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CONSENSUS SET OF CORE PATIENT-REPORTED OUTCOMES FOR MYELODYSPLASTIC SYNDROMES DEFINED BY PATIENTS AND HEMATOLOGISTS – EUMDS REGISTRY GROUP STUDY
Author(s): ,
Igor Stojkov
Affiliations:
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 in Tirol,Austria
,
Annette Conrads-Frank
Affiliations:
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 in Tirol,Austria
,
Ursula Rochau
Affiliations:
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 in Tirol,Austria
,
Karin Koinig
Affiliations:
Department of Internal Medicine V (Hematology and Oncology),Innsbruck Medical University,Innsbruck,Austria
,
Marjan Arvandi
Affiliations:
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 in Tirol,Austria
,
Sibylle Puntscher
Affiliations:
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 in Tirol,Austria
,
Corine van Marrewijk
Affiliations:
Department of Hematology,Radboud university medical center,Nijmegen,Netherlands
,
Pierre Fenaux
Affiliations:
Service d'Hématologie, Hôpital Saint-Louis,Assistance Publique des Hôpitaux de Paris (AP-HP) and Université Paris 7,Paris,France
,
Argiris Symeonidis
Affiliations:
Department of Medicine,Div. Hematology, University of Patras Medical School,Patras,Greece
,
Fatiha Chermat
Affiliations:
Groupe Francophone des Myélodysplasies,GFM,Paris,France
,
Hege Garelius
Affiliations:
Department of Medicine,Section of Hematology and Coagulation, Sahlgrenska University Hospital,Göteborg,Sweden
,
David Bowen
Affiliations:
St. James's Institute of Oncology,Leeds Teaching Hospitals,Leeds,United Kingdom
,
Moshe Mittelman
Affiliations:
Department of Medicine A,Tel Aviv Sourasky (Ichilov) Medical Center and Sackler Medical Faculty, Tel Aviv University,Tel Aviv,Israel
,
Elvira Mora
Affiliations:
Hospital Universitario y Politécnico La Fe, Valencia,Spain & Instituto de Investigación Sanitaria La Fe,Valencia,Spain
,
Theo de Witte
Affiliations:
Department of Tumor Immunology - Nijmegen Center for Molecular Life Sciences,Radboud university medical center,Nijmegen,Netherlands
,
Fabio Efficace
Affiliations:
Health Outcomes Research Unit,Gruppo Italiano Malattie Ematologiche dell'Adulto (GIMEMA),Rome,Italy
,
Uwe Siebert
Affiliations:
UMIT - University for Health Sciences, Medical Informatics and Technology, Dept. of Public Health, Health Services Research & HTA / Harvard Univ.,Dept. Health Policy & Management, Institute for Technology Assessment,Hall i.T. / Boston,Austria
Reinhard Stauder
Affiliations:
Department of Internal Medicine V (Hematology and Oncology),Innsbruck Medical University,Innsbruck,Austria
(Abstract release date: 05/14/20) EHA Library. Stojkov I. 06/12/20; 294734; EP817
Igor Stojkov
Igor Stojkov
Contributions
Abstract

Abstract: EP817

Type: e-Poster

Background
Patients with myelodysplastic syndromes (MDS) require individualized treatment approaches, integrating the perception of patients. Thus, patient-reported outcomes (PROs) have been introduced, which include any information that comes directly from the patient such as symptom severity or quality-of-life (QoL). So far, core PROs have not been defined in MDS.

Aims
We aim (1) to identify QoL measurement instruments (QoL-MI) relevant in MDS, as a comprehensive source of potential core PROs, (2) to nominate core PROs for MDS by patients and hematologists.

Methods
We performed a comprehensive systematic literature search (PubMed, Cochrane Library, Scopus, Web of Science) to identify QoL-MI. Studies were screened by two independent reviewers and the observed QoL-MI were summarized in an evidence table. The domains and items within each of the observed QoL-MI were extracted and categorized into potential PROs by experienced researchers and clinical experts. To nominate core PRO, a two-round Delphi survey was performed among MDS patients from the outpatient clinics of Saint Louis Hospital in Paris and physicians from 18 different countries as part of the MDS-RIGHT project and the EUMDS Registry. Each outcome was ranked for importance, using a nine-point agreement scale and predefined criteria according to the 1-3/7-9 scoring. Missing values were imputed by the items’ median ranking and new outcomes suggested by at least two participants were included in the next round. We used Spearman's rank correlation coefficient (ρ) to determine the correlation of the mean PRO rankings between groups.

Results
The literature search resulted in 2,863 studies; 81 studies were included in our review. Overall, we identified twelve generic, six cancer-specific and two MDS-specific QoL-MI. The most frequently used instruments were EORTC QLQ-C30 (26 studies), FACIT-An (17), SF-36 (16), QoL-E (10), and FACIT-BMT (7). These five instruments composed 68% of the total 112 QoL-MI applications, whereas the MDS-specific QoL-MIs composed only 10%. Forty potential core PROs were extracted from the QoL-MI and applied in the two-round Delphi survey. In the patients’ Delphi survey, we obtained 40 responses in the first round followed by 38 in the second round. Results showed an agreement for inclusion of “general QoL” into the core PROs set. Further outcomes ranked highly include “fatigue“, “general health“, “confidence in health care services“, “weakness“, and “disease knowledge“. Among the physicians, we obtained 38 responses in the first round and 32 in the second Delphi survey. Three PROs fulfilled the inclusion criteria: “general QoL”, “transfusion dependence”, and “ability to work/activities of daily living”. The Spearman coefficients showed moderate correlation between mean physician and patient scores (1st round: ρ=0.51, p<0.001; 2nd round: ρ=0.54, p<0.001). Lower agreement was observed for “disease knowledge” and “confidence in health care services”, both ranked notably higher by the patients.

Conclusion
QoL-MI, particularly MDS-specific QoL-MI are underused in current MDS practice. This first consensus set of core PROs selected by patients and hematologists is useful to promote quality of care in daily practice and forms the basis for patient-centered care.

Session topic: 10. Myelodysplastic syndromes - Clinical

Keyword(s): MDS, Outcome measurement

Abstract: EP817

Type: e-Poster

Background
Patients with myelodysplastic syndromes (MDS) require individualized treatment approaches, integrating the perception of patients. Thus, patient-reported outcomes (PROs) have been introduced, which include any information that comes directly from the patient such as symptom severity or quality-of-life (QoL). So far, core PROs have not been defined in MDS.

Aims
We aim (1) to identify QoL measurement instruments (QoL-MI) relevant in MDS, as a comprehensive source of potential core PROs, (2) to nominate core PROs for MDS by patients and hematologists.

Methods
We performed a comprehensive systematic literature search (PubMed, Cochrane Library, Scopus, Web of Science) to identify QoL-MI. Studies were screened by two independent reviewers and the observed QoL-MI were summarized in an evidence table. The domains and items within each of the observed QoL-MI were extracted and categorized into potential PROs by experienced researchers and clinical experts. To nominate core PRO, a two-round Delphi survey was performed among MDS patients from the outpatient clinics of Saint Louis Hospital in Paris and physicians from 18 different countries as part of the MDS-RIGHT project and the EUMDS Registry. Each outcome was ranked for importance, using a nine-point agreement scale and predefined criteria according to the 1-3/7-9 scoring. Missing values were imputed by the items’ median ranking and new outcomes suggested by at least two participants were included in the next round. We used Spearman's rank correlation coefficient (ρ) to determine the correlation of the mean PRO rankings between groups.

Results
The literature search resulted in 2,863 studies; 81 studies were included in our review. Overall, we identified twelve generic, six cancer-specific and two MDS-specific QoL-MI. The most frequently used instruments were EORTC QLQ-C30 (26 studies), FACIT-An (17), SF-36 (16), QoL-E (10), and FACIT-BMT (7). These five instruments composed 68% of the total 112 QoL-MI applications, whereas the MDS-specific QoL-MIs composed only 10%. Forty potential core PROs were extracted from the QoL-MI and applied in the two-round Delphi survey. In the patients’ Delphi survey, we obtained 40 responses in the first round followed by 38 in the second round. Results showed an agreement for inclusion of “general QoL” into the core PROs set. Further outcomes ranked highly include “fatigue“, “general health“, “confidence in health care services“, “weakness“, and “disease knowledge“. Among the physicians, we obtained 38 responses in the first round and 32 in the second Delphi survey. Three PROs fulfilled the inclusion criteria: “general QoL”, “transfusion dependence”, and “ability to work/activities of daily living”. The Spearman coefficients showed moderate correlation between mean physician and patient scores (1st round: ρ=0.51, p<0.001; 2nd round: ρ=0.54, p<0.001). Lower agreement was observed for “disease knowledge” and “confidence in health care services”, both ranked notably higher by the patients.

Conclusion
QoL-MI, particularly MDS-specific QoL-MI are underused in current MDS practice. This first consensus set of core PROs selected by patients and hematologists is useful to promote quality of care in daily practice and forms the basis for patient-centered care.

Session topic: 10. Myelodysplastic syndromes - Clinical

Keyword(s): MDS, Outcome measurement

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