Mavacamten in adolescent obstructive hypertrophic cardiomyopathy: SCOUTing a way forward.

摘要

Paediatric obstructive hypertrophic cardiomyopathy (oHCM) is a rare inherited cardiac disease that may present from childhood through adolescence and is associated with substantial long-term morbidity and mortality. Its management is based largely on observational studies and clinical experience and currently focusses on symptom improvement through medical therapy and, when indicated, septal reduction therapy to alleviate left ventricular outflow tract (LVOT) obstruction. In adult oHCM, mavacamten has established cardiac myosin inhibition as an effective strategy to reduce LVOT obstruction, but paediatric trial evidence has been lacking. The SCOUT-HCM trial is the first randomised placebo-controlled trial in adolescents to investigate the efficacy and safety of mavacamten for symptomatic oHCM. Among 44 adolescents (mean age 14.7 ± 1.7 years) mavacamten significantly reduced Valsalva-provoked LVOT gradients at 28 weeks, confirming short-term physiological efficacy. Secondary and exploratory findings, including NYHA functional class, cardiac structure and biomarkers, showed a trend to improvement, without a signal of left ventricular systolic dysfunction. The positive results of SCOUT-HCM, align with larger and longer adult oHCM trials and are likely to influence clinical practice, supporting a cautious integration of mavacamten in adolescent oHCM management. Longer follow-up and broader paediatric studies should determine durability of benefit, validated patient-reported outcomes, exercise capacity, arrhythmia burden, long-term safety and the need for septal reduction therapy in paediatric oHCM.