PMID- 26097149
OWN - NLM
STAT- MEDLINE
DCOM- 20160909
LR  - 20201216
IS  - 1099-0496 (Electronic)
IS  - 1099-0496 (Linking)
VI  - 51
IP  - 1
DP  - 2016 Jan
TI  - Cardiac phenotype determines survival in Duchenne muscular dystrophy.
PG  - 70-6
LID - 10.1002/ppul.23215 [doi]
AB  - OBJECTIVE: To identify determinants of survival by comparing cardiopulmonary 
      function in two patient groups: prolonged survivors of Duchenne muscular 
      dystrophy (DMD) versus DMD patients who experienced early death (ED). METHODS: 
      Retrospective chart review of our DMD patients from 1999 to 2013. Prolonged 
      Survival (PS) was defined as alive and >/=30 years old. Early death (ED) was 
      defined as death at < 30 years old. EXCLUSION CRITERIA: steroid therapy. RESULTS: 
      Eleven patients met criteria for PS and 14 patients for ED (mean age +/- SD: 
      34.3 +/- 4.3 years vs. 21.7 +/- 3.8 years, respectively; P < 0.001). Pulmonary 
      function was better in the ED patients: all PS patients had a vital capacity of 
      0 ml (n = 11) versus 23% (3/13) of the ED patients (P < 0.001). Thirteen of 14 ED 
      patients and all PS patients received assisted ventilation. Heart function was 
      worse in the ED patients: ejection fraction (EF) was 42.2 +/- 14.2% in the PS 
      patients (n = 11) versus 29.2 +/- 14.1% in the ED patients (n = 13; P = 0.035). 
      Dilated cardiomyopathy was present in 36% (4/11) of PS patients versus 78% 
      (11/14) of ED patients (P =0.048). Among ED patients, 57% (8/14) died from 
      progressive cardiomyopathy. CONCLUSION: In our study group, good heart function 
      was a pre-condition for PS and poor heart function was the primary cause of early 
      death. Our results suggest that, when DMD patients are treated with assisted 
      ventilation, heart function is the main determinant of their survival.
CI  - (c) 2015 Wiley Periodicals, Inc.
FAU - Birnkrant, David J
AU  - Birnkrant DJ
AD  - Department of Pediatrics, MetroHealth Medical Center, Case Western Reserve 
      University School of Medicine, Cleveland, Ohio.
FAU - Ararat, Erhan
AU  - Ararat E
AD  - Department of Pediatrics, MetroHealth Medical Center, Case Western Reserve 
      University School of Medicine, Cleveland, Ohio.
FAU - Mhanna, Maroun J
AU  - Mhanna MJ
AD  - Department of Pediatrics, MetroHealth Medical Center, Case Western Reserve 
      University School of Medicine, Cleveland, Ohio.
LA  - eng
PT  - Journal Article
DEP - 20150610
PL  - United States
TA  - Pediatr Pulmonol
JT  - Pediatric pulmonology
JID - 8510590
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Cardiomyopathies/*complications/physiopathology
MH  - Female
MH  - Heart/*physiopathology
MH  - Humans
MH  - Lung/*physiopathology
MH  - Male
MH  - Muscular Dystrophy, Duchenne/*complications/physiopathology
MH  - *Phenotype
MH  - Prognosis
MH  - Respiratory Function Tests
MH  - Retrospective Studies
MH  - Risk Factors
MH  - Young Adult
OTO - NOTNLM
OT  - Duchenne muscular dystrophy
OT  - cardiomyopathies
OT  - genes
OT  - genetic therapy
OT  - genotype
OT  - heart function tests
OT  - lung function tests
OT  - modifier
OT  - noninvasive ventilation
OT  - phenotype
OT  - survival
OT  - tracheostomy
EDAT- 2015/06/23 06:00
MHDA- 2016/09/10 06:00
CRDT- 2015/06/23 06:00
PHST- 2015/01/29 00:00 [received]
PHST- 2015/04/06 00:00 [revised]
PHST- 2015/04/23 00:00 [accepted]
PHST- 2015/06/23 06:00 [entrez]
PHST- 2015/06/23 06:00 [pubmed]
PHST- 2016/09/10 06:00 [medline]
AID - 10.1002/ppul.23215 [doi]
PST - ppublish
SO  - Pediatr Pulmonol. 2016 Jan;51(1):70-6. doi: 10.1002/ppul.23215. Epub 2015 Jun 10.