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Background: To capture the broad range of treatment burden issues experienced by adolescent and adult people with hemophilia (PWH), the Hemophilia Treatment Experience Measure (Hemo-TEM) was developed. We describe the development of this new hemophilia-specific patient-reported outcome (PRO) measure including concept elicitation, cognitive debriefing, and psychometric validation. Results: Concept elicitation interviews were conducted with 5 clinical experts and 30 adult PWH in the United States (US). The qualitative analysis of these interviews and a review of the literature informed the PRO measure development. The project team reviewed concept endorsement rates and generated a 27-item preliminary version of the Hemo-TEM. Cognitive debriefing interviews were conducted to ensure participant understanding and item relevance in samples of (adolescent (n = 20) and adult (n = 14)) PWH in the US. The refined, validation-ready version of the Hemo-TEM included 30 items. Lastly, data from 3 clinical trials comprised the 4 analysis sets used for the psychometric validation with a sample size of N = 88. Item reduction dropped 4 items resulting in a final 26-item measure. Factor analysis generated 5 domains in the Hemo-TEM [injection difficulties (3 items), physical impact (6 items), treatment bother (7 items), interference with daily life (4 items), and emotional impact (6 items)] and a total score. All scores were reliable [internally consistent (0.84–0.88)]. For convergent validity, with the exception of one domain, all hypothesized associations were met. Preliminary sensitivity to change effect sizes were between − 0.30 and − 0.70. Meaningful change thresholds ranged from 6 points (physical impact and emotional impact) to 10 points (treatment bother) with 8 points for the Hemo-TEM total score. Conclusions: Findings from the concept elicitation, cognitive debriefing, and psychometric validation phases provide evidence that the Hemo-TEM is a well-designed, valid, and reliable measure of the burden of hemophilia treatment, including treatment impact on adolescent and adult PWH. [ABSTRACT FROM AUTHOR]

Introduction: In a randomized controlled trial, we previously investigated if melanoma patients receiving checkpoint inhibitors had fewer severe immune-related adverse events (irAEs) when they reported symptoms using electronic patient-reported outcomes (ePRO) with triggered alerts as an add-on to standard care, compared to standard care alone. The aim of this study is to examine between-group differences in health-related quality of life (HRQoL) and associations between irAEs severity and HRQoL. Methods: The study population of 138 patients completed the EuroQol EQ-5D-5L Index and FACT-M questionnaires at baseline and weeks 24 and 48. We analyzed HRQoL from all patients who completed at least one questionnaire. Missing FACT-M items were imputed following existing guidelines. Results: There was no difference in HRQoL at baseline as measured EQ-5D-5L between the intervention and the control group. Between baseline and 48 weeks, mean EQ-5D-5L scores were unchanged among patients in the intervention group (p = 0.81) but decreased significantly among patients in the control group (p = 0.03). Consequently, patients in the intervention group had higher mean scores than those in the control group (p = 0.05) at 48 weeks. Mean FACT-M scores did not differ significantly between the two groups at any of the time points. There were observed no between-group differences in mean EQ-5D-5 and mean FACT-M scores between patients with severe irAEs and patients who had none. Conclusion: Melanoma patients receiving CPIs who self-reported irAEs using ePRO with triggered alerts as a supplement to standard care maintained their HRQoL compared to patients who received standard care alone. Patients in the intervention group had a significantly better HRQoL measured by EQ-5D-5L than controls 48 weeks after baseline. The results suggest that including ePRO in standard care increases melanoma patients´ well-being. Further and larger studies are needed to confirm this finding and examine the impact of severe irAEs on cancer patients' HRQoL. Trial registration: Clinicaltrials.gov NCT03073031 Registered 8 March 2017, Retrospectively registeredhttps://clinicaltrials.gov/. [ABSTRACT FROM AUTHOR]

Background: Although the incidence, severity and mortality of Clostridioides (Clostridium) difficile infection (CDI) have been increasing, patients' quality of life changes resulting from CDI have not been studied thoroughly. This study aimed at exploring the consequences of CDI on quality of life through patients' perspective. Methods: An observational, cross-sectional study involving 350 participants with a self-reported CDI diagnosis was conducted through an online self-administered survey. Participants were grouped into those who had active disease ("Current CDI") and those who had a history of CDI ("Past CDI"). Results: One hundred fifteen participants (33%) reported Current CDI and 235 (67%) reported Past CDI. A large majority of participants admitted that their daily activities were impacted by the infection (93.9% and 64.7% of Current and Past CDI respondents respectively, p < 0.05). Physical and psychological consequences of CDI were experienced by 63.5% and 66.1% of participants with active CDI. Despite the infection being cleared, these consequences were still frequently experienced in Past CDI cohort with similar rates (reported by 73.2% of respondents for both, physical consequences p = 0.08; psychological consequences p = 0.21). After the infection, 56.6% of respondents noted that post-CDI symptoms remained; 40.9% believed they would never get rid of them. Conclusions: While the societal burden of CDI is well described in the literature, our study is one of the first aimed at understanding the major burden of CDI on quality of life. Our results highlight the long-lasting nature of CDI and further reinforce the need for enhanced therapeutics in the prevention and treatment of this devastating infection. [ABSTRACT FROM AUTHOR]

Study design:Survey of expert opinion, feedback and final consensus.Objective:To describe the development and the variables included in the International Spinal Cord Injury (SCI) Spinal Interventions and Surgical Procedures Basic Data set.Setting:International working group.Methods:A committee of experts was established to select and define data elements. The data set was then disseminated to the appropriate committees and organizations for comments. All suggested revisions were considered and both the International Spinal Cord Society and the American Spinal Injury Association endorsed the final version.Results:The data set consists of nine variables: (1) Intervention/Procedure Date and start time (2) Non-surgical bed rest and external immobilization, (3) Spinal intervention-closed manipulation and/or reduction of spinal elements, (4) Surgical procedure-approach, (5) Date and time of the completion of the intervention or surgical closure; (6) Surgical procedure-open reduction, (7) Surgical procedure-direct decompression of neural elements, and (8 and 9) Surgical procedure-stabilization and fusion (spinal segment number and level). All variables are coded using numbers or characters. Each spinal intervention and procedure is coded (variables 1 through 7) and the spinal segment level is described (variables 8 and 9). Sample clinical cases were developed to illustrate how to complete it.Conclusion:The International SCI Spinal Interventions and Surgical Procedures Basic Data Set was developed to facilitate comparisons of spinal interventions and surgical procedures among studies, centers and countries. [ABSTRACT FROM AUTHOR]

To determine the influence of patient religiosity on the outcome of treatment of hepatitis C infection, a prospective, blinded, cohort study was performed on hepatitis C-infected patients categorized as 'higher religiosity' and 'lower religiosity' based on responses to a religiosity questionnaire. Comparisons were made between high and low religiosity patients on demographics, pre-treatment laboratory values, and response to treatment. Eighty-seven patients with complete questionnaires were placed in either higher (38) or lower (49) religiosity cohort. The patients (60% female) were ethnically diverse: African-American 39%; Hispanic 31%; white 29%. African-American race ( P = 0.001) and female gender ( P = 0.026) were associated with higher religiosity. The frequency of being offered treatment, accepting treatment, and completing treatment was similar in both religiosity cohorts ( P = 0.234, 0.809, 0.367). Fifty-six patients completed the 24- or 48-week treatment with peginterferon and ribavirin. Depression was more frequent in the low religiosity group (38.2% vs. 4.6%, P = 0.005). Sustained viral response rate at 3-6-month post-therapy was similar in the higher (50%) and lower (57.6%) religiosity cohorts ( P = 0.580; n = 55). Logistic regression modeling revealed that males having higher religiosity gave greater odds of SVR than those with lower religiosity (OR 21.3; 95% CI 1.1-403.9). The level of religiosity did not affect the decision to begin treatment for chronic HCV infection and was not associated with a better treatment outcome. A higher level of religiosity was associated with less depression among patients. [ABSTRACT FROM AUTHOR]

The objectives of this study are to assess the efficacy and safety of frovatriptan, and rizatriptan in the subgroup of women with menstrually related migraine of a multicenter, randomized, double blind, cross-over study. Each patient received frovatriptan 2.5 mg or rizatriptan 10 mg in a randomized sequence: after treating 3 episodes of migraine in not more than 3 months with the first treatment, the patient had to switch to the other treatment. Menstrually related migraine was defined according to the criteria listed in the Appendix of the last IHS Classification of Headache disorders. 99 out of the 125 patients included in the intention-to-treat analysis of the main study were of a female gender: 93 had regular menstrual cycles and were, thus, included in this analysis. A total of 49 attacks classified as menstrually related migraine were treated with frovatriptan and 59 with rizatriptan. Rate of pain relief at 2 h was 58% for frovatriptan and 64% for rizatriptan ( p = NS), while rate of pain free at 2 h was 31 and 34% ( p = NS), respectively. At 24 h, 67 and 81% of frovatriptan-treated, and 61 and 74% of rizatriptan-treated patients were pain free and had pain relief, respectively ( p = NS). Recurrence at 24 h was significantly ( p < 0.01) lower with frovatriptan (10 vs. 32% rizatriptan). Frovatriptan was as effective as rizatriptan in the immediate treatment of menstrually related migraine attacks while showing a favorable sustained effect with a lower rate of migraine recurrence. These results need to be confirmed by randomized, double-blind, prospective, large clinical trials. [ABSTRACT FROM AUTHOR]

Study design:A survey administered to 66 individuals with spinal cord injury (SCI) implementing a choice-based conjoint (CBC) analysis. Six attributes with three levels each were defined and used to generate choice sets with treatment scenarios. Patients were asked to choose the scenario that they preferred most.Objectives:To determine the utility weights for treatment characteristics as well as the overall preference for the three types of neural prostheses (NP), that is Brindley, rhizotomy-free Brindley, and pudendal nerve stimulation. Earlier studies have revealed the importance of restoration of bladder function, but no studies have been performed to determine the importance of NP features.Setting:Two academic affiliated medical systems' SCI outpatient and inpatient rehabilitation programs, Cleveland, OH.Methods:CBC analysis followed by multinomial logit modeling. Individual part-worth utilities were estimated using hierarchical Bayes.Results:Side effects had the greatest significant impact on subject choices, followed by the effectiveness on continence and voiding. NPs with rhizotomy-free sacral root stimulation were preferred (45% first choice) over pudendal afferent nerve stimulation (39% second choice) and sacral root stimulation with rhizotomy (53% third choice). Almost 20% did not want to have an NP at all times.Conclusion:CBC has shown to be a valuable tool to support design choices. The data showed that persons would prefer a bladder NP with minimally invasive electrodes, which would give them complete bladder function, with no side effects and that can be operated by pushing a button and they do not have to recharge themselves. [ABSTRACT FROM AUTHOR]

Background: The EORTC 24971/TAX 323, a phase III study of 358 patients with unresectable locoregionally advanced squamous cell carcinoma of the head and neck, showed an improved progression-free and overall survival (OS) with less toxicity when docetaxel (T) was added to cisplatin and 5-fluorouracil (PF) for induction and given before radiotherapy (RT). The impact of the addition of docetaxel on patients' health-related quality of life (HRQOL) and symptoms was investigated.Methods: HRQOL was assessed at baseline, at end of cycle 2, and 4, 6, and 9 months after completion of RT using the European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire C30 (QLQ-C30) and the EORTC QLQ Head and Neck Cancer-Specific Module (EORTC QLQ-H&N35). The primary HRQOL scale was global HRQOL per protocol.Results: Compliance to HRQOL assessments was 97% at baseline, but dropped to 54% by 6 months. Data were analysed up to 6 months. There was a trend towards improved global HRQOL during the treatment period. At 6 months after the end of RT, global HRQOL was higher in the TPF arm than in the PF arm, but the low compliance does not allow to draw definitive conclusions. Swallowing and coughing problems decreased more in the TPF arm than in the PF arm at the end of cycle 2, but to a limited extent.Conclusion: Induction chemotherapy with TPF before RT not only improves survival and reduces toxicity compared with PF but also seems to improve global HRQOL in a more sustainable manner. [ABSTRACT FROM AUTHOR]

Objective: To assess whether Medicare fee-for-service beneficiaries with depression and diabetes had a higher mortality rate over a 2-year period compared with beneficiaries with diabetes alone.Design: Evidence of depression was based on a physician diagnosis or self-reported prescription of an antidepressant in the year prior to screening, or a score of > or = 3 on the Patient Health Questionnaire two-item questionnaire. Mortality was assessed bi-monthly by checking Medicare claims and eligibility files or from information from telephone contact with the participant's family. Cox proportional hazard regression models were used to calculate adjusted hazard ratios of death in depressed versus nondepressed beneficiaries with diabetes.Participants: A total of 10,704 beneficiaries with diabetes enrolled in a disease management program were surveyed with a health assessment questionnaire and followed over a two-year period.Main Results: Comorbid depression in Medicare beneficiaries with diabetes participating in a disease management program was associated with an increased risk for all-cause mortality over a two-year period of approximately 36% to 38%, depending on the definition of depression that was used. No significant increase in rates of cause-specific mortality from macrovascular disease were found in depressed versus nondepressed beneficiaries.Conclusion: Among a large Medicare cohort of fee-for-service beneficiaries with diabetes, comorbid depression was associated with an increase in all-cause mortality over a two-year period. Future research will be required to determine whether the increase in mortality associated with depression is due to potential behavioral mediators (i.e., smoking, poor adherence to diet) or physiologic abnormalities (i.e., hypothalamic-pituitary axis dysregulation) associated with depression. [ABSTRACT FROM AUTHOR]

Background: Little research investigates the role of patient-physician communication in understanding racial disparities in depression treatment.Objective: The objective of this study was to compare patient-physician communication patterns for African-American and white patients who have high levels of depressive symptoms.Design, Setting, and Participants: This is a cross-sectional study of primary care visits of 108 adult patients (46 white, 62 African American) who had depressive symptoms measured by the Medical Outcomes Study-Short Form (SF-12) Mental Component Summary Score and were receiving care from one of 54 physicians in urban community-based practices.Main Outcomes: Communication behaviors, obtained from coding of audiotapes, and physician perceptions of patients' physical and emotional health status and stress levels were measured by post-visit surveys.Results: African-American patients had fewer years of education and reported poorer physical health than whites. There were no racial differences in the level of depressive symptoms. Depression communication occurred in only 34% of visits. The average number of depression-related statements was much lower in the visits of African-American than white patients (10.8 vs. 38.4 statements, p = .02). African-American patients also experienced visits with less rapport building (20.7 vs. 29.7 statements, p = .009). Physicians rated a higher percentage of African-American than white patients as being in poor or fair physical health (69% vs. 40%, p = .006), and even in visits where depression communication occurred, a lower percentage of African-American than white patients were considered by their physicians to have significant emotional distress (67% vs. 93%, p = .07).Conclusions: This study reveals racial disparities in communication among primary care patients with high levels of depressive symptoms. Physician communication skills training programs that emphasize recognition and rapport building may help reduce racial disparities in depression care. [ABSTRACT FROM AUTHOR]

Background: Few studies have evaluated the association between patient expectations for recovery and clinical outcomes, and no study has evaluated whether asking patients to choose their therapy modifies such an association.Objective: To evaluate the association between patients' expectations and functional recovery in patients with acute low back pain (LBP), and to determine whether that association is affected by giving patients choice of therapy.Design and Participants: A secondary analysis of a randomized controlled trial comparing usual care alone to usual care plus choice of chiropractic, acupuncture, or massage in 444 adults with acute LBP, lasting less than 21 days.Measurements and Main Results: Primary outcome was functional disability (Roland score) at 5 and 12 weeks. Patients' general expectations for improvement were associated with improvement in functional status (beta = 0.96, 95% CI = 0.56, 1.36). A 1-point increase in general expectations was associated with a 0.96-point improvement in Roland score. The association of expectation with outcome was 2-3 times greater in the usual care group than the choice group. However, these differences did not reach statistical significance.Conclusions: In patients with acute LBP, higher expectations for recovery are associated with greater functional improvement. Eliciting patient expectations for improvement may be a simple way to identify patients with the highest (or lowest) likelihood of experiencing functional improvement. Incorporating questions about patient expectations in future trials may clarify the role of this important correlate of clinical outcomes. [ABSTRACT FROM AUTHOR]

Background: No study has investigated the associations of migraine with pain symptoms over a ten-year period among outpatients with major depressive disorder (MDD). This study aimed to investigate this issue.Methods: At baseline, the study enrolled 290 outpatients with MDD and followed-up the patients at six-month, two-year, and ten-year time points. MDD and anxiety comorbidities were diagnosed using the Structured Clinical Interview for DSM-IV-text revision. Migraine was diagnosed based on the International Classification of Headache Disorders. The bodily pain subscale of the Short Form 36 (SF-BP) and the pain subscale (PS) of the Depression and Somatic Symptoms scale were also used. Generalized Estimating Equation models were employed to investigate the longitudinal impacts of migraine on pain symptoms.Results: MDD patients with migraine had lower SF-BP and higher PS scores than those without. Depression, anxiety, and headache indices were significantly correlated with SF-BP and PS scores. The higher the frequency of migraine, the more often patients suffered from pain symptoms. Patients with migraine at all investigated time points suffered from pain symptoms most of the time (ranging from 60.0% to 73.7%) over the 10 years. After controlling for depression and anxiety, migraine was independently associated with a decreased SF-BP score (by 8.93 points) and an increased PS score (by 1.33 points).Conclusion: Migraine was an important comorbidity associated with greater severities of pain symptoms during long-term follow-up. Migraine treatment should be integrated into the treatment of depression to improve pain symptoms and quality of life in the pain dimension. [ABSTRACT FROM AUTHOR]