Staphylococcus aureus, including community-acquired methicillin-resistant S. aureus, in a level III NICU: 2001 to 2008.

OBJECTIVE: To determine epidemiology and clinical characteristics of infants with methicillin-susceptible (MSSA) and -resistant Staphylococcus aureus (MRSA) in a level III neonatal intensive care unit (NICU). STUDY DESIGN: All NICU admissions (2001 to 2008) with any positive S. aureus culture were included as cases. Cases were further characterized as either colonized or infected with invasive disease. RESULTS: Four thousand three hundred four infants were admitted; 273 (6.3%) had at least one culture positive for S. aureus, including 198 with MSSA and 75 with MRSA. Invasive disease occurred in 23.2% of MSSA cases versus 29.3% MRSA (p = 0.298). Between the study periods 2001 to 2005 versus 2006 to 2008, the incidence of all MSSA cultures (colonization and invasive disease) decreased from 53.6 to 38.9/1000 admissions (p = 0.044), and that of MRSA increased from 13.7 to 24.77/1000 admissions (p = 0.010). The incidence of invasive MSSA (p = 0.49) and MRSA (p = 0.38) disease between the two periods remained similar. Infants with invasive MRSA versus MSSA had a longer duration of positive cultures (55 versus 19 days, p = 0.009). None of five available isolates collected prior to 2006 was characterized as USA300, but 11/21 isolates collected subsequently were USA300 (p = 0.053). CONCLUSION: The incidence of MRSA (colonization and infection) nearly doubled during the study period coinciding with emergence of community-acquired MRSA USA300.

The Interplay of Preference, Familiarity and Psychophysical Properties in Defining Relaxation Music.

BACKGROUND: The stress response has been well documented in past music therapy literature. However, hypometabolism, or the relaxation response, has received much less attention. Music therapists have long utilized various music-assisted relaxation techniques with both live and recorded music to elicit such a response. The ongoing proliferations of relaxation music through commercial media and the dire lack of evidence to support such claims warrant attention from healthcare professionals and music therapists. OBJECTIVE: The purpose of these 3 studies was to investigate the correlational relationships between 12 psychophysical properties of music, preference, familiarity, and degree of perceived relaxation in music. METHODS: Fourteen music therapists recommended and analyzed 30 selections of relaxation music. A group of 80 healthy adults then rated their familiarity, preference, and degree of perceived relaxation in the music. RESULTS: The analysis provided a detailed description of the intrinsic properties in music that were perceived to be relaxing by listeners. These properties included tempo, mode, harmonic, rhythmic, instrumental, and melodic complexities, timbre, vocalization/lyrics, pitch range, dynamic variations, and contour. In addition, music preference was highly correlated with listeners' perception of relaxation in music for both music therapists and healthy adults. The correlation between familiarity and degree of relaxation reached significance in the healthy adult group. CONCLUSIONS: Results from this study provided an in-depth operational definition of the intrinsic parameters in relaxation music and also highlighted the importance of preference and familiarity in eliciting the relaxation response.

Lactose-free infant formula does not change outcomes of neonatal abstinence syndrome (NAS): a randomized clinical trial.

OBJECTIVE: To determine if lactose-free formula, compared to lactose-containing formula, decreases the cumulative morphine dose required to treat neonatal abstinence syndrome (NAS). STUDY DESIGN: In a double-blind clinical trial, we randomized 74 infants (36-42 weeks gestation) at risk for developing NAS due to in-utero exposure to opioids to receive either lactose-free (Similac Sensitive®) or lactose-containing (Similac Advance®) infant formula. The primary outcome measure was the cumulative dose of morphine used for the treatment of NAS during the first 14 days of life. RESULTS: Data on 69 (4 withdrew consent and 1 ineligible)/74 randomized infants were analyzed. Patient characteristics between the infant groups fed lactose-free (n = 34) vs. lactose-containing (n = 35) infant formula were similar except more common maternal heroin abuse in the latter group (p = 0.013). Cumulative morphine dose (20.7 ± 19.8 vs. 23 ± 23.5 mg, p = 0.61) between the two groups were similar. CONCLUSION: Lactose-free vs. lactose-containing infant formula did not change the outcomes of infants with NAS.

Progressive left ventricular dysfunction and long-term outcomes in patients with Duchenne muscular dystrophy receiving cardiopulmonary therapies.

Objective: To describe the natural history of cardiomyopathy in patients with Duchenne muscular dystrophy (DMD) who are receiving contemporary therapies. Methods: This is a single-institution retrospective cohort study of 57 patients aged >15 years with DMD. Serial digital echocardiograms were performed over a median follow-up of 8 years. Left ventricular dysfunction (LVD) was defined as shortening fraction (SF) <29% plus focal wall motion abnormalities. Therapies included ACE inhibitors, beta-blockers and assisted ventilation. Results: The SF declined progressively in 53/57 patients (93%). LVD occurred in 40 of 57 patients (70%), with variable age at onset (median 18 years, IQR 14-21.5 years). Rate of SF decline (-1.51%±1.16%/year) was variable and unrelated to genotype. However, survival was shorter for patients with LVD onset at age <18 years vs onset at ≥18 years (death at 21.1±2.5 years vs 33.1±4.4 years; P<0.001). Death occurred in 27/57 (47%) patients at a median age of 26.3 years (IQR 20.6-31.5). Death was preceded by LVD in 22/27 patients (81%), 15 (68%) of whom developed class 4 heart failure (CHF). Time from CHF to death was brief (median 8.0 months). Conclusion: Despite contemporary therapies, SF declined progressively in almost all patients. Age at onset of LVD and age at death were variable and unrelated to genotype; however, survival was shortened for patients with LVD onset at age <18 years. Death was usually preceded by LVD. CHF was a sentinel event, with death occurring shortly thereafter.

Increased neonatal fat mass, not lean body mass, is associated with maternal obesity.

OBJECTIVE: The purpose of this study was to compare body composition measures in neonates of women who were overweight/obese (body mass index, > or = 25 kg/m2) versus women who were lean/average (body mass index, < 25 kg/m2), all of whom had normal glucose tolerance levels. STUDY DESIGN: Seventy-six neonates (34 female and 42 male) of singleton pregnancies of pregravid overweight/obese women and 144 neonates (67 female and 77 male) of lean/average women were assessed with anthropometric measures and total body electrical conductivity evaluation of body composition at birth. RESULTS: There was a borderline increase in birthweight (3436 +/- 567 g vs 3284 +/- 534 g; P = .051) but not lean body mass (3020 +/- 410 g vs 2950 +/- 400 g; P = .23) in the overweight/obese versus lean/average weight groups. However, there were significant increases in percent body fat (11.6% +/- 4.7% vs 9.7 +/- 4.3%; P = .003) and fat mass (420 +/- 220 g vs 380 +/- 170 g; P = .01) in neonates of overweight/obese women versus lean/average weight women. CONCLUSION: Overweight/obese women with normal glucose tolerance levels have neonates who are heavier than lean/average weight women because of increased adiposity. We speculate that this increased obesity in offspring of obese women with normal glucose tolerance levels is a significant risk for adolescent obesity and components of the metabolic syndrome.

Human Milk Analysis Using Mid-Infrared Spectroscopy.

BACKGROUND: The composition of human milk is known to vary with length of gestation, stage of lactation, and other factors. Human milk contains all nutrients required for infant health but requires fortification to meet the needs of low-birth-weight infants. Without a known nutrient profile of the mother's milk or donor milk fed to a baby, the composition of the fortified product is only an estimate. Human milk analysis has the potential to improve the nutrition care of high-risk newborns by increasing the information about human milk composition. Equipment to analyze human milk is available, and the technology is rapidly evolving. This pilot study compares mid-infrared (MIR) spectroscopy to reference laboratory milk analysis. METHODS: After obtaining informed consent, we collected human milk samples from mothers of infants weighing <2 kg at birth. Duplicate samples were analyzed for macronutrients by MIR and by reference laboratory analysis including Kjeldahl for protein, Mojonnier for fat, and high-pressure liquid chromatography for lactose. Intraclass correlation coefficients, Bland-Altman scatter plots, and paired t tests were used to compare the two methods. RESULTS: No significant differences were detected between the macronutrient content of human milk obtained by MIR vs reference laboratory analysis. CONCLUSIONS: MIR analysis appears to provide an accurate assessment of macronutrient content in expressed human milk from mothers of preterm infants. The small sample size of this study limits confidence in the results. Measurement of lactose is confounded by the presence of oligosaccharides. Human milk analysis is a potentially useful tool for establishing an individualized fortification plan.

Making a diagnosis of hypertension and defining treatment threshold in very low birth weight infants' need revision?

INTRODUCTION: Recent evidence suggests that preterm birth is a possible risk factor for high blood pressure (BP) in later life. The most widely quoted blood pressure centiles for very low birth weight (VLBW, ≤1500 g birth weight) infants at corrected term gestation is based on a cohort with mostly late preterm or term infants (Zubrow curves). OBJECTIVES: The objective of this study was to determine the clinical utility of the Zubrow curves in diagnosis of hypertension in VLBW infants at their term corrected gestational age (CGA). PATIENTS AND METHODS: In a case-control study, we compared BP in 75 VLBW infants at 40 weeks CGA (cases) to 69 full term infants admitted to neonatal intensive care unit (NICU) (controls). RESULTS: In spite of having lower weights, VLBW infants compared to term infants (2612.8 ± 546 vs. 3308.2 ± 373 g, P ≤ 0.001) had higher average systolic (88.8 ± 7.6 vs. 82.33 ± 8.5 mm Hg; P ≤ 0.001) and mean BP (61.2 ± 6.6 vs. 57.61 ± 6.9, P = 0.01). Although 41% (31/75) VLBW infants would have met the criteria for hypertension according to Zubrow curves only 4% (3/75) were diagnosed with hypertension. CONCLUSION: Since Zubrow BP centiles were based on a heterogeneous population of infants including preterm and term infants, new BP centiles based on chronological data from VLBW infants would allow a better definition of hypertension in these infants and identify the threshold BP for initiating treatment.

Outcome of children with severe traumatic brain injury who are treated with decompressive craniectomy.

OBJECT Decompressive craniectomy (DC) for the management of severe traumatic brain injury (TBI) is controversial. The authors sought to determine if DC improves the outcome of children with severe TBI. METHODS In a retrospective, case-control study, medical records of all patients admitted to the pediatric ICU between May 1998 and May 2008 with severe TBI and treated with DC were identified and matched to patients who were treated medically without DC. Medical records were reviewed for patients' demographic data and baseline characteristics. RESULTS During the study period, 17 patients with severe TBI treated with DC at a median of 2 hours (interquartile range [IQR] 1-14 hours) after admission were identified and matched to 17 contemporary controls. On admission, there were no differences between DC and control patients regarding age (10.2 ± 5.9 years vs 12.4 ± 5.4 years, respectively [mean ± SD]), sex, weight, Glasgow Coma Scale score (median 5 [IQR 3-7] vs 4 [IQR 3-6], respectively; p = 0.14), or the highest intracranial pressure (median 42 [IQR 22-54] vs 30 [IQR 21-36], respectively; p = 0.77). However, CT findings were significant for a higher rate of herniation and cerebral edema among patients with DC versus controls (7/17 vs 2/17, respectively, had herniation [p = 0.05] and 14/17 vs 6/17, respectively, had cerebral edema [p = 0.006]). Overall there were no significant differences in survival between patients with DC and controls (71% [12/17] vs 82% [14/17], respectively; p = 0.34). However, among survivors, at 4 years (IQR 1-6 years) after the TBI, 42% (5/12) of the DC patients had mild disability or a Glasgow Outcome Scale score of 5 vs none (0/14) of the controls (p = 0.012). CONCLUSIONS In this retrospective, small case-control study, the authors have shown that early DC in pediatric patients with severe TBI improves outcome in survivors. Future prospective randomized controlled studies are needed to confirm these findings.

Autonomic alterations in cocaine-exposed infants.

BACKGROUND: Heart rate variability (HRV) reflects autonomic control of the heart. After intrauterine cocaine exposure, asymptomatic newborn infants within 72 hours of life have decreased HRV. It is unknown whether these alterations are transient (acute effect) or persist in older infants and possibly reflect a teratogenic effect of cocaine. METHODS: This study prospectively evaluated HRV in 2- to 6-month-old infants who were exposed to cocaine in-utero (Group 1, n = 71). Their data were compared to normal controls (Group 3, n = 77) and to newborns exposed to drugs other than cocaine (Group 2, n = 89). Based on our previous study, heavy and light cocaine exposure was also defined a priori as the amount of cocaine used during the pregnancy that was more than or less than the 70th percentile, respectively. RESULTS: At the age of 2 to 6 months, infants with in-utero cocaine exposure had higher vagal tone and higher HRV (total power) than normal controls (no exposure to drugs). Most of this increase in vagal tone occurred in the light-cocaine-exposure group. HRV and vagal tone in the heavy-cocaine-exposure group were similar to the noncocaine-exposed group. CONCLUSIONS: At 2 to 6 months of age, asymptomatic infants exposed to cocaine in-utero have recovered from lower HRV seen within 72 hours of age. Infants exposed to light cocaine recovered by a rebound by increasing their vagal tone to above-normal levels. A similar response was blunted in heavily-cocaine-exposed infants. These alterations noted at follow up suggest a possible teratogenic effect of cocaine on the developing autonomic system.

Heart rate variability in healthy newborn infants.

In adults and older children, heart rate variability (HRV) is frequently used to study autonomic function noninvasively. Normal values of HRV in newborn infants, however, are not widely available. This problem may be partially attributed to the lack of standardization of different methods. This study assessed HRV in normal newborn infants using 24-hour Holter monitoring. From 1997 to 2000, we prospectively evaluated frequency- (spectral analysis), geometric-, and time-domain indexes of HRV in normal term infants. Ninety-six asymptomatic infants who were <72 hours old were studied. Frequency-domain parameters (power in the high, low, very low, ultra low, and total frequency domains), a geometric parameter (HRV triangular index), and time-domain parameters (SDNN, SDANN, SDNNi, r-MSSD, s-NN50) are reported as means +/- SD, medians, and 5th and 95th percentiles to establish the normative values for newborns. A high degree of correlation (r > or = 0.85, p <0.0001) was noted among the 3 vagal tone dependent parameters, such as high-frequency power (frequency domain), r-MSSD, and s-NN50 (time domain). Our study supports the use of vagal dependent time-domain parameters like r-MSSD and sNN50 as surrogates for high-frequency power in newborns. Because the data are reported as means +/- SD, medians, and 5th and 95th percentiles, their use facilitates the study of parasympathetic and sympathetic activity in comparable populations.

Diastolic filling abnormalities by color kinesis in newborns exposed to intrauterine cocaine.

Because cocaine crosses the placenta, we prospectively evaluated global and segmental systolic and diastolic cardiac function by color kinesis in clinically asymptomatic newborns who were exposed to cocaine in utero (group 1, n = 82). Their data were compared with normal controls (group 3, n = 87) and newborns exposed to drugs other than cocaine (group 2, n = 108). During left ventricular filling, newborns exposed to cocaine, compared with groups 2 and 3, had significantly (P <.05) higher global fractional area change (%) (76 +/- 10.3 vs 72 +/- 9.4 and 72 +/- 9.1, respectively), regional fractional area changes (%) for the anterior, septal, inferior, and lateral wall, and in the index of asynchrony (at 50% filling 13.2 +/- 5.8 vs 11.3 +/- 4.1 and 11.6 +/- 4.2, respectively). There were no significant differences in systolic function among the 3 groups. Prenatal cocaine exposure in asymptomatic infants leads to higher global and segmental fractional area changes and asynchrony during diastole. The significance and course of these alterations require further investigation.

Diastolic alterations in infants exposed to intrauterine cocaine: a follow-up study by color kinesis.

BACKGROUND: During the first 48 hours of life, newborn infants exposed to cocaine in utero have left ventricular diastolic segmental abnormalities. It is unknown whether these abnormalities are transient because of short-term effects or persist in older infants, possibly reflecting a teratogenic effect of cocaine. METHODS: This study prospectively evaluated global and segmental systolic and diastolic cardiac parameters by color kinesis. The patients were 2- to 6-month-old infants who were exposed to cocaine in utero (N = 56). Their data were compared with normal control patients with no intrauterine drug exposure (N = 60) and newborns exposed to drugs other than cocaine (N = 72). RESULTS: At the age of 2 to 6 months, there was no significant difference in the measured color kinesis parameters among the cocaine-exposed and the 2 control groups (infants prenatally exposed to other drugs and no drugs). Infants exposed to heavy cocaine prenatally, as compared with the noncocaine-exposed group, had a significant (P =.007) increase in septal fractional area change during left ventricular filling. CONCLUSIONS: At 2 to 6 months of age, infants have recovered from initial left ventricular diastolic segmental alterations seen in the first 48 hours of life except for the septal wall in the heavily cocaine-exposed group.

Quantification of regional left ventricular wall motion in newborns by color kinesis.

BACKGROUND: Normal values for regional left ventricular wall motion, although documented in adults, have not been reported in healthy newborns. METHODS: This study prospectively evaluated global and segmental systolic and diastolic cardiac function by color kinesis in clinically asymptomatic healthy newborns. RESULTS: Eighty-eight asymptomatic infants who were less than 48 hours old were studied. Systolic and diastolic parameters of global and regional left ventricular function are reported as means +/- SD, medians, 5th and 95th percentiles to establish the normative values for newborns. The reported fractional area changes during systole and diastole are similar to the reported normal values for older subjects. Higher body surface area significantly correlated with an increased peak velocity during systole, and fractional area changes during filling of the lateral wall. CONCLUSIONS: Our report of left ventricular regional wall-motion characteristics of healthy newborns, as evaluated by color kinesis, may help in the objective evaluation and management of newborns suspected to have global or segmental ventricular dysfunction.

Prenatal cocaine/polydrug exposure and infant performance on an executive functioning task.

Executive functioning in cocaine/polydrug (marijuana, alcohol, tobacco) exposed infants was assessed in a single session, occurring between 9.5 and 12.5 months of age. In an A-not-B task, infants searched, after performance-adjusted delays, for an object hidden in a new location. Overall, the cocaine-exposed (CE) infants did not differ from non-CE controls recruited from the same at-risk population. However, comparison of heavier-CE (n = 9) to the combined group of lighter-CE (n = 10) and non-CE (n = 32) infants revealed significant differences on A-not-B performance, as well as on global tests of mental and motor development. Covariates investigated included socioeconomic status, marital status, race, maternal age, years of education, weeks of gestation, birth weight, as well as severity of prenatal marijuana, alcohol, and tobacco exposure. The relationship of heavier-CE status to motor development was mediated by length of gestation, and the relationship of heavier-CE status to mental development was confounded with maternal gestational use of cigarettes. The relationship of heavier-CE status to A-not-B performance remained significant after controlling for potentially confounded variables and mediators, but was not statistically significant after controlling for the variance associated with global mental development.

Predicting outcome in neonatal hypoxic respiratory failure with the score for neonatal acute physiology (SNAP) and highest oxygen index (OI) in the first 24 hours of admission.

OBJECTIVE: To determine the clinical utility of SNAP score versus the highest oxygen index (OI) in first 24 hours of admission in predicting outcome of HRF. STUDY DESIGN: All admissions (1991 to 1999) > or =36 weeks gestation, ventilated for > or =12 hours with FiO(2)> or =0.50, without congenital anomalies were reviewed. Primary outcome measure was survival (without ECMO) versus ECMO and/or death. RESULTS: From 184 infants with HRF, 148 survived (without ECMO) versus 36 died and/or received ECMO. SNAP score and highest OI were similar in predicting outcome of HRF (area under ROC curve: 0.813+/-0.037 versus 0.814+/-0.041; P=0.72). Death and/or ECMO requirement were best predicted by a SNAP score of 19 (Sensitivity 75.0%, Specificity 71%) or an OI of 28 (Sensitivity 75.0%, Specificity 76.4%). CONCLUSION: Although both, the SNAP score and highest OI, are useful and similar in predicting outcome of HRF, OI is preferable because of its ease of use. We believe the predictive value of these parameters should be evaluated in a multicenter setting.

The use of the air leak test and corticosteroids in intubated children: a survey of pediatric critical care fellowship directors.

INTRODUCTION: Uncertainties exist regarding the value of the air leak test or use of steroids for preventing post-extubation stridor and extubation failure in children. OBJECTIVE: Determine the practice preferences of pediatric critical care physicians regarding the air leak test and administration of glucocorticosteroids to prevent airway edema. METHODS: A 14-question survey regarding the value of the air leak test, use of glucocorticosteroids, and management of airway edema in intubated children was sent to all North American pediatric critical care fellowship directors affiliated with medical school teaching hospitals. RESULTS: The response rate was 85% (58/68). Seventy-six percent (44/58) routinely check for air leak prior to extubation. The physicians who check for air leak were more likely to delay extubation in order to administer glucocorticosteroids (60% [26/43] vs 15% [2/13], p = 0.01). An air leak of >or= 30 cm H(2)O was more likely (than >or= 20 cm H(2)O) to result in delaying extubation (95% [35/37] vs 51% [19/37], p <0.001). Of the respondents who use steroids for airway edema prophylaxis, 73% (24/33) give steroids based on the air leak test. CONCLUSIONS: The majority of surveyed pediatric critical care fellowship program directors rely on the air leak test and use corticosteroids to prevent post-extubation stridor and extubation failure. At an air leak of >or= 30 cm H(2)O most of the surveyed physicians would delay extubation and initiate glucocorticosteroids.

Development of a 5-item parent questionnaire to screen preschool children for reading problems.

OBJECTIVE: To develop a brief tool for screening of emergent literacy skills in preschool children (3-5 years old) in pediatric clinics. METHODS: Parents were given an 8-item questionnaire, and the children were tested with the Get Ready to Read-Revised (GRTR-R) screener. With the GRTR-R score as gold standard, the parent questionnaire was optimized using various combinations of questions and response weights in one half of the sample. The resulting 5-item questionnaire was then validated using the other half of the sample. RESULTS: A total of 203 patients were enrolled. In the validation sample, the 5-item questionnaire had sensitivity and specificity vis-à-vis the GRTR-R of 100% and 78.6% in 5-year-olds (cutoff score of 8) and 78.6% and 68.2% in 4-year-olds (cutoff of 6). The questionnaire did not perform well in 3-year-olds. CONCLUSION: A very brief parent questionnaire may be useful as a first-line screener for early reading problems.

Metabolic bone disease: a continued challenge in extremely low birth weight infants.

BACKGROUND: Metabolic bone disease (MBD) is an important prematurity-related morbidity, but remains inadequately investigated in extremely low birth weight (ELBW) infants, the group most at risk. The objective was to describe the incidence and associated risk factors of MBD in ELBW infants. METHODS: Retrospective analysis of all ELBW infants admitted between January 2005 and December 2010 who survived > 8 weeks. MBD was defined as the presence of osteopenia or rickets in radiographs. RESULTS: Of the 230 infants included in the study, 71 (30.9%) developed radiological evidence of MBD (cases) of which 24/71 (33.8%) developed spontaneous fractures. MBD and fractures were noted at mean postnatal ages of 58.2 ± 28 and 100.0 ± 61 days, respectively. Compared with controls, cases were smaller at birth (664.6 ± 146 g vs 798.1 ± 129 g), more premature (25.0 ± 1.8 vs 26.4 ± 1.9 weeks), more frequently associated with mechanical ventilation, chronic lung disease, parenteral nutrition days, cholestasis, furosemide, postnatal steroids, and antibiotics use (all P < .01). Cases had lower average weekly intake of calcium, phosphorous, vitamin D, protein, and calories during the first 8 weeks of life compared with controls. Cases with MBD, compared with controls, had higher mortality (14.1 vs 4.4%) and longer hospital stay (140.2 ± 51 vs 101.0 ± 42 days; P < .01). CONCLUSIONS: MBD remains an important morbidity in ELBW infants despite advances in neonatal nutrition. Further research is needed to optimize the management of chronic lung disease and early nutrition in ELBW infants.

Left ventricular dysfunction in duchenne muscular dystrophy and genotype.

Prognosis in patients with Duchenne muscular dystrophy (DMD) is guarded, and most deaths are due to cardiac or respiratory causes. It is unclear if some DMD gene mutations might be predictive of either mild or severe cardiac dysfunction. We studied 75 patients with DMD followed at our institution. Cardiac function, as assessed by yearly echocardiography, showed marked variability in left ventricular (LV) function. Some patients in their 3rd decade had no or minimal dysfunction, whereas others in their 2nd decade had very severe dysfunction. Therefore, 4 severity groups were defined ranging from no or mild LV dysfunction to severe LV dysfunction using patient age at first abnormal echocardiographic finding and degree of LV dysfunction. Genetic data were collected for all patients. Most patients had mutations from exon 1 to 20 to exon 41 to 55. The distribution of the 4 severity groups of LV dysfunction did not significantly differ between these 2 mutation groups. An analysis based on the number of exons involved (<5 vs ≥5 exons) also found no significant difference in cardiac severity. When patients having identical mutations were compared with their cardiac course, concordance was often not evident. Steroid therapy had no apparent protection for the development of cardiomyopathy. In conclusion, 75 patients with DMD showed marked variability in the severity of LV dysfunction. Neither the age of onset nor the severity of cardiomyopathy correlated with any of the mutation groups.