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Jakinibs for the treatment of immune dysregulation in patients with gain-of-function signal transducer and activator of transcription 1 (STAT1) or STAT3 mutations Lisa R. Forbes, MD, Tiphanie P. Vogel, MD, PhD, Megan A. Cooper, MD, PhD, Johana Castro-Wagner, MD, Edith Schussler, MD, Katja G. Weinacht, MD, PhD, Ashley S. Plant, MD, Helen C. Su, MD, PhD, Eric J. Allenspach, MD, PhD, Mary Slatter, MD, Mario Abinun, MD, Desa Lilic, MD, Charlotte Cunningham-Rundles, MD, PhD, Olive Eckstein, MD, Peter Olbrich, MD, R. Paul Guillerman, MD, Niraj C. Patel, MD, Yesim Y. Demirdag, MD, Christa Zerbe, MD, Alexandra F. Freeman, MD, Steven M. Holland, MD, Paul Szabolcs, MD, Andrew Gennery, MD, Troy R. Torgerson, MD, PhD, Joshua D. Milner, MD, Jennifer W. Leiding, MD Journal of Allergy and Clinical Immunology Volume 142, Issue 5, Pages (November 2018) DOI: /j.jaci Copyright © 2018 American Academy of Allergy, Asthma & Immunology Terms and Conditions
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Fig 1 Improvement in disease manifestations in patients with STAT1 GOF and STAT3 GOF mutations after treatment with jakinib and tocilizumab. A, Patient 3 with severe TPN-dependent enteropathy. Stool output significantly decreased with initiation of ruxolitinib. B, Patient 15 with lymphocytic interstitial pneumonia. a, Chest computed tomographic (CT) imaging before tocilizumab shows diffuse ground-glass pulmonary opacities and a few tiny cysts. b, Posttocilizumab chest CT imaging shows a slight overall decrease in diffuse ground-glass pulmonary opacities. c, Preruxolitinib chest CT imaging shows an increase in diffuse ground-glass pulmonary opacities compared with both prior CT examinations. d, One month after starting ruxolitinib, there is a considerable decrease in diffuse ground-glass pulmonary opacities. e, One year after treatment with both tocilizumab, chest CT, and ruxolitinib, there is a further decrease in diffuse ground-glass pulmonary opacities. BiPAP, Bilevel positive airway pressure ventilation; HFNC, high-flow nasal cannula; NC, nasal cannula. Journal of Allergy and Clinical Immunology , DOI: ( /j.jaci ) Copyright © 2018 American Academy of Allergy, Asthma & Immunology Terms and Conditions
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