There is paucity of evidence-based data regarding the impact of therapies on quality of life and symptom burden of patients with RRMM. Inclusion of PRO in future studies of patients with RRMM is needed to better inform clinical decision-making.

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Some concerns have been raised about potential bias in patient-reported outcome (PRO) results from open-label cancer randomized controlled trials (RCTs). We investigated if open-label trials favor the experimental treatment over the standard treatment more frequently than blinded trials. We also examined if the effect of treatment concealment differs for distal vs more proximal PROs. We assessed 538 RCTs with a PRO endpoint conducted in the most prevalent cancers, of which 366 (68.0%) were open-label, 148 (27.5%) were blinded, and 24 (4.5%) were categorized as unclear. In our multivariable logistic regression model, we did not observe a statistically significant association of the independent variable treatment concealment (open-label vs blinded) on the dependent variable measuring the proportion of trials favoring the experimental treatment (adjusted odds ratio = 1.19, 95% confidence interval = 0.79 to 1.79, 2-sided P = .40. This was also the case when comparing distal and proximal PROs. Our findings provide novel evidence-based data that support the validity of PRO results from open label cancer RCTs.

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Myelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis and blood cytopenia with a variable risk of progression to acute myeloid leukemia. The main goal of therapy for the large majority of patients is to improve health-related quality of life (HRQoL). Its rigorous assessment is now recommended in international MDS guidelines. Our review provides an overview of HRQoL results from randomized controlled trials (RCTs) in MDS patients. The literature search undertaken in PubMed identified 10 RCTs with HRQoL endpoints (all secondary) published between August 2008 and September 2020. These RCTs have helped to better understand the impact of therapies from the patient perspective and have generated valuable information that can be used to further support clinical decisions. However, the number of RCTs in MDS patients, including HRQoL endpoints, is still low. Given the importance of symptom relief and HRQoL improvement in the treatment of MDS patients, the assessment of the patient perspective in future RCTs is highly recommended to keep expanding the knowledge of the impact of new MDS therapies.

Patient-reported Outcome (PRO) measures used in cancer research can assess a number of health domains. Our primary objective was to investigate which broad types of PRO domains, Functional health, Symptoms, and Global Quality of Life (QoL), most frequently yield significant differences between treatments in randomized controlled trials (RCTs). MethodsTwo-hundred-twenty-nine RCTs published between January 2004 and February 2019, conducted on patients diagnosed with most common solid malignancies, and using the EORTC QLQ-C30 questionnaire were considered. Studies were identified systematically using literature searches in key electronic databases. Unlike other PRO measures typically used in RCTs, the scoring algorithm of the multidimensional EORTC QLQ-C30 allows to clearly distinguish the three broad types of PRO domains. ResultsOne hundred thirty-four RCTs (58•5%) reported statistically significant differences between treatment arms for at least one of the QLQ-C30 domains. Most frequently differences were reported for two or all three broad types of PRO domains (78 of 134 trials, 58•2%). In particular, 35 trials (26•1%), found significant differences for Symptoms, Functional health, and Global QoL, 24 trials (17•9%) for Symptoms and Functional health, 11 trials (8•2%) for Functional health and Global QoL, and 8 trials (6•0%) for Symptoms and Global QoL. The likelihood of finding a statistically significant difference between treatment arms was not associated with key study characteristics, such as study design (i.e., open-label vs. blinded trials) and industry support. ConclusionsOur findings emphasize the importance of a multidimensional PRO assessment to most comprehensively capture the overall burden of therapy from the patients' standpoint. Highlights

Digital health tools are increasingly being used in cancer care and may include electronic patient-reported outcome (ePRO) monitoring systems. We examined physicians’ perceptions of usability and clinical utility of a digital health tool (GIMEMA-ALLIANCE platform) for ePRO monitoring in the real-life practice of patients with hematologic malignancies. This tool allows for the collection and assessment of ePROs with real-time graphical presentation of results to medical staff. Based on a predefined algorithm, automated alerts are sent to medical staff. Participating hematologists completed an online survey on their experience with the platform. Of the 201 patients invited to participate between December 2020 and June 2021 (cut-off date for current analysis), 180 (90%) agreed to enter the platform and had a median age of 57 years. Twenty-three hematologists with a median age of 42 years and an average of 17 years of experience in clinical practice were surveyed. All hematologists agreed or strongly agreed that the platform was easy to use, and 87%, agreed or strongly agreed that ePROs data were useful to enhance communication with their patients. The majority of physicians (78%) accessed the platform at least once per month to consult the symptom and health status profile of their patients. The frequency of access was independent of physician sex (p=0.393) and years of experience in clinical practice (p=0.404). In conclusion, our preliminary results support the clinical utility, from the perspective of the treating hematologist, of integrating ePROs into the routine cancer care of patients with hematologic malignancies.

Background Given the growing importance of patient‐reported outcomes (PROs) as part of “big data” in improving patient care, there is a need to provide a state‐of‐the‐art picture of the added value of using PROs in prostate cancer (PCa) randomized controlled trials (RCTs). We aimed to synthetize the most recent high‐quality PRO evidence‐based knowledge from PCa RCTs and to examine whether quality of PRO reporting in PCa research improved over time. Methods We conducted a systematic literature search using PubMed, from April 2012 until February 2019. For benchmarking purposes, we also included RCTs identified in our previously published review of RCTs (2004‐2012). Methodology for study identification and evaluation followed standardized criteria and a predefined data extraction form was used to abstract information. PRO quality of the studies was evaluated using the International Society of Quality of Life Research (ISOQOL) recommended criteria. Results A total of 55 new RCTs were published between April 2012 and February 2019. About 24 (43.6%) RCTs were found to be of high‐quality regarding PRO assessments. Of these, 13 (54.2%) have been reported in the most recent European Association of Urology (EAU) PCa Guidelines. Overall QoL and sexual, urinary, and bowel function were the most commonly reported PROs. FACT‐P, EPIC‐26, and EORTC QLQ‐C30 and/or its module PR25 were most frequently used as measurement tools. An overall improvement in the completeness of PRO reporting was noted over time. Conclusion Many PRO trials are currently not included in the EAU guidelines. Our findings suggest that there has to be a better consensus on the use of PRO data for PCa patients, which will then be reflected in the PCa Guidelines and future data collection. Homogeneity in PROs collection and measurement tools will in turn enable “big data” Consortia to increase the patients’ voice in clinical research.