High-dose Oral Corticosteroids Research Articles

Corticosteroids are the major contributors to the risk for serious infections in autoimmune disorders with severe renal involvement.

We sought to investigate the infection profile and associated risk factors in a compiled cohort of patients with autoimmune disorders with severe renal involvement treated with aggressive immunosuppressive (IS) regimens. A total of 162 patients with aggressive glomerulonephritis [101 with lupus nephritis (LN), 24 with cryoglobulinemic vasculitis (CryoVasc), and 37 with ANCA-associated vasculitis (AAV)] were retrospectively reviewed for any infection occurrence. Infection incidence, type, site, and grade (1-5) were recorded. Multivariate Cox proportional hazard regression analysis was performed to identify independent risk factors for infections. A total of 179 infection episodes occurred during a follow-up of 468 patient-years. Eighty-two patients (50.6%) had at least one infection. The incidence rates of infections and severe infections were 38.2 and 14.3 events per 100 patient-years. Patients with AAV had more infections than those with CryoVasc and LN (100.6, 47.5, and 26.6 infections per 100-patient-years, respectively; p = 0.002). Most patients developed infections early during the initial induction therapy (62.1% in the first 6 months of follow-up). In multivariate Cox regression analysis, high-dose oral corticosteroids (≥ 0.5 mg/kg/day in the first month of induction therapy) was an independent predictor of any infection (HR 2.66; 95% CI, 1.5-4.73), severe infections (HR 2.45; 95% CI, 1.03-5.82), and pulmonary infections (HR 2.91; 95% CI, 1.05-8.01). Pulmonary involvement increased the risk for pulmonary infections (HR 3.67; 95% CI, 1.32-10.1) and severe infections (HR 2.45; 95% CI, 1.01-5.92). Infections occur frequently with current IS regimens in aggressive glomerulonephritis. Pulmonary involvement and high-dose corticosteroid regimen were the most significant risk factors for infections. Key Points • Infections occur frequently with current immunosuppressive regimens in autoimmune aggressive glomerulonephritis. • High-dose corticosteroids are the major contributors to the risk for serious infections.

Cost-Effectiveness Analysis of Parenteral Methotrexate for the Treatment of Crohn's Disease.

Despite worldwide use of parenteral methotrexate (pMTX), health economic evidence for its use in Crohn's disease (CD) is limited. The low price of this generic drug has removed any commercial incentive to further invest in research. However, there is an unmet need for treatment of mild-to-moderate CD, since biological/targeted therapies are usually reserved for patients with more severe disease due to the higher costs of these treatments. To evaluate the cost-effectiveness of pMTX compared to the standard of care (SOC, i.e., high doses of oral corticosteroids (hdCS) followed by gradual tapering) for the treatment of mild-to-moderate CD in the Czech Republic. We developed a 3-year Markov model with a 1-week cycle length comprising five health states. The model projected quality-adjusted life-years (QALYs) and costs from the healthcare payers' perspective. Efficacy data were obtained from a systematic literature review of clinical trials and extrapolated using survival analysis. Over a 3-year time-horizon, pMTX yields additional 0.111 QALYs (1.798 vs. 1.687) at an additional cost of €513 (€3087 vs. €2574), with an incremental deterministic (probabilistic) cost-effectiveness ratio of €4627 (€4742)/QALY, far below the willingness-to-pay (WTP) threshold (≈ €47,000/QALY). The probabilistic sensitivity analysis showed that the probability of pMTX being cost-effective was 100%. A one-way sensitivity and scenario analysis confirmed the robustness of the base-case result. Parenteral MTX proved to be cost-effective in patients with mild-to-moderate CD. This is the first published cost-effectiveness analysis of pMTX for this indication. It also shows an example of a lack of valuation of generic therapy despite its cost-effectiveness and a clear benefit to the healthcare system.

The Successful Treatment of Combined Local and Systemic Steroids in a Patient with Brachial Plexus Neuritis: A Case report

Brachial plexus neuritis (BPN), also known as Parsonage-Turner syndrome, is an uncommon neurological disorder that can manifest with acute extreme upper arm pain followed by patchy muscle paralysis. There is no evidence supporting any recommended treatments for BPN from randomized trials. Non-randomized studies have supported the effectiveness of early administration of high-dose oral corticosteroids during the painful phase. We present a case of a 41-year-old-female with a history of acute left lateral shoulder pain and shoulder girdle weakness who was diagnosed with acute BPN. To reduce the risk of complications arising from high-dose oral steroid administration, we used an ultrasound-guided brachial plexus blockade combined with low-dose oral steroids. The combined treatment approach successfully restored shoulder function and effectively alleviated pain while avoiding complications associated with systemic steroid administration. Keywords: Brachial plexus neuritis; Interventional ultrasonography; Steroid

Methotrexate and prednisolone study in erythema nodosum leprosum (MaPs in ENL) protocol: a double-blind randomised clinical trial

IntroductionErythema nodosum leprosum (ENL) is an immunological complication of leprosy. ENL results in morbidity and disability and if it is not treated can lead to death. The current treatment consists...

Thalidomide in the treatment of erythema nodosum leprosum (ENL) in an outpatient setting: A five-year retrospective analysis from a leprosy referral centre in India.

Erythema nodosum leprosum (ENL), or type 2 lepra reaction, is a multi-system immune-mediated complication in patients with multibacillary leprosy, frequently associated with chronicity and recurrences. Management of ENL requires high doses of oral corticosteroids, which may not be universally effective and pose serious adverse effects. Thalidomide has proven to be a steroid-sparing agent and is useful in controlling the reactions. However, many centres do not employ it in outpatient settings due to adverse effects and teratogenicity risk. Hence, we studied the feasibility of treating ENLs and reported the therapeutic outcome.This is a five-year record-based analysis of ENL leprosy patients treated with thalidomide, includingdescriptive statistics of demographic variables. Clinical characteristics were stratified by treatment compliance status (yes/no). Incidence rates and rate ratios for recovery stratified by bacillary index, type of ENL presentation and MDT treatment status were calculated.Out of 102 ENL patients treated with thalidomide, 68 (66.7%) were compliant and improved. Among them, ENL recurrence was noted in 11(16.2%) patients. The commonest thalidomide side effect was pedal oedema (73.5%). Patients with bacillary index (BI) less than or equal to 4.0 had a 37% increase in the incidence of recovery. Patients with acute ENL were almost twice as likely to recover as those with chronic ENL. Also, the improvement was two and a half times greater among those who completed MDT as compared to those on MDT. The study showed that thalidomide treatment for patients with ENL is possible in outpatientclinics. We also successfully prevented pregnancies to a larger extent through counselling for contraception.We observed that early institution of thalidomide induces faster remission and prevents ENL recurrence.

NIVOLUMAB-INDUCED PULMONARY FIBROSIS: A NEW FINDING

SESSION TITLE: Medical Student/Resident Diffuse Lung Disease SESSION TYPE: Med Student/Res Case Rep Postr PRESENTED ON: October 18-21, 2020 INTRODUCTION: The growth of personalized medicine has increased survival in aggressive cancers. Despite their improved side effect profile relative to early chemotherapy, monoclonal antibodies still feature many potentially severe side effects. Here, we present the first reported case of nivolumab-induced rapidly progressive pulmonary fibrosis. CASE PRESENTATION: A 65 year old gentleman with chronic respiratory failure requiring supplemental oxygen, a history of smoking, and stage IV supraglottic laryngeal cancer was started on nivolumab after progression of disease on platinum-based chemotherapy and radiation. Three months after starting nivolumab, honey-combing, traction bronchiectasis, and pulmonary fibrosis with lower lobe predominance was noted on computed tomography (CT) imaging during a hospital admission for dyspnea. (Image 1) Nivolumab was stopped, and oral corticosteroids were started. Despite this, the patient’s respiratory status continued to decline, requiring multiple hospitalizations. After admission for septic shock secondary to pneumonia, the patient suffered respiratory failure requiring intubation, which the patient declined and died with comfort care. DISCUSSION: Nivolumab is an anti-PD1 checkpoint inhibitor human monoclonal antibody approved for second-line use in squamous cell head and neck cancer with disease progression after platinum-based therapy. For this indication, median survival time is increased by 2.4 months. (1) Pneumonitis is a rare adverse effect of nivolumab, seen in 5-6% of patients. The median time of onset ranges widely from days to over a year. Most cases of pneumonitis are mild, and most cases improve or resolve completely. Features associated with worse outcomes include severity at presentation, underlying lung disease, poor lung function, and smoking history. Mainstay of treatment is high dose oral corticosteroids, with severe cases requiring intravenous steroids or additional immunosuppression such as infliximab. (2) Our patient had many of the poor prognostic factors for nivolumab-induced pneumonitis, but instead developed fibrosis. Pneumonitis has been reported many times, but to our knowledge this is the first evidence of pulmonary fibrosis caused by nivolumab. Potentially, the maelstrom of baseline conditions contributed to fibrosis rather than pneumonitis. CONCLUSIONS: Given the prevalence of chronic lung conditions, increasing use of nivolumab, and rapidly progressive nature of this case—from diagnosis to death in six weeks and poor response to steroids—consideration of pulmonary fibrosis as an adverse event of nivolumab may necessitate future considerations. Reference #1: Opdivo. [package insert]. New York, NY: Bristol-Myers-Squibb; 2014. Reference #2: Naidoo J, Wang X, Woo KM, et al. Pneumonitis in Patients Treated With Anti-Programmed Death-1/Programmed Death Ligand 1 Therapy. J Clin Oncol. 2017;35(7):709-717. DISCLOSURES: No relevant relationships by Hazim Bukamur, source=Web Response No relevant relationships by Kendall Creed, source=Web Response My spouse/partner as a Consultant relationship with Abbvie Please note: $1-$1000 Added 05/27/2020 by Ebtesam Islam, source=Web Response, value=Consulting fee No relevant relationships by John Makram, source=Web Response No relevant relationships by Alexandra Wichmann, source=Web Response

P0380INFECTION PROFILE AND ASSOCIATED RISK FACTORS IN AGGRESSIVE GLOMERULONEPHRITIS TREATED WITH INDUCTION AND MAINTENANCE REGIMENS OF IMMUNOSUPPRESSIVE THERAPY

Abstract Background and Aims Infections remain an important contributor to the morbidity and mortality of immunosuppressive (IS) therapy in aggressive glomerulonephritis. We sought to investigate the infection profile and associated risk factors in a compiled cohort of patients with lupus nephritis (LN), cryoglobulinemic vasculitis (CryoVas) and ANCA-associated vasculitis (AAV) treated with induction and maintenance IS regimens. Method A total of 162 patients (101 with LN, 24 with CryoVas and 37 with AAV) were retrospectively reviewed for any infection that occurred from initiation of induction therapy. Infections were graded (1-5) according to the Common Terminology Criteria for Adverse Events. Infection site and type of microorganism were also recorded. Univariate and multivariate Cox proportional hazard regression analysis were performed in order to identify independent risk factors for infection. Results Eighty-two patients (50.6%) had at least one infection with a total 179 episodes of infection occurring during a median follow-up of 12 months (IQR:4-36.25 months). The majority of patients (64 of 82) had infections during the first 24 months since IS treatment initiation with a 24-month infection-free rate of 55%. The most common site was lung infection (in 32.7% of patients), while 39.5% of patients had bacterial infections (1.8% with Mycobacterium tuberculosis). 36.7% of patients had severe infections (grade 3 or higher) with 4.4% of infection-related deaths (8 patients). The most common induction regimen was cyclophosphamide in addition to corticosteroids (62%), while 43% received either mycophenolate mofetil or azathioprine in addition to corticosteroids as a maintenance regimen. In univariate Cox regression analysis, chronic obstructive pulmonary disease (HR 3.91; 95% CI, 1.76-8.68, p=0.001), pulmonary involvement in the setting of systemic disease (HR 2.35; 95% CI, 1.26-4.37, p=0.007), pulse methylprednisolone (HR 2.7; 95% CI, 1.7-4.31, p=0.001) and high-dose (≥30 mg/day) oral corticosteroids (HR 3.38; 95% CI, 2.11-5.43, p=0.001) were risk factors for infection. In multivariate Cox regression analysis, high-dose oral corticosteroids (HR 2.67; 95% CI, 1.5-4.76, p=0.001) remained an independent predictor of infection risk. Of the risk factors associated with severe infections (grade 3 or higher), in univariate analysis we identified pulmonary involvement in the setting of systemic disease (HR 3.65; 95% CI, 1.72-7.77, p=0.001), pulse methylprednisolone (HR 3.56; 95% CI, 1.7-7.3, p=0.001), high-dose (≥30 mg/day) oral corticosteroids (HR 3.56; 95% CI, 1.77-7.16, p=0.001), estimated GFR (HR 0.98; 95% CI, 0.98-0.99, p=0.01) and AAV (by comparison to CryoVas and LN) (HR 2.81; 95% CI, 1.39-5.66, p=0.004) as risk factors for infection. After multivariate adjustment, pulmonary involvement in the setting of systemic disease (HR 2.38; 95% CI, 1.01-5.73, p=0.05) and high-dose oral corticosteroids (HR 2.44; 95% CI, 1.04-5.72, p=0.04) were identified as independent predictors of infection risk. Conclusion Infections occur frequently with current immunosuppressive regimens in aggressive glomerulonephritis. In addition to pulmonary involvement in the setting of systemic disease, a high dose corticosteroid regimen was the most significant risk factor for infection.

Recurrent Henoch-Sch\xf6nlein Purpura with bullous rash and pulmonary nodules

BackgroundHenoch-Schönlein purpura (HSP) is the most common vasculitis of childhood. It has a characteristic rash described as palpable purpura that most frequently affects the distal lower extremities and buttocks. HSP rarely presents with bullous rash nor pulmonary nodules.Case presentationWe present a novel case of a 12-years-old female with recurrent pediatric HSP with a combination of the rare manifestations of bullous rash and pulmonary nodules. She initially presented with the bullous rash, chest pain, cough, and abdominal pain. Patient was successfully treated with intravenous pulse corticosteroids followed by a high dose oral corticosteroid taper, with resolution of the bullous rash and pulmonary nodules.ConclusionThe rare manifestations of scarring bullous rash and pulmonary nodules can be presenting features of pediatric HSP, the combination of which has not been previously reported. The treatment of intravenous corticosteroid resolved patient’s abdominal symptoms, rash and pulmonary nodules.

Pseudomonas aeruginosa scleritis initially presenting as idiopathic diffuse anterior scleritis.

Pseudomonas aeruginosa remains the most common cause of bacterial scleritis. This report illustrates an unusual presentation of P. aeruginosa scleritis, which initially presented as diffuse anterior scleritis with anterior uveitis. The detailed laboratory work-up of the patient was negative, and the initiation of high-dose oral corticosteroid therapy led to further deterioration of clinical condition, with the appearance of a yellowish-white nodule within 3 days. The aspirate from the nodule grew P. aeruginosa, and the scleral inflammation resolved with anti-microbial therapy.

An unusual myopathy with dual pathology: Case report

Immune-mediated necrotising myopathy (IMNM) and inclusion body myositis (IBM) belong to a spectrum of conditions called the idiopathic inflammatory myopathies. However, they both represent distinct conditions, which differ in their clinical presentation, immuno-pathogenesis and in the available treatment options. In this poster presentation, I report the case of a 69-year old gentleman who had presented with a gradually progressive, symmetric, proximal myopathy with preservation of distal muscle strength including the deep finger flexors. There was no rash, bulbar disturbance or other focal neurological deficits. A serum creatine kinase was significantly elevated (4935 IU/L) and serological testing revealed strongly positive anti-HMGCR antibodies. Although these clinical and biochemical features robustly support a diagnosis of IMNM, skeletal muscle biopsy showed evidence of dual pathology with histopathological features consistent with a combination of IMNM and IBM. Computed tomography (CT) of the chest, abdomen and pelvis showed no underlying occult malignancy. The patient subsequently demonstrated an excellent clinical and biochemical response to high dose oral corticosteroid treatment, with improved muscle strength and mobility, and was simultaneously commenced on azathioprine immunotherapy. This poster illustrates an unusual case of biopsy-proven dual IMNM and IBM pathology in skeletal muscle. Herein, I discuss valuable learning points derived from this case, including the importance of interpreting muscle biopsy-findings in the context of the relevant clinical and biochemical profile when making a diagnosis, and constructing treatment strategies, in the myopathic patient.

7. Life-threatening adult-onset Still’s disease

7. Life-threatening adult-onset Still’s disease

6. The Stillness after the storm: a challenging diagnostic and therapeutic case of adult-onset Still’s disease

6. The Stillness after the storm: a challenging diagnostic and therapeutic case of adult-onset Still’s disease

077 An unusual presentation of antibody negative autoimmune encephalitis: a case report

IntroductionImmune-mediated encephalitis can occur in the absence of measurable pathogenic antibodies in CSF or serum.Methods and resultsWe present the case of a 29-year-old Sudanese woman presented with four days history of meningism. She is three months post-partum with her fifth child and does not have significant past medical history. She is a current smoker with history of methamphetamine and heroin use until 5 years ago. There were no focal neurological deficits on examination. CT brain was normal and her CSF had 420×106/L leucocytes (Polymorphs 18, Mononuclears 402). Three days later, she deteriorated clinically with GCS 11 and developed left sided hemiplegia requiring intubation and ventilation in intensive care.The CSF culture along with autoimmune encephalitis panel, anti-neuronal antibodies, HSV, VZV, Enterovirus, cryptococcal antigen, TB quantiferon, HIV, Malaria were negative. Her immunology tests were unremarkable including rheumatoid factor, complements, anti-nuclear antibodies, dsDNA, PR3, MPO, RNP, Smith, Ro, La, Scleroderma 70 and Jo-1 antibodies. The MRI Brain showed persistent changes in right hemispheric cortical and subcortical gyriform FLAIR hyperintensities which were also present in DWI images. The EEG showed right temporo-occipital region 7–8 Hz rhythmic discharges with generalised diffuse slowing. She had right parietal lobe brain biopsy which shows perivascular and intramural inflammation by mononuclear cells almost comprised of CD3 T-cells consistent with encephalitis associated with secondary lymphocytic vasculitis. She was treated with intravenous pulsed methylprednisolone and IVIg followed by plasma exchange. Then given high dose oral corticosteroids with slow tapering and intravenous Cyclophosphamide four weekly. Significant neurological improvement noted over next 8 weeks with patient being alert and participating in ongoing multidisciplinary rehabilitation for hemiplegia.ConclusionA case of cerebral biopsy confirmed, CSF and serum antibody-negative encephalitis is presented.

Idiopathic orbital inflammation: An unusual presentation of benign essential blepharospasm

A case of a 48-year-old man with recurrent twitching of the right eye associated with the drooping of the upper eyelid, ipsilateral periorbital headache and blurring of vision aggravated by direct exposure to strong sunlight. Eye examination revealed intermittent twitching of the right upper eyelid, mild restriction of range of motion (downward gaze) and difficulty reading small prints (presbyopia). Computed tomographic scan showed thickened right superior rectus muscle and tendons. Based on the clinical and neuroimaging abnormalities a diagnosis of right orbital myositic inflammation was made. Patient was given high dose oral corticosteroids and remarkable improvement in his symptoms was recorded after 6 weeks of treatment.

Change in type-2 biomarkers and related cytokines with prednisolone in uncontrolled severe oral corticosteroid dependent asthmatics: an interventional open-label study

Type-2 biomarkers and related cytokines (IL-5, IL-13), lung function and asthma symptoms were measured in 44 poorly-controlled severe oral corticosteroid (OCS)-dependent asthmatics for up to 88 days after a 7-day...

Intravenous pulse methylprednisolone for induction of remission in severe ANCA associated Vasculitis: a multi-center retrospective cohort study

BackgroundIntravenous pulse methylprednisolone (MP) is commonly included in the management of severe ANCA associated vasculitis (AAV) despite limited evidence of benefit. We aimed to evaluate outcomes in patients who had, or had not received MP, along with standard therapy for remission induction in severe AAV.MethodsWe retrospectively studied 114 consecutive patients from five centres in Europe and the United States with a new diagnosis of severe AAV (creatinine > 500 μmol/L or dialysis dependency) and that received standard therapy (plasma exchange, cyclophosphamide and high-dose oral corticosteroids) for remission induction with or without pulse MP between 2000 and 2013. We evaluated survival, renal recovery, relapses, and adverse events over the first 12 months.ResultsFifty-two patients received pulse MP in addition to standard therapy compared to 62 patients that did not. There was no difference in survival, renal recovery or relapses. Treatment with MP associated with higher risk of infection during the first 3 months (hazard ratio (HR) 2.7, 95%CI [1.4–5.3], p = 0.004) and higher incidence of diabetes (HR 6.33 [1.94–20.63], p = 0.002), after adjustment for confounding factors.ConclusionsThe results of this study suggest that addition of pulse intravenous MP to standard therapy for remission induction in severe AAV may not confer clinical benefit and may be associated with more episodes of infection and higher incidence of diabetes.

P095 COMPARING ORAL CORTICOSTEROIDS ≥30MG/DAY AMONG ADULT AND PEDIATRIC PATIENTS WITH NEWLY DIAGNOSED ULCERATIVE COLITIS

Ulcerative Colitis (UC) has a relapsing–remitting disease course. Sparing use of oral corticosteroids (CS) ≥30 mg/day, as mono or combination therapy, are generally reserved for rapid relief of symptoms and induction of remission to achieve optimal therapeutic response. This analysis aimed to compare patient profiles of adult and pediatric patients newly diagnosed with UC who use ≥30 mg/day CS in a real world setting. The Truven Marketscan commercial administrative claims database was used to explore the real-world utilization of ≥30mg/day CS among pediatric (children 2- 11 years old and adolescents 12-17 years old) and adult (≥18 years old) UC patients. Patients were identified based on first diagnosis of UC during 01/01/2013 -12/31/2015 and were included in the analysis if they had an additional UC diagnosis during the 1-year post-index period. Patients with pre-specified, common inflammatory comorbid conditions associated with high doses of oral CS were excluded. The final sample was subsequently stratified based on number of days (d) [0, 1-7, 8-30, 31-90, 91-180, 181-365] on ≥30mg/day oral CS during follow-up (1-year post diagnosis). Pre-index and post-index patient characteristics (demographics, medications, and health care resource use (HCRU)) are described. A total of 10,443 UC patients were included in this analysis consisting of 91 children (2-11 years old), 290 adolescents (12-17 years old), and 10,062 adults (18+ years old). Demographic information such as age, gender, and geographic location for each subgroup is displayed in Table 1. Approximately, 40.66% of children and 46.90% of adolescents received corticosteroids during the 1-year post-index period, compared to only 14.62% of adults. In addition, 25.27% of children, 34.48% of adolescents, and 6.68% of adults received corticosteroids for more than 30 days during the post-index period. Among all subgroups, patients with ≥30d of ≥30mg/day CS use were more likely to be prescribed biologics. HCRU (including gastroenterologist visits, ER visits, and inpatient visits) was higher for patients receiving ≥30mg/day of CS at least 1 day during the post-index period when compared to those not receiving ≥30mg/day of CS. HCRU was higher among the pediatric subgroups (children and adolescents) when compared to adults (Table 1). During a 1-year follow-up period, UC patients with a longer duration of ≥30mg/day CS use were more likely to be on a biologic and had increased HCRU. This analysis of real-world data suggests that there is a subset of newly diagnosed pediatric and adult patients with UC who may require longer use of higher doses of oral CS to achieve optimal therapeutic response. This analysis also suggests that pediatric UC patients are more likely to be treated with corticosteroids and have higher HCRU when compared to the adult UC population.

P095 COMPARING ORAL CORTICOSTEROIDS ≥30MG/DAY AMONG ADULT AND PEDIATRIC PATIENTS WITH NEWLY DIAGNOSED ULCERATIVE COLITIS

Ulcerative Colitis (UC) has a relapsing–remitting disease course. Sparing use of oral corticosteroids (CS) ≥30 mg/day, as mono or combination therapy, are generally reserved for rapid relief of symptoms and induction of remission to achieve optimal therapeutic response. This analysis aimed to compare patient profiles of adult and pediatric patients newly diagnosed with UC who use ≥30 mg/day CS in a real world setting. The Truven Marketscan commercial administrative claims database was used to explore the real-world utilization of ≥30mg/day CS among pediatric (children 2- 11 years old and adolescents 12-17 years old) and adult (≥18 years old) UC patients. Patients were identified based on first diagnosis of UC during 01/01/2013 -12/31/2015 and were included in the analysis if they had an additional UC diagnosis during the 1-year post-index period. Patients with pre-specified, common inflammatory comorbid conditions associated with high doses of oral CS were excluded. The final sample was subsequently stratified based on number of days (d) [0, 1-7, 8-30, 31-90, 91-180, 181-365] on ≥30mg/day oral CS during follow-up (1-year post diagnosis). Pre-index and post-index patient characteristics (demographics, medications, and health care resource use (HCRU)) are described. A total of 10,443 UC patients were included in this analysis consisting of 91 children (2-11 years old), 290 adolescents (12-17 years old), and 10,062 adults (18+ years old). Demographic information such as age, gender, and geographic location for each subgroup is displayed in Table 1. Approximately, 40.66% of children and 46.90% of adolescents received corticosteroids during the 1-year post-index period, compared to only 14.62% of adults. In addition, 25.27% of children, 34.48% of adolescents, and 6.68% of adults received corticosteroids for more than 30 days during the post-index period. Among all subgroups, patients with ≥30d of ≥30mg/day CS use were more likely to be prescribed biologics. HCRU (including gastroenterologist visits, ER visits, and inpatient visits) was higher for patients receiving ≥30mg/day of CS at least 1 day during the post-index period when compared to those not receiving ≥30mg/day of CS. HCRU was higher among the pediatric subgroups (children and adolescents) when compared to adults (Table 1). During a 1-year follow-up period, UC patients with a longer duration of ≥30mg/day CS use were more likely to be on a biologic and had increased HCRU. This analysis of real-world data suggests that there is a subset of newly diagnosed pediatric and adult patients with UC who may require longer use of higher doses of oral CS to achieve optimal therapeutic response. This analysis also suggests that pediatric UC patients are more likely to be treated with corticosteroids and have higher HCRU when compared to the adult UC population.

An open-label randomized controlled trial of low-dose corticosteroid plus enteric-coated mycophenolate sodium versus standard corticosteroid treatment for minimal change nephrotic syndrome in adults (MSN Study)

First-line therapy of minimal change nephrotic syndrome (MCNS) in adults is extrapolated largely from pediatric studies and consists of high-dose oral corticosteroids. We assessed whether a low corticosteroid dose combined with mycophenolate sodium was superior to a standard oral corticosteroid regimen. We enrolled 116 adults with MCNS in an open-label randomized controlled trial involving 32 French centers. Participants randomly assigned to the test group (n=58) received low-dose prednisone (0.5 mg/kg/day, maximum 40 mg/day) plus enteric-coated mycophenolate sodium 720 mg twice daily for 24 weeks; those who did not achieve complete remission after week 8 were eligible for a second-line regimen (increase in the prednisone dose to 1 mg/kg/day with or without Cyclosporine). Participants randomly assigned to the control group (n=58) received conventional high-dose prednisone (1 mg/kg/day, maximum 80 mg/day) for 24 weeks. The primary endpoint of complete remission after four weeks of treatment was ascertained in 109 participants, with no significant difference between the test and control groups. Secondary outcomes, including remission after 8 and 24 weeks of treatment, did not differ between the two groups. During 52 weeks of follow-up, MCNS relapsed in 15 participants (23.1%) who had achieved the primary outcome. Median time to relapse was similar in the test and control groups (7.1 and 5.1 months, respectively), as was the incidence of serious adverse events. Five participants died from hemorrhage (n=2) or septic shock (n=3), including 2 participants in the test group and 3 in the control group. Thus, in adult patients, treatment with low-dose prednisone plus enteric-coated mycophenolate sodium was not superior to a standard high-dose prednisone regimen to induce complete remission of MCNS.

PGI23 - COST-EFFECTIVENESS ANALYSIS OF PARENTERAL METHOTREXATE FOR THE TREATMENT OF CROHN’S DISEASE IN THE CZECH REPUBLIC

Clinical evidence suggests that parenteral methotrexate (MTX) provides benefit for induction of remission and complete discontinuation of high-dose oral corticosteroid treatment (hdCS; >20mg/day) in patients with Crohn’s disease (CD). Our aim was to assess cost-effectiveness of MTX in the treatment of mild-to-moderate CD in comparison to hdCS/placebo with its gradual withdrawal in the Czech Republic. We developed a three-year Markov model with one-week cycle length in TreeAge. The model comprises four health states – initial state of mild-to-moderate CD (16-week treatment with MTX or hdCS), steroid-free remission, lack/loss of response and death. It projects quality-adjusted life-years (QALYs) and costs from healthcare payers’ perspective. Costs were based on list prices, reimbursement tariffs and previous pharmacoeconomic analyses as of 02/2018. Costs and outcomes were discounted by 3%. Transition probabilities between health states were provided by clinical trials and extrapolated using survival analysis. Mortality rate was taken from Czech mortality tables and adjusted to CD. Utilities were derived from the published mapping algorithm. One-way sensitivity analysis (OWSA) accompanied by scenario analysis (SA) was performed. Probability sensitivity analysis (PSA; 10,000 iterations) was run using an implicit willingness-to-pay threshold (WTP) of €47,000/QALY. Over a three-year time horizon, MTX yields additional 0.06 QALYs (1.92 vs. 1.86) at the additional total cost of €761 (€2263 vs. €1503) compared with hdCS, with the incremental cost-effectiveness ratio of €13,077/QALY. PSA showed that probability of MTX to be cost-effective was 99.89% at the WTP. OWSA and SA confirmed the robustness of the base-case result with all one-way changes and scenarios deeply below WTP. Parenteral MTX is a cost-effective therapy for patients with mild-to-moderate CD receiving hdCS. Therefore, it was swiftly and positively assessed by the local authority. To our knowledge, this is the first published cost-effectiveness analysis of parenteral MTX for this indication.