Artikel
Deriving Core Outcomes for Myelodysplastic Syndromes – Health Professionals’ Perspective
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Veröffentlicht: | 6. März 2018 |
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Gliederung
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Background and objectives: Myelodysplastic syndromes (MDS) comprise a diverse group of myeloid clonal hemopathies more prevalent in older patients. The decision on the treatment strategy, influenced by the advanced age at diagnosis and the common comorbidities, is additionally being undermined by the inconsistent outcome reporting and the lack of comparability among MDS studies. The aim of this research was to present highly important outcomes for the treatment of MDS from the perspective of health care practitioners (HCPs) and to initiate the development of a unified MDS-specific core outcome set (COS).
Methods: Potential MDS core outcomes were assessed by a previously performed comprehensive systematic literature review. The selection process included a three-round online Delphi survey among HCPs from 17 different countries as part of the MDS-RIGHT project. Each outcome was ranked using a scale from 1 to 9. Following recommended criteria, highly important outcomes were defined as those ranked 7-9 by at least 70% of participants and ranked 1-3 by not more than 15%. Outcomes were excluded if ranked 1-3 by at least 70% of the participants and 7-9 by not more than 15%. All outcomes proceeded to the next round and were presented together with the ratings of the previous round. The final, third round was intended for deriving consensus and defining the outcomes specifically for MDS.
Results: From 425 included studies, we extracted 1341 patient and/or clinically relevant outcomes that were condensed into 26 potential MDS core outcomes. 56 responses (24%) obtained in the first round resulted in 15 outcomes ranked as highly important, and one additional outcome, suggested by two participants. None of the outcomes could be excluded. 38 responses (17%) were analyzed in the following round, where six outcomes were ranked highly important. Still, no outcomes fulfilled the exclusion criteria, leading to the overall selection of six MDS core outcomes (quality of life, treatment-related mortality, overall survival, performance status, safety, hematological improvements). Final consensus on the remaining outcomes and definition of all included outcomes are ongoing.
Conclusion: Our study succeeded in identifying six outcomes that experts agree on being included in the MDS-COS. The selected outcomes show the experts’ awareness of the importance of patient-reported outcomes in MDS. Defining of the MDS-COS and inclusion of perspectives of other stakeholders (i.e., patients, industry representatives etc.) are currently ongoing.