Clinical and Economic Burden of Non-Obstructive Hypertrophic Cardiomyopathy: A Literature Review
Author(s)
Paulos Gebrehiwet, PhD1, Laura Cianciolo, MPA2, Jesse Ortendahl, MS2, Matthew Sussman, MA2, Mike B. Butzner, Jr., DrPH1, Sanatan Shreay, PhD1, Theodore Abraham, MD3;
1Cytokinetics, Inc., South San Francisco, CA, USA, 2Stratevi, Boston, MA, USA, 3Hypertrophic Cardiomyopathy Center, University of California, San Francisco, CA, USA
1Cytokinetics, Inc., South San Francisco, CA, USA, 2Stratevi, Boston, MA, USA, 3Hypertrophic Cardiomyopathy Center, University of California, San Francisco, CA, USA
Presentation Documents
OBJECTIVES: Hypertrophic cardiomyopathy (HCM) is a chronic, progressive disease characterized by left ventricular hypertrophy. Non obstructive (nHCM) is a type of HCM with an estimated prevalence of 0.05% in the United States. Previous studies report mixed findings on the burden severity for nHCM. The objective of this targeted literature review (TLR) was to review current evidence of the clinical and economic burden of nHCM.
METHODS: PubMed was used to identify English-language studies published from 2005-2024 in nHCM. Two distinct searches were conducted: 1) clinical burden, and 2) economic burden. A supplemental search for abstracts and white papers was conducted using Google, and review studies were excluded. Outcome of interest included epidemiology, healthcare resource utilization and direct medical costs.
RESULTS: Of 119 studies identified, 15 studies (including 4 from supplemental search) met criteria for inclusion (clinical search, n= 14; economic, n= 1). The 104 excluded studies were due to lack of relevance. Most studies used data from a single center or assessed a small sample, raising generalizability concerns. Amongst clinical studies, 9/14 (64%) were conducted in United States. Studies reported data on prevalence, incidence, mortality, and disease progression rate. Eight studies (57%) reported nHCM prevalence ranging from 0.01-0.17%. Five studies (36%) reported mortality rates ranging from 0.5-10%. Utilization outcomes were reported in nine studies, including hospitalization rates of 0.08-0.60%. The economic study reported annual per-patient costs for nHCM stratified by symptom status and presence of complications ($22,711-$73,630).
CONCLUSIONS: Results from this review indicate there is limited evidence characterizing clinical and economic outcomes for nHCM. Future research is necessary to measure the magnitude of the nHCM population and burden of disease as well as assess the potential impact of novel therapeutics in development for treatment of nHCM.
METHODS: PubMed was used to identify English-language studies published from 2005-2024 in nHCM. Two distinct searches were conducted: 1) clinical burden, and 2) economic burden. A supplemental search for abstracts and white papers was conducted using Google, and review studies were excluded. Outcome of interest included epidemiology, healthcare resource utilization and direct medical costs.
RESULTS: Of 119 studies identified, 15 studies (including 4 from supplemental search) met criteria for inclusion (clinical search, n= 14; economic, n= 1). The 104 excluded studies were due to lack of relevance. Most studies used data from a single center or assessed a small sample, raising generalizability concerns. Amongst clinical studies, 9/14 (64%) were conducted in United States. Studies reported data on prevalence, incidence, mortality, and disease progression rate. Eight studies (57%) reported nHCM prevalence ranging from 0.01-0.17%. Five studies (36%) reported mortality rates ranging from 0.5-10%. Utilization outcomes were reported in nine studies, including hospitalization rates of 0.08-0.60%. The economic study reported annual per-patient costs for nHCM stratified by symptom status and presence of complications ($22,711-$73,630).
CONCLUSIONS: Results from this review indicate there is limited evidence characterizing clinical and economic outcomes for nHCM. Future research is necessary to measure the magnitude of the nHCM population and burden of disease as well as assess the potential impact of novel therapeutics in development for treatment of nHCM.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
CO78
Topic
Clinical Outcomes
Topic Subcategory
Relating Intermediate to Long-term Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Cardiovascular Disorders (including MI, Stroke, Circulatory)