TMD pain: the effect on health related quality of life and the influence of pain duration.

OBJECTIVES: As impact of literature concerning this subject is scarce, the objectives of this study were to assess whether the Health Related Quality of Life (HRQoL) is decreased in patients with painful temporomandibular disorders as compared to the HRQoL in the general population, and to evaluate to what extent pain duration affects HRQoL. METHODS: Data concerning physical and mental health were retrieved from patients with painful temporomandibular disorders. Assessment tools used were: the Mandibular Function Impairment Questionnaire (MFIQ), the Short-Form-36 (SF-36), the Hospital Anxiety and Depression Schedule (HADS), and the General Health Questionnaire (GHQ). In order to examine the influence of the duration of pain on HRQoL, the total sample was divided into three different subgroups. Subgroup 1 consisted of patients with complaints existing less than one year. Patients with complaints from 1 to 3 years were allocated to the second group. The 3rd subgroup included patients with complaints longer than 3 years. RESULTS: The total sample consisted of 95 patients (90 females and 5 males). On most physical and social functioning items, groups 2 and 3 scored significantly worse than the general population. On the other hand, none of the groups differed from the general population when comparing the mental items. Duration of pain was significantly correlated with SF-36 subscale physical functioning and the mandibular impairment. CONCLUSION: Patients with TMD pain less than one year score better than compared to the population norm. With a longer duration of pain, mental health scores and role limitations due to emotional problems do not appear to be seriously affected by reduced physical health, while social functioning appears to be considerably affected.

Cost of liver transplantation: a systematic review and meta-analysis comparing the United States with other OECD countries.

Large cost variations of liver transplantation are reported. The aim of this study was to assess cost differences of liver transplantation and clinical follow-up between the United States and other Organization for Economic Cooperation and Development (OECD) countries. Eight electronic databases were searched, and 2,000 citations published after 1990 with more than 10 transplantations, and with original cost data, were identified. A total of 30 articles included 5,975 liver transplantations. Meta-analysis was used to derive a combined mean using a random-effects model to test for heterogeneity between studies. Estimated mean cost of a U.S. liver transplantation was US$163,438 (US$145,277-181,598) compared to US$103,548 (US$85,514-121,582) for other OECD countries. Patient characteristics, disease characteristics, quality of the health care provider, and methodology could not explain this cost difference. Health system characteristics differed between the U.S. and other OECD countries. Cost differences in liver transplantation between these two groups may be largely explained by health system characteristics.

Cost-effectiveness of a psychoeducational relapse prevention program for depression in primary care.

BACKGROUND: Major depression is a prevalent mental disorder with a high risk of relapses and recurrences, which are associated with considerable burden for patients and high costs for society. Despite these negative consequences, only few studies have focused on interventions aimed at the prevention of recurrences in primary care patients with depression. AIMS OF THE STUDY: To assess the cost-effectiveness of a psychoeducational prevention program (PEP) aimed at improving the long-term outcome of depression in primary care. METHODS: Recruitment took place in the northern part of the Netherlands, patients were referred by general practitioners. In total 267 patients were included in the study and randomly assigned to usual care (UC) or UC with one of three forms of PEP; PEP alone, psychiatric consultation followed by PEP (psychiatrist-enhanced PEP), and cognitive behavioral therapy followed by PEP (CBT-enhanced PEP). Costs and health outcomes were registered at three month intervals during the 36 months follow-up of the study. Primary outcome measure was the proportion of depression-free time. RESULTS: Mean total costs during the 36 months of the study were 8200 euros in the UC group, 9816 euros in the PEP group, 9844 euros in the psychiatrist-enhanced PEP group, and 9254 euros in the CBT-enhanced PEP group. Costs of productivity losses, hospital admissions, contacts with regional institutions for mental healthcare, and medication use contributed substantially to the total costs in each group. Results of the primary outcome measure were less positive for PEP than for UC, but slightly better in the enhanced PEP groups. If decision-makers are willing to pay up to 300 euros for an additional proportion of depression-free time, UC is most likely to be the optimal intervention. For higher willingness to pay, CBT-enhanced PEP seems most efficient. DISCUSSION: The basic PEP intervention was not cost-effective in comparison with UC. The economic impact of productivity losses associated with depression, and the importance of including these costs in economic studies, was illustrated by the findings of this study. Due to the drop-out of patients during the 36 months follow-up period, economic analyses had to account for missing data, which may complicate the interpretation of the results. Although Quality-Adjusted Life Years (QALYs) could not be assessed for all the patients, the results of analyses focusing on QALYs supported the overall conclusion that PEP is not cost-effective. IMPLICATIONS FOR HEALTH CARE PROVISION AND POLICIES: Results indicated that PEP should not be implemented in the Dutch healthcare system. Furthermore, is seems highly unlikely that PEP could be cost-effective in other (comparable) European healthcare systems. IMPLICATIONS FOR FURTHER RESEARCH: The relatively positive economic results for CBT-enhanced PEP imply that UC enriched with CBT (but without PEP) might be cost-effective in preventing relapses in primary care patients with depression. The actual consequences of CBT for relapse prevention will have to be studied in further detail, both from a clinical and economic point of view.

Pre-transplant quality of life does not predict survival after lung transplantation.

BACKGROUND: Currently, the goal of lung transplantation is not only to improve survival but also includes improvement of health-related quality of life (HRQL). Limited knowledge is available about the value of HRQL before lung transplantation with regard to predicting survival after lung transplantation. To maximize the benefits of transplantation, it is essential to gain knowledge about variables that predict both length and quality of survival. In this study we sought to determine whether HRQL before transplantation predicts survival after lung transplantation. METHODS: For each of the 200 lung transplant recipients included in this study, the HRQL questionnaire completed at the date closest to the transplant date was selected. Measures included were: the Nottingham Health Profile (NHP); State-Trait Anxiety Inventory (STAI); Self-Rating Depression Scale (SDS)-Zung; Karnofsky Performance Scale; and Index of Well-Being (IWB). Cox regression models were used to determine whether pre-transplant scores predicted post-transplant survival. RESULTS: Survival rates at 1, 3 and 5 years were 85%, 73% and 69%, respectively. Mean scores on all pre-transplant HRQL measures were unfavorable compared with reference values for the general population. No significant predictors for survival after lung transplantation were found. CONCLUSIONS: Results suggest that scores on the various HRQL measures before transplantation did not predict survival after lung transplantation. The present results do not support the usefulness of pre-transplant HRQL measures for the selection of lung transplant candidates or their urgency for transplantation.

The outcome of primary liver transplantation from deceased donors in children with body weight < or =10 kg.

Between November 1982 and March 2006, 67 children with body weight < or =10 kg had a primary liver transplantation from deceased donors in our unit. The aim of this study was to analyze the outcome in terms of patient and graft survival and to search for factors affecting this outcome. Overall, one-, three-, five-, and 10-yr primary patient and graft survival rates were 73%, 71%, 66%, 63% and 59%, 56%, 53%, 48%, respectively. Twenty-four of 67 (36%) children died and in the remaining 22 (33%), the first grafts failed and they were retransplanted. Cox regression analysis revealed that a need for retransplantation and urgent transplantation were important predictors for patient survival (p = 0.04 and p = 0.001, respectively). To assess whether the need for retransplantation can be influenced, all study variables were compared between surviving grafts and failed grafts. Cox regression analysis showed that only donor/recipient (D/R) weight ratio proved to be independent predictor for graft survival (p = 0.004). After comparison of graft survival with the long rank test according to different D/R weight ratios (3.0-7.0), the cut-off point for significantly different graft survival approached 4.0. The one-, three-, five-, and 10-yr graft survival for technical variant grafts with a D/R weight ratio <4.0 was 85%, 68%, 68%, and 68% compared with a D/R weight ratio >4.0 was 44%, 38%, 38%, and 30%, respectively (p = 0.02). In summary, patient survival in children with body weight < or =10 kg is determined by urgent transplantation and the need for retransplantation. Graft loss and retransplantation in small children can be prevented by adequate size matching of donor and recipient whereby a D/R weight ratio <4.0 seems to offer the favorable outcome.

Cost-effectiveness analysis in severe mental illness: outcome measures selection.

BACKGROUND: Most economic evaluations conducted in mental healthcare did not include widely recommended preference-based health outcomes like the QALY (Quality-Adjusted Life Years). Instead, studies have mainly been designed as cost-effectiveness analyses that include single outcome measures aimed at a (disease) specific aspect of health. AIMS OF THE STUDY: To raise awareness about the potential problems related to the selection of outcome measures for economic studies in patient populations with severe mental illness. Furthermore, to make suggestions that may prevent these problems in future economic evaluations. METHODS: Data of a previously conducted economic evaluation assessing the cost-effectiveness of the HIT (Hallucination focused Integrative Treatment) intervention in patients with schizophrenia were used for the analyses presented in the current paper. Economic analyses based on the results of the selected primary health outcome (Positive and Negative Syndrome Scale: PANSS) were compared with results based on various other health outcomes assessed during the study, including QALYs. RESULTS: No relevant differences between groups were found on the single primary health outcome initially included in the cost-effectiveness analysis. In contrast, relevant and significant differences were identified on three of the four additionally assessed health outcomes. Conclusions based on the results of multiple cost-effectiveness analyses and acceptability curves were strongly in favour of the experimental intervention when including these three additional instruments. QALY results did not show differences between groups. DISCUSSION: Selecting between outcome measures for cost-effectiveness analysis in the field of mental healthcare appears to be a complicated process, which may have considerable consequences for the results of economic studies and subsequent policy decisions. It was argued that inconsistent results across the selected primary health outcome and additionally assessed health outcomes should explicitly be presented to decision-makers. Until there is consensus on a preference-based instrument suited for severe mental illness, QoL instruments could be applied instead of instruments aimed at specific aspects of health. IMPLICATIONS FOR HEALTH POLICIES: Decision-makers in the field of mental healthcare should be careful when interpreting results of economic studies that included outcome measures aimed at a specific aspect of health. Such instruments may provide too narrow a view on relevant changes in health and findings may be difficult to generalise. Due to current reservations on the use of QALYs in mental healthcare, QALY outcomes should be considered in the context of the results of additionally assessed health outcomes.

Cost-effectiveness of neonatal screening for medium chain acyl-CoA dehydrogenase deficiency: the homogeneous population of The Netherlands.

OBJECTIVE: To assess the cost-effectiveness of neonatal screening on medium chain acyl-CoA dehydrogenase (MCAD) deficiency in a homogeneous population. STUDY DESIGN: For the scenario without neonatal screening, medical chart review and interviews were performed with physicians and families of 116 Dutch patients born between 1985 and July 2003 with clinically ascertained MCAD deficiency. For the scenario with neonatal screening, 66,205 unaffected and 11 affected newborns identified by prospective neonatal screening for MCAD deficiency in the northern part of the Netherlands were evaluated. The incremental cost-effectiveness ratio (ICER) used life years (LYs) as the outcome measure by combining both scenarios in a decision model with second-order Monte Carlo simulation. RESULTS: For the scenarios with and without neonatal screening for MCAD deficiency, costs were $6.10 and $4.22 per newborn, respectively. The main cost categories were institutionalization (64%), admissions (17%), special education (8%), laboratory testing (4%), and (para)medical contact (4%). The resulting ICER was $1653 per LY gained. Sensitivity analysis generated an ICER between $14,839 and $4345 per LY gained. CONCLUSIONS: Screening for MCAD deficiency in a well-defined population generates an ICER well within accepted boundaries for cost-effective interventions, even after sensitivity analysis.

The effect of intra-articular injection of ultracain in the temporomandibular joint in patients with preauricular pain: a randomized prospective double-blind placebo-controlled crossover study.

OBJECTIVES: To evaluate the distinguishing ability of intra-articular anesthesia from placebo in orofacial pain patients with pain located in the temporomandibular joint (TMJ) region, aiming at a validation of intra-articular anesthesia injection as a diagnostic test of pain in the TMJ region. MATERIALS AND METHODS: A randomized prospective double-blind, placebo-controlled crossover study was conducted among 19 patients (18 females, 1 male) with pain in the TMJ region. The short-term effects of intra-articular ultracain and saline injections on pain and maximum mouth opening were measured and analyzed. RESULTS: Compared with placebo injections, a statistically significant difference in pain scores was found after intra-articular injection of a local anesthetic on Visual Analog Scale values. The maximum mouth opening scores did, however, not differ between the 2 groups. DISCUSSION: TMJ injection with local anesthesia leads to the decrease of pain in patients with preauricular pain. To establish the source of pain, injection of a local anesthetic in the TMJ may be used as a diagnostic tool. However, the results of diagnostic injections should still be interpreted cautiously.

Long-term health-related quality of life after lung transplantation: different predictors for different dimensions.

BACKGROUND: Lung transplantation has proven to be an effective treatment option for patients with end-stage lung disease with profound effects on both survival and health-related quality of life (HRQL). Generally, studies have reported improved HRQL after lung transplantation. When assessing HRQL, physical, psychologic or social dimensions are usually included. However, it is unclear what predicts outcome, to what extent, and whether there are differences in predictors between dimensions of HRQL. Knowledge about these predictors may be useful when making choices regarding therapy. The research question in the present study was: What are the predicting variables of physical and psychologic dimensions of HRQL, and do they differ? METHODS: Results from studies of the physical dimension (the Nottingham Health Profile's energy and mobility scales) and the psychologic dimension (Zung depression and STAI anxiety scores) from 140 transplanted patients with a maximum follow-up of almost 10 years were assessed using mixed-model analysis. For both dimensions, the following variables were tested for their predictive value: age; gender; diagnosis; year of transplantation; time on the waiting list; type of transplantation; bronchiolitis obliterans syndrome; and pre-transplant HRQL scores. RESULTS: With regard to the physical dimension after lung transplantation, presence of bronchiolitis obliterans syndrome (BOS), age and pre-transplant scores on the measure under study were significant predictors for both energy and mobility. For mobility, gender appeared to be an additional predictor. With regard to the psychologic dimension after lung transplantation, BOS was a predictor for both anxiety and depression. Pre-transplant depression was an additional predictor for post-transplant depression, and age was an additional predictor for anxiety. CONCLUSIONS: Several variables were identified that predicted HRQL after lung transplantation. These variables differed between the physical and psychologic dimensions. The presence of BOS was a predictor for both dimensions. The present findings may be helpful when choosing or developing interventions aimed at improving HRQL after lung transplantation.

RAte Control Efficacy in permanent atrial fibrillation: a comparison between lenient versus strict rate control in patients with and without heart failure. Background, aims, and design of RACE II.

BACKGROUND: Recent studies demonstrated that rate control is an acceptable alternative for rhythm control in patients with persistent atrial fibrillation (AF). However, optimal heart rate during AF is still unknown. OBJECTIVE: To show that in patients with permanent AF, lenient rate control is not inferior to strict rate control in terms of cardiovascular mortality, morbidity, neurohormonal activation, New York Heart Association class for heart failure, left ventricular function, left atrial size, quality of life, and costs. METHODS: The RACE II study is a prospective multicenter trial in The Netherlands that will randomize 500 patients with permanent AF (< or = 12 months) to strict or lenient rate control. Strict rate control is defined as a mean resting heart rate < 80 beats per minute (bpm) and heart rate during minor exercise < 110 bpm. After reaching the target, a 24-hour Holter monitoring will be performed. If necessary, drug dose reduction and/or pacemaker implantation will be performed. Lenient rate control is defined as a resting heart rate < 110 bpm. Patients will be seen after 1, 2, and 3 months (for titration of rate control drugs) and yearly thereafter. We anticipate a 25% 2.5-year cardiovascular morbidity and mortality in both groups. RESULTS: Enrollment started in January 2005 in 29 centers in The Netherlands and is expected to be concluded in June 2006. Follow-up will be at least 2 years with a maximum of 3 years. CONCLUSION: This study should provide data how to treat patients with permanent AF.

Noninferiority testing in cost-minimization studies: Practical issues concerning power analysis.

OBJECTIVES: In cost-minimization studies, it is important to establish noninferiority in the clinical effect of the treatments under investigation. The relationship between the proportion of patients reaching the end point in a study, equivalence limit (delta), and power is investigated in the context of cost-minimization studies with dichotomous clinical end points. Two formulations of the null-hypothesis, absolute and relative formulations of delta, will be explored. METHODS: Sensitivity analysis was performed, in which the effect of the predicted proportions and delta on the power in a noninferiority setting was investigated. The patterns found are discussed in terms of the practical relevance within the cost-minimization framework. RESULTS: Sensitivity analyses show different patterns of results for both null-hypotheses. The differences in these results originate from the way delta is expressed. By expressing delta as absolute difference, power grows quite fast when sample proportions are smaller than expected. In the case of a proportional delta at small sample proportions, the power to establish noninferiority remains low. CONCLUSIONS: To obtain valid results from a cost-minimization study, care has to be taken to adapt the correct methodology for noninferiority testing in clinical outcomes. Defining delta in terms of absolute differences between treatments can lead to obscured results. Although conservative, the expression of delta as a proportion of the effectiveness of the treatment as usual is found to be closer to clinical practice. The inflated delta, resulting from smaller clinical effects than expected when absolute formulation is applied, thus can be avoided.

The effect of HLA mismatches, shared cross-reactive antigen groups, and shared HLA-DR antigens on the outcome after pediatric liver transplantation.

The aim of this study was to analyze the effect of human leukocyte antigen (HLA) class I and HLA-DR mismatching, sharing cross-reactive antigen groups (CREGs), and sharing HLA-DR antigens on the outcome after pediatric liver transplantation. Outcome parameters were graft survival, acute rejection, and portal fibrosis. A distinction was made between full-size (FSLTx) and technical-variant liver transplantation (TVLTx). A total of 136 primary transplants were analyzed. The effect of HLA on the outcome parameters was analyzed by adjusted multivariate logistic and Cox regression analysis. HLA mismatches, shared CREGs, and shared HLA-DR antigens affected neither overall graft survival nor survival after FSLTx. Survival after TVLTx was superior in case of 2 mismatches at the HLA-DR locus compared to 0 or 1 mismatch (P = 0.01) and in case of no shared HLA-DR antigen compared to 1 shared HLA-DR antigen (P = 0.004). The incidence of acute rejection was not influenced by HLA. The incidence of portal fibrosis could be analyzed in 62 1-yr biopsies and was higher after TVLTx than FSLTx (P = 0.04). The incidence of portal fibrosis after TVLTx with 0 or 1 mismatch at the HLA-DR locus was 100% compared to 43% with 2 mismatches (P = 0.004). After multivariate analysis, matching for HLA-DR and matching for TVLTx were independent risk factors for portal fibrosis. In conclusion, an overall beneficial effect of HLA matching, sharing CREGs, or sharing HLA-DR antigens was not observed. A negative effect was present for HLA-DR matching and sharing HLA-DR antigens on survival after TVLTx. HLA-DR matching might be associated with portal fibrosis in these grafts.

Incomplete quality of life data in lung transplant research: comparing cross sectional, repeated measures ANOVA, and multi-level analysis.

BACKGROUND: In longitudinal studies on Health Related Quality of Life (HRQL) it frequently occurs that patients have one or more missing forms, which may cause bias, and reduce the sample size. Aims of the present study were to address the problem of missing data in the field of lung transplantation (LgTX) and HRQL, to compare results obtained with different methods of analysis, and to show the value of each type of statistical method used to summarize data. METHODS: Results from cross-sectional analysis, repeated measures on complete cases (ANOVA), and a multi-level analysis were compared. The scores on the dimension 'energy' of the Nottingham Health Profile (NHP) after transplantation were used to illustrate the differences between methods. RESULTS: Compared to repeated measures ANOVA, the cross-sectional and multi-level analysis included more patients, and allowed for a longer period of follow-up. In contrast to the cross sectional analyses, in the complete case analysis, and the multi-level analysis, the correlation between different time points was taken into account. Patterns over time of the three methods were comparable. In general, results from repeated measures ANOVA showed the most favorable energy scores, and results from the multi-level analysis the least favorable. Due to the separate subgroups per time point in the cross-sectional analysis, and the relatively small number of patients in the repeated measures ANOVA, inclusion of predictors was only possible in the multi-level analysis. CONCLUSION: Results obtained with the various methods of analysis differed, indicating some reduction of bias took place. Multi-level analysis is a useful approach to study changes over time in a data set where missing data, to reduce bias, make efficient use of available data, and to include predictors, in studies concerning the effects of LgTX on HRQL.

Stress, psychological distress, and coping in patients on the waiting list for lung transplantation: an exploratory study.

Little information is available in literature on quality of life, stress and coping during the period patients are waiting for lung transplantation. This study explored potential stressful events that patients experience during the waiting period assessed the level of anxiety and depression and explored the use of coping strategies. Cross sectional analysis were performed. Between 3 and 27 months the number of patients that participated varied between 70 and 21. Measurements took place every 3 months until 27 months after waiting list placement. Instruments were the State Trait Anxiety Inventory, the Self-rating Depression Scale (SDS)-Zung and questions concerning stress and coping. Feeling tension caused by 'having to wear a beeper', and 'being afraid that the transplantation will come too late' were identified as important stress factors. Patients on the waiting list experienced more anxiety and depression, compared to the general population. The longer patients had been on the waiting list, the more anxiety and depression they experienced. Positive coping strategies like 'trying to relax' were more frequently used than negative ones like 'taking sedatives'. Stress, anxiety, and depression occur frequently in waiting list patients.

Rate control is more cost-effective than rhythm control for patients with persistent atrial fibrillation--results from the RAte Control versus Electrical cardioversion (RACE) study.

Aims To evaluate costs between a rate and rhythm control strategy in persistent atrial fibrillation. Methods and results In a prospective substudy of RACE (Rate control versus electrical cardioversion for persistent atrial fibrillation) in 428 of the total 522 patients (206 rate control and 222 rhythm control), a cost-minimisation and cost-effectiveness analysis was performed to assess cost-effectiveness of the treatment strategies. After a mean follow-up of 2.3+/-0.6 years, the primary endpoint (cardiovascular morbidity and mortality) occurred in 17.5% (36/202) of the rate control patients and in 21.2% (47/222) of the rhythm control patients. Mean costs per patient under rate control were euro 7386 and euro 8284 under rhythm control. Cost-effectiveness analysis showed that per avoided endpoint under rate control, the cost savings were euro 24944. Under rhythm control, more costs were generated due to electrical cardioversions, hospital admissions and anti-arrhythmic medication. Costs were higher in older patients, patients with underlying heart disease, those who reached a primary endpoint and women. Heart rhythm at the end of study, did not influence costs. Conclusions Rate control is more cost-effective than rhythm control for treatment of persistent atrial fibrillation. Underlying heart disease but not heart rhythm largely accounts for costs.

The effect of bronchiolitis obliterans syndrome on health related quality of life.

Bronchiolitis obliterans syndrome (BOS) is the most important factor limiting long-term survival after lung transplantation, and has a substantial impact on patients' daily life in terms of disability and morbidity. Aim of our study was to examine the effects of BOS on health related quality of life (HRQL) in lung transplantation patients. Data on HRQL from 29 patients who developed BOS at least 18 months earlier were studied longitudinally. HRQL measures were: the Nottingham Health Profile (NHP), the State Trait Anxiety Inventory (STAI), the Self-rating Depression Scale (ZUNG), and the Index of Well Being (IWB). Furthermore questions concerning activities of daily life and dyspnea were asked. The majority of the patients were male, and the most common diagnosis was emphysema. After the onset of BOS, significantly more restrictions were reported on the dimensions energy and mobility of the NHP. These restrictions appeared to increase over time. After the onset of BOS, STAI scores remained more or less stable and close to the value of the general population. ZUNG scores were significantly higher after the onset of BOS, and patients experienced a lower level of well being than the general population. The percentage of patients that reported to be able to perform activities of daily life without effort declined dramatically after the onset of BOS. Furthermore, the percentage of patients complaining of dyspnea increased after the onset of BOS. In conclusion, our study showed that HRQL was negatively affected by the onset of BOS. However, in spite of these less favorable long-term results, even patients who develop BOS may at least temporarily benefit from a lung transplantation.

Improved quality of life after lung transplantation in individuals with cystic fibrosis.

The aim of the present study was to assess the effect of lung transplantation (LgTX) on health-related quality of life (HRQL) in a group of patients with cystic fibrosis (CF), compared to patients with other diagnoses (non-CF). HRQL was assessed before transplantation in a group of 32 CF patients and 183 non-CF patients. After LgTX, we conducted a prospective longitudinal study among 10 CF patients and 35 non-CF patients who survived at least 31 months after LgTX. Measures were the Nottingham Health Profile (NHP), the State-Trait Anxiety Inventory (STAI), the Self-Rating Depression Scale (ZUNG), the Index of Well-Being (IWB), and the Karnofsky Performance Index. Patients in the CF group were younger, spent more days on the waiting list, and were more likely to be working or going to school than patients with other indications. Before transplantation, CF patients and non-CF patients experienced restrictions on almost all HRQL measures, compared to the general population. On the NHP dimensions of mobility and energy, CF patients had significantly better scores than non-CF patients. Between 1-4 months after transplantation, scores on the NHP, ZUNG, and Karnofsky performance indices improved, and STAI and IWB scores even occurred within the reference value in both groups. Significantly better scores in the CF group compared to the non-CF group were found on the NHP dimension of mobility 4 months after transplantation, and on the dimension of sleep 7 and 13 months after transplantation. Scores remained more or less stable over time in both groups. It may be concluded that patients in both groups experience major restrictions in HRQL before transplantation. However, pretransplant non-CF patients experience more restrictions than CF patients. After LgTX, both groups of patients showed substantial improvement in HRQL, and this improvement was maintained until 31 months after LgTX.

High-dose chemotherapy with hematopoietic stem-cell rescue for high-risk breast cancer.

BACKGROUND: The use of high-dose adjuvant chemotherapy for high-risk primary breast cancer is controversial. We studied its efficacy in patients with 4 to 9 or 10 or more tumor-positive axillary lymph nodes. METHODS: Patients younger than 56 years of age who had undergone surgery for breast cancer and who had no distant metastases were eligible if they had at least four tumor-positive axillary lymph nodes. Patients in the conventional-dose group received fluorouracil, epirubicin, and cyclophosphamide (FEC) every three weeks for five courses, followed by radiotherapy and tamoxifen. The high-dose treatment was identical, except that high-dose chemotherapy (6 g of cyclophosphamide per square meter of body-surface area, 480 mg of thiotepa per square meter, and 1600 mg of carboplatin per square meter) with autologous peripheral-blood hematopoietic progenitor-cell transplantation replaced the fifth course of FEC. RESULTS: Of the 885 patients, 442 were assigned to the high-dose group and 443 to the conventional-dose group. After a median follow-up of 57 months, the actuarial 5-year relapse-free survival rates were 59 percent in the conventional-dose group and 65 percent in the high-dose group (hazard ratio for relapse in the high-dose group, 0.83; 95 percent confidence interval, 0.66 to 1.03; P=0.09). In the group with 10 or more positive nodes, the relapse-free survival rates were 51 percent in the conventional-dose group and 61 percent in the high-dose group (P=0.05 by the log-rank test; hazard ratio for relapse, 0.71; 95 percent confidence interval, 0.50 to 1.00). CONCLUSIONS: High-dose alkylating therapy improves relapse-free survival among patients with stage II or III breast cancer and 10 or more positive axillary lymph nodes. This benefit may be confined to patients with HER-2/neu-negative tumors.

Long-term quality of life in patients surviving at least 55 months after lung transplantation.

The aim of this study was to examine the long-term effect of lung transplantation on Health Related Quality of Life by studying 28 patients who survived at least 55 months after lung transplantation. Measures included the Nottingham Health Profile, questions concerning lung-specific problems, the State-Trait Anxiety Inventory, the Self-rating Depression Scale, the Index of Well-Being, the Karnofsky performance index, and questions concerning activities of daily life. Furthermore, comorbid conditions were measured. Before transplantation patients reported restrictions on almost all quality of life measures. Until approximately 43 months after transplantation there were significant improvements on most dimensions of the Nottingham Health Profile and more patients could walk without dyspnea. Significant improvements occurred with regard to the levels of anxiety, depression, and well being, and the scores on the Karnofsky performance index improved. Activities of daily life could be performed without help by most patients. After approximately 43 months patients experienced more dyspnea, anxiety, depression, and a lower level of well being. The number of patients suffering from a decrease of kidney function, drug treated hyperlipidemia, insulin dependent diabetes mellitus and bronchiolitis obliterans syndrome increased. It may be concluded that patients experience a stable and better overall quality of life after transplantation. Long-term after lung transplantation patients experience a decline on several dimensions of quality of life, which may be explained by an increase of comorbid conditions and Bronchiolitis Obliterans Syndrome.

Graft loss after pediatric liver transplantation.

OBJECTIVE: To describe the epidemiology and causes of graft loss after pediatric liver transplantation and to identify risk factors. SUMMARY BACKGROUND DATA: Graft failure after transplantation remains an important problem. It results in patient death or retransplantation, resulting in lower survival rates. METHODS: A series of 157 transplantations in 120 children was analyzed. Graft loss was categorized as early (within 1 month) and late (after 1 month). Risk factors were identified by analyzing recipient, donor, and transplantation variables. RESULTS: Kaplan-Meier 1-month and 1-, 3-, and 5-year patient survival rates were 85%, 82%, 77%, and 71%, respectively. Graft survival rates were 71%, 64%, 59%, and 53%, respectively. Seventy-one of 157 grafts (45%) were lost: 18 (25%) by death of patients with functioning grafts and 53 (75%) by graft-related complications. Forty-five grafts (63%) were lost early after transplantation. Main causes of early loss were vascular complications, primary nonfunction, and patient death. Main cause of late graft loss was fibrosis/cirrhosis, mainly as a result of biliary complications or unknown causes. Child-Pugh score, anhepatic phase, and urgent transplantation were risk factors for early loss. Donor age, donor/recipient weight ratio, blood loss, and technical-variant liver grafts were risk factors for late loss. CONCLUSIONS: To prevent graft loss after pediatric liver transplantation, potential recipients should be referred early so they can be transplanted in an earlier phase of their disease. Technical-variant liver grafts are risk factors for graft survival. The logistics of the operation need to be optimized to minimize the length of the anhepatic phase.