MyMy Buu

All Publications

• Major Adverse Dystrophinopathy Events (MADE) Score as Marker of Cumulative Morbidity and Risk for Mortality in Boys with Duchenne Muscular Dystrophy. Progress in pediatric cardiology Kaufman, B. D., Garcia, A., He, Z., Tesi-Rocha, C., Buu, M., Rosenthal, D., Gordish-Dressman, H., Almond, C. S., Duong, T. 2023; 69

  Abstract

  Overlapping symptoms from cardiomyopathy, respiratory insufficiency, and skeletal myopathy confound assessment of heart failure in Duchenne Muscular Dystrophy. We developed an ordinal scale of multiorgan clinical variables that reflect cumulative disease burden-the Major Adverse Dystrophinopathy Event (MADE) Score. We hypothesized that a higher MADE score would be associated with increased mortality in boys with Duchenne Muscular Dystrophy. The Cooperative International Neuromuscular Research Group Duchenne Natural History Study dataset was utilized for validation.Duchenne Natural History Study variables were selected based on clinical relevance to prespecified domains: Cardiac, Pulmonary, Myopathy, Nutrition. Severity points (0-4) were assigned and summed for study visits. MADE score for cohorts defined by age, ambulatory status, and survival were compared at enrollment and longitudinally.Associations between MADE score and mortality were examined.Duchenne Natural History Study enrolled 440 males, 12.6 ±6.1 years old, with 3,559 visits over 4.6 ±2.8 years, 45 deaths. MADE score increased with age and nonambulatory status. Mean MADE score per visit was 19 ±10 for those who died vs. 9.8 ±9.3 in survivors p=0.03. Baseline MADE score >12 predicted mortality independent of age (78% sensitivity, CPE.70). Rising MADE score trajectory was associated with mortality in models adjusted for enrollment age, follow-up time, and ambulatory status, all p<.001.A multiorgan severity score, MADE, was developed to track cumulative morbidities that impact heart failure in Duchenne muscular dystrophy. MADE score predicted Duchenne Natural History Study mortality. MADE score can be used for serial heart failure assessment in males and may serve as an endpoint for Duchenne muscular dystrophy clinical research.

  View details for DOI 10.1016/j.ppedcard.2023.101639

  View details for PubMedID 37990740

  View details for PubMedCentralID PMC10659574

• Nocturnal hypoventilation as a respiratory complication of acute flaccid myelitis. The Journal of pediatrics Aziz-Bose, R., Bhargava, S., Buu, M., Bove, R., van Haren, K. 2022

  Abstract

  Detailed accounts of long-term respiratory complications among children with acute flaccid myelitis have not been systematically reported. We describe respiratory complications and outcomes in a single-center cohort of 19 children with acute flaccid myelitis. Significantly, 3 of the 19 children had a prolonged course of nocturnal hypoventilation that required intervention.

  View details for DOI 10.1016/j.jpeds.2022.05.032

  View details for PubMedID 35605645

• Consensus on Triple Endoscopy Data Elements Preparatory to Development of an Aerodigestive Registry. The Laryngoscope Boesch, R. P., de Alarcon, A., Piccione, J., Prager, J., Rosen, R., Sidell, D. R., Wootten, C., Balakrishnan, K., Aerodigestive Research Collaborative 2022

  Abstract

  OBJECTIVES/HYPOTHESIS: This study defines essential data elements to be recorded during an aerodigestive "triple endoscopy" to form the foundation of a standardized multicenter registry and to clearly define measurement of each consensus item.STUDY DESIGN: Modified Delphi process.METHODS: Modified Delphi consensus with six survey rounds. Twenty-four expert pediatric otolaryngology, pulmonology, and gastroenterology aerodigestive clinicians from eight large academic pediatric aerodigestive programs formed the Delphi panel. After achieving consensus through the Delphi process, outside validation was performed at 2019 national Aerodigestive Society conference. Consensus, near-consensus, or exclusion was obtained for each proposed data element. Concordance was then measured between expert panel conclusions and validation group conclusions.RESULTS: Overall response rate was 94.4%. 73/167 proposed items reached consensus in six domains (flexible bronchoscopy, bronchoalveolar lavage, microdirect laryngoscopy and bronchoscopy, esophagogastroduodenoscopy with biopsies, and esophageal impedance and pH probe). Measurement of all items was defined; classification/grading systems were selected for 11 items. Validation group endorsed importance of 82/167 data items; compared to expert consensus, overall, inclusion, and exclusion concordance rates were 94.5%, 98.7%, and 90.9%.CONCLUSION: Triple endoscopy is a central component of aerodigestive care. This study identifies and defines data elements to be recorded for all triple endoscopy procedures. The list is of usable length, and clear definitions were created for all items, with explicit classification/grading systems selected for 11 items. Face validity was confirmed with an independent multispecialty sample of aerodigestive providers. This consensus provides the foundation for a triple endoscopy registry but also is immediately applicable to standardize clinical documentation in aerodigestive care.LEVEL OF EVIDENCE: 5 Laryngoscope, 2022.

  View details for DOI 10.1002/lary.30038

  View details for PubMedID 35122443

• Advances in the therapy of Spinal Muscular Atrophy. The Journal of pediatrics Klotz, J., Rocha, C. T., Young, S. D., Duong, T., Buu, M., Sampson, J., Day, J. W. 2021

  View details for DOI 10.1016/j.jpeds.2021.06.033

  View details for PubMedID 34197889

• Gastrostomy Tubes Placed in Children With Neurologic Impairment: Associated Morbidity and Mortality. Journal of child neurology Lin, J. L., Rigdon, J. n., Van Haren, K. n., Buu, M. n., Saynina, O. n., Bhattacharya, J. n., Owens, D. K., Sanders, L. M. 2021: 8830738211000179

  Abstract

  Gastrostomy tube (G-tube) placement for children with neurologic impairment with dysphagia has been suggested for pneumonia prevention. However, prior studies demonstrated an association between G-tube placement and increased risk of pneumonia. We evaluate the association between timing of G-tube placement and death or severe pneumonia in children with neurologic impairment.We included all children enrolled in California Children's Services between July 1, 2009, and June 30, 2014, with neurologic impairment and 1 pneumonia hospitalization. Prior to analysis, children with new G-tubes and those without were 1:2 propensity score matched on sociodemographics, medical complexity, and severity of index hospitalization. We used a time-varying Cox proportional hazard model for subsequent death or composite outcome of death or severe pneumonia to compare those with new G-tubes vs those without, adjusting for covariates described above.A total of 2490 children met eligibility criteria, of whom 219 (9%) died and 789 (32%) had severe pneumonia. Compared to children without G-tubes, children with new G-tubes had decreased risk of death (hazard ratio [HR] 0.47, 95% confidence interval [CI] 0.39-0.55) but increased risk of the composite outcome (HR 1.21, CI 1.14-1.27). Sensitivity analyses using varied time criteria for definitions of G-tube and outcome found that more recent G-tube placement had greater associated risk reduction for death but increased risk of severe pneumonia.Recent G-tube placement is associated with reduced risk of death but increased risk of severe pneumonia. Decisions to place G-tubes for pulmonary indications in children with neurologic impairment should weigh the impact of severe pneumonia on quality of life.

  View details for DOI 10.1177/08830738211000179

  View details for PubMedID 33750232

• Pneumonia Prevention Strategies for Children With Neurologic Impairment. Pediatrics Lin, J. L., Van Haren, K. n., Rigdon, J. n., Saynina, O. n., Song, H. n., Buu, M. C., Thakur, Y. n., Srinivas, N. n., Asch, S. M., Sanders, L. M. 2019

  Abstract

  Children with neurologic impairment (NI) face high risk of recurrent severe pneumonia, with prevention strategies of unknown effectiveness. We evaluated the comparative effectiveness of secondary prevention strategies for severe pneumonia in children with NI.We included children enrolled in California Children's Services between July 1, 2009, and June 30, 2014, with NI and 1 pneumonia hospitalization. We examined associations between subsequent pneumonia hospitalization and expert-recommended prevention strategies: dental care, oral secretion management, gastric acid suppression, gastrostomy tube placement, chest physiotherapy, outpatient antibiotics before index hospitalization, and clinic visit before or after index hospitalization. We used a 1:2 propensity score matched model to adjust for covariates, including sociodemographics, medical complexity, and severity of index hospitalization.Among 3632 children with NI and index pneumonia hospitalization, 1362 (37.5%) had subsequent pneumonia hospitalization. Only dental care was associated with decreased risk of subsequent pneumonia hospitalization (adjusted odds ratio [aOR]: 0.64; 95% confidence interval [CI]: 0.49-0.85). Exposures associated with increased risk included gastrostomy tube placement (aOR: 2.15; 95% CI: 1.63-2.85), chest physiotherapy (aOR: 2.03; 95% CI: 1.29-3.20), outpatient antibiotics before hospitalization (aOR: 1.42; 95% CI: 1.06-1.92), clinic visit before (aOR: 1.30; 95% CI: 1.11-1.52), and after index hospitalization (aOR: 1.72; 95% CI: 1.35-2.20).Dental care was associated with decreased recurrence of severe pneumonia. Several strategies, including gastrostomy tube placement, were associated with increased recurrence, possibly due to unresolved confounding by indication. Our results support a clinical trial of dental care to prevent severe pneumonia in children with NI.

  View details for DOI 10.1542/peds.2019-0543

  View details for PubMedID 31537634

• Tear Down This Wall: Diversity and Disparities in Cystic Fibrosis AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE Buu, M. C., Milla, C. E. 2018; 198 (8): 983-984

  View details for DOI 10.1164/rccm.201806-1076ED

  View details for Web of Science ID 000447405000006

• Tear Down this Wall: Diversity and Disparities in Cystic fibrosis. American journal of respiratory and critical care medicine Buu, M. C., Milla, C. E. 2018

  View details for PubMedID 30063377

• Respiratory complications, management and treatments for neuromuscular disease in children CURRENT OPINION IN PEDIATRICS Buu, M. C. 2017; 29 (3): 326-333

  Abstract

  To summarize current literature describing the respiratory complications of neuromuscular disease (NMD) and the effect of respiratory interventions and to explore new gene therapies for patients with NMD.Measurements of respiratory function focus on vital capacity and maximal inspiratory and expiratory pressure and show decline over time. Management of respiratory complications includes lung volume recruitment, mechanical insufflation-exsufflation, chest physiotherapy and assisted ventilation. Lung volume recruitment can slow the progression of lung restriction. New gene-specific therapies for Duchenne muscular dystrophy and spinal muscular atrophy have the potential to preserve respiratory function longitudinally. However, the long-term therapeutic benefit remains unknown.Although NMDs are heterogeneous, many lead to progressive muscle weakness that compromises the function of the respiratory system including upper airway tone, cough and secretion clearance and chest wall support. Respiratory therapies augment or support the normal function of these components of the respiratory system. From a respiratory perspective, the new mutation and gene-specific therapies for NMD are likely to confer long-term therapeutic benefit. More sensitive and standard tools to assess respiratory function longitudinally are needed to monitor respiratory complications in children with NMD, particularly the youngest patients.

  View details for DOI 10.1097/MOP.0000000000000498

  View details for Web of Science ID 000401074000012

  View details for PubMedID 28338488

• Developing Gene-Specific Meta-Predictor of Variant Pathogenicity bioRxiv Rychkova, A., Buu, M., Scharfe, C., Lefterova, M., Odegaard, J., Schrijver, I., Milla, C., Bustamante, C. 2017

  View details for DOI 10.1101/115956

• Ocular manifestations of pulmonary diseases The Eye in Pediatric Systemic Disease Yoo, S., Buu, M. Springer International Publishing. 2017
• Evaluating Outcomes Disparities in the Hispanic Cystic Fibrosis Population A Need for a National Analysis Response CHEST Buu, M. C., Milla, C. E., Wise, P. H. 2016; 150 (3): 753

  View details for PubMedID 27613985

• Assessing Differences in Mortality Rates and Risk Factors Between Hispanic and Non-Hispanic Patients With Cystic Fibrosis in California CHEST Buu, M. C., Sanders, L. M., Mayo, J. A., Milla, C. E., Wise, P. H. 2016; 149 (2): 380-389

  Abstract

  Over the past 30 years, therapeutic advances have extended the median life span of patients with cystic fibrosis (CF). Hispanic patients are a vulnerable subpopulation with high of prevalence of risk factors for worse health outcomes. The consequences of these differences on health outcomes have not been well described. The objective of this study is to characterize the difference in health outcomes, including mortality rate, between Hispanic and non-Hispanic patients with CF.Retrospective analysis of CF Foundation patient registry data of California residents with CF, diagnosed during or after 1991, from 1991-2010. Ethnicity was self-reported. Primary outcome was mortality. Hazard ratios were estimated from a Cox regression model, stratified by gender and adjusted for socioeconomic status, clinical risk factors, and year of diagnosis.Of 1719, 485 (28.2%) self-identified as Hispanic. Eighty-five deaths occurred, with an overall mortality rate of 4.9%. Unadjusted mortality rate was higher among Hispanic patients than non-Hispanic patients (9.1% vs. 3.3%, p<0.0001). Compared with non-Hispanic patients, Hispanic patients had lower survival rate 18 years post-diagnosis (75.9% vs. 91.5%, p<0.0001). Adjusted for socioeconomic status and clinical risk factors, Hispanic patients had increased rate of death compared to non-Hispanic patients (HR 2.81, 95% CI 1.70-4.63).Hispanic patients with CF have a higher mortality rate than non-Hispanic patients, even after adjusting for socioeconomic status and clinical severity. Further investigation of mechanism for the measured difference in lung function will help inform interventions and improve the health of all CF patients.

  View details for DOI 10.1378/chest.14-2189

  View details for Web of Science ID 000369660400021

• Asthma, tobacco smoke and the indoor environment: a qualitative study of sheltered homeless families JOURNAL OF ASTHMA Buu, M. C., Carter, L., Bruce, J. S., Baca, E. A., Greenberg, B., Chamberlain, L. J. 2014; 51 (2): 142-148

  Abstract

  Asthma is common in homeless children with an incidence of 28-40%. There are few published studies investigating asthma in homeless children. This study examines the perspectives of both caregivers and shelter staff regarding challenges and opportunities of caring for children with asthma.A focus group of sheltered parents (n = 10) with children who have asthma was conducted to identify barriers to optimal asthma management. Key informant interviews (n = 6) were conducted with shelter staff to discuss the shelter systems and policies to address childhood asthma. Data were audio-recorded and transcribed. A representative analysis team performed qualitative theme analysis.Key themes across 5 domains were identified: asthma education, access to asthma medication and equipment, asthma action plans, structural barriers to asthma management and environmental triggers. Parents identified multiple asthma triggers present in the shelter environment but cited lack of control as a barrier to remediation. Shelter staff desired elimination of asthma triggers but refer to the lack of resources as the primary barrier. Shelter staff favored a smoking ban on shelter property but named challenges to policy implementation. Both parents and staff identified asthma education and increased access to medications would be helpful.Policies to reduce environmental exposures, such as a smoking ban, to asthma triggers has the potential to improve the health of sheltered children with asthma.

  View details for DOI 10.3109/02770903.2013.857682

  View details for Web of Science ID 000331908900005

  View details for PubMedID 24147583