Predictors of death in adults with duchenne muscular dystrophy-associated cardiomyopathy

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8 Citations (Scopus)

Abstract

Background--Duchenne muscular dystrophy (DMD) is frequently complicated by development of a cardiomyopathy. Despite significant medical advances provided to DMD patients over the past 2 decades, there remains a group of DMD patients who die prematurely. The current study sought to identify a set of prognostic factors that portend a worse outcome among adult DMD patients. Methods and Results--A retrospective cohort of 43 consecutive patients was followed in the adult UT Southwestern Neuromuscular Cardiomyopathy Clinic. Clinical data were abstracted from the electronic medical record to generate baseline characteristics. The population was stratified by survival to time of analysis and compared with characteristics associated with death. The DMD population was in the early 20s, with median follow-up times over 2 years. All the patients had developed a cardiomyopathy, with the majority of the patients on angiotensin-converting enzyme inhibitors (86%) and steroids (56%), but few other guideline-directed heart failure medications. Comparison between the nonsurviving and surviving cohorts found several poor prognostic factors, including lower body mass index (17.3 [14.8-19.3] versus 25.8 [20.8-29.1] kg/m2, P<0.01), alanine aminotransferase levels (26 [18-42] versus 53 [37-81] units/L, P=0.001), maximum inspiratory pressures (13 [0-30] versus 33 [25-40] cmH2O, P=0.03), and elevated cardiac biomarkers (N-terminal pro-brain natriuretic peptide: 288 [72-1632] versus 35 [21-135] pg/mL, P=0.03]. Conclusions--The findings demonstrate a DMD population with a high burden of cardiomyopathy. The nonsurviving cohort was comparatively underweight, and had worse respiratory profiles and elevated cardiac biomarkers. Collectively, these factors highlight a high-risk cardiovascular population with a worse prognosis.

Original languageEnglish (US)
Article numbere006340
JournalJournal of the American Heart Association
Volume6
Issue number10
DOIs
StatePublished - Oct 1 2017

Fingerprint

Duchenne Muscular Dystrophy
Cardiomyopathies
Population
Biomarkers
Thinness
Electronic Health Records
Brain Natriuretic Peptide
Alanine Transaminase
Angiotensin-Converting Enzyme Inhibitors
Body Mass Index
Heart Failure
Steroids
Guidelines
Survival

Keywords

  • Cardiac biomarkers
  • Cardiomyopathy
  • Duchenne muscular dystrophy
  • Heart failure therapy
  • Prognostic factors

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine

Cite this

@article{f207e0271b524ee593989f5b051ac219,
title = "Predictors of death in adults with duchenne muscular dystrophy-associated cardiomyopathy",
abstract = "Background--Duchenne muscular dystrophy (DMD) is frequently complicated by development of a cardiomyopathy. Despite significant medical advances provided to DMD patients over the past 2 decades, there remains a group of DMD patients who die prematurely. The current study sought to identify a set of prognostic factors that portend a worse outcome among adult DMD patients. Methods and Results--A retrospective cohort of 43 consecutive patients was followed in the adult UT Southwestern Neuromuscular Cardiomyopathy Clinic. Clinical data were abstracted from the electronic medical record to generate baseline characteristics. The population was stratified by survival to time of analysis and compared with characteristics associated with death. The DMD population was in the early 20s, with median follow-up times over 2 years. All the patients had developed a cardiomyopathy, with the majority of the patients on angiotensin-converting enzyme inhibitors (86{\%}) and steroids (56{\%}), but few other guideline-directed heart failure medications. Comparison between the nonsurviving and surviving cohorts found several poor prognostic factors, including lower body mass index (17.3 [14.8-19.3] versus 25.8 [20.8-29.1] kg/m2, P<0.01), alanine aminotransferase levels (26 [18-42] versus 53 [37-81] units/L, P=0.001), maximum inspiratory pressures (13 [0-30] versus 33 [25-40] cmH2O, P=0.03), and elevated cardiac biomarkers (N-terminal pro-brain natriuretic peptide: 288 [72-1632] versus 35 [21-135] pg/mL, P=0.03]. Conclusions--The findings demonstrate a DMD population with a high burden of cardiomyopathy. The nonsurviving cohort was comparatively underweight, and had worse respiratory profiles and elevated cardiac biomarkers. Collectively, these factors highlight a high-risk cardiovascular population with a worse prognosis.",
keywords = "Cardiac biomarkers, Cardiomyopathy, Duchenne muscular dystrophy, Heart failure therapy, Prognostic factors",
author = "Daniel Cheeran and Shaida Khan and Rohan Khera and Anish Bhatt and Sonia Garg and Grodin, {Justin L.} and Robert Morlend and Araj, {Faris G.} and Amin, {Alpesh A.} and Thibodeau, {Jennifer T.} and Sandeep Das and Drazner, {Mark H.} and Mammen, {Pradeep P.A.}",
year = "2017",
month = "10",
day = "1",
doi = "10.1161/JAHA.117.006340",
language = "English (US)",
volume = "6",
journal = "Journal of the American Heart Association",
issn = "2047-9980",
publisher = "Wiley-Blackwell",
number = "10",

}

TY - JOUR

T1 - Predictors of death in adults with duchenne muscular dystrophy-associated cardiomyopathy

AU - Cheeran, Daniel

AU - Khan, Shaida

AU - Khera, Rohan

AU - Bhatt, Anish

AU - Garg, Sonia

AU - Grodin, Justin L.

AU - Morlend, Robert

AU - Araj, Faris G.

AU - Amin, Alpesh A.

AU - Thibodeau, Jennifer T.

AU - Das, Sandeep

AU - Drazner, Mark H.

AU - Mammen, Pradeep P.A.

PY - 2017/10/1

Y1 - 2017/10/1

N2 - Background--Duchenne muscular dystrophy (DMD) is frequently complicated by development of a cardiomyopathy. Despite significant medical advances provided to DMD patients over the past 2 decades, there remains a group of DMD patients who die prematurely. The current study sought to identify a set of prognostic factors that portend a worse outcome among adult DMD patients. Methods and Results--A retrospective cohort of 43 consecutive patients was followed in the adult UT Southwestern Neuromuscular Cardiomyopathy Clinic. Clinical data were abstracted from the electronic medical record to generate baseline characteristics. The population was stratified by survival to time of analysis and compared with characteristics associated with death. The DMD population was in the early 20s, with median follow-up times over 2 years. All the patients had developed a cardiomyopathy, with the majority of the patients on angiotensin-converting enzyme inhibitors (86%) and steroids (56%), but few other guideline-directed heart failure medications. Comparison between the nonsurviving and surviving cohorts found several poor prognostic factors, including lower body mass index (17.3 [14.8-19.3] versus 25.8 [20.8-29.1] kg/m2, P<0.01), alanine aminotransferase levels (26 [18-42] versus 53 [37-81] units/L, P=0.001), maximum inspiratory pressures (13 [0-30] versus 33 [25-40] cmH2O, P=0.03), and elevated cardiac biomarkers (N-terminal pro-brain natriuretic peptide: 288 [72-1632] versus 35 [21-135] pg/mL, P=0.03]. Conclusions--The findings demonstrate a DMD population with a high burden of cardiomyopathy. The nonsurviving cohort was comparatively underweight, and had worse respiratory profiles and elevated cardiac biomarkers. Collectively, these factors highlight a high-risk cardiovascular population with a worse prognosis.

AB - Background--Duchenne muscular dystrophy (DMD) is frequently complicated by development of a cardiomyopathy. Despite significant medical advances provided to DMD patients over the past 2 decades, there remains a group of DMD patients who die prematurely. The current study sought to identify a set of prognostic factors that portend a worse outcome among adult DMD patients. Methods and Results--A retrospective cohort of 43 consecutive patients was followed in the adult UT Southwestern Neuromuscular Cardiomyopathy Clinic. Clinical data were abstracted from the electronic medical record to generate baseline characteristics. The population was stratified by survival to time of analysis and compared with characteristics associated with death. The DMD population was in the early 20s, with median follow-up times over 2 years. All the patients had developed a cardiomyopathy, with the majority of the patients on angiotensin-converting enzyme inhibitors (86%) and steroids (56%), but few other guideline-directed heart failure medications. Comparison between the nonsurviving and surviving cohorts found several poor prognostic factors, including lower body mass index (17.3 [14.8-19.3] versus 25.8 [20.8-29.1] kg/m2, P<0.01), alanine aminotransferase levels (26 [18-42] versus 53 [37-81] units/L, P=0.001), maximum inspiratory pressures (13 [0-30] versus 33 [25-40] cmH2O, P=0.03), and elevated cardiac biomarkers (N-terminal pro-brain natriuretic peptide: 288 [72-1632] versus 35 [21-135] pg/mL, P=0.03]. Conclusions--The findings demonstrate a DMD population with a high burden of cardiomyopathy. The nonsurviving cohort was comparatively underweight, and had worse respiratory profiles and elevated cardiac biomarkers. Collectively, these factors highlight a high-risk cardiovascular population with a worse prognosis.

KW - Cardiac biomarkers

KW - Cardiomyopathy

KW - Duchenne muscular dystrophy

KW - Heart failure therapy

KW - Prognostic factors

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U2 - 10.1161/JAHA.117.006340

DO - 10.1161/JAHA.117.006340

M3 - Article

C2 - 29042427

AN - SCOPUS:85032213533

VL - 6

JO - Journal of the American Heart Association

JF - Journal of the American Heart Association

SN - 2047-9980

IS - 10

M1 - e006340

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