Self-administered Patient-reported Outcome Research Articles

The Revised Reflux Symptom Index (R-RSI): development, internal and external validation study.

This study proposes a revised version of the Reflux Symptom Index (R-RSI), a seventeen-item questionnaire that was revised to increase the suspicion of laryngo-pharyngeal reflux disease (LPRD). Internal validation involved 213 participants, comprising160 subjects without a previous LPRD diagnosis and 53 subjects with a self-reported previous diagnosis of LPRD with or without gastro-esophageal reflux disease (GERD). Test-retest reliability and internal consistency were calculated. For the external validation, 56 patients (independent from the previous cohort) were enrolled to explore the R-RSI Screening Properties and determine a cut-off using 24-h MII-pH as the gold standard. R-RSI test-retest reliability was high, both for the total score (ICC: 0.970) and for each item (ranging from 0.876 to 0.980). Cronbach's alpha was 0.910, indicating excellent internal consistency of the questionnaire. Participants with a previous self-reported diagnosis scored significantly higher (mean 24.94 ± 7.4; median 26, IQR 20-29) than those without a previous diagnosis (mean 4.66 ± 5.3; median 4, IQR 1-6) (p-value < 0.0001). Participants with both previous LPRD and GERD diagnoses had higher scores (27.20 ± 7.8) compared to those with only LPRD (21.77 ± 5.5) (p-value=0.003). Using 24-h MII-pH diagnosis as a gold standard, the optimal R-RSI cut-off point was determined to be 18, with a sensitivity of 84.5% and a specificity of 81.8%, positive predictive value of 95% and negative predictive value of 60%. Our results suggest that the R-RSI may be useful to suspect LPRD, with or without GERD. The R-RSI is a self-administered patient-reported outcome questionnaire that demonstrates excellent reliability and high screening properties. Employing a cut-off of ≥18 the R-RSI can assist in diagnosing and monitoring LPRD.

Cross-cultural adaptation, validation and psychometric evaluation of the International Hip Outcome Tool 12 (iHOT12) to Hebrew

BackgroundThe “International Hip Outcome Tool 12” (iHOT12) is a self-administered patient-reported outcome tool for measuring health-related quality of life and physical functioning in young and active patients with hip pathology. Since the iHOT12 has become widely used, we sought to translate and validate it for Hebrew-speaking populations. The aims of this study were: (1) To translate and culturally adapt the iHOT12 into Hebrew using established guidelines. (2) To test the new Hebrew version for validity, and (3) reliability.MethodsThe iHOT12 was translated and culturally adapted from English to Hebrew (iHOT12-H) according to the COSAMIN guidelines. For validity, the iHOT12-H and Western Ontario and McMaster universities osteoarthritis index (WOMAC) were completed by 200 patients with hip pathology. Exploratory factor analysis was used to assess structural validity. Subsequently, 51 patients repeated the iHOT12-H within a 2-week interval. Intraclass Correlation Coefficient (ICC), Cronbach alpha, and Standard Error of Measurement (SEM) were calculated to assess reliability.ResultsConstruct validity: iHOT12-H correlated strongly to the WOMAC scores (r = -0.82, P < 0.001, Spearman). Factor analysis revealed a two-factor structure. Cronbach’s alpha was 0.953 confirming internal consistency to be highly satisfactory. Test–retest correlation of the iHOT12-H was excellent with an ICC = 0.956 (95% CI 0.924–0.974). There was no floor or ceiling effect.ConclusionThe iHOT12 Hebrew version has excellent reliability, good construct validity and can be used as a measurement tool for physical functioning and quality of life in young, physically active patients with hip pathology. This study will serve Israeli researchers in evaluating treatment effectiveness for these patients. Moreover, it will also enable multinational cooperation in the study of hip pathology.

Association of illness perception and alexithymia with fatigue in hemodialysis recipients: a single-center, cross-sectional study

Fatigue in hemodialysis recipients interferes with daily activities and renal rehabilitation, and its underlying causes and treatment remain unclear. Psychological factors, like illness perceptions and alexithymia, cause fatigue in other diseases; however, their contribution to hemodialysis-related fatigue is unknown. This cross-sectional study included 53 hemodialysis recipients. To assess participants’ fatigue, we used a self-administered patient-reported outcome questionnaire whose items have shown correlation with those of established scales, such as the Profile of Mood States and Visual Analogue Scales. The associations among the scores of the revised Illness Perceptions Questionnaire (IPQ-R), Toronto Alexithymia Scale (TAS-20), and Hospital Anxiety and Depression Scale and fatigue were analyzed using bivariable and multivariable analyses. Patients with fatigue had significantly higher median scores for the IPQ-R subscales “Identity” and “Negative emotional representation about illness” than those without fatigue, suggesting the association of specific illness perception with fatigue. Median scores for the TAS-20 subscale “Difficulty identifying feelings” were also significantly higher among fatigued patients, suggesting the association of alexithymia with fatigue. Depression was not associated with fatigue. Multivariable logistic regression revealed the association of a high “Identity” score with the risk of fatigue (adjusted odds ratio, 1.32; 95% confidence interval, 1.00–1.73; P = 0.04), while there were no significant association between a high “Difficulty identifying feelings” score and the risk of fatigue (adjusted odds ratio, 1.09; 95% confidence interval, 0.95–1.24). Specific illness perception and alexithymia were slightly associated with hemodialysis-related fatigue. Cognitive-behavioral therapy for these conditions could reduce fatigue and promote renal rehabilitation.

Psychometric evaluation of a patient-reported outcomes instrument for congenital thrombotic thrombocytopenic purpura

BackgroundCongenital thrombotic thrombocytopenic purpura (cTTP) is an ultra-rare, life-threatening hereditary disorder that causes patients to experience significant morbidity and decreased health-related quality of life (HRQoL). A cTTP disease-specific patient-reported outcome (PRO) instrument that is reflective of patients’ experiences with the disorder does not currently exist. The objective of this study was to evaluate and validate the psychometric properties of the Congenital Thrombotic Thrombocytopenic Purpura–Patient Experience Questionnaire (cTTP-PEQ), developed using a literature review, interviews with expert clinicians, and qualitative concept elicitation and cognitive debriefing interviews.MethodsThis prospective, observational study (NCT03519672) was conducted with patients diagnosed with cTTP currently receiving treatment. Patients were enrolled through investigator sites and direct-to-patient recruitment. Individuals completed electronic self-administered PRO measures, including the cTTP-PEQ, at baseline and Day 14 (+ up to 10 days). The cTTP-PEQ consisted of five multi-item domains (Pain/Bruising, Cognitive Impairment, Visual Impairment, Mood, Treatment Burden) and three single-item domains (Fatigue, Headache, Activity Limitation), and assessed symptoms and impact of cTTP in the previous 24 h, 7 days, and 2 weeks. Convergent and discriminant validity were evaluated using Spearman’s rank correlation coefficients. Known-groups validity was assessed between patient groups separated by Patient Global Impression of Severity (PGI-S; normal vs. mild/moderate/severe). Internal reliability was assessed using Cronbach’s alpha. Test–retest reliability was assessed using intraclass correlation coefficients (ICCs).ResultsThirty-six patients participated in this study. Convergent validity was confirmed with high-to-moderate correlations (r ≥ 0.4) for 12/15 hypothesized relationships between pairs of domains and/or total scores. Discriminant validity was confirmed with low correlations (r < 0.3) observed for 5/7 hypothesized relationships. Known-groups validity was confirmed with significant differences (p ≤ 0.05) in mean cTTP-PEQ scores between the two PGI-S groups for most domains and items at both timepoints. Cronbach’s alpha was 0.88 at baseline and 0.91 at Day 14, confirming internal consistency of the instrument. Test–retest reliability was also confirmed with a high ICC (0.96).ConclusionThis study validates the psychometric properties of the novel cTTP-PEQ for use in research and clinical practice to assess HRQoL among patients with cTTP. This instrument will be particularly useful when assessing cTTP disease burden and the impact of new treatments.

Outcomes of Patellar Replacement Vs Patellar Neurectomy in Total Knee Replacement

Objectives: To compare the outcomes of patellar replacement and patellar neurectomy in Patients undergoing Total Knee Replacement . Materials &amp; Methods: The design of this study was a cross sectional study design and this study was conducted in DHQ Teaching Hospital Gujranwala. Total 30 Patients were enrolled in this study and they were divided into two groups. Group A constitutes of 15 Patients underwent Post tibial neurectomy. And Group B consisting of 15 Patients went through Tibial Resection. All the patients have been assessed through the Tinel test on the painful area and self-administered Patient-Reported Outcome Measurements (PROMs): Patellar replacement also known as patellofemoral arthroplasty, is a surgical procedure that is performed in conjunction with total knee replacement (TKR) to relieve pain in the knee joint caused by patellofemoral arthritis or other conditions affecting the patella. All the patients of Group B were given anesthesia to numb the area. An incision was made in front of the knee to expose the patella and surrounding structures. Removal of the patella: The patella was carefully removed from the knee joint. The underside of the patella was reshaped to fit a prosthetic component that was implanted in its place. The patellar component was attached to the reshaped surface of the patella using bone cement. The patella was repositioned in the knee joint and attached to the femur using sutures. The incision was closed using sutures or staples. The goal of patellar replacement is to reduce pain and improve function in the knee joint. All the group A participants went through Patellar neurectomy. Patellar neurectomy is a surgical procedure that involves cutting or removing the nerve endings that supply sensation to the patella. Results: Total 30 Patients participated in this study. In Group A =15 Patients who underwent patellar Neurectomy and 15 Patients in Group B who experienced Tibia Resection. Total 5 months follow up was done. Group B participants showed excellent Results. NRS score decreased to 0 in Group B. While in patellar Neurectomy group A two patients experienced still moderate pain, Remaining 13 patients although did not experienced pain but felt numbness on limbs. Practical Implication: Patellar neurectomy involves cutting or removing the nerve endings that supply sensation to the patella, interrupting the transmission of pain signals from the patella to the brain. Although it is less time consuming but can cause numbness or altered sensation in the skin over the patella, which may affect the patient's quality of life Does not address underlying patellar instability or misalignment, which can still cause pain and functional limitations Can be associated with a risk of complications such as neuroma, infection, stiffness. Conclusion: Patellar Resection with TKA has better outcomes than Patellar Neurectomy. Patellar denervation with electrocautery is effective in reducing pain and opioid consumption after total knee replacement. Postoperative range of motion is significantly better in patellar denervation group, which means that patellar denervation significantly improves clinical outcome Keywords: Total Knee Replacement, Patellar Replacement, Neurectomy, Neurectomy, Tibia Resection, Pain, Prosthesis

Transcultural German translation of the reflux symptom score-12 questionnaire

In addition to clinical and instrumental methods as well as medical history, questionnaires are well-established tools in the diagnosis of laryngopharyngeal reflux (LPR). Recently, the reflux symptom score (RSS) as well as the short version of it, the RSS-12, which are self-administered patient-reported outcome questionnaires, have been published as an alternative to the well-established reflux symptom index (RSI). The RSS-12 assesses severity and frequency of LPR symptoms as well as the impact on quality of life and has been shown to have high reliability and validity. However, so far, only French, English, and Portuguese versions of the RSS-12 have been published. Hence, the aim of this study was to prepare atranscultural German translation of the RSS-12 (G-RSS-12) to provide anew clinical and scientific tool for the diagnosis of LPR in German-speaking regions.

Painful total knee arthroplasty: Infrapatellar branch of the saphenous nerve selective denervation. A case series

Introduction: Total knee arthroplasty (TKA) is a reliable and widespread solution for knee osteoarthritis treatment, but about 20% of the patients complains of persisting pain. Neuroma formation in the IPBSN (infrapatellar branch of the saphenous nerve) is an undervalued cause of persistent anterior pain after TKA. The aim of this study was to evaluate the effectiveness of the selective neuroma denervation on patients satisfaction and pain improvement.Matherial and methods: Between 2014 and 2020, we evaluated 13 patients suffering from persistent anterior knee pain and numbness after TKA who underwent a surgical neurectomy of the IPBSN. After clinical assessment and diagnosis confirmation, we carried out the surgery. Short Form 12 Health (SF12), Oxford Knee Score (OKS), and Numeric Rating Scale (NRS) have been collected before and after the procedure. After the surgical treatment, the symptom's resolution and patients’ satisfaction were attested by the achievement of the Minimal ClinicallyImportantDifference(MCID) of the self-administered patient-reported outcome measures (PROMs).Results: Our results showed a statistically significant improvement (p < 0.05) of patients pain perception (mean NRS improvement –4.2, SD 2.5) and overall satisfaction (with a mean increase in OKS of 14.5 points SD 9.4, and in SF12 of 6.5 points SD 4.0).Conclusions: Hence, our study suggests that selective neuroma denervation may be an effective solution to improve the quality of life of patients who develop this complication after TKA.

Psychometric properties of the itch numeric rating scale, skin pain numeric rating scale, and atopic dermatitis sleep scale in adult patients with moderate-to-severe atopic dermatitis

BackgroundThe Itch Numeric Rating Scale (NRS), Skin Pain NRS, and Atopic Dermatitis Sleep Scale (ADSS) are self-administered patient-reported outcome (PRO) instruments developed to assess symptoms in patients with atopic dermatitis (AD). The objective of this study was to evaluate the psychometric properties (reliability, validity, and responsiveness) and interpretability thresholds of these PROs using data from three pivotal Phase 3 studies in adults.MethodsBREEZE-AD1, BREEZE-AD2, and BREEZE-AD5 evaluated the safety and efficacy of baricitinib in adults with moderate-to-severe AD. Clinician-reported outcomes and other PROs commonly assessed in patients with AD were used to estimate meaningful changes and evaluate test–retest reliability, convergent and divergent validity, known-groups validity, responsiveness, and meaningful change thresholds (MCTs) of the Itch NRS, Skin Pain NRS, and ADSS.ResultsThe test–retest reliability of the Itch NRS, Skin Pain NRS, and ADSS was evidenced by generally large intraclass correlation coefficients (> 0.7) in stable groups of patients between baseline and Week 1 and Weeks 4 and 8. Moderate-to-large correlations (r > 0.4) at baseline and Week 16 were generally observed between each measure and other PROs measuring the same concept, supporting convergent validity. Small-to-moderate correlations with clinician-reported outcomes demonstrated divergent validity. Each instrument was able to distinguish between known groups of disease severity as assessed using other indicators of AD severity. The responsiveness of the Itch NRS, Skin Pain NRS, and ADSS scales was demonstrated through significant differences in their change scores from baseline to Week 16 between categories of change in another PRO also from baseline to Week 16. Thresholds for interpreting meaningful change were estimated as − 4.0 for the 0–10 Itch and Skin Pain NRS items; − 1.25 for the 0–4 ADSS Items 1 and 3 and; − 1.50 for the 0–29 ADSS Item 2, these equivalent to moderate degrees of change.ConclusionsResults of this study demonstrate that the psychometric properties of the Itch NRS, Skin Pain NRS, and ADSS are good to excellent. These findings support the use of these instruments in daily assessment of AD symptoms in adults with moderate-to-severe AD.Trial registration ClinicalTrials.gov numbers: NCT03334396, NCT03334422, and NCT03435081.

The ENDOPAIN 4D Questionnaire: A New Validated Tool for Assessing Pain in Endometriosis.

Objectives: To study the measurement properties, the responsiveness and the minimal clinically important difference of the ENDOPAIN-4D: a new questionnaire for assessing pain in endometriosis. Methods: A prospective, observational, multicentre study was conducted including all women ≥18 years consulting for symptomatic proven endometriosis between 1 January 2017 and 30 June 2018 and volunteering to participate. Each patient had to answer a new self-administered patient-reported outcome (PRO) questionnaires (the ENDOPAIN-4D) at inclusion (T0) and 12 months after medical or surgical treatment (T1). Criteria defined by COSMIN were used to validate the questionnaire’s measurement properties. The minimal clinically important difference was estimated by the anchor-based method. Results: The study included 199 women. The ENDOPAIN-4D score had a four dimensional structure with good internal consistency (measured by Cronbach α): (I) pain-related disability (α = 0.79), (II) painful bowel symptoms (α = 0.80), (III) dyspareunia (α = 0.83), and (IV) painful urinary tract symptoms (α = 0.77). They produced four subscores that can be summed to obtain a single score (α = 0.61). The ENDOPAIN-4D total score ranged from 0 to 94.00 (mean ± SD: 46.7 ± 22). The total score was significantly correlated with the PROs used in endometriosis. Sensitivity to change was good with large effect sizes (ES) (mean of the differences: 36.3 p = 1.8 10−7, ES 0.76). The minimal clinically important difference of the global score was determined to be 10.9. Conclusions: The ENDOPAIN-4D questionnaire is easy to use, valid, and effective in assessing patient reported pain symptoms in women treated for endometriosis. This new instrument can be used as the primary outcome for future clinical trials and as a tool for routine patient follow-up.

Association Between Non-Cystic Fibrosis Bronchiectasis and Quality of Life: A Single-Center Cross-Sectional Study.

IntroductionBronchiectasis is a chronic respiratory disease that can affect patients of all ages and significantly impact the quality of life (QOL) in patients who suffer from it. In spite of its widespread prevalence, and the significant impact on QOL, data on the quantitative impact of bronchiectasis on QOL is lacking. The Quality of Life-Bronchiectasis (QOL-B) is a self-administered patient-reported outcome measure, that was recently developed as a response to the emergent need for such measurement tools to study the impact of bronchiectasis on QOL.MethodsWe conducted a single-center cross-sectional study to study the correlation between QOL and various other outcome parameters such as exercise capacity, lung functions, co-morbidities, inflammatory markers, and body mass index (BMI). The secondary outcome was to find out various determinants of quality of life in non-cystic fibrosis bronchiectasis (NCFB).ResultsForty-four patients who determined the pre-determined criteria for NCFB were enrolled in this study. This study demonstrated a significant impact on the QOL of NCFB patients based on the QOL-B scoring system. Almost all domains of QOL-B were found to be adversely impacted as measured by one or more of the outcome parameters but the FEV1, age, colonization, extension, dyspnea (FACED) score, bronchiectasis severity index (BSI) score, six-minute walk test (6MWD), and FEV1 showed associations across most scales while the other outcome parameters showed varying associations.ConclusionsThe QOL is significantly reduced in NCFB and it may be quantified using the QOL-B questionnaire. The impact on QOL in NCFB may be assessed using validated tools such as the FACED and BSI scoring systems, as well as other well-established outcome parameters like 6MWD and FEV1 predicted.

Relevance of Diagnosed Depression and Antidepressants to PROMIS Depression Scores Among Hand Surgical Patients

We aimed to test the utility of screening for depressive symptoms in the hand surgical office focusing on chances of heightened depressive symptoms in patients with no history of diagnosed depression and by quantifying ongoing depressive symptoms among patients diagnosed with depression accounting for antidepressant use. The clinical importance of this study was predicated on the documented negative association between depressive symptoms and hand surgical outcomes. This cross-sectional study analyzed 351 patients presenting to a tertiary hand center between April 21, 2016, and November 22, 2017. Adult patients completed self-administered Patient-Reported Outcomes Measurement Information System (PROMIS) Depression computer adaptive tests at registration. Health records were examined for a past medical history of diagnosed depression and whether patients reported current use of prescription antidepressants. Mean PROMIS Depression scores were compared by analysis of variance (groups: no diagnosed depression, depression without medication, depression with medication). Four points represented a clinically relevant difference in PROMIS Depression scores between groups and Depression scores greater than 59.9 were categorized as having heightened depressive symptoms. Sixty-two patients (18%) had been diagnosed with depression. Thirty-four of these patients (55%) reported taking antidepressant medications. The PROMIS Depression scores indicated greater current depressive symptoms among patients with a history of diagnosed depression when not taking antidepressants (11 points worse than unaffected) and also among patients taking antidepressants (7 points worse than unaffected). Heightened depressive symptoms were detected in all groups but were more prevalent among those diagnosed with depression (36% with no medication, 29% with antidepressant medication) compared with unaffected patients (7%). Depression screening for heightened depressive symptoms identifies 1 in 14 patients without diagnosed depression and 1 in 3 patients diagnosed with depression as having currently heightened depressive symptoms. Hand surgeons can use PROMIS Depression screening in all patients and using this to guide referrals for depression treatment to ameliorate one confounder of hand surgical outcomes. Symptom prevalence study II.

Patient acceptability and usability of a self-administered electronic patient-reported outcome assessment in HIV care: relationship with health behaviors and outcomes

ABSTRACT We assessed acceptability/usability of tablet-based patient-reported outcome (PRO) assessments among patients in HIV care, and relationships with health outcomes using a modified Acceptability E-Scale (AES) within a self-administered PRO assessment. Using multivariable linear regression, we measured associations between patient characteristics and continuous combined AES score. Among 786 patients (median age=48; 91% male; 49% white; 17% Spanish-speaking) overall mean score was 26/30 points (SD: 4.4). Mean scores per dimension (max 5, 1=lowest acceptability, 5=highest): ease of use 4.7, understandability 4.7, time burden 4.3, overall satisfaction 4.3, helpfulness describing symptoms/behaviors 4.2, and enjoyability 3.8. Higher overall score was associated with race/ethnicity (+1.3 points/African-American patients (95%CI:0.3-2.3); +1.6 points/Latino patients (95%CI:0.9-2.3) compared to white patients). Patients completing PROs in Spanish scored +2.4 points on average (95%CI:1.6-3.3). Higher acceptability was associated with better quality of life (0.3 points (95%CI:0.2-0.5)) and adherence (0.4 points (95%CI:0.2-0.6)). Lower acceptability was associated with: higher depression symptoms (-0.9 points (95%CI:-1.4 to -0.4)); recent illicit opioid use (-2.0 points (95%CI:-3.9 to -0.2)); multiple recent sex partners (-0.8 points (95%CI:-1.5 to -0.1)). While patients endorsing depression symptoms, recent opioid use, condomless sex, or multiple sex partners found PROs less acceptable, overall, patients found the assessments highly acceptable and easy to use.

Development and Validation of the Short Version of the Reflux Symptom Score: Reflux Symptom Score-12.

To develop and validate a short version of the Reflux Symptom Score-the 12-question Reflux Symptom Score-12 (RSS-12)-for patients with laryngopharyngeal reflux disease (LPR). Prospective study. Multicenter academic hospitals. Patients with LPR diagnosed via multichannel intraluminal impedance pH monitoring were enrolled from 3 European hospitals. Healthy individuals completed the study. Individuals completed the Reflux Symptom Score, Reflux Symptom Index (RSI), and Voice Handicap Index (VHI) at baseline and 3 months posttreatment. The Reflux Symptom Score was completed twice within a 7-day period to assess test-retest reliability. Cronbach's α was used for assessing internal consistency. The RSS-12 was developed and validity assessed through a comparison of the RSS-12, RSI, and VHI. Responsiveness to change was evaluated through the pre- to posttreatment evolution of the RSS-12 total score. Receiver operating characteristic analysis was used to determine the RSS-12 threshold that is suggestive of LPR. The RSS-12 was characterized by high test-retest reliability (rs = 0.956) and adequate internal consistency reliability (α = 0.739). The RSS-12 was significantly correlated with the RSI (rs = 0.845), suggesting high external validity. The RSS-12 total and item scores were significantly higher in patients with LPR as compared with healthy individuals (P = .001), supporting high internal validity. RSS-12, VHI, and RSI significantly improved throughout treatment. Regarding the receiver operating characteristic curve, an RSS-12 score >11 is suggestive of LPR, exhibiting a sensitivity of 94.5% and a specificity of 86.2%. The RSS-12 is a shorter, reliable, and valid self-administered patient-reported outcome measure questionnaire that can be used in the outpatient setting to suggest and monitor LPR.

Validity and reliability of the Mouth Handicap of Systemic Sclerosis questionnaire in a UK population.

The objective of this study was to explore the psychometric properties of the Mouth Handicap in Systemic Sclerosis (MHISS) specifically exploring aspects of validity and reliability of MHISS in UK SSc patients. A total of 150 questionnaires were distributed in this study at the Oral Medicine Department of UCLHT Eastman Dental Hospital, the Outpatient Rheumatology Clinic of the Royal Free Hospital and Scleroderma Family Day. Participants were asked to complete three questionnaires (SF-36, OHIP-14 and MHISS) along with a pro forma of demographic and disease-specific questions. Both convergent validity and internal consistency reliability were examined. The convergent validity was assessed by Spearman's correlation coefficient, and internal consistency reliability was assessed by Cronbach's alpha. Of the 150 questionnaires distributed to participants, 68 respondents were included. With regard to construct validity, a low to moderate degree of convergent validity was found between MHISS total score and all SF-36 subscales. However, the total MHISS score was highly correlated to all OHIP subscales. MHISS questionnaire is a self-administered patient-reported outcome measure that assesses the mouth disability in SSc patients. However, improving the global and oral health of SSc patients is important as there is no cure for the disease; thus, instruments that record the impact of the condition and indicate the effect of treatment on the lives of patients are of paramount importance. Current results demonstrate good preliminary psychometric properties (validity and reliability) in a UK population with further exploration of psychometric properties with an emphasis on interpretability required.

PRO95 PSYCHOMETRIC EVALUATION OF A PATIENT-REPORTED OUTCOMES (PRO) INSTRUMENT FOR CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA (CTTP)

Congenital thrombotic thrombocytopenic purpura (cTTP, hereditary cTTP) is an ultra-rare, life-threatening disorder where patients experience substantial comorbidity and decreased health-related quality of life (HRQoL). Baxalta (a Takeda company) developed a cTTP-specific patient-reported outcome (PRO) instrument and confirmed its content validity using qualitative cognitive debriefing. This study evaluated the psychometric properties of a newly developed cTTP-specific PRO instrument. This US/UK observational study (NCT03519672) was conducted from July 2018-April 2019 in patients aged ≥18 years receiving treatment for cTTP. Patients were enrolled through investigator sites and direct-to-patient recruitment. Patients completed self-administered PRO measures electronically at baseline and day 14. The cTTP-specific instrument assessed symptoms and impact of cTTP and consists of 5 multiple-item domains (pain/bruising, cognitive impairment, visual impairment, mood, treatment burden) and 3 single-item domains (fatigue, headaches, activity limitation). Convergent and divergent validity were determined by assessing the relationship between cTTP-specific PRO domains and domains of 8 additional PROs using Spearman rank correlation coefficients. Known-groups validity was assessed by comparing two patient-reported impression of severity (PGI-S) groups using t-tests. Internal reliability was assessed using Cronbach’s alpha. Test–retest reliability was assessed using intraclass correlation coefficients (ICC) between the two timepoints. In the 36 patients evaluated, high-to-moderate correlations (r≥0.4) were observed for 10 of 13 pairs of domains and/or total scores, confirming convergent validity. Low correlations (r<0.3) were observed for 7 of 10 hypothesized relationships, confirming divergent validity. Differences in mean cTTP scores between the two PGI-S groups were significant (p≤0.05) for all domains/most items, confirming known-groups validity. Internal Consistency was confirmed with high Cronbach’s alpha (baseline, 0.88; day 14, 0.91). Test-retest reliability was confirmed with a high ICC (0.96). This study provided evidence for the psychometric properties of the newly developed cTTP-specific PRO instrument for use in research and clinical practice to assess HRQoL among cTTP patients.

Validity and reliability of the reflux symptom score.

To develop and validate the Reflux Symptom Score (RSS), a self-administered patient-reported outcome questionnaire for patients with laryngopharyngeal reflux (LPR). Prospective controlled study. A total of 113 patients with LPR were enrolled and treated with diet and 3 months of pantoprazole, alginate, and/or magaldrate depending on the LPR characteristics (acid, nonacid, or mixed). Eighty asymptomatic individuals completed the study. Patients and controls completed the RSS twice within a 7-day period to assess test-retest reliability. Internal consistency was measured using Cronbach's α for the RSS items in patients and controls. Validity was assessed by comparing the baseline RSS with the Reflux Symptom Index (RSI) and Voice Handicap Index (VHI). Seventy-seven patients completed the RSS at baseline and after 6 and 12 weeks of treatment to assess responsiveness to change. The RSS cutoff for determining the presence and absence of LPR was examined by receiver operating characteristic analysis. Test-retest reliability (rs = 0.921) and internal consistency reliability (α = 0.969) were high. RSS exhibited high external validity indicated by a significant correlation with the RSI (rs = 0.831). Internal validity was excellent based on the higher RSS in patients compared with controls (P = .001). RSS, RSI, and VHI scores significantly improved from pre- to posttreatment, indicating a high responsiveness to change. RSS >13 can be considered suggestive of LPR-related symptoms. RSS was not influenced by the occurrence of gastroesophageal reflux disease, LPR subtypes, or patient characteristics. RSS is a self-administered patient-reported outcome questionnaire that demonstrates high reliability and excellent criterion-based validity. RSS can be used in diagnosing and monitoring LPR disease. 3b Laryngoscope, 130:E98-E107, 2020.

Psychometric properties of patient-reported outcome questionnaires for patients with musculoskeletal disorders of the shoulder.

To evaluate the psychometric properties of self-administered patient-reported outcome (PRO) questionnaires which were used in non-surgical homogeneous populations with musculoskeletal shoulder disorders. The included studies were identified using eligibility criteria. The methodological quality of each article was assessed using the COSMIN checklist. The psychometric properties of original versions and translated versions of PROs were also assessed. Twenty articles were included. Two musculoskeletal shoulder disorders were identified that met the selection criteria: rotator cuff disease and glenohumeral instability. A total of 11 PROs were identified. In general, the methodological quality of the included studies is fair or poor. The Western Ontario Rotator Cuff Index (WORC) and the Shoulder Pain and Disability Index (SPADI) are the most frequently evaluated PROs for patients with rotator cuff disease, and their psychometric properties seem to vary according to what language that they are in. For glenohumeral instability, the Western Ontario Shoulder Instability Index (WOSI) and the Oxford Instability Shoulder Score (OISS) are the most frequently evaluated PROs, and their psychometric properties seem to be adequate. Using for rotator cuff disease is advised, for Norwegian users, the SPADI, WORC, Oxford Shoulder Score, and disabilities of the arm, shoulder and hand. Dutch and Persian users could use the WORC. For Greek speakers, the SPADI is recommended. Turkish users could use the rotator cuff quality-of-life measure. For glenohumeral instability, Dutch and Norwegian speakers could use the WOSI and the OISS. Italian, Japanese, and Turkish users could use the WOSI. For English users, the OISS and the Shoulder Rating Questionnaire are recommended. III.

Development of the Autosomal Dominant Polycystic Kidney Disease Impact Scale: A New Health-Related Quality-of-Life Instrument

The impact of autosomal dominant polycystic kidney disease (ADPKD) on health-related quality of life (HRQoL) is not well understood due to a lack of instruments specific to the condition. Content for a new self-administered patient-reported outcome (PRO) questionnaire to assess ADPKD-related HRQoL was developed through clinical expert and patient focus group discussions. The new PRO instrument was administered to study patients with ADPKD to evaluate its reliability and validity. 1,674 adult patients with ADPKD participated in this research: 285 patients in focus groups to generate questionnaire content, 15 patients in debriefing interviews to refine the PRO questionnaire, and 1,374 patients to assess the performance and measurement properties of the PRO questionnaire. A new PRO questionnaire. The ADPKD Impact Scale (ADPKD-IS), consisting of 14 items representing 3 conceptual domains (physical, emotional, and fatigue) plus 4additional questions, was developed. The instrument's reliability (regarding internal consistency and test-retest consistency) and validity (content and construct) were supported. Need for more responsiveness testing when more data from clinical use become available over time. Complex concepts such as ADPKD-related pain and impact on a patient's HRQoL need further evaluation. The ADPKD-IS is a new patient-centric tool that reliably and validly provides a standardized method for assessing HRQoL and overall disease burden in patients with ADPKD.

Implementation and first results of a tablet-based assessment referring to patient-reported outcomes in an inpatient cancer care unit

BackgroundInclusion of patient-reported outcomes (PROs) in routine cancer care is of key importance for individualized treatment, shared decision making and patient satisfaction. ObjectiveTo describe the implementation under routine conditions of an electronic self-administered PRO assessment and comparison of PROs before and after inpatient treatment in oncologic care. MethodsIn a tablet-based survey PROs on symptom burden, global health status/ quality of life (QoL) and health utility were collected twice (at hospital admission and discharge) in an inpatient oncological setting over a 17-month period using the EORTC QLQ-C30 and EQ-5D questionnaires. Data were linked to the hospital information system (HIS). Patient acceptability, recruitment rates, symptom burden, and clinically meaningful changes in PROs over time were analyzed. ResultsFrom a total of 384 hospitalized patients invited to participate at admission 371 (96.6 %) participated. At discharge, 195 patients were approached for a follow-up assessment, and 192 patients (98.5 %) participated. Despite strong acceptance among patients, recruitment rates were decreasing over time. During the hospital stay clinically meaningful improvements were observed for health utility (33.3 %, n = 64) and global health status/QoL (43.2 %, n = 83). Patients reported a variety of symptoms at admission and discharge. ConclusionsImplementation of PRO assessment in routine care and data integration into the HIS provides valuable information for the entire medical staff as symptom burden is present during the entire hospital stay. Implications for PracticeLong-term maintenance of PRO assessment in a clinical setting as a prerequisite of value-based healthcare requires continuous involvement of the nursing team, which can only be achieved by allocating resources to this task.

Validation of a German version of the International Hip Outcome Tool 12 (iHOT12) according to the COSMIN checklist.

BackgroundPatient Reported Outcome (PRO) measurements have become an important tool to evaluate disease-related quality of life. The “International Hip Outcome Tool” (iHOT12) is a self-administered patient-reported outcome tool, which includes questions on the patient’s symptoms, functional and sports limitations as well as social, emotional, and occupational limitations. The purpose of this study was to adapt and validate a German version of the iHOT12 according to the COSMIN checklist.MethodsIn order to validate the German translation of the iHOT12, we conducted a prospective multicenter cohort study on patients with hip disorders and a score ≥4 on the modified Tegner Activity Scale (mTAS). The patients completed the German iHOT12 questionnaire and other functional scores (Hip Outcome Score, modified Tegner Activity Scale, EuroQol-5D) twice at intervals of at least two weeks. Evaluation of psychometric properties was conducted following the COSMIN checklist for validation of health status measurement instruments. The methodical testing for reliability included internal consistency, test-retest reliability, and measurement error. For testing of validity, we analyzed construct validity, hypotheses testing, interpretability and responsiveness.ResultsBetween December 2013 and December 2014, eighty-three consecutive patients completed both questionnaires and were available for data analysis. Cronbach’s alpha was 0.94 (95 %-CI: 0.91, 0.95) confirming internal consistency and test-retest reliability of the iHOT-12 was high with an ICC = 0.94 (95 %-CI: 0.89, 0.97). All a priori hypotheses were confirmed. Further, no relevant floor- or ceiling effects occurred. The iHOT12 showed good responsiveness with a minimal important change (MIC) under 14 points.ConclusionsThe German translation of the iHOT-12 is a reliable, valid, and responsive tool for the evaluation of disease-related quality of life in active patients with a hip disorder. We could show that the minimal important change, a change of health condition the patient discerns, is less than 14 points in the iHOT12 scale.