Samya Z. Nasr

Transition program from pediatric to adult care for Cystic Fibrosis patients

A survey of adolescents and young adults with cystic fibrosis (CF) attending an adult CF center was conducted to evaluate a transition program as a means of transferring care from pediatric to adult setting. A total of 40 patients completed a self-administered questionnaire of whom 13 (32.5%) went through the transition program, and 21 (52.5%) received their care at the University of Michigan Cystic Fibrosis Center. Fourteen patients (35.0%) were cared for at other pediatric CF centers, and five (12.5%) were first diagnosed as adults. All those in the transition program approved of having the visit with the internal medicine physician in the pediatric clinic. Most thought that the transition program made the change from pediatric to adult care easier. All patients were comfortable leaving the pediatric clinic after the transition period. Of the 40 patients, 17 (42%) recommended that other patients go through the transition program, although 9 of these 17 patients did not themselves go through the transition program. Twenty-six patients (65%) preferred the adult program. These findings suggest that adolescents with CF should be encouraged to transfer their medical care to an adult CF Center once they have reached an agreed-upon age. This process should be smooth and should occur as part of the regular CF care. This is possible through a well-structured and well-organized transition program with committed pediatric and adult staff.

Identifying barriers to treatment adherence and related attitudinal patterns in adolescents with cystic fibrosis.

The treatment of cystic fibrosis (CF) is directed toward correction of organ dysfunction and relief of symptoms resulting from the disease. Lack of adherence to daily treatment regimens may have substantial short‐term and long‐term effects on patients with CF. In this study, we attempted to identify barriers to treatment adherence which could be predicted by objective measures and explore ways to improve adherence in adolescents with CF.

Evaluation of a cystic fibrosis transition program from pediatric to adult care.

As the cystic fibrosis (CF) patient population median survival increase, the need for transitioning their care to adult care centers increase as well. We have a structured transition program since the early 1980s. The purpose of this study is to evaluate the experiences and opinions of patients in our adult CF center who went through a formal transition versus those who did not, in an attempt to evaluate the overall process and to identify means for improvement.

EUR-1008 pancreatic enzyme replacement is safe and effective in patients with cystic fibrosis and pancreatic insufficiency

BACKGROUND EUR-1008 (Zenpep [pancrelipase]) is a new, enteric-coated, porcine-derived pancreatic enzyme product (PEP) developed for the treatment of cystic fibrosis (CF) patients with malabsorption associated with exocrine pancreatic insufficiency (EPI). Unlike currently marketed PEPs, EUR-1008 contains the label-claimed lipase content. Safety and efficacy were assessed in younger (<7 years) and older (> or =7 years) CF patients with EPI. METHODS Two multicenter studies were conducted: a randomized, double-blind, placebo-controlled, crossover trial in patients > or =7 years of age (N=34) and a supplemental, open-label study in children <7 years of age (N=19). Use of any medications altering gastric pH/motility was prohibited during the studies. Outcome measures in the randomized trial included changes in the coefficient of fat absorption (CFA), coefficient of nitrogen absorption (CNA), and signs/symptoms of malabsorption for EUR-1008 vs. placebo. Outcome measures in the supplemental study included safety and response (defined as no steatorrhea and no overt signs/symptoms of malabsorption) to EUR-1008 vs. previous enzyme treatment. RESULTS In the randomized trial, EUR-1008 treatment compared to placebo resulted in a significantly higher mean CFA (88.3% vs. 62.8%, respectively) and CNA (87.2% vs. 65.7%, respectively) (both p<0.001) and reduced the incidence of malabsorption signs and symptoms in 32 evaluable patients. In the supplemental study, 11 of 19 patients met the criteria for responder with EUR-1008 at the end of the study vs. 10 of 19 patients at screening (previous PEP), and improvements in clinical symptoms were reported with EUR-1008 treatment. EUR-1008 was safe and well tolerated, and no serious drug-related AEs were reported in either study. CONCLUSIONS EUR-1008 was safe, well tolerated, and effective in CF patients of all ages with EPI-associated malabsorption in two clinical trials. Treatment led to clinically and statistically significant improvements in CFA and CNA in the randomized study, and control of malabsorption and clinical symptoms in both studies.

Appetite Stimulants Use in Cystic Fibrosis

Cystic fibrosis (CF) is an autosomal recessive disease. It affects multiple body organs. The lungs and pancreas are the most affected which results in progressive lung damage and pancreatic insufficiency. Due to the disease process, CF patients require significantly higher caloric intake than recommended for other individuals. The nutritional goal for CF patients is to achieve normal growth and development and, once genetic potential is reached, to maintain good nutritional status throughout life. Evidence has shown that lung function is closely associated with nutritional status in CF and that nutritional status is an independent predictor of survival. Most CF patients are on a high calorie diet to help achieve normal growth and development and maintain good lung function. Inadequate caloric intake in CF can lead to malnutrition. Malnutrition in CF requires careful, multidisciplinary history taking, physical exam, and overall patient/family assessment. Only by determining the actual cause of the malnutrition can appropriate and safe therapies be used to treat it. Appetite stimulants, although efficacious in treating malnutrition in CF, should only be prescribed if decreased food intake secondary to inadequate appetite is the principal cause of the malnutrition and all other contributing factors have been assessed, ruled‐out or treated. In this review, we attempted to summarize the use of several appetite stimulants used in CF and other diseases to improve appetite and maximize caloric intake. Pediatr Pulmonol. 2008; 43:209–219.

Adherence to dornase alfa treatment among commercially insured patients with cystic fibrosis

Abstract Objective: To investigate adherence to dornase alfa therapy among commercially-insured patients with cystic fibrosis (CF) and to examine the impact of adherence on health and economic outcomes. Methods: This retrospective cohort analysis included CF patients with ≥1 dornase alfa (Pulmozyme) pharmacy claim between 1 October 2006 and 30 September 2008 and with continuous enrollment in the health insurance plan at least 1 year before and 1 year after their index dornase alfa claim. Adherence was measured with the medication possession ratio (MPR). Multivariate models were used to estimate the relationship between adherence and exacerbations, utilization, and cost. Results: Nine hundred and seven patients met the inclusion criteria. The mean age was 19.5 years (SD = 11.5) and 49.1% were female. Overall MPR was 0.59 and by age was 0.66 for patients of 5–12 years, 0.57 for 13–20 years, 0.54 for 21–30 years, and 0.56 for patients ≥31 years. Adherence was better in fall and winter than in spring and summer. There was no statistically significant difference in the proportion of patients with inpatient respiratory exacerbations across groups with low (<0.5), moderate (0.5–0.79), and high (≥0.8) adherence (24.5%, 22.3%, and 19.1%, respectively, p = 0.250). There was a trend toward higher total charges in more-adherent patients (mean

Treatment of Anorexia and Weight Loss With Megestrol Acetate in Patients With Cystic Fibrosis

58,612 in the least-adherent group and mean

Effect of Treatment of Cystic Fibrosis Pulmonary Exacerbations on Systemic Inflammation

69,427 in the most adherent group, p = 0.107). In multivariate models, MPR was not significantly associated with the risk of inpatient respiratory exacerbations (hazard ratio = 1.16 for MPR <0.5 vs ≥0.8; 95% CI = 0.83–1.61). Limitations: Study data were derived from insurance claims; adherence measures were based on prescription fills, not observed medication use. Conclusion: Adherence to dornase alfa was generally low, but varied by age and season. Adherence was not found to be significantly associated with respiratory exacerbations or total charges, but was associated with shorter hospital length of stay.

Tobramycin inhalation powder in cystic fibrosis patients: response by age group.

Four patients with severe cystic fibrosis lung disease, anorexia and weight loss, received Megestrol Acetate (MA), as an appetite stimulant. The initial dose was 400–800 mg daily and was continued for 6–15 months. Appetite was improved, with significant weight gain in all patients and an increase in their weight for age percentile from <5% at the start of the study to approximately the 25th percentile after 6 months of use and improvement in quality of life. One patient discontinued MA after 6 months, and subsequently appetite and weight were depressed. Pediatr Pulmonol. 1999; 28:380–382.

The use of high resolution computerized tomography (HRCT) of the chest in evaluating the effect of tobramycin solution for inhalation in cystic fibrosis lung disease

RATIONALE In cystic fibrosis (CF), pulmonary exacerbations present an opportunity to define the effect of antibiotic therapy on systemic measures of inflammation. OBJECTIVES Investigate whether plasma inflammatory proteins demonstrate and predict a clinical response to antibiotic therapy and determine which proteins are associated with measures of clinical improvement. METHODS In this multicenter study, a panel of 15 plasma proteins was measured at the onset and end of treatment for pulmonary exacerbation and at a clinically stable visit in patients with CF who were 10 years of age or older. MEASUREMENTS AND MAIN RESULTS Significant reductions in 10 plasma proteins were observed in 103 patients who had paired blood collections during antibiotic treatment for pulmonary exacerbations. Plasma C-reactive protein, serum amyloid A, calprotectin, and neutrophil elastase antiprotease complexes correlated most strongly with clinical measures at exacerbation onset. Reductions in C-reactive protein, serum amyloid A, IL-1ra, and haptoglobin were most associated with improvements in lung function with antibiotic therapy. Having higher IL-6, IL-8, and α1-antitrypsin (α1AT) levels at exacerbation onset were associated with an increased risk of being a nonresponder (i.e., failing to recover to baseline FEV1). Baseline IL-8, neutrophil elastase antiprotease complexes, and α1AT along with changes in several plasma proteins with antibiotic treatment, in combination with FEV1 at exacerbation onset, were predictive of being a treatment responder. CONCLUSIONS Circulating inflammatory proteins demonstrate and predict a response to treatment of CF pulmonary exacerbations. A systemic biomarker panel could speed up drug discovery, leading to a quicker, more efficient drug development process for the CF community.