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The use of item libraries for patient-reported outcome (PRO) measurement in oncology allows for the customisation of PRO assessment to measure key health-related quality of life concepts of relevance to the target population and intervention. However, no high-level recommendations exist to guide users on the design and implementation of these customised PRO measures (item lists) across different PRO measurement systems. To address this issue, a working group was set up, including international stakeholders (academic, independent, industry, health technology assessment, regulatory, and patient advocacy), with the goal of creating recommendations for the use of item libraries in oncology trials. A scoping review was carried out to identify relevant publications and highlight any gaps. Stakeholders commented on the available guidance for each research question, proposed recommendations on how to address gaps in the literature, and came to an agreement using discussion-based methods. Nine primary research questions were identified that formed the scope and structure of the recommendations on how to select items and implement item lists created from item libraries. These recommendations address methods to drive item selection, plan the structure and analysis of item lists, and facilitate their use in conjunction with other measures. The findings resulted in high-level, instrument-agnostic recommendations on the use of item-library-derived item lists in oncology trials.

Background An important issue for patients with cancer treated with novel therapeutics is how they weigh the effects of treatment on survival and quality of life (QOL). We compared QOL in patients enrolled to SWOG S1400I, a substudy of the LungMAP biomarker-driven master protocol. Methods SWOG S1400I was a randomized phase III trial comparing nivolumab plus ipilimumab vs nivolumab for treatment of immunotherapy-naïve disease in advanced squamous cell lung cancer. The primary endpoint was the MD Anderson Symptom Inventory–Lung Cancer severity score at week 7 and week 13 with a target difference of 1.0 points, assessed using multivariable linear regression. A composite risk model for progression-free and overall survival was derived using best-subset selection. Results Among 158 evaluable patients, median age was 67.6 years and most were male (66.5%). The adjusted MD Anderson Symptom Inventory–Lung Cancer severity score was 0.04 points (95% confidence interval [CI] = −0.44 to 0.51 points; P = .89) at week 7 and 0.12 points (95% CI = −0.41 to 0.65; P = .66) at week 13. A composite risk model showed that patients with high levels of appetite loss and shortness of breath had a threefold increased risk of progression or death (hazard ratio [HR] = 3.06, 95% CI = 1.88 to 4.98; P Conclusions We found no evidence of a benefit of ipilimumab added to nivolumab compared with nivolumab alone for QOL in S1400I. A risk model identified patients at high risk of poor survival, demonstrating the prognostic relevance of baseline patient-reported outcomes even in those with previously treated advanced cancer.

This is a longitudinal prospective study that tracked multiple symptom burden and functioning status for bladder cancer (BLC) patients for 3 months post-radical cystectomy at The University of Texas MD Anderson Cancer Center, using a validated disease-specific patient-reported outcome measure (PROM) tool, the MD Anderson Symptom Inventory (the MDASI-PeriOp-BLC). The feasibility of collecting an objective measure for physical functioning, using “Timed Up & Go test” (TUGT) and PRO scores at baseline, discharge and end of study, was tested. Patients (n = 52) received care under an ERAS pathway. The more severe scores of fatigue, sleep disturbance, distress, drowsiness, frequent urination and urinary urgency at baseline predicted poor functional recovery postoperatively (OR = 1.661, 1.039–2.655, p = 0.034); other more severe symptoms at discharge (pain, fatigue, sleep disturbance, lack of appetite, drowsiness, bloating/abdominal tightness) predicted poor functional recovery (OR = 1.697, 1.114–2.584, p = 0.014) postoperatively. Compliance rates at preoperative, discharge and end of study were 100%, 79% and 77%, while TUGT completion rates were 88%, 54% and 13%, respectively. This prospective study found that more severe symptom burden at baseline and discharge is associated with poor functional recovery post-radical cystectomy for BLC. The collection of PROs is more feasible than using performance measures (TUGT) of function following radical cystectomy.

The aim of this study was to define a threshold of minimal clinically important improvement (MCII) for interpreting patient condition following video-assisted thoracoscopic surgery (VATS).Patients undergoing VATS were recruited for this multicenter, prospective, observational cohort study. Symptoms were measured using the MD Anderson Symptom Inventory-Lung Cancer Module perioperatively. To define MCIIs, we first identified index symptoms, defined as the most severe symptoms showing the largest reduction from day 1 post-surgery to discharge. MCIIs for each index symptom were then obtained via an anchor-based approach. Symptom recovery was defined as an MCII after post-surgery day 1. Cox regression models were used to identify risk factors for unrecovered index symptoms.Using 366 patients, we identified pain and fatigue as index symptoms after VATS. MCII was defined as a 30% reduction in pain or fatigue. At discharge, 22.6% of patients had not recovered from pain and 22.4% had not recovered from fatigue. Cox models found that risk factors for unrecovered pain were Charlson Comorbidity Index score ≥1 (hazard ratio [HR] 1.36, 95% confidence interval [CI] 1.04-1.77; p = 0.02) and preoperative neoadjuvant therapy (HR 2.78, 95% CI 1.13-6.83; p = 0.02). Malignancy was a risk factor for unrecovered fatigue (HR 1.47, 95% CI 1.02-2.13; p = 0.04).Pain and fatigue can be used as index measures for symptom recovery in patients following VATS. A 30% MCII represented meaningful recovery after VATS and could identify patients who may need extensive care after discharge.

ObjectiveCurrent gaps in knowledge limit clinicians from fully implementing patient-reported outcomes in routine post-operative care.MethodsThis prospective study assessed symptoms via the gynecologic module of the MD Anderson Symptom Inventory (MDASI-PeriOp-GYN) in patients who underwent open laparotomy.ResultsAt discharge, patient-reported moderate to severe (≥4 on a 0–10 scale) abdominal bloating or abdominal cramping, combined with length of stay of ≥4 days, were found to be associated with a higher risk of 30-day post-operative grade II–IV complications by the Clavien-Dindo system (all p values ConclusionThis study defined the clinically meaningful symptoms that related to the risk of developing important complications after discharge from major open gynecological surgery.These findings support the integration of assessment of patient-reported outcomes into patient-centered post-operative care.

Qingsong Yu,1 Hongfan Yu,1 Wei Xu,1 Yang Pu,1 Yuxian Nie,2 Wei Dai,3 Xing Wei,3 Xin Shelley Wang,4 Charles S Cleeland,4 Qiang Li,3 Qiuling Shi1,2,5 1School of Public Health and Management, Chongqing Medical University, Chongqing, People’s Republic of China; 2State Key Laboratory of Ultrasound in Medicine and Engineering, Chongqing Medical University, Chongqing, People’s Republic of China; 3Department of Thoracic Surgery, Sichuan Cancer Hospital & Institute, Sichuan Cancer Center, School of Medicine, University of Electronic Science and Technology of China, Chengdu, Sichuan, People’s Republic of China; 4Department of Symptom Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA; 5Center for Cancer Prevention Research, Sichuan Cancer Hospital & Institute, Sichuan Cancer Center, School of Medicine, University of Electronic Science and Technology of China, Chengdu, Sichuan, People’s Republic of ChinaCorrespondence: Qiuling Shi, School of Public Health and Management, Chongqing Medical University, No. 1, Medical School Road, Yuzhong District, Chongqing, 400016, People’s Republic of China, Tel +86-18290585397, Fax +86-28-85420116, Email qshi@cqmu.edu.cnPurpose: Patient-reported outcome (PRO)-based symptom assessment with a threshold can facilitate the early alert of adverse events. The purpose of this study was to determine whether shortness of breath (SOB) on postoperative day 1 (POD1) can inform postoperative pulmonary complications (PPCs) for patients after lung cancer (LC) surgery.Methods: Data were extracted from a prospective cohort study of patients with LC surgery. Symptoms were assessed by the MD Anderson Symptom Inventory-lung cancer module (MDASI-LC) before and daily after surgery. Types and grades of complications during hospitalization were recorded. SOB and other symptoms were tested for a possible association with PPCs by logistic regression models. Optimal cutpoints of SOB were derived, using the presence of PPCs as an anchor.Results: Among 401 patients with complete POD1 MDASI-LC and records on postoperative complications, 46 (11.5%) patients reported Clavien–Dindo grade II-IV PPCs. Logistic regression revealed that higher SOB score on POD1 (odds ratio [OR]=1.13, 95% CI=1.01– 1.27), male (OR=2.86, 95% CI=1.32– 6.23), open surgery (OR=3.03, 95% CI=1.49– 6.14), and lower forced expiratory volume in one second (OR=1.78, 95% CI=1.66– 2.96) were significantly associated with PPCs. The optimal cutpoint was 6 (on a 0– 10 scale) for SOB. Patients reporting SOB < 6 on POD1 had shorter postoperative length of stay than those reporting 6 or greater SOB (median, 6 vs 7, P =0.007).Conclusion: SOB on POD1 can inform the onset of PPCs in patients after lung cancer surgery. PRO-based symptom assessment with a clinically meaningful threshold could alert clinicians for the early management of PPCs.Keywords: lung cancer, surgery, postoperative pulmonary complications, patient-reported outcomes, shortness of breath

Background In clinical trials and clinical practice, patient-reported outcomes are almost always assessed using multiple patient-reported outcome measures at the same time. This raises concerns about whether patient responses are affected by the order in which the patient-reported outcome measures are administered. Methods This questionnaire-based study of order effects included adult cancer patients from five cancer centers. Patients were randomly assigned to complete questionnaires via paper booklets, interactive voice response system, or tablet web survey. Linear Analogue Self-Assessment, Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events, and Patient-Reported Outcomes Measurement Information System assessment tools were each used to measure general health, physical function, social function, emotional distress/anxiety, emotional distress/depression, fatigue, sleep, and pain. The order in which the three tools, and domains within tools, were presented to patients was randomized. Rates of missing data, scale scores, and Cronbach’s alpha coefficients were compared by the order in which they were assessed. Analyses included Cochran–Armitage trend tests and mixed models adjusted for performance score, age, sex, cancer type, and curative intent. Results A total of 1830 patients provided baseline patient-reported outcome assessments. There were no significant trends in rates of missing values by whether a scale was assessed earlier or later. The largest order effect for scale scores was due to a large mean score at one assessment time point. The largest difference in Cronbach’s alpha between the versions for the Patient-Reported Outcomes Measurement Information System scales was 0.106. Conclusion The well-being of a cancer patient has many different aspects such as pain, fatigue, depression, and anxiety. These are assessed using a variety of surveys often collected at the same time. This study shows that the order in which the different aspects are collected from the patient is not important.

Although pain is the most feared symptomatic component of having cancer and being treated for it, cancer-related fatigue is the most severe and impairing symptom that cancer patients and cancer survivors face. If not forewarned by their clinical team, the severity of cancer-related fatigue (CRF) may come as an unexpected consequence both disease and treatment. Although the construct of fatigue is multidimensional, having physical and emotional components and the inability to perform planned activities, most patients are able to rate the severity of their fatigue using brief patient reported outcome (PRO) scales. Increasingly, based on supportive care guidelines from different organizations, it is strongly recommended that screening for severity of fatigue be part of oncology clinical visits, and monitoring symptoms between clinic visits is feasible using web-based symptom tracking on smart phones. Fatigue is a common result of physical illness, and the mechanistic bases of patients having impairing fatigue are often multiple. The relationship between CRF and in ammation is the subject of much current investigation. When patients are able, based on their physical capacity, several studies have shown that exercise can be of bene t for improving fatigue and improving function. Behavioral coaching in scheduling activities may also be of bene t. The role of psychostimulants is still controversial, and more research is needed. Non-traditional interventions such as Ginseng and acupuncture have shown a positive signal in some trials. Especially considering the major burden of CSF to a high percentage of patients and its persistence in survivorship, more research in both the mechanisms and the treatment of CSF is needed.

Patients with multiple myeloma (MM) experience substantial cancer/treatment-related symptom burden during maintenance therapy. This is a phase II randomized, double-blinded, placebo-controlled clinical trial to examine the effect of minocycline for symptom reduction by its potential anti-inflammatory effect. Eligible MM patients for maintenance therapy were randomized to receive minocycline (100 mg twice daily) or placebo. The MD Anderson Symptom Inventory for MM (MDASI-MM) was used to assess multiple symptoms weekly during the trial. Clinician-rated toxicities and blood samples were prospectively collected. The effect size, area under the curve (AUC), and t tests were used to determine the symptom burden between treatment groups and identify the 5 most-severe MDASI-MM symptoms. The longitudinal analysis compared the changes in symptom severity and associated inflammatory markers between groups over time. Sixty-nine evaluable MM patients (33 from the intervention group and 36 from the placebo group) were included. No grade 3+ adverse events related to study medication were noted. The AUCs for the 5 worst MDASI-MM symptoms (fatigue, pain, disturbed sleep numbness/tingling, and drowsiness) were not significantly different between two arms. Regardless of group assignment, pain reduction was positively associated with decreased serum levels of soluble tumor necrosis factor-α receptors 1 and 2 during therapy (all P < 0.05). This pPhase II randomized study observed no statistically significant positive signal impact from minocycline on symptom reduction or inflammatory markers during maintenance therapy for MM, although using minocycline was feasible and had a low toxicity profile.

OBJECTIVE We sought to construct a valid and reliable patient-reported outcome measure for patients with advanced malignancy and GIO. BACKGROUND Bowel obstruction is the most common indication for palliative surgical consultation in patients with advanced cancer; however, no validated patient-reported outcome measures exist for this population. METHODS A total of 125 patients with GIO and 64 patients without GIO who underwent palliative surgical consultation completed the MDASI-GIO questionnaire and a single global quality-of-life question. Summary statistics were used to assess the symptom burden of GIO patients. Outcome measures were validity (construct and criterion) and reliability (internal and test-retest) for the MDASI-GIO. RESULTS The majority of patients rated the severity of each of the 5 following GIO-specific symptoms as moderate to severe (rating of ≥5 on a 0 to 10 scale): "being unable to eat" (72%), "being unable to have a bowel movement" (65%), "abdominal discomfort" (62%), "stomach feeling full" (55%), and "abdominal cramping" (54%). The MDASI-GIO subscale Cronbach coefficient alpha values were 0.80-0.91, and intraclass correlations were 0.72-0.84. Correlations between MDASI-GIO subscales and global quality of life were -0.39 to -0.49 (P < 0.001 for all comparisons). GIO patients had significantly worse symptoms and higher interference than did non-GIO patients (all P < 0.05) with effect-size differences of ≥0.36, supporting known-group validity. CONCLUSIONS The MDASI-GIO shows initial validity and reliability for assessing the severity of symptoms of patients with GIO and the interference of these symptoms in patients' daily functioning.

Relapsed or refractory multiple myeloma (RRMM) is labeled “symptomatic” based on laboratory values, but not relevant to quantitative measure of patient’s perspectives. This study aimed to describe symptom burden, health status, and quality of life in RRMM patients. The cross-sectional study included 184 MM patients (141 RRMM cases and 43 MM patients on follow-up without diagnosis/treatment of RRMM disease as controls), while 64 RRMM patients also provided longitudinal patient-reported outcomes (PROs) data. Symptomatic status was based on clinical measures of disease activity. PROs included the MD Anderson Symptom Inventory multiple myeloma module (MDASI-MM), single-item quality of life (SIQOL), and EuroQol-5D (EQ-5D). Wilcoxon rank test and effect size were used for comparisons. Regression models were used to describe symptom trajectory and to identify predictors of high symptom burden during 3 months of RRMM therapy. Most patients were clinically identified as symptomatic (93%). RRMM patients tended to report more severe symptoms, with significantly lower QOL scores and more severe fatigue, poor appetite, and lower enjoyment of life compared with controls (all p

Objective The MD Anderson Symptom Inventory for Brain Tumor (MDASI-BT) module is a widely used instrument for measuring symptom burden and interference of daily activities in brain tumor patients. This study aims to develop and validate its Japanese version (MDASI-BT-Japanese). Methods Following forward and backward translation of the original MDASI-BT into Japanese, understandability and feasibility were assessed by cognitive debriefing. Subsequently, patients with brain tumors were asked to fill out MDASI-BT-Japanese and European Quality of Life-5 Dimensions (EQ-5D). Feasibility, reliability and validity of MDASI-BT-Japanese were assessed. Results Cognitive debriefing confirmed overall ease of completion and good understandability. The study population composed of 140 patients with brain tumors (most commonly gliomas). The mean symptom severity score and mean interference score were 1.9 ± 1.7 and 2.8 ± 2.7, respectively. The top items included distress and drowsiness for symptom severity and general activity and work for interference. The median time required was 4 minutes (range, 0.5–30), and missing values were seen in 1%. Internal consistency was proven by excellent Cronbach’s coefficient alpha (0.94 for symptom severity, 0.92 for interference). Test–retest reliability was assessed with acceptable intra-class correlation coefficient (mean, 0.76). Correlation efficient ranged between 0.7 and 0.9 for convergent validity. Known-group validity was confirmed by significantly different mean symptom severity score and mean interference score among patients with different performance status. As evidence of concurrent validity, MDASI-BT-Japanese correlated with EQ-5D in the hypothesized magnitude and direction. Conclusions The newly developed MDASI-BT-Japanese has demonstrated feasibility, reliability and validity in evaluation of clinical benefit in Japanese-speaking brain tumor patients.

Background The validation of the Treatment-induced Neuropathy Assessment Scale (TNAS v2.0), a patient-reported outcome measure of symptoms associated with cancer treatment-induced peripheral neuropathy (TIPN), was previously reported. Further patient input (qualitative interviewing, cognitive debriefing) suggested that the measure should be modified to better reflect the TIPN experience. We report the performance of a revised version (TNAS v3.0) for assessing TIPN across cancer treatments. This TNAS version incorporates extensive patient input, in accordance with FDA guidance on the development of patient-reported outcomes measures. Patients with multiple myeloma, colorectal cancer, or gynecological cancer treated with bortezomib, oxaliplatin, or taxane–platinum combination therapy, respectively, completed the TNAS v3.0, European Organization for Research and Treatment of Cancer Chemotherapy-Induced Peripheral Neuropathy (EORTC-CIPN20), and a cognitive debriefing survey during a scheduled clinic visit. Patients also participated in in-depth qualitative interviews about their TIPN symptoms. The psychometric properties of the TNAS v3.0 were evaluated. Results Cognitive debriefing survey results were summarized and showed that most patients found the items easy to complete, comprehensible, acceptable, and not redundant. A notable change from TNAS v2.0 was the separation of “numbness” from “tingling,” although these 2 items remained the most severe, followed by a new “pain” item. The Cronbach coefficient alphas for the 9-item TNAS were 0.88 and 0.90 at the first and second administrations, respectively, indicating good reliability. The test–retest reliability of the TNAS was 0.97. The correlation coefficients for the 9-item TNAS and the EORTC-CIPN20 were 0.69 for the sensory subscale, 0.70 for the motor subscale, and 0.32 for the autonomic subscale, indicating good validity. Conclusion This psychometric evaluation showed that the TNAS v3.0 is valid and reliable. Further research is needed to determine clinically meaningful differences in TNAS v3.0 scores and demonstrate its responsiveness over time.

Purpose In patients with non-small cell lung cancer (NSCLC), concurrent chemoradiation therapy (CRT) exacerbates a cluster of difficult-to-manage symptoms, especially cancer-related fatigue. Minocycline is a readily available, low-cost antibiotic with antiinflammatory properties. We conducted a phase 2 randomized, double-blinded, placebo-controlled trial to investigate the effect of minocycline in reducing CRT-symptom burden in NSCLC. Methods and Materials Patients with NSCLC scheduled to receive CRT provided consent and were randomized to receive either minocycline (100 mg twice daily) or a matching placebo during 6 to 7 weeks of CRT. Patient-reported fatigue and other symptoms were assessed on MD Anderson Symptom Inventory weekly from the start of CRT for 12 weeks. The primary outcome was 12-week (±2 days) area under the curve for symptom burden, which was compared between treatment groups. Results Forty of 49 enrolled patients (80%) were evaluable (19 on minocycline and 21 on placebo). There were no grade 3 + adverse events related to the study medication. Fatigue was significantly reduced in the minocycline group compared with placebo group during the 12-week trial period (area under the curve = 31.2 ± 14.2 vs 45.0 ± 20.9, P = .011), with a large effect size (Cohen’s d = 0.77). Pain (Cohen’s d = 0.54) and shortness of breath (Cohen’s d = 0.55) were also significantly reduced in the minocycline group (all P Conclusions Minocycline during CRT for NSCLC was feasible, had a low toxicity profile, and yielded a clinically and statistically significant positive signal in reducing symptom burden related to NSCLC and CRT. This study is a proof of concept so a larger trial in CRT patients is warranted.

CONTEXT: Increasing numbers of oncology therapies are being approved based on early-phase single-arm studies. Yet, little is known regarding the use of patient-reported outcomes in single-arm oncology trials testing novel therapies. OBJECTIVES: We examined patient-reported symptom severity and symptom interference with activity- (WAW: work, general activity, walking) and mood-(REM: relations with others, enjoyment of life, mood) related functioning, and their association with factors known to influence symptom severity reporting in early-phase clinical trials clinic patients. METHODS: Patients completed the validated MD Anderson Symptom Inventory, containing 13 severity items and six interference items, each rated on a 0–10 scale (higher scores=worse symptom severity/interference). Performance status (ECOG-PS) and age were ascertained. Multiple linear regression was performed. RESULTS: In 248 phase I patients (51% female, 90% ECOG 0–1, and 74% ≤65 years), 67% of patients had ≥seven concurrent symptoms of any severity level, and 51% of patients described ≥three concurrent symptoms as moderate-to-severe (severity rating≥5). Composite symptom severity, WAW and REM were worse in patients with ECOG-PS≥2 vs. 0–1, and worse in patients with ECOG-PS=1 than in patients with ECOG-PS=0. Compared with patients over 65y, adolescent and young adult (AYA) patients (18y-39y) and patients aged 40y to 65y had worse composite symptom severity. As expected, being employed full-time/retired was associated with better symptom profiles in phaseI patients. CONCLUSION: The variation of symptom burden by performance status and age suggest that these factors need to be considered in the design of early-phase trials, particularly if patient-reported symptoms are used as primary/secondary/exploratory endpoints.

Fatigue is the most reported and most distressing symptom among patients with cancer. However, no questionnaire that measures fatigue and fatigue interference with life has been translated into Arabic.This study aimed to translate and validate the Arabic version of the Brief Fatigue Inventory (BFI-A).The BFI was translated into Arabic using the forward-backward translation technique. This cross-sectional study collected data from cancer patients through a self-administered questionnaire that included the BFI-A, Insomnia Severity Index (ISI), Zung Depression Scale (ZDS), MD Anderson Symptom Inventory total score (MDASI), and Medical Outcome Study Short Form 36 (SF-36) Vitality Subscale. Descriptive and inferential statistics were used including mean, standard deviation, internal consistency, and correlation coefficient using Pearson's correlation.A total of 79 patients were recruited in Amman, Jordan, in 2015. Mean of the total BFI-A was 4.01 (2.4), showing that 83.5% had nonsevere fatigue. Cronbach's α coefficient of the BFI-A was 0.93. The correlations between total BFI-A scores and BFI-A items were significant (P0.05) and ranged from 0.75 to 0.86. BFI-A showed a significant correlation (P0.05) with the following tools: ISI = 0.70, ZDS = 0.69, MDASI = 0.75, and SF-36 Vitality Subscale = -0.57.This study suggests that the BFI-A is a reliable and valid tool to assess fatigue among Arab cancer patients.دراسة للتحقق من النسخة العربية لمقياس التعب الوجيز (BFI–A).خالد سليمان، محمود الكلالدة، لؤي أبو شحرور، برنيس ييتس، آن بيرجر، تيتو مندوزا، ملكة ملك، أيمن بني سلامة، شارلز كليلاند، أحمد المنشاوي.يُعدّ التعب أكثر الأعراض التي يشكو منها مرضى السرطان وأكثرها تكديراً لهم. ومع ذلك، لم يُترجَم إلى اللغة العربية أي استبيان يقيس التعب ومدى إعاقته لحياة المريض.كانت هذه الدراسة تهدف إلى ترجمة "مقياس التعب الوجيز" (BFI-A) والتحقق من نسخته العربية.تُرجم المقياس إلى اللغة العربية بطريقة الترجمة المباشرة والعكسية. وجمعت هذه الدراسة المقطعية بيانات من مرضى السرطان من خلال استبيان ملأه المرضى بأنفسهم وتضمَّن "مقياس التعب الوجيز" (BFI-A)، و"مؤشر شدة الأرق" (ISI)، و"مقياس زونج للاكتئاب" (ZDS)، ومجموع درجات "قائمة أعراض" (MDASI)، و"المقياس الفرعي للحيوية في دراسة النتائج الطبية المختصرة المكونة من 36 بنداً" (SF-36). واستُخدمت إحصاءات وصفية واستنتاجية، منها المتوسط، والانحراف المعياري، والاتساق الداخلي، ومعامل الارتباط باستخدام ارتباط بيرسون.استعين اجمالا بتسعة وسبعين مريضا في مدينة عمان الاردنية عام 2015. وكان متوسط مجموع درجات « مقياس التعب الوجيز (BFI-A) هو 4.1)، مما يدل على ان 83.5٪ كانوا يعانون تعبا غير شديد. وكان معامل كرونباخ الفا لمقياس التعب الوجيز هو 0.93. وكانت علاقات الترابط بين مجموع درجات « مقياس التعب الوجيز » وبنود هذا المقياس علاقات وثيقة (0.05P)، وتراوحت من 0.75 الى 0.86. وتبين وجود ترابط وثيق (0.05P) بين « مقياس التعب الوجيز BFI-A)» والمقاييس التالية: مؤشر شدة الارق (0.70) (ISI)، ومقياس زونج للاكتئاب (0.69) (ZDS)، وقائمة الاعراض (0.75) (MDASI)، والمقياس الفرعي للحيوية في دراسة النتائج الطبية المختصره المكونه من 36 بندا (0.57-) (SF-36).تشير هذه الدراسة إلى أن (BFI-A) أداة موثوقة وصالحة لتقييم التعب لدى مرضى السرطان الناطقين بالعربية.Étude de validation de la version arabe du Brief Fatigue Inventory (BFI-A).La fatigue est le symptôme le plus fréquent et le plus éprouvant chez les patients atteints de cancer. Cependant, aucun questionnaire mesurant la fatigue et son interférence avec la vie quotidienne n’a été traduit en arabe.La présente étude visait à traduire et à valider la version arabe du Brief Fatigue Inventory (BFI-A).Le BFI a été traduit en arabe à l’aide de la technique de traduction/rétro-traduction. La présente étude transversale a permis de collecter des données de patients atteints de cancer en recourant à un auto-questionnaire comprenant le BFI-A, l’index de Sévérité de l’Insomnie (ISI), l’échelle d’auto-évaluation de la dépression de Zung (ZDS), l’inventaire des symptômes M.D. Anderson (MDASI) et la sous-échelle vitalité de la forme abrégée du questionnaire généraliste SF-36 (qualité de vie) du Medical Outcome Study. Des statistiques descriptives et inférentielles ont été utilisées, y compris la moyenne, l’écart type, la cohérence interne et le coefficient de corrélation de Pearson.79 patients au total ont été recrutés à Amman (Jordanie) en 2015. La moyenne totale du BFI-A était de 4,01 (2,4), indiquant que 83,5 % des patients présentaient un état de fatigue non sévère. Le coefficient alpha de Cronbach du BFI-A était de 0,93. Les corrélations entre les scores totaux et les rubriques du BFI-A étaient significatives (p0,05) et étaient comprises entre 0,75 et 0,86. Le BFI-A affichait une corrélation importante (p0,05) avec les instruments suivants : ISI = 0,70 ; ZDS = 0,69 ; MDASI = 0,75 et la sous-échelle vitalité du SF-36 (qualité de vie) = −0,57.La présente étude suggère que le BFI-A est un outil valable et fiable pour évaluer la fatigue chez les patients arabes atteints de cancer.

Context The most debilitating symptoms during oxaliplatin-based chemotherapy in patients with colorectal cancer (CRC) are neuropathy and fatigue. Inflammation has been suggested to contribute to these symptoms, and the anti-inflammatory agent minocycline is safe and readily available. Objectives This proof-of-concept study investigated minocycline's capacity to reduce treatment-related neuropathy and fatigue and its impact on inflammatory markers during chemotherapy in a Phase II randomized, double-blind, placebo-controlled clinical trial. Methods Patients with locally advanced or metastatic CRC who were scheduled for oxaliplatin-based chemotherapy were randomly assigned to receive either minocycline (100 mg twice daily) or placebo over four months from started chemotherapy. Toxicity assessments and blood samples were prospectively collected monthly. The severity of fatigue and numbness/tingling was assessed weekly using the MD Anderson Symptom Inventory. The primary endpoint, area under the curve for numbness/tingling and fatigue over approximately four months, was compared between the two arms. Results Of 66 evaluable participants, 32 received minocycline and 34 placebo. There was no observed significant symptom reduction on both fatigue and numbness/tingling in either arm, nor was there a difference in levels of serum proinflammatory and anti-inflammatory markers between arms. No Grade 3 adverse events nor disparity mediating effects on intervention were observed. Conclusion Minocycline treatment is feasible and has a low-toxicity profile. However, with 200 mg/day, it did not reduce numbness/tingling or fatigue nor moderate inflammatory biomarkers from this Phase II randomized study. Our results do not support further exploration of minocycline for fatigue or neuropathy symptom intervention in patients treated for CRC.

BACKGROUND While women diagnosed with breast cancer have increased survival when compared with other cancers, survivorship may include residual symptom burden from treatment and continuing endocrine therapies. OBJECTIVE The objective of this study was to identify subgroups of breast cancer survivors experiencing similar symptom severity. METHODS Participants were 498 women with breast cancer, not on active treatment. Symptom severity was self-reported using the MD Anderson Symptom Inventory. Target symptoms were included in a latent profile analysis. Factors related to subgroup membership and differences in quality of life (QOL) and functioning were explored using logistic regression. RESULTS Mean age was 60.11 (SD, 11.32) years, 86.1% were white, and 79.1% were receiving endocrine therapy. Target symptoms included fatigue (reported at ≥5 by 22.8% of women), sleep disturbance (24.8%), and trouble remembering (17.2%). Two subgroups were identified: low symptom severity (77.0% of women) and high (23.0%). Older women (odds ratio [OR], 0.971; 95% confidence interval [CI], 0.952-0.989) and employed women (OR, 0.621; 95% CI, 0404-0.956) were less likely to be in the high subgroup; women with poorer performance status (OR, 1.653; 95% CI, 1.188-2.299) were more likely to be in the high subgroup. Women in the high subgroup reported lower QOL (P = .000) and greater interference with functioning (P = .000). CONCLUSIONS Two subgroups of women with distinct symptom severity were identified. IMPLICATIONS FOR PRACTICE Identification of women at risk for high symptoms during survivorship may allow clinicians to intensify their approach to symptom management, thereby mitigating poor outcomes and impairments in QOL.

Background The complexity of disease- and treatment-related symptoms causes profound distress and deterioration of health-related quality of life among patients with brain tumors. Currently, there is no Danish validated disease-specific instrument that focuses solely on measures of both neurologic and cancer-related symptoms of patients with brain tumors. The MD Anderson Symptom Inventory Brain Tumor Module (MDASI-BT) is a validated patient self-report questionnaire that measures symptom prevalence, intensity, and interference with daily life. The aim of the present study was to determine the psychometric validity of the Danish translation of the MDASI-BT, and to test its utility in 3 cohorts of Danish patients across the spectrum of the brain cancer disease and treatment trajectory. Methods A linguistic validation process was conducted. Danish patients with malignant primary brain tumors were included to establish the psychometric validity and reliability of the Danish MDASI-BT. Cognitive debriefing interviews were conducted to support the psychometric properties. Results A total of 120 patients participated in this study. Coefficient αs for the symptom and interference subscales indicate a high level of reliability across all items. Corresponding symptom and interference or functional items and subscales in the MDASI-BT and European Organisation for Research and Treatment of Cancer Brain Tumor Module BN20 were significantly correlated. Cognitive debriefing provided evidence for content validity and questionnaire utility as participants were comfortable answering the questions and had no problem with the understandability or number of questions asked. Conclusion The MDASI-BT is a simple, concise symptom assessment tool useful for assessing the symptom severity and interference of Danish-speaking patients with brain cancer.

In recent years, the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Adult Cancer Pain have undergone substantial revisions focusing on the appropriate and safe prescription of opioid analgesics, optimization of nonopioid analgesics and adjuvant medications, and integration of nonpharmacologic methods of cancer pain management. This selection highlights some of these changes, covering topics on management of adult cancer pain including pharmacologic interventions, nonpharmacologic interventions, and treatment of specific cancer pain syndromes. The complete version of the NCCN Guidelines for Adult Cancer Pain addresses additional aspects of this topic, including pathophysiologic classification of cancer pain syndromes, comprehensive pain assessment, management of pain crisis, ongoing care for cancer pain, pain in cancer survivors, and specialty consultations.

Background Symptom assessment requires psychometrically validated questionnaires that are easy to use, relevant to the disease, and quick to administer. The MD Anderson Symptom Inventory for malignant pleural mesothelioma (MDASI-MPM) was adapted from the general (core) MDASI to assess the severity of cancer-related and treatment-related symptoms specific to patients with this condition. The MDASI-MPM includes the 13 core MDASI symptoms, which are experienced by most cancer patients, and 6 MPM-specific items developed via qualitative interviewing, a method favored by the US Food and Drug Administration for instrument item generation and development. Qualitative interviewing that summarizes the item generation and development for the MDASI-MPM is detailed in a separate report. The psychometric study reported here was the next step in developing the validation dossier for the MDASI-MPM. Results In this secondary analysis of data from a Phase II trial, 248 patients provided MDASI-MPM data at multiple timepoints during therapy. Over time, fatigue, pain, shortness of breath, feeling of malaise, and muscle weakness were consistently the worst symptoms reported; symptoms interfered most with work and general activity and least with relations with others. Cronbach coefficient alpha values for all MDASI-MPM subscales were at least 0.88 at baseline and 0.91 during treatment, indicating good internal consistency reliability. Intraclass correlations of at least 0.86 for all MDASI-MPM subscales administered a cycle apart (n = 82) were indicative of good test-retest reliability. Correlations between MDASI-MPM subscales and LCSS-Meso scores were at least 0.70 (P

Attribution of adverse events (AEs) is critical to oncology drug development and the regulatory process. However, processes for determining the causality of AEs are often sub-optimal, unreliable, and inefficient. Thus, we conducted a toxicity-attribution workshop in Silver Springs MD to develop guidance for improving attribution of AEs in oncology clinical trials. Attribution stakeholder experts from regulatory agencies, sponsors and contract research organizations, clinical trial principal investigators, pre-clinical translational scientists, and research staff involved in capturing attribution information participated. We also included patients treated in oncology clinical trials and academic researchers with expertise in attribution. We identified numerous challenges with AE attribution, including the non-informative nature of and burdens associated with the 5-tier system of attribution, increased complexity of trial logistics, costs and time associated with AE attribution data collection, lack of training in attribution for early-career investigators, insufficient baseline assessments, and lack of consistency in the reporting of treatment-related and treatment-emergent AEs in publications and clinical scientific reports. We developed recommendations to improve attribution: we propose transitioning from the present 5-tier system to a 2-3 tier system for attribution, more complete baseline information on patients' clinical status at trial entry, and mechanisms for more rapid sharing of AE information during trials. Oncology societies should develop recommendations and training in attribution of toxicities. We call for further harmonization and synchronization of recommendations regarding causality safety reporting between FDA, EMA and other regulatory agencies. Finally, we suggest that journals maintain or develop standardized requirements for reporting attribution in oncology clinical trials.

Objectives Cutpoints are specific numeric values used to create discrete categories for patient-reported outcome (PRO) items or scales. Cutpoints are widely used in both clinical research and practice. This article offers a definition for cutpoints, describes strategies for determining actionable cutpoints, and discusses considerations related to interpreting cutpoints in clinical applications. Methods We clarify the definition of cutpoints for PRO measures and summarize the major statistical approaches for identifying cutpoints, including multivariate analysis of variance and receiver operating characteristic and regression modeling. Discussion We review issues related to cutpoint determination and interpretation that should be considered when integrating PROs into clinical research and practice, including the selection of anchors, variability of cutpoints, and clinical burden that may be generated when a cutpoint is used as a threshold for further clinical action. Key points Cutpoints are widely used to categorize PRO responses in both clinical research and practice. Cutpoints can be derived for PRO measures regardless of the response scale used; however, the mild, moderate, and severe categories generated from numeric cutpoints are distinct from the mild, moderate, and severe categories found in some PRO measures that use verbal descriptors as response options. Bootstrap analysis is recommended to quantify the variability of cutpoints. The application of cutpoints is limited by how well the anchors are chosen and how cutpoints developed using group-level data are applied at the individual level.

PURPOSE: Local/systemic symptoms during cancer therapy may be exacerbated by dysregulated inflammation and its downstream toxic effects. Minocycline can suppress proinflammatory cytokine release; therefore, we investigated its potential to reduce patient-reported symptom severity during radiotherapy (RT) for head and neck cancer (HNC). METHODS: Eligible patients for this blinded, placebo–controlled trial were adults with T0–3, N-any, and M0 HNC receiving single-modality RT. Participants were randomized 1:1 to either minocycline (200 mg/day) or placebo during RT. The primary endpoint was the area under the curve (AUC) of 5 prespecified symptoms (pain, fatigue, disturbed sleep, poor appetite, difficulty swallowing/chewing) during RT, assessed with the MD Anderson Symptom Inventory for HNC (MDASI-HN). RESULTS: We analyzed data from 20 evaluable patients per arm. Overall, 75% had oropharyngeal cancer and 78% were male. No grade 3+ adverse events potentially related to study medication were observed. Two minocycline patients required a feeding tube during RT vs 5 placebo patients (P = 0.21). The average daily AUC during RT for the 5 MDASI-HN symptoms was 3.1 (SD =1.0) for minocycline and 3.7 (SD = 1.7) for placebo (P = 0.16); the 0.37 effect size was less than our 0.70 target. AUC comparisons for several individual symptoms and symptom interference favored minocycline but were not statistically significant. The greatest numerical differences occurred for systemic symptoms, larger toward treatment end, and in early post-RT recovery. CONCLUSIONS: Minocycline was feasible, well tolerated, and achieved a positive signal toward reducing patient-reported symptom severity during RT for HNC, particularly for systemic symptoms. This justifies additional study and informs future trial design.

OBJECTIVE: Using patient-reported outcomes (PROs) in perioperative care is increasingly common. We report the development, validation, and application of an MD Anderson Symptom Inventory version for use in patients undergoing surgery for gynecologic cancer or benign conditions (MDASI-PeriOp-GYN). METHODS: Our process included: (1) generating PeriOp-GYN–specific candidate items from qualitative interviews with patients, followed by input from an expert panel; (2) dropping items that lacked independent clinical relevance; (3) validating psychometric properties (reliability, validity) of the resulting MDASI-PeriOp-GYN; and (4) conducting cognitive debriefing interviews with patients to confirm ease of comprehension, relevance, and acceptability. RESULTS: Qualitative interviews with 40 patients generated 9 new PeriOp-GYN symptom items (bloating, abdominal cramping, constipation, hot flashes, dizziness, grogginess/confusion, urinary pain, difficulty urinating, and diarrhea) that, along with the core MDASI items, formed the new MDASI-PeriOpGYN. A total of 150 patients (minimally invasive surgery (MIS)=69, open surgery=81) participated in the validation study; 123 also provided retest data. Cronbach alphas were 0.89 for symptoms and 0.86 for interference. Test-retest reliability was 0.88 for all symptom severity items. Known-group validity was supported by the detection of significant differences in symptom and interference levels by performance status (P

Patients with newly diagnosed lung cancer who have not yet begun treatment may already be experiencing major symptoms produced by their disease. Understanding the symptomatic effects of cancer treatment requires knowledge of pretreatment symptoms (both severity and interference with daily activities). We assessed pretreatment symptom severity, interference, and quality of life (QOL) in treatment-naive patients with lung cancer and report factors that correlated with symptom severity. This was a retrospective analysis of data collected at initial intake. Symptoms/interference were rated on the MD Anderson Symptom Inventory (MDASI) between 30 days prediagnosis and 45 days postdiagnosis. We examined symptom severity by disease stage and differences in severity by histology. Linear regression analyses identified significant predictors of severe pain and dyspnea. Of 460 eligible patients, 256 (62%) had adenocarcinoma, 30 (7%) had small cell carcinoma, and 100 (24%) had squamous cell carcinoma; > 30% reported moderate-to-severe (rated ≥ 5, 0–10 scale) pretreatment symptoms. The most-severe were fatigue, disturbed sleep, distress, pain, dyspnea, sadness, and drowsiness. Symptoms affected work, enjoyment of life, and general activity (interference) and physical well-being (QOL) the most. Patients with advanced disease (n = 289, 63%) had more-severe symptoms. Cancer stage was associated with pain severity; both histology and cancer stage were associated with severe dyspnea. One third of lung cancer patients were symptomatic at initial presentation. Quantification of pretreatment symptom burden can inform patient-specific palliative therapy and differentiate disease-related symptoms from treatment-related toxicities. Poorly controlled symptoms could negatively affect treatment adherence and therapeutic outcomes.

PURPOSE Based on the MD Anderson Symptom Inventory (MDASI), we developed a Patient-reported outcomes tool for hepatectomy perioperative care (MDASI-PeriOp-Hep). METHODS To establish the content validity, we generated PeriOp-Hep-specific candidate items from qualitative interviews of patients (n = 30), and removed items that lacked clinical relevance on the basis of input from panels of patients, caregivers, and clinicians. The psychometric properties of the MDASI-PeriOp-Hep were validated (n = 150). The cognitive debriefing and clinical interpretability were assessed to confirm the ease of comprehension, relevance, and acceptability of the tool. RESULTS Five symptoms specific to hepatectomy (abdominal bloating, tightness, or fullness; abdominal cramping; muscle weakness, instability, or vertigo; constipation; and incisional tightness) were identified as module items to form the MDASI-PeriOp-Hep. The Cronbach αs for symptoms and for interference were 0.898 and 0.861, respectively. The test-retest reliability was 0.887 for all 18 symptom severity items. Compared to other commonly used tools, correlation of MDASI-PeriOp-Hep scores to performance status (all, P

Purpose Symptom monitoring and management using patient-reported outcome (PRO) measures improves outcomes for individuals with cancer. The purpose of the current study was to provide a qualitative assessment of preferences of individuals with cancer for PRO measures. Participants & setting 15 patients receiving systemic therapy at the University of Texas MD Anderson Cancer Center. Methodologic approach Participants completed three PRO measures. Qualitative interviews were conducted, and content analysis was used to identify relevant themes. Findings Identified themes were the importance of communicating various aspects of the disease and treatment experience to the oncology team, the importance of systematic PRO assessments, congruence among PRO questionnaires and questions clinicians ask at clinic visits, concerns about the length of PRO questionnaires, the importance of the response options available in PRO questionnaires, and willingness to complete PRO measures frequently. Implications for nursing Oncology nurses are critical facilitators of the systematic use of PRO measures across the cancer care continuum.

OBJECTIVE: To evaluate the utility of patient-reported outcomes (PROs) to measure physical functioning in perioperative care for patients with gynecological (GYN) tumors. METHODS: 180 patients with GYN tumors undergoing open surgery participated in this longitudinal study. The physical functioning was measured by a subjective PRO tool, the Interference subscales of the MD Anderson Symptom Inventory (MDASI-I); as well as by an objective tool, the Timed Up & Go test (TUGT), perioperatively. Longer time (>20 seconds) needed to complete the TUGT was defined as “Prolonged”. Patients completed EuroQoL-5D as well. The association between the scores of MDASI-I items and TUGT was assessed via the Spearman correlation coefficient. The known-group validity was assessed using the t-test and Cohen’s D effect size. RESULTS: Compliance rates at preoperative, discharge and postoperative time points of MDASI-I were 98%, 95%, 96%; while TUGT completion rates were 92%, 75%, and 80%, respectively. Patients who had refused TUGT at discharge reported a significantly worse “MDASI-general activity” score compared to patients who completed TUGT (mean score of 7.00 vs. 5.38, P=0.020). Patient-reported “Walking” on MDASI-I significantly differentiated patients with prolonged vs. those with frail/normal TUGT at discharge (mean score of 4.89 vs. 2.79, Cohen’s d effect size = 0.82, P < 0.001) MDASI-I demonstrated excellent known-group validity per performance status and for the EuroQoL-5D subscales. CONCLUSION: Patient-reported physical functioning impairment after GYN surgery correspond with observed worse scores of the objective functioning measure test (TUGT). MDASI-I assessment represents a feasible and valid tool to evaluate functional status and warrants further implementation in the perioperative setting.

IntroductionImmunotherapies have revolutionized the treatment of various cancers, but little is known about their symptomatic toxicity. Assessing these symptoms is best accomplished by asking the patients themselves. However, such reports are subjective and may face challenges as bonafide scientific data. Demonstrating the validity of symptom assessment tools, mainly through the reduction of measurement errors, has the potential to improve patient care if these tools are widely adopted. To that end, we present herein the psychometric properties of the Immunotherapy for Early-Phase Trials module of the MD Anderson Symptom Inventory (MDASI-Immunotherapy EPT) in patients receiving various immunotherapies in early phase trials at a major cancer center.MethodsOne hundred forty-five patients completed the inventory at baseline, with 85 of them also doing so after 9 weeks of treatment. The mean (±SD) age of the patients was 57.0±12.9 years. Also, 56% of the patients were women, 79% identified as white, and 49% had at least some college education.ResultsThe internal consistency reliability of the MDASI-Immunotherapy EPT was excellent, as the Cronbach’s alphas for all of its subscales were at least 0.88 (range 0.88–0.95). Known-group validity based on Eastern Cooperative Oncology Group performance status groupings was excellent at 9 weeks after the start of an immunotherapy trial for the MDASI-Immunotherapy EPT severity (effect size, 0.96) and interference (effect size, 0.82) subscales. We found substantial changes in the symptom items difficulty remembering (effect size, −0.85), fever and/or chills (effect size, −0.63), disturbed sleep (effect size, −0.52), diarrhea (effect size, −0.42), and swelling of hands, legs, or feet (effect size, −0.39).ConclusionsIn conclusion, the MDASI-Immunotherapy EPT is a valid, reliable, and sensitive tool for measuring symptomatic toxicity.

PURPOSE: Symptom monitoring has demonstrated improved outcomes in patients with cancer, including quality of life, resource utilization, ability to continue treatment, and survival. The use of disease-specific patient-reported outcome (PRO) measures facilitates symptom monitoring. While the MD Anderson Symptom Inventory–Lung Cancer (MDASI-LC), a PRO measure of symptom burden in lung cancer, is psychometrically validated for use in patients with NSCLC, its content validity has not been verified through direct patient input. Our purpose is to describe the symptom experience of patients with NSCLC and to confirm that the MDASI-LC contains the key symptoms specific to NSCLC from the patient perspective. METHODS: Patients with NSCLC described their symptom experience in single qualitative interviews. Content analysis was used to define the content domain for a PRO measure of NSCLC symptom burden. RESULTS: Participants (N = 40) had a mean age of 66.1 years (standard deviation, 10.9 years); 60.0% were male, 77.5% were White, and 56.4% had stage IV disease. Thirty-two symptoms were described, with 6 reported by ≥ 20% of participants. Symptom variations were noted by treatment modality but not by stage of disease. Patients with NSCLC commonly reported shortness of breath, cough, distress, fatigue, pain, and constipation. In patients receiving chemotherapy, treatment-related symptoms, including neuropathy and sore mouth, were commonly noted. The presence of these symptoms resulted in interference with daily activities, relationships, life plans, treatment adherence, and mood. CONCLUSION: The symptoms included in the MDASI-LC are important components of the content domain of an NSCLC symptom burden measure. The presence of these symptoms affect daily life and, therefore, is of clinical consequence.

PURPOSE: Symptom monitoring is attracting attention as a way to improve adherence to cancer therapy, reduce treatment-related toxicities, and possibly improve overall survival. How reporting thresholds affect symptom alert generation and clinical outcomes is poorly understood. PATIENTS AND METHODS: We analyzed data from 38 US health care institutions collected for the prospective Eastern Cooperative Oncology Group–American College of Radiology Imaging Network E2Z02 Symptom Outcomes and Practice Patterns study. Participants were outpatients receiving chemotherapy for breast (n = 642), colorectal (n = 486), or lung cancer (n = 340) who rated symptom severity using the MD Anderson Symptom Inventory at 2 assessment points 1 month apart. Percentages of patients with pain, dyspnea, fatigue, or distress at different thresholds (score of 4-7 on a 0-10 scale) were compared. The percentage of patients whose performance status had worsened at follow-up was used to estimate risk for missing clinically important symptom data by using higher severity thresholds RESULTS: At the guideline-recommended threshold of ≥ 4, suprathreshold rates were 60% for any of the 4 symptoms at the initial survey; performance status worsened at follow-up for 27% of all patients with any symptom rated ≥ 4 at the initiate survey. When the threshold was increased to ≥ 7, approximately half of patients (51%) with worsened performance status were not identified. CONCLUSION: The burden to clinicians from an alert threshold of ≥ 4 (per many current guidelines) would be substantial. However, setting higher alert thresholds may miss a large percentage of patients who need clinical intervention. These results may inform resource planning when implementing electronic symptom screening at an institutional or practice level.

Patient-reported outcomes (PROs), such as symptoms, function, and other health-related quality-of-life aspects, are increasingly evaluated in cancer randomised controlled trials (RCTs) to provide information about treatment risks, benefits, and tolerability. However, expert opinion and critical review of the literature showed no consensus on optimal methods of PRO analysis in cancer RCTs, hindering interpretation of results. The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium was formed to establish PRO analysis recommendations. Four issues were prioritised: developing a taxonomy of research objectives that can be matched with appropriate statistical methods, identifying appropriate statistical methods for PRO analysis, standardising statistical terminology related to missing data, and determining appropriate ways to manage missing data. This Policy Review presents recommendations for PRO analysis developed through critical literature reviews and a structured collaborative process with diverse international stakeholders, which provides a foundation for endorsement; ongoing developments of these recommendations are also discussed.

Context Although it is well known that patients with advanced pancreatic cancer (PC) experience significant symptom burden, few strategies for effective symptom intervention are available for them. Objectives To investigate the efficacy of minocycline, an anti-inflammatory agent, for symptom reduction in patients with advanced PC. Methods We conducted Phase II, randomized, and placebo-controlled trial to obtain preliminary estimates of the effects on symptom reduction with 100 mg of minocycline or placebo given twice a day. Eligible patients had diagnosed advanced PC and were scheduled for standard chemotherapy. Patient-reported symptoms were measured weekly during the eight-week trial using the MD Anderson Symptom Inventory (MDASI) module in patients with gastrointestinal cancer. The primary outcome measure was the area under the curve values of the five most severe symptoms in the two arms. Results Of the 44 patients recruited, 31 (71%) were evaluable for the primary efficacy analysis, with 18 received minocycline and 13 placebo. Fatigue, pain, disturbed sleep, lack of appetite, and drowsiness were the most severe symptoms reported by both groups. No significant differences in area under the curve values over time between the study arms were found for the composite MDASI score or single-item scores of the five most severe MDASI items. No treatment-related deaths were reported, and no Grade 3–4 toxicities were observed. Conclusion Minocycline is safe for use in patients receiving treatment for PC. There is no observed symptom reduction with minocycline on the major symptom burden associated with advanced PC compared with placebo. Attrition because of rapid disease progression impacted the study significantly.

PURPOSE: Patient-reported outcome measures (PROMs) that assess how patients feel and function have potential for evaluating quality of care. Stakeholder recommendations for PRO-based performance measures (PMs) were elicited, and feasibility testing was conducted at six cancer centers. METHODS: Interviews were conducted with 124 stakeholders to determine priority symptoms and risk adjustment variables for PRO-PMs and perceived acceptability. Stakeholders included patients and advocates, caregivers, clinicians, administrators, and thought leaders. Feasibility testing was conducted in six cancer centers. Patients completed PROMs at home 5-15 days into a chemotherapy cycle. Feasibility was operationalized as ≥ 75% completed PROMs and ≥ 75% patient acceptability. RESULTS: Stakeholder priority PRO-PMs for systemic therapy were GI symptoms (diarrhea, constipation, nausea, vomiting), depression/anxiety, pain, insomnia, fatigue, dyspnea, physical function, and neuropathy. Recommended risk adjusters included demographics, insurance type, cancer type, comorbidities, emetic risk, and difficulty paying bills. In feasibility testing, 653 patients enrolled (approximately 110 per site), and 607 (93%) completed PROMs, which indicated high feasibility for home collection. The majority of patients (470 of 607; 77%) completed PROMs without a reminder call, and 137 (23%) of 607 completed them after a reminder call. Most patients (72%) completed PROMs through web, 17% paper, or 2% interactive voice response (automated call that verbally asked patient questions). For acceptability, > 95% of patients found PROM items to be easy to understand and complete. CONCLUSION: Clinicians, patients, and other stakeholders agree that PMs that are based on how patients feel and function would be an important addition to quality measurement. This study also shows that PRO-PMs can be feasibly captured at home during systemic therapy and are acceptable to patients. PRO-PMs may add value to the portfolio of PMs as oncology transitions from fee-for-service payment models to performance-based care that emphasizes outcome measures.