Care Of Cystic Fibrosis Patients Research Articles

Pulmonary surgery in cystic fibrosis

Cystic fibrosis (CF) has a variety of pulmonary manifestations that include pneumonia, pulmonary abscess, bronchiectasis, hemoptysis, and pneumothorax. Although newer therapies have greatly improved survival of patients with CF, surgical interventions for definitive treatment of these sequelae are often required. Several reports have illustrated that, with the current advances in the perioperative treatment and care of CF patients, surgical interventions for these pulmonary manifestations may be performed safely, resulting in a greatly improved quality of life. Also, although improvements in lung transplantation offer new hope for definitive treatment of those patients with cystic fibrosis, special considerations regarding other surgical issues, such as the prevalence of gastroesophageal reflux, need to be addressed.

Diagnosing and managing cystic fibrosis in the developing world

Cystic fibrosis (CF), earlier believed to be non existent in non Caucasians, is now a pan ethnic disease, having being reported from various regions of the world over last one decade. Apart from limited resources, the major problems in diagnosis and management of CF in developing countries include: lack of awareness among pediatricians, absence of facilities for diagnosis (sweat chloride estimation and genetic studies), lack of trained manpower for care of specific problems, and non availability of appropriate drugs. Care of children with CF may not be a priority for governments in countries where childhood mortality rates are high, predominantly due to acute infections. An indigenously developed and relatively inexpensive method of sweat collection and chloride estimation using pilocarpine iontophoresis and titration, respectively, may be an alternative to the commercially available costly equipment. Having a team of trained nurse, physiotherapist, and dietician for optimal care of CF patients may not be feasible due to inadequate resources. Training a single person (e.g. nurse) to deliver comprehensive CF care may be a feasible alternative. To overcome problems of non availability of appropriate drugs (enzymes, inhaled antibiotics, DNAse, etc), locally available drugs may be used. Examples include use of hypertonic saline in place of DNAse, enteric coated enzyme tablets in place of enteric coated spherules, etc. Factors that are associated with decreased survival in CF patients from developing countries are age of onset of symptoms <2 months, severe malnutrition at the time of diagnosis, colonization with Pseudomonas at the time of diagnosis and frequency of pneumonia >4 episodes/year. All these factors can be modified except onset of symptoms before 2 months of age, by early diagnosis and appropriate treatment. Many of the above mentioned hurdles have been successfully overcome by us to establish CF services in a resource-limited setting. We conclude that education of pediatricians about the disease, early diagnosis using indigenous technology and aggressive physiotherapy with nutritional management and judicious use of antibiotics can improve the quality of life and survival in CF patients in resource-limited settings.

Isolation of Infectious Cystic Fibrosis Patients: Results of a Systematic Review

Respiratory tract infections significantly contribute to morbidity and mortality among cystic fibrosis (CF) patients. Therefore, pathogen transmission needs to be prevented. There are several guidelines for the care of CF patients, but no transparent systematic literature review has been published. We conducted a systematic literature review (January 1966 to September 2004) dealing with segregation of CF patients colonized with Burkholderia cepacia species, Pandoraea species, Pseudomonas aeruginosa, Stenotrophomonas maltophilia, or Alcaligenes species. Quality of studies was evaluated by taking patient population size, existence of control-patients, patient randomization, diagnostic approach, and bacteria typing methods into account. One hundred ninety-nine studies were found. Evidence and quality of 102 publications were evaluated. In 99 publications, recommendations concerning segregation measures for infectious CF patients were determined including a total of 11,576 patients. No randomized, controlled trials had been conducted. Fifty of 56 authors strongly recommended isolation of CF patients infected with B. cepacia or Pandoraea species. In 31 of 39 studies, interpatient spread of Pseudomonas aeruginosa was documented or had been brought to an end by isolation of patients. Only five studies had addressed S. maltophilia or Alcaligenes species. Patients colonized with B. cepacia or Pandoraea species are to be separated from noncolonized patients in single rooms. Patients harboring multidrug-resistant Pseudomonas aeruginosa, S. maltophilia, or Alcaligenes species may not share a room with immunocompromised patients, in intensive care units, or with other CF patients anywhere in the hospital.

Mucoviscidose

Cystic fibrosis (CF) is the most frequent severe genetic disease in Caucasian populations. The modified gene codes for the CFTR (cystic fibrosis transmembrane conductance regulator) protein, a chloride channel which is expressed in many organs. Clinical manifestations begin early in life and are dominated by the respiratory disease which conditions morbidity and mortality. The clinical symptoms are secondary to bronchiectasis and chronic bronchial infection with Staphylococcus aureus and Haemophilus influenzae, then with Pseudomonas aeruginosa, leading to acute exacerbations and finally to chronic respiratory insufficiency. The digestive tract symptoms are the second more frequent symptoms, mainly due to exocrine pancreatic insufficiency, but intestinal or liver disease is possible. Rhino-sinusitis, diabetes, osteoporosis and bilateral absence of the vas deferens are other manifestations of CF. The diagnosis is based on the sweat test and genotyping. Late diagnosis in adulthood is possible. Neonatal screening has been generalised in France in 2002. Care for CF patients is given in paediatric and adult CF centres by a multidisciplinary team who works with a network of caregivers in the hospital and at home. The treatment is based on chest physiotherapy and antibiotics in order to fight bronchial infection, and on digestive and nutritional measures. Lung transplantation is indicated in case of severe respiratory insufficiency. The improvement in the care of CF leads to improvement in survival (median survival above 30 years) and to an increase in the number of adult patients (1/3 of patients) requiring a specific follow-up.

Median household income and mortality rate in cystic fibrosis.

Poverty has been shown to be a determinant of health outcomes in many epidemiologic studies. The goal of this study was to assess the association between household income and the mortality rate in cystic fibrosis (CF) patients. We selected white patients diagnosed before 18 years old and having 1 or more records in the Cystic Fibrosis Foundation Patient Registry since 1991. These 23 817 patients were linked to the 1990 US Census by their zip code of residence. The median household income was adjusted for state level differences in cost of living using the 1998 Consumer Price Index. None. We examined the association between categories of the median household income and the mortality rate. We examined the association between income categories and age-related changes in pulmonary function and body weight as well as specific nutritional and pulmonary therapies. We found a strong monotonic association between the median household income and the mortality rate. The test of trend was significant, and this effect was maintained after adjustment for a variety of patient and disease characteristics. When the lowest income category (<20 000 dollars) is compared with the highest (> or = 50 000 dollars), the adjusted incidence rates were 90.3 and 62.6 per 10 000 person years, respectively; this represents a 44% increased risk of death in the lowest income category. Patients living in areas with lower median household income also had consistently lower pulmonary function and body weight than did those living in higher income areas. The differences in weight percentiles and forced expiratory volume in 1 second are substantial in magnitude, they appear at an early age, and they persist into adulthood for these CF patients. Prescribed nutritional treatments and screening for CF-related diabetes were significantly higher among patients living in areas with lower median household income. Prescription of deoxyribonuclease and inhaled tobramycin was not significantly associated with median household income. There was a strong association between lower household income and increased mortality rate among CF patients. Additional understanding of this effect will require more complete and direct measurement of socioeconomic status and a better understanding of treatment adherence, local environmental conditions, and especially the care of CF patients during the early years of life.

Physical training for cystic fibrosis.

Physical training may form an important part of the care package for patients with cystic fibrosis. To determine whether a prescribed regime of physical training produces improvement or prevents deterioration in physiological and clinical outcomes in cystic fibrosis compared to no training. We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group specialist trials register which comprises references identified from comprehensive electronic database searches, handsearching relevant journals and handsearching abstract books of conference proceedings. Date of the most recent search: August 2001. All randomised controlled clinical trials in which a prescribed regime of physical training is compared to no physical training in patients with cystic fibrosis. Two reviewers independently selected trials for inclusion, assessed methodological quality and extracted data. Of the sixteen trials identified six trials, which included 184 patients, met the inclusion criteria. Exercise tolerance in the short term, during an acute admission was improved in patients who received physical training. In a three year study there was no improvement in exercise tolerance at the end of the study period. Over three years there was a significantly greater mean annual rate of decline in FVC in the control group compared to the exercise group WMD 2.17 (95%CI 0.47, 3.87). Changes in other lung function parameters showed a similar trend, but these were not statistically significant. Conclusions about the efficacy of physical training in cystic fibrosis are limited by the small size, short duration and incomplete reporting of most of the trials included in this review. Physical training is already part of the care package offered to most patients with cystic fibrosis and there is no evidence to actively discourage this. Further research is needed to assess comprehensively the net benefit of the addition of physical training to the care of cystic fibrosis patients.

Identification and antimicrobial susceptibility of Alcaligenes xylosoxidans isolated from patients with cystic fibrosis.

In the past decade, potential pathogens, including Alcaligenes species, have been increasingly recovered from cystic fibrosis (CF) patients. Accurate identification of multiply antibiotic-resistant gram-negative bacilli is critical to understanding the epidemiology and clinical implications of emerging pathogens in CF. We examined the frequency of correct identification of Alcaligenes spp. by microbiology laboratories affiliated with American CF patient care centers. Selective media, an exotoxin A probe for Pseudomonas aeruginosa, and a commercial identification assay, API 20 NE, were used for identification. The activity of antimicrobial agents against these clinical isolates was determined. A total of 106 strains from 78 patients from 49 CF centers in 22 states were studied. Most (89%) were correctly identified by the referring laboratories as Alcaligenes xylosoxidans. However, 12 (11%) strains were misidentified; these were found to be P. aeruginosa (n = 10), Stenotrophomonas maltophilia (n = 1), and Burkholderia cepacia (n = 1). Minocycline, imipenem, meropenem, piperacillin, and piperacillin-tazobactam were the most active since 51, 59, 51, 50, and 55% of strains, respectively, were inhibited. High concentrations of colistin (100 and 200 microg/ml) inhibited 92% of strains. Chloramphenicol paired with minocycline and ciprofloxacin paired with either imipenem or meropenem were the most active combinations and inhibited 40 and 32%, respectively, of strains. Selective media and biochemical identification proved to be useful strategies for distinguishing A. xylosoxidans from other CF pathogens. Standards for processing CF specimens should be developed, and the optimal method for antimicrobial susceptibility testing of A. xylosoxidans should be determined.

Outcomes of intensive care unit care in adults with cystic fibrosis.

Cystic fibrosis (CF) causes progressive respiratory failure and death in more than 90% of patients. Mechanical ventilation has been discouraged in CF because of poor outcomes, but improved survival and the availability of lung transplantation have increased the indications for care of CF patients in the intensive care unit (ICU). We studied the outcomes of all CF patients admitted to the University of North Carolina Hospitals Medical ICU from January 1990 through December 1998. Seventy-six patients, ranging in ages from 17 to 45 yr (mean: 27 yr), and of whom 53% were female, had 136 admissions for exacerbations of CF with respiratory failure (RF, n = 65), hemoptysis (n = 33), antibiotic desensitization (n = 30), pneumothorax (n = 3), or other reasons (n = 5). Eighty-six percent of the patients with hemoptysis and all of those with desensitization and pneumothorax were alive 1 yr after ICU discharge. Of the 42 patients with RF, 37 (88%) required assisted ventilation. Twenty-three (55%) of the patients with RF survived to ICU discharge and 19 (45%) died. Seventeen (40%) of the patients with RF received lung transplants and 14 were alive 1 yr later. Without transplantation, three (7%) of the patients with RF were alive and three (7%) were dead 1 yr later. Sex, body mass index, and respiratory bacteria did not correlate with survival. We conclude that ICU care for adults with CF who have reversible complications is appropriate and effective. Ventilatory support is appropriate for some transplant candidates.

Cystic Fibrosis: End-Stage Care in Canada

To determine the circumstances in which individuals with cystic fibrosis (CF) die, the role of different caregivers, and the extent of palliative care for CF patients. Mailed survey of CF physicians. CF centers in Canada. All CF deaths in 1996 known to centers in Canada. The mean age (+/- SD) at death of the 45 individuals included in the study was 25.8 +/- 13.5 years. The major cause of death was respiratory (34 patients; 75.5%). Nutritional concerns were common. Lung transplantation was considered in 42 patients (93.2%), with 7 patients (17.1%) being entered on a list, but it was carried out in only 2 patients (4.4%). Autopsies were performed on only 10 patients (22.2%). Most patients died in hospital (37 patients; 82.2%), and 7 patients (15.6%) died in ICUs while receiving intermittent positive-pressure ventilation. Palliative care was never discussed in 10 patients (25%). In a further 16 patients (40%), it was not discussed until the last month before death. Respiratory disease remains the most common cause of death in CF patients. Lung transplantation is frequently considered, but most patients die without having had a transplant. Discussions on end-of-life care could be considered sooner.

Distribution of genes encoding putative transmissibility factors among epidemic and nonepidemic strains of Burkholderia cepacia from cystic fibrosis patients in the United Kingdom.

In the last 15 years, Burkholderia cepacia has emerged as a significant pathogen in cystic fibrosis (CF) patients, mainly due to the severity of infection observed in a subset of patients and the fear of transmission of the organism to noncolonized patients. Although patients who deteriorate rapidly cannot be predicted by microbiological characteristics, three genetic markers have been described for strains that spread between patients. These are the cblA gene, encoding giant cable pili; a hybrid of two insertion sequences, IS1356 and IS402; and a 1.4-kb open reading frame known as the B. cepacia epidemic strain marker (BCESM). The latter two are of unknown function. An epidemic strain lineage was previously identified among CF patients in the United Kingdom that apparently had spread from North America and that was characterized by a specific random amplified polymorphic DNA (RAPD) pattern. We searched for the described genetic markers using specific PCR assays with 117 patient isolates of B. cepacia from 40 United Kingdom hospitals. Isolates were grouped according to genomovar and epidemic strain lineage RAPD pattern with a 10-base primer, P272. A total of 41 isolates from patients in 12 hospitals were classified as the epidemic strain, and 40 of these were distributed in genomovars IIIa (11 isolates), IIIb (1 isolate), and IIIc (28 isolates). All isolates of the epidemic strain were positive for the cblA gene and BCESM, but two lacked the insertion sequence hybrid. None of the 76 sporadic isolates contained cblA or the insertion sequence hybrid, but 11 of them were positive for BCESM. Nonepidemic isolates were distributed among genomovars I or IV (9), II (49), IIIa (11), IIIb (3), and IIIc (4). There were three clusters of cross-infection (one involving two patients and two involving three patients) with isolates of genomovar II. We conclude that in the United Kingdom, a single clonal lineage has spread between and within some hospitals providing care for CF patients. The presence of the cblA gene is the most specific marker for the epidemic strain. We recommend that all isolates of B. cepacia from CF patients should be screened by PCR to influence segregation and infection control strategies.

Management of adolescent and adult inpatients with cystic fibrosis

A program in which pharmacists help care for cystic fibrosis patients is described. The Egleston Cystic Fibrosis Center at Emory University houses outpatient clinic facilities and a 10-bed inpatient unit and is affiliated with Egleston Children's Hospital. The center provides full-service care for nearly 500 patients. Patients with mild to moderate exacerbations of pulmonary problems can receive their entire course of therapy at the center, and those with severe illness may complete their hospital stay there. A care team consisting of pharmacists, physicians, nurses, and others provides preventive and acute care. Patients can choose a "care partner" who will assist them with their care during any hospitalizations and at home. Both patient and care partner are taught drug administration, nutrition, and physical therapy and meet regularly with the care team. Patients must receive their medication education from a pharmacist before they can administer their own drugs. Pharmacists at the center also evaluate serum drug concentrations, stock the automated dispensing device, monitor for drug interactions, answer drug information questions, and attend multidisciplinary rounds. Pharmacy residents can work with the care team through rotations and clinic experience. Pharmacists at a cystic fibrosis center provide clinical services to patients and promote self-care.

Prenatal Screening for Cystic Fibrosis Carriers: An Economic Evaluation

The cloning of the CFTR gene has made it technically possible to avert the unwanted birth of a child with cystic fibrosis (CF). Several large trials offering prenatal CF carrier screening suggest that such screening is practical and that identified carriers generally use the information obtained. Therefore, a critical question is whether the cost of such screening is justified. Decision analysis was performed that used information about choices that pregnant women were observed to make at each stage in the Rochester prenatal carrier-screening trial. The cost of screening per CF birth voluntarily averted was estimated to be $1,320,000-$1,400,000. However, the lifetime medical cost of the care of a CF child in today's dollars was estimated to be slightly>$1,000,000. Therefore, despite both the high cost of carrier testing and the relative infrequency of CF conceptions in the general population, the averted medical-care cost resulting from choices freely made are estimated to offset approximately 74%-78% of the costs of a screening program. At present, if it is assumed that a pregnancy terminated because of CF is replaced, the marginal cost for prenatal CF carrier screening is estimated to be $8,290 per quality-adjusted life-year. This value compares favorably with that of many accepted medical services. The cost of prenatal CF carrier screening could fall to equal the averted costs of CF patient care if the cost of carrier testing were to fall to $100.

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 agreedupon 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.

HOW TO EVALUATE CLINICAL PARAMETERS AT A GLANCE

Schiøtz, P.O., Beck, B. and Pedersen, M. (Paediatric Department TG, Rigshospitalet, Copenhagen, Denmark). How to evaluate clinical parameters at a glance. Acta Paediatr Scand 1982; suppl 301: 121‐124. — A brief introduction is given to the practical approach in cystic fibrosis patient care, which is used at the Danish Cystic Fibrosis centre employing Dr. Flensborg's flowsheets.

PROGNOSIS FOR SURVIVAL WITH CYSTIC FIBROSIS: THE EFFECTS OF EARLY DIAGNOSIS AND CYSTIC FIBROSIS CENTER CARE

Warwick, W.J. (Department of Pediatrics, University of Minnesota Hospitals, Minneapolis, Minnesota, 55455, U.S.A.) Prognosis for survival with cystic fibrosis: the effects of early diagnosis and cystic fibrosis center care. Acta Paediatr Scand 1982; suppl 301: 27‐31. — Improved diagnosis, i.e. more cystic fibrosis (CF) patients diagnosed at an early age, is possible but will require a great increase in the sensitivity of physicians, providing care for infants and young children, to the possibility that early symptoms could be CF at a time a baby is “too well to have CF” coupled with a simple fast screening type of sweat test. Prognosis for survival for CF patients treated at CF Centers is improved with early diagnosis but the data concerning newborn screening are still too few. The general care of CF patients around the world in the absence of a CF Center shows expected survival of 50 percent to 4 or 5 years of age; addition of a CF Center and a CF Care Team increases that 50 percent survival to 20 years. Improvement in early diagnosis or newborn screening is needed to reduce the number of CF patients who die undiagnosed. CF Center and CF team care increases the survival age of CF patients by 4 to 6 times. Early diagnosis before pulmonary complications have begun more than double the effectiveness of CF Center care. In Denmark, the Paediatric Department TG provides the leadership and training and clinical care and research needed to maintain progress and to improve upon the prognosis for CF. Data from Denmark obtained through the central Danish CF Registry confirm in Denmark these observations from around the world of the benefits of early diagnosis and center treatment.Improvement in early diagnosis or discovery of and general adoption of a newborn screening program is what is needed to reduce the number of CF patients who die undiagnosed. CF Center and CF Team care increases the survival age of CF patients by 4 to 6 times. Early diagnosis before pulmonary complications have begun more than double the effectiveness of CF Center care. In Denmark, the Paediatric Department TG provides the leadership and training and clinical care and research needed to maintain progress and to improve upon the prognosis for CF.