Abstract
Introduction: Cardiomyopathy is a common cause of morbidity and death in patients with Duchenne muscular dystrophy (DMD). Methods: This investigation was a cross-sectional cross-sectional analysis of clinical data from the multi-institutional Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study of 340 DMD patients aged 2-28 years. Cardiomyopathy was defined as shortening fraction (SF) <28% or ejection fraction (EF) <55%. Results: Two hundred thirty-one participants reported a prior clinical echocardiogram study, and 174 had data for SF or EF. The prevalence of cardiomyopathy was 27% (47 of 174), and it was associated significantly with age and clinical stage. The association of cardiomyopathy with age and clinical stage was not changed by glucocorticoid use as a covariate (P>0.68). In patients with cardiomyopathy, 57% (27 of 47) reported not taking any cardiac medications. Cardiac medications were used in 12% (15 of 127) of patients without cardiomyopathy. Conclusions: We found that echocardiograms were underutilized, and cardiomyopathy was undertreated in this DMD natural history cohort.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 250-256 |
| Number of pages | 7 |
| Journal | Muscle and Nerve |
| Volume | 50 |
| Issue number | 2 |
| DOIs | |
| State | Published - Aug 1 2014 |
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Keywords
- Cardiomyopathy
- Duchenne muscular dystrophy
- Echocardiogram
- Glucocorticoid
- Natural history
Cite this
Cooperative international neuromuscular research group duchenne natural history study demonstrates insufficient diagnosis and treatment of cardiomyopathy in duchenne muscular dystrophy. / Spurney, Christopher; Shimizu, Reiko; Morgenroth, Lauren P.; Kolski, Hanna; Gordish-Dressman, Heather; Clemens, Paula R.; Cregan, Michelle; Goude, Erica; Glick, Merete; Johnson, Linda; Han, Jay; Joyce, Nanette; Kilmer, David; Nicorici, Alina; Chidambaranathan, C.; Kumar, Suresh; Eliasoph, Laila; Hosaki, Elizabeth; Gonzales, Angela; Harris, Vivienne; Chiu, Angela; Thannhauser, Jennifer; Walker, Lori; Wright, Caitlin; Yousefi, Mehrnaz; Alhander, Anne Christine; Wahlgren, Lisa; Ekstrom, Anne Berit; Kroksmark, Anna Karin; Sterky, Ulrika; Birkmeier, Marissa; Duong, Tina; Kaminski, Sarah; Carroll, Kate; DeValle, Katy; Kennedy, Rachel; Kornberg, Andrew; Rodriguez, Victoria; Villano, Dani; Leve, Adina Bar; Wisband, Elana; Yaffe, Debbie; Andreone, Luz; Corderi, Jose; Mesa, Lilia; Levi, Lorena; Coleman-Wood, Krista; Hoffman, Ann; Korn-Petersen, Wendy; Kuntz, Nancy; Selcen, Duygu; Abdel-Hamid, Hoda; Bise, Christopher; Craig, Ann; Nguyen, Casey; Weimer, Jason; Al-Lozi, M.; Florence, Julaine; Golumbak, Paul; Lopate, Glenn; Malane, Justin; Malkus, Betsy; Renna, Renee; Schierbacker, Jeanine; Seiner, Catherine; Wulf, Charlie; Blair, Susan; Grillo, Barbara; Jones, Karen; Monasterio, Eugenio; Barrett-Adair, Meegan; Clift, Judy; Feliciano, Cassandra; Young, Rachel; Rose, Kristy; Webster, Richard; Wicks, Stephanie; Chen, Lucia; Kennedy, Cameron; Deliz, Brenda; Espada, Sheila; Fuste, Pura; Luciano, Carlos; Beneggi, Maria; Capone, Luca; Molteni, Alessandro; Morettini, Valentina; Gupta, Anjali; McNeil, Robert; Erickson, Amy; Margolis, Marcia; Naughton, Cameron; Parry, Gareth J; Walk, David; Arrieta, Adrienne; Bartley, Naomi; Canelos, Paola; Casper, Robert; Cnaan, Avital; Duong, Tina; Hu, Fengming; Sund, Zoë; Zimmerman, Angela.
In: Muscle and Nerve, Vol. 50, No. 2, 01.08.2014, p. 250-256.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Cooperative international neuromuscular research group duchenne natural history study demonstrates insufficient diagnosis and treatment of cardiomyopathy in duchenne muscular dystrophy
AU - Spurney, Christopher
AU - Shimizu, Reiko
AU - Morgenroth, Lauren P.
AU - Kolski, Hanna
AU - Gordish-Dressman, Heather
AU - Clemens, Paula R.
AU - Cregan, Michelle
AU - Goude, Erica
AU - Glick, Merete
AU - Johnson, Linda
AU - Han, Jay
AU - Joyce, Nanette
AU - Kilmer, David
AU - Nicorici, Alina
AU - Chidambaranathan, C.
AU - Kumar, Suresh
AU - Eliasoph, Laila
AU - Hosaki, Elizabeth
AU - Gonzales, Angela
AU - Harris, Vivienne
AU - Chiu, Angela
AU - Thannhauser, Jennifer
AU - Walker, Lori
AU - Wright, Caitlin
AU - Yousefi, Mehrnaz
AU - Alhander, Anne Christine
AU - Wahlgren, Lisa
AU - Ekstrom, Anne Berit
AU - Kroksmark, Anna Karin
AU - Sterky, Ulrika
AU - Birkmeier, Marissa
AU - Duong, Tina
AU - Kaminski, Sarah
AU - Carroll, Kate
AU - DeValle, Katy
AU - Kennedy, Rachel
AU - Kornberg, Andrew
AU - Rodriguez, Victoria
AU - Villano, Dani
AU - Leve, Adina Bar
AU - Wisband, Elana
AU - Yaffe, Debbie
AU - Andreone, Luz
AU - Corderi, Jose
AU - Mesa, Lilia
AU - Levi, Lorena
AU - Coleman-Wood, Krista
AU - Hoffman, Ann
AU - Korn-Petersen, Wendy
AU - Kuntz, Nancy
AU - Selcen, Duygu
AU - Abdel-Hamid, Hoda
AU - Bise, Christopher
AU - Craig, Ann
AU - Nguyen, Casey
AU - Weimer, Jason
AU - Al-Lozi, M.
AU - Florence, Julaine
AU - Golumbak, Paul
AU - Lopate, Glenn
AU - Malane, Justin
AU - Malkus, Betsy
AU - Renna, Renee
AU - Schierbacker, Jeanine
AU - Seiner, Catherine
AU - Wulf, Charlie
AU - Blair, Susan
AU - Grillo, Barbara
AU - Jones, Karen
AU - Monasterio, Eugenio
AU - Barrett-Adair, Meegan
AU - Clift, Judy
AU - Feliciano, Cassandra
AU - Young, Rachel
AU - Rose, Kristy
AU - Webster, Richard
AU - Wicks, Stephanie
AU - Chen, Lucia
AU - Kennedy, Cameron
AU - Deliz, Brenda
AU - Espada, Sheila
AU - Fuste, Pura
AU - Luciano, Carlos
AU - Beneggi, Maria
AU - Capone, Luca
AU - Molteni, Alessandro
AU - Morettini, Valentina
AU - Gupta, Anjali
AU - McNeil, Robert
AU - Erickson, Amy
AU - Margolis, Marcia
AU - Naughton, Cameron
AU - Parry, Gareth J
AU - Walk, David
AU - Arrieta, Adrienne
AU - Bartley, Naomi
AU - Canelos, Paola
AU - Casper, Robert
AU - Cnaan, Avital
AU - Duong, Tina
AU - Hu, Fengming
AU - Sund, Zoë
AU - Zimmerman, Angela
PY - 2014/8/1
Y1 - 2014/8/1
N2 - Introduction: Cardiomyopathy is a common cause of morbidity and death in patients with Duchenne muscular dystrophy (DMD). Methods: This investigation was a cross-sectional cross-sectional analysis of clinical data from the multi-institutional Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study of 340 DMD patients aged 2-28 years. Cardiomyopathy was defined as shortening fraction (SF) <28% or ejection fraction (EF) <55%. Results: Two hundred thirty-one participants reported a prior clinical echocardiogram study, and 174 had data for SF or EF. The prevalence of cardiomyopathy was 27% (47 of 174), and it was associated significantly with age and clinical stage. The association of cardiomyopathy with age and clinical stage was not changed by glucocorticoid use as a covariate (P>0.68). In patients with cardiomyopathy, 57% (27 of 47) reported not taking any cardiac medications. Cardiac medications were used in 12% (15 of 127) of patients without cardiomyopathy. Conclusions: We found that echocardiograms were underutilized, and cardiomyopathy was undertreated in this DMD natural history cohort.
AB - Introduction: Cardiomyopathy is a common cause of morbidity and death in patients with Duchenne muscular dystrophy (DMD). Methods: This investigation was a cross-sectional cross-sectional analysis of clinical data from the multi-institutional Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study of 340 DMD patients aged 2-28 years. Cardiomyopathy was defined as shortening fraction (SF) <28% or ejection fraction (EF) <55%. Results: Two hundred thirty-one participants reported a prior clinical echocardiogram study, and 174 had data for SF or EF. The prevalence of cardiomyopathy was 27% (47 of 174), and it was associated significantly with age and clinical stage. The association of cardiomyopathy with age and clinical stage was not changed by glucocorticoid use as a covariate (P>0.68). In patients with cardiomyopathy, 57% (27 of 47) reported not taking any cardiac medications. Cardiac medications were used in 12% (15 of 127) of patients without cardiomyopathy. Conclusions: We found that echocardiograms were underutilized, and cardiomyopathy was undertreated in this DMD natural history cohort.
KW - Cardiomyopathy
KW - Duchenne muscular dystrophy
KW - Echocardiogram
KW - Glucocorticoid
KW - Natural history
UR - http://www.scopus.com/inward/record.url?scp=84904674913&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84904674913&partnerID=8YFLogxK
U2 - 10.1002/mus.24163
DO - 10.1002/mus.24163
M3 - Article
C2 - 24395289
AN - SCOPUS:84904674913
VL - 50
SP - 250
EP - 256
JO - Muscle and Nerve
JF - Muscle and Nerve
SN - 0148-639X
IS - 2
ER -