Factors that impact health-related quality of life in adults with celiac disease： A multicenter study

AIM： TO evaluate the factors involved in the impairment of health-related quality of life （HRQOL） in patients with celiac disease. METHODS： A multicenter, cross-sectional prospective study was performed in patients with celiac disease who completed two HRQOL questionnaires： the gastrointestinal quality of life index （GIQLI） and the EuroQol-5D （EQ）. RESULTS： Three hundred and forty patients （163 controlled with a gluten-free diet, and 177 newly diagnosed with a normal diet） were included. The GIQLI score was significantly better in patients on a gluten- free diet （GFD） than in non-treated patients on their usual diet, both in terms of the overall score （3.3 vs 2.7, respectively; P 〈 0.001）, as well as on the individual questionnaire dimensions. Both the preference value of the EQ as the visual analogue scale were significantly better in treated than in non-treated patients （0.93 vs 0.72 P 〈 0.001 and 80 vs 70 P 〈 0.001, respectively）. Variables significantly associated with a worse HRQOL score were female gender, failure to adhere to a GFD, and symptomatic status. CONCLUSION： In untreated celiac disease, the most important factors that influence patient perception ofhealth are the presence of symptoms and a normal diet. HRQOL improves to levels similar to those described in the general population in celiac disease patients well controlled with a GFD.     

Palliation of malignant biliary obstruction: a prospective trial examining impact on quality of life

BACKGROUND: Endoscopic decompression is used for palliation of patients with malignant biliary obstruction. Little is known of its effect on quality of life. The aims of this study were to determine clinical characteristics that have the greatest adverse impact on quality of life in patients with malignant biliary obstruction, and to quantify changes in the quality of life of patients with malignant biliary obstruction after successful decompression with a plastic stent. METHODS: Patients with malignant biliary obstruction without liver metastases considered nonsurgical candidates and referred to a tertiary university medical center for palliative endoscopic decompression were sequentially enrolled in this prospective cohort study. The SF-36 Health Survey questionnaire at baseline and 1 month after stent insertion was used to quantify quality of life. Results were correlated to clinical and laboratory parameters. Multivariate analyses were carried out to determine independent predictors of baseline quality of life and improvement after stent insertion. RESULTS: Fifty patients (20 men, 30 women; mean [SD] age 72.6 [10.6] years) with a mean weight of 62.4 (12.9) kg and mean body mass index of 23.4 (4.3) kg/m(2) were enrolled. Two thirds had a distal malignant lesion, 12.5% had mid bile duct obstruction, and the rest either hilar or intrahepatic cholangiocarcinoma. At baseline, 70% complained of pruritus and 98% were jaundiced (mean total bilirubin 15 [7] mg/dL). Mean duration of symptoms before decompression was 23 (25) days. Weight loss, and elevated bilirubin level had the greatest impact on baseline quality of life domains in both univariate and multivariate analysis. After biliary drainage, complete follow-up information was available for 51% of the initial cohort. Among these 26 patients, a 33% improvement in bilirubin level was documented in 84% of patients and was associated with significant improvements in social function (relative risk = 0.11; 95% CI [0.03, 0.19]) and mental health (relative risk = 0.036; 95% CI [0.011, 0.08]). A baseline bilirubin of greater than 14 mg/dL was associated with lack of improvement in social function at 1-month follow-up (p = 0.03). CONCLUSIONS: Weight loss and hyperbilirubinemia are strongly predictive of poor quality of life before endoscopic decompression. Successful biliary drainage after stent insertion is associated with improvements in quality of life, although this is less true among patients with a baseline bilirubin over 13 mg/dL. These results may lead to better selection of patients for palliative biliary decompression and require prospective validation.     

Auranofin therapy and quality of life in patients with rheumatoid arthritis. Results of a multicenter trial

In a six-month, randomized, double-blind study at 14 centers, auranofin (3 mg twice daily) was compared with placebo in the treatment of patients with classic or definite rheumatoid arthritis. All patients had unremitting disease for at least the previous six months and at least three months of therapy with nonsteroidal anti-inflammatory drugs (NSAIDs). NSAIDs, oral steroids, and analgesics were allowed throughout the trial. Efficacy was analyzed in 154 patients who received auranofin and 149 who received placebo. To reflect an expanded view of outcome assessment, the measures used included some 20 nontraditional measures of functional performance, pain, global impression, and utility (worth or value) in addition to five standard clinical measures of rheumatoid synovitis (e.g., number of tender joints). The nontraditional measures were mainly in the form of structured questionnaires administered by trained interviewers. To minimize the statistical problem of multiple comparisons, most of the measures were grouped into four composites--clinical (standard measures), functional, global, and pain--and the treatment effect for each composite was tested at the 0.0125 level of significance. Auranofin was superior to placebo in the clinical (p = 0.003), functional (p = 0.001), and global (p = 0.007) composites and trended similarly in the pain composite (p = 0.021). Individual measures within the composites consistently favored auranofin. Other measures, not part of the composites, also favored auranofin, including a patient utility measure designed for this study, the PUMS (p = 0.002). Results confirm the hypothesis that the favorable effect of auranofin on clinical synovitis is accompanied by improvements across a range of outcomes relevant to the patient's quality of life.     

Primarily chronic progressive and relapsing/remitting multiple sclerosis: two immunogenetically distinct disease entities

HLA class II gene polymorphism was investigated in 100 patients with clinically definite multiple sclerosis (MS) by restriction fragment length polymorphism analysis of Taq I-digested DNA using DRB, DQA, and DQB cDNA probes. Twenty-six patients had primarily chronic progressive MS and 74 had relapsing/remitting MS. The latter group included patients with a secondary progressive evolution of symptoms. Both clinical forms of MS were found to be associated with the DRw15,DQw6 haplotype. In addition, primarily chronic progressive MS was positively associated with the DQB1 restriction fragment pattern seen in DR4,DQw8, DR7,DQw9, and DRw8, DQw4 haplotypes, as well as negatively associated with the Taq I DQB1 allelic pattern corresponding to the serological specificity DQw7. Relapsing/remitting MS was positively associated with the DQB1 allelic pattern observed in the DRw17,DQw2 haplotype. These three DQB1 alleles are in strong negative linkage disequilibria with DRw15. The two susceptibility markers of each clinical form of MS act additively in determining the genetic susceptibility, as the relative risks for individuals carrying both markers roughly equal the sum of respective risks. Different alleles of the DQB1 locus defined by restriction fragment length polymorphisms contribute to susceptibility and resistance to primarily chronic progressive MS as well as to susceptibility to relapsing/remitting MS. The observed immunogenetic heterogeneity between the different clinical forms of MS favors the hypothesis that primarily chronic progressive MS and relapsing/remitting MS are two distinct disease entities.     

Autologous stem cell transplantation in children with severe progressive systemic or polyarticular juvenile idiopathic arthritis: long-term follow-up of a prospective clinical trial

OBJECTIVE: To assess the safety and efficacy of intensive immunosuppression followed by T cell-depleted autologous hematopoietic stem cell transplantation (ASCT) for induction of disease remission in children with refractory progressive juvenile idiopathic arthritis (JIA). METHODS: Twenty-two patients with progressive refractory JIA were followed up over a median period of 80 months after pretreatment with intensive immunosuppression followed by ASCT in a multicenter, prospective, phase II clinical trial. Hematopoietic stem cells were harvested from the patients' bone marrow, depleted of T cells, and kept frozen until used for ASCT. Pretreatment of patients consisted of a combination of antithymocyte globulin, cyclophosphamide, and low-dose total body irradiation. Patients were followed up for ASCT-related complications, recovery of hematologic and immune system parameters, and disease outcomes. RESULTS: Reconstitution of hematologic values to normal range was rapid. Recovery of immune system parameters, especially normalization of CD4+, CD45RA+ naive T cells, was delayed, occurring at >/=6 months after ASCT. The prolonged period of immune deficiency resulted in a large number of viral infections and may have contributed to the development of macrophage activation syndrome (MAS), leading to death, in 2 patients. After ASCT, 8 of the 20 evaluable patients reached complete clinical remission of their JIA, 7 were partial responders, and 5 experienced a relapse of their disease (occurring 7 years after ASCT in 1 patient). Later during followup, 2 of the patients whose disease relapsed died from infections that developed after restarting immunosuppressive medication. CONCLUSION: Intensive immunosuppression followed by ASCT resulted in sustained complete remission or marked improvement in 15 of 22 patients with progressive refractory JIA. The procedure, however, is associated with significant morbidity and risk of mortality due to prolonged and severe depression of T cell immunity. After fatal complications due to MAS were observed in some patients, the protocol was amended in 1999, to ensure less profound depletion of T cells, better control of systemic disease before transplantation, antiviral prophylaxis after transplantation, and slow tapering of corticosteroids. Following these protocol modifications, no additional ASCT-related deaths were observed among the 11 patients who received the modified treatment.     

Impairment of health-related quality of life in patients with inflammatory bowel disease: a Spanish multicenter study

BACKGROUND: Inflammatory bowel disease impairs patients' perception of health and has a negative impact on health-related quality of life (HRQOL). Most studies include patients from a single hospital. This may bias limit results through the use of small patient samples and/or samples within a restricted disease spectrum. METHODS: HRQOL was measured in patients with ulcerative colitis (UC) and Crohn's disease (CD) from 9 hospitals located in different geographical areas in Spain using 2 questionnaires: the Spanish version of the Inflammatory Bowel Disease Questionnaire (IBDQ) and the EuroQol. Results are expressed as medians. RESULTS: The study included 1156 patients (528 patients with UC and 628 with CD; median age, 35 yr; slight predominance of women, 617 versus 539). HRQOL worsened in parallel with disease severity to a similar extent in both UC (IBDQ scores of 6.1, 4.7, and 4.0 for the 3 disease severity groups, respectively) and CD (IBDQ scores of 6.1, 5.0, and 4.1, respectively). A similar inverse relation between clinical activity and quality of life was observed when EuroQol preference values were used. All 5 dimensions of the IBDQ showed significantly lower scores in patients with active UC and CD than in patients in remission. The pattern of scores by IBDQ dimensions differed between patients in relapse (who scored worse on the digestive symptoms dimension) and patients in remission. Variables related with disease activity, time of evolution since diagnosis and female sex, were significantly associated with having a worse perception of HRQOL. The type of disease or geographical area of residence did not influence results on the IBDQ. CONCLUSIONS: UC and CD impair patients' HRQOL, and the degree of impairment depends on disease activity but is independent of the type of disease and place of residence.     

Validity of a new quality of life scale for functional dyspepsia: a United States multicenter trial of the Nepean Dyspepsia Index

OBJECTIVE: The lack of a suitable disease-specific, health-related quality of life instrument for dyspepsia prompted the development of the Nepean Dyspepsia Index (NDI). The utility of the NDI in functional dyspepsia is unknown. We aimed to assess the validity of this new quality of life instrument for the first time in United States patients with documented functional dyspepsia. METHODS: The long form of the NDI contains a symptom index and 42 items designed to measure impairment of subjects' ability to engage in and to enjoy relevant aspects of their life because of dyspepsia, as well as a ranking of the individual importance of each aspect. Patients (n = 101, mean age 51 yr, 62% female) who had a history of functional dyspepsia for > or = 1 month and a negative endoscopy within the prior 1 yr were followed for 14 days. Patients completed the NDI and the validated Speilberger State-Trait Anxiety Inventory, Beck Depression Inventory, Short Form-36, and a global assessment of symptoms and quality of life at baseline and 14 days later; the NDI was also retested at 48 h and 2 wk. RESULTS: Five clinically relevant factors (subscales), namely, tension/sleep, interference, eating/drinking, knowledge/control, and work/study were identified by factor analysis, after incorporating individual importance ratings (25 items total). All subscales had excellent face validity and internal consistency (Chronbach's alpha, all >0.85). Reliability was also excellent (intraclass correlations all >0.84). There were modest typically negative correlations between a number of the NDI subscales and the Short Form-36, anxiety, and depression, indicating that the NDI is disease-specific and supporting its validity. Changes in NDI scales correlated moderately with global assessment of change (total score r = -0.49), indicating initial responsiveness. CONCLUSIONS: The Nepean Dyspepsia Index is a valid, disease-specific index for functional dyspepsia, measuring symptoms and health-related quality of life.     

Autologous bone marrow transplantation for progressive non-Hodgkin's lymphoma: clinical impact of immunophenotype and in vitro purging

From 1983 to 1989 we performed a prospective trial of 70 consecutive, in vitro purged autologous bone marrow transplants (BMT) for patients with progressive non-Hodgkin's lymphoma. Forty-nine patients had responsive disease at the time of transplantation while 21 others had refractory high risk lymphoma. Forty-two patients with B-lineage lymphoma received autologous marrow purged in vitro with monoclonal antibody (anti CD9, CD10, CD24) plus complement, 12 with T-lineage lymphoma received monoclonal antibody immunotoxins (anti CD5, CD7-ricin conjugates) along with 4-hydroperoxycyclophosphamide purging and 16 received unpurged marrow. All received cyclophosphamide, 57 with fractionated total body irradiation, and 13 with BCNU and cytarabine. Hematologic engraftment was prompt and unaffected by phenotype (B vs. T) or by in vitro purging used (B vs. T vs. none) although nine of 16 non-relapse deaths were related to poor graft function. Fifty-one patients (73%) were alive in complete remission (CR) 1 month following transplantation while 15 patients (12 with initially refractory disease) had persistent disease. Subsequently, 41 +/- 18% (by Kaplan-Meier estimate; +/- 95% confidence limits) of those who achieved CR remained relapse free 1-6.4 (median 3) years post-BMT. Neither risk group, purging, nor immunophenotype predicted subsequent post-transplant relapse. Among those 51 who achieved CR, 13 of 43 (27 +/- 14%) with responsive disease survive disease free while three of eight (38 +/- 34%) refractory patients survive disease free (p = 0.96). Overall, 24 patients survive, 16 in continuous complete remission 1-6.5 years following transplantation.(ABSTRACT TRUNCATED AT 250 WORDS)     

Accessibility to biologics and its impact on disease activity and quality of life in patients with rheumatoid arthritis in Kuwait

Clinical Rheumatology - Biologics are indicated in rheumatoid arthritis (RA) in case of persistent high disease activity despite conventional disease-modifying anti-rheumatic drugs (cDMARDs) or...