TY - JOUR
T1 - Core set of patient-reported outcomes for myelodysplastic syndromes
T2 - an EUMDS Delphi study involving patients and hematologists
AU - Stojkov, Igor
AU - Conrads-Frank, Annette
AU - Rochau, Ursula
AU - Koinig, Karin A.
AU - Arvandi, Marjan
AU - Puntscher, Sibylle
AU - van Marrewijk, Corine
AU - Fenaux, Pierre
AU - Symeonidis, Argiris
AU - Chermat, Fatiha
AU - Garelius, Hege
AU - Bowen, David
AU - Mittelman, Moshe
AU - Mora, Elvira
AU - de Witte, Theo
AU - Efficace, Fabio
AU - Siebert, Uwe
AU - Stauder, Reinhard
N1 - Publisher Copyright:
ß 2021 by The American Society of Hematology. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.
PY - 2022/1/11
Y1 - 2022/1/11
N2 - 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 5 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.
AB - 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 5 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.
UR - http://www.scopus.com/inward/record.url?scp=85123090067&partnerID=8YFLogxK
U2 - 10.1182/bloodadvances.2021004568
DO - 10.1182/bloodadvances.2021004568
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C2 - 34492684
AN - SCOPUS:85123090067
SN - 2473-9529
VL - 6
SP - 1
EP - 12
JO - Blood advances
JF - Blood advances
IS - 1
ER -