scholarly journals Consensus-based recommendations for the management of juvenile systemic sclerosis

Rheumatology ◽  
2020 ◽  
Author(s):  
Ivan Foeldvari ◽  
Roberta Culpo ◽  
Francesca Sperotto ◽  
Jordi Anton ◽  
Tadej Avcin ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Treatment Approach ◽  
Standard Of Care ◽  
Access Point ◽  
International Consensus ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Assessment And Treatment ◽  
Juvenile Systemic Sclerosis

Abstract Juvenile systemic sclerosis (JSSc) is a rare disease of childhood and currently no international consensus exists with regard to its assessment and treatment. This SHARE (Single Hub and Access point for paediatric Rheumatology in Europe) initiative, based on expert opinion informed by the best available evidence, provides recommendations for the assessment and treatment of patients with JSSc with a view to improving their outcome. Experts focused attention not only on the skin assessment but also on the early signs of internal organ involvement whose proper treatment can significantly affect the long-term outcome. A score for disease severity is proposed in order to perform a structured assessment of outcome over time but a validation in a wider patient population is recommended. Finally, a stepwise treatment approach is proposed in order to unify the standard of care throughout Europe with the aim to reduce morbidity and mortality in this disease.

The Ethics of Infant Dialysis

1996 ◽  
Vol 16 (1_suppl) ◽  
pp. 505-509 ◽  
Author(s):  
Timothy E. Bunchman
Keyword(s):  
Standard Of Care ◽  
Health Care Team ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Number Of Factors ◽  
Long Term Prognosis ◽  
Infant Dialysis ◽  
Stage Renal Disease ◽  
End Stage

The proper treatment of an infant with end-stage renal disease depends upon a number of factors including parental willingness to take on the task, experience of the health-care team, local and regional resources, and society's willingness to accept this support as a standard of care. Whereas the abilityto keep infants aliveon peritoneal dialysis (PD) is obtainable, it is not without physical, financial, as well as emotional cost. In order for a family to agree to take on such a task, an understanding of the risks and long-term prognosis should be offered. This “informed consent” is difficult to obtain in such a highly charged situation when emotions often dictate choice independently of logic. Long-term outcome of infants on PD has improved over time, yet is still fraught with complications. Options of treatment or nontreatment are explored.

scholarly journals POS0834 LONG-TERM OUTCOME OF SSC ASSOCIATED ILD: IMPROVED SURVIVAL IN PPI TREATED PATIENTS

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 670.2-671
Author(s):  
M. Kreuter ◽  
F. Bonella ◽  
K. Kathrin ◽  
J. Henes ◽  
E. Siegert ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Progression Free Survival ◽  
Basic Research ◽  
Research Support ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Single Breath ◽  
Kaplan Meier ◽  
Prospective Trials

Background:Gastroesophageal reflux disease (GERD) occurs frequently in patients with systemic sclerosis (SSc) and SSc-associated interstitial lung disease (SSc-ILD). PPI use has to been shown to improve survival in patients with idiopathic pulmonary fibrosis, whereas to date there are no data on the use of PPI in SSc-ILD.Objectives:This study was aimed to assess whether use of PPI is associated with progression of SSc-ILD and survival.Methods:We retrospectively analysed 1931 patients with SSc and SSc-ILD from the German Network for Systemic Sclerosis (DNSS) database (2003 onwards). Kaplan–Meier analysis compared overall survival (OS) and progression-free survival (PFS) in patients with vs. without GERD (SSc and SSc-ILD), and PPI vs. no PPI use (SSc-ILD only). Progression was defined as a decrease in either % predicted forced vital capacity ≥10% or single-breath diffusing capacity for carbon monoxide ≥15%, or death.Results:GERD was not associated with decreased OS or PFS in patients with either SSc or SSc-ILD. In patients with SSc-ILD, PPI use was associated with improved OS vs. no PPI use after 1 year (98.4% [95% confidence interval: 97.6–99.3]; n=760 vs. 90.8% [87.9–93.8]; n=290) and after 5 years (91.4% [89.2–93.8]; n=357 vs. 70.9% [65.2–77.1]; n=106; p<0.0001). PPI use was also associated with improved PFS vs. no PPI use after 1 year (95.9% [94.6–97.3]; n=745 vs. 86.4% [82.9–90.1]; n=278) and after 5 years (66.8% [63.0–70.8]; n=286 vs. 45.9% [39.6–53.2]; n=69; p<0.0001).Conclusion:GERD had no effect on survival in SSc or SSc-ILD. PPIs improved survival in patients with SSc-ILD; however, controlled, prospective trials are needed to confirm this finding.Disclosure of Interests:Michael Kreuter Speakers bureau: Boehringer, Consultant of: Boehringer, Grant/research support from: Boehringer, Francesco Bonella Speakers bureau: Boehringer, Roche, GSK, Consultant of: Boehringer, Roche, GSK, Grant/research support from: Boehringer, Kuhr Kathrin: None declared, Jörg Henes Speakers bureau: Abbvie, Boehringer, Chugai, Roche, Janssen, Novartis, SOBI, Pfizer and UCB, Consultant of: Boehringer, Celgene, Chugai, Roche, Janssen, Novartis, SOBI, Grant/research support from: Chugai, Roche, Janssen, Novartis, SOBI, Pfizer, Elise Siegert: None declared, Gabriela Riemekasten Speakers bureau: Novartis, Janssen, Roche, GSK, Boehringer, Consultant of: Janssen, Actelion, Boehringer, Norbert Blank Consultant of: Sobi, Novartis, Roche, UCB, MSD, Pfizer, Actelion, Abbvie, Boehringer, Grant/research support from: Novartis, Sobi, Christiane Pfeiffer: None declared, Ulf Müller-Ladner: None declared, Alexander Kreuter Speakers bureau: MSD, Boehringer, InfectoPharm, Paid instructor for: MSD, PETER KORSTEN Consultant of: Glaxo, Abbvie, Pfizer, BMS, Chugai, Sanofi, Lilly, Boehringer, Novartis, Grant/research support from: Glaxo, Aaron Juche: None declared, Marc Schmalzing Speakers bureau: Chugai Roche, Boehringer, Celgene, Medac, UCB, Paid instructor for: Novartis, Abbvie, Astra Zeneca, Chugai Roche, Janssen, Consultant of: Chugai Roche, Hexal Sandoz, Gilead, Abbvie, Janssen, Boehringer, Margitta Worm Speakers bureau: Boehringer, Ilona Jandova Speakers bureau: Boehringer, Novartis, Abbvie, Laura Susok Speakers bureau: MSD, Novartis, BMS, Sunpharma, Consultant of: MSD, Tim Schmeiser Consultant of: Abbvie, Boehringer, Novartis, UCB, Claudia Guenther Paid instructor for: Advisory Board Boehringer January 2020, Employee of: Novartis 2002-2005, Gernot Keyszer Consultant of: Boehringer, Jan Ehrchen Speakers bureau: Boehringer, Janssen, Chugai, Sobi, Employee of: Pfizer, Actelion (now Janssen), Andreas Ramming Speakers bureau: Boehringer, Gilead, Janssen, Pfizer, Roche, Consultant of: Boehringer, Pfizer, Grant/research support from: Novartis, Pfizer, Ina Kötter Speakers bureau: several companies, Consultant of: several companies, Grant/research support from: several companies, Hanns-Martin Lorenz Speakers bureau: Abbvie, Astra Zeneca, Actelion, Alexion Amgen, Bayer Vital, Baxter, Biogen, Boehringer, BMS, Celgene, Fresenius, Genzyme, GSK, Gilead, Hexal, Janssen, Lilly, Medac, MSD, Mundipharm, Mylan, Novartis, Octapharm, Pfizer, Roche Chugai, Sandoz, Sanofi, Shire SOBI, Thermo Fischer, UCB, Grant/research support from: basic research studies: Pfizer, Novartis, Abbvie, Gilead, Lilly, MSD, Roche Chugai, Pia Moinzadeh Speakers bureau: Boehringer, Actelion, Grant/research support from: Actelion, Nicolas Hunzelmann Speakers bureau: Boehringer Janssen, Roche, Sanofi, Consultant of: Boehringer

Arkansas Autotransplant (AT) Experience with > 2500 Myeloma (MM) Patients: Follow-Up Extending to >15 Years.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 1149-1149
Author(s):  
Frits van Rhee ◽  
Guido Tricot ◽  
Elias Anaissie ◽  
Maureen Reiner ◽  
Maurizio Zangari ◽  
...  
Keyword(s):  
Cox Regression ◽  
Standard Of Care ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Kaplan Meier ◽  
Long Term Follow Up ◽  
Previously Treated Patients ◽  
The University

Abstract Background: AT’s have become the standard of care for MM. Long-term follow-up studies from large centers are critical to understand who benefits most and who should be considered for alternative treatment approaches. Patients and Methods: 2,605 MM patients receiving at least one AT at the University of Arkansas were considered for this study. Kaplan-Meier analysis was used to estimate median event-free (EFS) and overall survival (OS). Cox regression was used to evaluate independent prognostic factors of EFS and OS from AT. Results: Of the 2,605 patients, 891 were enrolled into front line Total Therapy (TT) protocols TT1/2/3 (TT); 1,012 were treated on protocols for previously treated patients (non-TT); and 702 were treated off protocol due to significant co-morbidities or patient/MD preference (non-P). Median EFS and OS for all patients are 29 mo and 51 mo; 10-yr EFS and OS are 18% and 23%; 12% survived &gt;15yr. Features independently predicting superior survival included TT (HR 0.51, p&lt;.001), absence of cytogenetic abnormalities (no CA) (HR 0.47, p&lt;.001), timely application of 2nd transplant (&lt; 6 months of 1st transplant) (HR 0.71, p&lt;.001) as well as B2M &lt; 3mg/L, CRP &lt; 6mg/dL, albumin &gt;=3g/dL, platelet count &gt;=100.000/microL (all p&lt;.001) and age &lt;65yr (p=.008). The figure depicts survival (landmarked at 6 months after 1st transplant) according to the number of favorable features present of the 5 strongest predictors (TT, 2 transplants within 6 months, no CA, low B2M, low CRP). Conclusion: This large single institution experience demonstrates that &gt; 10yr survival can be accomplished in over one-half of the patients presenting without CA (14%), with low levels of B2M and CRP and receiving TT and timely 2nd autotransplant. The worst constellation affected 5% of all patients presenting with at most 1 good-risk feature whose 5-yr survival was only 8%. Collectively, these data should serve as a standard for MM investigators and patients alike, against which long-term outcome of newer treatments should be measured. Figure Figure

scholarly journals SAT0328 OUTCOME OF INTERSTITIAL LUNG DISEASE (ILD) IN ANTI-PM/SCL PATIENTS WITH SYSTEMIC SCLEROSIS: RESULTS FROM AN EUSTAR CASE-CONTROL STUDY.

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1109.2-1110
Author(s):  
M. G. Lazzaroni ◽  
C. Campochiaro ◽  
E. Marasco ◽  
J. De Vries-Bouwstra ◽  
F. Franceschini ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Disease Duration ◽  
Case Control Study ◽  
Age At Onset ◽  
Control Group ◽  
Research Support ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Control Study

Background:The main clinical associations of anti-PM/Scl in Systemic Sclerosis (SSc) so far reported include calcinosis, myositis and interstitial lung disease (ILD). Nevertheless, data regarding the long-term outcome of ILD in these patients are lacking. A single centre Spanish cohort reported a better functional outcome in 14 SSc-ILD patients anti-Pm/Scl+ as compared to 49 anti-Topo I after a mean follow-up of 7 years (1).Objectives:To analyze the long-term outcome of ILD in a large multicentre EUSTAR study dedicated to anti-Pm/Scl SSc patients.Methods:A case-control study within the EUSTAR cohort collected 165 anti-PM/Scl+ SSc cases and 257 anti-PM/Scl- SSc controls, matched for sex, cutaneous subset, disease duration, and age at onset. Data for ILD at HRCT were available for 162/165 cases and 249/257 controls. Data for pulmonary function tests (PFT) at the baseline (T0), 1 year after diagnosis (T1) and at the last visit (LV) were analyzed.Results:A significantly higher frequency of ILD was reported in anti-Pm/Scl+ cases vs anti-Pm/Scl- controls (62.3% vs 39.4%, p:<0.0001, OR 95%, CI 2.55, 1.70-3.83). Complete PFTs data were available for 81/101 ILD anti-Pm/Scl+ cases and 78/98 anti-Pm/Scl- ILD controls, with similar age at onset and female/male ratio and disease duration at LV (112±81 months vs. 115±64 months, p:0.77). Diffuse cutaneous involvement was less frequent in cases than in controls (27.2% vs. 44.9%, p:0.03).In ILD cases, %pFVC tended to improve from T0 (85.1±18.3) to T1 (89.5±16.5, p:0.045) and to LV (87.9±16.9, p:0.057), while in ILD controls remained stable from T0 (90.4±18.5) to T1 (91.1±16.5, p:0.38) and significantly declined to LV (85.0±18.0, p:0.0002). %pDLCO remained stable from T0 (60.5±16.8) to T1 (60.1±17.6, p:0.87) and to LV (60.4±16.9, p:0.77) in ILD cases, while significantly declined from T0 (67.0±18.9) to T1 (62.7±18.2, p:0.0016) and to LV (59.6±18.4, p<0.0001) in the control group. Mean %pFVC and %pDLCO at the 3 time points were not significantly different between the two groups.Delta %pFVC (LV-T0) was 2.85±11.3 for the anti-Pm/Scl+ group vs -5.42±13.4 in the control group (p:0.0004) with a significant smaller proportion of patients with FVC loss ≥10% from T0 to LV in the anti-PM/Scl group (12.3% vs. 39.7%, p:0.0001). Delta %pDLCO (LV-T0) was -0.13±10.8 for the anti-PM/Scl+ group vs -7.38±14.6 in the control group (p:0.0015), with a significant smaller proportion of patients with DLCO loss ≥10% from T0 to LV in the anti-PM/Scl+ group (13.6% vs. 42.3%, p<0.0001).Conclusion:In this multicenter real-life study, the long-term pulmonary functional outcome in SSc-ILD patients with anti-Pm/Scl positivity seems to be more favorable than in patients without anti-Pm/Scl antibodies.References:[1]Guillen-Del Castillo A, Semin Arthritis Rheum 2014, 44 (3), 331-7.Disclosure of Interests: :Maria Grazia Lazzaroni: None declared, Corrado Campochiaro Speakers bureau: Novartis, Pfizer, Roche, GSK, SOBI, Emiliano Marasco: None declared, Jeska de Vries-Bouwstra: None declared, Franco Franceschini: None declared, Francesco Del Galdo: None declared, Christopher Denton Grant/research support from: GlaxoSmithKline, CSL Behring, and Inventiva, Consultant of: Medscape, Roche-Genentech, Actelion, GlaxoSmithKline, Sanofi Aventis, Inventiva, CSL Behring, Boehringer Ingelheim, Corbus Pharmaceuticals, Acceleron, Curzion and Bayer, Lorenzo Cavagna: None declared, Oliver Distler Grant/research support from: Grants/Research support from Actelion, Bayer, Boehringer Ingelheim, Competitive Drug Development International Ltd. and Mitsubishi Tanabe; he also holds the issued Patent on mir-29 for the treatment of systemic sclerosis (US8247389, EP2331143)., Consultant of: Consultancy fees from Actelion, Acceleron Pharma, AnaMar, Bayer, Baecon Discovery, Blade Therapeutics, Boehringer, CSL Behring, Catenion, ChemomAb, Curzion Pharmaceuticals, Ergonex, Galapagos NV, GSK, Glenmark Pharmaceuticals, Inventiva, Italfarmaco, iQvia, medac, Medscape, Mitsubishi Tanabe Pharma, MSD, Roche, Sanofi and UCB, Speakers bureau: Speaker fees from Actelion, Bayer, Boehringer Ingelheim, Medscape, Pfizer and Roche, Yannick Allanore Grant/research support from: BMS, Inventiva, Roche, Sanofi, Consultant of: Actelion, Bayer AG, BMS, BI, Paolo Airò: None declared

Long-term outcome of patients with systemic sclerosis requiring home parenteral nutrition

2015 ◽  
Vol 34 (5) ◽  
pp. 991-996 ◽  
Author(s):  
Elizabeth Harrison ◽  
Ariane L. Herrick ◽  
Martyn Dibb ◽  
John T. McLaughlin ◽  
Simon Lal
Keyword(s):  
Systemic Sclerosis ◽  
Parenteral Nutrition ◽  
Home Parenteral Nutrition ◽  
Term Outcome ◽  
Long Term Outcome
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