TY - JOUR
T1 - Guidelines for the Use and Reporting of Patient-Reported Outcomes in Multiple Myeloma Clinical Trials
AU - Laane, Edward
AU - Salek, Sam
AU - Oliva, Esther Natalie
AU - Bennink, Christine
AU - Clavreul, Solène
AU - Richardson, Paul G.
AU - Scheid, Christof
AU - Weisel, Katja
AU - Ionova, Tatyana
AU - on behalf of the European Hematology Association Specialized Working Group on Quality of Life and Symptoms
N1 - Publisher Copyright:
© 2023 by the authors.
PY - 2023/12/8
Y1 - 2023/12/8
N2 - In the era of personalized medicine there is an increasing need for the assessment of patient-reported outcomes (PROs) to become a standard of patient care. Patient-reported outcome measures (PROM) are important in assessing significant and meaningful changes as a result of an intervention based on a patient’s own perspective. It is well established that active multiple myeloma (MM) can be characterized by a high burden of disease and treatment-related symptoms, with considerable worsening of quality of life (QoL). In general, and over the past decade, the focus has shifted to obtaining the most durable remissions with the best QoL as primary goals for MM treatment. Patients place considerable value on their QoL and communicating about QoL data prior to treatment decisions allows them to make informed treatment choices. Consequently, optimization of QoL of patients with MM is an important therapeutic goal and the incorporation of PROs into clinical trials has the potential of improving treatment outcomes. In this regard, guidance for the use and reporting of PROMs in MM in clinical trials is warranted. Under the auspices of the European Hematology Association, evidence-based guidelines for the use and reporting of PROs in patients with MM have been developed according to the EHA’s core Guidelines Development Methodology. This document provides general considerations for the choice of PROMs in MM clinical trials as well as a series of recommendations covering a selection of PROMs in MM clinical trials; the mode of administration; timing of assessments; strategies to minimize missing data; sample size calculation; reporting of results; and interpretation of results.
AB - In the era of personalized medicine there is an increasing need for the assessment of patient-reported outcomes (PROs) to become a standard of patient care. Patient-reported outcome measures (PROM) are important in assessing significant and meaningful changes as a result of an intervention based on a patient’s own perspective. It is well established that active multiple myeloma (MM) can be characterized by a high burden of disease and treatment-related symptoms, with considerable worsening of quality of life (QoL). In general, and over the past decade, the focus has shifted to obtaining the most durable remissions with the best QoL as primary goals for MM treatment. Patients place considerable value on their QoL and communicating about QoL data prior to treatment decisions allows them to make informed treatment choices. Consequently, optimization of QoL of patients with MM is an important therapeutic goal and the incorporation of PROs into clinical trials has the potential of improving treatment outcomes. In this regard, guidance for the use and reporting of PROMs in MM in clinical trials is warranted. Under the auspices of the European Hematology Association, evidence-based guidelines for the use and reporting of PROs in patients with MM have been developed according to the EHA’s core Guidelines Development Methodology. This document provides general considerations for the choice of PROMs in MM clinical trials as well as a series of recommendations covering a selection of PROMs in MM clinical trials; the mode of administration; timing of assessments; strategies to minimize missing data; sample size calculation; reporting of results; and interpretation of results.
UR - http://www.scopus.com/inward/record.url?scp=85180643421&partnerID=8YFLogxK
U2 - 10.3390/cancers15245764
DO - 10.3390/cancers15245764
M3 - Article
C2 - 38136310
AN - SCOPUS:85180643421
SN - 2072-6694
VL - 15
JO - Cancers
JF - Cancers
IS - 24
M1 - 5764
ER -