PMID- 34268723
OWN - NLM
STAT- MEDLINE
DCOM- 20211021
LR  - 20220531
IS  - 1897-4279 (Electronic)
IS  - 0022-9032 (Linking)
VI  - 79
IP  - 9
DP  - 2021
TI  - Mavacamten - a new disease-specific option for pharmacological treatment of 
      symptomatic patients with hypertrophic cardiomyopathy.
PG  - 949-954
LID - 10.33963/KP.a2021.0064 [doi]
AB  - Current pharmacotherapy for hypertrophic cardiomyopathy (HCM) is not 
      disease-specific and has suboptimal efficacy, often necessitating interventional 
      treatment. EXPLORER-HCM was a phase 3, randomized, double-blind, 
      placebo-controlled, multicenter clinical trial investigating the effects of 
      mavacamten, a first-in-class selective cardiac myosin inhibitor, in patients with 
      HCM, left ventricular outflow tract obstruction (LVOTO) and New York Heart 
      Association (NYHA) class II or III symptoms. The primary endpoint was defined as 
      either a >/=1.5 ml/kg/min increase in peak oxygen consumption (pVO(2)) and >/=1 NYHA 
      class reduction or a >/=3.0 ml/kg/min pVO2 increase without NYHA class worsening. 
      Secondary endpoints evaluated changes in post-exercise LVOT gradient, pVO2, NYHA 
      class, Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score 
      (KCCQ-CSS), and Hypertrophic Cardiomyopa-thy Symptom Questionnaire 
      Shortness-of-Breath subscore (HCMSQ-SoB). A total of 251 patients were randomized 
      to receiving mavacamten or placebo. The primary endpoint and all secondary 
      endpoints were met significantly more frequently in the mavacamten arm versus 
      placebo. The safety profile of mavacamten was similar to that of placebo. In 
      conclusion, disease-specific treatment with mavacamten in patients with 
      obstructive HCM led to reduced LVOTO and improvement in both objective functional 
      parameters and patient-related health status.
FAU - Pysz, Piotr
AU  - Pysz P
AD  - Division of Cardiology and Structural Heart Diseases, Medical University of 
      Silesia, Katowice, Poland.
FAU - Rajtar-Salwa, Renata
AU  - Rajtar-Salwa R
AD  - Department of Cardiology and Cardiovascular Interventions, University Hospital, 
      Krakow, Poland.
FAU - Smolka, Grzegorz
AU  - Smolka G
AD  - Division of Cardiology and Structural Heart Diseases, Medical University of 
      Silesia, Katowice, Poland.
FAU - Olivotto, Iacopo
AU  - Olivotto I
AD  - Cardiomyopathy Unit, Azienda Ospedaliera Universitaria Careggi, Florence, Italy.
FAU - Wojakowski, Wojciech
AU  - Wojakowski W
AD  - Division of Cardiology and Structural Heart Diseases, Medical University of 
      Silesia, Katowice, Poland.
FAU - Petkow-Dimitrow, Pawel
AU  - Petkow-Dimitrow P
AD  - Department of Cardiology and Cardiovascular Interventions, University Hospital, 
      Krakow, Poland.
AD  - 2nd Department of Cardiology, Jagiellonian University Medical College, Krakow, 
      Poland.
LA  - eng
PT  - Clinical Trial, Phase III
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
DEP - 20210716
PL  - Poland
TA  - Kardiol Pol
JT  - Kardiologia polska
JID - 0376352
RN  - 0 (Benzylamines)
RN  - 0 (MYK-461)
RN  - 56HH86ZVCT (Uracil)
SB  - IM
MH  - Benzylamines/*therapeutic use
MH  - *Cardiomyopathy, Hypertrophic/drug therapy
MH  - *Heart Defects, Congenital
MH  - Humans
MH  - Uracil/analogs & derivatives/*therapeutic use
MH  - *Ventricular Outflow Obstruction/drug therapy
OTO - NOTNLM
OT  - hypertrophic cardiomyopathy
OT  - left ventricular outflow obstruction
OT  - mavacamten
EDAT- 2021/07/17 06:00
MHDA- 2021/10/26 06:00
CRDT- 2021/07/16 06:51
PHST- 2021/07/09 00:00 [received]
PHST- 2021/07/09 00:00 [accepted]
PHST- 2021/07/17 06:00 [pubmed]
PHST- 2021/10/26 06:00 [medline]
PHST- 2021/07/16 06:51 [entrez]
AID - VM/OJS/J/84660 [pii]
AID - 10.33963/KP.a2021.0064 [doi]
PST - ppublish
SO  - Kardiol Pol. 2021;79(9):949-954. doi: 10.33963/KP.a2021.0064. Epub 2021 Jul 16.