A型肉毒毒素注射治疗对痉挛型脑瘫患儿运动功能及健康相关生活质量的影响

目的:探讨下肢A型肉毒毒素(botulinum toxin type A,BTX-A)注射治疗对痉挛型双瘫患儿躯体运动功能和健康相关生活质量的影响.方法:51例2岁1个月～9岁6个月痉挛型双瘫患儿随机分为BTX-A注射组26例和对照组25例.患儿均给予系统性康复治疗6个月,注射组在入组第一天给予1次BTX-A注射治疗.在治疗前,治疗后1个月、3个月、6个月对患儿运动功能和生活质量进行评估.结果:注射组与对照组相比用改良Ashworth量表(Modified Ashworth Scale,MAS)量化评分组间差异有统计学意义(P＜0.05);注射组与对照组相比粗大运动功能评分(Gross Motor Function Measure,GMFM)走跑跳功能评分组间差异有统计学意义(P＜0.05);儿科生活质量调查表(The Pediatric Quality of Life Inventory Measurement Models,PedsQL)生理领域的时间因素以及时间因素和分组的交互作用比较有统计学意义(P＜0.05),注射组提高趋势更明显,而心理领域差异无统计学意义(P＞0.05).结论:A型肉毒毒素辅助治疗能更有效改善脑瘫患儿的肌肉痉挛和运动功能,但是短时间内对其社会、心理、情感等方面的功能无明显改善作用.     

Clinical outcomes and health-related quality of life (HRQOL) following haemopoietic stem cell transplantation (HSCT) for paediatric leukaemia

Background Haemopoietic stem cell transplantation (HSCT) is a life‐saving but intensive procedure associated with potentially severe adverse late effects. We aimed to determine morbidity and health‐related quality of life (HRQOL) in a sample of survivors aged 8–18 years at least 1 year post HSCT for paediatric acute leukaemia, compared with a non‐transplanted group of survivors matched for age, gender, initial disease and time since treatment. Methods Families (N = 54; HSCT n= 29) recruited from four UK centres completed measures of child behaviour and school attendance, HRQOL and finances. Mothers completed measures of their own well‐being. Clinical outcome data were extracted from medical records. Results Children in the HSCT group had significantly more late effects and had received more tests for vision, bone, dental and skin health, and thyroid, lung, and gonadal function than the non‐transplanted group. HRQOL scores for the HSCT group were significantly lower in all domains compared with the non‐transplanted group and population norms, but were not significantly related to clinical indices. Mothers in the HSCT group had significantly poorer mental well‐being than population norms. Conclusion Significant morbidity and compromised HRQOL was found in survivors of HSCT. The burden of caring for a child after HSCT has a continuing toll on mothers' well‐being.The importance of counselling families about possible long‐term consequences is emphasized.     

Impact of COPD exacerbations on patient-centered outcomes

BACKGROUND: Frequent exacerbations are associated with a faster decline in FEV(1), impaired health status, and worse survival. Their impact and temporal relationship with other outcomes such as functional status, dyspnea, and the multidimensional body mass index, obstruction, dyspnea, exercise capacity (BODE) index remain unknown. HYPOTHESIS: We reasoned that exacerbations affect the BODE index and its components, and that changes in the BODE index could be used to monitor the effect of exacerbations on the host. STUDY DESIGN: Prospective observational study in a Veterans Affairs medical center. METHODS: We studied 205 patients with COPD (mean [+/- SD] FEV(1), 43 +/- 15% predicted), and recorded the body mass index, FEV(1) percent predicted, modified Medical Research Council dyspnea scale, 6-min walk distance, and the BODE index at baseline, during the exacerbation, and at 6, 12, and 24 months following the first episode, and documented all exacerbations for 2 years after the first acute exacerbation. RESULTS: From the cohort, 130 patients (63%) experienced 352 exacerbations or (0.85 exacerbations per patient per year); 48 patients (23%), experienced one episode, 82 patients (40%) experienced 2 or more exacerbations, and 50 patients required hospitalization. At study entry, exacerbators had a worse mean baseline BODE index score (4.2 +/- 2.1 vs 3.57 +/- 2.3, respectively; p < 0.03). The BODE index score worsened by 1.38 points during the exacerbation, and remained 0.8 and 1.1 points above baseline at 1 and 2 years, respectively. There was little change in BODE index score at 2 years in nonexacerbators. CONCLUSION: COPD exacerbations negatively impact on the BODE index and its components. The BODE index is a sensitive tool used to assess the impact of exacerbations and to monitor COPD disease progression.     

Challenges and recommendations for advancing the state-of-the-science of quality of life assessment in symptom management trials

Major findings are presented from a workshop on Quality of Life Assessment in Cancer Symptom Management Trials, sponsored by the National Cancer Institute. Data-driven research reports focused on 3 topics, 1) the rationale and utility of health-related quality of life (HRQOL) assessment, 2) conceptual models, and 3) measurement and design issues. Recommendations for including HRQOL assessment cited the potential value of: capturing additional treatment effects (eg, fatigue + depression); describing the patient experience; predicting patient prognosis; identifying potential adverse effects; observing interactions among symptoms; calculating quality adjusted survival and cost-effectiveness; and generating new hypotheses. Recommendations for developing more fully developed conceptual models focused on maintaining clear distinctions among symptoms, function, summary measures of HRQOL, and global HRQOL assessments; identifying symptom clusters; pursuing hypotheses about whether clustering is better explained as symptom-related or as patient-related (genetic predispositions); and gaining a better understanding of the dynamic and reciprocal influences of symptoms on each other. With respect to measurement and design issues, because different HRQOL measures cover different domains with various degrees of sensitivity, there is a need to select measures that are carefully tailored to the study's hypotheses. Finally, there is a growing appreciation that trials must be powered to test for effects on secondary endpoints.     

Validity and reliability of the Medical Outcomes Study Short-Form Health Survey version 2 (SF-12v2) among adults with autism

BackgroundThe purpose of the study was to assess the validity and reliability of the Medical Outcomes Study Short Form-12 version 2 (SF-12v2) instrument among adults with autism.MethodsStudy data was collected using a cross-sectional online survey of adults with autism enrolled with the Interactive Autism Network (N = 291). Factorial validity was assessed using confirmatory factor analysis technique. Item–scale correlations were examined for convergent validity. Known-groups validity was assessed by examining the variation in Physical Component Summary (PCS) and Mental Component Summary (MCS) scores by autism severity. Cronbach's alpha was determined for internal consistency reliability. Floor and ceiling effects were also assessed.ResultsA two-factor model with correlated error terms was found to have a good fit. The PCS scale strongly correlated with the underlying items representing the scale. The MCS scale had strong to moderate correlation with its underlying items. For known-groups validity, the MCS score varied as expected with lower score observed among adults with high severity as compared to low severity; however, PCS score varied inversely. Internal consistent reliability of the SF-12v2 was good, and there were no floor and ceiling effects.ConclusionsExcept for known-groups validity, all other psychometric indicators performed well for the SF-12v2.     

A comparative study of health related quality of life, psychological well-being, impact of illness and stigma in epilepsy and migraine

This study aimed to show the difference of epilepsy from another chronic neurological disorder with episodic manifestations (CDEM): migraine. Seventy persons with epilepsy (PWE), 56 persons with migraine (PWM) and 45 healthy control participants (HCs) were included. The groups were compared in terms of demographics, quality of life, depression and self-esteem. The PWE and PWM were also compared with regard to stigma, impact of the illness, disclosure, application of spiritual/traditional healing methods, limitations, most affected life areas, and restrictions. Results showed that the PWM had lower quality of life values than the PWE and the HCs, while there was no difference among the groups in depression and self-esteem. Results also show higher unemployment levels and lower marriage rates for the PWE, where education levels were equal. Although the PWM had higher impact of illness values, the PWE were shown to have higher stigma, more concealment behavior, and higher traditional/spiritual healing application ratios. Also, the PWM emphasized the importance of being “able to fulfil daily living, social and work activities”, while the PWE emphasized the need for “independence” constantly. These results indicate that, although both migraine and epilepsy are CDEMs, they have different levels of impact on patients’ lives. The impacts are socially greater in epilepsy and extend beyond the neurological condition itself.     

Health-related quality of life in adults with drug-resistant focal epilepsy treated with modified Atkins diet in a randomized clinical trial

Ketogenic diet, a high-fat, low-carbohydrate diet, is an established treatment for patients with severe epilepsy. We have previously reported a moderate reduction in seizure frequency after treatment with a modified Atkins diet. This study aimed to see whether dietary therapy impacts patients' health-related quality of life (HRQOL). In a randomized controlled design, we compared the change in self-reported HRQOL among adults with difficult-to-treat epilepsy after a 12-week diet intervention. Thirty-nine patients with drug-resistant focal epilepsy (age = 16–65 years) were randomized to eat a modified Atkins diet with maximum 16 g of carbohydrate per day (diet group, n = 19) or to continue eating habitual diet (control group, n = 20). No changes to the other epilepsy treatments were allowed. Patient-reported HRQOL was assessed with the Quality of Life in Epilepsy Inventory-89 (QOLIE-89). The diet group experienced a statistically significant improvement in mean total score of QOLIE-89 of 10 points compared to controls (p = .002). Moreover, although not statistically significant when using a cutoff of 50% seizure reduction, our data suggest an association between diet-induced reduction in seizure frequency and improvement in HRQOL. The improvement in HRQOL was not associated with diet-induced weight reduction.