An Observational Multicenter Study to Assess the Cost of Illness and Quality of Life in Patients with Myelodysplastic Syndromes in Italy
Authors: Santini Valeria , Sanna Alessandro , Bosi Alberto , Alimena Giuliana , Loglisci Giuseppina , Levis Alessandro , Salvi Flavia , Finelli Carlo , Clissa Cristina , Specchia Giorgina , Ricco Alessandra , Cortelezzi Agostino , Ferla Valeria , Sciumé Mariarita , Nobile Francesco , Oliva Esther , Lazzaro Carlo , Martelli Elisa 
Published: October 2019

Abstract 1023

Background:

The annual incidence rate of myelodysplastic syndromes (MDS) in the general population is estimated at around 3–5/100,000. Because of a lack of specific therapies, until recently small attention has been devoted to costs of treatment of MDS. A specific Diagnosis-Related Group is still missing. In the US the mean annual cost for MDS patients ranges from $19,811 to $51,066.

Aims:

To assess the annual cost of illness (COI) and quality of life (QoL) of patients with MDS in Italy.

Methods:

The Costo Sociale delle Sindromi Mielodisplastiche e Qualità della vita in Italia (CoSMIQ) is an observational, cross-sectional, retrospective, prevalence-based, multicenter study based on an International Prognostic Scoring System (IPSS)-stratified sample of patients with MDS ≥18 years of age seen in standard clinical practice at 7 hematologic institutions across Italy (3 in the North; 2 in the Center; and 2 in the South).

Demographics, clinical history, health care and non-healthcare resource consumption, and patients' and caregivers' productivity losses were collected by physicians using clinical records and information provided by patients. COI (in Euro (€) 2010) was determined utilizing a societal perspective. QoL was assessed via EORTC QLQ-C30 and QOL-E v.2 questionnaires, the latter being a new specific tool for the assessment of health-related QoL in patients with MDS.

Kendall's tau rank correlation, chi-squared test, multivariate analysis of variance (ANOVA; MANOVA) and multiple linear regressions were performed when appropriate.

Results:

In all, 225 of 234 patients who met the inclusion criteria were analyzed (IPSS low risk: 124 patients; intermediate-1 (Int-1) risk: 75 patients; Int-2 and high risk: 26 patients) (Table).

The total COI reached €27,980.4 ± 28,322.2 (mean ± standard deviation) (Figure). Cost-drivers of COI were antianemics (lower-risk) and antineoplastics (higher-risk). The Italian National Health Service (INHS) funded the greatest share of COI (from 97.4% to 99.5%).

In general, CoSMIQ patients perform worse than the general population in all EORTC QLQ-C30 domains; the total global health status (QL) domain reached 65.1 ± 22.2 (low risk: 64.2 ± 22.3; Int-1 risk: 67.4 ± 22.1; higher-risk: 62.5 ± 22.3). QL was negatively correlated with COI (p=0.049) and patients' age (p=0.089), and positively correlated with disease duration (p=0.203).

Keeping the other predictors constant, RBC transfusion dependence predicts increased COI (p=0.006) and lower QL (p=0.009).

Regarding the QOL-E questionnaire, the QOL-E MDS-specific domain (MDSS) was positively correlated with total COI (p=0.0002), and patients' age (p=0.348) and negatively correlated with disease duration (p=0.013).

The total QOL MDSS was 27.5 ± 19.8 (low risk: 25.3 ± 19.4; Int-1 risk: 29.6 ± 19.3; higher-risk: 31.6 ± 22.2). The total treatment-outcome index domain (QOL TOI) was 49.4 ± 14.4 (low risk: 49.1 ± 14.7; Int-1 risk: 48.3 ± 14.7; higher-risk: 53.9 ± 11.7). QOL TOI was negatively correlated with total COI (p=0.379), and patients' age (p=0.0003) and positively correlated with disease duration (p=0.162). Differences by IPSS group were not statistically significant for COI (p=0.191) or for any domain of the EORTC QLQ-C30 or QOL-E questionnaires (p=0.124; p=0.467).

Conclusion:

MDS dramatically increases INHS budgets while negatively impacting patients' QoL. The results of the CoSMIQ study highlight the strong correlation between RBC transfusion dependence and both COI and QL.

Disclosures:

Lazzaro: Celgene Srl: Research Funding. Martelli:Celgene Srl: Employment, Equity Ownership.