Measurement of health-related quality of life in patients with amyotrophic lateral sclerosis in clinical trials of new therapies.

OBJECTIVES: Recent trials of amyotrophic lateral sclerosis (ALS) therapies have included the Sickness Impact Profile (SIP) to evaluate health-related quality of life (HQL). The purpose of this study was to assess the feasibility, psychometric properties, and interpretation of the Sickness Impact Profile in this setting. METHODS: The Sickness Impact Profile was administered at baseline, 3, 6, and 9 months during a double-blind, placebo-controlled study of recombinant human insulin-like growth factor I. The frequency of missing Sickness Impact Profile data and administration time were recorded. Patients' scores on the Appel ALS (AALS) Rating Scale were used to identify a stable subgroup for reliability testing and clinically distinct groups for validity testing. Internal consistency reliability and reproducibility were evaluated using Cronbach's alpha and intraclass correlation coefficients, respectively. Analysis of variance (ANOVA) models and t tests were used to assess validity. Effect sizes and the responsiveness index were used to assess responsiveness. RESULTS: At baseline, 259 (97%) patients completed a 30-minute Sickness Impact Profile interview. At subsequent assessments, response rates ranged from 92% to 97% and mean administration times ranged from 25 to 27 minutes. The overall Sickness Impact Profile score demonstrated alpha reliability and 3-month stability coefficients of 0.94 and 0.80, respectively. Baseline overall Sickness Impact Profile scores discriminated between patients in the two AALS-defined groups with a mean of 13.0+/-7.8 and 24.0+/-11.7 in the better and worse AALS groups, respectively. Similarly, mean overall SIP change scores discriminated patients progressing at different rates (slow to moderate = 4.00+/-7.97; rapid = 10.74+/-8.76). With few exceptions, dimension and category scores met similar criteria. Responsiveness statistics for the physical and overall Sickness Impact Profile scores were lower at 3 months and higher at 6 and 9 months. CONCLUSIONS: The feasibility, psychometric, and interpretive findings support the validity of the Sickness Impact Profile for assessing outcomes of amyotrophic lateral sclerosis and its treatment. Based on these findings, we recommend including the Sickness Impact Profile in future amyotrophic lateral sclerosis clinical trials.     

Quality of life in amyotrophic lateral sclerosis

Although quality of life is difficult to define clearly, a number of instruments and questionnaires have been developed and applied to patients who have amyotrophic lateral sclerosis (ALS). This article reviews the spectrum of quality-of-life questionnaires and instruments used for ALS and the data generated from them. It discusses positive and negative factors that can affect quality of life for the patient and caregiver and concludes with suggestions for ongoing management to enhance quality of life.     

Survival and quality of life after tracheostomy for acute respiratory failure in patients with amyotrophic lateral sclerosis

Background

Acute respiratory failure (ARF) is a common event in the advanced stage of amyotrophic lateral sclerosis (ALS) and may be rarely a presenting symptom. Frequently, such patients require intubation and mechanical ventilation (MV) and, in a large proportion, receive tracheostomy, as a consequence of weaning failure. In our study, we investigated postdischarge survival and quality of life (QoL) after tracheostomy for ARF in patients with ALS.

Methods

Design: This study is a retrospective chart review combined with prospective evaluation of QoL and degree of depression. Setting: The study was conducted in an adult, respiratory intensive care unit in a university hospital. Patients: Amyotrophic lateral sclerosis patients with tracheostomy for ARF between January 1, 1995 and April 30, 2008 were investigated. Intervention and measurements: (a) A retrospective chart review was used and (b) prospective administration of the 11-item short-form Life Satisfaction Index (LSI-11) and Beck Depression Inventory (BDI) questionnaires to survivors, at least 1 month after discharge from hospital, was performed.

Results

Sixty patients were studied retrospectively. None of the patients died in the hospital after tracheostomy. Forty-two patients (70%) were discharged completely MV dependent, and 17 patients (28.3%) were partially MV dependent. One patient (1.6%) was liberated from MV.The median survival after tracheostomy was 21 months (range, 0-155 months). The survival rate was 65% by 1 year and 45% by 2 years after tracheostomy. Survival was significantly shorter in patients older than 60 years at tracheostomy, with a hazard ratio of dying of 2.1 (95% confidence interval, 1.1-3.9). All 13 survivors completed the LSI-11 and BDI. The mean (SD) cumulative score on the LSI-11 was 9.3 (3.6; range, 0-22; higher values indicating better QoL), similar to that obtained from a control group consisting of individuals with ALS who had not received tracheostomy (9.3 ± 4.3) and to that reported for persons in the general population. Only 15% of the tracheostomized patients (2/13) were severely depressed, according to BDI; 11 of 13 patients reported a positive view of tracheostomy and said that they would want to undergo this procedure if they could make the decision again.

Conclusions

Patients with ALS have a high chance of long-term survival after tracheostomy for ARF. Although administered at the time of a respiratory crisis without being discussed in advance, tracheostomy shows good acceptance and results in acceptable QoL.     

Correlation of visual function with health-related quality of life in glaucoma patients

Objectives  The purpose of this study was to assess visual impairment and quality of life (QOL), the relationship between visual function and health-related and vision-related QOL, and to identify factors significantly impacting QOL for glaucoma patients.
Methods  One-on-one interviews of 280 glaucoma patients at a regional hospital in Taiwan from 1 April 2005 to 30 September 2005. Data collected included demographics, clinical examinations and treatment status. QOL data collected utilized the Chinese (Taiwanese) version of the Medical Outcomes Study Short-Form 36 Health Survey (SF-36) and the National Eye Institute Visual Function-25 (NEIVFQ-25).
Results  The majority of study participants had open-angle glaucoma (54.3%) and were classified as moderate stage (33.6%). The higher the health-related QOL scores for both questionnaires, the fewer difficulties experienced by the patients. Moderate correlation was found between the SF-36 and NEIVFQ-25 subscales and visual function. Significant predicting factors for the SF-36 were gender, age, employment status, presence of systemic disease, better-eye visual acuity and better-eye mean defect. For the NEIVFQ-25, these were general vision, near and distance activities, colour vision and peripheral vision scores.
Conclusions  A combination of demographic data, clinical examination and QOL questionnaires may help clinicians better ascertain the impact of disease severity on glaucoma patients' QOL. This information may help in patient education, treatment compliance and selection of treatment options.     

肌萎缩侧索硬化患者焦虑和抑郁状况及其影响因素调查

目的了解肌萎缩侧索硬化(amyotrophic lateral sclerosis,ALS)患者焦虑、抑郁状况及其影响因素。方法采用Zung编制的焦虑自评量表(self-rating anxiety scale,SAS)、抑郁自评量表(self-rating depression scale,SDS),对174例ALS患者进行调查。了解ALS患者焦虑、抑郁状况及其影响因素。结果 174例患者中,发生焦虑21例,占12.07%;发生抑郁65例,占37.36%。患者SAS、SDS得分均高于全国常模,组间比较,均P0.001,差异具有统计学意义。女性患者抑郁发生率较男性患者高(P0.05)。结论 ALS患者焦虑、抑郁心理问题较正常人更为突出,治疗过程中要重视对患者的心理支持和疏导,特别应关注女性患者的心理健康状况。     

End-of-life decision making in 42 patients with amyotrophic lateral sclerosis

OBJECTIVE: To determine when end-of-life issues were discussed with patients afflicted with amyotrophic lateral sclerosis (ALS). METHODS: This was a retrospective analysis of ALS patients referred to the neuromuscular clinic at Georgetown University Hospital. Patients were seen by a pulmonologist and a neurologist at the initial diagnosis or referral, and every 2-3 months thereafter. End-of-life discussions were addressed at each visit. Other variables recorded included the amount of time afflicted with ALS, serial pulmonary function test results, and the subjective level of bulbar dysfunction. RESULTS: We saw 43 patients (age range 39-94 y) between June 1999 and September 2004. One patient was on a ventilator at the initial visit, and was therefore excluded from the study. Discussion about the patients' end-of-life care preferences were initiated at the first pulmonary visit with 40 patients. With 2 patients, end-of-life decisions were discussed at the second office visit. Twenty-five patients chose do-not-resuscitate and do-not-intubate (DNR/DNI) orders after the initial end-of-life discussion with the pulmonologist. Five other patients chose DNR/DNI orders during subsequent clinic visits. Four patients were still undecided at their last clinic visit. Six patients were lost to follow-up before a decision was made. Two patients requested full ventilatory support. Both the forced vital capacity and the level of bulbar dysfunction were not statistically different between the patients who chose DNR/DNI and the patients who were either undecided or requested full ventilatory support. CONCLUSIONS: Decisions about end-of-life care are often delayed in patients with ALS. These patients' final decisions seem to be independent of their level of respiratory insufficiency or bulbar function, and most related to the physician addressing end-of-life care decisions in a timely manner.     

Quality of life and psychosocial issues in ventilated patients with amyotrophic lateral sclerosis and their caregivers

Non-invasive ventilation (NIV) is an efficient palliative measure for symptoms of chronic hypoventilation in patients with amyotrophic lateral sclerosis (ALS), and can also lengthen survival. A subset of ALS patients undergoes tracheostomy ventilation (TV) for life prolongation. We investigated the quality of life (QOL) and psychosocial situation of 52 home ventilated ALS patients and their caregivers. The battery included sociodemographic, generic, and disease-specific variables, as well as the Profile of Mood States and the Munich Quality of Life Dimensions List. Data were compared between the NIV (n=32) and the TV (n=21) groups. Mean ventilation time was 14 months for NIV and 35 months for TV. Eighty-one percent of TV patients had been tracheotomized without informed consent. The data show a good overall QOL for both NIV and TV patients, but a very high burden of care for TV caregivers, 30% of whom rated their own QOL lower than their patient's QOL. Sexuality was an important issue. Thus, any assessment of QOL in a home palliative care situation should include the primary caregivers.     

Daily physical activity in adults with congenital heart disease is positively correlated with exercise capacity but not with quality of life

Objective  

With the fear of heart failure and sudden death, many patients with congenital heart disease (CHD) traditionally were withheld from exercise activities. Much is reported on sedentary lifestyle with diminished exercise capacity in children with CHD which results in an increased cardiovascular risk. This study aimed to assess whether the sedentary lifestyle is also present in adulthood and whether this is associated with exercise capacity and quality of life (QoL) limitations.     

Total antioxidant status is increased in the serum of amyotrophic lateral sclerosis patients

Oxidative stress may play an important role in the etiopathogenesis of amyotrophic lateral sclerosis (ALS). The aim of the study was to investigate serum total antioxidant status (TAS) level, a measure of peroxyl‐scavenging capacity, in ALS patients. TAS level was determined by the colorimetric method in the serum from 28 ALS patients and 20 healthy control group subjects. The study revealed that serum TAS level was significantly higher in ALS patients compared with that in controls (p<0.05). There was no significant difference in TAS level between the groups of patients classified according to their age and sex, clinical state, type of ALS onset and duration of disease (p>0.05). The change of TAS level in ALS patients observed in this study suggests that oxidative stress may play an important role in neurodegeneration in ALS. An increase in plasma TAS level may be the result of endogenous protection mechanisms against free radical toxicity, but serum TAS level in ALS patients cannot be a marker of ALS duration, clinical state of patients and type of ALS onset.     

Total antioxidant status is increased in the serum of amyotrophic lateral sclerosis patients

Oxidative stress may play an important role in the etiopathogenesis of amyotrophic lateral sclerosis (ALS). The aim of the study was to investigate serum total antioxidant status (TAS) level, a measure of peroxyl-scavenging capacity, in ALS patients. TAS level was determined by the colorimetric method in the serum from 28 ALS patients and 20 healthy control group subjects. The study revealed that serum TAS level was significantly higher in ALS patients compared with that in controls (p<0.05). There was no significant difference in TAS level between the groups of patients classified according to their age and sex, clinical state, type of ALS onset and duration of disease (p>0.05). The change of TAS level in ALS patients observed in this study suggests that oxidative stress may play an important role in neurodegeneration in ALS. An increase in plasma TAS level may be the result of endogenous protection mechanisms against free radical toxicity, but serum TAS level in ALS patients cannot be a marker of ALS duration, clinical state of patients and type of ALS onset.     

Health problems and health-related quality of life in people with multiple sclerosis

OBJECTIVE: To explore the interrelationship between a number of common multiple sclerosis-related problems and health-related quality of life independent of the effect of physical disease impact. DESIGN: A cross-sectional postal survey. SETTING: The study was conducted in seven treatment centres within England. SUBJECTS: Nine hundred and twenty-nine people with multiple sclerosis. MEASURES: The severity of the multiple sclerosis problems (fatigue, pain, urinary incontinence, bowel, depression, pressure ulcers, sexual and employment problems) was assessed using 5-point self-report ordinal scales. Disease impact was measured using the Multiple Sclerosis Impact Scale-29. Health-related quality of life was measured using the 36-Item Short Form Health Survey, version 2 (SF-36). The relationship between the problems, disease impact and health-related quality of life was determined using multivariate analysis of covariance. RESULTS: Seventy four per cent of the sample experienced four or more of the selected problems. The extent and severity of problems increased with the level of disease impact (P< 0.001). Fatigue, pain, employment, depression and relationship problems were all found to have negative effect on the SF-36 scales independent to the effect of disease impact. SF-36 scores were most compromised in patients experiencing multiple problems. The data suggest an interdependent relationship between depression, fatigue and pain. CONCLUSION: The data suggest that many multiple sclerosis complications negatively affect health-related quality of life, independent of the effect of overall disease impact.