scholarly journals Validation and equivalency of electronic clinical outcomes assessment systems

2020 ◽  
Vol 7 (4) ◽  
pp. 271
Author(s):  
Sarah T. Gary ◽  
Antonio V. Otero ◽  
Kenneth G. Faulkner ◽  
Nadeeka R. Dias
Keyword(s):  
Clinical Outcomes ◽  
Patient Reported Outcomes ◽  
Bring Your Own Device ◽  
Equivalence Testing ◽  
Patient Reported ◽  
The Us ◽  
Different Types ◽  
Fit For Purpose ◽  
Clinical Outcomes Assessment ◽  
Recent Patient

<p>The US food and drug administration (FDA) has long called for clinical outcomes assessments (COA), such as patient-reported outcomes (PRO), to be ‘fit-for-purpose’ meaning the COA has been validated to support the context of use. The FDA’s recent patient-focused drug development guidance series has renewed the importance of ensuring that COA is ‘fit-for-purpose’ and valid.  In addition, the FDA has recommended that COA be collected electronically and that the electronic (eCOA) system and devices also be validated. Advancing technology requires eCOA systems and devices to evolve; eCOA devices may change over time. As bring your own device (BYOD) models gain popularity and acceptance, devices may also be mixed within trials. Changes in eCOA devices or mixing devices may require equivalence testing to prove validity across platforms. The aim of this article is to provide an overview of the different types of validation at both the assessment level and the eCOA system (device) level to help clinical trial sponsors determine the appropriate level of validation or equivalence testing required for COA used in their clinical trials.   </p>

scholarly journals Linguistic validation of the simplified Chinese version of the US National Cancer Institute’s patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE™)

BMC Cancer ◽  
2020 ◽  
Vol 20 (1) ◽  
Author(s):  
Cheng KKF ◽  
S. A. Mitchell ◽  
N. Chan ◽  
E. Ang ◽  
W. Tam ◽  
...  
Keyword(s):  
Adverse Events ◽  
Cancer Patients ◽  
Patient Reported Outcomes ◽  
A Priori ◽  
Cognitive Interviews ◽  
Simplified Chinese ◽  
Patient Reported ◽  
The Us ◽  
The Common ◽  
Response Choices

Abstract Background The aim of this study was to translate and linguistically validate the U.S. National Cancer Institute’s Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE™) into Simplified Chinese for use in Singapore. Methods All 124 items of the English source PRO-CTCAE item library were translated into Simplified Chinese using internationally established translation procedures. Two rounds of cognitive interviews were conducted with 96 cancer patients undergoing adjuvant treatment to determine if the translations adequately captured the PRO-CTCAE source concepts, and to evaluate comprehension, clarity and ease of judgement. Interview probes addressed the 78 PRO-CTCAE symptom terms (e.g. fatigue), as well as the attributes (e.g. severity), response choices, and phrasing of ‘at its worst’. Items that met the a priori threshold of ≥20% of participants with comprehension difficulties were considered for rephrasing and retesting. Items where < 20% of the sample experienced comprehension difficulties were also considered for rephrasing if better phrasing options were available. Results A majority of PRO-CTCAE-Simplified Chinese items were well comprehended by participants in Round 1. One item posed difficulties in ≥20% and was revised. Two items presented difficulties in < 20% but were revised as there were preferred alternative phrasings. Twenty-four items presented difficulties in < 10% of respondents. Of these, eleven items were revised to an alternative preferred phrasing, four items were revised to include synonyms. Revised items were tested in Round 2 and demonstrated satisfactory comprehension. Conclusions PRO-CTCAE-Simplified Chinese has been successfully developed and linguistically validated in a sample of cancer patients residing in Singapore.

scholarly journals Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome

2015 ◽  
Vol 3 ◽  
pp. 232640981557618 ◽  
Author(s):  
Christina Lampe ◽  
Mohit Jain ◽  
Andrew Olaye ◽  
Bianca Meesen ◽  
Celeste Decker ◽  
...  
Keyword(s):  
Clinical Outcomes ◽  
Patient Reported Outcomes ◽  
Morquio A ◽  
Patient Reported ◽  
Morquio A Syndrome

Fit for purpose and modern validity theory in clinical outcomes assessment

2017 ◽  
Vol 27 (7) ◽  
pp. 1711-1720 ◽  
Author(s):  
Michael C. Edwards ◽  
Ashley Slagle ◽  
Jonathan D. Rubright ◽  
R. J. Wirth
Keyword(s):  
Clinical Outcomes ◽  
Outcomes Assessment ◽  
Fit For Purpose ◽  
Clinical Outcomes Assessment ◽  
Validity Theory

scholarly journals Prospective, observational study to evaluate the relation between patient-reported outcomes and component rotation in total knee replacement: protocol for the CRACK study

BMJ Open ◽  
2019 ◽  
Vol 9 (11) ◽  
pp. e031166
Author(s):  
Hans-Peter van Jonbergen ◽  
Ellie Landman ◽  
Maarten Horst ◽  
Robin Westerbeek ◽  
Ydo Kleinlugtenbelt
Keyword(s):  
Total Knee Replacement ◽  
Clinical Outcomes ◽  
Outcome Measures ◽  
Knee Replacement ◽  
Tibial Component ◽  
Patient Reported Outcomes ◽  
Persistent Pain ◽  
Component Rotation ◽  
Patient Reported ◽  
Total Knee

IntroductionTotal knee replacement (TKR) for osteoarthritis results in a satisfactory outcome in the majority of patients, although up to one in five patients may be dissatisfied with the outcome. Persistent pain is a main contributor to patient dissatisfaction, and femoral and tibial component malrotation have been identified as a potential cause for both persistent pain and patellofemoral problems. Based on the assumption that component malrotation is the causative factor for persistent pain, early revision for patients with symptomatic malrotated components has been advocated in the literature. However, convincing evidence that component malrotation indeed results in less than optimal outcomes is lacking. This study aims to assess the relation between knee prosthesis component rotation and patient-reported outcomes in a large group of patients and to determine the range of femoral, tibial and combined rotation that results in the best clinical outcomes.Methods and analysisIn this single-centre, prospective observational cohort study, a total of 500 patients will undergo TKR. All patients will have a 3D-CT assessment of femoral and tibial component rotation within 8 weeks after surgery. Outcome measures will include the Oxford Knee Score, the Knee Injury and Osteoarthritis Outcome Score, EQ-5D, visual analogue scale for pain, the American Knee Society Score and the knee joint range of motion. We will assess the relation between femoral, tibial and combined component rotation and patient-reported outcome measures at 8 weeks and 1 year of follow-up, and we will determine the range of femoral, tibial and combined rotation that results in the best clinical outcomes.Ethics and disseminationEthical approval for this study has been granted by the Isala Hospital ethics committee. The results will be published in a peer-reviewed journal and presented at relevant meetings.Trial registration numberNL7635.

scholarly journals Association of Patient-reported Outcomes With Clinical Outcomes After Distal Humerus Fracture Treatment

2020 ◽  
Vol 4 (2) ◽  
pp. e19.00122
Author(s):  
Abhiram R. Bhashyam ◽  
Yassine Ochen ◽  
Quirine M.J. van der Vliet ◽  
Luke P.H. Leenen ◽  
Falco Hietbrink ◽  
...  
Keyword(s):  
Clinical Outcomes ◽  
Patient Reported Outcomes ◽  
Distal Humerus ◽  
Humerus Fracture ◽  
Fracture Treatment ◽  
Distal Humerus Fracture ◽  
Patient Reported

scholarly journals The HOPE Pilot Study: Harnessing Patient-Reported Outcomes and Biometric Data to Enhance Cancer Care

2018 ◽  
pp. 1-12 ◽  
Author(s):  
Alexi A. Wright ◽  
Nikita Raman ◽  
Patrick Staples ◽  
Stephanie Schonholz ◽  
Angel Cronin ◽  
...  
Keyword(s):  
Physical Activity ◽  
Health Status ◽  
Clinical Outcomes ◽  
Mobile Health ◽  
Symptom Management ◽  
Patient Reported Outcomes ◽  
Perceived Effectiveness ◽  
Activity Data ◽  
Patient Reported ◽  
Wearable Accelerometers

Purpose Integrating patient-reported outcomes (PROs) into clinical practice is an increasingly promising strategy for improving patients’ symptoms, communication, and clinical outcomes. The objective of the current study was to assess the feasibility, acceptability, and perceived effectiveness of a mobile health intervention that was designed to collect PROs and activity data as a measure of health status. Patients and Methods This work was a pilot intervention with 10 patients with gynecologic cancers who received palliative chemotherapy. The HOPE (Helping Our Patients Excel) study used wearable accelerometers to assess physical activity and the Beiwe research platform to collect PROs, stratify patient responses by risk, provide tailored symptom management, and notify patients and clinicians of high-risk symptoms. Feasibility and acceptability were assessed through enrollment and adherence rates, and perceived effectiveness was evaluated by patients and oncologists at study completion. Results The approach-to-consent rate was 100%, and participants were 90% and 70% adherent to the wearable accelerometers and smartphone surveys, respectively. Participants’ mean daily step count was 3,973 (standard deviation [SD], 2,305 steps) and increased from week 1 (mean, 3,520 steps; SD, 1,937 steps) to week 3 (mean, 4,136 steps; SD, 1,578 steps). Active monitoring of participants’ heart rates, daily steps, and PROs throughout the study identified anomalies in participants’ behavior patterns that suggested poor health for two patients (20%). Patients and clinicians indicated that the intervention improved physical activity, communication, and symptom management. Conclusion A mobile health intervention that collects PROs and activity data as a measure of health status is feasible, acceptable, and was perceived to be effective in improving symptom management in patients with advanced gynecologic cancers. A larger, multisite, randomized clinical trial to assess the efficacy of the HOPE intervention on patients’ symptoms, health-related quality of life, clinical outcomes, and health care use is warranted.

scholarly journals Two Year Clinical Outcomes of the Subchondroplasty® Procedure for Treatment of Symptomatic Bone Marrow Lesions of the Knee

2019 ◽  
Vol 7 (7_suppl5) ◽  
pp. 2325967119S0029
Author(s):  
Christopher Hajnik ◽  
Sam Akhavan ◽  
Douglas J. Wyland ◽  
Steven B. Cohen ◽  
Laith M. Jazrawi ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Clinical Outcomes ◽  
Conversion Rate ◽  
Bone Substitute ◽  
Patient Reported Outcomes ◽  
Bone Substitute Material ◽  
Bone Marrow Lesions ◽  
Patient Reported ◽  
Substitute Material ◽  
One Year

Objectives: Bone Marrow Lesions (BML) are a common finding on knee MRI. In the knee, BML have a strong correlation to patient-reported pain, function, joint deterioration and rapid progression to TKR. Histologic analyses of BML have demonstrated findings consistent with fracture and bony remodeling of the trabeculae. The Subchondroplasty (SCP®) Procedure aims to treat the bone defects present in the BML by percutaneously filling them with a bone substitute material, designed to flow through intact bone, harden at body temperature and then heal through natural bone turnover. Previous retrospective, single-center case series have demonstrated improvements in patient-reported outcomes. The purpose of this prospective, multi-center study is to evaluate the 2-year clinical and radiographic outcomes of patients with BML of the knee treated with the Subchondroplasty Procedure. Methods: Seventy patients were treated between 2012 and 2017 for BML of the tibial plateau and/or femoral condyle. Self-drilling cannulas were inserted into the BML using arthroscopic and fluoroscopic guidance, then injected with AccuFill® Bone Substitute Material. All patients also underwent arthroscopy to aid in targeting the underlying bony lesion and address intra-articular pathology. MRIs and radiographs were obtained pre-operatively, at 6, 12 and 24 months, with additional radiographs collected at 6 weeks and 3 months. Patient-reported outcomes, including VAS pain, IKDC and KOOS were collected pre-operatively, 2 and 6 weeks, and 3, 6, 12 and 24 months post-operatively. Results: Seventy patients (36 males and 34 females), average age 57 were consented and enrolled in the study. Preoperative K-L grade included 1.4% Grade 0, 2.9% Grade 1, 27.1% Grade 2, 55.7% Grade 3 and 7.1% Grade 4. Fifty eight tibial plateaus and 41 femoral condyles were treated (29 bipolar lesions treated). VAS Pain scores improved from a mean of 6.2/10 pre-op to 2.9/10 at 1 year. IKDC scores improved from mean 33.9 pre-op to 61.3 at 1 year. KOOS scores improved from baseline to 1 year (Fig. 1) with mean KOOS Pain from 45.8 to 73.9, ADL 52.9 to 79.2, Symptoms 49.7 to 71.9, Sports 21.2 to 49.9 and Quality of Life 18.1 to 52.3. All patient-reported outcomes showed statistically significant improvement at one year. Two year outcomes collected to date appear to follow the same trend. The last study subject is due to return in January 2019 at which point the final 2 year analysis will be completed. Six patients (8.6%) converted to arthroplasty (1 UKA and 5 TKA) at one year. To date, the 24 month conversion rate is 16.1% out of 62 subjects. The final conversion rate for 24 months will be calculated after the final subject returns. Radiographs and MRIs demonstrated good incorporation of the AccuFill material through 12 months with evidence of early remodeling and a lack of OA progression in the majority of subjects. Twenty-four month MRIs demonstrate continued remodeling of the AccuFill material. Conclusion: This study presents statistically and clinically-meaningful evidence of improvements in clinical outcomes following Subchondroplasty procedure for BML of the knee. The low conversion rate suggests this less-invasive procedure may delay the need for knee arthroplasty. MR imaging demonstrates good incorporation of the BSM and evidence of remodeling and reduction in material volume over time. [Figure: see text][Table: see text]

Sign in / Sign up

Export Citation Format

Share Document