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. 2022 Jan 11;6(1):1-12.
doi: 10.1182/bloodadvances.2021004568.

Core set of patient-reported outcomes for myelodysplastic syndromes: an EUMDS Delphi study involving patients and hematologists

Igor Stojkov  1 , Annette Conrads-Frank  1 , Ursula Rochau  1 , Karin A Koinig  2 , Marjan Arvandi  1 , Sibylle Puntscher  1 , Corine van Marrewijk  3 , Pierre Fenaux  4 , Argiris Symeonidis  5 , Fatiha Chermat  6 , Hege Garelius  7 , David Bowen  8 , Moshe Mittelman  9 , Elvira Mora  10   11 , Theo de Witte  12 , Fabio Efficace  13 , Uwe Siebert  1   14   15 , Reinhard Stauder  2
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Core set of patient-reported outcomes for myelodysplastic syndromes: an EUMDS Delphi study involving patients and hematologists

Igor Stojkov et al. Blood Adv. .

Abstract

Patient-reported outcomes (PROs) are relevant and valuable end points in the care of patients with myelodysplastic syndromes (MDS). However, a consensus-based selection of PROs for MDS, derived by both patients and hematologists, is lacking. We aimed to develop a core set of PROs for patients with MDS as part of the prospective European LeukemiaNet MDS (EUMDS) Registry. According to international guidelines, candidate PROs were identified from a comprehensive literature search of MDS studies. Overall, 40 PROs were selected and evaluated in a two-round Delphi survey by 40 patients with MDS and 38 hematologists in the first round and 38 patients and 32 hematologists in the second round. Based on an agreement scale and predefined inclusion criteria, both patients and hematologists selected "general quality of life" as a core PRO. Hematologists also selected "transfusion-dependency burden" and "ability to work/activities of daily living" as core PROs. The second Delphi round increased PRO rating agreements. Statistically significant rating differences between patients and hematologists were observed for 28 PROs (Mann-Whitney U test; P < .05) in the first round and for 19 PROs in the second round, with "disease knowledge" and "confidence in health care services" rated notably higher by patients. The overall mean PRO ratings correlation between the 2 groups was moderate (Spearman's rank correlation coefficient = 0.5; P < .05). This first consensus on a core set of PROs jointly developed by patients and hematologists forms the basis for patient-centered care in daily practice and clinical research.

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Figures

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Graphical abstract
Figure 1.
Figure 1.
PRISMA flowchart of the screening process. Systematic literature search to identify relevant QoL instruments used in MDS studies, as a potential source of patient-reported outcomes. n, number of studies.
Figure 2.
Figure 2.
Two-round Delphi survey PRO ratings, based on COMET categorization. Ratings of the patients and hematologists in the 2-round Delphi survey for identifying core PROs for MDS. Green squares: PROs rated dominantly (>70%) between 7 and 9; red squares: PROs rated dominantly (>70%) between 1 and 3. *Fear of MDS progression or transformation to AML. ADL, activities of daily living; HCS, health care services; SLP, speech-language problems.
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References

    1. Malcovati L, Hellström-Lindberg E, Bowen D, et al. ; European Leukemia Net . Diagnosis and treatment of primary myelodysplastic syndromes in adults: recommendations from the European LeukemiaNet. Blood. 2013;122(17):2943-2964. - PMC - PubMed
    1. Siebert U. When should decision-analytic modeling be used in the economic evaluation of health care? Eur J Health Econ. 2003;4(3):143-150.
    1. Stauder R. The challenge of individualised risk assessment and therapy planning in elderly high-risk myelodysplastic syndromes (MDS) patients. Ann Hematol. 2012;91(9):1333-1343. - PMC - PubMed
    1. Cheson BD, Bennett JM, Kantarjian H, et al. ; World Health Organization(WHO) international working group . Report of an international working group to standardize response criteria for myelodysplastic syndromes. Blood. 2000;96(12):3671-3674. - PubMed
    1. Patel SS, Gerds AT. Patient-reported outcomes in myelodysplastic syndromes and MDS/MPN overlap syndromes: stepping onto the stage with changing times. Curr Hematol Malig Rep. 2017;12(5):455-460. - PubMed

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