Patient Global Assessment Scores Research Articles

Real-world safety and efficacy of biologics in elderly patients with psoriasis: A multicenter observational study.

Clinical trials of biologics have frequently excluded elderly patients, resulting in inadequate data on their safety and efficacy. Additionally, evidence of their safety and efficacy remains limited, despite some real-world studies. To assess the safety and efficacy of biologics in elderly patients with psoriasis, we compared these outcomes in younger patients using data from the West Japan Psoriasis Registry (WJPR). The WJPR consists of approximately 30 facilities in Western Japan, including various healthcare settings. This study enrolled 1395 patients who participated in the 2022 follow-up survey of the WJPR and were either using or had used biologics during the survey. These included 456 patients in the elderly group (≥65 years) and 939 patients in the younger group (<65 years). Treatment-ending adverse events (TEAEs) occurred in 15.8% and 11.3% of elderly and younger patients, respectively. The incidence rate per 1000 patient-years (PY) for TEAEs was significantly higher in elderly patients than in younger patients (32.9 vs 23.2, p = 0.0234). Infectious diseases were more prevalent in the elderly group than the younger group; however, no significant difference in the frequency of infectious diseases was found between the two groups (p = 0.0807). Malignant neoplasms occurred significantly more frequently in the elderly group than in the younger group (p = 0.0169). Our results indicate a few concerns about infection when prescribing biologics to elderly patients. Biologics were effective for both elderly and younger patients. We found no significant differences in the proportion of patients with a body surface area score ≤3%, Physician's Global Assessment score 0/1, or Patient's Global Assessment score 0/1, as well as in the mean Dermatology Life Quality Index and the Itch Numerical Rating Scale between the younger and the elderly groups. Overall, our results confirm the appropriateness of using biologics in elderly patients with regard to safety and efficacy.

A Case Report of Complex Korean Medicine Treatment Including Modified Gamiguibi-tang in a Paraneoplastic Cerebellar Degeneration Patient with Dysarthria and Weakness of Distal Extremity

Objectives: This study investigated the treatment response to Korean medicine of a paraneoplastic cerebellar degeneration (PCD) patient with dysarthria and weakness of distal extremity.Case presentation: A 53-year-old female diagnosed with PCD complained of dysarthria and weakness of distal extremity. During 32-day hospitalization, she was treated with Korean medicine, namely, herbal medicine (modified Gamiguibi-tang), acupuncture (15 minutes twice a day at CV23, HT7, LI4, LR3, ST36, SP6, GB20, TE17, PC6, GV20, Ex-HN1, GV24 etc.), moxibustion, and cupping, combined with Western medicine (prednisolone, azathioprine) and physical therapy. Post-treatment, the patient global assessment (PGA) score of dysarthria fell from 100 to 60; grasp power rose from 15 kg to 19 kg and 13 kg to 17 kg in the right and left hands, respectively; and the 5-level EQ-5D version (EQ-5D-5L), EQ visual analogue scale (EQ-VAS), and Beck depression inventory (BDI-II) scores changed from 10 to 8, 0 to 80, and 34 to 7 respectively. Follow-up visits continued for about a month after discharge, improvement in symptoms maintained, and there were no significant side effects.Conclusions: Given the lack of standard treatment for PCD, Korean medicine can be tried clinically for the treatment of PCD patients with dysarthria and weakness of distal extremity. However, further studies with control groups are needed.

Addition of constitutional symptoms to the SLEDAI-2K improves overall disease activity assessment: A pilot study.

Constitutional symptoms (fatigue, lymphadenopathy, and weight loss) are not included in the SLE disease activity index-2000 (SLEDAI-2K). In this pilot study, we assessed the concurrent and construct validity of a revised SLEDAI-2K (SLED-R) that included these symptoms with the original SLEDAI-2K (SLED-O), using the physician global assessment of disease activity (PGA) as the reference. Our revised SLED-R substituted the SLED-O's fever descriptor with a constitutional descriptor that included fever, fatigue, lymphadenopathy, and/or weight loss. SLED-O, SLED-R, PGA and patient global assessment (PtGA) scores were collected prospectively. Bland-Altman correlations for repeated measures were calculated and Meng's z-test was used to compare correlations between dependent and overlapping correlation coefficients. Associations between constitutional symptoms and disease activity measures were analyzed using Mann-Whitney U, Kruskal-Wallis, Chi-square tests and repeated measures correlations. 1123 SLED-O, SLED-R, PGA, and 1066 PtGA were collected in 239 subjects. The new descriptor was scored in 45 subjects (18.8%) and 92 instances (8.1%), while the original descriptor, fever, was scored in only 4 subjects (1.7%) and 5 instances (0.4%). Mean SLED-O, PGA and PtGA scores were higher when the constitutional descriptor was scored versus not (p < .001). The correlation between SLED-R and PGA was marginally higher than between SLED-O and PGA (p < .001). Fatigue contributed most to this increase (p = .001) and associated with both higher PGA and PtGA scores (p < .001). Mean SLED-O and PGA scores were higher when ≥1 constitutional symptom(s) were scored versus not (p < .002). Correlations between PGA and PtGA when the new descriptor was scored versus not were similar (p = .860). The frequency of concordance between PGA and PtGA was lower when the new descriptor was scored (55%) versus not (72.5%), with PGA > PtGA when the new descriptor was scored (p < .001). The addition of constitutional symptoms to SLEDAI-2K, particularly fatigue, resulted in a marginal increase in its correlation with PGA, and new constitutional symptoms associated with higher SLED-O and PGA scores. As fatigue is subjective and difficult to attribute to SLE, its validity and inter-rater reliability in scoring remains uncertain. The clinical utility of SLED-R remains unclear, and further studies of its validity and reliability are needed.

E058 Disease activity measurement in rheumatoid arthritis - simpler in not always better!

Abstract Background/Aims Rheumatoid arthritis is one of the commonest inflammatory arthritis (1). The traditional method of disease activity assessment would incorporate activity measures like DAS 28, CDAI and SDAI which have been validated in clinical research (2). All these indices use 28 joint assessment method. These measures use different aspects of disease activity including swollen joint count, tender joint count, patient global scores and physician global assessment scores and inflammatory markers are included in some of the measures. We conducted a small study to compare these outcome measures in patients with moderate RA (per DAS criteria) who were recruited for disease activity assessment through MSK ultrasound. Methods Of the 30 biologic naive patients recruited, 27 were included in this study. Patients with moderate disease activity (per DAS criteria) were recruited for MSK ultrasound evaluation by a single rheumatologist. Patients with a diagnosis of fibromyalgia were excluded. These patients had been reviewed by two assessors prior to USS assessment and disease activity measures updated. Patient data was collected through electronic patient records. Data was analysed through MS Excel. Results The median age was 63. There were 66% females and 34% male patients. Majority were sero positive for either RhF, Anti CCP or both (77%). Mean DAS-28 score was 4.4 and corresponding mean CDAI and SDAI scores were 22.8 and 23.5 respectively. Mean HAQ score 1.08 and median CRP was 8.6. The median composite EULAR OMERACT synovitis score was 9.18. The average physician global assessment score for disease activity before USS was 4.9/10 and it correlated well with decision to escalate treatment in 41% patients as a result of USS assessment. Conclusion With a small sample size, this project shows that simplified disease activity measures do not necessarily provide clear evidence of disease activity and are heavily influenced by patient reported outcome measures (tender joint count and global pain scores). CDAI scores can be slightly higher compared to DAS and SDAI scores for the similar patients. Physician assessment and ultrasound can provide robust evaluation of disease activity and help in decision-making. A multicentric study with a large simple size to evaluate these findings would be helpful. Judicious use of clinical evaluation and imaging can help in tailoring treatment according to patient’s clinical condition and can also save money. Disclosure H. Crawshaw: None. E. Gates: None. C. Acebes: None. M. Jamal: None.

Clinical value of the Patient Global Assessment with Ankylosing Spondylitis: A cross-sectional study.

To analyze the factors associated with the overall patient condition and explore the clinical value of the Patient Global Assessment (PGA) index for assessing the disease state in patients with Ankylosing Spondylitis (AS). This cross-sectional study used a standardized questionnaire to record the basic information of patients with AS. The collected data included the Ankylosing Spondylitis Disease Activity Score (ASDAS)-C-reactive protein (CRP), ASDAS-erythrocyte sedimentation rate (ESR), Bath Ankylosing Spondylitis Disease Activity Index (BASDAI), PGA, and other clinical indicators. Statistical analysis was performed using SPSS 25.0 software, and the scale was assessed for retest reliability and structural validity. The Kruskal-Wallis H test and Spearman or Pearson correlation analysis were used to analyze the factors influencing PGA scores. The receiver operator characteristic (ROC) curve was used to identify the cutoff value of the PGA for predicting disease activity in AS. The patient age, disease duration, family history, and history of ocular inflammation significantly differed between PGA groups (P < .05). The median PGA was significantly lower in patients with disease remission than in those with disease activity (P < .01). The various clinical indexes significantly differed between PGA groups (P < .01). The PGA was significantly correlated with various clinical indicators (P < .01). The area under the ROC curve (AUC) for disease activity based on the ASDAS-CRP was 0.743 (P < .01) with a PGA cutoff value of 1.38; the AUC for disease activity based on the BASDAI was 0.715 (P < .01) with a PGA cutoff value of 1.63. The PGA was significantly correlated with patient-reported outcomes, disease activity, function, and psychological status, and may indicate the level of inflammation in patients with AS. A PGA of around 1.5 indicates disease activity.

Patient-Reported Outcomes (PROs) and PRO Remission Rates in 12,262 Biologic-Naïve Patients With Psoriatic Arthritis Treated With Tumor Necrosis Factor Inhibitors in Routine Care.

To evaluate patient-reported outcomes (PROs) after initiation of tumor necrosis factor inhibitor (TNFi) treatment in European real-world patients with psoriatic arthritis (PsA). Further, to investigate PRO remission rates across treatment courses, registries, disease duration, sex, and age at disease onset. Visual analog scale or numerical rating scale scores for pain, fatigue, patient global assessment (PtGA), and the Health Assessment Questionnaire-Disability Index (HAQ-DI) from 12,262 patients with PsA initiating a TNFi in 13 registries were pooled. PRO remission rates (pain ≤ 1, fatigue ≤ 2, PtGA ≤ 2, and HAQ-DI ≤ 0.5) were calculated for patients still on the treatment. For the first TNFi, median pain score was reduced by approximately 50%, from 6 to 3, 3, and 2; as were fatigue scores, from 6 to 4, 4, and 3; PtGA scores, from 6 to 3, 3, and 2; and HAQ-DI scores, from 0.9 to 0.5, 0.5, and 0.4 at baseline, 6, 12, and 24 months, respectively. Six-month Lund Efficacy Index (LUNDEX)-adjusted remission rates for pain, fatigue, PtGA, and HAQ-DI scores were 24%, 31%, 36%, and 43% (first TNFi); 14%, 19%, 23%, and 29% (second TNFi); and 9%, 14%, 17%, and 20% (third TNFi), respectively. For biologic-naïve patients with disease duration < 5 years, 6-month LUNDEX-adjusted remission rates for pain, fatigue, PtGA, and HAQ-DI scores were 22%, 28%, 33%, and 42%, respectively. Corresponding rates for patients with disease duration > 10 years were 27%, 32%, 41%, and 43%, respectively. Remission rates were 33%, 40%, 45%, and 56% for men and 17%, 23%, 24%, and 32% for women, respectively. For patients aged < 45 years at diagnosis, 6-month LUNDEX-adjusted remission rate for pain was 29% vs 18% for patients ≥ 45 years. In 12,262 biologic-naïve patients with PsA, 6 months of treatment with a TNFi reduced pain by approximately 50%. Marked differences in PRO remission rates across treatment courses, registries, disease duration, sex, and age at onset of disease were observed, emphasizing the potential influence of factors other than disease activity on PROs.

Patient and physician assessment in difficult-to-treat rheumatoid arthritis: patterns of subjective perception at early stages of b/tsDMARD treatment

ObjectivesTo analyse the trajectories of Disease Activity Score 28 (DAS28), patient global assessment (PGA) and physician global assessment (PhGA) and to assess their predictive capabilities on difficult-to-treat rheumatoid arthritis (D2TRA)...

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ZY-IL1 in Three Patients with Cryopyrin-Associated Periodic Syndromes.

We present the first results of the proof-of-concept phase 2a study of oral NLRP3 inflammasome inhibitor in subjects with cryopyrin-associated periodic syndromes (CAPS). Three adult subjects with a confirmed diagnosis of CAPS were enrolled and administered 50mg of ZYIL1 twice daily for 7 days. A total of 5 treatment-emergent adverse events (TEAEs) were reported in 2 subjects. All 5 TEAEs were mild in severity and considered unrelated to the study drug. At steady state, the average plasma concentration and trough concentration ranged from 2.5 to 4.2 and 1.4 to 2.5µg/mL, respectively. Inflammatory markers and disease activity (physician and patient global assessment score) decreased notably 12 hours post-last dose.

Disease activity and disease-related factors are drivers of patient global assessment in rheumatoid arthritis: a real-life cross-sectional study

Despite that the Patient Global Assessment (PGA) is widely used for measuring Rheumatoid Arthritis (RA) disease activity to define the remission state of the disease, the primary contributors influencing patients’ ratings are still debated. This study aims to determine which clinical, sociodemographic and lifestyle-related contextual factors might be key drivers of PGA in RA. This single-center cross-sectional study recruited 393 consecutive adult RA patients. Median age 60 years, females 306 (77.9%). Data related to disease activity were assessed by using Simplified Disease Activity Index (SDAI), severity by Health Assessment Questionnaire (HAQ), and impact by RA Impact of Disease (RAID). Sociodemographic/lifestyle features were collected. Disease remission was calculated using Boolean-based criteria 1.0 and 2.0. Quantile regression models were used for univariate and multivariate analysis. The remission rate progressively increased from 15% by using SDAI with a Boolean 1.0-based definition to 43.5% using a Boolean 2.0-based remission. Among factors related to disease activity, the use of low-dose corticosteroids, the RAID items pain and sleep difficulties were predictive for worse PGA scores (p = 0.01). Among factors related to disease severity HAQ score and RAID total were independent factors associated with higher median PGA (p = 0.02 and p < 0.001). RAID’s physical well-being was related to PGA scores (p = 0.01). An increasing trend in PGA was observed in longstanding diseases (> 15 years). Our results confirmed that there is no unambiguous interpretation of the PGA score. It is a measure related to some disease activity parameters, but it is also influenced by contextual factors related to disease severity and impact. These data highlighted that PGA should have a broad interpretation, thus supporting the proposal of a dual targets (biological and impact) approach to obtain a more accurate estimate of disease activity.

E022 Effect of upadacitinib in a patient with primary Sjogren syndrome: a case report

Abstract Background/Aims Primary Sjogren syndrome (pSS) is a chronic autoimmune multi-system disease characterized by inflammation of exocrine glands, particularly the lacrimal and salivary glands, as well as multiple extra-glandular involvements. Treatment is often challenging, and until now, no disease-modifying anti-rheumatic drugs (DMARDs) have been approved for this condition. There is a lack of data about the efficacy of Janus kinase inhibitors in pSS. Here we report a patient with pSS successfully responding to upadacitinib. Methods A 68-year-old woman presented with pain in multiple joints for 16 years with intermittent use of NSAIDs. She had no low back pain and no self or family history of psoriasis or red eye. She denied dry mouth but required frequent water drinking during meals to aid swallowing and occasionally used artificial tears. MTX was started for her two-month articular flare and positive rheumatoid factor (RF). MTX caused severe vomiting and hyponatremia, and she was hospitalized. After the correction of hyponatremia, she was put on tofacitinib. We reevaluated the case when she came to us. She had grade 2-3 tenderness of most peripheral joints and was bedridden. Immunological parameters revealed RF 127 IU/ml (reference value &amp;lt;14), anti SSA antibody 147 U/ml (normal &amp;lt;3.2), anti SSB antibody 149 U/ml (reference value &amp;lt;8) and anti CCP antibody; ANA, anti-ds-DNA Ab were negative. There was no erosion on the X-ray hands. Schirmer's test was positive (3 mm bilaterally). Unstimulated salivary flow after five minutes was 0.04 ml/min (≤0.1 ml/min indicates salivary hypofunction). She fulfilled the 2016 ACR/EULAR classification criteria, and we diagnosed her with primary Sjogren syndrome. Hydroxychloroquine was initiated, but she discontinued it for anorexia. Considering her age, she was switched to upadacitinib, as European Medicines Agency restricted the use of tofacitinib beyond 65 years. Results Her EULAR Sjogren Syndrome's Disease Activity Index (ESSDAI) was six at baseline, indicating moderate disease activity. Her joint pain improved significantly within two months with infrequent use of pilocarpine in the sixth month. Six months after treatment with upadacitinib, ESSDAI came down to 2; the Patient's Global Assessment score was 02 and achieved STAR (Sjogren's Tool for Assessing Response) response 7 (STAR responder if score ≥5). Conclusion To our knowledge, this is the first case report on the efficacy of upadacitinib in pSS. Further high-quality clinical trials are required to confirm the benefit of upadacitinib in pSS. Disclosure M. Momen Majumder: None. S. Haq: None.

Comparative effectiveness and safety of topical methimazole 5% monotherapy versus combination of Q-Switched Nd: YAG Laser and topical methimazole 5% in patients with refractory melasma.

Melasma is an acquired pigmentary disorder which currently has no definitive treatment. Although topical drugs containing hydroquinone are the basis of treatments, they are usually associated with recurrence. We aimed to evaluate the effectiveness and safety of monotherapy with topical methimazole 5% versus combination of Q-Switched Nd: YAG Laser and topical methimazole 5% in patients with refractory melasma. A total of 27 women with refractory melasma were included. We applied topical methimazole 5% (once a day) with three passes of QSNd: YAG laser (Wavelength: 1064 nm, pulse energy: 750 mJ, fluence: 1.50 J/cm2 , spot size: 4× 4 mm, hand piece: fractional, JEISYS company) for six sessions on the right half of the face, and topical methimazole 5% (once a day) on the left half of the face, for each patient. The treatment course was 12 weeks. Evaluation of effectiveness was done with the Physician Global Assessment (PGA), Patient Global Assessment (PtGA), Physician satisfaction (PS), Patients satisfaction (PtS), and mMASI score. PGA, PtGA, and PtS were not significantly different between the two groups at any time (p> 0.05). PS in the laser plus methimazole group was significantly better than methimazole group at 4th, 8th, and 12th weeks (p< 0.05). The rate of PGA improvement in the combination group was significantly better than the monotherapy over time (p< 0.001). The changes of mMASI score between the two groups did not significantly differ at any time (p> 0.05). There was no significant difference in the adverse events between the two groups. Combination therapy with topical methimazole 5% and QSNY laser can be considered as an effective way to treat refractory melasma.

Lupus low disease activity state attainment in the phase 3 TULIP trials of anifrolumab in active systemic lupus erythematosus

ObjectivesIn patients with systemic lupus erythematosus (SLE), lupus low disease activity state (LLDAS) attainment is associated with improved outcomes. We investigated LLDAS attainment in anifrolumab-treated patients.MethodsWe performed post hoc analysis...

An Open-Label Non-Randomized Preliminary Noninferiority Study Comparing Home-Based Handheld Narrow-Band UVB Comb Device with Standard Hospital-Based Whole-Body Narrow-Band UVB Therapy in Localized Vitiligo.

Narrow-band ultraviolet B (NB-UVB) is the standard therapy for vitiligo. The objective of this study is to compare the safety and clinical efficacy of a handheld NB-UVB comb device with the standard whole-body NB-UVB therapy in localized stable vitiligo. Thirty-one vitiligo patients were allocated to either daily therapy with a home-based handheld comb device (group A, n = 17) or thrice-weekly hospital-based whole-body NB-UVB therapy (group B, n = 14) for 4 months, based on their preference. The primary and secondary outcomes were assessed at each follow-up, and appropriate statistical tools were used for analysis. Of the 31 patients enrolled, 26 patients (study groups A/B: 15/11) completed the study. Primary outcome: Median percentage repigmentation of the representative patch in groups A and B were 51.35% and 63.85%, respectively (P = 0.64). The median size reduction of the representative patch in both groups was statistically significant (P < 0.05). The mean difference between "per protocol analysis" and "intention to treat" showed noninferiority. Secondary outcomes: Both groups were comparable on Lund and Browder score, patient global assessment and investigator global assessment scores, adverse events, color match, and change in the quality of life. The comparison group had a significantly greater number of missed sessions (P = 0.02). The majority of patients had a "good" response in both groups. Handheld NB-UVB comb device daily with a fixed dose of fluence was found to be noninferior with better compliance to standard whole-body NB-UVB therapy.

Efficacy and Safety of Autologous Serum Therapy in Chronic Spontaneous Urticaria in the Pediatric Population: A Prospective Pilot Study.

Chronic spontaneous urticaria (CSU) in children is mostly spontaneous in onset (57%). Treatment comprises long-term antihistaminic therapy without need for elaborate investigations. A subset of such patients don't respond to conventional treatment and novel therapies to help reduce pill burden is the need of the hour. To determine the efficacy and safety of autologous serum therapy (AST) in pediatric patients with chronic spontaneous urticaria. All pediatric patients, aged between 6-16 years, attended to our OPD from March 2019 to March 2020 were recruited. Clinico-demographic data and baseline investigations of all patients were performed. Two-weekly AST therapy was given for 8 visits with levocetrizine tablet 5mg on an on-demand basis. Urticaria activity score (UAS) sheet was provided to record and return every 2 weeks. Statistical analysis was done using the IBM SPSS 26 software package. Autologous serum skin test (ASST) was positive in 63% patients. Both the ASST positive and ASST negative group showed significant reduction in UAS7 score at week 14 compared to baseline. The reduction in mean UAS7 score was associated with a decreased pill burden and positive response in the patient and physician global assessment scale. No statistically significant difference between the two groups in terms of mean UAS7 reduction was found. This study has explored the efficacy and safety of autologous serum therapy in the pediatric CSU patients. Both ASST positive and ASST negative group respond to AST therapy.

Clinical Efficacy and Skin Bioengineering Evaluations of a Semi-Occlusive Healing Ointment as an Adjuvant Therapy in Hand Eczema

Background: Hand Eczema (HE) is the most common skin problem during SARS-CoV-2 pandemic which has impaired quality of life, impact work ability and cause hand dysfunction. The use of Topical Corticosteroid (TCS) alone can delay HE healing. Objective: To determine the efficacy of a semi-occlusive ointment containing panthenol, glycerol and bisabolol as an adjuvant therapy to TCS in mild-to-moderate HE. Methods: An open-label prospective study was conducted of 60 patients with mild-to-moderate HE. The tested product was applied to both hands, two or three times a day every 4 to 6 hours for 8 weeks. There was then a 4-week cessation period. Disease severity was assessed by physician/patient scoring systems, Corneometer, Tewameter and Visioscan that were collected at week 0, 2, 4, 8 and 12. Results: Fifty-six patients completed the study. The patients had a mean age of 42.8 years and were mostly female. The median duration of HE was 12.0 years. The physician and patient global assessment scores of clinical severity; erythema, dryness, itching and functional impairment, were significantly reduced starting at week 2 compared with baseline. After the 4-week cessation of the tested product, patient loosed the product efficacies. The proportion of patients who used TCS tended to decrease during the study period. Skin hydration was significantly improved at week 4. No unwanted effects found. Conclusion: A semi-occlusive healing ointment with panthenol, glycerol and bisabolol was effective and safe for treating mild-to-moderate HE. Our study identified an adjuvant ointment choice for HE treatment other than TCS.

Low-dose rituximab is efficacious in refractory idiopathic inflammatory myopathies.

Rituximab (RTX) use early in the course of refractory idiopathic inflammatory myopathy (IIM) is not well studied. This study sought to determine the short-term efficacy of RTX in a registry-based cohort of refractory IIM. Registry-based observational data about IIM patients receiving RTX between 2018 and 2021 were included. Total improvement score was calculated from the core set measures as per International Myositis Assessment and Clinical Studies group (IMACS) at baseline, 6 months and 12 months of follow-up. Forty-two patients (F:M, 29:13), with a mean (s.d.) age of 39.5 (11.5) years were studied. Majority of patients received RTX for refractory myositis, after a median (interquartile range) duration of 8 (4,18) months. Twenty-eight received RTX at a dosage of 1 g × two doses, while 14 received 500 mg × two doses with an interval of 15 days. At 6 months and 12 months post-RTX, the improvement was recorded in manual muscle testing (MMT-8) scores, physician global assessment (PGA), patient global assessment (PtGA) and median steroid dosage as compared with the baseline (P < 0.01 for all). A mean (s.d.) improvement of 44.5 (16) and 48.7 (19.2) in total improvement score was recorded at 6 and 12 months, respectively. The change in MMT-8, PGA and PtGA scores from baseline between the two dosage regimens of RTX were comparable at 6 and 12 months. Severe lower respiratory tract infections requiring hospitalization occurred in three patients of the cohort. RTX improved IMACS core set measures and had steroid sparing efficacy at 6 and 12 months in patients with IIM in this registry-based study. Rituximab as an induction regimen of two doses of 500 mg can be as efficacious as 1 g at 6 months and 12 months of follow-up.

Coping strategies, emotional distress and perceived disease severity in a cohort of patients with rheumatoid arthritis: a mediation analysis.

Few data are available on the role of emotional distress as a possible mediator of the relationship between coping strategies and the Patient Global Assessment (PGA). This study aims to investigate, in a large cohort of patients affected by RA, the relationship between specific copying strategies and PGA, and the role of emotional distress as a mediator. A total of 490 patients with RA completed a set of standardized assessments including the self-reported PGA, the Coping Orientation to the Problems Experienced (COPE-NVI) and the Hospital Anxiety and Depression Scale (HADS). A mediation analysis was conducted to investigate the role of emotional distress. The effect of coping strategies on the PGA score was significantly mediated by the emotional distress for religious (total effect mediated 42.0%), planning (total effect mediated 17.5%), behavioural disengagement (total effect mediated 10.5%), and focus on and venting of emotions (total effect mediated 9.8%). Seven coping strategies (acceptance, positive reinterpretation and growth, active coping, denial, humour, substance use-mental disengagement) resulted directly associated to PGA total score, but no mediation effect was found. The remaining four coping strategies were not associated to the PGA score. This study suggests that coping strategies could be an important factor in the perceived disease severity. Consequently, in order to reduce PGA in patients with RA, a useful tool could be represented by the implementation of psychological interventions aiming to modify the specific coping styles. Moreover, to prevent or treat emotional distress seems to further reduce PGA.

238 Cutaneous dermatomyositis area and severity index activity score (CDASI-A) and associated patient-reported outcomes in a phase 2 clinical trial in dermatomyositis

Retrospective reviews of clinical databases from two sites have identified strong associations between skin activity in dermatomyositis (DM), as measured by CDASI-A, and patient-reported quality-of-life (QoL). We hypothesized that associations between CDASI-A and QOL outcomes could also be demonstrated in the clinical trial setting. We evaluated correlations between CSADI-A and PROMIS Short Form domain scores, Skindex domain scores, Patient Global Assessment (PtGA) scores, and Physician Global Assessment (PGA) scores in a double-blind, randomized, placebo-controlled clinical trial. Data were from a 5-visit, 16-week Phase 2 trial of lenabasum, a cannabinoid receptor type 2 agonist, in 22 subjects with DM. CDASI-A scores and QOL outcomes collected at each study visit were correlated using linear mixed effect models to account for within-subject variability and repeated measures. As our goal was to correlate CDASI and QoL throughout the trial, analysis was performed without regard to treatment. Improvement in CDASI-A significantly correlated with PROMIS social role (p≤0.05). No other PROMIS domain scores correlated with the CDASI. The CDASI-A also significantly correlated with all Skindex scores (symptoms, functioning, emotions, and itch), all PtGA scores (global disease, itch, pain, and global skin), and all PGA scores (global disease, skin activity, and global skin), p<0.001 each. All reported correlations were in the appropriate direction. Our findings support that CDASI-A reflects both skin disease and QOL and is an appropriate outcome in DM clinical trials. Most PROMIS domains did not correlate with skin activity suggesting that Skindex and PtGA scores may better relate to skin activity as measured by the CDASI.

Calcipotriol: A novel tool in treatment of acne vulgaris.

Retinoids and active vitamin D3 analogues regulate the proliferation and differentiation of keratinocytes. Retinoids are the main stay in the treatment of acne vulgaris through their comedolytic and anti-inflammatory effects. However, the effect of calcipotriol on the different forms of acne lesions has not been reported. This split face prospective study aimed to detect the efficacy of topical calcipotriol in the treatment of acne lesions in comparison with that of adapalene. Forty patients with acne vulgaris were treated with topical calcipotriol (0.005%) cream and 0.1% adapalene gel on the right and left sides of the face respectively. Clinical and histological assessment of the used treatments was done 2months after the start of treatment. Two months after treatment, there was significant reduction of all acne lesions with significant decrease of physician global assessment and patient global assessment scores (p=0.0001) on both sides of the face with no significant difference between both sides. Histologically, there was significant decrease in the density of inflammatory infiltrate, which was more significant on the right side (p < 0.0001). Topical calcipotriol can serve a significant role in the treatment of acne vulgaris, through its anti-inflammatory effect which was comparable to that of adapalene.

Factors influencing patient-reported outcomes in ANCA-associated vasculitis: correlates of the Patient Global Assessment

BackgroundPatient-reported outcomes (PROs) are currently poorly integrated in the clinical evaluation of disease activity in patients with ANCA-associated vasculitis (AAV). ObjectivesTo assess the distribution of the Patient Global Assessment (PtGA) in patients with AAV in stable remission, and to identify correlates of PtGA; to assess the discordance between PtGA score and Physician Global Assessment (PhGA). MethodsPatients with a diagnosis of AAV in stable remission (BVAS=0) and with a Physician Global Assessment (PhGA)=0 were included. The following PROs on a 0-100 visual analogue scale (VAS) were assessed: PtGA, fatigue, pain, general health, sleep quality, and chronic damage according to the patient's opinion. The Cragg Hurdle model was used to assess the predictors of PtGA. Results65 patients were included, female 57%, mean age 61±12 years. Median disease duration 6 years (IQR 3-12). Vasculitis damage index (VDI) was 4.4±2.3. Despite having been classified as being in remission, PtGA was elevated in 37% of patients. The PtGA was not associated with older age, comorbidities, educational level, the type of AAV diagnosis, number of organ-systems involved, previous relapses, disease duration, nor higher VDI. Female sex was significantly associated with PtGA: 51% of female patients reported an elevated PtGA compared to 18% of males (p=0.009). PtGA significantly correlated with all the other assessed PROs. Pain and fatigue were the main determinants of an elevated PtGA. ConclusionsA significant proportion of patients with AAV considered to be in remission by the physician still declares to have persistent aspects of uncontrolled disease. PtGA is significantly influenced by pain and fatigue, which should receive more attention in the future assessment of patients with AAV.