Longitudinal Study on Determinants of Health-Related Quality of Life in Patients with Myelodysplastic Syndromes

Stojkov, Igor

Oral and Poster Abstracts
637. Myelodysplastic Syndromes—Clinical Studies: Poster I

Diseases, MDS, Myeloid Malignancies, Clinically relevant, Quality Improvement

Igor Stojkov, MSc¹^*, Annette Conrads-Frank, PhD¹^*, Ursula Rochau, MD, PhD¹^*, Marjan Arvandi, PhD¹^*, Karin A Koinig, PhD²^*, Michael Schomaker, PhD¹^*, Pierre Fenaux, MD, PhD³, David Bowen, MD⁴, Argiris Symeonidis, MD, PhD⁵, Moshe Mittelman, MD⁶^*, Jaroslav Cermak, MD, PhD⁷^*, Guillermo F Sanz, MD, PhD⁸, Eva Hellstrom Lindberg, MD, PhD⁹, Luca Malcovati, MD¹⁰, Saskia Langemeijer, MD, PhD¹¹^*, Ulrich Germing¹²^*, Mette Skov Holm, MD, PhD¹³^*, Krzysztof Madry, MD, PhD¹⁴^*, Agnes Guerci Bresler, MD¹⁵^*, Dominic J. Culligan, MD¹⁶^*, Ioannis Kotsianidis, MD, PhD¹⁷^*, Laurence Sanhes, MD¹⁸^*, Juliet Mills, MD¹⁹^*, Sibylle Puntscher, PhD¹^*, Daniela Schmid, PhD²⁰^*, Corine Van Marrewijk, PhD¹¹^*, Alexandra Smith, PhD²¹^*, Theo de Witte, MD, PhD²²^*, Uwe Siebert, MD, ScD²³^* and Reinhard Stauder, MD, MSc²

¹Institute 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
²Department of Internal Medicine V (Hematology and Oncology), Innsbruck Medical University, Innsbruck, Austria
³Service d’Hématologie Séniors, Hôpital Saint-Louis, Assistance Publique des Hôpitaux de Paris and Université Paris 7, Paris, France
⁴St. James's Institute of Oncology, Leeds Teaching Hospitals, Leeds, United Kingdom
⁵Department of Medicine, Div. Hematology, University of Patras Medical School, Patras, Greece
⁶Department of Medicine A, Tel Aviv Sourasky (Ichilov) Medical Center and Sackler Medical Faculty, Tel Aviv University, Tel Aviv, Israel
⁷Department of Clinical Hematology, Inst. of Hematology & Blood Transfusion, Praha, Czech Republic
⁸Department of Haematology, Hospital Universitario y Politécnico La Fe, Valencia, Spain
⁹Department of Medicine, Division of Hematology, Karolinska Institutet, Stockholm, Sweden
¹⁰Department of Hematology Oncology, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Piazzale Golgi 2, Pavia, Italy
¹¹Department of Hematology, Radboud university medical center, Nijmegen, Netherlands
¹²Department of Haematology, Oncology and Clinical Immunology, Universitätsklinik Düsseldorf, Düsseldorf, Germany
¹³Department of Haematology, Aarhus University Hospital, Aarhus, Denmark
¹⁴Department of Haematology, Oncology and Internal Medicine, Warszawa Medical University, Warszawa, Poland
¹⁵Service d'Hématologie Clinique, Centre Hospitalier Universitaire Brabois, Nancy, France
¹⁶Department of Haematology, Aberdeen Royal Infirmary, Aberdeen, United Kingdom
¹⁷Department of Hematology, Democritus University of Thrace, University Hospital of Alexandroupolis, Alexandroupolis, Greece
¹⁸Haematology Department of Perpignan, Saint Jean Hospital, Perpignan, France
¹⁹Leeds Institute for Cancer Studies and Pathology, University of Leeds, Leeds, United Kingdom
²⁰Division for Quantitative Methods in Public Health and Health Services Research, Department of Public Health, Health Services Research and Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology, Hall In Tirol, Austria
²¹Epidemiology and Cancer Statistics Group, Department of Heath Sciences, University of York, York, United Kingdom
²²Department of Tumor Immunology - Nijmegen Center for Molecular Life Sciences, Radboud university medical center, Nijmegen, Netherlands
²³UMIT, Dept. of Public Health, Health Services Research & HTA / Harvard Univ., Dept. Health Policy & Management, Institute for Technology Assessment, Hall i. T. (Austria) /, Boston, MA

Background: Myelodysplastic Syndromes (MDS) are associated with ageing, bone marrow dysplasia, peripheral blood cytopenias with varying severity and impaired health-related quality of life (HRQoL). HRQoL, a unique source of patient information, represents a valuable parameter for assessing treatment burden and therapy response in MDS.

Aim: To support individualized treatment planning and long-term improvements of HRQoL, we identified disease-specific and patient-related determinants of impaired HRQoL in patients with MDS.

Methods: We used longitudinal data from the EUMDS Registry, including 2205 patients at baseline (within 100 days from diagnosis) and two follow-up visits with a six-month average interval. EuroQol 5-Dimension 3-Level (EQ-5D-3L) index and visual analogue scale (VAS) score were used as two primary outcomes, with “low HRQoL” assigned below their observed medians of 0.78 and 0.70 respectively. Moreover, the EQ-5D dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) were used as secondary outcomes to assess impairment (“no problem” versus the remaining two categories).

We used bivariate logistic generalized estimating equations (GEE) to fit a marginal model and estimate odds ratios (OR) with 95% confidence intervals (CI) for the associations of disease-specific and patient-related parameters with HRQoL. To consider potential variable correlation, multivariate analyses were performed for each of the two primary outcomes. Missing values were imputed by multiple imputation using the chained equation method. The GEE longitudinal method accounts for the dynamic changes over time and produces an average population estimate within the analyzed period.

Results: From the 2205 patients at baseline, 5572 observations were analyzed along the three visits. We identified the following characteristics as relevant determinants of low EQ-5D index and VAS score, respectively (ORs and [95%CI]): higher age (1.04 [1.03-1.05]; 1.05 [1.05-1.06]), female sex (1.61 [1.37-1.89]; 1.23 [1.05-1.45]), advanced IPSS-R scoring (1.08 [1.02-1.14]; 1.18 [1.11-1.25]), increased serum ferritin levels ≥1000 μg/L (1.73 [1.37-2.19]; 1.81 [1.47-2.33]), increased neutrophil counts x10⁹/L (1.04 [1.02-1.06]; 1.03 [1.01-1.06]), transfusion dependence (1.44 [1.24-1.66]; 1.75 [1.50-2.04]), MDS-specific comorbidity index (MDS-CI) (1.19 [1.11-1.27]; 1.23 [1.15-1.32]), and Sorror comorbidity index (1.15 [1.10-1.19]; 1.14 [1.10-1.19]). In contrast, higher hemoglobin levels (0.87 [0.84-0.90]; 0.82 [0.80-0.85]) and high Karnofsky performance index (KPI) (0.59 [0.55-0.63]; 0.49 [0.45-0.52]) were associated with better HRQoL (Figure 1).

In the multivariate analysis, age above 75 years (1.84 [1.39-2.45]; 1.44 [1.07-1.92]), female sex (1.70 [1.43-2.03]; 1.22 [1.02-1.46]), serum ferritin levels ≥1000μg/L (1.41 [1.06-1.87]; 1.37 [1.02-1.84]), increased MDS-CI (1.11 [1.02-1.20]; 1.14 [1.05-1.25]) and high KPI (0.62 [0.58-0.67]; 0.53 [0.49-0.57]) remained relevant determinants for both low EQ-5D index and VAS score (Figure 2).

Regarding the secondary outcomes, around 30% of patients reported impairments in four EQ-5D dimensions and 10-11% in the dimension “self-care”. Female sex, KPI, Sorror comorbidity index, hemoglobin count and transfusion-related variables (transfusion dependence, cumulative number of transfusions and transfusion density) are major determinants of impairments in all five dimensions. Advanced age increased the risk for low HRQoL, particularly in the dimension “mobility” and “self-care”. An overall shorter list of relevant determinants was observed for the impaired “anxiety/depression” dimension. The variables: age, ferritin level, neutrophils count and MDS-CI, which were found significant in the other four dimensions, were not associated with the “anxiety/depression” dimension. Moreover, IPSS-R and female sex were not shown relevant for the “pain/discomfort” dimension.

Conclusion: Overall, we identified female sex, KPI, comorbidities, advanced age and increased ferritin levels as key determinants associated with poor HRQoL in newly diagnosed patients with MDS. These determinants provide valuable knowledge towards improved personalized treatment approach and early preventive measures in MDS. Studies addressing the predictive value of HRQoL after specific interventions are underway.

Disclosures: Stojkov: Erasmus Mundus-Western Balkans (ERAWEB) programme: Other: Doctoral scholarship; MDS-RIGHT (Providing the right care to the right patient with MyeloDysplastic Syndrome at the right time) project: Research Funding. Conrads-Frank: MDS-RIGHT (Providing the right care to the right patient with MyeloDysplastic Syndrome at the right time) project: Research Funding. Rochau: MDS-RIGHT (Providing the right care to the right patient with MyeloDysplastic Syndrome at the right time) project: Research Funding. Arvandi: MDS-RIGHT (Providing the right care to the right patient with MyeloDysplastic Syndrome at the right time) project: Research Funding. Fenaux: BMS: Honoraria, Research Funding; Novartis: Honoraria, Research Funding; Abbvie: Honoraria, Research Funding; Jazz: Honoraria, Research Funding. Symeonidis: Amgen: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; GenesisPharma: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Takeda: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Merck Sharp & Dohme: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Pfizer: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Roche: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Sanofi/Genzyme: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Janssen: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Gilead: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; WinMedica: Research Funding; Bristol-Myers Squibb: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene: Honoraria, Research Funding; Astellas: Research Funding; Abbvie: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding. Sanz: LaHoffman Roche Ltd.: Membership on an entity's Board of Directors or advisory committees; Helsinn: Membership on an entity's Board of Directors or advisory committees; Abbvie Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees; Takeda Pharmaceutical Ltd.: Membership on an entity's Board of Directors or advisory committees. Culligan: Celgene: Consultancy, Honoraria, Speakers Bureau; Novartis: Consultancy, Honoraria; Abbvie: Consultancy, Honoraria, Speakers Bureau; Diiachi-Sankyo: Consultancy, Honoraria; Jazz: Consultancy, Honoraria; Pfizer: Consultancy. Van Marrewijk: EUMDS and MDS-RIGHT (Providing the right care to the right patient with MyeloDysplastic Syndrome at the right time) project: Other: Project manager of the EUMDS Registry. de Witte: Amgen; Celgene; Novartis: Other: Project coordinator of the EUMDS Registry, Research Funding. Siebert: MDS-RIGHT (Providing the right care to the right patient with MyeloDysplastic Syndrome at the right time) project: Research Funding. Stauder: Celgene: Honoraria, Membership on an entity's Board of Directors or advisory committees; Teva: Research Funding; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees.

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1279 Longitudinal Study on Determinants of Health-Related Quality of Life in Patients with Myelodysplastic Syndromes