PS 4-38 SYSTEMATIC OVERVIEW OF RESPONSE CRITERIA AMONG PATIENTS WITH MYELODYSPLASTIC SYNDROMES

Wednesday, October 26, 2016
Bayshore Ballroom ABC, Lobby Level (Westin Bayshore Vancouver)
Poster Board # PS 4-38

Igor Stojkov, MPharm1, Ursula Rochau, MD, MSc2, Helena Borba, MPharm3, Milica Jevdjevic, DMD1, Karin Koinig, Dr4, Marjan Arvandi, MS5, Fabio Efficace, Dr6, Reinhard Stauder, Dr4 and Uwe Siebert, MD, MPH, MSc, ScD7, (1)UMIT - University for Health Sciences, Medical Informatics and Technology, Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment, Hall i. T., Austria, (2)UMIT, Dept. of Public Health, Health Services Research & HTA / ONCOTYROL - Center for Personalized Cancer Medicine, Area 4 HTA and Bioinformatics, Hall i. T./Innsbruck, Austria, (3)Department of Pharmacy, Pharmaceutical Sciences Postgraduate Research Program, Federal University of Parana, Curitiba, Brazil, (4)Department of Internal Medicine V (Hematology and Oncology), Innsbruck Medical University, Innsbruck, Austria, (5)Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology, Hall i.T., Austria, (6)Health Outcomes Research Unit, Gruppo Italiano Malattie Ematologiche dell’Adulto (GIMEMA), Rome, Italy, (7)UMIT, Dept. of Public Health, Health Services Research & HTA / Harvard Univ., Dept. Health Policy & Management, Institute for Technology Assessment / Oncotyrol - Center for Personalized Cancer Medicine, Hall in Tirol (Austria) / Boston (USA), Austria
Purpose: Myelodysplastic syndromes (MDS) are a group of clonal hematopoietic stem cell malignancies mainly affecting elderly people. In addition to older age, several factors including, for example, the need of red blood cell transfusions or the high prevalence of comorbidities, contribute to making treatment decisions highly challenging in routine care. To initiate the establishment of a MDS-specific core outcome set, the aim of this work was to provide an overview of already accepted and implemented outcomes for MDS patients.

Method: We performed a comprehensive systematic literature search in the ClinicalTrials.gov database and four clinical trial registries (ISRCTN, ICTRP, EU-CTR and NCI). Studies evaluating MDS patient-relevant outcomes registered in the last four years were included. We 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 (IS/UR/HB/MJ).

Result: A total of 1014 studies were found. After removal of duplicates and full-text screening, 425 studies (observational and interventional) remained included. Overall, 1341 MDS patient-relevant outcomes were evaluated. Observed outcomes (primary and secondary) were stratified in five outcome groups according to the intervention, which was evaluated: “Drug”: 66.4%, “Drug and transplantation”: 22.4%, “Transplantation”: 5.7%, “Transfusion”: 1.7%, “Health and molecular prognostic value”: 2.5%, and “Procedures”: 1.3%. Overall, the five most frequently analyzed outcome parameters were overall survival (15.4%), adverse events (11.7%), acute/chronic graft-versus-host disease (10.1%), hematological improvements of cytopenias (9.6%) and remission rates (7.3%). Quality-of life (QoL) (3.3%) was ranked eleventh out of the 25 detected outcomes.

Conclusion: A broad range of reported outcomes was observed. Whereas conventional response parameters were applied frequently, patient-reported outcomes (PROs) were missing in the majority of studies. In order to establish a specific MDS core outcome set and ensure its practical application, we will provide further classification and redefinition of the outcomes in collaboration with the clinical experts. Taking into account the importance of evaluating QoL among MDS patients, we initiated a systematic review focusing on publications reporting the application of QoL measurement instruments in MDS.

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