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040			_aBMG
041			_aeng
100			_aRossano, Joseph W. _eAutor _953796
245			_aMavacamten in adolescents with obstructive hypertrophic cardiomyopathy
300			_apáginas: 362-373
520			_aBackground: Approved pharmacologic therapies for pediatric hypertrophic cardiomyopathy are lacking, and surgical intervention may be indicated in patients with left ventricular outflow tract obstruction. The efficacy and safety of mavacamten, a cardiac myosin inhibitor that is available for adults, warrant evaluation in adolescents. Methods: We conducted a phase 3, double-blind, randomized, placebo-controlled trial involving symptomatic adolescents (12 to <18 years of age) with New York Heart Association class II or III obstructive hypertrophic cardiomyopathy. The patients were randomly assigned in a 1:1 ratio to receive mavacamten or placebo. The primary end point was the change from baseline to week 28 in left ventricular outflow tract pressure gradient provoked by the Valsalva maneuver. Results: A total of 44 patients underwent randomization; 23 patients (8 [35%] of whom were female) were assigned to mavacamten group, and 21 (5 [24%] of whom were female) were assigned to the placebo group. The mean (±SD) age of the patients was 14.7±1.7 years in the mavacamten group and 14.6±1.7 years in the placebo group, and the mean Valsalva left ventricular outflow tract gradient at baseline was similar in the two groups (78.4±34.1 mm Hg and 80.8±47.4 mm Hg, respectively). At week 28, the least-squares mean change in the Valsalva left ventricular outflow tract gradient was −48.5 mm Hg in the mavacamten group and −0.5 mm Hg in the placebo group (difference, −48.0 mm Hg; 95% confidence interval, −67.7 to −28.3; P<0.001). The incidence of adverse events was similar in the two groups. Two patients in each group had serious adverse events; in the mavacamten group, 1 patient had two episodes of syncope, and another had an inappropriate shock delivered by an implantable cardioverter–defibrillator; in the placebo group, 1 patient had chest pain, and another had depression with suicidal ideation. No patient had a reduction in the left ventricular ejection fraction to less than 50%. No deaths occurred during the trial. Conclusions: Among adolescent patients with obstructive hypertrophic cardiomyopathy, the reduction in left ventricular outflow tract obstruction was significantly greater with mavacamten than with placebo over a 28-week period.
650			_aMEDICINA DEL ADOLESCENTE _95533
650			_aCARDIOLOGÍA _911301
650			_aINSUFICIENCIA CARDIACA _911699
650			_aPEDIATRIA _96401
650			_aCARDIOMIOPATÍA _937409
700			_aCanter, Charles _953797
700			_aWolf, Cordula M. _953798
700			_aPapez, Andrew _953799
700			_aGambra, Marta _953800
700			_aBryant, Randall M. _953801
773	0		_022717 _922637 _dMassachusetts NEJM Group _oNEJM004 _tThe New England Journal of Medicine _wESSALUD _x0028-4793
942			_cARTICULOS _e2026-07-31 _zSQB
999			_c22747 _d22747