Raising the standards of patient‐centered outcomes research in myelodysplastic syndromes: Clinical utility and validation of the subscales of the QUALMS from the MDS‐RIGHT project

Efficace, Fabio; Koinig, Karin A.; Cottone, Francesco; Bowen, David; Mittelman, Moshe; Sommer, Kathrin; Langemeijer, Saskia; Culligan, Dominic; Filanovsky, Kalman; Storck, Michael; Smith, Alexandra; Marrewijk, Corine van; Dugas, Martin; Stojkov, Igor; Siebert, Uwe; Witte, Théo de; Stauder, Reinhard

doi:10.1002/cam4.5487

Cited by 6 publications

(12 citation statements)

References 42 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The majority of patients were enrolled in Italian centers thereby hampering a thorough analysis on potential differences by geographical regions. Also, our HRQoL profile description is confined to aspects measured by the EORTC QLQ-C30 and other MDS-specific HRQoL questionnaires [36][37][38] could have revealed additional valuable information.…”

Section: Discussionmentioning

confidence: 99%

Health-related Quality of Life Profile of Newly Diagnosed Patients With Myelodysplastic Syndromes by Age, Sex, and Risk Group: A Real-world Study by the GIMEMA

Efficace,

Al Essa,

Platzbecker

et al. 2023

HemaSphere

Self Cite

View full text Add to dashboard Cite

Health-related quality of life (HRQoL) is an important goal of therapy for patients with myelodysplastic syndromes (MDS); however, little is known about HRQoL of these patients at clinical presentation. We report HRQoL profile of newly diagnosed patients with MDS across both the the International Prognostic Scoring System (IPSS) and IPSS-Revised (IPSS-R) classifications, stratified by sex and age group categories, aiming to also establish European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core30 (EORTC QLQ-C30) reference values for these patients. Analysis was based on 927 patients with a median age of 73.3 years (interquartile range, 66.0–79.2), of whom 506 and 421 with lower- and higher-risk disease respectively, according to the IPSS classification. HRQoL was assessed with the EORTC QLQ-C30 and substantial differences by age groups and sex, between and within lower- and higher-risk disease categories were observed. For example, within higher-risk disease patients, the youngest group (ie, 30–59 years) tended to report clinically meaningful worse outcomes across various functional and symptom domains compared with older age groups. We also developed 2 regression models allowing for the prediction of EORTC QLQ-C30 reference scores for patients classified according to either the IPSS or the IPSS-R. Investigation of prevalence rates for clinically important problems and symptoms at diagnosis revealed a substantial burden of the disease with >50% of patients reporting clinically important problems with physical functioning and dyspnea in both lower- and higher-risk disease. Our findings may help to enhance the interpretation of HRQoL outcomes in future MDS studies and to better contextualize HRQoL data from routine practice settings.

show abstract

Section: Discussionmentioning

confidence: 99%

Health-related Quality of Life Profile of Newly Diagnosed Patients With Myelodysplastic Syndromes by Age, Sex, and Risk Group: A Real-world Study by the GIMEMA

Efficace,

Al Essa,

Platzbecker

et al. 2023

HemaSphere

Self Cite

View full text Add to dashboard Cite

show abstract

Section: Key Selected Aspects To Consider During Protocol Development...mentioning

confidence: 99%

“…For example, the recently developed QUALMS has three subscales assessing physical burden (QUALMS‐P), emotional burden (QUALMS‐E), and benefit finding (QUALMS‐BF), plus a total QUALMS score. 64 , 65 Therefore, a hypothetical trial using only this questionnaire should clearly identify which specific scale will be used as the primary endpoint of the PRO analysis. If a new drug is felt to ameliorate anemia and hopefully reduce the physical burden of fatigue, then the QUALMS‐P is appropriate; if a new oral form of an older drug is felt to reduce the stress of disease management by decreasing clinic visit burden, then the QUALMS‐E or total QUALMS is likely more appropriate.…”

Section: Key Selected Aspects To Consider During Protocol Development...mentioning

confidence: 99%

Toward a more patient‐centered drug development process in clinical trials for patients with myelodysplastic syndromes/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS)

Efficace,

Buckstein,

Abel

et al. 2024

HemaSphere

Self Cite

View full text Add to dashboard Cite

Notable treatment advances have been made in recent years for patients with myelodysplastic syndromes/neoplasms (MDS), and several new drugs are under development. For example, the emerging availability of oral MDS therapies holds the promise of improving patients' health‐related quality of life (HRQoL). Within this rapidly evolving landscape, the inclusion of HRQoL and other patient‐reported outcomes (PROs) is critical to inform the benefit/risk assessment of new therapies or to assess whether patients live longer and better, for what will likely remain a largely incurable disease. We provide practical considerations to support investigators in generating high‐quality PRO data in future MDS trials. We first describe several challenges that are to be thoughtfully considered when designing an MDS‐focused clinical trial with a PRO endpoint. We then discuss aspects related to the design of the study, including PRO assessment strategies. We also discuss statistical approaches illustrating the potential value of time‐to‐event analyses and their implications within the estimand framework. Finally, based on a literature review of MDS randomized controlled trials with a PRO endpoint, we note the PRO items that deserve special attention when reporting future MDS trial results. We hope these practical considerations will facilitate the generation of rigorous PRO data that can robustly inform MDS patient care and support treatment decision‐making for this patient population.

show abstract

“…The relatively recent development of a validated MDS-specific quality of life assessment tool, the QUALMS score (https://qualms.dana-farber.org/), is expected to improve the reliability of these assessments in clinical trials. This instrument has been endorsed by the international MDS community and regulatory bodies [11 ▪▪ ,34 ▪ ,35 ▪ ].…”

Section: Transfusing Quality Of Lifementioning

confidence: 99%

“…New approaches to the measurement of quality of life in MDS reveal that not all patients benefit equally from transfusions; our transfusion goals may be too high in some and too low in others [10 ▪▪ ]. The recent development of a simple MDS-specific standardized instrument (QUALMS) for measurement of quality of life may help us to unravel some of the complexity in this regard [11 ▪▪ ]. Thus, effectively measuring whether transfused patients are deriving clinical benefit, and identifying and treating those who have developed or are developing iron overload are immediately actionable targets for optimized management in our patients with lower risk MDS.…”

Section: Introductionmentioning

confidence: 99%

Transfusion avoidance in myelodysplastic neoplasms

Griffiths

2023

Current Opinion in Hematology

View full text Add to dashboard Cite

Purpose of review Myelodysplastic neoplasms (MDS) are diseases of stem cell aging associated with complications from inadequate hematopoiesis (red cells, neutrophils and platelets) and variable risk for transformation to acute myeloid leukemia. Those with low-risk disease also suffer and die from MDS-related complications. Among the most challenging is development of anemia and transfusion dependence, which impacts quality of life and is associated with reduced survival. Appreciating and measuring the quality-of-life impact, preventing (if possible), treating, and managing the complications from anemia in MDS are of critical importance. Recent findings Recent developments in basic science highlight the potential deleterious impact of iron overload within the developing red cell niche. Iron overload can compromise red cell maturation from healthy as well as malignant clones and produces an environment favoring expansion of mutant clonal cells, potentially driving disease progression. Observational studies in nontransfusion dependent MDS highlight that iron overload occurs even in the nontransfusion dependent. The newly approved (and established) therapies for management of MDS-related anemia work best when begun before patients become heavily transfusion-dependent. Summary Iron overload is detrimental to hematopoiesis. Understanding the benefit afforded by transfusion is critical to optimal application and patient reported outcomes can inform this. Recently developed therapies are active and optimized application may improve response.

show abstract

Raising the standards of patient‐centered outcomes research in myelodysplastic syndromes: Clinical utility and validation of the subscales of the QUALMS from the MDS‐RIGHT project

Abstract: This is a repository copy of Raising the standards of patient-centered outcomes research in myelodysplastic syndromes : Clinical utility and validation of the subscales of the QUALMS from the MDS-RIGHT project.

Cited by 6 publications

References 42 publications

Health-related Quality of Life Profile of Newly Diagnosed Patients With Myelodysplastic Syndromes by Age, Sex, and Risk Group: A Real-world Study by the GIMEMA

Health-related Quality of Life Profile of Newly Diagnosed Patients With Myelodysplastic Syndromes by Age, Sex, and Risk Group: A Real-world Study by the GIMEMA

Toward a more patient‐centered drug development process in clinical trials for patients with myelodysplastic syndromes/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS)

Transfusion avoidance in myelodysplastic neoplasms

Contact Info

Product

Resources

About