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Rare Diseases

Avacopan for ANCA Vasculitis Goes in Front of the FDA

MedPage Today
May 07, 2021

An FDA advisory panel will consider whether to recommend approval of an oral C5a inhibitor for antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis, with agency staff raising questions over the "clinical meaningfulness" of key study data.

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RheumNow Podcast – Falling or Falling in Love?

May 06, 2021

Dr. Jack Cush reviews the best journal articles from the past week on

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Childhood Lupus May Hamper Adult Careers

May 04, 2021

Long-term outcomes of kids with childhood-onset systemic lupus erythematosus (cSLE) are uncommon, but this cSLE cohort analysis suggests that SLE significantly impacts their future education, vocation, and employment as they reach adulthood.

A cohort of 106 cSLE (93% female, 73% White

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Uric Acid: A Major Player in the Metabolic Syndrome

May 03, 2021

An open source, free read from the European Journal of Internal Medicine discusses how uric acid has evolved from the toxic cause of gout and nephrolithiasis and evolved, by way of science, into a pleiotropic recidivist in the evolution of obesity, diabetes, insulin resistance, renal disease,

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Tofacitinib in Ankylosing Spondylitis

Apr 28, 2021

A phase III trial assessing the efficacy and safety of tofacitinib in adults with with active ankylosing spondylitis (AS) shows that JAK inhibitiion was superior to placebo in a 48 week study.

A total of 269 adult AS patients with a prior an inadequate response or intolerance to ≥2

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BSRBR: Demyelinating Disease Following Anti-TNFα Therapy

Apr 21, 2021

Analysis of patients receiving anti–tumor necrosis factor α (TNFi) therapy in the British Society for Rheumatology Biologics Register (BSRBR) shows that the risk of demyelination is rare but is marginally incrased by taking TNFi therapy. 

From their large registry (n=13,489) they

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Raynaud and Microvascular Complications with New Migraine Meds

Apr 20, 2021

A new study of calcitonin gene-related peptide (CGRP) antagonists for migraine headaches found that a small subset manifested Raynaud phenomenon and other had microvascular complications, suggesting that CGRP antagonists should be used in caution in patients with Raynauds. 

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New, Improved Therapy for Pompe Disease

Apr 16, 2021

Researchers from the University of Cincinnati Gardner Neuroscience Institute will present data on a new treatment for Pompe disease at the American Academy of Neurology on April 20th; the results show that treatment with avalglucosidase alfa yielded substantial clinical improvements and a more

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RheumNow Podcast – Mistakes with Steroids

Apr 08, 2021

Dr Jack Cush gives his spin on this week's RheumNews and Rheum Research from

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Overview of New VEXAS Syndrome

Apr 08, 2021

Medscape offers up an informative overview of the newly described syndrome called VEXAS discovered by researchers at the National Institutes of Health. These patients were previously misdiagnosed with Sweets syndrome, relapsing polychondritis, myelodysplastic syndrome, MGUS, or polyarteritis

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RheumNow Podcast – Upadacitinib Wins in PsA (4.2.2021)

Apr 02, 2021

Dr Jack Cush reviews the news and Journal reports and takes "Back Talk" questions from viewers.

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Serious Infections Still Common in Vasculitis

MedPage Today
Mar 29, 2021

Serious infections occurred more frequently among patients with anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis who were given induction therapy with cyclophosphamide compared with rituximab (Rituxan), Greek researchers found.

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RheumNow Podcast – Infection in ANCA-Associated Vasculitis (3.26.2021)

Mar 26, 2021

Dr. Jack Cush reviews the news, FDA Denials and journal articles from the past week on

  1. Analysis of CPRD registry and 15,670 MTX treated pts (46,571 PYs) showed discontinuation for abnormal or severely abnormal labs was 42/1000PY and 6/1,000PY and half that

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RheumNow Podcast – Great Associations (3.19.2021)

Mar 18, 2021

Dr. Jack Cush rants on the journal reports, news and FDA actions featured this past week on

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MDA-5 Antibodies and Juvenile Dermatomyositis

Mar 17, 2021

A multicentre, retrospective study examined MSA profiles (by immunoprecipitation) from 96 juvenile idiopathic inflammatory myopathies (JIIM) at nine pediatric rheumatology centres in Japan. MSA were found in 85 of 96 cases with over 90% of patients having one of the following three MSA types

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COVID-19-Related Deaths in Rheumatic Disease Patients

Mar 11, 2021

There is a growing body of evidence regarding COVID-19 outcomes (including death) in rheumatic disease patients; analysis of a German registry shows that poor control, rituximab, sulfasalazine and other immunosuppressives could be risk factors for COVID-related death.

The early data

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Actemra FDA Approved for Systemic Sclerosis Interstitial Lung Disease

Mar 08, 2021

Genentech announced Thursday that the US Food and Drug Administration approved tocilizumab (Actemra) for the treatment of Systemic Sclerosis-associated Interstitial Lung Disease (SSc-ILD), specifically indicated for slowing the rate of decline in pulmonary function in adults SSc-ILD.

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RheumNow Podcast – Pounds of Prevention (3.5.2021)

Mar 04, 2021

Dr. Jack Cush reviews and discusses nearly a dozen news reports and journal articles from the past week on

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RheumNow Podcast – Sweet Lies or Hard Truths? (2.26.2021)

Feb 26, 2021

Dr. Jack Cush reviews and discusses the news and Journal reports from the past week on

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Lung Preservation with Tocilizumab in Early Systemic Sclerosis

Feb 21, 2021

Arthritis & Rheumatology has published the results of a subanalysis from tocilizumab (TCZ) clinical trials showing that Il-6 inhibition was associated with preservation of lung function patients with early systemic sclerosis (SSc).

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High Risk Cancer Profiling with Inflammatory Myositis

Feb 18, 2021

A recent metanalysis of studies suggest you may be able to use certain clinical factors to stratify cancer risk in patients with idiopathic inflammatory myopathies (IIMs).

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Avacopan - Is it Really a Win for ANCA-Associated Vasculitis?

Feb 18, 2021

The NEJM has reported the results of a study showing that avacopan, a C5a receptor inhibitor, is as effective as steroids at treating patients with antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis (AAV).

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Alveolar Hemorrhage and Mortality in Lupus

Feb 17, 2021

Metanalysis of patients with diffuse alveolar hemorrhage (DAH), show this to be a rare and life-threatening complication of systemic lupus erythematosus (SLE), that is more apt to affect older, severe lupus patients with active infection.

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Checkpoint Inhibitor Use with Pre-existing Autoimmune Disease

Feb 17, 2021

Annals of Internal Medicine reports treating advanced melanoma with immune checkpoint inhibitors (ICI) in patients with pre-existing autoimmune disease (AID) was as effective and safe in those with AID compared to those without AID, although severe colitis occurred more frequently among

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MIRROR Study: Pegloticase With Methotrexate in Uncontrolled Gout

Feb 15, 2021

Botson et al has reported that the combination of methotrexate (MTX) and IV pegloticase is safe and effective in patients with uncontrolled gout.

This exploratory, open-label clinical trial screened 17 patients and treated 14 patients (all men, 49.3 ± 8.7 years) with IV pegloticase with MTX (15

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