JOURNAL CLUB

PRESENTER – DR NITHYA RAJ

MODERATOR –DR ASWIN LAL
AFICAMTEN FOR SYMPTOMATIC OBSTRUCTIVE

HYPERTROPHIC CARDIOMYOPATHY

THE NEW ENGLAND JOURNAL OF MEDICINE

(MAY 13, 2024)
 VOLUME 390, NUMBER 20

AUTHORS : MARTIN S MARON, M.D.AHMAD MASRI, M.D.

MICHAEL E. NASIF, ROBERTO BARRIALES – VILLA, M.D.PhD,
MICHAEL ARAD, M.D.,NUNO CARDIM, M.D.PhD, LUBNA
CHOUDHURY,M.D.
BACKGROUND

• AFICAMTEN – ORAL SELECTIVE CARDIAC MYOSIN

INHIBITOR
• REDUCES LV OUTFLOW TRACT GRADIENTS BY
MITIGATING CARDIAC HYPERCONTRACTILITY
METHODS

• STUDY DESIGN – PHASE 3 RANDOMISED , DOUBLE BLIND,

PLACEBO - CONTROLLED STUDY
• STUDY POPULATION – 282 PATIENTS ( AGE 18-85 YEARS )
• STUDY DURATION – 24 WEEKS
THE TRIAL

• 282 PATIENTS WITH CONFIRMED CLINICAL DIAGNOSIS

• ORAL AFICAMTEN (STARTING DOSE – 5MG , MAX DOSE – 20MG) OR
PLACEBO OD FOR 24 WEEKS
• PRIMARY END POINT – CHANGE IN PEAK O2 UPTAKE FROM
BASELINE TO WEEK 24
• CARDIOPULMONARY EXERCISE TESTING
• SECONDARY END POINTS

- CHANGE IN KANSAS CITY CARDIOMYOPATHY QUESTIONNAIRE

CLINICAL SUMMARY SCORE AT WEEK 24
- IMPROVEMENT OF AT LEAST 1 NYHA CLASS AT WEEK 24
- CHANGE IN LV OUTFLOW TRACT GRADIENT AFTER VALSALVA
MANEUVER AT WEEK 24
RESULTS

• 142 IN AFICAMTEN GROUP, 140 IN THE PLACEBO GROUP

• 59 % WERE MEN AND 41% WERE WOMEN
• BASELINE MEAN RESTING LV OUTFLOW TRACT GRADIENT WAS
55.1 mmHg
• BASELINE MEAN LV EF WAS 74.8%
• AT 24 WEEKS MEAN CHANGE IN PEAK O2 UPTAKE WAS 1.8/ml/Kg/min
IN AFICAMTEN GROUP & 0.0 ml/Kg/min IN PLACEBO GROUP
LIMITATIONS

• SHORT PERIOD
• NO ASSESSMENT OF LONGER TERM CV OUTCOMES
• SOME SHOWED NO IMPROVEMENT IN NYHA CLASS
• FURTHER TRIALS REQUIRED TO DETERMINE SYMPTOM RELIEF
WITH LONGER TREATMENT
CONCLUSION

• IN PATIENTS WITH SYMPTOMATIC OBSTRUCTIVE HCM ,

TREATMENT WITH AFICAMTEN RESULTED IN GREATER
IMPROVEMENT IN PEAK O2 UPTAKE OVER A 24 WEEK TREATMENT
PERIOD THAN PLACEBO
THANK YOU