Rheumatology Commons^™

Second Case Of Hoip Deficiency Expands Clinical Features And Defines Inflammatory Transcriptome Regulated By Lubac., Hirotsugu Oda, David B. Beck, Hye Sun Kuehn, Natalia Sampaio Moura, Patrycja Hoffmann, Maria Ibarra, Jennifer Stoddard, Wanxia Li Tsai, Gustavo Gutierrez-Cruz, Massimo Gadina, Sergio D. Rosenzweig, Daniel L. Kastner, Luigi D. Notarangelo, Ivona Aksentijevich

Manuscripts, Articles, Book Chapters and Other Papers

Background: HOIP is the catalytic subunit of the linear ubiquitination chain assembly complex (LUBAC) that is essential for NF-κB signaling and thus proper innate and adaptive immunity. To date only one patient with HOIP deficiency has been reported with clinical characteristics that include autoinflammation, immunodeficiency, amylopectinosis, and systemic lymphangiectasia. Case: We sought to identify a genetic cause of a disease for an 8 year-old girl who presented with early-onset immune deficiency and autoinflammation. Methods: Targeted next generation sequencing of 352 immune-related genes was performed. Functional studies included transcriptome analysis, cytokine profiling, and protein analysis in patients' primary cells. Results: We …

Association Of Slco1b1 *14 Allele With Poor Response To Methotrexate In Juvenile Idiopathic Arthritis Patients, Laura B. Ramsey, Halima Moncrieffe, Chelsey Smith, Marc Sudman, Miranda C. Marion, Carl D. Langefeld, Mara L. Becker, Susan D. Thompson

Manuscripts, Articles, Book Chapters and Other Papers

Objective: Variants in the SLCO1B1 gene, encoding a hepatic methotrexate (MTX) transporter, affect clearance of high-dose MTX. We tested whether in the *14 and *15 alleles of SLCO1B1 influenced the response to low-dose MTX in juvenile idiopathic arthritis (JIA) patients.

Methods: The study included 310 JIA patients genotyped for three single nucleotide polymorphisms (SNPs) in SLCO1B1 (rs4149056, rs2306283, and rs11045819). A patient's SLCO1B1 diplotype was determined by combining the SNPs into the *1a, *1b, *4, *5, *14, and *15 alleles. Number of active joints at follow-up (visit closest to 6 months of treatment and prior …

Diagnostic Dilemma Of Disseminated Histoplasmosis Mimicking Hemophagocytosis Lymphohistiocytosis In Patient With Rheumatoid Arthritis On Anti-Tnf Therapy: Case Report And Review Of The Literature, Saurav Suman, Aleksander Lenert

Internal Medicine Faculty Publications

Tumor necrosis factor inhibitors (TNFi) have become the cornerstone for the treatment of rheumatoid arthritis and other systemic autoimmune conditions. However, these biologic DMARDs can lead to various opportunistic infections such as viral infection, tuberculosis, and histoplasmosis. Furthermore, these biologics can also cause severe systemic inflammatory reactions known as hemophagocytosis lymphohistiocytosis (HLH) that can lead to multiorgan failure and high mortality. Due to overlapping clinical features and time-intensive microbiological culture methods, distinguishing between HLH and opportunistic infections can be challenging early in the disease course. We present a similar situation with our patient where the patient met the diagnostic criteria …

Comparative Analysis Of Us Real-World Dosing Patterns And Direct Infusion-Related Costs For Matched Cohorts Of Rheumatoid Arthritis Patients Treated With Infliximab Or Intravenous Golimumab., Lorie A. Ellis, Elisabetta Malangone-Monaco, Helen Varker, Diana Stetsovsky, Maureen Kubacki, Raphael J. Dehoratius, Shelly Kafka

Department of Medicine Faculty Papers

Purpose: The objectives of this study were to evaluate and compare treatment patterns and infusion-related health care resource expenditures for rheumatoid arthritis (RA) patients initiating golimumab for intravenous use (GLM-IV) and infliximab (IFX) therapy and to assess cost implications from the commercial perspective.

Methods: Adult RA patients with a new episode of GLM-IV or IFX treatment between January 1, 2014 and March 31, 2016 were identified from MarketScan databases and evaluated for maintenance infusion intervals and related costs of treatment. IFX and GLM-IV patients were matched 1:1 on index medication treatment duration, gender, payer type, prior biologic use, and post-index …

Phase Iii, Multicentre, Randomised, Double-Blind, Placebo-Controlled, 104-Week Study Of Subcutaneous Belimumab Administered In Combination With Rituximab In Adults With Systemic Lupus Erythematosus (Sle): Bliss-Believe Study Protocol, Y. K. Onno Teng, I. N. Bruce, B. Diamond, R. A. Furie, R. F. Van Vollenhoven, D. Gordon, J. Groark, R. B. Henderson, M. Oldham, P. P. Tak

Journal Articles

© 2019 Author(s) (or their employer(s)). Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ. Introduction Belimumab, an anti-B-lymphocyte-stimulator antibody, is approved for the treatment of active, autoantibody-positive systemic lupus erythematosus (SLE). Rituximab, a B cell-depleting anti-CD20 antibody, remains in the SLE treatment armamentarium despite failed trials in lupus nephritis and extrarenal lupus. These biologics, which operate through complementary mechanisms, might result in an enhanced depletion of circulating and tissue-resident autoreactive B lymphocytes when administered together. Thus, belimumab and rituximab combination may be a highly effective treatment of SLE. This study aims to evaluate …

An Interim Report Of The Scleroderma Clinical Trials Consortium Working Groups, M. Baron, B. Kahaleh, E. J. Bernstein, L. Chung, P. J. Clements, C. Denton, R. T. Domsic, N. Ferdowsi, S. Narain, A. Vacca, +12 Additional Authors

Journal Articles

© The Author(s) 2018. The Scleroderma Clinical Trials Consortium represents many of the clinical researchers in the world who are interested in improving the efficiency of clinical trials in systemic sclerosis. The Scleroderma Clinical Trials Consortium has established 11 working groups to develop and validate better ways of measuring and recording multiple aspects of this heterogeneous disease. These include groups working on arthritis, disease damage, disease activity, cardiac disease, juvenile systemic sclerosis, the gastrointestinal tract, vascular component, calcinosis, scleroderma renal crisis, interstitial lung disease, and skin measurement. Members of the Scleroderma Clinical Trials Consortium may join any one or more …

Monoclonal Antibody Targeting Bdca2 Ameliorates Skin Lesions In Systemic Lupus Erythematosus, R. Furie, V. P. Werth, J. F. Merola, L. Stevenson, T. L. Reynolds, H. Naik, W. Wang, R. Christmann, A. Gardet, N. Franchimont, +6 Additional Authors

Journal Articles

No abstract provided.

Delivering Challenging News: An Illness-Trajectory Communication Curriculum For Multispecialty Oncology Residents And Fellows, D. Cannone, M. Atlas, A. Fornari, M. L. Barilla-Labarca, M. Hoffman

Journal Articles

Introduction: Published curricula to teach communication skills for postgraduate fellows in oncology are few in number despite the fact that oncologists conduct many difficult discussions with their patients and their families. Such discussions may include disclosing initial diagnosis or relapse of a patient's cancer or relaying a poor prognosis or change to palliative care. Methods: An eight-module course on communication in oncology practice was delivered over 2 months for palliative and oncology fellows and radiation oncology residents. Learners were given a precourse survey in which they were asked to rate their proficiency in various communication tasks. Each learner then participated …

Safety And Efficacy Of Belimumab Plus Standard Therapy For Up To Thirteen Years In Patients With Systemic Lupus Erythematosus, D. J. Wallace, E. M. Ginzler, J. T. Merrill, R. A. Furie, W. Stohl, W. W. Chatham, A. Weinstein, J. D. Mckay, W. J. Mccune, A. Heath, +4 Additional Authors

Journal Articles

© 2019 The Authors. Arthritis & Rheumatology published by Wiley Periodicals, Inc. on behalf of American College of Rheumatology. Objective: To investigate the long-term safety and efficacy of intravenous (IV) belimumab plus standard of care (SOC) therapy for systemic lupus erythematosus (SLE) in patients with active, autoantibody-positive SLE. Methods: The study was designed as a multicenter, open-label, continuation study of IV belimumab given every 4 weeks in conjunction with SOC therapy in patients with SLE who completed a phase II, double-blind study. Adverse events (AEs) and laboratory data were monitored from the first belimumab dose (in either study) until 24 …

Randomised Prospective Trial To Assess The Clinical Utility Of Multianalyte Assay Panel With Complement Activation Products For The Diagnosis Of Sle., D. J. Wallace, R. V. Alexander, T. O'Malley, A. Khosroshahi, M. Hojjati, K. Loupasakis, J. Alper, Y. Sherrer, S. Narain, T. Dervieux, +6 Additional Authors

Journal Articles

Objective: We compared the physician-assessed diagnostic likelihood of SLE resulting from standard diagnosis laboratory testing (SDLT) to that resulting from multianalyte assay panel (MAP) with cell-bound complement activation products (MAP/CB-CAPs), which reports a two-tiered index test result having 80% sensitivity and 86% specificity for SLE.

Methods: Patients (n=145) with a history of positive antinuclear antibody status were evaluated clinically by rheumatologists and randomised to SDLT arm (tests ordered at the discretion of the rheumatologists) or to MAP/CB-CAPs testing arm. The primary endpoint was based on the change in the physician likelihood of SLE on a five-point Likert scale collected before …

Predicting Adherence To Therapy In Rheumatoid Arthritis, Psoriatic Arthritis Or Ankylosing Spondylitis: A Large Cross-Sectional Study., Josef S Smolen, Dafna Gladman, H Patrick Mcneil, Philip J Mease, Joachim Sieper, Maja Hojnik, Pascal Nurwakagari, John Weinman

Articles, Abstracts, and Reports

Objective: This analysis explored the association of treatment adherence with beliefs about medication, patient demographic and disease characteristics and medication types in rheumatoid arthritis (RA), psoriatic arthritis (PsA) or ankylosing spondylitis (AS) to develop adherence prediction models.

Methods: The population was a subset from ALIGN, a multicountry, cross-sectional, self-administered survey study in adult patients (n=7328) with six immune-mediated inflammatory diseases who were routinely receiving systemic therapy. Instruments included Beliefs about Medicines Questionnaire (BMQ) and 4-item Morisky Medication Adherence Scale (MMAS-4

Results: A total of 3390 rheumatological patients were analysed (RA, n=1943; PsA, n=635; AS, n=812). Based on the strongest significant …

Participation In An Innovative Patient Support Program Reduces Prescription Abandonment For Adalimumab-Treated Patients In A Commercial Population., Diana Brixner, Manish Mittal, David T Rubin, Philip Mease, Harry H Liu, Matthew Davis, Arijit Ganguli, A Mark Fendrick

Articles, Abstracts, and Reports

Purpose: Nonadherence to indicated therapy reduces treatment effectiveness and may increase cost of care. HUMIRA Complete, a Patient Support Program (PSP), aims to reduce nonadherence in patients prescribed adalimumab (ADA). The objective of this study was to assess the relationship between participation in the PSP and prescription abandonment rates among ADA-treated patients.

Patients and methods: This longitudinal study using patient-level data from AbbVie's PSP linked with medical and pharmacy claims data included patients ≥18 years with an ADA-approved indication, ≥1 pharmacy claim for ADA, and available data ≥3 months before and ≥6 months after the index date (defined as the …

Effect Of Tofacitinib On Patient-Reported Outcomes In Patients With Active Psoriatic Arthritis And An Inadequate Response To Tumour Necrosis Factor Inhibitors In The Phase Iii, Randomised Controlled Trial: Opal Beyond., Vibeke Strand, Kurt De Vlam, Jose A Covarrubias-Cobos, Philip Mease, Dafna D Gladman, Linda Chen, Elizabeth Kudlacz, Joseph Wu, Joseph C Cappelleri, Thijs Hendrikx, Ming-Ann Hsu

Articles, Abstracts, and Reports

Objectives: Tofacitinib is an oral Janus kinase inhibitor for treatment of psoriatic arthritis (PsA). Patient-reported outcomes (PROs) were evaluated in patients with PsA with inadequate responses to tumour necrosis factor inhibitors (TNFi-IR) in a 6-month, phase III randomised controlled trial (OPAL Beyond [NCT01882439]).

Methods: Patients (N=394) received tofacitinib 5 or 10 mg twice daily or placebo (advancing to tofacitinib 5 or 10 mg twice daily at month 3). Least squares mean changes from baseline and percentages of patients reporting improvements ≥minimum clinically important differences and scores ≥normative values were determined in Patient Global Assessment of disease activity (PtGA), Pain, Patient …

Understanding The Association Between Skin Involvement And Joint Activity In Patients With Psoriatic Arthritis: Experience From The Corrona Registry., Philip Mease, Carol J Etzel, William J Huster, Talia M Muram, April W Armstrong, Jeffrey R Lisse, Sabrina Rebello, Rhiannon Dodge, Mwangi James Murage, Jeffrey D Greenberg, William N Malatestinic

Articles, Abstracts, and Reports

Objective: To compare the characteristics of patients with psoriatic arthritis among patient groups stratified by degree of skin and joint involvement, and to evaluate the relationship between skin severity and joint activity.

Methods: Body surface area (BSA) and Clinical Disease Activity Index (CDAI) at enrolment were analysed. Patient characteristics were stratified by skin severity and joint activity. Baseline patient characteristics, clinical and disease characteristics and patient-reported outcomes were compared. The strength of the relationship of skin severity and joint activity was evaluated using methods for categorical variables (χ

Results: 1542 adult patients in the Corrona Psoriatic Arthritis/Spondyloarthritis Registry enrolled between …

Body Mass Index And Treatment Response To Subcutaneous Abatacept In Patients With Psoriatic Arthritis: A, Iain B Mcinnes, Gianfranco Ferraccioli, Maria-Antonietta D'Agostino, Manuela Le Bars, Subhashis Banerjee, Harris A Ahmad, Yedid Elbez, Philip Mease

Articles, Abstracts, and Reports

Objective: This

Methods: In ASTRAEA, patients with active PsA were randomised (1:1) to receive blinded weekly SC abatacept 125 mg or placebo for 24 weeks. Treatment response at week 24 was assessed by the proportions of patients achieving American College of Rheumatology 20% improvement response, Disease Activity Score in 28 joints (DAS28 (C reactive protein (CRP))) ≤3.6 and

Results: Of 212/213 and 210/211 patients with baseline BMI data in the abatacept and placebo groups, respectively, 15% and 19% were underweight/normal, 36% and 27% were overweight, and 49% and 54% were obese. After adjusting for baseline characteristics, there were no significant …

Discontinuation And Switching Patterns Of Tumour Necrosis Factor Inhibitors (Tnfis) In Tnfi-Naive And Tnfi-Experienced Patients With Psoriatic Arthritis: An Observational Study From The Us-Based Corrona Registry., Philip Mease, Chitra Karki, Mei Liu, Youfu Li, Bernice Gershenson, Hua Feng, Peter Hur, Jeffrey D Greenberg

Articles, Abstracts, and Reports

Objective: To examine patterns of tumour necrosis factor inhibitor (TNFi) use in TNFi-naive and TNFi-experienced patients with psoriatic arthritis (PsA) in the USA.

Methods: All patients aged ≥18 years with PsA enrolled in the Corrona Psoriatic Arthritis/Spondyloarthritis Registry who initiated a TNFi (index therapy) between March 2013 and January 2017 and had ≥1 follow-up visit were included. Times to and rates of discontinuation/switch of the index TNFi were compared between TNFi-naive and TNFi-experienced cohorts. Patient demographics and disease characteristics at the time of TNFi initiation (baseline) were compared between cohorts and between patients who continued versus discontinued their index TNFi …

Tofacitinib Or Adalimumab Versus Placebo: Patient-Reported Outcomes From Opal Broaden-A Phase Iii Study Of Active Psoriatic Arthritis In Patients With An Inadequate Response To Conventional Synthetic Disease-Modifying Antirheumatic Drugs., Vibeke Strand, Kurt De Vlam, Jose A Covarrubias-Cobos, Philip Mease, Dafna D Gladman, Daniela Graham, Cunshan Wang, Joseph C Cappelleri, Thijs Hendrikx, Ming-Ann Hsu

Articles, Abstracts, and Reports

Objectives: Tofacitinib is an oral Janus kinase inhibitor for treatment of psoriatic arthritis (PsA). We evaluated patient-reported outcomes (PROs) in patients with PsA refractory to ≥1 conventional synthetic disease-modifying antirheumatic drug (csDMARD-IR) and tumour necrosis factor inhibitor-naïve in a 12-month, phase III randomised controlled trial (OPAL Broaden [NCT01877668]).

Methods: Patients (N=422) received tofacitinib 5 mg or 10 mg twice daily, adalimumab 40 mg subcutaneously every 2 weeks or placebo advancing to tofacitinib 5 mg or 10 mg twice daily at month 3. Least squares mean changes from baseline and percentages of patients reporting improvements ≥minimum clinically important differences (MCID); and …

Measurement Properties Of The Minimal Disease Activity Criteria For Psoriatic Arthritis., Laura C Coates, Vibeke Strand, Hilary Wilson, Dennis Revicki, Brad Stolshek, Ahmed Samad, James B Chung, Dafna Gladman, Philip Mease

Articles, Abstracts, and Reports

Objective: To comprehensively assess evidence on the measurement properties of the minimal disease activity (MDA) criteria, a composite measure of the state of disease activity in psoriatic arthritis (PsA).

Methods: A targeted literature review was conducted to identify studies that informed the validity and/or ability of the MDA to detect change among patients known to have experienced a change in clinical status. The search was conducted using MEDLINE and Embase databases (published as of October 2017). Pertinent articles provided by investigators and identified from select conference proceedings were also evaluated.

Results: A total of 20 publications met the inclusion criteria. …

Does Adjuvant Therapy With Omega-3 Fatty Acids (Fish Oil) Improve Joint Tenderness In Patients With Rheumatoid Arthritis?, Eileen Fauteux

PCOM Physician Assistant Studies Student Scholarship

OBJECTIVE: The objective of this selective EBM review is to determine whether or not adjuvant therapy with Omega-3 fatty acids (fish oil) improves joint tenderness in patients with Rheumatoid Arthritis.

STUDY DESIGN: Review of 3 double-blind, randomized controlled trials from 2010- 2017.

DATA SOURCES: Three peer-reviewed journal articles written in English were found using PubMed. Articles were selected based on their relevance to the question and whether the outcome measured was patient-oriented evidence that matters.

OUTCOME(S) MEASURED: All articles analyzed the effectiveness of adjunctive treatment with omega-3 fatty acids on the improvement of joint tenderness in patients with rheumatoid arthritis. …

Is Hyaluronic Acid Intra-Articular Injection Better Than Corticosteroid Intra Articular Injection At Reducing Pain In Patients With Knee Osteoarthritis?, Madison Cohen

PCOM Physician Assistant Studies Student Scholarship

OBJECTIVE: The objective of this selective EBM review is to determine whether or not “Hyaluronic Acid intra articular injection is better than Corticosteroid intra articular injection at reducing pain in patients with knee osteoarthritis.”

STUDY DESIGN: Review of three double-blind, randomized control trials published between 2014 and 2016. All studies were published in English language in peer reviewed journals.

DATA SOURCES: All three randomized control trials were found via PubMed.

OUTCOMES MEASURED: The primary outcomes measured by the patient and investigator being improvement of knee pain severity, knee function, and range of motion after hyaluronic acid intra articular (IA) injection …