OP95 Are Patient-Reported Outcome Measures Meeting Today's Standards?

2018 ◽  
Vol 34 (S1) ◽  
pp. 34-35
Author(s):  
Elisabeth Oehrlein ◽  
Eleanor Perfetto ◽  
T. Rose Love ◽  
Yujin Chung ◽  
Parima Ghafoori
Keyword(s):  
Outcome Measures ◽  
Patient Engagement ◽  
Rating Scale ◽  
Psychometric Validation ◽  
Cognitive Interviewing ◽  
Patient Centered ◽  
Concept Elicitation ◽  
Patient Diary ◽  
Patient Reported ◽  
Disease Specific

Introduction:Over the past decade, health technology assessment (HTA) agencies have become interested in improving the patient-centeredness of their assessments. A common approach has been to prioritize patient-reported outcomes (PROs), often describing PROs as patient-relevant or patient-oriented. However, it is often unclear whether and to what degree PRO measures (PROMs) truly reflect what is important to patients. This review examined the pedigree of a sample of measures used as primary or secondary endpoints in trials and discussed in Food and Drug Administration (FDA) approved product labels between 2003 and 2014.Methods:We examined all 26 PROs included in chapters 1 (Office of Microbial Products) and 2 (Office of Drug Evaluation I) of the FDA's Pilot Clinical Outcome Assessment (COA) Compendium. Three reviewers independently searched PubMed and Google to identify publications or other relevant materials related to method and stage of measure development where patient engagement took place.Results:Among 26 evaluated PROMs, we were unable to locate any information on development or validation for 12 (patient diary=9; rating scale=3). Among the remaining 14 PROMs, 5 did not include any evidence of patient engagement (questionnaire=1; patient diary=2; rating scale=2); 3 engaged patients during concept elicitation or psychometric validation only (disease-specific questionnaires=3); and 6 engaged patients during both concept elicitation and cognitive interviewing (disease-specific questionnaires=6). PROMs either previously qualified or submitted for qualification by FDA were more likely to include patient engagement.Conclusions:PROs can provide patient-centered data useful for HTA; however, patient-reported information is not inherently patient-centered. This study found that only a minority of sampled PROMs engaged patients during both concept elicitation and cognitive interviewing. To facilitate patient-centered HTA, manufacturers should ensure that PROMs incorporated into clinical trials measure concepts important to patients. Similarly, HTAs should request data on development and validation of all outcome measures incorporated into trials.

scholarly journals Patients’ Experiences With Staphylococcus aureus and Gram-negative Bacterial Bloodstream Infections: A Qualitative Descriptive Study and Concept Elicitation Phase To Inform Measurement of Patient-reported Quality of Life

2020 ◽  
Author(s):  
Heather A King ◽  
Sarah B Doernberg ◽  
Julie Miller ◽  
Kiran Grover ◽  
Megan Oakes ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Staphylococcus Aureus ◽  
Bloodstream Infection ◽  
Bloodstream Infections ◽  
Patient Centered ◽  
Gram Negative ◽  
Concept Elicitation ◽  
Patient Reported ◽  
Disease Specific

Abstract Background Although Staphylococcus aureus and gram-negative bacterial bloodstream infections (SAB/GNB) cause substantial morbidity, little is known regarding patient perceptions’ of their impact on quality of life (QOL). Guidance for assessing QOL and disease-specific measures are lacking. We conducted a descriptive qualitative study to gain an in-depth understanding of patients’ experiences with SAB/GNB and concept elicitation phase to inform a patient-reported QOL outcome measure. Methods We conducted prospective one-time, in-depth, semi-structured, individual, qualitative telephone interviews 6– 8 weeks following bloodstream infection with either SAB or GNB. Patients were enrolled in an institutional registry (tertiary academic medical center) for SAB or GNB. Interviews were audio-recorded, transcribed, and coded. Directed content analysis identified a priori and emergent themes. Theme matrix techniques were used to facilitate analysis and presentation. Results Interviews were completed with 30 patients with SAB and 31 patients with GNB. Most patients were at or near the end of intravenous antibiotic treatment when interviewed. We identified 3 primary high-level concepts: impact on QOL domains, time as a critical index, and sources of variability across patients. Across both types of bloodstream infection, the QOL domains most impacted were physical and functional, which was particularly evident among patients with SAB. Conclusions SAB/GNB impact QOL among survivors. In particular, SAB had major impacts on multiple QOL domains. A combination of existing, generic measures that are purposefully selected and disease-specific items, if necessary, could best capture these impacts. Engaging patients as stakeholders and obtaining their feedback is crucial to conducting patient-centered clinical trials and providing patient-centered care.

scholarly journals Minimal Clinically Important Difference of Patient Reported Outcome Measures of Lower Extremity Injuries in Orthopedics

2017 ◽  
Vol 5 (2_suppl2) ◽  
pp. 2325967117S0007 ◽  
Author(s):  
Derya Çelik ◽  
Özge Çoban ◽  
Önder Kılıçoğlu
Keyword(s):  
Lower Extremity ◽  
Outcome Measures ◽  
Minimal Clinically Important Difference ◽  
Rating Scale ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Foot And Ankle ◽  
Clinically Important Difference ◽  
Outcome Score ◽  
Patient Reported

Purpose: MCID scores for outcome measures are frequently used evidence-based guides to gage meaningful changes. To conduct a systematic review of the quality and content of the the minimal clinically important difference (MCID) relating to 16 patient-rated outcome measures (PROM) used in lower extremity. Methods: We conducted a systematic literature review on articles reporting MCID in lower extremity outcome measures and orthopedics from January 1, 1980, to May 10, 2016. We evaluated MCID of the 16 patient reported outcome measures (PROM) which were Harris Hip Score (HHS), Oxford Hip Score (OHS), Hip Outcome Score (HOS), Hip Disability and Osteoarthritis Outcome Score (HOOS), The International Knee Documentation Committee Subjective Knee Form (IKDC), The Lysholm Scale, The Western Ontario Meniscal Evaluation Tool (WOMET), The Anterior Cruciate Ligament Quality of Life Questionnaire (ACL-QOL), The Lower Extremity Functional Scale (LEFS), The Western Ontario and Mcmaster Universities Index (WOMAC), Knee İnjury And Osteoarthritis Outcome Score (KOOS), Oxford Knee Score (OKS), Kujala Anterior Knee Pain Scale, The Victorian Institute of Sports Assessment Patellar Tendinosis (Jumper’s Knee) (VİSA-P), Tegner Activity Rating Scale, Marx Activity Rating Scale, Foot And Ankle Outcome Score (FAOS), The Foot Function Index (FFI), Foot And Ankle Ability Measure (FAAM), The Foot And Ankle Disability Index Score and Sports Module, Achill Tendon Total Rupture Score(ATRS), The Victorian İnstitute Of Sports Assesment Achilles Questionnaire(VİSA-A), American Orthopaedic Foot and Ankle Society (AOFAS). A search of the PubMed/MEDLINE, PEDro and Cochrane Cen¬tral Register of Controlled Trials and Web of Science databases from the date of inception to May 1, 2016 was conducted. The terms “minimal clinically important difference,” “minimal clinically important change”, “minimal clinically important improvement” “were combined with one of the PROM as mentioned above. Results: A total of 223 abstracts were reviewed and 119 articles chosen for full text review. Thirty articles were included in the final evaluation. The MCID was mostly calculated for WOMAC and frequently reported in knee and hip osteoartritis, knee and hip atrhroplasties, femoraasetabular impingement syndrome and focal cartilage degeneration. In addition, Receiver Operating Characteristic (ROC) analysis was the most used method to report MCID. Conclusions: MCID is an important concept used to determine whether a medical intervention improves perceived outcomes in patients. Despite an abundance of methods reported in the literature, little work in MCID estimation has been done in the PRAM related to lower extremity. There is a need for future studies in this regard.

An interdisciplinary intensive outpatient pain program is associated with improved patient activation and key outcomes

2020 ◽  
Vol 10 (5) ◽  
pp. 307-318
Author(s):  
Barbara K Bujak ◽  
Christine E Blake ◽  
Paul F Beattie ◽  
Shana Harrington ◽  
Courtney M Monroe ◽  
...  
Keyword(s):  
Outcome Measures ◽  
Patient Reported Outcomes ◽  
Rating Scale ◽  
Patient Activation ◽  
Persistent Pain ◽  
Outpatient Program ◽  
Patient Reported ◽  
Patient Activation Measure ◽  
Intensive Outpatient Program ◽  
Intensive Outpatient

Aim: To examine the change in the Patient Activation Measure and physical and psychosocial outcome measures in a military interdisciplinary intensive outpatient program for persistent pain. Materials & methods: Pre- and post-intervention measures, which were also stratified by gender and baseline activation, included patient-reported outcomes and physical function assessment, obtained from 2017 to 2018 program database. Results: The majority of the participants were male (70.9%), with an average age of 29.18 years and pain duration of 4.78 years (n = 103). Patient activation, majority of the patient reported outcomes and functional assessments improved in the overall sample with fewer changes in females on the Defense and Veterans Pain Rating Scale. Conclusion: Improvements were noted on the Patient Activation Measure and majority of the other outcome measures suggesting that service members with persistent pain at any level of patient activation or baseline function, may benefit from an intensive outpatient program.

scholarly journals Cross-Cultural Adaptation and Psychometric Validation of the Dutch Version of the Core Outcome Measures Index for the Neck in Patients Undergoing Surgery for Degenerative Disease of the Cervical Spine

Neurospine ◽  
2021 ◽  
Vol 18 (4) ◽  
pp. 798-805
Author(s):  
Pravesh S. Gadjradj ◽  
Timothy C. Chin-See-Chong ◽  
Daphne Donk ◽  
Paul Depauw ◽  
Maurits W. van Tulder ◽  
...  
Keyword(s):  
Outcome Measures ◽  
Roc Curve ◽  
Psychometric Validation ◽  
Core Outcome ◽  
Dutch Version ◽  
Retest Reliability ◽  
The Core ◽  
Patient Reported ◽  
Core Outcome Measures ◽  
Test Retest Reliability

Objective: To perform the psychometric validation of the Dutch version of the Core Outcome Measures Index (COMI) for the neck.Methods: A total of 178 patients, who had an indication for surgery due to degenerative cervical spinal disease, were enrolled in the study. They filled in a baseline booklet containing the Dutch version of the COMI-neck, Likert-scales for neck and arm/shoulder pain, the Neck Disability Index (NDI), the EuroQol-5 dimensions (EQ-5D) and the 12-item Short Form health survey (SF-12). Aside from analyzing construct validity using the Spearman correlation test, test-retest reliability, and responsiveness at 3 months were assessed using the intraclass correlation coefficient (ICC) and the receiver-operating characteristic (ROC) curve, respectively.Results: The COMI-neck showed good acceptability with missing data ranging from 0% to 4.5% and some floor/ceiling effects for 3 of the domains at baseline. The COMI-summary score showed good to very good correlation with the EQ5D (ρ = -0.43), the physical component summary of the SF-12 (ρ = -0.47) and the NDI (ρ = 0.73). Individual domains showed correlations of -0.28 to 0.85 with the reference questionnaires. Test-retest reliability analysis showed an ICC of 0.91 with a minimal detectable change of 1.7. Responsiveness analysis of the COMI-neck showed an area under 0.79 under the ROC-curve. The standardized response mean for a good outcome was 1.24 and for a poor outcome 0.37.Conclusion: The current study shows that the Dutch version of the COMI-neck is a valid, reliable and responsive Patient-Reported Outcome Measure, among patients undergoing surgery for degenerative cervical spinal disorders.

scholarly journals 12-Month Cohort Study to Investigate Changes in Patient-Reported Outcomes Pain after Intra-articular Injection of Micro-Fragmented Adipose Tissue for Knee Osteoarthritis

2021 ◽  
Vol 3 (1) ◽  
pp. e19-e28
Author(s):  
Nathan Hogaboom ◽  
Ella D'Amico ◽  
Ken Mautner ◽  
Christopher Rogers ◽  
Gerard Malanga
Keyword(s):  
Quality Of Life ◽  
Adipose Tissue ◽  
Knee Osteoarthritis ◽  
Outcome Measures ◽  
Knee Pain ◽  
Rating Scale ◽  
Numerical Rating Scale ◽  
Symptomatic Knee ◽  
Patient Reported

BackgroundTo evaluate changes in pain, function, and quality of life after treatment with injected micro-fragmented adipose tissue (MFAT) for knee osteoarthritis in a large cohort of individuals treated at multiple centers. MethodsOne hundred ten individuals were recruited from three private outpatient clinics. Participants had to be diagnosed with symptomatic knee OA (defined by persistent knee pain associated with clinical symptoms of OA and/ or classic imaging findings) and who had not received prior knee surgery or treatment with platelet-rich plasma, cortisone, or hyaluronic acid within the previous 6 weeks. Data from 120 knees were included in the analysis. Outcome measures included Knee Injury and Osteoarthritis Outcome Score (KOOS) subscales (pain, symptoms, activities of daily living [ADL], sports and recreation, quality of life [QOL]) and an 11-point Numerical Rating Scale (NRS) for average knee pain over the past week. Outcomes were collected at baseline and 3, 6, and 12 months. ResultsSignificant increases and decreases in KOOS subscale and NRS scores were observed, respectively, in the cohort as a whole (p< .05). Lower BMI was associated with more significant improvements in pain, sports/recreation, and ADL KOOS subscale scores (p< .05). Greater age was associated with more significant improvements in symptoms and QOL subscale scores (p< .05). ConclusionsA single injection of MFAT improved pain, function, and QOL outcome measures up to 12 months in this cohort for more than half of the participants. Greater BMI and lower age negatively influenced outcomes. It is not known whether improvements continue after this timeframe or why many participants reported little-to-no improvement.

scholarly journals Development of Patient-Reported Outcome Measures (Symptoms and Impacts) in Adults with Pyruvate Kinase Deficiency

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3447-3447
Author(s):  
Sam Salek ◽  
Audra N. Boscoe ◽  
Chris Evans ◽  
Shayna Egan ◽  
Ted Wells ◽  
...  
Keyword(s):  
Pyruvate Kinase ◽  
Research Funding ◽  
Patient Reported Outcome ◽  
Psychometric Validation ◽  
Specific Patient ◽  
Patient Reported ◽  
Item Wording ◽  
Eortc Qlq C30 ◽  
Eortc Qlq ◽  
Disease Specific

Introduction: Pyruvate kinase (PK) deficiency is a rare, congenital autosomal recessive hemolytic anemia managed with supportive treatments, including transfusion, splenectomy, and iron chelation. Disease-directed treatments, including a small molecule PK activator and gene therapy, are currently in development. No disease-specific patient-reported outcome (PRO) measures have been validated for use in this patient population. The objective of this initiative was to develop PRO measures for assessing symptoms and impacts of PK deficiency and compare them to existing, non-disease-specific measures previously recommended for use in this disease area. Methods: A targeted literature review was conducted to inform the development of a preliminary hypothesized conceptual framework to identify signs, symptoms, and impacts commonly experienced by patients with PK deficiency and to inform the direction and content of interviews with such patients. Concept elicitation interviews were conducted with 21 adults with PK deficiency from the US, Netherlands, and Germany. Draft items were then tested in cognitive interviews with 20 adults with PK deficiency to further establish content validity and revised based on the results. A comparison was conducted between concepts included in the newly developed PK deficiency disease-specific measures and the domain structure and item concepts included in the EORTC QLQ-C30 and SF-36v2 to evaluate the extent of differences and conceptual overlap with instruments that had previously been recommended in this population. Specific attributes compared included face validity (i.e., conceptual coverage and inclusion of proximal symptoms and/or impacts) and measurement characteristics (i.e., item wording, recall, and response options). Results: Two measures, the PK Deficiency Diary (PKDD), a 7-item measure of the core signs and symptoms of PK deficiency, and PK Deficiency Impact Assessment (PKDIA), a 14-item measure of the impacts of PK deficiency on patients' HRQoL, were developed. A comparison of the newly drafted measures to the EORTC QLQ-C30 and SF-36v2 demonstrated minimal similarities in concepts, domains, item wording, and recall period. Of the 7 concepts in the PKDD, only 3 were common to the EORTC QLQ-C30, 4 were common to the SF36v2, and 2 were related but did not match exactly (i.e., "bone pain" and "pain"). Of the 12 distinct concepts in the PKDIA, only 5 were common to the EORTC QLQ-C30, 2 were common to the SF-36v2, and 3 were related but did not match exactly (i.e., difficulty starting things, difficulty finishing things, and difficulty performing moderate physical activity). Conclusions: This research demonstrates that the EORTC-QLQ-C30 and SF-36v2 lack the appropriate conceptual relevance and coverage of disease-specific signs, symptoms, and impacts most relevant and burdensome to patients with PK deficiency. The newly developed PKDD and PKDIA may be useful tools in clinical trials in patients with PK deficiency. Psychometric validation of these measures is currently underway. Disclosures Salek: Pfizer: Honoraria, Speakers Bureau; Merck: Consultancy; Agios Pharmaceuticals, Inc.: Consultancy, Honoraria. Boscoe:Agios Pharmaceuticals, Inc.: Employment, Equity Ownership. Evans:Agios Pharmaceuticals, Inc.: Consultancy, Research Funding. Egan:Agios Pharmaceuticals, Inc.: Consultancy, Research Funding. Wells:Agios Pharmaceuticals, Inc.: Consultancy, Research Funding. Piantedosi:Agios Pharmaceuticals, Inc.: Employment. Grace:Agios Pharmaceuticals, Inc: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Research Funding. Storm:Agios: Employment.

scholarly journals Patient-Reported Outcome Measures (PROMs) in Pediatric Non-Malignant Hematology: A Systematic Review

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 2180-2180
Author(s):  
Robert J Klaassen ◽  
Julia Y. Kinahan ◽  
Johann M. I. Graham ◽  
Yamilée V. Hébert ◽  
Katie O'Hearn
Keyword(s):  
Quality Of Life ◽  
Systematic Review ◽  
Outcome Measures ◽  
Well Being ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Clinical Environment ◽  
Patient Reported ◽  
Disease Specific

Introduction: Patient reported outcome measures (PROMs) are questionnaires completed by patients or caregivers without interpretation by healthcare professionals. As such, they allow patient concerns about a variety of healthcare issues to be identified and addressed in an efficient and actionable manner. PROMs can be generic, with questions relevant to multiple disease groups or disease-specific, with questions targeting the symptoms, limitations, and feelings common to the disease group. This systematic review identified generic and disease-specific PROMs for monitoring symptoms and health-related quality of life (HRQoL) in 4 pediatric non-malignant hematologic disease groups: thalassemia, hemophilia, immune thrombocytopenia (ITP), and sickle cell disease (SCD). Methods: Databases (MEDLINE, Embase, HaPI, CINAHL, and PsycTESTS) were searched to identify publications that either validated or used PROMs as an outcome measure in the four disease groups. Articles were excluded when <30% of the population was pediatric (<18 years), when the study setting was inpatient, when the tool had not been validated, or when the article did not report the use of a PROM for monitoring symptoms or HRQoL. Notably, hemophilia records published prior to 2016 were not screened as a systematic review by Limperg et al. (2017) identified validated PROMs in the pediatric hemophilia population and was used to include relevant articles. Results: The search identified 1176 unique records, with 902 records remaining for title and abstract screening after removal of 274 hemophilia articles published prior to the systematic review. Including hemophilia records identified from the 2017 review, 217 articles met inclusion criteria incorporating 107 generic and 20 disease-specific PROMs. Of the generic tools, the most frequent categories identified include psychological well-being (26 tools), general quality of life (19 tools), and family impact (19 tools). The most frequently used tool was the PedsQL 4.0 Generic Core Scales (66 studies), appearing 33 times in SCD, 25 times in thalassemia, 5 times in ITP, and 3 times in hemophilia. Other commonly used generic tools include the Short Form Health Survey, Child Health Questionnaire, PROMIS Health Measures, and Child Behaviour Checklist (Table). Disease-specific tools identified in the review include the PedsQL SCD Module, Kids ITP Tool, Haemo-QoL, CHO-KLAT, and TranQol (Table). In addition, 10 studies reported on pain diaries and 9 of these studies were SCD focused, the other being hemophilia focused. Conclusion: This systematic review identified several generic and disease-specific PROMs that have been used in pediatric non-malignant hematology. Although generic tools have been used more frequently, many disease-specific tools have been validated and are available for use in the clinical environment. We are currently conducting focus groups with patients, parents, and clinicians to determine the optimal choice of tools for monitoring symptoms and HRQoL in the pediatric non-malignant clinical environment. Disclosures No relevant conflicts of interest to declare.

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