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Persisting symptoms despite optimal medical treatment in patients with obstructive HCM not eligible for septal reduction therapy: insights from an international registry
Session:
Comunicações Orais - Sessão 21 - Miocardiopatia hipertrófica
Speaker:
Mariana Silva Brandão
Congress:
CPC 2023
Topic:
F. Valvular, Myocardial, Pericardial, Pulmonary, Congenital Heart Disease
Theme:
17. Myocardial Disease
Subtheme:
17.7 Myocardial Disease - Other
Session Type:
Comunicações Orais
FP Number:
---
Authors:
Mariana S. Brandão; Niccolo' Maurizi; Brian Claggett; Euan A. Ashley; Adam S. Helms; Neal Lakdawala; Michelle Michels; Alexandre C. Pereira; Sara Saberi; Christopher Semsarian; James S. Ware; Sharlene M. Day; Carolyn Y. Ho; Iacopo Olivotto; on Behalf of The Share Registry Investigators (Sarcomeric Human Cardiomyopathy Registry)
Abstract
<p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><strong>Introduction: </strong>Patients with hypertrophic cardiomyopathy (HCM) and left ventricular outflow tract obstruction (LVOTO) who remain symptomatic despite optimal medical therapy (OMT) may not be eligible to, or compliant with, septal reduction therapies (SRT), including myectomy and alcohol septal ablation. This subset represents an unmet clinical need, potentially amenable to cardiac myosin inhibitors (CMI), new agents targeting the molecular basis of HCM.</span></span></p> <p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><strong>Aim: </strong>To evaluate the prevalence, in an international consortium, of patients with obstructive HCM with persisting symptoms despite OMT, fulfilling the enrollment criteria for the EXPLORER-HCM trial.</span></span></p> <p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><strong>Methods: </strong>In this cross-sectional analysis, we enrolled HCM patients in New York Heart Association (NYHA) class ≥II at the most recent evaluation, aged ≥18 years, with LVOT gradient ≥50mmHg and left ventricular ejection fraction [LVEF]≥55%. Patients on disopyramide were excluded, according to EXPLORER-HCM criteria.</span></span></p> <p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><strong>Results: </strong>Of 10.225 HCM patients (85% Caucasian, 61.4% male, mean age at diagnosis 44<span style="color:black">±20 years), 8.874 patients with complete data were included in the analysis, of whom 2067 (23%) had obstructive HCM [Fig1].</span></span></span></p> <p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><span style="color:black">Of these, 48% had symptoms at baseline, and 39.7% remained symptomatic despite OMT. SRT, mostly myectomy, was performed in 346 patients (42%) of this group. The remaining 474 (59%) patients were not treated invasively (age at diagnosis 54</span><span style="color:black">±</span><span style="color:black">16 years, 47% male, 15% with pathogenic/likely pathogenic gene variants, mean LVEF 69</span><span style="color:black">±7%, mean LVOT gradient 77±39mmHg). Of these 474, 16.7% (n=79) were on Disopyramide.</span></span></span></p> <p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><span style="color:black">According to the EXPLORER-HCM enrollment criteria, 192 of the 474 patients (40%) were potentially eligible for CMI. Eligible patients were mostly female (51%), Caucasian (92%), and were older at diagnosis (56 <em>vs </em>53 years, p=.038). Most (75%) were in NYHA class II. </span>At baseline, mean maximal wall thickness was 19±5 mm, and<span style="color:black"> LVOT gradient was 91±31mmHg; mean LVEF was 70</span>±7%; 83.3% had late gadolinium enhancement on cardiac magnetic resonance. </span></span></p> <p style="text-align:justify"><span style="font-size:11pt"><span style="font-family:Calibri,sans-serif"><strong><span style="color:black">Conclusions: </span></strong><span style="color:black">Most patients with obstructive HCM present persisting symptoms despite optimal medical therapy. Of these, less than half undergo SRT; 40% of the remaining symptomatic would be potential candidates for CMI according to the EXPLORER-HCM criteria. More liberal criteria for CMI introduction (such as the possibility to combine them with disopyramide) would expand the indication by a further 17% in this group. Optimization of care in this pt subset is warranted.</span></span></span></p>
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