Final Patient-reported Outcome Research Articles

Does increasing age impact clinical and radiographic outcomes following lumbar spinal fusion?

Background ContextDespite the growing senior population within the United States, there is a lack of consensus regarding the safety and efficacy of performing lumbar spinal fusion for this population. PurposeTo evaluate the clinical and radiographic outcomes in different age cohorts following lumbar spinal fusion. Study DesignRetrospective cohort analysis. Patient SampleAnalysis of 1,184 patients who underwent posterolateral lumbar fusion from 2011 to 2018. Surgery was indicated after failure of conservative treatment to address radiculopathy and/or neurogenic claudication. Patients were excluded if they were under 18 years of age at the time of surgery, had a lumbar fracture, tumor, or infection, or had fusions involving the thoracic spine, high-grade spondylolisthesis, or concomitant deformity. Of the 1,184 patients, 850 patients were included. Patients were divided into three roughly equal groups for analysis: young (18–54 years), middle-aged (55–69 years), and senior (≥70 years). Outcome MeasuresVisual Analog Scale Back/Leg pain, and Oswestry Disability Index (ODI) were collected, and achievement of minimal clinically important difference was evaluated. Lumbar lordosis (LL), pelvic tilt (PT), pelvic incidence (PI), and PI-LL difference were measured on radiographs. Rates of postoperative complications were analyzed. MethodsSeveral radiographic parameters were measured using plain radiographs obtained at preoperative, immediately postoperative (standing radiographs performed on postoperative day 1), and most recent follow-up visits. Preoperative and final patient-reported outcomes, along with demographic information, were obtained all patients. Binary outcome variables were compared between groups with multivariate logistic regression, and continuous outcome variables were compared using multivariate linear regression, with age 18 to 54 years used as the reference. Multivariate regressions were used to compare outcomes between cohorts while controlling baseline characteristics. ResultsA total of 850 patients were included; 330 young (38.80%), 317 middle-aged (37.30%), and 203 senior (23.90%). Seniors had higher postoperative length of stay compared to younger patients (p<.001). Younger patients had worse final ODI scores compared to middle-aged patients (p=.002). Seniors had higher rates of proximal ASD (p=.002) compared to young patients. There was no difference in achievement of minimal clinically important differences (MCID) between all three groups. ConclusionsSenior patients have significant improvement in patient-reported clinical outcomes, despite having greater comorbidities, and longer length of stay. However, given a general lack of achievement of MCID across all cohorts, these findings suggest the need for a critical re-evaluation of the role of lumbar spinal fusion in the management of patients with refractory radiculopathic and/or neurogenic claudication symptoms.

Patients with predominantly back pain at the time of lumbar fusion for low-grade spondylolisthesis experience similar clinical improvement to patients with predominantly leg pain: mid-term results

BACKGROUND CONTEXTPatients with back pain predominance (BPP) have traditionally been thought to derive less predictable symptomatic relief from lumbar fusion surgery. PURPOSETo compare postoperative clinical outcomes as well as degree of improvement in clinical outcome measures between patients with BPP and patients with leg pain predominance (LPP) undergoing open posterior lumbar fusion. STUDY DESIGNRetrospective cohort study. PATIENT SAMPLEAnalysis of patients who underwent an open posterior lumbar fusion for low-grade (Meyerding Grade I or II) degenerative or isthmic spondylolisthesis from 2011 to 2018 was conducted. Surgery was indicated after failure of conservative treatment to address radiculopathy and/or neurogenic claudication. Patients were excluded if they were under 18 years of age at the time of surgery, had less than 6 months of follow-up, presented with a lumbar vertebral body fracture, tumor, or infection, or underwent a fusion surgery that extended to the thoracic spine, high-grade spondylolisthesis, or concomitant deformity. OUTCOME MEASURESRadiographs obtained at preoperative, immediate postoperative, and final visits were evaluated for presence or absence of fusion. Patient-reported outcomes were recorded at preoperative and final clinic visits that included: visual analog scale (VAS) back/leg pain, and Oswestry disability index (ODI). Achievement of minimal clinically important difference (MCID) was analyzed, along with rates of postoperative complication and reoperation. METHODSPreoperative and final patient-reported outcomes were obtained. Achievement of MCID was evaluated using following thresholds: ODI 14.9, VAS-back pain 2.1, VAS-leg pain 2.8. For analysis, patients were divided into two groups based on predominant location of pain: predominantly VAS-back pain (BPP) and predominantly VAS-leg pain (LPP). RESULTSOne hundred forty-one patients met inclusion criteria. Of these, 71 had LPP, and 70 had BPP. Patients with preoperative LPP experienced greater improvements in VAS-leg (p<.001) compared to those with BPP, whereas patients with preoperative BPP experienced greater improvements in VAS-back (p=.011) postoperatively compared to those with LPP. There were no differences in the final clinical outcomes. Additionally, LPP achieved MCID for VAS-leg (p=.027) at significantly higher proportion than BPP and BPP achieved MCID for VAS-back (p=.050) at significantly higher proportion than LPP. CONCLUSIONSPatients with low-grade spondylolisthesis who underwent an open posterior lumbar fusion had improvement in symptoms regardless of presentation with BPP or LPP. In properly indicated patients, posterior spinal fusion is effective for those with BPP in the setting of experiencing both leg and back pain, and clinicians can use this information for perioperative discussions and surgical decision-making.

Strategies to improve patient-reported outcome completion rates in longitudinal studies

PurposeThe quality of patient-reported outcome (PRO) data can be compromised by non-response (NR) to scheduled questionnaires, particularly if reasons for NR are related to health problems, which may lead to unintended bias. The aim was to investigate whether electronic reminders and real-time monitoring improve PRO completion rate.MethodsThe population-based study “Quality of life in Danish multiple myeloma patients” is a longitudinal, multicentre study with consecutive inclusion of treatment-demanding newly diagnosed or relapsed patients with multiple myeloma. Education of study nurses in the avoidance of NR, electronic reminders, 7-day response windows and real-time monitoring of NR were integrated in the study. Patients complete PRO assessments at study entry and at 12 follow-up time points using electronic or paper questionnaires. The effect of the electronic reminders and real-time monitoring were investigated by comparison of proportions of completed questionnaires before and after each intervention.ResultsThe first 271 included patients were analysed; of those, 249 (85%) chose electronic questionnaires. Eighty-four percent of the 1441 scheduled PRO assessments were completed within the 7-day response window and 11% after real-time monitoring, achieving a final PRO completion rate of 95%. A significant higher proportion of uncompleted questionnaires were completed after the patients had received the electronic reminder and after real-time monitoring.ConclusionsElectronic reminders and real-time monitoring contributed to a very high completion rate in the study. To increase the quality of PRO data, we propose integrating these strategies in PRO studies, however highlighting that an increase in staff resources is required for implementation.

232. Importance of sagittal alignment in cervical spondylotic myelopathy: an observational study from the Canadian Spine Outcomes and Research Network

BACKGROUND CONTEXT Recent evidence suggests that sagittal alignment may play a role in the presentation and management of patients with cervical spondylotic myelopathy (CSM) but its importance has not been fully established. PURPOSE Our objectives were to evaluate the extent to which (1) preoperative sagittal alignment is associated with baseline function and symptoms, (2) surgery leads to changes in sagittal alignment, and (3) postoperative sagittal alignment is associated with function and symptoms at follow-up. STUDY DESIGN/SETTING We performed an observational cohort study using prospectively collected data from the Canadian Spine Outcomes and Research Network (CSORN). PATIENT SAMPLE We included consecutive patients who were diagnosed with CSM by participating surgeons at seven centers between 2015 and 2017. OUTCOME MEASURES mJOA, NDI, VAS, SF12 and EQ5D. METHODS We measured sagittal alignment using two methods: surgeon classification and Ishihara's Cervical Curvature Index (ICCI). We measured function and symptoms with multiple Patient Reported Outcome Measures (PROMs), and we adjusted for age, gender, severity, and comorbidities using multiple linear regression. RESULTS Among 474 patients, baseline alignment was neutral in 196 (41%), lordotic in 190 (40%), and kyphotic in 88 (19%). In unadjusted analyses, baseline kyphosis was associated with significantly worse EuroQol 5D (p CONCLUSIONS In patients with CSM, baseline alignment is not associated with differences in impairment of health-related quality of life after adjusting for potential confounders. Surgery for CSM increases lordosis and improves symptoms and function regardless of baseline alignment, but postoperative sagittal alignment does not seem to be associated with final patient-reported outcomes. Our findings suggest that sagittal alignment restoration may not be critical for improving PROMs in the surgical management of patients with CSM, and that surgeons may prioritize the technical goals of neurological decompression and stabilization. FDA DEVICE/DRUG STATUS This abstract does not discuss or include any applicable devices or drugs.

32. Does the number of patient-reported allergies impact clinical outcomes after lumbar spinal fusion?

BACKGROUND CONTEXT A gap in the literature exists regarding the association of clinical and radiographic outcomes with specific number of allergies for patients undergoing lumbar spinal fusions; which is necessary to close in order to better understand the prognostic value of patient-reported allergies. PURPOSE The purpose of this present study was therefore to determine the impact of specific number of allergies with clinic and radiographic outcomes following lumbar spinal fusion. STUDY DESIGN/SETTING Retrospective cohort analysis. PATIENT SAMPLE Analysis of patients who underwent an open posterior lumbar fusion from 2011 to 2017 was conducted. Surgery was indicated after failure of conservative treatment to address radiculopathy and/or neurogenic claudication. Patients were excluded if they were under 18 years of age at the time of surgery, presented with a lumbar vertebral body fracture, tumor, or infection, or underwent a fusion surgery that extended to the thoracic spine, high-grade spondylolisthesis, or concomitant deformity. OUTCOME MEASURES Patient-reported outcomes were recorded at preoperative and final clinic visits that included: Visual Analog Scale (VAS) Back/Leg pain, and Oswestry Disability Index (ODI). Achievement of minimal clinically important difference (MCID) was analyzed, along with rates of postoperative complication and reoperation. METHODS Preoperative and final patient-reported outcomes were obtained. Achievement of MCID was evaluated using following thresholds: ODI 14.9, VAS-back pain 2.1, VAS-leg pain 2.8. For analysis, patients were divided into two groups based on number of allergies: less than three allergies and greater than or equal to three allergies. RESULTS A total of 504 patients met inclusion criteria. There were 472 (93.65%) patients who reported fewer than three allergies, while 32 (6.35%) patients presented with three or more allergies. Patients with three or more reported allergies were more often older (p=0.026), female (p CONCLUSIONS Patients with more than three reported allergies have significantly less improvement of ODI scores from preoperative to final assessment and have significantly high ODI MCID scores postoperatively despite no significant difference in psychiatric condition or radiographic measurements between the two groups. These results suggest a lower perceived improvement of disability in patients with more than three reported allergies when compared with patients with a lower number of allergies. This study underscores the importance of considering the number of allergies as a prognostic factor, similar to psychiatric disorders. Furthermore, these findings can be used in discussion with patients regarding expectations of disability improvement prior to surgery. FDA DEVICE/DRUG STATUS This abstract does not discuss or include any applicable devices or drugs.

Clostridium difficile Infection\u2013Daily Symptoms (CDI-DaySyms\u2122) questionnaire: psychometric characteristics and responder thresholds

BackgroundThe purpose of the current study was to determine the final content validation, psychometric characteristics, clinically meaningful improvement, and responder thresholds of the Clostridium difficile infection (CDI)–Daily Symptoms (CDI-DaySyms™) patient-reported outcome (PRO) questionnaire.MethodsThis validation study was part of two phase III studies (NCT01987895 and NCT01983683) conducted in patients with mild-to-moderate or severe CDI who completed the CDI-DaySyms™ daily throughout the treatment period. The questionnaire was evaluated in three stages: final PRO item content validation (Stage I); psychometric evaluation of reliability and construct validity (Stage II); and determination of clinically meaningful improvement and responder thresholds using distribution-based methods (Stage III).ResultsThe analysis included 168 patients. Most patients were female and Caucasian with mild-to-moderate CDI. The mean age was 57.1 years. Initial item analysis supported by confirmatory factor analysis demonstrated the relevance of 10 items grouped into three distinct domains (Diarrhea Symptoms, Abdominal Symptoms, and Systemic/Other Symptoms). Domain scores demonstrated acceptable internal consistency and test-retest reliability, were sensitive to change, and correlated in expected directions with other relevant symptom and disease-severity measures. Responder thresholds were defined as score changes of − 1.00, − 0.80, and − 0.70 in the Diarrhea Symptoms, Abdominal Symptoms, and Systemic/Other Symptoms domains, respectively.ConclusionsThe CDI-DaySyms™ is a valid measure of patient-reported CDI symptoms, with good measurement properties, which supports its utility as an endpoint in clinical studies. Further studies confirming responder thresholds based on anchor-based methods are required.Trial registrationNCT01987895, registered November 20, 2013; NCT01983683, registered November 14, 2013.

Bilateral Hip Arthroscopy: Can Results From Initial Arthroscopy for Femoroacetabular Impingement Predict Future Contralateral Results?

To determine the degree of correlation of radiographic measurements, degree of correlation of intraoperative pathology, and difference in outcomes between sides of patients requiring staged bilateral hip arthroscopy. Two high-volume hip preservation centers retrospectively reviewed hip preservation databases for staged bilateral hip arthroscopies conducted between 2008 and 2015. Patients were separated into those who presented with bilateral hip pain and those that presented with unilateral pain and developed contralateral pain >2years later. Patients were analyzed for radiographic correlation (alpha angle, lateral center edge angle, anterior center edge angle, magnetic resonance imaging alpha angle, Tönnis grade) and correlation of intraoperative pathology (acetabular labrum articular disruption grade, Outerbridge grade/location, Villar class ligamentum teres tears, labral tear location, symmetry of Seldes tear types, and the differences between operative procedures). Patient-reported outcomes were analyzed (modified Harris Hip Score, Non-Arthritic Hip Score, International Hip Outcome Tool-12, hip outcome score-sport specific subscale, visual analog scale, patient satisfaction). Correlative tests included Pearson and Spearman; univariate and multivariate analysis for differences included χ-square test and Student t tests for ordinal and continuous variables respectively. A total of 133 of 2,705 patients (4.6%) underwent bilateral hip arthroscopy. Radiographic alpha angle, magnetic resonance imaging alpha angle, lateral center edge angle, and anterior center edge angle demonstrated strong correlation (Pearson's coefficients 0.651, 0.648, 0.644, 0.667, respectively, P < .0001). Tönnis grade was weakly correlated (Pearson's coefficient 0.286, P= .001). Intraoperative pathology was moderately correlated (Pearson's coefficients for acetabular Outerbridge location, 0.300, P= .0170; acetabular labrum articular disruption, 0.490, P < .0001; acetabular Outerbridge; 0.530; P < .0001; femoral head Outerbridge, 0.459, P < .0001; Villar class, 0.393, P < .0001; and labral tear location, 0.468, P < .0001). Labral tear Seldes type was compared with Bowker's symmetry test and there was no significant difference between sides. There were no significant differences in surgical interventions performed between sides. Patients with bilateral hip arthroscopies significantly improved in all measured patient-reported outcomes and had a high patient satisfaction after both procedures. Final patient-reported outcomes and change in patient-reported outcomes were not different between procedures; follow up ranged from 3months to 8years. This study demonstrated an incidence of 4.6% of patients who require bilateral hip arthroscopy. These patients can expect significant improvement after surgical intervention. Patients that had 1 side done gained similar improvement when the contralateral side was performed. Preoperative radiographic, intraoperative pathology, and procedures performed were similar between hips. Level III, retrospective cohort study.

Arthroscopic hip surgery with a microfracture procedure of acetabular full-thickness chondral damage. Cohort study with a 3-year follow-up

Introduction and purposeManagement of injuries to the articular cartilage is complex and challenging. Our purpose was to assess outcomes of a cohort of patients who underwent hip arthroscopy with full-thickness chondral damage treated with microfracture and compare these outcomes with those from a similar cohort of patients who did not. Material and methodsWe prospectively gathered the data of 31 hips treated with microfracture from January 2009 to January 2016. In the cohort of hips without chondral damage there were 49 hips. All patients were assessed pre- and postoperatively with 4 patient-reported outcome instruments. ResultsThe mean follow-up was 36.2 months in the cohort of patients with full-thickness chondral damage, and 36.6 months in the cohort of patients without chondral damage. Both groups demonstrated significant improvement in all patient-reported outcome instruments between preoperative and final follow-up. There was no statistically significant difference between both cohorts at final follow-up. DiscussionThis study showed that patients undergoing microfracture during hip arthroscopy had significant improvement in all patient-reported outcome instruments during follow-up. The greatest improvement was noted at 6 months postoperatively. Both groups showed no significant difference in final patient-reported outcome instruments scores.

Tratamiento artroscópico mediante microfracturas de la lesión cartilaginosa acetabular de espesor completo. Estudio de cohortes con seguimiento medio a 3 años

Introduction and purposeManagement of injuries to the articular cartilage is complex and challenging. Our purpose was to assess outcomes of a cohort of patients who underwent hip arthroscopy with full-thickness chondral damage treated with microfracture and compare these outcomes with those from a similar cohort of patients who did not. Material and methodsWe prospectively gathered the data of 31 hips treated with microfracture from January 2009 to January 2016. In the cohort of hips without chondral damage there were 49 hips. All patients were assessed pre- and postoperatively with 4 patient-reported outcome instruments. ResultsThe mean follow-up was 36.2 months in the cohort of patients with full-thickness chondral damage, and 36.6 months in the cohort of patients without chondral damage. Both groups demonstrated significant improvement in all patient-reported outcome instruments between preoperative and final follow-up. There was no statistically significant difference between both cohorts at final follow-up. DiscussionThis study showed that patients undergoing microfracture during hip arthroscopy had significant improvement in all patient-reported outcome instruments during follow-up. The greatest improvement was noted at 6 months postoperatively. Both groups showed no significant difference in final patient-reported outcome instruments scores.

Ovarian cancer study dropouts had worse health-related quality of life and psychosocial symptoms at baseline and over time.

Participant drop out is a major barrier to high-quality patient-reported outcome (PRO) data analysis in cancer research as patients with worsening health are more likely to dropout. To test the hypothesis that ovarian cancer patients with worse PROs would drop out earlier, we examined how patients differed by time of dropout on health-related quality of life (HRQOL), anxiety, depression, optimism and insomnia. This analysis included 619 participants, stratified by time of dropout, from the Australian Ovarian Cancer Study - Quality of Life substudy, in which participants completed PRO questionnaires at three-monthly intervals for 21 months. Trends in PROs over time were examined. Pearson correlations examined the relationship between time of dropout and baseline PROs. Multiple linear regression models including age, disease stage and time since diagnosis examined relationships between baseline and final PRO scores, and final PRO scores and dropout group. Participants who dropped out earlier had significantly worse baseline HRQOL (p<0.0001) and higher depression (p<0.0001). For all five PROs, final scores were significantly associated with baseline scores (p<0.0001). Time of dropout was significantly associated with final HRQOL (p=0.003), anxiety (p=0.05), depression (p=0.02) and optimism (p=0.02) scores. Depression, HRQOL and anxiety worsened at a faster rate overtime in dropouts than study completers. Poorer HRQOL and higher depression at baseline, and final HRQOL, anxiety, depression and optimism scores were predictive of time of dropout. These results highlight the importance of collecting auxiliary data to inform careful and considered handling of missing PRO data during analysis, interpretation and reporting.

Microfracture in the Hip

Background: Microfracture in hip preservation surgery has demonstrated favorable outcomes, but studies with a higher level of evidence assessing microfracture are warranted. Purpose: To assess 2-year outcomes of patients who underwent hip arthroscopy with full-thickness chondral damage treated with microfracture and compare these outcomes with those of a control group from a similar cohort of patients who did not have full-thickness chondral damage and who were not treated with microfracture. Study Design: Cohort study; Level of evidence, 3. Methods: Between February 2008 and May 2012, prospectively gathered data for patients undergoing microfracture during hip arthroscopy with a 2-year follow-up were reviewed. All patients were assessed pre- and postoperatively at 3 months, 1 year, and 2 years with 4 patient-reported outcome (PRO) instruments. A matched cohort of patients who did not have full-thickness chondral damage and hence did not receive microfracture was selected on a 1:2 ratio. Matching criteria were sex, age within 6 years, workers’ compensation status, concomitant labral treatment, and radiographic parameters. Statistical analyses were performed to compare the change in PROs in both groups. Results: A total of 79 hips were included in the microfracture group and 158 in the control group. There was no significant difference in PRO scores preoperatively between the groups. Both groups demonstrated significant improvement in all postoperative PRO scores at all time points. There was no statistically significant difference in postoperative PRO scores between the microfracture and control groups, except for the visual analog scale (VAS) score at 2 years, which was higher (P = .02) in the microfracture group (mean ± SD, 3.63 ± 2.50) than in the control group (2.82 ± 2.76). Patient satisfaction was 7.2 for the microfracture group and 8.04 for the control group, which was statistically different (P < .05). The mean change in all PRO scores was similar between groups at 3 months and 1 year postoperatively but significantly lower in the microfracture group at 2 years postoperatively. The greatest improvement in both groups was noted at 3 months postoperatively. Conclusion: This study showed that patients undergoing microfracture during hip arthroscopy had equivalent PRO scores compared with the control group at 2 years postoperatively. The change in PRO scores from preoperatively to 2 years postoperatively was significantly lower in the microfracture group compared with the control group. The VAS scores and satisfaction were inferior by 0.81 and 0.84 units, respectively, in the microfracture group compared with the control group, likely due to lack of full-thickness chondral defects in the latter. However, both groups showed significant improvement in all PRO scores after surgery, with no significant difference in final PRO scores.

A Review of FDA Warning Letters and Notices of Violation Issued for Patient-Reported Outcomes Promotional Claims between 2006 and 2012

ObjectiveTo ascertain the frequency and types of patient-reported outcome (PRO) violations made in US pharmaceutical promotional materials between 2006 and 2012 and determine whether there were increases in violation warnings after issuance of the Food and Drug Administration (FDA) draft and final PRO Guidance. MethodsAll warning letters (WLs) or notices of violation (NOVs) issued by the FDA’s Office of Prescription Drug Promotion were reviewed for PRO violations (n = 213). Each letter containing a PRO violation was reviewed to determine the type of violation: 1) PRO measure not fit for purpose, 2) study design/interpretation of results, 3) statistical analysis, and 4) no treatment benefit. ResultsForty-one (19%) letters contained information about PRO infringements. Noticeable spikes in letters were shown in 2007 (37%) and 2010 (31%) after the issuance of the draft and final PRO Guidance, respectively. The most common violation was PRO measure not fit for purpose (54%), specifically: use of individual items (45%), insufficient evidence of content validity (36%), and broadening of the claim beyond what the PRO measures (27%). Issues with study design/interpretation of results were also high (49%), particularly broadening of claim beyond what was measured in the trial (55%) and no PRO measure used (50%). ConclusionsA fifth of the letters issued to companies contained PRO violations, with most related to poor selection of the PRO measure used or trying to broaden the claim. More guidance from the Office of Prescription Drug Promotion about what is considered “substantial evidence” in this area could help reduce the number of letters issued.

Oncology labels with clinical outcomes data since the final PRO guidance.

88 Background: FDA released draft Guidance on Patient-Reported Outcomes (PROs) in 2006 and finalized it in 2009. The Guidance applies equally to clinician and observer outcomes (ClinROs, ObsROs; collectively, clinical outcome assessments - COAs), and defines regulatory expectations for COA instruments in pivotal clinical trials and the inclusion of COA data in approved labeling. The purpose of this review was to survey labels for oncologic drugs approved in the three years since the “PRO Guidance” was finalized and to characterize any COAs in these labels. Methods: CenterWatch maintains a list of FDA approved drugs following definitions established by the Tufts Center for the Study of Drug Development, including only drugs or NMEs newly approved by the FDA Center for Drug Evaluation and Research. From 2010 through 2013, 45 oncologic drugs were approved by the FDA. The labels and other information from the Drugs@FDA website were reviewed. Results: Few of the approved labels included any reference to COAs or data collected using PROs, ClinROs, or ObsROs. Several (e.g., carfilzomib) had clear statements indicating approval for the product was based on a predefined response rate rather than “improvement in survival or symptoms.” The exceptions are fentanyl tablets and spray, and abiraterone, all with PRO data on pain. The number of PROs in the labels reviewed was low, but the trend suggests that products reviewed by Divisions outside the Office of Hematology and Oncology Products were more likely to have a PRO. Abiraterone was an exception as it was reviewed by Oncology Products I and included patient reported pain. Conclusions: There are several explanations for the low rate of COAs in oncology labels, e.g., oncology trials tend to be unblinded. The FDA has stated that open label designs cannot support PRO claims. Yet there is still a heavy reliance on outcomes such as progression free and overall survival. Three labels were identified with PRO data on pain, two of which were specifically indicated for pain rather than tumor control. This review suggests there are opportunities for sponsors and the FDA to increase the degree to which the patient’s voice is heard during the regulatory decision process, particularly, in programs reviewed by the oncology divisions.

Development of a New Patient-Reported Outcome Measure in Sarcopenia

The objective of this study was to develop a patient-reported outcome (PRO) to assess reduced muscle strength in sarcopenia. Qualitative research study. University of Arkansas for Medical Sciences. Subjects with sarcopenia. Adults aged 55 years and older with sarcopenia (n = 12) attended open-ended, concept elicitation interviews to characterize the functional effects of reduced muscle strength on their lives. The resulting qualitative data were analyzed using a qualitative analysis software program (Atlas.ti [Atlas.ti GmbH, Berlin, Germany]) and a common set of codes was developed to summarize the data. Subsequently, the initial PRO measure was drafted. Cognitive interviews were then conducted with additional sarcopenia subjects (n = 12) to refine the measure. Qualitative interviews identified key concepts (eg, impacts) in the areas of activities of daily living, emotions, social activities, energy, balance, coordination, sleep, and strength. Based on data from the cognitive debriefing interviews (eg, understandability, relevance, suggestions to reword items), the PRO measure development team came to consensus on which items or parts of the instructions to retain, revise, or delete. The final measure included 14 items. The final PRO measure, the Age-Related Muscle Loss Questionnaire, can be used in both clinical practice and clinical trial settings to assess functional impacts of reduced muscle strength in sarcopenia.