Development of a core outcome set for myelodysplastic syndromes – a Delphi study from the EUMDS Registry Group

Rochau, Ursula; Stojkov, Igor; Conrads-Frank, Annette; Borba, Helena Hiemisch Lobo; Koinig, Karin A.; Arvandi, Marjan; Marrewijk, Corine van; Garelius, Hege; Germing, Ulrich; Symeonidis, Argiris; Sanz, Guillermo; Fenaux, Pierre; Witte, Théo de; Efficace, Fabio; Siebert, Uwe; Stauder, Reinhard

doi:10.1111/bjh.16654

Cited by 12 publications

(14 citation statements)

References 58 publications

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“…For clinical trials, the core set of PROs should be integrated into the recently developed general core outcome set for MDS by Rochau et al 14 as a set of minimum standardized outcomes for clinical studies.…”

Section: Discussionmentioning

confidence: 99%

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

Stojkov¹,

Conrads-Frank²,

Rochau³

et al. 2021

Blood Advances

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Patient-reported outcomes (PROs) are relevant and valuable endpoints 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. Following international guidelines, candidate PROs were identified from a comprehensive literature search in 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, and 38 and 32 in the second round, respectively. 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-value <0.05) 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 two groups was moderate (Spearman's rank correlation coefficient=0.5; p-value <0.05). 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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Section: Discussionmentioning

confidence: 99%

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

Stojkov¹,

Conrads-Frank²,

Rochau³

et al. 2021

Blood Advances

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“…This set comprises overall survival (OS), performance score (PS), hematological improvement (HI), treatment-related mortality (TRM), safety, and quality of life (PROs). 38 Except for TA (safety and TRM), all other outcomes are included in our final set of GBIs. PROs reached the required threshold for acceptance only after rechallenge in the second round.…”

Section: Discussionmentioning

confidence: 99%

Guideline-based indicators for adult patients with myelodysplastic syndromes

et al. 2020

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Myelodysplastic syndromes (MDSs) represent a heterogeneous group of hematological stem cell disorders with an increasing burden on health care systems. Evidence-based MDS guidelines and recommendations (G/Rs) are published but do not necessarily translate into better quality of care if adherence is not maintained in daily clinical practice. Guideline-based indicators (GBIs) are measurable elements for the standardized assessment of quality of care and, thus far, have not been developed for adult MDS patients. To this end, we screened relevant G/Rs published between 1999 and 2018 and aggregated all available information as candidate GBIs into a formalized handbook as the basis for the subsequent consensus rating procedure. An international multidisciplinary expert panel group (EPG) of acknowledged MDS experts (n = 17), health professionals (n = 7), and patient advocates (n = 5) was appointed. The EPG feedback rates for the first and second round were 82% (23 of 28) and 96% (26 of 27), respectively. A final set of 29 GBIs for the 3 domains of diagnosis (n = 14), therapy (n = 8), and provider/infrastructural characteristics (n = 7) achieved the predefined agreement score for selection (>70%). We identified shortcomings in standardization of patient-reported outcomes, toxicity, and geriatric assessments that need to be optimized in the future. Our GBIs represent the first comprehensive consensus on measurable elements addressing best practice performance, outcomes, and structural resources. They can be used as a standardized instrument with the goal of assessing, comparing, and fostering good quality of care within clinical development cycles in the daily care of adult MDS patients.

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“…Due to advanced age and/or co-existing medical conditions, patients are often ineligible for this procedure. Therefore, treatment decisions for lower risk IPSS-R MDS often focus on improvement of quality of life (QoL) [9].…”

Section: Introductionmentioning

confidence: 99%

The anemia-independent impact of myelodysplastic syndromes on health-related quality of life

Wouters

Conrads-Frank²,

Koinig

et al. 2021

Ann Hematol

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Myelodysplastic syndromes (MDS) are in the majority of cases characterized by anemia. Both anemia and MDS per se may directly contribute to impairments in health-related quality of life (HRQoL). In this study, we aimed to investigate the anemia-independent impact of MDS on HRQoL. We evaluated participants (≥ 50 years) from the large population-based Lifelines cohort (N = 44,694, mean age 59.0 ± 7.4 years, 43.6% male) and the European MDS Registry (EUMDS) (N = 1538, mean age 73.4 ± 9.0 years, 63.0% male), which comprises a cohort of lower-risk MDS patients. To enable comparison concerning HRQoL, SF-36 scores measured in Lifelines were converted to EQ-5D-3L index (range 0–1) and dimension scores. Lower-risk MDS patients had significantly lower HRQoL than those from the Lifelines cohort, as illustrated in both the index score and in the five different dimensions. Multivariable linear regression analysis demonstrated that MDS had an adjusted total impact on the EQ-5D index score (B = − 0.12, p < 0.001) and an anemia-independent “direct” impact (B = − 0.10, p < 0.001). Multivariable logistic regression analysis revealed an anemia-independent impact of MDS in the dimension mobility, self-care, usual activities, and anxiety/depression (all except pain/discomfort). This study demonstrates that the major part of the negative impact of lower-risk MDS on HRQoL is not mediated via anemia. Thus, the therapeutic focus should include treatment strategies directed at underlying pathogenic mechanisms to improve HRQoL, rather than aiming predominantly at increasing hemoglobin levels.

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Development of a core outcome set for myelodysplastic syndromes – a Delphi study from the EUMDS Registry Group

Cited by 12 publications

References 58 publications

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

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

Guideline-based indicators for adult patients with myelodysplastic syndromes

The anemia-independent impact of myelodysplastic syndromes on health-related quality of life

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