Economic and Humanistic Burden in Patients With Obstructive Hypertrophic Cardiomyopathy - A Systematic Literature Review


Spoorendonk JA1, Şahbaz Gülser S1, Arca` E2, Hurst M3, Zema C4, Contente M3, Krause T3
1OPEN Health, Rotterdam, Netherlands, 2OPEN Health, Rotterdam, ZH, Netherlands, 3Bristol Myers Squibb, Uxbridge, UK, 4Bristol Myers Squibb, Lawrenceville, NJ, USA

OBJECTIVES: To systematically identify evidence on the cost-effectiveness (CE), healthcare costs and resource use (HCRU), quality of life (QoL) and health-state utilities (HSU) associated with treatment of obstructive hypertrophic cardiomyopathy (HCM).

METHODS: Medline, Embase, PsychINFO, and EconLit were searched with no language or geographic restrictions from database inception until 3 December 2021 for relevant CE, HCRU, QoL and HSU studies evaluating obstructive HCM treatment for adults. CE studies were restricted to assessments of pharmacological interventions and septal reduction therapies (SRT), while other types of data were allowed pertaining to any intervention. Grey literature was searched from 2019 until the same date.

RESULTS: 2,286 records were identified and 35 studies met inclusion criteria. No studies assessing the CE of treatments were found. 27 studies reported HCRU: 1 each from Belgium, Chile, Sweden and Spain, 2 each from Japan and the United Kingdom, and 19 from the United States (US). 21 studies reported resource use (RU) associated with SRT or pacing, while 3 studies reported on disease state. 3 studies included both costs and RU (2 SRT, 1 disease state). Only direct costs were reported in the 10 studies reporting costs only. Costs were predominantly from the US; 6 studies focussed on SRT or pacing and 4 on disease costs. 11 studies reported QoL and HSUs: the KCCQ, HCMSQ, EQ-5D-5L, EQ-VAS, SF-36, Karolinska questionnaire, Karolinska QoL VAS, SF-12, MLHFQ scales were included. 1 study reported data associated with mavacamten treatment, while 10 studies reported QoL associated with SRT or pacing. No estimates for disutilities were found.

CONCLUSIONS: There were no CE studies identified, and other economic data, where available, pertained mostly to SRT, were heterogeneous in nature, and should be interpreted with caution. Further research is needed to generate evidence on the economic and humanistic burden of obstructive HCM.

Conference/Value in Health Info

2022-11, ISPOR Europe 2022, Vienna, Austria

Value in Health, Volume 25, Issue 12S (December 2022)




Study Approaches

Topic Subcategory

Literature Review & Synthesis


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