scholarly journals Trial in Progress: A Real-World Cohort Study Reporting Patient Profiles, Clinical Outcomes, and Unmet Medical Need of Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Eculizumab in the Essen Center in Germany

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 38-39
Author(s):  
Katharina Versmold ◽  
Carina Raiser ◽  
Imi Faghmous ◽  
Pablo Katz ◽  
Aijing Shang ◽  
...  
Keyword(s):  
Clinical Outcomes ◽  
Clinical Data ◽  
Laboratory Data ◽  
Third Party ◽  
University Hospital ◽  
Red Blood Cell Transfusion ◽  
Clone Size ◽  
Medical Need ◽  
Source Data Verification

Background Paroxysmal nocturnal hemoglobinuria (PNH) is a life-threatening disease of dysregulated complement activation. It is a rare disease with an estimated incidence of 1 to 1.5 cases per million people globally. Eculizumab is a humanized monoclonal anti-complement component 5 antibody that was approved for the treatment of patients with PNH in the United States and European Union in 2007, yet unmet medical needs remain. Up to half of patients continue to require blood transfusions despite treatment with eculizumab, and hemolytic activity remains detectable in many patients (Brodsky et al. Blood. 2008). Eculizumab is not available in many countries. In places where treatment is approved, there are further impediments to access, such as cost of treatment, reimbursement issues, infrastructure limitations, and patient restrictions (Risitano et al. Am J Hematol. 2018; Risitano et al. Front Immunol. 2019). Data published on real-world outcomes of eculizumab are limited. Here we describe a study that will retroactively analyze data from patients with PNH treated with eculizumab at the Essen University Hospital in Germany. Study Design and Methods This retrospective, secondary data use, cohort study will include all patients at the Essen University Hospital who were diagnosed with PNH and treated with eculizumab prior to April 2018. Clinical data from medical records were entered into an electronic case report form (eCRF). Source data verification has been performed for all clinical data. Laboratory data were extracted directly from the hospital computer system. The Essen University hospital also checked and verified missing laboratory data. Patient-level data in the eCRF and laboratory data were fully anonymized. The primary objective of the study is to understand the remaining unmet medical need by describing the eculizumab dose and frequency of dose adjustment and describing the proportions of patients who experience intravascular and extravascular hemolysis while on treatment. The secondary objectives include explorations of the association between lactate dehydrogenase (LDH) and hemoglobin stabilization with clinical outcomes (eg, breakthrough hemolysis and the need for red blood cell transfusion), the association between PNH clone size and clinical outcomes and the risk of thrombosis, the changes in LDH and hemoglobin levels over time, the need for red blood cell transfusion during eculizumab treatment, and the proportion of eculizumab-treated patients with positive monospecific Coombs test results. In addition, opportunities to apply machine-learning methodologies to predict patients who may not respond to eculizumab will be explored. Many of the analyses will be descriptive. The associations between LDH and hemoglobin with clinical outcomes will be evaluated using rank correlation coefficients or logistic regression. Multivariable regression will be used to explore the prognostic value of clone size on clinical outcomes and thrombosis events. This retrospective study includes 85 patients with PNH with complete clinical and laboratory data (Table). The median age of the cohort was 38 years old, and the cohort was split evenly between men and women. Many patients received a diagnosis of PNH prior to the availability of eculizumab, as the year of diagnosis was 2010 or earlier for 53 patients (62%). The median years of follow-up from initiation of eculizumab was 4.7 years. Overall, 34% had aplastic anemia at diagnosis, and symptoms of fatigue, abdominal pain, and kidney failure were reported in 60%, 34%, and 15%, respectively. At data cutoff, 92% of patients were still alive. Summary The patient demographics in this study are comparable to other studies in PNH, suggesting a representative population. With median follow-up time of nearly 5 years, this study will allow for a long-term assessment of the patient experience with eculizumab. High-quality study data is ensured via full source data verification of clinical data and verification of missing laboratory data. These study results will help to address many research questions in PNH, identify the remaining unmet medical need, and also inform new drug development. Disclosures Versmold: F. Hoffmann-La Roche Ltd: Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Raiser:F. Hoffmann-La Roche Ltd: Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Faghmous:F. Hoffmann-La Roche Ltd: Ended employment in the past 24 months, Other: All authors received medical writing support for this abstract, furnished by Scott Battle, funded by F. Hoffmann-La Roche Ltd, Basel, Switzerland. ; Kite Pharma: Current Employment. Katz:F. Hoffmann-La Roche Ltd: Current Employment, Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Shang:F. Hoffmann-La Roche Ltd: Current Employment, Current equity holder in publicly-traded company, Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Xu:F. Hoffmann-La Roche Ltd: Current Employment, Other: All authors received support for third party writing assistance, furnished by Scott Battle, PhD, provided by F. Hoffmann-La Roche, Basel, Switzerland.. Röth:Novartis: Consultancy, Honoraria; Roche: Consultancy, Honoraria, Research Funding; Sanofi: Consultancy, Honoraria; Biocryst: Consultancy, Honoraria; Apellis: Consultancy, Honoraria; Alexion Pharmaceuticals Inc.: Consultancy, Honoraria, Research Funding.

scholarly journals Clinical Significance of Epidurography Contrast Patterns after Adhesiolysis during Lumbar Percutaneous Epidural Neuroplasty

2018 ◽  
Vol 2018 ◽  
pp. 1-8 ◽  
Author(s):  
Sang-Hyuk Park ◽  
Gyu Yeul Ji ◽  
Pyung Goo Cho ◽  
Dong Ah Shin ◽  
Young Sul Yoon ◽  
...  
Keyword(s):  
Clinical Outcomes ◽  
Short Form ◽  
University Hospital ◽  
Leg Pain ◽  
Vas Scores ◽  
The University ◽  
Procedural Outcomes ◽  
Short Form Health

Background. The correlation between epidurography contrast patterns and the clinical outcomes of percutaneous epidural neuroplasty (PEN) remains unclear. Objective. To analyze the correlation between postadhesiolysis epidurography contrast patterns and the clinical outcomes of patients who undergo lumbar PEN. Design. This study is a retrospective analysis of 78 consecutive patients who underwent lumbar PEN between April 2012 and March 2013. Setting. The analysis was done in the university hospital center. Method. The clinical outcomes of all patients were assessed before and 1, 3, 6, and 12 months after undergoing lumbar PEN. Specifically, the intensity of back and leg pain, quality of life, and procedural outcomes were evaluated using a visual analog scale (VAS), the Oswestry Disability Index (ODI), and the 12-Item Short-Form Health Survey (SF-12). Results. The VAS scores for back and leg pain, ODI score, and SF-12 score exhibited a significant improvement during the follow-up period (P<0.01 versus preprocedural scores). At most follow-up time points, patients exhibiting extraforaminal contrast distribution n=22 on postadhesiolysis epidurograms exhibited a similar improvement in VAS scores and a significantly better improvement in ODI and SF-12 scores compared with patients exhibiting intracanal contrast distribution n=56. Conclusion. Extraforaminal contrast distribution during lumbar PEN may be associated with better functional outcomes.

SP6.1.1 Cholecystectomy or expectant management after endoscopic sphincterotomy and bile duct stone removal?

2021 ◽  
Vol 108 (Supplement_7) ◽  
Author(s):  
Kate Toogood ◽  
Thomas Pike ◽  
Peter Coe ◽  
Simon Everett ◽  
Giles Toogood ◽  
...  
Keyword(s):  
Bile Duct ◽  
Clinical Outcomes ◽  
Expectant Management ◽  
University Hospital ◽  
Management Group ◽  
Nice Guidance ◽  
Cbd Stone ◽  
Definitive Management ◽  
Complex Surgery

Abstract Aims Following ERCP, NICE guidance suggests that surgically fit patients undergo cholecystectomy to prevent recurrence of choledocholithiasis and its complications. However, for many patients who are deemed unfit or who choose not to have surgery, ERCP is their definitive management. This study examines the clinical outcomes and costs of expectant management (EM) or cholecystectomy following ERCP. Methods All patients that underwent ERCP, sphincterotomy and common bile duct (CBD) clearance at St James’s University Hospital between January 2015 and December 2018 were identified from a prospectively maintained ERCP database. The clinical outcomes for patients that had undergone an ERCP, sphincterotomy and CBD clearance for gallstones were identified from their electronic patient record. A cost analysis for the complete patient pathway was performed. Results 820 patients underwent ERCP and CBD clearance for gallstones with a median 3.9 year follow up. 222 patients had undergone a cholecystectomy prior to ERCP and were excluded from analysis. 203 patients underwent planned cholecystectomy with 15% (31 patients) requiring complex surgery and 12% (24 patients) needing readmission. 395 patients received expectant management (EM). 9 (2.3%) patients returned with CBD stone symptoms, 6 (1.5%) went on to laparoscopic cholecystectomy (LC). The readmission rate in the EM group was 9%. The average cost per patient in the expectant management group was £7,487 and in the cholecystectomy group was £10,584. Conclusion The results from this study suggest that the need for cholecystectomy following ERCP is uncertain, with similar rates of biliary re-admissions in both groups.

Follow-up neutrophil-lymphocyte ratio after stroke thrombectomy is an independent biomarker of clinical outcome

2020 ◽  
pp. neurintsurg-2020-016342 ◽  
Author(s):  
Moustafa Aly ◽  
Ramez N Abdalla ◽  
Ayush Batra ◽  
Ali Shaibani ◽  
Michael C Hurley ◽  
...  
Keyword(s):  
Logistic Regression ◽  
Clinical Outcomes ◽  
Temporal Change ◽  
Laboratory Data ◽  
Multivariable Logistic Regression Model ◽  
Anterior Circulation ◽  
Neutrophil Lymphocyte Ratio ◽  
Lymphocyte Ratio ◽  
Multivariable Logistic Regression

BackgroundAdmission neutrophil-lymphocyte ratio (NLR) is significantly correlated to clinical outcomes in acute ischemic stroke (AIS). We investigated follow-up NLR and temporal changes in NLR after endovascular thrombectomy (EVT) with respect to successful revascularization, clinical outcomes, symptomatic intracranial hemorrhage (sICH) and mortality.MethodsRetrospective analysis of EVT for anterior circulation emergent LVO was performed with both admission (NLR1) and 3–7 day follow-up NLR (NLR2) laboratory data. Patient demographics, National Institutes of Health Stroke Scale (NIHSS) presentations, reperfusion efficacy (modified Thrombolysis in Cerebral Infarction (mTICI) score), sICH, and clinical outcomes (modified Rankin Scale (mRS)) at 90 days were studied. Univariate analyses correlated NLR1, NLR2, and temporal change in NLR (NLR2-NLR1) with successful reperfusion (mTICI ≥2b), favorable outcomes (mRS ≤2), sICH, and mortality. Multivariable logistic regression model evaluated the independent effects of NLR2 on favorable outcomes.Results142 AIS patients with median NIHSS 17 underwent EVT within 24 hours, and met NLR laboratory inclusion criteria. Lower follow-up NLR2 and less temporal change in NLR over 3–7 days, but not admission NLR1, inversely correlated with successful reperfusion (p<0.05) and favorable clinical outcomes (p<0.001). Higher follow-up NLR2 and greater temporal change in NLR was significantly associated with sICH and mortality (p≤0.05). In multivariable logistic regression, lower follow-up NLR2 remained a predictor of favorable outcomes (OR 0.785, p=0.001), independent of age or successful reperfusion.ConclusionsFollow-up NLR is a readily available and modifiable biomarker that correlates with the degree of reperfusion after mechanical stroke thrombectomy. Lower follow-up NLR2 at 3–7 days is associated with successful reperfusion and an independent predictor of favorable clinical outcomes, with reduced risk for sICH and mortality.

scholarly journals The Association Between Biomarkers and Clinical Outcomes in Novel Coronavirus (COVID-19) Pneumonia in a U.S. Cohort

2020 ◽  
Author(s):  
Shant Ayanian ◽  
Juan Reyes ◽  
Lei Lynn ◽  
Karolyn Teufel
Keyword(s):  
Clinical Outcomes ◽  
Laboratory Data ◽  
Clinical Deterioration ◽  
University Hospital ◽  
Risk Of Death ◽  
Markers Of Inflammation ◽  
Increased Risk ◽  
Icu Transfer ◽  
Novel Coronavirus ◽  
D Dimer

AbstractBackgroundThe global pandemic caused by COVID-19 remains poorly understood by clinicians. Identifying biologic markers associated with prognosis can help clinicians recognize disease severity.ObjectiveTo describe the association between D-dimer, CRP, IL-6, ferritin, LDH, and clinical outcomes in a cohort of COVID-19 patients treated on the inpatient medical service at a university hospital in Washington, DC.DesignIn this retrospective study, we included all adults admitted to the inpatient medicine service at George Washington University Hospital between March 12, 2020 and May 9, 2020 with laboratory confirmed COVID-19. Clinical and laboratory data were extracted from electronic medical records and compared between survivors not requiring ICU transfer, survivors requiring ICU transfer, survivors requiring intubation, and non-survivors.Key Results299 patients were included in our study, of whom 69 required transfer to the ICU, 39 required intubation, and 71 died. Threshold values for IL-6 (≥50 pg/mL), D-dimer (≥3 mcg/mL), ferritin (≥450 ng/mL), CRP (≥100 mg/L), and LDH (1,200 u/L) were found to be statistically significant and independently associated with higher odd of clinical deterioration and death. Hypertension, CVA and heart disease independently had an increased risk of all three outcomes, while CKD had only an increased risk of death. Patient co-morbidities had no effect on the different biomarkers’ significant association with poor patient clinical outcomes, except cancer.ConclusionLaboratory markers of inflammation and coagulopathy can help clinicians identify patients who are at high risk for clinical deterioration, independent of clinically significant medical comorbidities.

The Georges Pompidou University Hospital Clinical Data Warehouse: A 8-years follow-up experience

2017 ◽  
Vol 102 ◽  
pp. 21-28 ◽  
Author(s):  
Anne-Sophie Jannot ◽  
Eric Zapletal ◽  
Paul Avillach ◽  
Marie-France Mamzer ◽  
Anita Burgun ◽  
...  
Keyword(s):  
Data Warehouse ◽  
Clinical Data ◽  
University Hospital ◽  
Clinical Data Warehouse

scholarly journals Temporal change in Syndecan-1 as a therapeutic target and a biomarker for the severity classification of COVID-19

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Fumihiro Ogawa ◽  
Yasufumi Oi ◽  
Kento Nakajima ◽  
Reo Matsumura ◽  
Tomoki Nakagawa ◽  
...  
Keyword(s):  
Clinical Data ◽  
Therapeutic Target ◽  
Time Course ◽  
Temporal Change ◽  
Medical Center ◽  
Laboratory Data ◽  
University Hospital ◽  
Endothelial Glycocalyx ◽  
Severity Classification

Abstract Background Coronavirus disease 2019 (COVID-19) pneumonitis associated with severe respiratory failure is associated with high mortality. The pathogenesis of COVID-19 is associated with microembolism or microvascular endothelial injuries. Here, we report that syndecan-1 (SDC-1), a component of the endothelial glycocalyx, may be a biomarker of severity classification for COVID-19 related to endothelial injury. Methods and analysis We analyzed the data of COVID-19 patients for 1 year from February 2020 at Yokohama City University Hospital and Yokohama City University Medical Center Hospital. We selected COVID-19 patients who required admission care, including intensive care, and analyzed the classification of severe and critical COVID-19 retrospectively, using various clinical data and laboratory data with SDC-1 by ELISA. Results We analyzed clinical and laboratory data with SDC-1 in five severe COVID-19 and ten critical COVID-19 patients. In the two groups, their backgrounds were almost the same. In laboratory data, the LDH, CHE, and CRP levels showed significant differences in each group (P = 0.032, P < 0.0001, and P = 0.007, respectively) with no significant differences in coagulation-related factors (platelet, PT-INR, d-dimer, ISTH score; P = 0.200, 0.277, 0.655, and 0.36, respectively). For the clinical data, the SOFA score was significantly different from admission day to day 14 of admission (p < 0.0001). The SDC-1 levels of critical COVID-19 patients were significantly higher on admission day and all-time course compared with the levels of severe COVID-19 patients (P = 0.009 and P < 0.0001, respectively). Conclusions Temporal change of SDC-1 levels closely reflect the severity of COVID-19, therefore, SDC-1 may be a therapeutic target and a biomarker for the severity classification of Covid-19.

scholarly journals P0427IGA GLOMERULONEPHRITIS WITH MASSIVE PROTEINURIA- CASE SERIES WITH CLINICAL AND PATHOHISTOLOGICAL REVIEW

2020 ◽  
Vol 35 (Supplement_3) ◽  
Author(s):  
Miroslav Tisljar ◽  
Luka Toric ◽  
Dino Kasumović ◽  
Matija Crnogorac ◽  
Ivica Horvatić ◽  
...  
Keyword(s):  
Diabetes Mellitus ◽  
Renal Insufficiency ◽  
Renal Biopsy ◽  
Clinical Outcomes ◽  
Case Series ◽  
University Hospital ◽  
Iga Vasculitis ◽  
Average Percentage ◽  
Replacement Surgery

Abstract Background and Aims IgA nephropathy (IgAN) is the most common glomerulonephritis worldwide. The classic manifestation of IgAN is episodic hematuria and proteinuria. Nephrotic syndrome (NS) is not common in IgAN. It is reported to occur in 5-10% of patients with IgAN. Clinical presentation with massive proteinuria is exceptional. Method We retrospectively analyzed patients with pathohistologically proven IgAN who initially presented with 24h proteinuria greater than 10 gr. Here we presented their clinical, biochemical and pathohistological (electron microscopy-EM, was provided in all cases) characteristics with clinical outcomes. Data were taken from Renal biopsy Registry of Department of Nephrology Dubrava University hospital. Results All together 12 patients (10 M, 2 F) average age 53 (range 25-81) years were included. This represents 3,2% of all IgAN patients in our Renal biopsy Registry (presently there are more than 2100 patients in total, 366 with IgAN). Arterial hypertension had 10 out of 12 patients and three patients had diabetes mellitus type 2. Other significant comorbidities included lung adenocarcinoma in one patient, history of preeclampsia, mitral valve replacement surgery and liver cirrhosis, each in one patient. Average serum creatinine level at presentation was 223,9 (90-549) µmol/L and average 24h proteinuria was 14,6 (10,2-32) gr. All patients had haematuria. Renal insufficiency was found in 7 patients and additionally in two patients rapidoprogressive clinical pattern was recognized. Oxford classification was applied for all patients. In all except one, mesangial hypercellularity (M1) was found, endocapillary proliferation (E1) in 4 and cellular crescents in 5 patients (C1 in 4, C2 in 1 patient). The average percentage of globally sclerosing glomeruli was 32% (10-85%) and IFTA was 33% (10-60%), respectively. On EM diffuse podocyte foot processes effacement was documented only in two patients. In patients with diabetes mellitus there were not signs of diabetic nephropathy (i.e. thickened GBM on EM). Two patients who presented with purpuric rash and arthritis were considered to have IgA vasculitis (Henoch Schoenlein purpura). Considering treatment, glucocorticosteroids and RAAS blockators (apart from those who were dialysis dependent) were introduced. Five patients were also treated with cyclophosphamide. Average time of follow-up was 53 months (2-156). Regarding clinical outcomes, 4 patients achieved remission (2 partial and 2 complete). One of those experienced clinical relaps and underwent kidney rebiosy. In one patient stationary course and in another one progression of renal insufficiency were noted. Both patients diagnosed as IgA vasculitis were dialysis depended at presentation and during follow-up period unfortunately died. Two more patients died latter on as a consequence of a non-renal disorders. One more patient who developed ESRD was transplanted but afterwards he rejected the graft. One patient was lost from follow-up. Conclusion Proteinuria is well known independent progression risk factor in IgAN. In presented study we showed that almost one third of IgAN patients who manifested with massive proteinuria ended up in ESRD in relatively short follow-up period.

scholarly journals Annual Changes in Serum Syndecan-1 as a Therapeutic Target and a Biomarker for the Severity Classification of COVID-19

2021 ◽  
Author(s):  
Fumihiro Ogawa ◽  
Yasufumi Oi ◽  
Kento Nakajima ◽  
Reo Matsumura ◽  
Tomoki Nakagawa ◽  
...  
Keyword(s):  
Clinical Data ◽  
Therapeutic Target ◽  
Time Course ◽  
Medical Center ◽  
Laboratory Data ◽  
University Hospital ◽  
Endothelial Glycocalyx ◽  
Severity Classification ◽  
Annual Changes

Abstract Background: Coronavirus disease 2019 (COVID-19) pneumonitis associated with severe respiratory failure is associated with high mortality. The pathogenesis of COVID-19 is associated with microemboli or microvascular endothelial injuries. Here, we report that serum syndecan-1 (SDC-1), a component of the endothelial glycocalyx, may be a biomarker of severity classification for COVID-19 related to endothelial injury.Methods and analysis: We analyzed the data of COVID-19 patients for one year from February 2020 at Yokohama City University Hospital and Yokohama City University Medical Center Hospital. We selected COVID-19 patients who required admission care, including intensive care, and analyzed the classification of severe and critical COVID-19 retrospectively, using various clinical data and laboratory data with serum SDC-1 by ELISA. Results: We analyzed clinical and laboratory data with SDC-1 in five severe COVID-19 and ten critical COVID-19 patients. In the two groups, their backgrounds were almost the same. In laboratory data, the LDH, CHE, and CRP levels showed significant differences in each group (P=0.032, P <0.0001, and P = 0.007, respectively) with no significant differences in coagulation-related factors (platelet, PT-INR, D-dimer, ISTH score; P=0.200, 0.277, 0.655, and 0.36, respectively). For the clinical data, the SOFA score was significantly different from admission day to day 14 of admission (p<0.0001). The SDC-1 levels of critical COVID-19 patients were significantly higher on admission day and all-time course compared with the levels of severe COVID-19 patients (P=0.009 and P <0.0001, respectively).Conclusions: Annual changes in serum SDC-1 levels closely reflect the severity of COVID-19, therefore, serum SDC-1 may be a therapeutic target and a biomarker for the severity classification of Covid-19.

Sign in / Sign up

Export Citation Format

Share Document