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Facilitating Evaluation of Hemolytic Uremic Syndrome Long-Term Health Outcomes Through Social Media Support Groups.

ABSTRACT: Individual burden and cost of hemolytic uremic syndrome (HUS)-a medical condition characterized by acute kidney failure-can be substantial when accounting for long-term health outcomes (LTHOs). Because of the low incidence of HUS, evaluation of associated LTHOs is often restricted to physician and outbreak cohorts, both of which may not be representative of all HUS cases. This exploratory study recruited participants from private social media support groups for families of HUS cases to identify potential LTHOs and costs of HUS that are not currently measured. Additionally, this study sought to identify case characteristics that may confound or modify these LTHOs and costs of HUS. Respondents self-selected to complete an online cross-sectional survey on acute and chronic illness history, treatments, and public health follow-up for HUS cases. Posttraumatic stress among respondents (typically case parents) was also evaluated. Responses were received for 74 HUS cases from 71 families representing all geographic regions, and levels of urbanicity within the US self-reported symptoms were typical for HUS, while 35.1% of cases reported antibiotic treatment at any point during the acute illness. Hospital transfers were reported by 71.6% of cases introducing possible delays to care. More than 70% of cases reported experiencing at least one LTHO, with 45% of cases reporting renal sequelae. Posttraumatic stress symptoms were frequently reported by respondents indirectly affected by HUS. Potentially large economic costs that are not addressed in existing analyses were identified including both financial and more general welfare losses (lost utility). While biases in the study design limit the generalizability of results to all HUS cases, this study provides new insights into unmeasured LTHOs and costs associated with HUS. These results suggest that robustly designed cohort studies on HUS should include measures of psychosocial impacts on both the affected individual and their family members.

SUBMITTER: Beczkiewicz ATE

PROVIDER: S-EPMC7719744 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature

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Facilitating Evaluation of Hemolytic Uremic Syndrome Long-Term Health Outcomes Through Social Media Support Groups.

Beczkiewicz Aaron T E ATE Scharff Robert L RL Kowalcyk Barbara B BB

Frontiers in public health 20201123

Individual burden and cost of hemolytic uremic syndrome (HUS)-a medical condition characterized by acute kidney failure-can be substantial when accounting for long-term health outcomes (LTHOs). Because of the low incidence of HUS, evaluation of associated LTHOs is often restricted to physician and outbreak cohorts, both of which may not be representative of all HUS cases. This exploratory study recruited participants from private social media support groups for families of HUS cases to identify ...[more]

PMID: 33330302

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Project description:BackgroundThere are limited long-term outcome data in eculizumab-treated patients with atypical hemolytic uremic syndrome (aHUS). We report final results from the largest prospective, observational, multicenter study of patients with aHUS treated with eculizumab.MethodsPatients with aHUS who participated in any of five parent eculizumab trials and received at least one eculizumab infusion were eligible for enrollment in a long-term follow-up study. Rates of thrombotic microangiopathy (TMA) manifestations off versus on eculizumab were evaluated. Additional endpoints included change from baseline estimated glomerular filtration rate (eGFR), long-term renal outcomes, and serious targeted treatment-emergent adverse events.ResultsAmong 93 patients (0-80 years of age), 51 (55%) remained on eculizumab and 42 (45%) discontinued; for those who discontinued, 21 (50%) reinitiated therapy. Patients who reinitiated eculizumab had similar baseline clinical characteristics to patients who remained on eculizumab, with higher likelihood of genetic/autoimmune complement abnormalities, more prior TMAs, and longer disease course versus those who did not reinitiate. Mean eGFR improved rapidly and remained stable for up to 6 years on eculizumab. In patients who discontinued, there was a trend toward decreasing renal function over time from discontinuation. Additionally, off-treatment TMA manifestation rates were higher in those aged < 18 years at diagnosis, with identified genetic/autoimmune complement abnormalities, or history of multiple TMAs prior to eculizumab initiation. The safety profile was consistent with previous studies. Three definite and one possible meningococcal infections related to eculizumab were reported and resolved with treatment. Three deaths unrelated to eculizumab were reported.ConclusionsThe current study confirms the efficacy and safety of eculizumab in aHUS, particularly with regard to long-term renal function and TMA events. Pediatric age at disease onset and presence of genetic or autoimmune complement abnormalities are risk factors for TMA events off treatment. Overall, patients who discontinue eculizumab may be at risk for additional TMA manifestations and renal function decreases. Discontinuation of eculizumab, with careful monitoring, is an option in select patients with consideration of patient preference, organ function normalization, and risk factors for relapse, including mutational analysis, age of onset, and history of multiple TMA episodes.Trial registrationClinicalTrials.gov NCT01522170 , January 31, 2012.

Project description:The hemolytic-uremic syndrome consists of the triad of microangiopathic hemolytic anemia, thrombocytopenia, and renal failure. The common form of the syndrome is triggered by infection with Shiga toxin-producing bacteria and has a favorable outcome. The less common form of the syndrome, called atypical hemolytic-uremic syndrome, accounts for about 10% of cases, and patients with this form of the syndrome have a poor prognosis. Approximately half of the patients with atypical hemolytic-uremic syndrome have mutations in genes that regulate the complement system. Genetic factors in the remaining cases are unknown. We studied the role of thrombomodulin, an endothelial glycoprotein with anticoagulant, antiinflammatory, and cytoprotective properties, in atypical hemolytic-uremic syndrome.We sequenced the entire thrombomodulin gene (THBD) in 152 patients with atypical hemolytic-uremic syndrome and in 380 controls. Using purified proteins and cell-expression systems, we investigated whether thrombomodulin regulates the complement system, and we characterized the mechanisms. We evaluated the effects of thrombomodulin missense mutations associated with atypical hemolytic-uremic syndrome on complement activation by expressing thrombomodulin variants in cultured cells.Of 152 patients with atypical hemolytic-uremic syndrome, 7 unrelated patients had six different heterozygous missense THBD mutations. In vitro, thrombomodulin binds to C3b and factor H (CFH) and negatively regulates complement by accelerating factor I-mediated inactivation of C3b in the presence of cofactors, CFH or C4b binding protein. By promoting activation of the plasma procarboxypeptidase B, thrombomodulin also accelerates the inactivation of anaphylatoxins C3a and C5a. Cultured cells expressing thrombomodulin variants associated with atypical hemolytic-uremic syndrome had diminished capacity to inactivate C3b and to activate procarboxypeptidase B and were thus less protected from activated complement.Mutations that impair the function of thrombomodulin occur in about 5% of patients with atypical hemolytic-uremic syndrome.

Dataset Information

Facilitating Evaluation of Hemolytic Uremic Syndrome Long-Term Health Outcomes Through Social Media Support Groups.

Publications

Facilitating Evaluation of Hemolytic Uremic Syndrome Long-Term Health Outcomes Through Social Media Support Groups.

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