April 2018 Issue

Nearly every major medical breakthrough over the past 50 years can be attributed to new discoveries that were made possible through meticulous biomedical research. Many diseases and conditions that previously took a heavy toll on patients in terms of both morbidity and mortality are now either treatable or preventable thanks in large part to painstaking research that expanded the frontiers of scientific knowledge and moved the most promising basic research developments into tangible health benefits through human clinical trails. Ongoing research is also the key that will open the door to additional major medical advances and breakthroughs in the future.

Our staff at Medifocus is committed to keeping our subscribers to the Medifocus Digest Alert on Scleroderma abreast of the latest new research developments that have recently been published in the medical literature for this condition. In this issue of the Digest Alert, you will find a focused list of hand-picked journal article references that represent the latest advances in basic and clinical research for Scleroderma. These articles represent the current state-of-the-art of the research that will hopefully lead to additional major advances and breakthroughs in the clinical management of Scleroderma. You can access the summaries of each article referenced below by simply clicking on the article Title.

We will continue to update you with the latest scientific and clinical developments through the Medifocus Digest Alert on Scleroderma every 3 months. Thank you for your ongoing subscription to this publication.

Elliot Jacob, Ph.D.
Vice-President - Content, Inc.

1:Myeloablative Autologous Stem-Cell Transplantation for Severe Scleroderma.
Authors:Sullivan KM, Goldmuntz EA, Keyes-Elstein L, McSweeney PA, Pinckney A, Welch B, Mayes MD, Nash RA, Crofford LJ, Eggleston B, Castina S, Griffith LM, Goldstein JS, Wallace D, Craciunescu O, Khanna D, Folz RJ, Goldin J, St Clair EW, Seibold JR, Phillips K, Mineishi S, Simms RW, Ballen K, Wener MH, Georges GE, Heimfeld S, Hosing C, Forman S, Kafaja S, Silver RM, Griffing L, Storek J, LeClercq S, Brasington R, Csuka ME, Bredeson C, Keever-Taylor C, Domsic RT, Kahaleh MB, Medsger T, Furst DE
Institution:From the Duke University Medical Center (K.M.S., O.C., E.W.S.C.) and RTI International (D.W.), Durham, and Rho Federal Systems Division, Chapel Hill (L.K.-E., A.P., B.E., S.C.) - all in North Carolina; National Institute of Allergy and Infectious Diseases, Bethesda, MD (E.A.G., B.W., L.M.G., J.S.G.); Colorado Blood Cancer Institute, Denver (P.A.M., R.A.N.); University of Texas McGovern Medical School (M.D.M.) and M.D. Anderson Cancer Center (C.H.) - both in Houston; Vanderbilt University, Nashville (L.J.C., K.P.); University of Michigan, Ann Arbor (D.K., J.R.S.); Case Western Reserve University and University Hospitals, Cleveland (R.J.F.); University of Alabama, Birmingham (S.M.); Boston University, Boston (R.W.S.); University of Virginia, Charlottesville (K.B.); University of Washington (M.H.W., D.E.F.) and the Fred Hutchinson Cancer Research Center (G.E.G., S.H.) - both in Seattle; University of California, Los Angeles, Los Angeles (J.G., S.K., D.E.F.); City of Hope National Medical Center, Duarte, CA (S.F.); Medical University of South Carolina, Charleston (R.M.S.); Mayo Clinic, Scottsdale, AZ (L.G.); University of Calgary, Calgary, AB, Canada (J.S., S.L.); Washington University, St. Louis (R.B.); Medical College of Wisconsin, Milwaukee (M.E.C., C.K.-T.); Ottawa Hospital Research Institute, Ottawa (C.B.); University of Pittsburgh, Pittsburgh (T.M., R.T.D.); and University of Toledo Medical Center, Toledo, OH (M.B.K.).
Journal:N Engl J Med. 2018 Jan 4;378(1):35-47.

2:Efficacy and Safety of Selexipag in Adults With Raynaud's Phenomenon Secondary to Systemic Sclerosis: A Randomized, Placebo-Controlled, Phase II Study.
Authors:Denton CP, Hachulla E, Riemekasten G, Schwarting A, Frenoux JM, Frey A, Le Brun FO, Herrick AL
Institution:Royal Free Hospital, London, UK. Internal Medicine, Hopital Huriez, University of Lille, Lille, France. Kreuznach, Germany. Manchester Musculoskeletal Biomedical Research Centre, Central Manchester NHS Foundation Trust, Manchester Academic Health Science Centre, Manchester, UK.
Journal:Arthritis Rheumatol. 2017 Dec;69(12):2370-2379. doi: 10.1002/art.40242.

3:Patient acceptable symptom state in scleroderma: results from the tocilizumab compared with placebo trial in active diffuse cutaneous systemic sclerosis.
Authors:Arnold MB, Khanna D, Denton CP, van Laar JM, Frech TM, Anderson ME, Baron M, Chung L, Fierlbeck G, Lakshminarayanan S, Allanore Y, Riemekasten G, Steen V, Muller-Ladner U, Spotswood H, Burke L, Siegel J, Jahreis A, Furst DE, Pope JE
Institution:Schulich School of Medicine and Dentistry, Western University, London, ON, Canada. School, London, UK. Netherlands. City, UT, USA. UK. Health Care System, Palo Alto, CA, USA. Berlin, Germany. University Giessen, Kerckhoff Clinic, Bad Nauheim, Germany. Canada.
Journal:Rheumatology (Oxford). 2018 Jan 1;57(1):152-157. doi: 10.1093/rheumatology/kex396.

4:Prognostic significance of computed tomography criteria for pulmonary veno-occlusive disease in systemic sclerosis-pulmonary arterial hypertension.
Authors:Connolly MJ, Abdullah S, Ridout DA, Schreiber BE, Haddock JA, Coghlan JG
Institution:National Pulmonary Hypertension Service, Royal Free London NHS Foundation Trust. Child Health, University College London, London, UK.
Journal:Rheumatology (Oxford). 2017 Dec 1;56(12):2197-2203. doi: 10.1093/rheumatology/kex351.

5:The limited cutaneous form of systemic sclerosis is associated with urinary incontinence: an international multicentre study.
Authors:John G, Allanore Y, Polito P, Piantoni S, Fredi M, Avouac J, Franceschini F, Truchetet ME, Cozzi F, Airo P, Chizzolini C
Institution:Department of Internal Medicine, Hopital neuchatelois, La Chaux-de-Fonds. Hospitals (HUG), Geneva, Switzerland. Italy. Italy. Italy. Bordeaux, France. Italy. Switzerland.
Journal:Rheumatology (Oxford). 2017 Nov 1;56(11):1874-1883. doi: 10.1093/rheumatology/kex230.

6:Epigenetics: The Future Direction in Systemic Sclerosis.
Authors:Walczyk M, Paradowska-Gorycka A, Olesinska M
Institution:Department of Connective Tissue Diseases, National Institute of Geriatrics, Rheumatology and Rehabilitation, Warsaw, Poland. Geriatrics, Rheumatology and Rehabilitation, Warsaw, Poland. Rheumatology and Rehabilitation, Warsaw, Poland.
Journal:Scand J Immunol. 2017 Dec;86(6):427-435. doi: 10.1111/sji.12595. Epub 2017 Sep 19.

7:Humanistic and cost burden of systemic sclerosis: A review of the literature.
Authors:Fischer A, Zimovetz E, Ling C, Esser D, Schoof N
Institution:University of Colorado School of Medicine, Denver, CO, USA. Electronic address:
Journal:Autoimmun Rev. 2017 Nov;16(11):1147-1154. doi: 10.1016/j.autrev.2017.09.010. Epub 2017 Sep 9.

8:Mapping and predicting mortality from systemic sclerosis.
Authors:Elhai M, Meune C, Boubaya M, Avouac J, Hachulla E, Balbir-Gurman A, Riemekasten G, Airo P, Joven B, Vettori S, Cozzi F, Ullman S, Czirjak L, Tikly M, Muller-Ladner U, Caramaschi P, Distler O, Iannone F, Ananieva LP, Hesselstrand R, Becvar R, Gabrielli A, Damjanov N, Salvador MJ, Riccieri V, Mihai C, Szucs G, Walker UA, Hunzelmann N, Martinovic D, Smith V, Muller CS, Montecucco CM, Opris D, Ingegnoli F, Vlachoyiannopoulos PG, Stamenkovic B, Rosato E, Heitmann S, Distler JHW, Zenone T, Seidel M, Vacca A, Langhe E, Novak S, Cutolo M, Mouthon L, Henes J, Chizzolini C, Muhlen CAV, Solanki K, Rednic S, Stamp L, Anic B, Santamaria VO, De Santis M, Yavuz S, Sifuentes-Giraldo WA, Chatelus E, Stork J, Laar JV, Loyo E, Garcia de la Pena Lefebvre P, Eyerich K, Cosentino V, Alegre-Sancho JJ, Kowal-Bielecka O, Rey G, Matucci-Cerinic M, Allanore Y
Institution:Rheumatology A department, Paris Descartes University, INSERM U1016, Sorbonne Paris Cite, Cochin Hospital, Paris, France.; Department of Cardiology, Paris XIII University, INSERM UMR S-942, Bobigny Hospital, Paris, France.; Unit of Clinical Research, Paris Seine Saint Denis University, Bobigny, France.; Department of Internal Medicine, Hopital Claude Huriez, University Lille Nord-de-France, Lille Cedex, Lille, France.; B Shine Rheumatology Unit, Rambam Health Care Campus, Rappaport Faculty of Medicine, Technion-Institute of Technology, Haifa, Israel.
Journal:Ann Rheum Dis. 2017 Nov;76(11):1897-1905. doi: 10.1136/annrheumdis-2017-211448. Epub 2017 Aug 23.

9:How I treat patients with systemic sclerosis in clinical practice.
Authors:Rossi D, Zanatta E, Marson P, Sciascia S, Polito P, Roccatello D, Cozzi F
Institution:Center of Research of Immunopathology and Rare Diseases - Coordinating Center of the Network for Rare Diseases of Piedmont and Aosta Valley, Department of Clinical and Biological Sciences and University of Turin, Italy; SCU Nephrology and Dialysis Unit, S. Giovanni Bosco Hospital and University of Turin, Turin, Italy. Electronic address:; Rheumatology Unit, University Hospital, Padua, Italy. Electronic address:; Apheresis Unit, University Hospital, Padua, Italy.
Journal:Autoimmun Rev. 2017 Oct;16(10):1024-1028. doi: 10.1016/j.autrev.2017.07.018. Epub 2017 Aug 1.

10:Mortality Risk Prediction in Scleroderma-Related Interstitial Lung Disease: The SADL Model.
Authors:Morisset J, Vittinghoff E, Elicker BM, Hu X, Le S, Ryu JH, Jones KD, Haemel A, Golden JA, Boin F, Ley B, Wolters PJ, King TE Jr, Collard HR, Lee JS
Institution:Department of Medicine, University of California, San Francisco, San Francisco, CA. Electronic address: Francisco, San Francisco, CA. CA. CA. CA. Francisco, CA. CA. CA. CA. CA. CA. CA. CA; Department of Medicine, University of Colorado Denver, Denver, CO.
Journal:Chest. 2017 Nov;152(5):999-1007. doi: 10.1016/j.chest.2017.06.009. Epub 2017 Jun 16.

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