Patient-Reported Outcome Measures in Cancer Care: An Updated Systematic Review and Meta-Analysis.

Importance: Patient-reported outcome measures (PROMs) come directly from the patient, without clinician interpretation, to provide a patient-centered perspective. Objective: To understand the association of PROM integration into cancer care with patient-related, therapy-related, and health care utilization outcomes. Data Sources: Searches included MEDLINE and MEDLINE Epub ahead of print, in-process, and other nonindexed citations; Embase databases (OvidSP); PsychINFO; CENTRAL; and CINAHL from January 1, 2012 to September 26, 2022. Study Selection: Randomized clinical trials (RCTs) that enrolled adult patients (ages 18 years and older) with active cancer receiving anticancer therapy using a PROM as an intervention. Data Extraction and Synthesis: Pairs of review authors, using prepiloted forms, independently extracted trial characteristics, disease characteristics, and intervention details. The Preferred Reporting Items for Systematic Reviews and Meta-Analyses reporting guideline was followed. Random-effects analyses were conducted. Main Outcomes and Measures: Overall mortality, health-related quality of life (HRQoL) measures, and hospital utilization outcomes. Results: From 1996 to 2022, 45 RCTs including 13 661 participants addressed the association of PROMs with outcomes considered important to patients. The addition of a PROM likely reduced the risk of overall mortality (HR, 0.84; 95% CI, 0.72-0.98; moderate certainty), improved HRQoL (range 0-100) at 12 weeks (mean difference [MD], 2.45; 95% CI, 0.42-4.48; moderate certainty). Improvements of HRQoL at 24 weeks were not significant (MD, 1.87; 95% CI, -1.21 to 4.96; low certainty). There was no association between the addition of a PROM and HRQoL at 48 weeks. The addition of a PROM was not associated with reduced ED visits (OR, 0.74; 95% CI, 0.54-1.02; low certainty) or hospital admissions (OR, 0.86; 95% CI, 0.73-1.02; low certainty). Conclusion and Relevance: The findings of this study suggest that the integration of PROMs into cancer care may improve overall survival and quality of life.

A Prognostic Survival Model Incorporating Patient-Reported Outcomes for Transplant-Ineligible Patients With Multiple Myeloma.

Developing prognostic tools specifically for patients themselves represents an important step in empowering patients to engage in shared decision-making. Incorporating patient-reported outcomes may improve the accuracy of these prognostic tools. We conducted a retrospective population-based study of transplant-ineligible (TIE) patients with multiple myeloma (MM) diagnosed between January 2007 and December 2018. A multivariable Cox regression model was developed to predict the risk of death within 1-year period from the index date. We identified 2356 patients with TIE MM. The following factors were associated with an increased risk of death within 1 year: age > 80 (HR 1.11), history of heart failure (HR 1.52), "CRAB" at diagnosis (HR 1.61), distance to cancer center (HR 1.25), prior radiation (HR 1.48), no proteosome inhibitor/immunomodulatory therapy usage (HR 1.36), recent emergency department (HR 1.55) or hospitalization (HR 2.13), poor performance status (ECOG 3-4 HR 1.76), and increasing number of severe symptoms (HR 1.56). Model discrimination was high with C-statistic of 0.74, and calibration was very good. To our knowledge, this represents one of the first prognostic models developed in MM incorporating patient-reported outcomes. This survival prognostic tool may improve communication regarding prognosis and shared decision-making among older adults with MM and their health care providers.

Important questions for the malignant hematologist to consider when designing or evaluating a study with patient-reported outcome measures (PROMs).

Patient-reported outcome measures (PROMs), which are measures of symptom burden, health-related quality of life (HRQoL), and therapy effectiveness have become increasingly important in clinical research. They are unique in that they are reported directly from the patient, without clinician interpretation, thereby avoiding clinician bias. With an increased focus on the patient at the center of health care, PROMs have been increasingly incorporated into clinical research, systematic reviews, and clinical guidelines. Despite the recognition of the importance of including PROMs into clinical haematologic cancer research, barriers have prevented their integration into cancer research. This review highlights the value of including PROMs into clinical haematologic cancer research and addresses the methodological challenges in using and evaluating PROMs. We propose important questions for the malignant haematologist to consider when designing or evaluating a study that includes PROMs.

Trajectory of Symptoms in Patients Undergoing Autologous Stem Cell Transplant for Multiple Myeloma: A Population-Based Cohort Study of Patient-Reported Outcomes.

BACKGROUND: Autologous stem cell transplant (ASCT) is an established treatment for patients with newly diagnosed multiple myeloma (NDMM). Understanding the symptom burden associated with ASCT may be an important consideration for patients with NDMM when selecting treatment options. PATIENTS AND METHODS: We conducted a population-based study of patients who underwent an ASCT for NDMM in Ontario, Canada, between 2007 and 2018. The patient-reported outcome, Edmonton Symptom Assessment System (ESAS) score, which captures nine common cancer-associated symptoms and is routinely collected at all outpatient visits, was linked to provincial administrative healthcare data. The monthly prevalence of moderate or severe symptoms (ESAS ≥ 4) each month in the first year following ASCT was analyzed. A multivariable logistic regression model was used to identify factors associated with moderate to severe symptoms. RESULTS: In our final cohort of 1969 patients who had undergone an ASCT, a total of 12,820 unique assessments were captured. Symptom burden was highest at 1 month post-ASCT, with moderate to severe tiredness and impaired well-being being the two most common symptoms. Symptom burden substantially improved by 3 months post-ASCT, reaching a new baseline for the year following. On multivariable analysis, female sex, increased co-morbidities, earlier year of diagnosis, and myeloma-related end-organ damage (specifically, bone and kidney disease) were associated with a higher odds of reporting moderate to severe symptoms. CONCLUSION: In this large population-based study using patient-reported outcomes, there was a substantial burden of symptoms noted among NDMM patients 1 month post-ASCT, which improved over time. Tailored supportive care interventions should focus on strategies to optimize management of identified symptoms.

Exploring the components of bleeding outcomes in transfusion trials for patients with hematologic malignancy.

Clinically significant bleeding in patients with hematologic malignancies is a heterogeneous composite outcome currently defined as World Health Organization (WHO) bleeding Grades 2, 3, and 4. However, the clinical significance of some minor bleeds categorized as WHO Grades 1 and 2 remains controversial. We analyzed the number and frequency of individual signs and symptoms of WHO Grades 1 and 2 bleeds and explored their association with more severe incident bleeds graded as WHO Grades 3 and 4. STUDY DESIGN AND METHODS: We aggregated daily bleeding assessment data from three randomized controlled trials conducted in patients with hematologic malignancies that used bleeding as an outcome. Cox proportional hazard regression analysis was used to identify signs and symptoms categorized as WHO Grades 1 and 2 bleeds that were associated with more severe bleeds (Grades 3 and 4). RESULTS: We collected data from 315 patients (n = 5476 daily bleeding assessments; 3383 [61.8%] with a bleed documented). A total of 98.3% (3326/3383) were Grade 1 and 2 bleeds and 1.7% (57/3383) were Grades 3 and 4. Grade 1 and 2 bleeds were composed of 20 different bleeding signs and symptoms. Hematuria (hazard ratio, 16.1; 95% confidence interval, 4.4-59.2; P < .0001) was associated with incident Grade 3 or 4 bleeds. CONCLUSION: In patients with hematologic malignancy, only hematuria (microscopic and/or macroscopic) was associated with more severe incident bleeds. This findings require validation in independent data sets.

Maintenance therapy in transplant ineligible adults with newly-diagnosed multiple myeloma: A systematic review and meta-analysis.

BACKGROUND: The role of maintenance therapy in transplant ineligible multiple myeloma (MM) patients following a period of fixed duration induction therapy remains unclear. OBJECTIVES: We performed a systematic review and meta-analysis of randomized controlled trials (RCTs) examining maintenance therapy compared to observation. METHODS: We conducted a comprehensive search including MEDLINE, Embase, and the Cochrane database up to February 28, 2020, for RCTs comparing maintenance therapy to observation in newly diagnosed transplant ineligible MM patients. Two authors independently screened studies for eligibility, extracted data, and assessed risk of bias. We performed meta-analyses using a random-effects model and assessed certainty using GRADE methodology. MAIN RESULTS: We included five RCTs with a total of 1139 patients. Patients receiving maintenance therapy had improved progression-free survival (PFS) compared to observation (Hazard ratio [HR] 0.48, 95% confidence interval [CI] 0.38 to 0.62, high certainty); however, there was no difference in overall survival (HR 0.96, 95% CI 0.76-1.2, moderate certainty). Adverse events were higher in the maintenance group compared to observation (very low to moderate certainty). CONCLUSION: Maintenance therapy increases PFS in transplant ineligible MM patients following a fixed period of induction therapy; however, this must be weighed against the increased risk of adverse events.

Thrombovascular events affect quality of life in patients with systemic lupus erythematosus.

OBJECTIVE: To compare health-related quality of life (HRQOL) of patients with antiphospholipid syndrome (APS) and systemic lupus erythematosus (SLE) with and without previous thrombovascular events (TE). METHODS: The Medical Outcomes Study Short-Form 36 (SF-36) was used to assess HRQOL in 5 patient groups: (1) primary APS (PAPS; n = 35); (2) APS associated to SLE (SAPS; n = 37); (3) SLE+TE without persistent positive antiphospholipid antibody (SLE+TE-aPL; n = 75); (4) SLE-TE+aPL (n = 71); and (5) SLE-TE-aPL (n = 608). RESULTS: The data on both mental component summary and physical component summary (PCS) scores showed an impaired quality of life in all patient groups. Patients in the SLE+TE-aPL group had a lower PCS score compared to patients in the SLE-TE+aPL group. CONCLUSION: The combination of SLE and TE has a more negative influence on reported HRQOL, compared to having SLE or APS alone.