scholarly journals MO273A NOVEL APPROACH TO ASSESS THE DEMOGRAPHIC AND CLINICAL CHARACTERISTICS OF IMMUNOGLOBULIN A NEPHROPATHY (IGAN) PATIENTS IN A US REAL-WORLD SETTING

2021 ◽  
Vol 36 (Supplement_1) ◽  
Author(s):  
Raymond Przybysz ◽  
Rina Mehta ◽  
Gisela Rovira Tomas ◽  
Carolina Aldworth ◽  
Jim Doherty ◽  
...  
Keyword(s):  
Kidney Function ◽  
Clinical Characteristics ◽  
Index Date ◽  
Immunoglobulin A ◽  
Significant Proportion ◽  
Immunoglobulin A Nephropathy ◽  
Targeted Interventions ◽  
The Us ◽  
The Mean ◽  
Severe Deterioration

Abstract Background and Aims Immunoglobulin A nephropathy (IgAN) is a chronic glomerular disease that affects approximately 100,000-200,000 people in the US. Approximately 15-40% of IgAN patients will eventually progress to end stage kidney disease (ESKD) within 10-20 years of diagnosis, and there is currently no targeted therapy for this disease. Decreased kidney function, persistent proteinuria and hypertension are some of the clinical manifestations of IgAN, and with demographic aspects (e.g. ethnicity) are considered as predictors of disease progression. The aim of this study is to better understand the demographic and clinical characteristics of IgAN patients in the US identified via physician notes. Method This is a descriptive, retrospective study of adult (≥ 18 years) IgAN patients in Optum® Electronic Health Records (EHRs), between January 2007 and December 2019. Optum® EHRs contain de-identified clinical and medical administrative data from 96 million people in 50 states that come from more than 140,000 providers at 740 hospitals and over 7,000 clinics. Identification of IgAN patients is challenging because there are no specific ICD codes for this disease so we used natural language processing of physician notes and chose patients with at least two IgAN records with the first one considered to be the index date, and no negative mention, as well as a biopsy procedure. Here, we present the baseline demographic and clinical characteristics of the identified patients up to 12 months before and at the index date. Results A total of 1803 patients with a biopsy record (22% of all patients with at least two IgAN records in their EHRs in our study) were included in this analysis; results are presented in Table 1. The mean age was 48 years, and the majority of patients were male (60.9%) and white (75.7%). Proteinuria levels of ≥1 g/day were found in 34% of patients. The mean eGFR was 45 ml/min/1.73m2 and 21.6% of patients had severe deterioration of kidney function (eGFR <15). The mean creatinine level was 3 mg/dL. Pain, edema and fatigue/tiredness were reported in 39.6%, 18.1% and 13.4% of patients, respectively. Hypertension was reported in 73% of patients. Based on the ICD 9 (585.6) and ICD-10 (N18.6) codes for diagnosis, 17.5% of patients had ESKD in our study. Conclusion In our cohort, a significant proportion of patients were found to have high proteinuria levels and severe deterioration of kidney function or ESKD. Moreover, edema and fatigue were recorded for a noticeable proportion of patients. In addition to the commonly reported symptoms, our study also found that pain was reported in a high proportion of patients. These findings highlight the clinical and symptom burden to patients with IgAN, suggesting that future targeted interventions are needed to reduce the burden and delay the progression of this disease.

PP268 Eliciting Meaningful Patient Preferences In Rare Diseases – Swing Weighting With Immunoglobulin A Nephropathy Patients In The United States And China

2020 ◽  
Vol 36 (S1) ◽  
pp. 24-24
Author(s):  
Kevin Marsh ◽  
Nancy Zaour ◽  
Kerrie-Anne Ho ◽  
Ankit Joshi ◽  
Rachel Lo ◽  
...  
Keyword(s):  
United States ◽  
Patient Preferences ◽  
Qualitative Data ◽  
Immunoglobulin A ◽  
The United States ◽  
Small Sample ◽  
Immunoglobulin A Nephropathy ◽  
Sample Sizes ◽  
Trade Offs ◽  
The Us

IntroductionReimbursement agencies are increasingly using patient preference data to evaluate health technologies. Discrete choice experiments (DCE) are commonly used to elicit patient preferences, but they require large sample sizes to obtain meaningful results. For this reason, it is often not possible to use DCE to elicit patient preferences in rare diseases. This study assessed a swing weighting method for eliciting preferences from a small sample: patients with immunoglobulin A nephropathy (IgAN) in the United States (US) and China.MethodsAttributes and levels were selected based on a review of clinical studies and qualitative research on patients. Computer-assisted, interview-based swing weighting exercises were piloted in a focus group with five participants each from the US and China. Preferences were then elicited in interviews with twenty-five patients in the US and fifteen patients in China. Consistency tests were used to assess internal validity. Qualitative data were collected on the reasons for patients’ preferences.ResultsPreference consistency: The weights for one attribute were elicited twice. The difference between initial and consistency test weights was not statistically significant (p < 0.1), although this may partly reflect the small sample sizes. Trade-offs: Qualitative data were used to demonstrate the validity of interpreting participants’ ratings as trade-offs. Using the partial value function for end-stage renal disease as an example, qualitative data demonstrated that patients were able to provide face-valid reasons for different shaped, non-linear preference functions. Robustness of treatment evaluation: Three hypothetical treatment profiles (using the attribute swings) were constructed. Preferences for these treatment profiles were robust to variations in patients’ preferences; all patients preferred one specific profile. This finding was not sensitive to changes in weights.ConclusionsThis study supports the feasibility of collecting valid and robust preference data from small groups of patients using swing weighting. Further work could be done to test the performance of swing weighting in larger sample sizes.

Atlantal hemi-rings and craniocervical instability: identification, clinical characteristics, and management

2011 ◽  
Vol 8 (4) ◽  
pp. 357-362 ◽  
Author(s):  
Douglas L. Brockmeyer ◽  
Meghan M. Brockmeyer ◽  
Taryn Bragg
Keyword(s):  
Clinical Characteristics ◽  
Lateral Displacement ◽  
Significant Proportion ◽  
Neurological Status ◽  
Dynamic Imaging ◽  
Patient Age ◽  
Patient Âgé ◽  
Craniocervical Instability ◽  
Mass Displacement ◽  
The Mean

Object Congenital craniovertebral anomalies are relatively common, but anomalies leading to overt craniocervical instability may be difficult to recognize and treat. The authors present a series of patients with atlantal hemi-rings, a disorder resulting in congenital craniovertebral instability. Presentation, treatment, imaging, and follow-up data obtained in patients with atlantal hemi-rings were assessed to identify factors relevant to craniocervical instability. Methods Nineteen patients were identified with atlantal hemi-rings, defined as a bony discontinuity of the C-1 ring in conjunction with lateral displacement of the C-1 lateral masses (as seen on coronal CT scans). Clinical and radiological characteristics were analyzed, including patient age at presentation, extent of occipitocervical motion, amount of C-1 lateral mass displacement, associated craniocervical anomalies, integrity of the transverse ligament, and neurological status. Results The mean patient age at presentation was 22 months (range birth to 9 years). The mean amount of occipitocervical translation seen on dynamic imaging was 9 mm (range 2–20 mm). Four patients required occipitocervical fusion at presentation. The remaining 15 patients were monitored for a mean of 20 months, and 9 ultimately underwent fusion. Surgery was also recommended for 4 of the remaining 6 children. Conclusions This report describes the radiological and clinical characteristics of patients with atlantal hemirings and craniocervical instability. The authors believe that this anomaly is the underlying cause of progressive instability in a significant proportion of patients with craniocervical abnormalities. The presence of atlantal hemi-rings should prompt immediate and thorough neurosurgical evaluation.

scholarly journals Real-World Treatment Patterns and Outcomes in Patients with Hemophagocytic Lymphohistiocytosis (HLH) and Other Clinical Conditions Treated with Emapalumab: The Real-HLH Study Design

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4991-4991
Author(s):  
Carl E. Allen ◽  
Jennifer Leiding ◽  
Shanmuganathan Chandrakasan ◽  
Kelly J. Walkovich ◽  
Abiola Oladapo ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Real World ◽  
Clinical Characteristics ◽  
Index Date ◽  
Treatment Patterns ◽  
Patient Identification ◽  
Advisory Committees ◽  
The Real ◽  
The Us ◽  
Clinical Conditions

Abstract Introduction: Hemophagocytic lymphohistiocytosis (HLH) is a rare, life-threatening hyperinflammatory syndrome characterized by overactivation of the immune system due to systemic release of proinflammatory cytokines, especially of interferon gamma (IFNγ), and persistent activation of macrophages/histiocytes and T cells. Emapalumab, a fully human, anti-IFNγ monoclonal antibody that binds to both free and receptor-bound IFNy, neutralizing its biologic activity, was approved by the Food and Drug Administration (FDA) in 2018 for the treatment of adult and pediatric patients with primary HLH with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. The safety and efficacy of emapalumab was based on results from a pivotal phase 2/3 trial which reported a 63% overall response in patients treated with emapalumab. Since approval, no study has evaluated the use of emapalumab in a larger cohort of patients in the real-world clinical setting. Aim: To assess real-world treatment patterns and outcomes, clinical and demographic characteristics among patients treated with emapalumab. Method: This retrospective, non-interventional, observational medical chart review study will include patients treated across treatment centers in the US with emapalumab in a non-clinical trial setting. The study aims to include more than 100 patients who have been treated with at least one dose of emapalumab between November 20, 2018 and December 31, 2020 (patient identification period). The date that the patient initiates treatment with emapalumab within the patient identification period is defined as the index date. The post-index date is defined as the period from the index date through to the study end date (June 30, 2021), end of data availability for the patient, or date of death, whichever occurs first. Patients will be classified into three groups (i.e. primary HLH, secondary HLH, or non-HLH) based on the information obtained from their charts, the HLH 2004 diagnostic criteria, and adjudication by the REAL-HLH Steering Committee. Results: The primary objective of the study is to describe treatment patterns in patients with HLH treated with emapalumab in a real-world clinical setting, including emapalumab dose, treatment duration, and reasons for initiating or discontinuing treatment. The secondary objectives are to describe demographic and clinical characteristics of patients with HLH treated with emapalumab, and their outcomes. The exploratory objectives include detailing the demographic, clinical characteristics, treatment patterns, and outcomes of patients with non-HLH clinical conditions treated with emapalumab (Table 1). Demographics and clinical characteristics will be analyzed at the time of diagnosis. Treatment patterns and outcomes will be analyzed during the post-index date. Conclusion: The study aims to assess treatment patterns and outcomes, clinical and demographic characteristics among patients treated with emapalumab, a novel IFNγ blocking agent, in real-world clinical settings in the US. Figure 1 Figure 1. Disclosures Allen: Sobi: Consultancy. Leiding: Sobi: Consultancy. Chandrakasan: Sobi: Consultancy. Walkovich: X4 Pharmaceuticals: Other: Local PI for clinical trial involving mavorixafor and patients with neutropenia; Swedish Orphan Biovitrum AB (Sobi): Consultancy, Honoraria; Pharming: Honoraria, Membership on an entity's Board of Directors or advisory committees; Horizon Pharmaceuticals: Honoraria, Membership on an entity's Board of Directors or advisory committees. Oladapo: Sobi: Current Employment. Yee: Sobi: Current Employment. Pednekar: Sobi: Consultancy; PRECISIONheor: Current Employment. Raza: Sobi: Consultancy; PRECISIONheor: Current Employment. Jordan: Sobi: Consultancy.

scholarly journals Epidemiologic and Clinical Characteristics of Thalassemia (Thal) Intermedia (TI) in the United States

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 3279-3279
Author(s):  
Elliott P. Vichinsky ◽  
Janet Kwiatkowski ◽  
Patricia J. Giardina ◽  
Carole Paley ◽  
Francis Vekeman ◽  
...  
Keyword(s):  
Serum Ferritin ◽  
Research Funding ◽  
Clinical Characteristics ◽  
Index Date ◽  
Growth Failure ◽  
The United States ◽  
Compound Heterozygous ◽  
Foreign Born ◽  
Liver Iron ◽  
The Us

Abstract Introduction TI is increasingly prevalent in the US due to changing immigration patterns. It is underdiagnosed, leading to inadequate or delayed management. This study reviewed the prevalence, epidemiology, and clinical characteristics of TI in patients (pts) in the US. Methods Medical records from 1/1997- 4/2014 at 4 US hematology centers were reviewed. Index date was the 1st TI visit at a center on or after 1/1/1997. Eligible pts had a TI diagnosis (≤8 mean packed red blood cell transfusions per year (yr) over a ≥3-y period after index date) and ≥12 months of follow-up. Data spanned from index date to death or last record. Descriptive analyses of demographic and clinical data were done by TI subtype. Results Of 138 pts enrolled, 84 had α-thal, 39 had β-thal, and 15 had E/β-thal. 74% of α-thal pts had deletional (del) Hb H, and 26% had non-deletional (ndel) Hb H. 59% of β-thal pts had homozygous or compound heterozygous β-globin mutations (8% with α deletion, 51% without), 20% had a single β mutation with α-gene triplication, 13% had autosomal dominant β thal, and 8% had other β-globin mutations. Of the E/β-thal pts, 80% had E/β0 and 20% had E/β+. Median age at index date was 2.3 yr (1.64 del; 6.1 ndel) in the α-thal group, 9.2 yr in the β-thal group, and 2.2 yr in the E/β-thal group. Most α-thal (77%) and E/β-thal (87%) pts were Asian; most β-thal pts were White (46%) or African-American (36%). Most α-thal (56%) and E/β-thal (53%) pts were of Southeast Asian origin; most β-thal pts were of Mediterranean (31%) or African (21%) origin. 21%, 10%, and 20% of α, β, and E/β-thal pts, respectively, were foreign-born, and 5%, 3% and 7%, respectively, were transfused outside of the US. Observation length was similar across subtypes (median: 5.3 yr). Clinical comorbidities are shown in Table 1. 22% of pts received ≥1 transfusion, while 7% of pts received ≥8 transfusions in any 1 yr to treat anemia, acute hemolysis, or cardiac abnormality; increased transfusions were initiated due to growth failure, acute hemolysis, or unspecified reasons. β-thal pts had a higher mean number of transfusions per pt per yr (PPPY) (α: 0.4 (0.0 del; 1.5 ndel), β: 0.9, E/β: 0.2) and higher mean serum ferritin (ng/mL) (204.3; 511.7; 362.4), and more often had iron chelation therapy (ICT) (11%; 28%; 7%). There was an association between higher serum ferritin, more frequent transfusions, and older age. In pts <10 yr, mean number of transfusions PPPY for regularly and ever transfused was 4.0 and 2.1, and serum ferritin for regularly, ever, and never transfused was 723.7, 438.0, and 146.4 ng/mL. In pts >18 yr, these values were 9.8 and 4.6 transfusions PPPY and 1166.8, 1042.2, and 521.4 ng/mL. An association also existed between ICT and higher mean serum ferritin (ICT: 769.9; no ICT: 463.7 ng/mL). 22% of pts had ≥1 liver iron test, and 18% had ≥1 cardiac iron test. Tested pts were older than those not tested (median yr, liver iron: 26.1 vs 7.4; cardiac: 20.1 vs 4.6). Among tested pts, 78% (25/32) had abnormal liver iron results. Median (range) LIC based on R2 or SQUID was 10.8 (2.5-18.2) mg/g dw, with corresponding within-12-month serum ferritin of 494 (127-1770) ng/mL. 49% (17/35) of pts tested had abnormal cardiac results based on electrocardiogram or echocardiogram. Table 1. Clinical comorbidities All α-thal α-thal del α-thal ndel β-thal E/β-thal N=138 N=84 N=62 N=22 N=39 N=15 Splenomegaly 54% 55% 39% 100% 51% 60% Extramedullary hematopoiesis 28% 24% 13% 55% 36% 33% Growth retardation 21% 23% 18% 36% 13% 33% Hepatomegaly 21% 24% 13% 55% 15% 20% Infections needing hospitalization/IV antibiotics 21% 25% 19% 41% 15% 13% Hypoparathyroidism/hypocalcemia 15% 17% 8% 41% 10% 20% Osteopenia/osteoporosis 13% 11% 5% 27% 21% 7% Bone deformities 9% 6% 5% 9% 21% 0% Splenectomy and/or cholecystectomy 9% 5% 2% 14% 21% 0% Cardiomegaly 4% 6% 2% 18% 0% 7% Conclusion TI in the US affects a diverse population. Our data showed a higher prevalence in African-Americans than previously documented. Rates of comorbidity and transfusion frequency increased with age. 18% and 4% of pts were born and transfused outside of the US, potentially leading to additional transfusion-related morbidity. Consistent with extant data, serum ferritin in TI often underestimated actual LIC, rendering more pts potentially eligible for ICT than observed. Morbidities observed in this study underscore the need for better and earlier diagnosis, substantiating the need for nationwide TI screening/surveillance to optimize management. Disclosures Vichinsky: Novartis: Research Funding. Kwiatkowski:Novartis: Research Funding; Sideris Pharmaceuticals: Consultancy; Shire Pharmaceuticals and Sideris Pharmaceuticals: Consultancy; ISIS: Membership on an entity's Board of Directors or advisory committees. Paley:Novartis: Employment. Vekeman:Novartis: Research Funding. Cheng:Novartis: Research Funding. Damron:Novartis: Research Funding. McCormick:Novartis: Research Funding. Sasane:Novartis: Employment. Qiu:Novartis: Employment. Duh:Novartis: Research Funding. Thompson:Bluebird bio: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; Mast: Research Funding; Apopharma: Consultancy; Baxter: Consultancy, Research Funding.

scholarly journals Deliberate Self-Poisoning Presenting to an Emergency Medicine Network in South-East Melbourne: A Descriptive Study

2014 ◽  
Vol 2014 ◽  
pp. 1-7 ◽  
Author(s):  
Asheq Rahman ◽  
Catherine Martin ◽  
Andis Graudins ◽  
Rose Chapman
Keyword(s):  
Emergency Department ◽  
Emergency Medicine ◽  
Length Of Stay ◽  
Clinical Audit ◽  
Significant Proportion ◽  
Patient Characteristics ◽  
Descriptive Study ◽  
Short Stay ◽  
Targeted Interventions ◽  
The Mean

Background. Deliberate self-poisoning (DSP) comprises a small but significant proportion of presentations to the emergency department (ED). However, the prevalence and patient characteristics of self-poisoning attendances to EDs in Victoria have not been recently characterised.Aim. To identify and compare the characteristics of adult patients presenting to the three EDs of Monash Health following DSP.Methods. Retrospective clinical audit of adult DSP attendances between 1st July 2009 and 30th June 2012.Results. A total of 3558 cases over three years were identified fulfilling the search criteria. The mean age of patients was 36.3 years with the largest numbers aged between 18 and 30 (38%). About 30% of patients were born overseas. Forty-eight percent were discharged home, 15% were admitted to ED short stay units, and 5% required ICU admission. The median ED length of stay was 359 minutes (IQR 231–607). The most frequently reported substances in DSP were benzodiazepines (36.6%), paracetamol (22.2%), and antipsychotics (12.1%). Exposure to more than one substance for the episode of DSP was common (47%).Conclusion. This information may help identify the trends in poisoning substances used for DSP in Victoria, which in turn may provide clinicians with information to provide more focused and targeted interventions.

scholarly journals Gut microbiota in Immunoglobulin A Nephropathy: a Malaysian Perspective

2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Agni Nhirmal Kumar Sugurmar ◽  
Rozita Mohd ◽  
Shamsul Azhar Shah ◽  
Hui-min Neoh ◽  
Rizna Abdul Cader
Keyword(s):  
Gut Microbiota ◽  
Gut Microbiome ◽  
Immunoglobulin A ◽  
Alpha Diversity ◽  
Rrna Gene ◽  
Healthy Controls ◽  
Control Cohort ◽  
Immunoglobulin A Nephropathy ◽  
Ckd Stage ◽  
The Mean

Abstract Introduction The alteration of the gut microbiome in the gut-kidney axis has been associated with a pro-inflammatory state and chronic kidney disease (CKD). A small-scaled Italian study has shown an association between the gut microbiome and Immunoglobulin A Nephropathy (IgAN). However, there is no data on gut microbiota in IgAN in the Asian population. This study compares the gut microbial abundance and diversity between healthy volunteers and Malaysian IgAN cohort. Methods A comparative cross-sectional study was conducted involving biopsy-proven IgAN patients in clinical remission with matched controls in a Malaysian tertiary centre. Demographic data, routine blood and urine results were recorded. Stool samples were collected and their DNA was extracted by 16S rRNA gene sequencing to profile their gut microbiota. Results Thirty-six IgAN patients (13 male; 23 female) with the mean age of 45.5 ± 13.4 years and median estimated glomerular filtration rate (eGFR) of 79.0 (62.1–92.2) mls/min/1.73m2 with median remission of 7 years were analysed and compared with 12 healthy controls (4 male; 8 female) with the mean age of 46.5 ± 13.5 years and eGFR of 86.5 (74.2–93.7) mls/min/1.73m2. Other demographic and laboratory parameters such as gender, ethnicity, body mass index (BMI), haemoglobin, serum urea and serum albumin were comparable between the two groups. There were no significant differences seen in the Operational Taxonomic Unit (OTU) and alpha diversity (Shannon index) between IgAN and healthy controls. Alpha diversity increased with increasing CKD stage (p = 0.025). Firmicutes/Bacteroidetes (F/B) ratio was low in both IgAN and healthy cohort. Fusobacteria phylum was significantly increased (p = 0.005) whereas Euryarchaoeota phylum was reduced (p = 0.016) in the IgAN group as compared to the control cohort. Conclusion Although we found no differences in OTU and alpha diversity between IgAN in remission and control cohort, there were some differences between the two groups at phylum level.

Relation of Statistically Significant, Abnormal, and Typical WAIS-R VIQ-PIQ Discrepancies to Full Scale IQs

2000 ◽  
Vol 16 (2) ◽  
pp. 107-114 ◽  
Author(s):  
Louis M. Hsu ◽  
Judy Hayman ◽  
Judith Koch ◽  
Debbie Mandell
Keyword(s):  
Graphical Method ◽  
The United States ◽  
Full Scale ◽  
True Score ◽  
Absolute Value ◽  
Normative Population ◽  
The Us ◽  
The Mean ◽  
And Performance ◽  
Quadratic Models

Summary: In the United States' normative population for the WAIS-R, differences (Ds) between persons' verbal and performance IQs (VIQs and PIQs) tend to increase with an increase in full scale IQs (FSIQs). This suggests that norm-referenced interpretations of Ds should take FSIQs into account. Two new graphs are presented to facilitate this type of interpretation. One of these graphs estimates the mean of absolute values of D (called typical D) at each FSIQ level of the US normative population. The other graph estimates the absolute value of D that is exceeded only 5% of the time (called abnormal D) at each FSIQ level of this population. A graph for the identification of conventional “statistically significant Ds” (also called “reliable Ds”) is also presented. A reliable D is defined in the context of classical true score theory as an absolute D that is unlikely (p < .05) to be exceeded by a person whose true VIQ and PIQ are equal. As conventionally defined reliable Ds do not depend on the FSIQ. The graphs of typical and abnormal Ds are based on quadratic models of the relation of sizes of Ds to FSIQs. These models are generalizations of models described in Hsu (1996) . The new graphical method of identifying Abnormal Ds is compared to the conventional Payne-Jones method of identifying these Ds. Implications of the three juxtaposed graphs for the interpretation of VIQ-PIQ differences are discussed.

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