Contributions
Abstract: PB2100
Type: Publication Only
Background
Myelodysplastic syndromes (MDS) represent a complex group of myeloid clonal disorders. Decision making in MDS is complicated by advanced age of MDS-patients and a high prevalence of comorbidities. Moreover, development of treatment strategies is complicated by the inconsistent outcome reporting in MDS studies and by the resulting lack of comparability.
Aims
The aims of this research were (1) to provide an overview of accepted and implemented outcomes in studies in MDS patients, (2) to identify the most important outcomes for the treatment of MDS from the perspective of health care practitioners (HCPs) and (3) to initiate the development of an unified MDS-specific core outcome set (COS).
Methods
To identify potential MDS core outcomes we performed a comprehensive systematic literature review in the ClinicalTrials.gov database and four clinical trial registries (ISRCTN, ICTRP, EU-CTR and NCI). We included studies evaluating MDS patient-relevant outcomes registered between 2012 and 2016 and excluded studies focusing solely on pharmacokinetics, pharmacodynamics or molecular research. Data regarding the registration year, funding, intervention, study population characteristics, primary and secondary outcomes were extracted using a predefined data extraction form and summarized in a comprehensive evidence table. All studies were screened by at least two independent reviewers. After summarizing the data, researchers together with MDS clinical experts actively participated in the discussion and derived a consensus on the potential MDS core outcomes. 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 (observational and interventional) included studies, we extracted 1,341 patient and/or clinically relevant outcomes that were condensed into 26 potential MDS core outcomes. 56 responses (24% response rate) 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. 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 is ongoing.
Conclusion
Our study succeeded in identifying six outcomes that experts agreed on being included in the MDS-COS. The selected outcomes show the experts’ awareness of the importance of patient-reported outcomes in MDS. MDS-specific definition of the MDS core outcomes and inclusion of perspectives of different stakeholders, namely of patients and health-care providers are currently ongoing.
Session topic: 10. Myelodysplastic syndromes – Clinical
Keyword(s): MDS, Outcome measurement
Abstract: PB2100
Type: Publication Only
Background
Myelodysplastic syndromes (MDS) represent a complex group of myeloid clonal disorders. Decision making in MDS is complicated by advanced age of MDS-patients and a high prevalence of comorbidities. Moreover, development of treatment strategies is complicated by the inconsistent outcome reporting in MDS studies and by the resulting lack of comparability.
Aims
The aims of this research were (1) to provide an overview of accepted and implemented outcomes in studies in MDS patients, (2) to identify the most important outcomes for the treatment of MDS from the perspective of health care practitioners (HCPs) and (3) to initiate the development of an unified MDS-specific core outcome set (COS).
Methods
To identify potential MDS core outcomes we performed a comprehensive systematic literature review in the ClinicalTrials.gov database and four clinical trial registries (ISRCTN, ICTRP, EU-CTR and NCI). We included studies evaluating MDS patient-relevant outcomes registered between 2012 and 2016 and excluded studies focusing solely on pharmacokinetics, pharmacodynamics or molecular research. Data regarding the registration year, funding, intervention, study population characteristics, primary and secondary outcomes were extracted using a predefined data extraction form and summarized in a comprehensive evidence table. All studies were screened by at least two independent reviewers. After summarizing the data, researchers together with MDS clinical experts actively participated in the discussion and derived a consensus on the potential MDS core outcomes. 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 (observational and interventional) included studies, we extracted 1,341 patient and/or clinically relevant outcomes that were condensed into 26 potential MDS core outcomes. 56 responses (24% response rate) 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. 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 is ongoing.
Conclusion
Our study succeeded in identifying six outcomes that experts agreed on being included in the MDS-COS. The selected outcomes show the experts’ awareness of the importance of patient-reported outcomes in MDS. MDS-specific definition of the MDS core outcomes and inclusion of perspectives of different stakeholders, namely of patients and health-care providers are currently ongoing.
Session topic: 10. Myelodysplastic syndromes – Clinical
Keyword(s): MDS, Outcome measurement