patients receiving oral medication for type 2 diabetes. After being trained to use each device, participants performed all steps of injection preparation and administered mock injections into an injection pad. Time-to-train (TTT) for each device was assessed in a subset. RESULTS: There were 310 evaluable participants (48.4% female; mean age, 60.0 years; 78 participants in the TTT subgroup). More participants preferred the dulaglutide device than the semaglutide device (84.2% vs. 12.3%; P < 0.0001). More participants perceived the dulaglutide device to have greater ease of use (86.8% vs. 6.8%; P < 0.0001). After preparing and using the devices, more participants were willing to use the dulaglutide device (93.5%) than the semaglutide device (45.8%). Training participants to use the dulaglutide device required less time than the semaglutide device (3.38 vs. 8.14 minutes; P < 0.0001). CONCLUSIONS: Participants with type 2 diabetes preferred the dulaglutide injection device to the semaglutide injection device. If patients prefer a device, they may be more willing to use the medication, which could result in better health outcomes. Furthermore, a shorter training time for injection devices may be helpful in busy clinical practice settings.

Psychometric validation of the 1-month recall Uterine Fibroid Symptom and Health-Related Quality of Life questionnaire (UFS-QOL).

BACKGROUND: To evaluate the psychometric characteristics of the 1-month recall Uterine Fibroid Symptom and Health-Related Quality of Life questionnaire (UFS-QOL), including the Revised Activities subscale. METHODS: VENUS I and II were phase III, randomized, double-blind, placebo-controlled trials of ulipristal acetate in women with uterine fibroids (UF) and abnormal uterine bleeding. Women completed the 1-month recall UFS-QOL at baseline and after 12 weeks' treatment. Uterine bleeding was assessed via a daily diary (both studies); the Patient Global Impression of Improvement scale (PGI-I) was completed in VENUS II. Psychometric analyses examined internal consistency reliability and construct validity of the UFS-QOL; confirmatory factor analysis (CFA) compared model fit of the original and Revised Activities subscales. Analyses were conducted separately for VENUS I and II. RESULTS: One hundred and fifty-seven patients in VENUS I and 429 in VENUS II were included. Changes in mean Symptom Severity and health-related quality of life (HRQoL) scale scores indicated symptom burden reductions and HRQoL improvements. Cronbach's alpha coefficients were high at baseline and after 12 weeks' treatment (all ≥0.76, meeting the >0.70 threshold), demonstrating strong internal consistency reliability. Correlations between UFS-QOL scores and bleeding diary responses (range: -0.35 to -0.63), and UFS-QOL scores and PGI-I responses (range: -0.48 to -0.70), ranged from moderate to strong after 12 weeks' treatment (all p < 0.0001). Patients with absence of bleeding or controlled bleeding after 12 weeks' treatment scored significantly better (p < 0.001) on each UFS-QOL scale than patients not achieving those end points, supporting construct validity. CFA confirmed model fit for the Revised Activities subscale. CONCLUSIONS: The 1-month recall UFS-QOL, including the Revised Activities subscale, is a valid, reliable measure to assess UF symptoms and their impact on HRQoL. TRIAL REGISTRATION: ClinicalTrials.gov, NCT02147197 . Registered May 26, 2014; retrospectively registered. ClinicalTrials.gov, NCT02147158 . Registered May 26, 2014; retrospectively registered.

Qualitative analysis and reproducibility assessment of the Scalp Itch Numeric Rating Scale among patients with moderate to severe plaque psoriasis of the scalp.

Background: Pruritus is a prevalent and bothersome symptom of scalp psoriasis. Validated scales assessing scalp itch are needed to evaluate treatment efficacy. Objective: To evaluate comprehensibility and reproducibility of the Scalp Itch Numeric Rating Scale (NRS), a novel scale being used in a phase 3 study of apremilast. Methods: The Scalp Itch NRS, Modified Whole Body Itch NRS, Global Assessment of Psoriasis Severity-Scalp (GAPS-S), and Global Impression of Change-Scalp Itch (GIC-SI) were assessed among patients with moderate to severe scalp psoriasis. Convergent validity and test-retest reliability between two visits (7 ± 3 days apart) were assessed using intra-class and Spearman's correlations. Results: Patients found the Scalp Itch NRS easy to use and understand. Convergent validity (Modified Whole Body Itch NRS Visit 1: rs = 0.71, Visit 2: rs = 0.92, p< .0001; GAPS-S Visit 1: rs = 0.62, Visit 2: rs = 0.63, p< .0001), and consistency with changes (Modified Whole Body Itch NRS: rs = 0.69, p< .0001; GAPS-S: rs = 0.42, p = .0029) were demonstrated. The Scalp Itch NRS showed strong test-retest reliability (intra-class correlation coefficient = 0.87; rs = 0.89). Change scores on the Scalp Itch NRS were consistent with change scores on the GIC-SI. Conclusions: The Scalp Itch NRS is a valid and reproducible measure of scalp itch in patients with moderate to severe scalp psoriasis. ClinicalTrials.gov: NCT03123471.

Psychometric evaluation of the Diabetes Injection Device Experience Questionnaire (DID-EQ) and Diabetes Injection Device Preference Questionnaire (DID-PQ).

BACKGROUND: Previous research has examined patient perceptions of insulin injection devices. However, a range of injectable medications other than insulin are now used to treat type 2 diabetes. No patient-reported outcome (PRO) instruments have been developed taking into account the perceptions of patients using newer injection devices, which are often different from those used in the past. Therefore, the primary purpose of this study was to evaluate a new PRO instrument focusing on patients' experiences with injection devices, including those used for newer treatments such as GLP-1 receptor agonists. METHODS: Patients with T2D treated with non-insulin injectable medications were recruited via advertisements and six clinical sites in the US. All participants completed the draft Diabetes Injection Device - Experience Questionnaire (DID-EQ) and additional measures administered for validity assessment. Participants who had experience with two non-insulin injection devices also completed the draft Diabetes Injection Device - Preference Questionnaire (DID-PQ). Analyses focused on item reduction (item performance, exploratory factor analysis), reliability, and validity. RESULTS: One hundred fourty two patients (mean age = 63.0y; 56.3% female) participated. Item reduction yielded a 10-item version of the DID-EQ, including a 7-item Device Characteristics subscale and three global items assessing satisfaction, ease of use, and convenience of the injection device. The DID-EQ demonstrated good internal consistency reliability (Cronbach's alpha of Device Characteristics subscale = 0.80) and 7-day test-retest reliability (ICCs: 0.92 for Device Characteristics subscale; 0.65 to 0.91 for the three global items). Construct validity was demonstrated via correlations with previously validated instruments (e.g., correlations with the DTSQ treatment satisfaction subscale ranged from 0.56 to 0.60, all p < 0.0001; correlations with the TRIM-D Device ranged from 0.63 to 0.77, all p < 0.0001). Descriptive analyses of the DID-PQ were conducted with a subset of 27 participants who were able to use it to compare two devices. CONCLUSIONS: This psychometric evaluation supports the reliability and validity of the DID-EQ, while providing initial information on the performance of the DID-PQ. These brief questionnaires complement measures of treatment efficacy and provide a more thorough picture of patients' experiences with non-insulin injectable treatments for type 2 diabetes.

Patient perceptions of injection devices used with dulaglutide and liraglutide for treatment of type 2 diabetes.

OBJECTIVE: Liraglutide and dulaglutide have demonstrated similar glycemic efficacy and safety. However, they differ in treatment administration and injection devices. The purpose of this study was to examine and compare patient perceptions of the injection devices used with liraglutide and dulaglutide. METHODS: Patients with type 2 diabetes treated with liraglutide or dulaglutide were recruited from across the US. Patients completed the Diabetes Injection Device Experience Questionnaire (DID-EQ) to rate their current injection device. Patients who had experience with both treatments also completed the Diabetes Injection Device Preference Questionnaire (DID-PQ) to report preferences between the two devices. ANCOVAs were conducted to compare DID-EQ scores between dulaglutide and liraglutide patients, while controlling for covariates. Descriptive statistics are presented for preferences reported on the DID-PQ. RESULTS: A total of 404 patients were recruited from 49 states (mean age = 60.7 years; 54.0% female; 204 liraglutide; 200 dulaglutide). Mean DID-EQ item scores for both treatments were high, ranging from 3.48 to 3.90 on a 4 point scale. ANCOVAs found significantly higher scores for dulaglutide than liraglutide on DID-EQ global items assessing ease of use (3.82 vs. 3.73, p = .040) and convenience (3.79 vs. 3.66, p = .004). Among the 58 patients who had used both devices, more patients reported a preference for the dulaglutide device than the liraglutide device on every item of the DID-PQ. CONCLUSIONS: High DID-EQ scores indicate positive perceptions of both the liraglutide and dulaglutide injection devices. The dulaglutide device was associated with slightly higher scores for ease of use and convenience than the liraglutide device.

Psychometric validation of the electronic chronic pain questions (eCPQ) in a primary care setting.

OBJECTIVE: Collecting data that helps evaluate different types of pain may improve physicians' decision-making with regard to treatment selection and on-going monitoring of patients. To date, no chronic pain assessments have been widely implemented in primary care. The aim of this study was to psychometrically validate the electronic Chronic Pain Questions (eCPQ) in a primary care setting. RESEARCH DESIGN AND METHODS: All men and women ≥18 years arriving at two similar primary care clinics in southeastern Michigan were invited to participate. Clinic staff verbally administered the eCPQ to patients and recorded their answers into the electronic medical record (EMR) prior to physician consultation with results available for physician review. Concurrent validity was assessed using Spearman correlations between eCPQ and patient-completed ancillary measures. Known-group validity was assessed by stratifying patients on self-reported chronic pain as well as by pain diagnosis (i.e. ICD-9 codes). To compare patients with chronic pain versus no chronic pain t-tests and chi-square tests were performed. Reproducibility was assessed between interviewer- and self-administration over time. RESULTS: A total of 534 patients were invited to participate and 455 patients consented to take part in the study (85.2% response rate); 395 patients had analyzable eCPQ data; 70.1% were Caucasian; 68.1% female; mean age was 43.4; 52.7% (n = 208) self-reported chronic pain. Correlations between eCPQ and ancillary measures supported concurrent validity. Excellent discrimination between groups was evidenced based on self-reported chronic pain and ICD-9 diagnosis. Patients with self-reported chronic pain reported significantly (p < .0001) higher pain ratings and greater interference with usual activities, sleep, and mood than those without chronic pain. Test-retest reliability between modes (interviewer- vs. self-administration) was excellent as was reproducibility based on self-administration of the eCPQ at two separate time points. Key limitations: Discriminant validity was determined by comparing participants based on ICD codes. Utilizing ICD codes to identify individuals with chronic pain may not be a reliable approach as it is dependent upon providers accurately and consistently entering chronic pain diagnoses in the EMR. CONCLUSIONS: The eCPQ has sound psychometric measurement properties, including concurrent validity, discriminant validity, and reproducibility. The eCPQ appears to be useful to identify patients with chronic pain and to assess and monitor symptoms over time.

Discriminating between neuropathic pain and sensory hypersensitivity using the Chronic Pain Questions (CPQ).

OBJECTIVE: The Chronic Pain Questions (CPQ) were developed for clinical use with the aim of supporting primary care physicians in the screening, assessment and monitoring of patients with chronic pain. the purpose of this study was to examine the ability of the cpq CPQ to discriminate between patients with neuropathic pain (nep) versus those with sensory hypersensitivity (sh). METHODS: Adult men and women with a diagnosis of a NeP or SH condition were recruited from 5 clinical sites across the United States. Participants completed a series of self-administered questionnaires, including the CPQ. Continuous variables were compared between groups with independent t-tests; categorical variables were compared with chi-square analyses. A series of exploratory logistic regressions were performed to discern optimal screening criteria for SH using CPQ responses. RESULTS: 98 participants, 68 with physician-confirmed diagnoses of SH and 30 with NeP, participated. 81.6% were female, 73.5% Caucasian, and mean (± SD) age was 58.4 ± 12.6 years. SH participants included significantly more females compared to the NeP group (94.1% vs. 53.3%). Differences in CPQ responses between groups were statistically significant for six of the 14 CPQ items with SH participants having significantly lower scores on specific pain quality questions and significantly higher scores on trouble thinking/remembering (5.3 ± 3.5 vs. 3.0 ± 2.8) and sensitivity to lights/noises/smells (4.8 ± 3.5 vs. 2.7 ± 3.0). No significant differences were found between groups for chronic pain intensity, pain made worse with touch, pain limited to joints, or pain interference with usual activity, sleep or mood. Logistic regression analyses revealed strong c indices (≥0.89) for all models. Consistent findings demonstrated that younger age, female gender, and scores ≥6 for the CPQ question on sensitivity to lights/noises/smells were all predictive of SH. CONCLUSIONS: The CPQ can help differentiate between patients with NeP and SH. More research is warranted.

Development of the chronic obstructive pulmonary disease morning symptom diary (COPD-MSD).

BACKGROUND: The morning tends to be the most difficult time of day for many patients with chronic obstructive pulmonary disease (COPD) when symptoms can limit one's ability to perform even simple activities. Morning symptoms have been linked to higher levels of work absenteeism, thereby increasing the already substantial economic burden associated with COPD. A validated patient-reported outcome (PRO) instrument designed to capture morning symptoms will allow for a more comprehensive approach to the evaluation of treatment benefit in COPD clinical trials. METHODS: A qualitative interview study was conducted among a sample of symptomatic adults with COPD. Concept elicitation interviews (n = 35) were conducted to identify COPD morning symptoms, followed by cognitive interviews (n = 21) to ensure patient comprehension of the items, instructions and response options of the draft COPD Morning Symptom Diary (COPD-MSD). All interview transcript data were coded using ATLAS.ti software for content analysis. RESULTS: Mean age of the concept elicitation and cognitive interview sample was 65.0 years (±7.5) and 62.3 years (±8.3), respectively. The study sample represented the full range of COPD severity (Global Initiative for Chronic Lung Disease [GOLD] classifications I-IV) and included a mix of racial backgrounds, employment status and educational achievement. During the concept elicitation interviews, the three most frequently reported morning symptoms were shortness of breath (n = 35/35; 100 %), phlegm/mucus (n = 31/35; 88.6 %), and cough (n = 30/35; 85.7 %). A group of clinical and instrument development experts convened to review the concept elicitation data and develop the initial 32-item draft COPD-MSD. Cognitive interviews indicated subjects found the draft COPD-MSD to be comprehensive, clear, and easy to understand. The COPD-MSD underwent minor editorial revisions and streamlining based on cognitive interviews and input from the experts to yield the final 19-item daily diary. CONCLUSIONS: This study supports the content validity of the new COPD-MSD and positions the diary for quantitative psychometric testing.