Stanley B. Fiel

Life Beyond Pediatrics: Transition of Chronically III Adolescents from Pediatric to Adult Health Care Systems

Transition from pediatric to adult health care is fraught with difficulties. On the one hand, the adult care system is not properly prepared to receive patients who are survivors of the so-called childhood disorders. On the other hand, patients and families have difficulty leaving the protective environment created by pediatric caregivers, who in turn may have mixed feelings about letting the patients go. Normalization of development and social adaptation for chronically ill adolescents, however, should include a change in the environment in which health care is received. This article examines some of the issues surrounding transition and transfer from pediatric to adult health care systems and propose some avenues toward implementation of such programs.

Efficacy of Short-Term Corticosteroid Therapy in Outpatient Treatment of Acute Bronchial Asthma

A group of adults with 76 episodes of acute asthma needing emergency therapy, but not requiring hospitalization, were discharged from an emergency department following standardized therapy with bronchodilators. Upon discharge, the patients were treated with a controlled regimen of oral theophylline, and were randomly assigned in double-blind manner to either a placebo treatment (42 patient episodes) or a corticosteroid treatment group (34 patient episodes). The latter were given an intravenous bolus of methylprednisolone followed by an eight-day tapering course of oral methylprednisolone, starting at 32 mg twice a day. Follow-up was carried out seven or 10 days after treatment in the emergency department. Relapse could not be predicted on the basis of peak expiratory flow rates measured during care in the emergency department. Those patients who received corticosteroids had a decrease in the need for repeated emergency care (5.9 percent versus 21 percent for placebo) and fewer respiratory symptoms (15.6 percent versus 36.4 percent for placebo). It is concluded that a short course of high-dose corticosteroids in outpatients reduces the relapse rate and symptoms following an acute asthmatic attack.

Clinical features, survival rate, and prognostic factors in young adults with cystic fibrosis☆

The medical records of 142 patients with cystic fibrosis were reviewed. The patient group included 78 males and 64 females; three patients were black. Periods of observation ranged from two to 25 years (mean, 14.5 years). The analysis focused on clinical evaluation at age 18 years and included information gained at an earlier age. Evaluation at age 18 years was based on Shwachman and Kulczyckis (S-K) scoring system, Brasfield chest roentgenographic scoring system, pulmonary function measurements, height-adjusted weight percentile, sputum bacteriologic results, number of hospitalizations for treatment of pulmonary infections prior to the age of 18 years, time of onset of clubbing, and frequency of complications. There were no significant differences between the sexes in clinical features. Median survival from the time of diagnosis to the conclusion of the study period (1955 to 1984) was 22 years for females and 25 years for males (NS). Median length of survival beyond the age of 18 years was eight years for females and 12 years for males (NS). Stepwise logistic regression and Cox regression analysis applied to 11 variables identified the S-K clinical score at 18 years of age as the best predictor of survival to the age of 23 years. The median durations of survival after the age of 18 years for patients with clinical scores of 30 to 49, 50 to 64, and 65 to 75 at age 18 were five, seven and a half, and 12 years, respectively (p less than 0.0001). Low clinical score, low weight percentile, and Pseudomonas cepacia colonization of the lower respiratory tract at the age of 18 years indicated a poor prognosis. On the other hand, high clinical score, good weight percentile, and colonization with Staphylococcus aureus alone were likely to be found in patients with mild disease and an increased likelihood of long-term survival with preserved pancreatic function.

Vitamin D deficiency is associated with pulmonary dysfunction in cystic fibrosis

BACKGROUND Vitamin D deficiency is common in CF. Whether vitamin D affects pulmonary function in CF is unknown. METHODS Data were abstracted from clinically stable CF patients who had pulmonary function studies and serum 25-hydroxyvitamin D [25(OH)D, ng/ml] levels drawn within 2 months of each other. Findings were adjusted for multiple variables known to affect pulmonary function in CF. RESULTS Enrollees totaled 597. Overall mean 25(OH)D level was 29.6±12.8 ng/ml (SD). Serum 25(OH)D levels showed a significant correlation with forced expiratory volume in 1s (FEV1) % predicted (r=0.20, p<0.0001) and forced vital capacity % predicted (r=0.13, p=0.0019). Multivariate analysis revealed that serum 25(OH)D remained an independent predictor of FEV1 % predicted even after controlling for multiple other factors known to affect CF lung function. CONCLUSIONS Serum 25(OH)D levels are significantly associated with pulmonary function in CF. Further study is required to determine whether this association is causal.

Chronic Obstructive Pulmonary Disease

SummaryChronic obstructive pulmonary disease (COPD) affects an estimated 15 million Americans and is the fourth most common cause of death in the US. Despite an improved understanding of the disease, treatment has changed little over the past 20 to 30 years and COPD remains a major health problem worldwide.Smoking is the major risk factor for COPD, accounting for some 90% of cases. Therefore, smoking cessation remains the most important intervention. Other available pharmacotherapeutic options include corticosteroids, bronchodilators, antibiotics for the treatment of acute exacerbations, mucokinetic agents, and oxygen for the advanced stages of the disease. Recent data investigating the use of dornase alfa (recombinant human deoxyribonuclease I) in patients with acute exacerbations of COPD were disappointing and failed to demonstrate a mortality benefit.While it is likely that ongoing research will optimise the use of older agents and identify new treatment options for patients with COPD, smoking cessation will probably remain the most important intervention and should be a key focus of future initiatives aimed at controlling this disease.

Aerosolized antibiotics in cystic fibrosis: an update

Inhaled antibiotic therapy, targeting Pseudomonas aeruginosa, is a fundamental component of cystic fibrosis (CF) management. Tobramycin inhalation solution (TIS) was approved in the United States (US) in 1998. Subsequent research efforts focused on developing products with a reduced treatment time burden. Aztreonam for inhalation solution (AZLI), administered via a more efficient nebulizer than TIS, was approved in the US in 2010. Dry powder for inhalation (DPI) formulations provide alternatives to nebulized therapy: tobramycin powder for inhalation (also known as TIP™) was approved in the US in 2013, and colistimethate sodium DPI received European approval in 2012. Other aerosolized antibiotics and regimens combining inhaled antibiotics are in development. Inhaled antibiotic rotation (e.g., TIS alternating with AZLI) is an important concept being actively tested in CF.

New treatment modalities for cystic fibrosis.

Refinements in standard therapy for cystic fibrosis have led to dramatic increases in survival and quality of life over the past three decades. Standard therapy has consisted of oral and intravenous antibiotics, chest percussion with postural drainage, and aerosol bronchodilator therapy. The discovery of the cystic fibrosis gene and elucidation of the underlying biochemical defect have broadened our understanding of the pathophysiology of cystic fibrosis and provided a rationale for many new and innovative therapies. Modulation of airway epithelial ion transport may improve mucociliary clearance and delay colonization by infective organisms. Anti-inflammatory therapy may decrease lung injury that results from the hosts attempt to limit airway infection. Supplementation of airway antiproteases may limit the destructive effects of unopposed proteases on pulmonary architecture. Genetic biotechnology has already produced agents that preserve pulmonary function and decrease infectious exacerbations by altering the viscoelastic properties of sputum from patients with cystic fibrosis. Both active and passive immunotherapy are currently being investigated as a measure to delay or combat endobronchial infection with Pseudomonas spp. Aerosolized aminoglycoside antibiotics are being increasingly employed to control pulmonary infection while minimizing systemic toxicity. These treatment modalities, combined with the prospects for gene therapy, provide a brighter outlook for the patient with cystic fibrosis than ever before.