A comparison of quality of life of patients on automated and continuous ambulatory peritoneal dialysis.

OBJECTIVE: Data on health-related quality of life (HRQOL) of automated peritoneal dialysis (APD) patients are scarce. The objectives of this study were (1) to explore HRQOL of APD patients and compare it with HRQOL of continuous ambulatory peritoneal dialysis (CAPD) patients and a general population sample, and (2) to study the relationship between HROOL assessment outcomes and background variables. DESIGN: Home interviews of APD and CAPD patients. HRQOL, social-demographic, clinical, and treatment-related background data were collected at the interview and from patient charts. Multiple regression analysis and logistic regression analysis were used to study the relationship of HRQOL assessment outcomes with background variables. SETTING: Sixteen Dutch dialysis centers. PATIENTS: Convenience sample of 37 APD patients and 59 CAPD patients matched for total time on dialysis. MAIN OUTCOME MEASURES: Four HRQOL instruments: Short-Form 36, EuroQol EQ-5D, Standard Gamble, and Time Trade Off. RESULTS: Physical functioning of both APD and CAPD patients was impaired compared with the general population; mental functioning was not different. In multivariate analyses, the mental health of APD patients was found to be better than that of CAPD patients. In addition, APD patients were less anxious and depressed than CAPD patients. With respect to physical aspects of HRQOL and role-functioning, no differences were observed between APD and CAPD patients. Other variables to explain HRQOL assessment outcomes were age, the number of comorbid diseases, and primary kidney disease. CONCLUSIONS: HRQOL of APD patients is at least equal to HRQOL of CAPD patients.

The effect of medical care by a helicopter trauma team on the probability of survival and the quality of life of hospitalised victims.

In 1995, an experiment was started to give extra medical help by helicopter to patients who needed emergency treatment. The aim of the experiment was not to reduce the transportation time to the hospital, but to bring specialised medical care directly to patients as soon as possible. An evaluation study was carried out to assess the effect of the treatment given by the Helicopter Trauma Team (HTT) on survival and quality of life. The study focused on hospitalised patients suffering from polytrauma. A direct comparison between an experimental and control group was not possible, because the HTT group consisted of more severely injured patients. A refined severity index was constructed on the basis of the Revised Trauma Scale (RTS) and the Injury Severity Scale (ISS) and their sub-scores. Using this index, it was possible to make a clear distinction between three groups of patients, i.e. those with a high probability of survival (with or without special medical treatment), those with a very low probability of survival and the patients in between. It was shown that the HTT-treatment was effective. The survival rate increased for patients in the 'in between' group, but not for patients with a low probability of survival. There was no difference in the quality of life of patients from the HTT and non-HTT groups 15 months after the accident. These findings refute the hypothesis that only the most severely injured patients with a low quality of life profit from HTT-treatment.

Sensitivity and perspective in the valuation of health status: whose values count?

The literature was studied on the existence of differences in valuation for hypothetical and actual health states between patients and other-rater groups. It was found that nine different study designs have been used to study this question and two of these designs were applied in a study involving dialysis patients and other rater groups. In the first study, both dialysis patients and students had to value hypothetical health states with Standard Gamble (SG) and Time Trade Off (TTO). Patients assigned higher values to hypothetical health states than students did. In the second study, dialysis patients who were being treated with four different dialysis modalities were asked to value their own health state with SG, TTO and a visual analogue scale (EQ(VAS)), and to describe their health state on the EQ-5D(profile). Several EQ-5D(index) values (health index values derived from general population samples) were calculated for the four dialysis treatment groups, based on the EQ-5D(profile). These health indexes could discriminate between treatment groups, according to clinical impressions. Treatment groups could not be differentiated based on patients' valuations of own health state. The results suggest that general population samples, using EQ-5D(index) values, may be more able to discriminate between patient groups than the patients themselves are. The implications of this finding for valuation research and policy-making are discussed.

Effect of simultaneous pancreas-kidney transplantation on mortality of patients with type-1 diabetes mellitus and end-stage renal failure.

BACKGROUND: Long-term prognosis of patients with type-1 diabetes mellitus and end-stage renal failure appears to be better after kidney transplantation compared with dialysis. Controversy exists about the additional benefit of a simultaneously transplanted pancreatic graft. We studied the effect on mortality of simultaneous pancreas-kidney transplantation compared with kidney transplantation alone from regional differences in transplantation protocols. METHODS: All 415 patients with type-1 diabetes (aged 18-52 years) who started renal-replacement therapy in the Netherlands between 1985 and 1996 were included in the analysis. Patients were allocated to a centre based on their place of residence at onset of renal failure. In the Leiden area, the primary intention to treat was with a simultaneous pancreas-kidney transplantation, whereas in the non-Leiden area, kidney transplantation alone was the predominant type of treatment. All patients were followed up to July, 1997. Analyses, mortality, and graft failure were by Cox proportional-hazard model adjusted for age and sex. FINDINGS: Simultaneous pancreas-kidney transplantation was done in 41 (73%) of 56 transplanted patients in the Leiden area compared with 59 (37%) of 158 transplanted patients in the non-Leiden area (p<0.001). The hazard ratio for mortality after the start of renal-replacement therapy was 0.53 (95% CI, 0.36-0.77, p<0.001) in the Leiden area compared with the non-Leiden area. When just the transplanted patients were analysed the mortality ratio was 0.4 (95% CI 0.20-0.77, p=0.008) and was independent of duration of dialysis and early transplant-related deaths. Equal survival was found for patients on dialysis only. INTERPRETATION: These data support the hypothesis that simultaneous pancreas-kidney transplantation prolongs survival in patients with diabetes and end-stage renal failure.

Economic evaluation of end stage renal disease treatment.

This paper examines the cost-effectiveness of end stage renal disease (ESRD) treatments. Empirical data on costs of treatment modalities and quality of life of patients were gathered alongside a clinical trial and combined with data on patient and technique survival from the Dutch Renal Replacement Registry. A Markov-chain model, based on the actual Dutch ESRD program as of January 1st 1997, predicted the cost-effectiveness and cost-utility of dialysis and transplantation over the 5-year period 1997-2001. Total annual costs amounted to DFL 650 million (1.1% of the health care budget). Centre Haemodialysis was found to be the least cost-effective treatment, while transplantation and Continuous Ambulatory Peritoneal Dialysis (CAPD) were the most cost-effective treatments. The Markov-chain model was used to study the influence of substitutive policies on the overall cost-effectiveness of the ESRD treatment program. The influence of such policies was found to be modest in the Dutch context, where a high percentage of patients is already being treated with more cost-effective treatment modalities. In countries where Centre Haemodialysis is still the only or the major treatment option for ESRD patients, substitutive policies might have a more substantial impact on cost-effectiveness of ESRD treatment.

Quality of life in short adults.

The use of (costly) growth hormone (GH) treatment in short children is often justified by the assumption that short stature considerably reduces quality of life in adults. We tested this assumption in 5 groups of short adults: 25 patients with isolated GH deficiency; 17 male patients with childhood onset renal failure; 25 women with Turner syndrome and 26 patients who were presented as a child to a paediatrician for idiopathic short stature. A group of 44 short individuals with presumably idiopathic short stature, who had not been presented to a paediatrician for short stature, was sampled from the general population ('normal shorts'). We measured quality of life in terms of socio-economic variables, the Nottingham Health Profile and time trade-off. The mean height of most groups was close to the 3rd percentile. The chance of having a partner was low for all groups, except for the normal shorts. Problems with job application were only reported in Turner syndrome. The scores on the Nottingham Health Profile were all within the normal range, but GH-deficient adults had a higher score on the domain energy than normal shorts. Women with Turner syndrome, individuals with renal failure, and those with idiopathic short stature had a wish to be taller, with an estimated reduction in quality of life of 2-4% (time trade-off). As the normal shorts did not show any sign of a reduced quality of life, we falsify the assumption of a direct relation between short stature and quality of life. The complaints of patients with idiopathic short stature around the 3rd percentile seem to be the result of unsuccessful coping strategies.

Measuring the quality of life before and after bilateral lung transplantation in patients with cystic fibrosis.

Lung transplantation is an important topic today in healthcare policy because the technique is new and costly. One of the important issues in the evaluation of lung transplantation is quality of life. The quality of life after lung transplantation must be relatively high compared with other forms of medical care to legitimize the high costs of transplantation. Quantifying the quality of life after lung transplantation and other medical therapies is possible with general measurements of quality of life. In a pilot study of six patients with cystic fibrosis, the quality of life, both before and after lung transplantation, was measured by the following five instruments: (1) standard gamble, (2) time trade-off, (3) the Karnofsky performance status, (4) the EuroQol visual analog scale, and (5) the Nottingham health profile. This pilot study demonstrates that the introduced methodology is feasible. The preliminary results suggest that the improvement in quality of life for patients with cystic fibrosis after bilateral lung transplantation is comparable to the improvement in quality of life after heart transplantation.

Systems of donor transfer.

The development of medical knowledge has resulted in a demand in society for donor organs, but the recruitment of donor organs for transplantation is difficult. This paper aims to provide some general insights into the complex interaction processes involved. A laissez-faire policy, in which market forces are relied on, is not acceptable from an ethical and legal point of view in most western European countries. Especially at the demand side of the exchange of donor organs, commercialism is to be opposed. We judge the use of commercial incentives at the supply side less unacceptable in theory but not feasible in western European countries. Since market forces are deemed unacceptable as instruments for coordinating demand and supply of donor organs, donor procurement has to be considered as a collective good, and therefore governments are faced with the responsibility of making sure that alternative interaction and distribution mechanisms function. The role of organ procurement agencies (OPAs) in societal interaction concerning postmortem organ donation is described using a two-dimensional conceptualisation scheme. Medical aspects of living organ donation are described. An international comparative description of legal systems to regulate living organ donation in western European countries completes this survey.

The utility of health at different stages in life: a quantitative approach.

Thirty students and thirty-five elderly people compared the quality of life of imaginary patients of different ages suffering from end-stage renal disease. By manipulating the time the imaginary patients had to be on a transplantation waiting list, the utility of health at different periods of life could be compared. Except for the very young, respondents found health in the early periods of life to be twice as important as in the last decade of life. Health at age 35 had an utility somewhere between these two extremes. The responses of the elderly people showed remarkable resemblance to the students' responses, suggesting that the results reflect a general ethical standard. The values found were tested by means of a factorial design and found to fulfill the qualifications of an interval scale.

Orthotopic liver transplantation in The Netherlands. The results and impact of a medical technology assessment.

In 1985 Dutch health care authorities and health insurance companies initiated a large-scale technology assessment (TA) of liver transplantation (LTx) in The Netherlands. The 10-year experience of the existing programme in the University Hospital Groningen was investigated. Topics included were patient flow, selection policies, survival, quality-of-life, costs, need, supply of donor organs and organisational aspects. Estimation of the consequences of a non-transplantation scenario allowed for the execution of a cost-effectiveness analysis. Results showed clear improvement by LTx of survival and quality-of-life, though to a lesser degree than expected. Costs of the first transplantation year amounted to Dfl 180,000 (approx US $90,000). The cost-effectiveness ratio ranged from Dfl 47,000 to Dfl 133,000 per life year gained. No overt imbalance between need and donor supply existed or was expected in the near future. The impact of this study is related to the informational value and to the contribution to the decision-process. Even at its appearance in 1988, the final report provided health policy makers with new information. Health policy concerning LTx was considerably influenced, as a rule in agreement with the study conclusions. We conclude the Dutch case study to be an example of a useful and efficient TA.

[Technology assessment of liver transplantation; a study of the liver transplantation program in Groningen 1977-1987]. / Een 'technology assessment' van levertransplantatie; een onderzoek naar het Groningse levertransplantatieprogramma van 1977-1987.

The liver transplantation programme of the University Hospital of Groningen, the Netherlands, was evaluated on behalf of the Dutch Sick Fund Council. From 1978 to 1987 561 patients were put forward for liver transplantation (LTX). During this period, 76 orthotopic liver transplants were carried out, 8 of which were retransplantations. Survival proved to depend on, among other things, diagnosis and age. One-year survival was 100% in children with biliary atresia and 60% in other diagnosis and age groups. The number of life-years gained by LTX depends on the stage of disease. After LTX the quality of life improves quickly. One year after LTX most survivors experience a virtually normal quality of life. The need of LTX in the Netherlands was estimated to be in the range of 25 to 69 transplantations on an annual basis. The annual supply of donor livers is expected to be about adequate. Costs amount to approx. Hfl 250,000.--per transplanted patient including costs of follow-up for up to five years. The cost-effectiveness ratio for all forms of cirrhosis was estimated at Hfl 47,000.--to 133,000.--per life year gained. The results of this first technology assessment on liver transplantation proved relevant for clinicians as well as health politicians.