scholarly journals PRO62 SYSTEMATIC LITERATURE REVIEW OF DISEASE BURDEN, COSTS AND MODELS IN TRANSTHYRETIN AMYLOID CARDIOMYOPATHY (ATTR-CM)

2019 ◽  
Vol 22 ◽  
pp. S852
Author(s):  
A. Granghaud ◽  
V. Famelart ◽  
A. Waeckel ◽  
C. Geffroy ◽  
O. Demarcq ◽  
...  
2018 ◽  
Vol 2018 ◽  
pp. 1-14 ◽  
Author(s):  
Vijayalaxmi V. Mogasale ◽  
Enusa Ramani ◽  
Vittal Mogasale ◽  
Ju Yeon Park ◽  
Thomas F. Wierzba

Background. Unsafe water is a well-known risk for typhoid fever, but a pooled estimate of the population-level risk of typhoid fever resulting from exposure to unsafe water has not been quantified. An accurate estimation of the risk from unsafe water will be useful in demarcating high-risk populations, modeling typhoid disease burden, and targeting prevention and control activities. Methods. We conducted a systematic literature review and meta-analysis of observational studies that measured the risk of typhoid fever associated with drinking unimproved water as per WHO-UNICEF’s definition or drinking microbiologically unsafe water. The mean value for the pooled odds ratio from case-control studies was calculated using a random effects model. In addition to unimproved water and unsafe water, we also listed categories of other risk factors from the selected studies. Results. The search of published studies from January 1, 1990, to December 31, 2013 in PubMed, Embase, and World Health Organization databases provided 779 publications, of which 12 case-control studies presented the odds of having typhoid fever for those exposed to unimproved or unsafe versus improved drinking water sources. The odds of typhoid fever among those exposed to unimproved or unsafe water ranged from 1.06 to 9.26 with case weighted mean of 2.44 (95% CI: 1.65–3.59). Besides water-related risk, the studies also identified other risk factors related to socioeconomic aspects, type of food consumption, knowledge and awareness about typhoid fever, and hygiene practices. Conclusions. In this meta-analysis, we have quantified the pooled risk of typhoid fever among people exposed to unimproved or unsafe water which is almost two and a half times more than people who were not exposed to unimproved or unsafe water. However, caution should be exercised in applying the findings from this study in modeling typhoid fever disease burden at country, regional, and global levels as improved water does not always equate to safe water.


2017 ◽  
Vol 33 (6) ◽  
pp. 1017-1031 ◽  
Author(s):  
Huamao Mark Lin ◽  
Xin Gao ◽  
Catherine E. Cooke ◽  
Deborah Berg ◽  
Richard Labotka ◽  
...  

Author(s):  
Rupert W. Jakes ◽  
Namhee Kwon ◽  
Beth Nordstrom ◽  
Rebecca Goulding ◽  
Kyle Fahrbach ◽  
...  

Abstract Eosinophilic granulomatosis with polyangiitis (EGPA) is a rare disease associated with vascular inflammation and multisystem organ damage. The literature reporting EGPA incidence or prevalence is limited. We performed a systematic literature review and meta-analysis to describe the incidence, prevalence, and disease burden associated with EGPA. Real-world, observational, English-language studies in MEDLINE, MEDLINE In-Process, and Embase up to 6 June, 2019, were included. A single investigator screened all identified titles/abstracts and extracted data; an additional, independent investigator repeated the screening and validated the extracted data. A random-effects meta-analysis was conducted to generate pooled estimates for EGPA incidence and prevalence. Data from 100 eligible publications were extracted (32 with incidence/prevalence data, 65 with morbidity/healthcare resource data; 3 with both types of data). Significant evidence of between-study heterogeneity for reported incidence (p = 0.0013–0.0016) and prevalence (p = 0.0001–0.0006) estimates was observed. Global and European pooled estimates (95% confidence interval) of EGPA incidence were 1.22 (0.93, 1.60) and 1.07 (0.94, 1.35) cases per million person-years, respectively; global and European pooled estimates (95% confidence interval) for EGPA prevalence were 15.27 (11.89, 19.61) and 12.13 (6.98, 21.06) cases per million individuals, respectively. The proportions of patients experiencing relapses, or who had nasal polyps or severe asthma, varied considerably across studies. EGPA healthcare resource use was high, with inpatient admissions and emergency department visits reported for 17–42% and 25–42% of patients, respectively. Our results indicate that although global and European EGPA incidence and prevalence is low, the associated disease burden is substantial. Key points• We performed a systematic literature review and meta-analysis of real-world, observational studies describing the incidence, prevalence, and disease burden associated with eosinophilic granulomatosis with polyangiitis (EGPA).• Based on meta-analysis data from 35 eligible studies reporting incidence and prevalence, the incidence and prevalence of EGPA were low (globally 1.22 cases per million person-years and 15.27 cases per million individuals, respectively).• Among the 49 studies with morbidity and/or healthcare resource data, most reported a large proportion of patients with EGPA relapses and comorbidities of nasal polyps and severe asthma.• Healthcare resource use was also high among patients with EGPA in these studies, with inpatient admissions and emergency department visits reported for 17–42% and 25–42% of patients, respectively. Taken together, these data indicate the substantial disease burden associated with EGPA.


2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Jean Yuh Tang ◽  
M. Peter Marinkovich ◽  
Eleanor Lucas ◽  
Emily Gorell ◽  
Albert Chiou ◽  
...  

Abstract Background/objective Recessive dystrophic epidermolysis bullosa (RDEB) is a genetic collagen disorder characterized by skin fragility leading to blistering, wounds, and scarring. There are currently no approved curative therapies. The objective of this manuscript is to provide a comprehensive literature review of the disease burden caused by RDEB. Methods A systematic literature review was conducted in MEDLINE and Embase in accordance with PRISMA guidelines. Observational and interventional studies on the economic, clinical, or humanistic burden of RDEB were included. Results Sixty-five studies were included in the review. Patients had considerable wound burden, with 60% reporting wounds covering more than 30% of their body. Increases in pain and itch were seen with larger wound size. Chronic wounds were larger and more painful than recurrent wounds. Commonly reported symptoms and complications included lesions and blistering, anemia, nail dystrophy and loss, milia, infections, musculoskeletal contractures, strictures or stenoses, constipation, malnutrition/nutritional problems, pseudosyndactyly, ocular manifestations, and dental caries. Many patients underwent esophageal dilation (29–74%; median dilations, 2–6) and gastrostomy tube placement (8–58%). In the severely affected population, risk of squamous cell carcinoma (SCC) was 76% and mortality from SCC reached 84% by age 40. Patients with RDEB experienced worsened quality of life (QOL), decreased functioning and social activities, and increased pain and itch when compared to other EB subtypes, other skin diseases, and the general population. Families of patients reported experiencing high rates of burden including financial burden (50–54%) and negative impact on private life (79%). Direct medical costs were high, though reported in few studies; annual payer-borne total medical costs in Ireland were $84,534 and annual patient-borne medical costs in Korea were $7392. Estimated annual US costs for wound dressings ranged from $4000 to $245,000. Patients spent considerable time changing dressings: often daily (13–54% of patients) with up to three hours per change (15–40%). Conclusion Patients with RDEB and their families/caregivers experience significant economic, humanistic, and clinical burden. Further research is needed to better understand the costs of disease, how the burden of disease changes over the patient lifetime and to better characterize QOL impact, and how RDEB compares with other chronic, debilitating disorders.


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