METHODS: HELIOS-A was a phase 3, global, open-label study comparing the efficacy and safety of vutrisiran with an external placebo group (APOLLO study). Patients were randomized 3:1 to subcutaneous vutrisiran 25 mg every 3 months (Q3M) or intravenous patisiran 0.3 mg/kg every 3 weeks (Q3W) for 18 months.
RESULTS: HELIOS-A enrolled 164 patients (vutrisiran, n = 122; patisiran reference group, n = 42); external placebo, n = 77. Vutrisiran met the primary endpoint of change from baseline in modified Neuropathy Impairment Score +7 (mNIS+7) at 9 months (p = 3.54 × 10-12), and all secondary efficacy endpoints; significant improvements versus external placebo were observed in Norfolk Quality of Life-Diabetic Neuropathy, 10-meter walk test (both at 9 and 18 months), mNIS+7, modified body-mass index, and Rasch-built Overall Disability Scale (all at 18 months). TTR reduction with vutrisiran Q3M was non-inferior to within-study patisiran Q3W. Most adverse events were mild or moderate in severity, and consistent with ATTRv amyloidosis natural history. There were no drug-related discontinuations or deaths.
CONCLUSIONS: Vutrisiran significantly improved multiple disease-relevant outcomes for ATTRv amyloidosis versus external placebo, with an acceptable safety profile.
CLINICALTRIALS.GOV: NCT03759379.
MATERIALS AND METHODS: Systematic searches of PubMed, Scopus and Web of Science databases were conducted. Independent reviewers screened the title, abstract and full texts according to predetermined inclusion and exclusion criteria. Articles published in English from January 2012 to March 2022 reporting the QoL of non-syndromic young patients aged 7-18 years with CL/P were included. Review articles and articles reporting the psychological adjustment of parents or other family members with CL/P were excluded.
RESULTS: 975 publications were identified, of which 20 studies met our inclusion criteria. The majority of studies reported that the CL/P condition has a negative impact on the QoL. Psychological health, functional well-being, socialemotional well-being and school environment are domains that are affected. Compared with typically developing young patients, those with CL/P had lower QoL scores even though QoL was assessed using different instruments across studies. The impact of CL/P on overall QoL scores varied by age but not gender or cleft type.
CONCLUSION: Our reviews had shown the presence of CL/P negatively affects the QoL of young patients. Psychological health is the most affected QoL domain. Understanding the impacted domain will help in planning and delivering better health care for individuals with CL/P and reducing the stigma commonly associated with CL/P. Future studies should target intervention on psychological health and consider resilience factors towards positive adjustment.
MATERIALS AND METHODS: Post-COVID-19 clinic attendees, consisting of post-COVID-19 patients discharged from major hospitals in the state of Selangor during the third pandemic wave of COVID-19 in Malaysia, were enrolled in this retrospective study for 6 months. Physician-scored Modified Medical Research Council (mMRC), patient self-reported quality of life (EQ-VAS) score and follow-up CT scan were evaluated.
RESULTS: Our cohort comprised 131 patients, with a median age of 52 (IQR 39-60) years and median BMI of 29.40 (IQR 25.59-34.72). Majority (72.5%) had co-morbidities, and 97.7% had severe disease requiring supplementary oxygen support during the acute COVID-19 episode. 56.5% required intensive care; among which one-third were invasively ventilated. Median equivalent dose of methylprednisolone prescribed was 2.60 (IQR 1.29-5.18) mg/kg during admission, while the median prednisolone dose upon discharge was 0.64 (IQR 0.51-0.78) mg/kg. It was tapered over a median of 8.0 (IQR 5.8-9.0) weeks. Upon follow-up at 11 (IQR 8-15) weeks, one-third of patients remained symptomatic, with cough, fatigue and dyspnoea being the most reported symptoms. mMRC and EQ-VAS scores improved significantly (p<0.001) during follow-up. Repeat CT scans were done in 59.5% of patients, with 94.8% of them demonstrating improvement. In fact, 51.7% had complete radiological resolution. Intensive care admission and mechanical ventilation are among the factors which were associated with poorer radiological outcomes, p<0.05.
CONCLUSION: Approximately one-third of patients with SARSCoV- 2-related OP remained symptomatic at 3 months of follow-up. Majority demonstrated favourable radiological outcomes at 5-month reassessment, except those who required intensive care unit admission and mechanical ventilation.
METHODS: An electronic data search in Web of Science, SCOPUS, and PubMed for relevant papers; those written in English; those reporting quantitative data; and those including more than one hundred patients who received just chemotherapy were included. Studies involving participants that received other kinds of anti-neoplastic therapies were excluded.
RESULTS: A total of 35 papers published between January 2000 and December 2021 were retrieved from the search databases of which (11612 patients) met the inclusion criteria. Findings showed that fatigue negatively affected QOL with a pooled prevalence of 49% (95% CI; 25.00-74.00) and the significant heterogeneity between articles was (I²=98%, P <0.001). Further, breast cancer contributed to the majority of selected articles with about 55 % (95%CI; 9:00- 94:00), followed by cancer (unspecified) 44% (95%CI; 5:00 - 92:00). Most studies (71%) (95%CI; 4:00 - 99:00) used the brief fatigue inventory (BFI) tool to assess severity of fatigue and 39% (95%CI; 17:00 -68:00) employed the European Organisation for the Research and Treatment of Cancer Quality of Life Questionnaire to evaluate QOL among cancer survivors.
CONCLUSION: The prevalence of fatigue in cancer patients is high and fatigue has a negative impact on QOL of cancer patients receiving chemotherapy. Directionality, fatigue needs to be addressed and treated properly for better improvement of health status and QOL in cancer patients.
METHODS: A cross-sectional study of consecutive adult patients who underwent glucose hydrogen breath test was conducted. Factors associated with SIBO were evaluated. Symptom severity and HRQoL of IBS patients with and without SIBO were compared. The independent factors associated with severe IBS were explored.
RESULTS: A total of 160 patients were included (median age 40 years, males 31.3%). IBS was present among 53.8% of subjects, with 33.8% having diarrhea-predominant IBS (IBS-D). SIBO was diagnosed in 22.5% of the study population. Patients with SIBO were more commonly diagnosed with IBS-D than those without (50.0% vs 29.0%, P = 0.019). Severe IBS was associated with SIBO (36.4% vs 15.6%, P = 0.043). SIBO was associated with poorer HRQoL (Euroqol five-dimensional utility score: 0.73 vs 0.80, P = 0.024). SIBO (44.4% vs 20.6%, P = 0.043), anxiety (77.8% vs. 39.7%, P = 0.004), and depression (50.0% vs 19.1%, P = 0.011) were associated with severe IBS in the univariate analysis. However, SIBO was the only independent factor associated with severe IBS in the multivariate analysis (adjusted odds ratio 3.83, 95% confidence interval CI 1.02-14.34, P = 0.046).
CONCLUSIONS: There was a significant association between IBS-D and SIBO. The coexistence of SIBO had a significant negative impact on IBS patients.
METHODS: This multinational cross-sectional survey was conducted in rehabilitation facilities in Malaysia, Indonesia, and Thailand. Community-dwelling adults with traumatic or nontraumatic spinal cord injury participating in the International Spinal Cord Injury Community Survey from 2017 to 2018 were enrolled. Data regarding bladder management/care, presence of bladder dysfunction, urinary tract infection, and quality of life score were extracted from the International Spinal Cord Injury Community Survey questionnaire. The impact of bladder care and urinary complications on quality of life was determined using univariable and multivariable regression analysis.
RESULTS: Questionnaires from 770 adults were recruited for analysis. After adjusting for all demographic and spinal cord injury-related data, secondary conditions, as well as activity and participation factors, urinary tract infection was an independent negative predictive factor of quality of life score ( P = 0.007, unstandardized coefficients = -4.563, multivariable linear regression analysis, enter method).
CONCLUSIONS: Among bladder care and urinary complication factors, urinary tract infection is the only factor negatively impacting quality of life. These results address the importance of proper bladder management and urinary tract infection prevention in persons with spinal cord injury to improve their quality of life.
METHODS: This retrospective cross-sectional study uses data from the Malaysian Psoriasis Registry (MPR) from January 2007 to December 2018.
RESULTS: Of 21 735 psoriasis patients, 34 (0.16%) had coexistent LE. The male to female ratio among psoriasis patients with coexistent LE was 1:5.8 versus 1.3:1 in patients with psoriasis but without LE. Nearly 70% presented with LE preceding psoriasis. Psoriasis patients with LE had an earlier age of psoriasis onset (27.56 ± 11.51 versus 33.31 ± 16.94 years, P = 0.006), a higher rate of psoriatic arthropathy (26.5% versus 13.0%, P = 0.02), and a significantly greater impairment of quality of life (Dermatology Quality of Life Index >10; 57.6% versus 40.3%, P = 0.04) compared with psoriasis patients without LE. The majority (87.5%) had systemic LE. The incidences of lupus nephritis (72.7% versus 40%) and hematological abnormalities (50% versus 20%) were higher among patients with LE preceding psoriasis compared with those with psoriasis preceding LE. Antinuclear antibody and double-stranded DNA were positive in 59.4% and 28.1% of psoriasis patients with LE, respectively. Hydroxychloroquine triggered the onset of psoriasis in 7 (24.1%) patients. Patients with LE were more likely to receive systemic treatment for psoriasis compared with those without LE (30.3% versus 14.2%, P = 0.008).
CONCLUSIONS: Psoriasis patients with coexistent LE were uncommon, displayed a female preponderance, were more likely to have joint involvement, and had greater quality of life impairment than those without LE. LE preceded psoriasis in most of these patients, and systemic LE was the most common subtype.