Real-World Implementation and Clinical Outcomes of Mavacamten in an Underserved Urban Cohort With Obstructive Hypertrophic Cardiomyopathy
- 1Morristown Medical Center
Abstract
BackgroundReal-world data regarding mavacamten use in underserved and racially diverse populations remain limited, particularly for long-term therapy implementation. Structural barriers such as financial instability, fragmented healthcare access, and challenges adhering to REMS-related monitoring requirements may affect longitudinal therapy implementation.
MethodsWe performed a retrospective observational study of consecutive patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) evaluated for mavacamten therapy at a tertiary HCM center in Bronx, NY, between 6/2022-10/2025. Patients were followed for up to 48 weeks after treatment initiation. Clinical characteristics, treatment interruptions, adverse events, echocardiographic and functional outcomes, and implementation barriers were assessed descriptively.
ResultsSeventy-two patients with symptomatic oHCM were evaluated for mavacamten; sixty-seven initiated treatment. Mean age was 66.9{+/-}15.3 years, 72% were female, and 61% identified as Black or Hispanic. Median resting left ventricular outflow tract (LVOT) gradient decreased from 51mmHg (IQR30-88) at baseline to 7mmHg (IQR5-10) at 48 weeks. Median Valsalva LVOT gradient decreased from 74mmHg (IQR50-104) to 20mmHg (IQR10-29). At final follow-up, 80% of patients improved by [≥]1 NYHA functional class. LVEF declined below 50% in five patients and recovered in all cases. Approximately one-quarter experienced treatment interruption or discontinuation, nearly half related to insurance instability, non-adherence, and REMS-related monitoring barriers.
ConclusionsMavacamten demonstrated efficacy and safety outcomes comparable to those reported in clinical trials and prior registries in this predominantly underserved minority urban cohort. However, structural and healthcare-access barriers substantially affected long-term treatment implementation. These findings highlight the need to address healthcare-system barriers to ensure equitable implementation of contemporary HCM therapies.
Related
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- Clinical effectiveness of mavacamten on cardiac function and disease-related indicators in patients with obstructive hypertrophic cardiomyopathy
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- Mavacamten shows broad benefit in human and mouse models of MYBPC3-related hypertrophic cardiomyopathy
- Mavacamten improves energy balance in a pre-clinical model of RASopathy-associated hypertrophic cardiomyopathy
BibTeX
@misc{bock2026realworld,
title = {{Real-World Implementation and Clinical Outcomes of Mavacamten in an Underserved Urban Cohort With Obstructive Hypertrophic Cardiomyopathy}},
author = {J. Bock and S. Murthy and A. L. Schenone and A. Weissler-Snir},
howpublished = {medRxiv},
year = {2026},
month = sep,
doi = {10.64898/2026.09.29.26364363},
url = {https://www.medrxiv.org/content/10.64898/2026.09.29.26364363v1},
note = {CC BY-NC-ND},
}
RIS
TY - UNPB
TI - Real-World Implementation and Clinical Outcomes of Mavacamten in an Underserved Urban Cohort With Obstructive Hypertrophic Cardiomyopathy
AU - J. Bock
AU - S. Murthy
AU - A. L. Schenone
AU - A. Weissler-Snir
PY - 2026
DA - 2026/09/29
PB - medRxiv
DO - 10.64898/2026.09.29.26364363
UR - https://www.medrxiv.org/content/10.64898/2026.09.29.26364363v1
AB - BackgroundReal-world data regarding mavacamten use in underserved and racially diverse populations remain limited, particularly for long-term therapy implementation. Structural barriers such as financial instability, fragmented healthcare access, and challenges adhering to REMS-related monitoring requirements may affect longitudinal therapy implementation. MethodsWe performed a retrospective observational study of consecutive patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) evaluated for mavacamten therapy at a tertiary HCM center in Bronx, NY, between 6/2022-10/2025. Patients were followed for up to 48 weeks after treatment initiation. Clinical characteristics, treatment interruptions, adverse events, echocardiographic and functional outcomes, and implementation barriers were assessed descriptively. ResultsSeventy-two patients with symptomatic oHCM were evaluated for mavacamten; sixty-seven initiated treatment. Mean age was 66.9{+/-}15.3 years, 72% were female, and 61% identified as Black or Hispanic. Median resting left ventricular outflow tract (LVOT) gradient decreased from 51mmHg (IQR30-88) at baseline to 7mmHg (IQR5-10) at 48 weeks. Median Valsalva LVOT gradient decreased from 74mmHg (IQR50-104) to 20mmHg (IQR10-29). At final follow-up, 80% of patients improved by [≥]1 NYHA functional class. LVEF declined below 50% in five patients and recovered in all cases. Approximately one-quarter experienced treatment interruption or discontinuation, nearly half related to insurance instability, non-adherence, and REMS-related monitoring barriers. ConclusionsMavacamten demonstrated efficacy and safety outcomes comparable to those reported in clinical trials and prior registries in this predominantly underserved minority urban cohort. However, structural and healthcare-access barriers substantially affected long-term treatment implementation. These findings highlight the need to address healthcare-system barriers to ensure equitable implementation of contemporary HCM therapies.
ER -