Cystic fibrosis: current survival and population estimates to the year 2000.

BACKGROUND: Survival from cystic fibrosis is increasing rapidly. Estimates of the extent of this improvement should allow health care facilities to be planned to deal with the expanding population of patients with cystic fibrosis. Estimates of life expectancy are also essential if accurate information on current prognosis is to be given to parents of an affected child, or to prospective parents deciding whether to proceed with a pregnancy where the fetus may be affected. METHODS: Survival trends in the national data on cystic fibrosis have been analysed to produce estimates of the likely size of the cystic fibrosis population over the next decade and to predict the life expectancy of children born with cystic fibrosis in the years up to 1990. RESULTS: In England and Wales the estimated number of patients with cystic fibrosis is at present about 5200, of whom 3300 (63%) are aged under 16 years. By the year 2000 the total population will increase to 6000, with 3400 (57%) aged under 16. Thus the number of children with cystic fibrosis will remain fairly constant over the next 10 years, whereas adult numbers will increase by about 36% (from 1901 to 2577). The median life expectancy of children with cystic fibrosis born in 1990 is estimated to be 40 years, double that of 20 years ago. CONCLUSION: This study suggests that health service provision for children will not need to change substantially over the next 10 years whereas services for adults will need to increase by about a third. Parents can be counselled that the median life expectancy of a newborn child with cystic fibrosis is currently likely to be of the order of 40 years.     

Effects of hospital volume on life expectancy after selected cancer operations in older adults: a decision analysis

BACKGROUND: In addition to lower operative mortality, patients undergoing selected cancer operations at high volume centers have improved longterm survival. We sought to determine the overall effect of hospital volume on life expectancy after cancer surgery. STUDY DESIGN: We used a Markov decision analysis model to estimate life expectancy for patients undergoing resection for pancreatic, lung, or colon cancer. Model inputs included probabilities of operative mortality and longterm survival. For input data, we examined operative mortality (in-hospital or within 30 days) stratified by volume in over 400,000 patients undergoing resection for these three cancers using the national Medicare database (1994-1999). Risks of late mortality were abstracted from published studies (MEDLINE, 1966 to present) to model the effect of hospital volume on longterm survival. In analysis, we first calculated life expectancy for patients undergoing surgery at very low, low, medium, high, and very high volume hospitals. We then explored the effects of various regionalization strategies. RESULTS: Life expectancy increased steadily with hospital volume for all three cancers. Life expectancy after pancreatic cancer resection increased linearly with hospital volume: from 1.9 years at very low volume centers to 3.6 years at very high volume centers. For lung cancer, life expectancy ranged from 5.4 to 6.6 years. Increases in life expectancy for colon cancer were not as dramatic: from 6.8 at very low volume hospitals to 7.4 years at very high volume hospitals. Differences in life expectancy across volume strata were largely attributable to differences in longterm survival, not operative mortality. From a policy perspective, regionalizing surgery for colon cancer would produce the greatest overall life-expectancy gains, but it would require moving most patients. CONCLUSIONS: Patients aged 65 and older with pancreatic, lung, and colon cancer have substantially greater life expectancy after cancer resection at higher volume hospitals. Further work is needed to understand the mechanisms underlying differences in performance across hospitals in cancer care.     

The prevalence,clinical status and genotype of cystic fibrosis patients living in Cuba using national registry data

BackgroundWe aimed to establish a national cystic fibrosis (CF) registry for Cuba, a developing country.MethodsRegional centres that deliver care for all CF patients provided information for a national database.FindingsThe prevalence of CF in Cuba is 26.3 cases per 1,000,000 population. The median age at diagnosis is 2 years, and the median age of the total population was 15 years. Of those aged 16 years or older, the prevalence of Pseudomonas aeruginosa infection was 46%, the prevalence of Staphylococcus aureus infection was 36%, and 80% of individuals were receiving oral azithromycin. The commonest gene mutation was F508del which was observed in 50% of patients.InterpretationThese data demonstrate that it is possible to establish a national CF registry in a developing country such as Cuba. This provides baseline data to permit evaluation of health care delivery enable the spread of good clinical practice nationally.     

Survival estimates for adults with cystic fibrosis born in the United Kingdom between 1947 and 1967. The UK Cystic Fibrosis Survey Management Committee

BACKGROUND: The UK has published observed cohort survival figures for subjects with cystic fibrosis born since 1968. Prior to 1968 cohorts cannot be established directly from routine data as cystic fibrosis was classified with a number of unrelated conditions in ICD7. Reported here are interrupted survival curves from 1978 for patients with cystic fibrosis born before 1968. METHODS: Life tables for the three year cohorts born between 1947 and 1967 were constructed by firstly estimating the numbers of patients with cystic fibrosis born in each cohort from live birth data and the disease incidence. The number of the estimated cohort that had survived to 1978 is known, which enables the proportion surviving to 1978 to be calculated. The survival of these cohorts after 1978 can be calculated in the usual way. RESULTS: The survival for each successive cohort was better than that of the previous one, but most of the improvements appear to have taken place up to the age of about 20 years. Only 3% of the 1947-49 cohort survived to 30 years of age compared with 21% for the 1965-67 cohort, and 3% of the 1953-55 cohort survived to 40 years of age. For the later cohorts the mortality rate for those aged between 26 and 30 years appears to be about 50 per 1000 per year. CONCLUSIONS: While the trend in the numbers surviving into later adulthood is upwards, the mortality rates for these ages does not appear to be improving. It is not possible to tell from these data whether the high mortality rates in adulthood will improve with better resourced adult clinics or with improved treatment during childhood.     

Amyloidosis in cystic fibrosis: a case series.

As the life expectancy of patients with cystic fibrosis increases, unusual complications including amyloidosis are increasingly recognised. We report three cases of amyloidosis including a unique case presenting with a hemorrhagic and thrombotic diatheisis. This complication of amyloidosis has never been reported in cystic fibrosis.     

Getting the Job Done: Analysis of the Impact and Effectiveness of the SmileTrain Program in Alleviating the Global Burden of Cleft Disease

Background

The study measured the success of SmileTrain, the largest cleft charity globally, in alleviating the global burden of disease (GBD). It was done by estimating averted disability-adjusted life years (DALYs) and delayed averted DALYs because of the global backlog in cleft procedures.

Methods

Anonymized data for all procedures in the SmileTrain global database were analyzed by age, sex, country, region, and surgery type. DALYs averted were calculated using life expectancy tables and established and estimated disability weights. The cost-effectiveness analysis used mean SmileTrain procedural disbursement figures. Sensitivity analysis was performed using various cleft incidence rates, life expectancy tables, and disability weights.

Results

During 2003–2010 a total of 536,846 operations were performed on 364,467 patients—86 % in Southeast Asia and the western Pacific region. Procedure numbers increased yearly. Mean age at primary surgery—6.2 years (9.8 years in Africa)—remained fairly constant over time in each region. Globally, 2.1–4.7 million DALYs were averted through the operations at a total estimated cost of US$196 M. Mean DALYs per patient were 3.8–9.0, and mean cost per DALY was $72–$134. Total delayed GBD due to advanced age at surgery was 191,000–457,000 DALYs.

Conclusions

Despite an unparalleled number of surgeries performed and yearly increase by one charity, the unmet and delayed averted cleft GBD remains significant in all regions. Large geographic disparities reflect varied challenges regarding access to surgery. Cleft surgeries are cost-effective interventions to reduce the global burden of disease (GBD). Future challenges include increased collaboration among cleft care providers and a focus on remote global areas by building infrastructure and local training.     

Long term prognosis of patients with cystic fibrosis in relation to early detection by neonatal screening and treatment in a cystic fibrosis centre.

BACKGROUND--A study was undertaken to evaluate whether an early diagnosis by neonatal screening may improve the long term prognosis of patients with cystic fibrosis and to assess the influence of expert management started immediately after the diagnosis. METHODS--Comparative clinical follow up in three birth cohorts of patients with cystic fibrosis was performed at the Cystic Fibrosis centre in Groningen in close collaboration with paediatricians in general hospitals in the north-eastern part of the Netherlands. The first birth cohort (n = 19) was detected by screening and the two other cohorts were detected clinically, one (n = 30) consisting of patients born during the screening programme and the other (n = 32) of patients born during the six years immediately after the screening programme ended. The total number of patients in the three birth cohorts included all patients with cystic fibrosis born in this area during a 12 year period. Cumulative survival rates and the variation with time of lung function, the levels of immunoglobulins, and growth patterns were used as main outcome measures. RESULTS--Patients born during the screening programme but detected clinically appeared to have a reduced life expectancy compared with patients detected by screening. The patients detected by screening showed less deterioration in lung function (annual decrease 1.2% of FEV1 % pred), a smaller increase in immunoglobulin levels, and minimal catch-up growth compared with an annual decrease of 3.25% of FEV1 % pred in the non-screened birth cohort of the same age, a higher rise in immunoglobulins leading to increased levels at the end of the observation period, and catch-up growth for weight as well as height. Differences between patients treated in a cystic fibrosis centre and those not referred to a specialist centre were smaller but similar, in favour of treatment at a cystic fibrosis clinic. CONCLUSIONS--Expert management started immediately after an early diagnosis of cystic fibrosis by neonatal screening results in important beneficial effects on the outcome and clinical course of the condition. The institution of very early treatment may be critical for the outcome and long term prognosis for most patients with cystic fibrosis. Neonatal screening programmes for cystic fibrosis should be introduced more widely.     

Variceal hemorrhage and cystic fibrosis: outcomes and implications for liver transplantation.

Autopsy and imaging studies show that liver involvement is common in cystic fibrosis. However, complications of chronic liver disease including portal hypertension and variceal bleeding are infrequently encountered, and the degree to which variceal hemorrhage affects prognosis in cystic fibrosis is unclear. This uncertainty has lead to debate as to whether liver transplantation is indicated in these patients. We describe a case series of 18 patients and compare their survival with a control group of cystic fibrosis patients without liver disease. The median age at first bleed was 20.0 years (range 9.7-30.9). The median survival after first bleed was 8.4 years, compared to 13.0 years in the control group (P = 0.15). A total of 14 patients have died, 9 from respiratory disease with no discernable contribution from their liver disease. Liver disease contributed to 4 deaths. Only 1 patient suffered a fatal hemorrhage, which may have been either variceal or bronchial in origin. Long-term survival is a frequent occurrence in patients with cystic fibrosis who suffer variceal hemorrhage, and age at death is comparable to the general cystic fibrosis population. In conclusion, this suggests that liver transplantation is not indicated in these patients without additional features of liver decompensation.     

Burden of Surgically Correctable Disabilities Among Children in the Dadaab Refugee Camp

Background

Surgery is increasingly recognized as a means to reduce the morbidity and mortality of disabling impairments in resource-limited environments. We sought to estimate the burden of surgically correctable disabling impairments and the cost-effectiveness of their treatment among children in a large refugee camp.

Methods

This is a chart review of all patients aged 0–18 years from Dadaab Refugee Camp (Kenya) treated at a facility primarily responsible for providing pediatric surgical care in the region. Total disability-adjusted life years (DALYs) averted were calculated using life expectancy tables and established or estimated disability weights. A sensitivity analysis was performed using various life expectancy tables. Delayed averted DALYs caused by delay in care were also estimated. Inpatient costs were collected to perform a cost-effectiveness analysis.

Results

Between 2005 and 2011 a total of 640 procedures were performed on 341 patients. The median age at surgery was 4.6 years, and 33 % of the children treated were female. Only 13.5 % of surgeries estimated as required for common congenital surgical conditions were actually performed. The total number of DALYs averted ranged from 4,136 to 9,529 (6.4–14.8 per patient), depending on the calculation method used. Cost-effectiveness analysis resulted in values of $40–$88 per DALY.

Conclusions

The burden of pediatric surgical disabling impairments in refugee camps is substantial. Surgical intervention to address this burden is both feasible and cost-effective. Such intervention can significantly decrease the burden of disability among children affected by armed conflicts.     

Quality of life in patients with cystic fibrosis and their parents: what is important besides disease severity?

BACKGROUND: Cystic fibrosis is the most common inherited disease with a fatal outcome in industrialised nations. With the improvement in life expectancy, supporting patients and their families in adapting to life with this chronic progressive disease has become increasingly important. The aim of the present study was to investigate the relationship between health related quality of life (HRQOL) in this population, severity of disease, and cognitive/behavioural factors such as subjective health perception and ways of coping. METHODS: A sample of 89 adolescent and adult patients with cystic fibrosis and 125 parents of younger patients with cystic fibrosis completed questionnaires on health related quality of life and on ways of coping with the illness. Parents were asked to fill out the questionnaires regarding their own quality of life and coping. Multiple regression analyses were performed to examine the relationship between different predictor variables and quality of life. RESULTS: After accounting for the impact of disease severity and hours of treatment per day, the subjective health perception of patients significantly explained variance in their quality of life. Ways of coping were also significantly correlated with HRQOL. In parents the most important factor in explaining variance of HRQOL seems to be the coping style, whereas disease severity of the child and subjective health perception did not show any influence. CONCLUSIONS: The findings support the important role of cognitive and behavioural factors in specific subjective health perception and ways of coping in the adaptation to this severe chronic disease, both in patients themselves and in parents. The results call for a careful assessment of issues of coping and professional support for families of patients with cystic fibrosis in the early course of disease.     

The burden of bladder cancer in men and women: analysis of the years of life lost

Study Type – Harms (individual cohort) Level of Evidence 2a What’s known on the subject? and What does the study add? Previously, it was appreciated that men lost about 12 years of expected remaining life if they died of bladder cancer. However, nothing was known about how exposure history, gender, age and stage at diagnosis, and other factors affected life expectancy of bladder cancer patients. The impact of these factors was now taken into consideration when determining years of potential life lost from bladder cancer death and the loss in median life expectancy at bladder cancer diagnosis. Compared with men, women lose more years of life and a greater fraction of their life expectancy to bladder cancer.

OBJECTIVE

? To estimate the average loss in life expectancy (LE) due to bladder cancer (BC) in men and women in the USA.

PATIENTS AND METHODS

? Cancer records for 51 528 patients diagnosed with BC during 1988–1997 were obtained from the Surveillance, Epidemiology, and End Results database. ? Potential follow‐up ranged from 10 to 20 years (median 14 years). Loss in median LE at BC diagnosis was computed as the difference between expected median survival and observed median survival. ? Expected survival was calculated using two methods: method 1 used age, sex, and race‐specific LE in the general population, method 2 used the hazard of death from non‐BC causes in patients with BC (to account for past exposures and treatment‐related toxicities not present in the general population).

RESULTS

? During the study period, BC death occurred in 17% of men and 23% of women and non‐BC death occurred in 53% of men and 47% of women. ? Using LE in the general population as the reference (method 1), loss in median LE at BC diagnosis was 3.9 years for men (33% of their potential remaining years of life) and 6.5 years for women (47% of their potential remaining years of life). ? Using non‐BC‐specific hazard as the reference (method 2), loss in median LE was 2.7 years for men (26% of their potential remaining years of life) and 4.1 years for women (36% of their potential remaining years of life).

CONCLUSION

? Compared with men, women loose more years of life and a greater fraction of their life expectancy to BC.     

Meconium ileus: a critical review of treatment and eventual prognosis

Forty-six patients with meconium ileus were treated from 1951 to 1969, of whom 23 (50%) patients died in the immediate postoperative period. Fourteen of the remaining are now alive and are 2 to 14 years of age. Long-term survival does not differ from other patients with cystic fibrosis. One-half of the patients survived to leave the hospital, one-half of the remainder succumbed to cystic fibrosis, leaving one-quarter of the total to live a reasonably normal life but under the constant regimen to which all children with cystic fibrosis must submit.     

The Prevalence of Osteoporosis, Osteopenia, and Fractures Among Adults with Cystic Fibrosis: A Systematic Literature Review with Meta-Analysis

Observational studies have indicated a high but heterogeneous prevalence of low bone mineral density for adult patients with cystic fibrosis. Fracture complications were also described. The objective of this study was to determine the prevalence of osteoporosis, osteopenia, and fractures among adult patients with cystic fibrosis. A systematic literature review was conducted using electronic databases. The keywords used were “cystic fibrosis [MeSH] AND bone density.” Original studies were eligible if they reported the prevalence of osteoporosis and/or osteopenia and/or fractures in adult patients with cystic fibrosis. A meta-analysis of pooled proportions was performed. Heterogeneity was tested with the Cochran Q statistic, and in the case of heterogeneity a random effect model was used. Of 117 studies, 12 were selected, i.e., that represented a total of 1055 patients. Mean age ranged from 18.5 to 32 years (median: 28.2 years). Mean body mass index ranged from 19.9 to 22.4 (median: 20.7); 53.8% were men. The pooled prevalence of osteoporosis in adults with cystic fibrosis was 23.5% (95% CI, 16.6–31.0). The pooled prevalence of osteopenia was 38% (95% CI, 28.2–48.3). The pooled prevalences of radiological vertebral fractures and nonvertebral fractures were 14% (95% CI, 7.8–21.7) and 19.7% (95% CI, 6.0–38.8), respectively. In conclusion, this systematic literature review with meta-analysis emphasized the high prevalence of osteopenia and osteoporosis in young adults with cystic fibrosis. The prevalence of fracture was also high.     

Listing for lung transplantation: life expectancy and transplant effect, stratified by type of end-stage lung disease, the Eurotransplant experience.

BACKGROUND: Increased referral for lung transplantation, persistent shortage of donor lungs, and moderate transplant outcome call not only for adequate listing criteria, but also for an optimal allocation scheme. We used global cohort survival after listing and survival benefit from transplantation to study the effect of a lung allocation scheme, primarily driven by waiting time, on the different types of end-stage lung disease. METHODS: We followed all adult patients consecutively listed for first, lung-only transplantation between 1990 and 1996 (n = 1,208) for at least 2 years, with an additional 2-year follow-up after transplantation (n = 744). We used the competing risk method, the Kaplan-Meier method, and a time-dependent non-proportional hazards model to analyze waiting-list outcome and global mortality after listing, post-transplant survival, and transplant effect, respectively. Each analysis was stratified for type of end-stage lung disease. RESULTS: At 2 years, 57% of the total cohort had received lung transplants, whereas 25% had died on the waiting list. The 2-year survival post-transplant was 55%. The global mortality of the cohort, since listing, amounted to 46% at 2 years. Compared with continued waiting, patients experienced benefit from transplantation by Day 100, which lasted until the end of the 2-year analysis period. We noticed the highest global mortality rates for patients with pulmonary fibrosis and pulmonary hypertension (54% and 52%); emphysema patients had the lowest (38%). Patients with pulmonary fibrosis and cystic fibrosis had much earlier benefit from transplantation, 55 and 90 days, respectively. Transplantation also benefited emphysema patients by Day 260. CONCLUSIONS: Lung transplantation conferred transplant benefit in a Western European cohort of adults, in particular for patients with pulmonary fibrosis and cystic fibrosis, but also for patients with emphysema. The global survival rate, reflecting the real life expectancy for a newly listed transplant candidate, is poor for patients with pulmonary fibrosis and pulmonary hypertension. Allocation algorithms that lessen the impact of waiting time and take into account the type of end-stage lung disease should be developed.     

Quality of life in adults with cystic fibrosis.

BACKGROUND: Cystic fibrosis is an inherited condition with a high mortality and morbidity. The aims of this study were to assess quality of life in a population of adults with cystic fibrosis, to compare quality of life with published scores from a healthy population and other patient groups, and to examine the relation between quality of life and other measured clinical variables. METHODS: Patients over 16 years of age attending an adult cystic fibrosis outpatient clinic were surveyed at a time when they were clinically stable. A self-complete questionnaire was administered which comprised the Nottingham Health Profile (NHP) together with six additional questions related to cystic fibrosis. RESULTS: Completed questionnaires were obtained from 240 subjects (100 women) of median age 26 years (range 16-56). Mean (SD) forced expiratory volume in one second (FEV1) was 49 (26)% predicted, forced vital capacity (FVC) was 68 (26)% predicted, and the FEV1:FVC ratio was 59 (16)%. In this cross sectional study different patterns of perceived quality of life were seen in men and women. In part 1 of the NHP there was an age related trend compared with norms in men, with more distress/disability in the dimensions of emotion, sleep, and social isolation in the older age groups. In women there was no age related trend in the degree of distress/disability compared with norms. The mean score was different from norms in the dimensions of pain, emotion and sleep. For the patients with cystic fibrosis as a whole the scores in part 1 were comparable with published scores of patients with minor non-acute conditions. Scores in part 2 of the NHP for men were different from norms in six of the seven areas of daily living (all except home life). For women the scores were different from norms in the areas of looking after the home, social life, hobbies, and holidays. There were correlations between several of the quality of life dimensions and other measured variables such as FEV1, breathlessness score, and the time spent on home treatment. CONCLUSIONS: Men and women with cystic fibrosis have different patterns of perceived quality of life, and there is an age related trend of perceived quality of life in men in some dimensions. Quality of life scores in this group, as assessed by the NHP, are similar to those reported in subjects with minor non-acute conditions.     

Clinical outcomes of a large cohort of individuals with the F508del/5T;TG12 CFTR genotype

BackgroundIn recent years, patients with cystic fibrosis (CF) conductance regulator (CFTR) variant poly(T) sequences have been increasingly reported with a wide spectrum of clinical severity. We describe the long-term clinical outcomes and progression to a CF diagnosis over time in a large Italian cohort of patients carrying the CFTR F508del/5T;TG12 genotype.MethodsA retrospective analysis of subjects from 10 CF centres in Italy with the F508del/5T;TG12 genotype was performed. Demographic, clinical, microbiological, and biochemical data, as well as information about the follow-ups and complications of the enroled patients, were collected.ResultsA total of 129 subjects (54 females; median age: 15.0 years, range: 0–58 years; 59 older than 18 years) were included. In terms of initial diagnoses, 30 were CF (23.3%), 41 were CFTR-related disorder (CFTR-RD) (31.7%), and 58 were CF transmembrane conductance regulator-related metabolic syndrome/cystic fibrosis screen positive, inconclusive diagnosis (CRMS/CFSPID) (45.0%). After a median follow-up of 6.7 years (range 0.2–25 years), 15 patients progressed to CF, bringing the total number of CF diagnoses to 45/129 (34.9%). Most of these patients had mild lung diseases with pancreatic sufficiency and a low prevalence of CF-related complications.ConclusionsAt the end of the study, 34.9% of subjects with the CFTR F508del/5T;TG12 genotype were diagnosed with CF. We suggest including patients with the F508del/5T;TG12 genotype in long-term follow-ups.     

Long-term survival and nutritional data in patients with cystic fibrosis treated in a Danish centre.

BACKGROUND: Adequate nutrition and optimal treatment of bronchopulmonary infections are both of critical importance in maintaining the health of patients with cystic fibrosis. The cystic fibrosis centre in Copenhagen has followed a regimen of very early and aggressive antimicrobial treatment, especially against Pseudomonas aeruginosa infection. An unrestricted diet of low fat and high protein without hyperalimentation was recommended before 1985 which was then changed to a high fat, high calorie intake. METHODS: The overall impact of the treatment regimen was evaluated by a cross sectional analysis of all 223 patients who attended the centre in 1989. Growth and nutritional parameters were combined with lung function parameters and with a retrospective analysis of chronic P aeruginosa infection and its duration. Survival curves for all 313 patients treated at the centre since 1949 were calculated. RESULTS: All the patients with cystic fibrosis had normal height, although the final height was achieved a little later than in healthy controls. Body weight was lower than normal in males above 15 and in females above 10 years of age. The body mass index (BMI), which was approximately 98% of normal in the younger patients, declined to 90% in adult men and to 83% in adult women with cystic fibrosis, and was strongly correlated with lung function parameters. In 1989 the median age of survival of all patients treated in the centre since 1949 was 30 years (32 years in males and 29 years in females). CONCLUSIONS: The overall treatment regimen in the cystic fibrosis centre in Copenhagen is associated with growth and survival rates that are at least equal to those in other cystic fibrosis centres in other countries.     

Factors influencing the acquisition and eradication of early Pseudomonas aeruginosa infection in cystic fibrosis

In recent years considerable improvements have been made in increasing the life expectancy of patients with cystic fibrosis. New highly effective modulator therapies targeting the underlying defect in the cystic fibrosis transmembrane conductance regulator protein are expected to enhance lifespan even further. However, chronic Pseudomonas aeruginosa pulmonary infections continue to threaten CF patient lung health and mortality rates. Early and aggressive antibiotic eradication therapies targeting P. aeruginosa are standard practice, but these eradication therapies fail in 10–40% of patients. The reasons for P. aeruginosa eradication failure remain unclear. Thus, this review summarizes the evidence to date for pseudomonal acquisition and eradication failure in the cystic fibrosis lung. A complex combination of host and bacterial factors are responsible for initial establishment of P. aeruginosa pulmonary infections. Moreover, host and pseudomonal factors, polymicrobial interactions, and antimicrobial limitations in relation to P. aeruginosa eradication therapy failure are summarized.     

Improved results after heart-lung transplantation: a 17-year experience

OBJECTIVE: In selected patients with severe end-stage combined cardiopulmonary diseases, heart-lung transplantation (HLTx) remains the only therapeutical option for improving survival and quality of life. PATIENTS AND METHODS: Since 1983, 51 HLTx were done at our institution. Mean patient age was 27+/-12 years with a mean donor age of 25+/-11 years. Indications for HLTx were primary pulmonary hypertension (PPH) in 49% of patients, congenital heart disease in 39%, cystic fibrosis in 6%, and repeat-HLTx in 6%. Eleven patients were younger than 14 years. Among these pediatric patients, the indications were PPH in 55% of patients, pulmonary atresia with severe pulmonary artery hypoplasia in 27%, and cystic fibrosis and cardiomyopathy with fixed pulmonary hypertension in 9% of patients each. Two patients had additional liver transplantation because of chronic aggressive virus hepatitis. For organ preservation, Euro-Collins solution (lung perfusion) and cardioplegic solution according to Bretschneider (heart perfusion) were used until 1994. The University of Wisconsin solution replaced Bretschneider's solution in 1994. Since 1996, Perfadex, a low-potassium dextran-based preservation solution, replaced Euro-Collins. All transplantations were done through a median sternotomy until 1994. Thereafter, a transverse thoracotomy was used in patients with suspected adhesions. Until 1995, cyclosporine A, azathioprine, and prednisolone were used for immunosuppression. Since then, tacrolimus replaced cyclosporine A. RESULTS: From 1983 until 1993, perioperative mortality was 35% (6/19). From 1994 on perioperative mortality decreased to 12.5% (4/32). Early mortality was caused by graft failure (n=5), severe bleeding (n=2), multi-organ failure (n=2), and acute rejection (n=1). Cumulative survival rates were 81% after 30 days, 63% after 1 year, and 54% after 5 years, respectively. Since 1994, cumulative survival rates were markedly improved to 87% after 30 days, 81% after 6 months, and 78% after 1 year. There was no death during the first postoperative year among the 11 pediatric patients. Late death was mainly caused by obliterative bronchiolitis (OB; 76%); two patients died because of multi-organ failure or septic complications, respectively, and one patient died within the first postoperative year because of aspergillosis. CONCLUSION: Changes in organ preservation management, surgical techniques, and immunosuppressive therapy significantly improved the short- and mid-term results after HLTx. Long-term results can only be improved in cases of successful prevention and treatment of OB.     

The Overlapping Risk Profile Between Dialysis Patients Listed and Not Listed for Renal Transplantation

The survival advantage of kidney transplantation extends to many high-risk ESRD patients; however, numerous factors ultimately determine which patients are evaluated and listed for the procedure. Broad goals of patient evaluation comprise identifying patients who will benefit from transplantation and excluding patients who might be placed at risk. There is limited data detailing whether current access limitations and screening strategies have achieved the goal of listing the most appropriate patients. The study estimated the life expectancy of adult patients in the United States prior to transplantation with ESRD onset from 1995 to 2003. Factors associated with transplant listing were examined based on patient prognosis after ESRD. Approximately one-third of patients listed for transplantation within 1 year of ESRD had ≤5-year life expectancy on dialysis. In contrast, one-third of patients not listed had >5-year life expectancy. The number of patients not listed with 'good' prognosis was significantly higher than those listed with 'poor' prognosis (134 382 vs. 16 807, respectively). Age, race, gender, insurance coverage and body mass index (BMI) were associated with likelihood for listing with 'poor' prognosis and not listing with 'good' prognosis. Over the past decade, many ESRD patients viable for transplantation have not listed for transplantation while higher-risk patients have listed rapidly.