A Set of Systematic and Targeted Reviews to Identify the Burden of Iron Chelation Therapy in Myelodysplastic Syndromes
Author(s)
Oliva E1, Huey K2, Deshpande S3, Turner M4, Chitnis M4, Schiller E4, Tang D2, Jones S2, Shah F5
1Grande Ospedale Metropolitano Bianchi Melacrino Morelli, Reggio Calabria, Italy, 2Bristol Myers Squibb, Princeton, NJ, USA, 3Evidera, London, UK, 4Evidera, Waltham, MA, USA, 5Whittington Health NHS Trust, Isleworth, LON, UK
Presentation Documents
OBJECTIVES: Treatment with iron chelation therapy (ICT) has shown benefits in patients with myelodysplastic syndromes (MDS). A systematic literature review (SLR) was conducted to investigate the burden associated with ICT. METHODS: Systematic searches were conducted in MEDLINE and Embase to identify SLRs assessing clinical burden, health-related quality of life (HRQoL), and economic burden of ICT in adult patients with MDS published between January 1, 2015 and May 10, 2020. To fill remaining gaps in data, a subsequent targeted search was conducted. RESULTS: Of 488 references identified at the title and abstract level, 60 were screened at the full-text level, with 3 SLRs ultimately eligible for inclusion. These SLRs reported results of observational studies in patients with lower-risk MDS and transfusion-dependent patients and primarily evaluated mortality; 2 reported limited safety data and cardiac events. Patients receiving ICT experienced a median of 137 months of cardiac event-free survival. No data were systematically identified on HRQoL, economic outcomes, and treatment adherence. Targeted searches provided additional details on ICT burden. One study of 35 patients in the UK reported the expected 10-year costs (in US dollars from 2000 to 2010) of deferasirox, desferrioxamine, and deferiprone; projected costs were $2,064,800, $526,880, and $561,056, respectively, across all patients. ICT carried potential for adverse events; drug-related adverse events included renal, gastrointestinal, and liver toxicity, and joint pain. An Italian open-label study evaluating deferasirox in transfusion-dependent patients over time found no significant changes from baseline EORTC QLQ-C30 score; patients remained stable approximately 10 points below the normal global quality of life score for the Italian population, indicating reduced HRQoL. CONCLUSIONS: The few SLRs reporting on the clinical burden of ICT indicate improved survival by comparison, but do not explore the economic or HRQoL burden experienced. Details on the patient HRQoL and economic outcomes remain underreported.
Conference/Value in Health Info
2021-05, ISPOR 2021, Montreal, Canada
Value in Health, Volume 24, Issue 5, S1 (May 2021)
Code
PCN78
Topic
Economic Evaluation
Disease
Oncology