Corticosteroid Monotherapy Research Articles

Retrospective analysis of the effectiveness of local corticosteroid therapy in children with oligoarticular juvenile idiopathic arthritis

Background . Despite the progress in diagnosis and treatment of chronic rheumatic diseases in children, the choice of anti-inflammatory drugs in case of the onset of oligoarticular juvenile idiopathic arthritis (JIA) still remains relevant. Till present, pediatric rheumatologists have not reached a consensus on this issue yet. The aim of this study was to search for predictors of early failure of local steroid therapy and assessment of its feasibility in patients with oligoarticular JIA. Materials and methods. In a retrospective study, 92 children aged 11 months – 9 years with chronic oligoarticular JIA without extra-articular manifestations were monitored. The features of the clinical, instrumental and laboratory diagnosis during the disease onset were studied, along with the dynamics of the articular syndrome and the effectiveness of intra-articular administration of corticosteroid drugs. Results and discussion. The data on 92 children with 164 active joints who received 218 local intra-articular injections of triamcinolone acetonide at the onset of the disease were analyzed. Intra-articular injections of triamcinolone acetonide at a dose of 20–40 mg were performed with an interval of 3, 6, and 12 months, depending on the intensity of the disease. In about one third of children with oligoarticular JIA, arthritis became inactive on average after two intra-articular injections of triamcinolone acetonide. The study did not reveal the predictors of early ineffective topical corticosteroid monotherapy in children. No clinical, instrumental, and laboratory signs were identified that would directly indicate the need for early therapy with methotrexate. Conclusion . Triamcinolone acetonide is an effective and safe drug for children with oligoarticular JIA. Despite the widespread use of biological, gene, and other innovative therapies, application of local corticosteroids as the firstline therapy in children with oligoarticular JIA should not be neglected.

Clinical outcome of CIDP one\xa0year after start of treatment: a prospective cohort study

ObjectiveTo assess clinical outcome in treatment-naive patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).MethodsWe included adult treatment-naive patients participating in the prospective International CIDP Outcome Study (ICOS) that fulfilled the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) diagnostic criteria for CIDP. Patients were grouped based on initial treatment with (1) intravenous immunoglobulin (IVIg), (2) corticosteroid monotherapy or (3) IVIg and corticosteroids (combination treatment). Outcome measures included the inflammatory Rasch-built overall disability scale (I-RODS), grip strength, and Medical Research Council (MRC) sum score. Treatment response, treatment status, remissions (improved and untreated), treatment changes, and residual symptoms or deficits were assessed at 1 year.ResultsForty patients were included of whom 18 (45%) initially received IVIg, 6 (15%) corticosteroids, and 16 (40%) combination treatment. Improvement on ≥ 1 of the outcome measures was seen in 31 (78%) patients. At 1 year, 19 (48%) patients were still treated and fourteen (36%) patients were in remission. Improvement was seen most frequently in patients started on IVIg (94%) and remission in those started on combination treatment (44%). Differences between groups did not reach statistical significance. Residual symptoms or deficits ranged from 25% for neuropathic pain to 96% for any sensory deficit.ConclusionsImprovement was seen in most patients. One year after the start of treatment, more than half of the patients were untreated and around one-third in remission. Residual symptoms and deficits were common regardless of treatment.

Preliminary Results from Accelerate, an International, Web-Based, Natural History Registry of Castleman Disease

Introduction: Castleman disease (CD) describes a heterogeneous group of disorders that share characteristic histopathological lymph node features. Unicentric CD (UCD) involves localized lymphadenopathy whereas multicentric CD (MCD) involves generalized lymphadenopathy, cytokine-driven systemic inflammatory symptoms, and life-threatening multiple organ system dysfunction. Uncontrolled Human Herpesvirus 8 (HHV-8) infection causes approximately half of MCD cases (HHV-8-associated MCD). The etiology of HHV-8-negative MCD is unknown or idiopathic (iMCD). Currently, there is a lack of clinical and laboratory data describing the 3 subtypes of CD and their natural histories. Further, a systematic analysis of treatments for each subtype has not been performed. Acknowledging these gaps, the field prioritized the development of a natural history study of CD, ACCELERATE, which began enrollment in 2016. The primary objective of ACCELERATE is to collect real world data on the natural history and treatment of patients with CD.Methods: ACCELERATE employs a unique two-armed approach to enroll individuals, who have had a lymph node pathology report suggesting “Castleman disease.” The “physician-directed arm” (PDA) consists of 10 study sites located in 6 European countries, a traditional paper-based patient consent, and physician data entry via a web-based portal. In contrast, the “patient-powered arm” (PPA) involves a web-based platform through which participants located anywhere outside of Europe can enroll directly at www.CDCN.org/ACCELERATE. Once enrolled in the PPA, medical records, including pathology images, are obtained and data is extracted into the web-based portal by the ACCELERATE Registry Team (ART). Analyses of demographic, select clinical and laboratory, and treatment data were performed in cases with complete extracted records. A Certification & Access Subcommittee will review all cases to grade the quality of data that exist in favor of a CD diagnosis. iMCD diagnosis is based on recently-published consensus criteria.Results: Since launching in August 2016, 51 subjects have consented, satisfied inclusion criteria, and completed all enrollment forms. The ART has completed extraction of medical records into the database for 15 patients, including 9 iMCD participants and 6 UCD participants. There were 4 females with iMCD and 3 with UCD. The median age at diagnosis was 31 (range: 14-58) for iMCD and 38.5 (range: 10-52) for UCD. Six iMCD patients demonstrated hyaline vascular or hypervascular histopathological features, 2 had mixed features, and 1 had plasmacytic features. All 6 UCD patients demonstrated hyaline vascular features. A greater number of clinical and laboratory abnormalities were observed in iMCD patients compared to UCD patients (Table 1). Four of the UCD patients received surgical excision as the sole therapy, and 2 were treated with surgical excision plus medical therapy with a corticosteroid or rituximab. Twenty-two different medications, including 4 different corticosteroids, were used to treat iMCD patients. Medications used simultaneously were classified as regimens. Twenty unique regimens were used, 7 of which included the anti-IL-6 monoclonal antibody, siltuximab, which is the only FDA-approved iMCD treatment. On average, each iMCD patient was treated with approximately 4.3 different regimens after diagnosis (mean time: 3.3 years). Regimens administered more than once to the iMCD cohort included: corticosteroid (CS) monotherapy (n=5), siltuximab+CS (n=5), rituximab monotherapy (n=4), tocilizumab+CS (n=4), siltuximab monotherapy (n=3), rituximab+CS (n=3), and rituximab+cyclophosphamide+CS (n=2).Conclusions: The ACCELERATE Registry is a powerful tool for gathering international clinical and laboratory data for CD. It will allow investigators to describe the natural history of CD and provide a database for testing new hypotheses. Information gathered from the registry may lead to refinements in the diagnostic criteria and improved insights into disease management. Based on this preliminary analysis, it is evident that CD is a heterogeneous disease that is treated with a variety of therapeutic strategies. As sample size increases, we will gain critical insights into treatment effectiveness. We are actively seeking increased enrollment of participants around the world at www.CDCN.org/ACCELERATE. [Display omitted] DisclosuresFajgenbaum:Janssen Pharmaceuticals: Research Funding. Liu:Evercore ISI: Employment. Lukac:PAREXEL International Czech Republic s.r.o, on behalf of Janssen Pharmaceuticals: Employment. Guilfoyle:Janssen: Employment, Equity Ownership. Tendler:Janssen Pharmaceuticals: Employment, Equity Ownership. Uldrick:National Institutes of Health: Patents & Royalties: immunomodulatory compound for KSHV malignancies (Inst); Merck: Research Funding; Celgene: Research Funding; Roche: Research Funding.

Inflammatory arthritis-associated pyoderma gangrenosum: a systematic review.

Pyoderma gangrenosum (PG) is a rare, rapidly progressive neutrophilic dermatosis commonly associated with systemic inflammatory diseases. We aimed to characterize the association of PG and inflammatory arthritis, as little is known outside of case reports and small cohort studies. We performed a systematic review in PubMed, EMBASE, and Scopus from inception to present using the terms arthritis and pyoderma gangrenosum. Patient demographics, clinical presentation, and treatment outcomes were recorded. Descriptive statistics and stratified analysis were used to compare factors of interest by type of arthritis. A total of 1399 articles were screened, and 129 patients with inflammatory arthritis and PG were included in the review. The most common types of arthritis were rheumatoid arthritis (RA) (50.4%), inflammatory bowel disease (IBD)-associated arthritis (10.9%), and psoriatic arthritis (8.5%). In the vast majority of cases, joint symptoms preceded PG, by a median of 10years (inter-quartile range [IQR] 5-16). Corticosteroid monotherapy and biologic therapies, used alone or in combination, resulted in improvement or complete resolution of ulcers 71.4% and 67.3% of the time, respectively. Within the latter, infliximab, adalimumab, and anakinra were most successful in inducing remission overall. RA and non-RA did not differ significantly in treatment success or healing time. This study shows that PG is frequently preceded by inflammatory arthritis, most commonly RA. Clinicians used a wide variety of treatment regimens with variable outcomes. While larger studies are needed to standardize the treatment of inflammatory arthritis-associated PG, this study suggests that in addition to systemic corticosteroids, biologic medications can be effective treatment options for these patients. • Inflammatory arthritis, most commonly rheumatoid arthritis, often precedes rather than follows pyoderma gangrenosum. • Other forms of arthritis associated with PG included IBD-associated arthritis and psoriatic arthritis. • Biologic therapies, such as infliximab, adalimumab, and anakinra, were largely successful in treating arthritis-associated pyoderma gangrenosum and may play an important role in corticosteroid-sparing therapy or in a maintenance regimen for this subset of patients. • The type of inflammatory arthritis associated with pyoderma gangrenosum may not be a helpful treatment guide as it was not significantly associated with treatment outcomes or healing time.

Impact of Immunosuppressive Agents on Clinical Manifestations and Outcome of Staphylococcus aureus Bloodstream Infection: A Propensity Score-Matched Analysis in 2 Large, Prospectively Evaluated Cohorts.

Staphylococcus aureus bloodstream infection (SAB) is a common, life-threatening infection. The impact of immunosuppressive agents on the outcome of patients with SAB is incompletely understood. Data from 2 large prospective, international, multicenter cohort studies (Invasive Staphylococcus aureus Infections Cohort [INSTINCT] and International Staphylococcus aureus Collaboration [ISAC]) between 2006 and 2015 were analyzed. Patients receiving immunosuppressive agents were identified and a 1:1 propensity score-matched analysis was performed to adjust for baseline characteristics of patients. Overall survival and time to SAB-related late complications (SAB relapse, infective endocarditis, osteomyelitis, or other deep-seated manifestations) were analyzed by Cox regression and competing risk analyses, respectively. This approach was then repeated for specific immunosuppressive agents (corticosteroid monotherapy and immunosuppressive agents other than steroids [IMOTS]). Of 3188 analyzed patients, 309 were receiving immunosuppressive treatment according to our definitions and were matched to 309 nonimmunosuppressed patients. After propensity score matching, baseline characteristics were well balanced. In the Cox regression analysis, we observed no significant difference in survival between the 2 groups (death during follow-up: 105/309 [33.9%] immunosuppressed vs 94/309 [30.4%] nonimmunosuppressed; hazard ratio [HR], 1.20 [95% confidence interval {CI}, .84-1.71]). Competing risk analysis showed a cause-specific HR of 1.81 (95% CI, .85-3.87) for SAB-related late complications in patients receiving immunosuppressive agents. The cause-specific HR was higher in patients taking IMOTS (3.69 [95% CI, 1.41-9.68]). Immunosuppressive agents were not associated with an overall higher mortality. The risk for SAB-related late complications in patients receiving specific immunosuppressive agents such as IMOTS warrants further investigations.

Treatment Strategies for GLILD in Common Variable Immunodeficiency: A Systematic Review.

IntroductionBesides recurrent infections, a proportion of patients with Common Variable Immunodeficiency Disorders (CVID) may suffer from immune dysregulation such as granulomatous-lymphocytic interstitial lung disease (GLILD). The optimal treatment of this complication is currently unknown. Experienced-based expert opinions have been produced, but a systematic review of published treatment studies is lacking.GoalsTo summarize and synthesize the published literature on the efficacy of treatments for GLILD in CVID.MethodsWe performed a systematic review using the PRISMA guidelines. Papers describing treatment and outcomes in CVID patients with radiographic and/or histologic evidence of GLILD were included. Treatment regimens and outcomes of treatment were summarized.Results6124 papers were identified and 42, reporting information about 233 patients in total, were included for review. These papers described case series or small, uncontrolled studies of monotherapy with glucocorticoids or other immunosuppressants, rituximab monotherapy or rituximab plus azathioprine, abatacept, or hematopoietic stem cell transplantation (HSCT). Treatment response rates varied widely. Cross-study comparisons were complicated because different treatment regimens, follow-up periods, and outcome measures were used. There was a trend towards more frequent GLILD relapses in patients treated with corticosteroid monotherapy when compared to rituximab-containing treatment regimens based on qualitative endpoints. HSCT is a promising alternative to pharmacological treatment of GLILD, because it has the potential to not only contain symptoms, but also to resolve the underlying pathology. However, mortality, especially among immunocompromised patients, is high.ConclusionsWe could not draw definitive conclusions regarding optimal pharmacological treatment for GLILD in CVID from the current literature since quantitative, well-controlled evidence was lacking. While HSCT might be considered a treatment option for GLILD in CVID, the risks related to the procedure are high. Our findings highlight the need for further research with uniform, objective and quantifiable endpoints. This should include international registries with standardized data collection including regular pulmonary function tests (with carbon monoxide-diffusion), uniform high-resolution chest CT radiographic scoring, and uniform treatment regimens, to facilitate comparison of treatment outcomes and ultimately randomized clinical trials.

Efficacy and Safety of Long-Term Corticosteroid Monotherapy in 26 Cases of Nephrotic Syndrome with Biopsy-Proven Membranous Nephropathy Induced by Seronegative Hepatitis B Virus-Associated Glomerulonephritis

Background: Hepatitis B virus-associated glomerulonephritis (HBV-GN) can occur in patients with negative HBV serological antigens. Little is known about the treatment of seronegative HBV-GN (sn HBV-GN). The aim of this prospective study was to evaluate the efficacy and safety of corticosteroids in the treatment of sn HBV-GN. Methods: Twenty-six patients with nephrotic syndrome induced by seronegative HBV-associated membranous nephropathy were enrolled. The patients were given methylprednisolone (0.8 mg/kg/day) for 12–24 weeks, tapered by a 2-mg reduction every 1–3 months. Patients were followed up for 6–36 months. Complete remission (CR) was defined as proteinuria <0.3 g/24 h. Partial remission (PR) was defined as proteinuria of 0.3–3.5 g/24 h that was reduced ≥50% of the baseline level. Results: The effective remission (including CR and PR) rates of nephrotic syndrome were 23.1%, 61.5%, 73.1%, 76.2%, 90.5%, and 81.0%, respectively, after 1, 3, 6, 12, 24, and 36 months. Nineteen patients achieved effective remission after 11.68 ± 7.15 months. The level of serum albumin improved from 24.34 ± 6.71 g/L at baseline to 39.61 ± 7.45 g/L at the 36th month significantly. After treatment, the level of serum Cr was similar to the baseline. Only 2 patients relapsed. The primary adverse reaction was infection. None of the patients showed evidence of HBV replication. Conclusion: The long-term middle-dose corticosteroid therapy without antiviral drugs is effective and safe for membranous sn HBV-GN patients. For sn HBV-GN patients, the monitoring of HBV DNA and HBV markers in the serum is necessary during the corticosteroid monotherapy. Trial Registration: The Chinese Clinical Trial Registry (ChiCTR1900022518).

A Review of the Current Practice of Diagnosis and Treatment of Idiopathic Membranous Nephropathy in China.

Idiopathic membranous nephropathy (IMN), a common pathological type of nephrotic syndrome, is one of the main causes of kidney failure. With an increasing prevalence, IMN has received considerable attention in China. Based on recent studies, we discuss advances in the diagnosis of IMN and the understanding of its genetic background. Although the pathogenesis of IMN remains unclear, our understanding has been substantially enhanced by the discovery of new antigens such as phospholipase A2 receptor, thrombospondin type-1 domain-containing 7A, exostosin1/exostosin2, neural epidermal growth factor-like 1 protein, neural cell adhesion molecule 1, semaphorin 3B, and factor H autoantibody. However, due to ethnic, environmental, economic, and lifestyle differences and other factors, a consensus has not yet been reached regarding IMN treatment. In view of the differences between Eastern and Western populations, in-depth clinical evaluations of biomarkers for IMN diagnosis are necessary. This review details the current treatment strategies for IMN in China, including renin-angiotensin system inhibitors, corticosteroid monotherapy, cyclophosphamide, calcineurin inhibitors, mycophenolate mofetil, adrenocorticotropic hormone, and traditional Chinese medicine, as well as biological preparations such as rituximab. In terms of management, the 2012 Kidney Disease Improving Global Outcomes (KDIGO) clinical practice guidelines do not fully consider the characteristics of the Chinese population. Therefore, this review aims to present the current status of IMN diagnosis and treatment in Chinese patients, and includes a discussion of new approaches and remaining clinical challenges.

Disease Burden and Treatment Patterns Associated With Eosinophilic Esophagitis in the United States: A Retrospective Claims Study.

This US-based, retrospective claims study aimed to investigate disease burden and treatment patterns in patients with eosinophilic esophagitis (EoE), and to compare health care resource use (HCRU) in patients with EoE and matched controls without EoE. Patients with a diagnosis of EoE and ≥12 months of prediagnosis data were identified from the Truven Health MarketScan Research databases (January 2008 to September 2016) and followed up from the diagnosis date until termination of eligibility for a health plan. Patient clinical characteristics and HCRU were recorded in the 12 months before diagnosis; HCRU and treatment patterns were recorded during follow-up. HCRU in patients with EoE and matched controls was compared during the 12-month postdiagnosis period. Among the 23,003 patients with EoE (mean age: 34.3 y; 64.8% male), gastroesophageal reflux disease was the most common prediagnosis condition (34.6%). After diagnosis, the most common off-label, first-line treatments were proton pump inhibitor monotherapy (52.8%) and topical corticosteroid monotherapy (21.5%). Overall, 3336 patients (14.5%) received at least 3 lines of off-label pharmacotherapy. Outpatient visits (recorded in 99.9% of patients on and postdiagnosis) were most frequently to gastroenterologists/pediatric gastroenterologists (49.5% prediagnosis, 72.6% on and postdiagnosis). Inpatient admissions and outpatient and emergency room visits were more likely in patients with EoE than in matched controls (P<0.0001). Patients with EoE in the USA experience a high disease burden both before and after diagnosis, which requires significant HCRU. Our findings highlight the unmet need for adequate control of EoE-related symptoms.

Home-based narrowband UVB, topical corticosteroid or combination for children and adults with vitiligo: HI-Light Vitiligo three-arm RCT.

Systematic reviews suggest that narrowband ultraviolet B light combined with treatments such as topical corticosteroids may be more effective than monotherapy for vitiligo. To explore the clinical effectiveness and cost-effectiveness of topical corticosteroid monotherapy compared with (1) hand-held narrowband ultraviolet B light monotherapy and (2) hand-held narrowband ultraviolet B light/topical corticosteroid combination treatment for localised vitiligo. Pragmatic, three-arm, randomised controlled trial with 9 months of treatment and a 12-month follow-up. Sixteen UK hospitals - participants were recruited from primary and secondary care and the community. Adults and children (aged ≥ 5 years) with active non-segmental vitiligo affecting ≤ 10% of their body area. Topical corticosteroids [mometasone furoate 0.1% (Elocon®, Merck Sharp & Dohme Corp., Merck & Co., Inc., Whitehouse Station, NJ, USA) plus dummy narrowband ultraviolet B light]; narrowband ultraviolet B light (narrowband ultraviolet B light plus placebo topical corticosteroids); or combination (topical corticosteroids plus narrowband ultraviolet B light). Topical corticosteroids were applied once daily on alternate weeks and narrowband ultraviolet B light was administered every other day in escalating doses, with a dose adjustment for erythema. All treatments were home based. The primary outcome was self-assessed treatment success for a chosen target patch after 9 months of treatment ('a lot less noticeable' or 'no longer noticeable' on the Vitiligo Noticeability Scale). Secondary outcomes included blinded assessment of primary outcome and percentage repigmentation, onset and maintenance of treatment response, quality of life, side effects, treatment burden and cost-effectiveness (cost per additional successful treatment). In total, 517 participants were randomised (adults, n = 398; and children, n = 119; 52% male; 57% paler skin types I-III, 43% darker skin types IV-VI). At the end of 9 months of treatment, 370 (72%) participants provided primary outcome data. The median percentage of narrowband ultraviolet B light treatment-days (actual/allocated) was 81% for topical corticosteroids, 77% for narrowband ultraviolet B light and 74% for combination groups; and for ointment was 79% for topical corticosteroids, 83% for narrowband ultraviolet B light and 77% for combination. Target patch location was head and neck (31%), hands and feet (32%), and rest of the body (37%). Target patch treatment 'success' was 20 out of 119 (17%) for topical corticosteroids, 27 out of 123 (22%) for narrowband ultraviolet B light and 34 out of 128 (27%) for combination. Combination treatment was superior to topical corticosteroids (adjusted risk difference 10.9%, 95% confidence interval 1.0% to 20.9%; p = 0.032; number needed to treat = 10). Narrowband ultraviolet B light was not superior to topical corticosteroids (adjusted risk difference 5.2%, 95% confidence interval -4.4% to 14.9%; p = 0.290; number needed to treat = 19). The secondary outcomes supported the primary analysis. Quality of life did not differ between the groups. Participants who adhered to the interventions for > 75% of the expected treatment protocol were more likely to achieve treatment success. Over 40% of participants had lost treatment response after 1 year with no treatment. Grade 3 or 4 erythema was experienced by 62 participants (12%) (three of whom were using the dummy) and transient skin thinning by 13 participants (2.5%) (two of whom were using the placebo). We observed no serious adverse treatment effects. For combination treatment compared with topical corticosteroids, the unadjusted incremental cost-effectiveness ratio was £2328.56 (adjusted £1932) per additional successful treatment (from an NHS perspective). Relatively high loss to follow-up limits the interpretation of the trial findings, especially during the post-intervention follow-up phase. Hand-held narrowband ultraviolet B light plus topical corticosteroid combination treatment is superior to topical corticosteroids alone for treatment of localised vitiligo. Combination treatment was relatively safe and well tolerated, but was effective in around one-quarter of participants only. Whether or not combination treatment is cost-effective depends on how much decision-makers are willing to pay for the benefits observed. Development and testing of new vitiligo treatments with a greater treatment response and longer-lasting effects are needed. Current Controlled Trials ISRCTN17160087. This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 24, No. 64. See the NIHR Journals Library website for further project information.

Systemic Steroid and Nebulized Budesonide Combination Therapy Versus Systemic Steroid Monotherapy in Patients with Acute Exacerbation of Chronic Obstructive Pulmonary Disease in a Community Hospital: A Retrospective Cohort Study.

Purpose: To evaluate clinical outcomes and costs of inhaled corticosteroid (ICS) and systemic corticosteroid combination therapy versus systemic corticosteroid monotherapy for treatment of acute exacerbations of chronic obstructive pulmonary disease (AECOPD). Methods: Hospitalized patients aged 41 to 85 years old who received ≥40 mg/day of systemic prednisone equivalents between April 3, 2017 to July 31, 2017 and April 3, 2018 to July 31, 2018 with a primary discharge diagnosis of AECOPD. Two cohorts were identified: those who received >2 doses of ICS (combination therapy) and those who received ≤2 doses of ICS (monotherapy) while on systemic corticosteroid therapy. Primary outcomes were progression of respiratory support or ≥20% increase in daily dose of systemic corticosteroids. Secondary outcomes were hospital length of stay (LOS), COPD 30-day readmissions, in-hospital mortality, and nebulized budesonide costs. Results: One hundred twenty-eight patients met inclusion criteria. Daily corticosteroid dose increases were similar between the combination and monotherapy cohorts (4% vs. 5%, P = 0.76) as was progression in ventilatory support (12% vs. 8%, P = 0.53). In-hospital mortality (4% vs. 1%, P = 0.36) and COPD 30-day readmissions (16% vs. 9%, P = 0.22) were not significantly different, however, patients in the combination arm had longer lengths of stay (4.8 days vs. 3.9 days, P = 0.04). Total nebulized budesonide costs were $1857 with a mean of $37 per patient stay for combination therapy cohort. Conclusion: Outcomes showed no clinical difference between combination therapy and monotherapy. This study suggests monotherapy may be more cost-effective while providing similar outcomes for the treatment of hospitalized patients with AECOPD.

Pulse oral corticosteroids in pediatric chronic inflammatory demyelinating polyneuropathy.

Childhood onset chronic inflammatory demyelinating polyneuropathy (CIDP) often requires long-term immunomodulatory therapy. We report a comprehensive review of our treatment of pediatric CIDP with a focus on high-dose weekly corticosteroids ("pulse oral corticosteroids"), a treatment method that is not commonly reported. We retrospectively reviewed medical records of pediatric patients with CIDP treated at our center between 2000 and 2018 for whom we had at least 12 mo follow-up. Here, we describe the demographics, disease course, treatment regimens, and long-term outcomes of these patients. Twenty-five patients were identified for analysis. Pulse oral corticosteroid monotherapy was the predominant maintenance treatment in 56% of patients. Patients were followed for a median of 4 y. Side effects were seen in a minority of patients. The probability of a normal exam or being off treatment at last follow-up was similar regardless of predominant maintenance therapy. Pulse oral corticosteroid therapy is a safe and effective long-term treatment option in children with CIDP.

AB1009 THE EFFICACY OF TOCILIZUMAB ON THE TREATMENT OF TAKAYASU ARTERITIS IN CHINESE CHILDREN

Background:Takayasu arteritis (TA) is the most prevalent large-vessel vasculitis in children. Patients with TA have a high mobidity and mortality.It remains a therapeutic challenge because corticosteroids monotherapy can rarely cure TAK and the relapse rate is high during GC tapering.Objectives:The aim of this study is to investigate the efficacy and safety of tocilizumab (TCZ)in Chinese children with Takayasu arteritis(TAK).Methods:We retrospectively studied 6 TAK children treated with TCZ in our hospital from July 2017 to October 2018. The demographic and clinical data, laboratory examination results and vascular imaging data were collected.Results:Six pediatric patients with critical or refractory TAK treated with TCZ were analyzed, including 3 males and 3 females.The diagnosis age was ranging in age from 2 to 13 years(median age:7 years).Three patients were initially treated with TCZ and Mycophenolate Mofetil(MMF) as the first-line regimen without corticosteroid or with a quite rapid GC taper duration,two of which had lifte-threatening coronary arteries involved and heart failure.The other three paitients were swcithed to TCZ from conventional disease modifying anti-rheumatic drugs (DMARDs) or other biologics due to being refractory to them and recurrent relapses.Four patients were given TCZ at 4 weeks regular intervals for 10 to 22 months,while two patients withdrew TCZ because of disease deterioration and unbearable abdominal or chest pain after the second dose.After 6 months follow-up,four patients experienced significant clinical and biological improvement with angiographically progression in one patient. A corticosteroid-sparing effect is obvious. Drug-related side effects occur in 1 patients manifesting as a mild elevated liver fuction. Neither neutropenia nor infection was observed.Conclusion:Our study shows a clinical, biological, and radiological response in patients with refractory TAK treated with TCZ.References :[1]Hellmich B, Agueda A, Monti S,et al.2018 Update of the EULAR recommendations for the management of large vessel vasculitis. Ann Rheum Dis 2019;0:1–12. doi:10.1136/annrheumdis-2019-215672.[2]BravoMancheño B, Perin F, Guez Vázquez Del ReyMDMR, García Sánchez A, Alcázar Romero PP. Successful tocilizumab treatment in a child with refractory Takayasu arteritis.Pediatrics 2012;130(6):e1720-724.[3]Goel R, Danda D, Kumar S, Joseph G. Rapid control of disease activity by tocilizumab in 10 «difficult-to-treat» cases of Takayasu arteritis. Int J Rheum Dis 2013;16(6):754–61.[4]Cañas CA, Cañas F, Izquierdo JH, Echeverri A-F, Mejía M, Bonilla-Abadía F, et al. Efficacy and safety of anti-interleukin 6 receptor monoclonal antibody (tocilizumab) in Colombian patients with Takayasu arteritis. J Clin Rheumatol Pract Rep Rheum Musculoskelet Dis 2014;20(3):125–9.[5]Batu ED, Sönmez HE, Hazirolan T, Özaltin F, Bilginer Y, Özen S. Tocilizumab treatment in childhood Takayasu arteritis: case series of four patients and systematic review of the literature. Semin Arthritis Rheum 2017 Feb;46(4):529–35.Disclosure of Interests:None declared

Indicators of active disease and steroid dependency in patients with inflammatory bowel diseases not treated with biologics in a German real-world-setting

Background and aimsWhile a minority of inflammatory bowel disease (IBD) patients receives biologics in Germany, little is known about therapeutic needs of patients receiving non-biologic therapies. This study aimed to identify indicators of active disease/steroid dependency in patients with moderate to severe Crohn’s disease (CD) and ulcerative colitis (UC) treated with conventional therapies and to describe health care resource use (HCRU)/cost.MethodsCD/UC patients treated with immunosuppressants (IS) and/or systemic or locally acting oral corticosteroids (CS) were identified in German claims data (2013–2017) and followed for 12 months post-therapy start. Indicators of active disease/steroid dependency during follow-up period were (i) ≥ 2 prescriptions of CS (sensitivity ≥ 4) or (ii) ≥ 1 IBD-related surgery or (iii) > 7 days IBD-related hospitalization(s).ResultsOf 9871 included IBD patients (5170 CD, 4701 UC), 25.7%/19.9% (CD/UC) received ≥ 2 prescriptions of CS (sensitivity, 17.4%/15.7%) (i), 3.2% experienced IBD-related surgeries (ii), and 2.5% > 7 days of hospitalizations (iii). Altogether, 44.4% had indicators of active disease/steroid dependency (sensitivity, 23.9%). Among patients with active disease/steroid dependency, 78.0% received CS monotherapy at baseline. Of these, 89.6% received a CS monotherapy in the follow-up period, too. Proportionally, fewer patients with CS monotherapy (57.4%) than IS therapy (91.0%) visited a specialist. HCRU/cost per patient year was significantly higher in patients with than without active disease/steroid dependency.ConclusionsA substantial percentage of biologic-naïve IBD patients suffers from active disease/steroid dependency. The majority receives a monotherapy with systemic CS. Referral to gastroenterologists for treatment optimization is recommended, also because active disease/steroid dependency is associated with increased HCRU/cost.

Response to topical corticosteroid monotherapy in mycosis fungoides

Topical corticosteroids alone or in combination with other therapies are widely used to treat mycosis fungoides (MF), but data on response rates to their use as monotherapy in MF are limited. To evaluate the efficacy of topical corticosteroid monotherapy in MF; compare sex, age, stage distributions, and histopathologic features between responders and nonresponders. A retrospective cross-sectional review of patients with MF from 2013 to 2019 treated at Thomas Jefferson University was conducted. Patients with biopsy-proven MF, all stages, who received topical corticosteroid monotherapy were included. Response rates were determined by percent change in body surface area (BSA) involvement and modified Severity-Weighted Assessment Tool (mSWAT). Of the 163 patients with MF in our database, 23% (37/163) initially received topical steroid monotherapy. Of these, 73% (27/37) improved, with an average 65% decrease in BSA (67% in mSWAT); 27% (10/37) did not respond/progressed, with an average 51.6% increase in BSA (57% in mSWAT); and 33% (12/37) had a complete response (BSA, 0%) with prolonged topical steroid use. Early-stage MF and female sex were more represented in responders. Single-center retrospective design. Topical steroid monotherapy in early-stage MF can produce measurable improvements in BSA and mSWAT scores and achieve complete remission in a limited subset of patients.

Vogt-Koyanagi-Harada disease: recurrence rates after initial-onset disease differ according to treatment modality and geographic area.

Literature was reviewed for studies on the evolution of initial-onset VKH disease, looking at treatment modalities and proportion of cases with chronic evolution and/or sunset-glow fundus (SGF). PubMed search yielded 1249 references containing the term of Vogt-Koyanagi-Harada. Twenty references (15 from outside of Japan and 5 from Japan) contained information on the evolution of treated initial-onset disease. For the "international" group, percentage of chronic evolution after systemic corticosteroid monotherapy was 61%, and after combined steroidal and non-steroidal therapy it fell to 2% (0% in 3/4 studies). In the Japanese studies where all patients received systemic corticosteroids alone, chronic evolution was reported in 25%; however, SGF amounted to 61%. In the world at large, chronic evolution of initial-onset VKH disease treated with corticosteroids alone concerned two-thirds of patients. Japanese studies showed that chronic evolution was substantially less frequent, indicating possibly less severe disease in Japan. This proportion fell to almost zero when dual steroidal and non-steroidal immunosuppression was given at onset.

Therapeutic management of idiopathic recurrent serositis: a retrospective study.

Idiopathic recurrent serositis (IRS) is the most frequent serositis encountered in real-life medical sceneries, and its management represents a therapeutic challenge. There are few epidemiologic data related to IRS, though most studies have focused on recurrent pericarditis, revealing that 70% of all forms of pericarditis are idiopathic and caused by innate immunity abnormalities. The aim of this study was to evaluate outcome and recurrence rates of patients with IRS, assessing management modalities used in our Periodic Fever Centre of the Gemelli Hospital, Rome, Italy, in comparison with previous treatments in other centres. Retrospectively, we analyzed the medical charts of 57 unselected patients with history of IRS managed during the period 1998-2017. A strong heterogeneity emerged by evaluating treatments of this cohort. In particular, in our Centre there was a larger use of combined therapies: 14 patients out of 27 (52%) were treated with nonsteroidal anti-inflammatory drugs (NSAIDs) and colchicine, compared to only 2 patients (7.4%) previously treated with combined treatments. We used corticosteroid monotherapy only in 1 case, against 7 from other centres. The mean duration of NSAID treatment in other hospitals was 43.8 days (SD ±27.40) and 191.25 days (SD ±42.23) in our Centre; the mean duration of corticosteroid treatment in other hospitals was 101.5 days (SD ±56.40) and 180.7 days (SD ±84.87) in our Centre. Colchicine was administered in other hospitals for the same duration of NSAIDs, and corticosteroids with an average duration of 111 days (SD ±30); conversely, we administered colchicine for an average duration of 250.12 days (SD ±80.7). Relapses of IRS were reported in 1/3 of cases who had discontinued therapies. The overall duration of treatments to manage IRS has a weight in terms of patients' outcome. A reduced duration of therapy with corticosteroids and a longer duration of therapy with NSAIDs determine a longer disease-free interval. A significant discriminating effect in terms of risk of IRS recurrence relies in an earlier combination therapy with colchicine independently from the start with either NSAIDs or corticosteroids. Finally, the evaluation of genes causing autoinflammatory diseases has not revealed any pathogenetic variants in a subcohort of 20/57 patients with IRS.

Treatment Pathways Leading to Biologic Therapies for Ulcerative Colitis and Crohn's Disease in the United States.

OBJECTIVES:Biologic therapies have been available for inflammatory bowel disease for >20 years, but patient outcomes have not changed appreciably over this time period. To better understand medication utilization for this disease, we evaluated a novel technique for visualizing treatment pathways, including initial treatment, switching, and combination therapies.METHODS:This retrospective, observational study used administrative claims data from the Truven Health MarketScan Commercial and Medicare Database. Adult patients with ≥2 consecutive health claims and newly diagnosed with ulcerative colitis (UC) or Crohn's disease (CD) were evaluated. Treatment pathways were visualized using Sankey diagrams representing the number of patients receiving treatment and duration of each treatment.RESULTS:In all, 28,119 patients with UC and 16,260 patients with CD were identified. The most common initial treatment for UC was 5-aminosalicylic acid monotherapy (61% of the patients), followed by corticosteroid monotherapy (25%); <1% of patients were initially treated with biologics. The most common initial treatment for CD was corticosteroid monotherapy (42%), followed by 5-aminosalicylic acid monotherapy (35%); <5% of the patients were initially treated with biologics. Significantly fewer patients followed biologic vs nonbiologic treatment pathways (UC: 6% vs 94%, CD: 19% vs 81%, both P < 0.05).DISCUSSION:Significantly fewer patients with inflammatory bowel disease followed treatment pathways that included biologic therapies compared with nonbiologic therapies, and very few patients were ever initiated on biologic therapy. Although we have made significant progress in treatment, our most effective medications are only being used in a small proportion of patients, suggesting barriers prevent optimized patient management.

Clinical features and treatment of 7 Chinese TAFRO syndromes from 96 de novo Castleman diseases: a 10-year retrospective study

BackgroundCastleman disease (CD) is a rare polyclonal lymphoproliferative disorder with unknown etiology. TAFRO syndrome is now regarded as a specific subtype of CD, and is still a huge challenge for clinicians.MethodsTo clarify the clinical features and management of TAFRO syndrome in China, we retrospectively analyzed 96 patients with HIV-negative CD (52 with unicentric CD and 44 with multicentric CD), who were diagnosed and treated at our center between 2008 and 2017. Specially, we systematically reviewed the 7 TAFRO syndrome cases based on the 2015 criteria proposed by Masaki.ResultsAmong the 7 cases, there were 3 men and 4 women, and the median age was 53 years. The main symptoms included thrombocytopenia (7/7), anasarca (7/7), fever (4/7), renal dysfunction (7/7), and organomegaly (6/7). One patient was treated with corticosteroid monotherapy, one received RD (Rituximab, dexamethasone), and 5 received CHOP/COP like chemotherapy as first-line treatment, 2 of the 5 combined with Rituximab. Four patients needed hemodialysis or CRRT because of progressive renal failure. The outcome for TAFRO syndrome was significantly worse compared to other types of CD. Although 3 patients improved after early treatment, 4 patients died due to disease progression, and only one patient achieved complete resolution of all the symptoms after changing to lenalidomide based regimen.ConclusionsThis study reveals that TAFRO syndrome is more severe and has more systemic symptoms than other iMCD, most cases need active treatment, and their prognoses are poor. Lenalidomide based regimen may be as a promising new therapy for TAFRO syndrome.

Clinical characteristics and treatment of IgG4-related diseases: a report of 48 cases

Forty eight patients with IgG-related disease (IgG-RD) were treated in Beijing Luhe Hospital, Capital Medical University between January 2014 and January 2017. The clinical features, organ involvement, laboratory findings and treatment of patients were analyzed. The median age of patients was 59 (29-78) years old. The most common symptoms were submandibular gland enlargement (20.8%), abdominal pain (18.8%), eyelid mass or edema (18.8%) and jaundice (16.7%). The involved organs mainly were submandibular glands (62.5%), lymph nodes (54.2%), lacrimal glands (45.8%), parotid glands (41.7%) and pancreas (31.3%). PET/CT was performed in 9 cases (18.8%), the SUVmax value was not correlated with the number of affected organs (r=0.511, P=0.160) and IgG4 (r=0.385, P=0.306). Histopathology showed increased lymphocytes and plasma cells in 46 cases (95.8%), eosinophilia in 15 cases (31.3%), and fibrous hyperplasia in 26 cases (54.2%). Twenty-three patients (47.9%) were treated with single glucocorticoid, 20 patients (41.6%) were treated with glucocorticoid combined immunosuppressants, 3 patients (6.3%) received surgical treatment, and 2 (4.2%) were observed with watch and wait regimen. Forty (97.9%) patients responded well to the initial treatment. Ten out of 23 cases (43.5%) in corticosteroid monotherapy group relapsed, while 6 out of 20 cases (30.0%) in corticosteroids plus immunosuppressive group relapsed, for whom the full dose of corticosteroid and immunosuppressive agents were still effective. IgG4-RD is a chronic inflammatory and fibrous sclerotic disease with multiple organ involvement, increase serum IgG4 level and tissue infiltration with large number of IgG4+cells. Hormone and immunosuppressive therapy is effective, and the overall prognosis is good. Key words: Immunoglobulin G; IgG4-related disease; Clinical characteristics; Treatment outcome