European cystic fibrosis society consensus on standards a roadmap to bbest careq

Transcription

1 Journal of Cystic Fibrosis 4 (2005) 1 5 European cystic fibrosis society consensus on standards a roadmap to bbest careq 1. Remarkable improvement in prognosis There has been a steady and impressive improvement in the outlook for people with cystic fibrosis (CF) since the first clear description of the condition in 1938 [1]. Progress has been particularly impressive over the last 20 years [2 4]. There are still many who remember the time when CF was a short-lived tragic disease of early childhood treated, almost reluctantly, by general paediatricians who knew the inevitable outcome a short-lived miserable existence for the infant, increasingly distressing to the family. But nowadays the situation is quite different and the spectrum of the condition extends from the detection of the carrier state in a healthy individual, to management of an adult with CF in the years following a bilateral lung transplant. Virtually every system in the body is involved at some stage of life and optimal treatment is wide ranging and complex and requires the expertise of a large number of health care professionals. 2. General medical advances and more effective treatment at CF centers Why has the outlook for people with CF become so much better? Of course, CF is not the only condition to have a radically improved outlook e.g. acute lymphoblastic leukaemia is often compared a previously fatal disease now more often than not curable. These and similar conditions all have in common a vigorous approach to treatment by specialised teams, who spend much of their time treating the disease in question. In doing so they gain increasing experience in the practical management of the disease and its complications. The availability of new and better drugs, more efficient methods of delivery and new complications resulting from extended survival, have all increased the complexity and knowledge required to deliver the best available treatment for people with cystic fibrosis. So although, in part, due to general medical and scientific advances, most would agree that the improving prognosis has been largely due to the development, introduction and evaluation of more effective treatment regimens for the respiratory, nutritional and other problems, by teams of professionals working at large specialist CF centers [5 10]. 3. A new positive attitude to treatment and higher expectations Another important factor has been the more positive attitude on the part of those responsible for treating people with CF and their reluctance to accept that decline and premature death were inevitable [7]. The previous hopeless attitude was by no means unusual and even in 1987 Prof. Tim David complained that ba passive fatalistic approach to treatment is a major obstacle to progress and the attitude that CF is a hopeless incurable disease is all too prevalentq [11]. Also the expectations of the patients and families have increased as the benefits of more aggressive treatment became apparent. 4. The concept of the bpoint of no returnq In most people with CF the presence and severity of chronic pulmonary infection is the major determinant of their health and survival [12,13]. Observing the progress of many people with CF over many years reinforces the concept of there being a bpoint of no returnq [14], which coincides with the onset of chronic pulmonary infection. The distinction between bcf diseaseq (physicochemical abnormality within uninfected airways due to absence or dysfunction of CFTR) and blung diseaseq (the situation after the onset of chronic infection and excessive inflammation) is a useful concept. Once dlung diseaset is established, the infection cannot be eradicated and there is a gradual but relentless deterioration of respiratory /$ - see front matter D 2005 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved. doi: /j.jcf

2 2 function, the speed of which is determined mainly by the intensity of treatment [15]. 5. Close monitoring and effective treatment must follow early diagnosis It has become apparent that the progressive deterioration of respiratory function described in the older CF literature is not inevitable. If respiratory infection is identified early and eradicated by appropriate antibiotic treatment, chronic infection can be prevented or certainly delayed for many years [15 18]. Also the early malnutrition and later growth problems can be avoided when enzyme and nutritional intervention is started soon after birth [19]. To achieve this for all infants with CF, neonatal screening followed by early diagnosis, close monitoring and effective treatment is mandatory. The CF multidisciplinary teams have a responsibility to undertake clinical audit and research in a condition, such as CF, where treatment is continually improving, changing and becoming increasingly more effective. In practice these are more easily accomplished at CF centers where many patients are treated useful CF clinical research from small clinics at local hospitals is virtually non-existent due to lack of patients, time and expertise. At the CF center general policy and the management of individual patients are discussed at weekly team meetings and ward rounds. Experience gained from treating many people with CF is shared first between team members and colleagues in related specialities and subsequently between colleagues in other centers and at international meetings. The results must be assessed in the long term for although pulmonary damage, sustained before the diagnosis is made or as a result of suboptimal treatment after diagnosis, can be contained with modern antibiotic treatment for a few years, it will eventually result in a more rapid decline in pulmonary function during adolescence or early adult life. Unfortunately, the results of delayed diagnosis and suboptimal treatment in the paediatric clinic may be more easily identified by the CF physician and his team at the adult clinic and may not always be apparent to the paediatric team. This is a good reason for the paediatric and adult CF staff to share their experience and even their staffs to some extent as occurs in some centers [20]. 6. A communication and information revolution It is essential that all who are responsible for CF care are familiar with the well-established and latest advances in treatment; but even in this age of information technology this is not always easy. The past 20 years has seen an explosive increase in the available information relating to cystic fibrosis. Medline citations increased from almost 5000 in the decade between 1986 and 1995 to nearly 10,000 between 1996 and Also between 1966 and 1997 there was a 30-fold increase in clinical trials relating to CF of which over a third were only reported at meetings [21]. So the previous legitimate complaint that there were too few clinical trials in CF and that much of the treatment is based largely on anecdotal experience is no longer true. The more stringent review of the published work on CF by the Cochrane Cystic Fibrosis and Genetics Disorders Group has been a welcome advance but cannot cover all aspects of CF care particularly relating to some of the practical but vitally important details of everyday management [22]. An overwhelming amount of scientific and quasi-scientific material and advice, much of dubious quality, is generally available on the Internet [23]. 7. Delay in implementing new information Unfortunately, despite all this information there is still a considerable delay in important advances influencing the treatment of some patients. It has been suggested that there may be too many sources of evidence, compiled with a variable mix of scientific rigour and opinion, resulting in confusing messages [24]. Even when evidence is deemed to support an intervention, current health policy may prevent clinicians from providing it. It is certainly true that an increasingly important factor in the delivery of optimal care is the ability of the providers to fund the increasingly expensive drugs and this varies between CF centers and countries. So although the main principles of treatment are readily available to those treating people with CF, it appears to be the fine details of management, which may differ between centers that account for some of the differences in the condition of their patients. 8. Marked differences in condition of people with CF at different centers Despite the wealth of information on the treatment of CF it is very obvious both from clinical experience and the data from CF Registries, that there were in the past, and continue to be, striking differences in the condition of people attending different CF Centers [25 27]. These differences are far more marked than can be accounted for by climate, socio-economic circumstances, age of diagnosis or variation in prevalent gene mutations. The unpublished study of Wood and Piazza [28] reflected very marked differences in care and outcomes throughout the seventies at three recognised US CF Centers where median survivals ranged from 9.5, 18.1 to 22.8 years. Analysis showed differences reflecting the closeness of supervision (number of clinic visits) and intensity of treatment (days of intravenous antibiotics) the patients received. The superiority of CF center care over care in general paediatric clinics was reflected in significantly better

3 3 survival in Victoria, Australia where CF center care was already well-established, than in England and Wales [9]. More recently, surprisingly large differences were observed between centers ranked within the upper and lower quartile values for respiratory function and nutritional status. bthe results suggested that the centers reporting higher values for FEV 1 and weight for age monitored patients more frequently, reviewed spirometry more often and treated patients more aggressivelyq [29]. It has been observed that the 10% difference in predicted FEV 1 %, seen between centers, is of the same order as achieved by new treatments, which are considered to show a significant benefit for patients. It is important that busy CF teams, although at times reluctant to submit data, appreciate that patient registries are absolutely essential to identify these significant differences in clinical state and outcomes. 9. Some treatment differences are ba matter of degree rather than kindq What are the differences in management that seem to be so important? Unfortunately many of the bfine detailsq of management have not been subjected to clinical trials. In 1958, a young paediatrician in Cleveland, Dr LeRoy Matthews, was charged with planning and initiating a bcomprehensive and prophylactic (preventive) treatment programmeq for the treatment of cystic fibrosis [30]. The programme subsequently incorporated many of the components now regarded as essential for modern CF center care e.g. early correct diagnosis, a comprehensive programme to deal with all the aspects of the disease and data collection to validate the impact of the treatment on morbidity and survival [5,6,31]. In 1960, Dr Terrence Gillespie, the CF Center Director from Halifax, Nova Scotia reflected on why the results were so much better in LeRoy Mathews s CF center in Cleveland than in Halifax, Nova Scotia as both clinics prescribed similar treatment. After he had worked in both places he concluded, bthe fundamental difference (in the treatment) was a matter of degree rather than kind. Dr Matthews had developed the concept of starting full treatment on the day of diagnosis on every patient, regardless of the clinical conditionq [32]. This seems to be the most likely explanation for these persisting differences, which still remain a major cause for continuing concern. 10. An bepidemicq of standards, protocols and guidelines In an attempt to ensure that patients are receiving the most appropriate and acceptable treatment, various national committees and working parties and many CF centers have developed their own standards for general care and also more detailed guidelines for various aspects of treatment [33 38]. Guidelines are defined as bsystematically developed statements to assist practitioner and patient decisions about appropriate health care for specific clinical circumstancesq [39]. Guidelines should be developed and endorsed by the relevant professional bodies, be based on good evidence of effectiveness, practicable and affordable, multidisciplinary where appropriate and always taking account of patients choices and wishes. Both guidelines, and the standards it is intended they should achieve, are usually produced by consensus groups of professionals, widely experienced in dealing with various aspects of cystic fibrosis. They provide guidance for those with less experience and also ensure the team members give consistent advice to patients and carers. Unfortunately the literature on guidelines in general can be overpowering to the clinician who sees them as a tool for making care more consistent and efficient and for closing the gap between what clinicians do and what scientific evidence supports [40]. The importance of the correct development of guidelines has been stressed and there is now extensive published work on the many aspects of guidelines development summarised clearly in the Guidelines Finder section of the National Electronic Library for Health ( The inclusion of parents and patients as user-representatives on consensus groups is welcome and now routine in many countries. Also the standards and guidelines should be available to all patients and parents to reassure them they are receiving the most appropriate treatment and to reduce the need for their consulting less reliable sources. However, not all are impressed by the recent epidemic of advice observing bwhat passes today for the standards of clinical care thousands of practice guidelines, often conflicting, sometimes disreputable, always a mixture of opinions and biases and, sometimes, evidence is a messq [41]. It is has been suggested that it would be preferable to start with what we know for certain, rather than producing an indigestible survey of every article available and that the bpurveyors of systematic reviews can begin to reverse this trend by promoting practice recommendations instead: a frequently updated compendium of all (and only) those clinical practices whose evidence of benefit is undisputed with the result that many health care professionals, goaded by unimpeachable evidence, will try harder to translate it into practiceq [41]. Such advice has much to commend it. 11. ECFS consensus on standards of care for people with cystic fibrosis In March 2004 Prof. Gerd Doering arranged a 2-day European CF Society consensus meeting on Standards of Care for People with Cystic Fibrosis. The final consensus document is published in the present issue [42]. The 34 members of the consensus group were from most European

4 4 countries and two from N. America; they represented the many disciplines involved in CF care. The group met for 2 days in Artimino to agree standards of care and also to answer some commonly asked and highly relevant questions. Some indication of the strength of evidence was given to the answers. The facilities and functions of a CF center were discussed also the staff required. The routines of CF care were agreed including outpatients, inpatients, annual reviews, details of various aspects of care, including the newly diagnosed patient, the patient diagnosed in adult life and atypical patients. The importance and role of other specialists in CF care were detailed. The document finishes with the 35 practical questions the answers to which were agreed by the consensus group (some after considerable, at times heated, discussion!). The ECFS consensus group has achieved their aim of defining standards for the routine evaluation, monitoring and treatment of people with CF in Europe which, if adopted by all centers, will provide a quality assurance instrument and a basis for audit of CF care. 12. Major efforts towards bbest careq for all It is general experience that the anticipated benefits of guidelines are rarely fully realised in an everyday setting and this is reflected in the variations in the outcome of the patients. The variability in outlook is marked and the median age of death in the US CF centers varies from 28.2 years in the bbestq centers to 23.2 years for the rest [43]. Future research, using the CFF encounter-based database, Port CF, will concentrate on improving care in three areas growth and nutrition, pulmonary maintenance and exacerbations and CF related diabetes [44]. There were a considerable number of studies reported at the 2004 North American CF Conference aimed at improving the nutritional status [45] and other aspects of care of patients in the poorly performing centers [46 48]. Schechter describes several key strategies for improving care including appreciating the system must be changed, working with an appropriate model of a system of clinical care, making small sequential changes then spreading the effective ones, using data to obtain feedback on effectiveness and collaboration and stealing good ideas from others [49]. Delivering suboptimal care to people with CF will undoubtedly reduce their life span, in fact the CF Foundation believes that improving the care of all people with CF to that of the best centers would increase the life expectancy by 7 years. 13. Conclusions The outlook for people with CF has improved remarkably due to more effective treatment developed at CF centers. But the treatment regimens are increasingly complex and the results achieved vary significantly between centers as revealed by CF registries. It is unacceptable that some people with CF are still receiving suboptimal treatment, which will compromise their quality of life and survival. This is the present unsatisfactory situation despite the availability of numerous guidelines and a wealth of other information. However, it is encouraging and timely that the eventual recognition and acceptance of the marked variability in care, condition and outlook of people with CF has now provided a stimulus to investigate the reasons and has initiated an attempt to bring the standards of the bbestq to all centers. The latest ECFS consensus document sets out the standards to which all centers should be aiming and which it is hoped all European centers will adopt. 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Littlewood Chairman, UK Cystic Fibrosis Trust, Bromley, UK Honorary Paediatrician, St James University Hospital, Leeds, UK address: jimlittlewood@btopenworld.com. Tel./fax: