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Scleroderma/Raynauds

New Treatments for Systemic Sclerosis

EurekAlert!

Autologous hematopoietic stem cell transplantation (aHSCT) for SSc has been proven the most effective treatment strategy with regard to overall and event free survival in selected patients.2 But a key limitation is its toxicity, and new treatment options are needed. Two abstracts

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#Scleroderma updated #EULAR2024 #recommendations @eular_org SKIN Are they good enough? Great work by task force @EUSTAR_org Do we alter initial Rx if v high mRSS? Ex #MMF not much improvement on skin vs #RTX #Cyclo Or combo Rx? We need to improve #SoC ?CAR-T ? RCT @RheumNow https://t.co/WhEzDdjVeu
Janet Pope @Janetbirdope( View Tweet )
#Scleroderma updated #EULAR2024 #recommendations @eular_org Are they good enough? Great work by task force @EUSTAR_org #PAH my thoughts Where does #sotaracept in #PAH fit in Not mentioned ?#selexipag before #epoprostenol For sure there is country variant @RheumNow https://t.co/3dJ6eusSkU
Janet Pope @Janetbirdope( View Tweet )
Agree! Liz Volkmann said it straight Unsustainable RCTs in ILD trials in #scleroderma Why Can’t recruit ~same pool of early #dcSSc design RCT UNETHICAL a to have a progressing #scleroderma-ILD Pt on failed background #MMF + placebo for 1 yr 🙏🏻 #EULAR2024 @RheumNow @eular_org https://t.co/29oFEazwmM
Janet Pope @Janetbirdope( View Tweet )
#EULAR2024 OP0295 Approriate patient stratification matters in trials. H2H RCT of Tocilizumab vs Rituximab in refractory #SSc-ILD (failed CyC), stratified based on RNA-seq of lungs biopsy showed improved efficacy in TCZ with low B-cell lineage expression signature @RheumNow https://t.co/7ME3Bd2PeM
Md Yuzaiful Md Yusof @Yuz6Yusof( View Tweet )
#EULAR2024 OP0288 Patients with Scleroderma shouldn’t be excluded from Lung Transplantation because of a concern with post-surgery complications/long term complications. Data from Italy of N=13 pts showed 93% survival, improved disesase activity & FVC over 2 years @RheumNow https://t.co/zPFB1MFxdc
Md Yuzaiful Md Yusof @Yuz6Yusof( View Tweet )
#EULAR2024 OP0256 Phase 2 RCT ReSScue data of fecal microbiota transplantation did not meet primary endpoint (bloating & diarrhoea) in scleroderma. How about impact on faecal incontinence symptoms? This improved throughout 20wks. Could be due improved stool consistency @RheumNow https://t.co/1t7d3M5S4Z
Md Yuzaiful Md Yusof @Yuz6Yusof( View Tweet )
#SARD #ILD Who to screen for #rheumatic #diseases For ILD #ACR guidelines Screening May also depend on ILD prevalence Not sure if frequency of screening and how to screen @jeffsparks #ORA24 @ORAexec https://t.co/BcyyYLKvr6
Janet Pope @Janetbirdope( View Tweet )

Is Cannabis Safe? (6.7.2024)

Dr. Jack Cush reviews FDA approval, news and journal articles -- and it’s the week before EULAR 2024 in Vienna!!

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Systemic sclerosis sine scleroderma in children is more aggressive than in adults. 18 juvenile vs 38 adults - ssJSSc more female (39% vs 89%), more digital pitting scars/ulcers (35 vs 10.5%), Pulm (50 vs 24) & CV (50 vs 3%); w/ more deaths https://t.co/l4f5bH1IX1 https://t.co/viQcD3tN9V
Dr. John Cush @RheumNow( View Tweet )
Untangling the relationship between smoking and systemic sclerosis: an analysis of the EUSTAR cohort https://t.co/IB0cOAFnwl https://t.co/zpwm3JRKU4
Nelly ZIADE 🍀 @Nellziade( View Tweet )

The Window of Opportunity (5.24.2024)

Dr. Jack Cush reviews the journal reports and news from the past week on RheumNow.com. This week: blockbuster drugs, opioids in rheumatology and nontreatment of inflammatory arthritis?

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Review article of Interstitial Lung Dz Assoc w/ Systemic Sclerosis. SSc-ILD prevalence ranges 25-95%; ILD is a main cause of morbidity and mortality, contributing to 35% of deaths https://t.co/8mMFbDu4gi https://t.co/nQ4GfNcMWt
Dr. John Cush @RheumNow( View Tweet )

"Don't You Know Who I am?" (5.17.2024)

Dr. Jack Cush reviews the news and journal reports from the past 2 weeks. This week's question: can we prevent gout, ILD or psoriasis?

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Who is confused about what to do for #GI symptoms in #scleroderma? @TimKanieckiMD does a masterful breakdown of gut involvement for @jhrheumatology rounds. You can read further in: https://t.co/xw6EElGIWl @ZsuzsannaM55365 would be proud! #MentoredResearchProjects #dysmotility https://t.co/yLVD7Vo1SJ
Johns Hopkins Rheumatology Fellows @JHRheumFellows( View Tweet )
IL-1 recept accessory protein (IL1RAP) shown to block mediators (IL-1, IL33, IL36) that may drive systemic sclerosis. In vitro, IL1RAP inhibited fibroblasts and endothelial cells; in mouse models of SSc anti-IL1RAP decr dermal & pulmonary fibrosis. https://t.co/6YE02P4rjR https://t.co/mzw7t88Jzl
Dr. John Cush @RheumNow( View Tweet )
Epigenetics and Autoimmune Skin Disease The central dogma proposed that genetic information predominantly transfers from DNA to RNA during gene expression to make a functional product protein. https://t.co/VgQE2yYYmq https://t.co/fAWBifCyWv
Dr. John Cush @RheumNow( View Tweet )
Cabaletta Bio announced FDA has granted Orphan Drug Designation to CABA-201, a 4-1BB-containing human CD19-CAR T cell agent, to treat systemic sclerosis. RESET (REstoring SElf-Tolerance) trial program includes the Phase 1/2 RESET-SSc trial https://t.co/Vo5jIHBrPj https://t.co/TXvFypbZdw
Dr. John Cush @RheumNow( View Tweet )
FDA grants orphan drug designation to CAR T-cell therapy for systemic sclerosis https://t.co/KZKSy2sbNk https://t.co/5abz8MeYGK
Dr. John Cush @RheumNow( View Tweet )
Review of autoimmune Dz & adverse preg outcomes: Incr risk of: - Miscarriage w/ Sjögrens (RR 8.85) & SLE (OR 4.9) - Preeclampsia w/ SLE (OR 3.2) - IUGR w/ scleroderma (OR 3.2) & celiac (OR 1.71) - Preterm birth w/ SLE (OR 2.79). https://t.co/bll2x2aS9Z https://t.co/Eg0sVBcPmR
Dr. John Cush @RheumNow( View Tweet )

Immunosuppression Increasing (2.23.2024)

Dr. Jack Cush reviews the news and journal reports from the past 2 weeks on RheumNow.com; but begins with a discussion of what constitutes "immunosuppression" and how it applies to our patients.





  1. 2021 NHIS Survey data suggests the prevalence of immunosuppression in the USA

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CD19 CAR T-Cell Therapy in Autoimmune Disease

The NEJM has published the results of CD19 B cell depletion (by CAR-T cell therapy) in autoimmune disease patients, demonstrating it to be feasible, safe, and efficacious.



The rationale to CD19 CAR-T cell transfer is to reset aberrant autoimmunity via deep depletion of B cells with

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Study of 43 SSc patients w/ gangrene shows Smoking Hx ; positive-ACA, ANCA, APL Abs & high ESR were independent risk factors for digital gangrenes in SSc. Vasculitis and macrovascular disease may contribute to the progression of digital gangrene https://t.co/3DY2Hx19s0 https://t.co/BfXANmckwF
Dr. John Cush @RheumNow( View Tweet )
Marketing analysis of systemic sclerosis prevalence estimates, from 7 major countries, ~150,000 SSc pts in 2022 (47% from US; 20% Japan; 10% UK). https://t.co/0zJFdq05Xd https://t.co/HWM3LNtHbS
Dr. John Cush @RheumNow( View Tweet )