Physician Visual Analog Scale Research Articles

A single‐blinded, randomized, controlled trial comparing efficacy between low‐fluence alexandrite 755‐nm picosecond laser and low‐fluence neodymium‐doped yttrium aluminum garnet (Nd:YAG) 1064‐nm picosecond laser for the treatment of ultraviolet B‐induced hyperpigmentation

Hyperpigmentation is a common concern of patients in dermatology clinics. Although there are many treatment options, lasers are considered a promising therapy for various hyperpigmentary conditions. This study aims to evaluate the efficacy of alexandrite 755-nm picosecond and neodymium-doped yttrium aluminum garnet (Nd:YAG) 1064-nm picosecond lasers for the treatment of ultraviolet B (UVB)-induced hyperpigmentation in Asians. A randomized, single-blinded study was conducted. UVB-induced hyperpigmentation was performed in three spots by narrowband UVB. After 2weeks, these three spots were allocated into 755-treated, 1064-treated, and control sites. Patients received weekly laser treatments for five sessions. Follow-ups were scheduled at 1 and 2months after the last session. Twenty patients attended the study. Overall, 755-nm and 1064-nm picosecond lasers showed a significant improvement in the mean lightness index (L*) compared to the control site, which started at Day49 and Day77, respectively. The mean L* of the 755-nm-treated site was also higher than that of the 1064-nm-treated site at Day105 (p≤0.001). Initially, the mean L*, physician's visual analog scale (VAS), and patient satisfaction with the 1064-nm picosecond laser were better than those with the 755-nm picosecond laser. Nevertheless, an inversion of the mean L* and VAS was noted at Day49, whereas the mean patient satisfaction was noted at Day77. In the subgroup analysis, a 755-nm picosecond laser effectively treated Fitzpatrick skin types (FPTs) III and IV. However, the mean L* of the 1064-nm picosecond laser was not significantly different from that of the control for FPT4. The alexandrite 755-nm picosecond and Nd:YAG 1064-nm picosecond lasers appear to be effective and safe modalities for treating UVB-induced hyperpigmentation. With the setting employed in this study, the outcome after the 755-nm picosecond laser treatment seemed superior to that of the 1064-nm picosecond laser treatment, especially for FPT4.

Comparative Study of Efficacy and Safety of Microneedling with Topical Autologous Platelet Rich Plasma Versus Microneedling Alone in Facial Atrophic Acne Scars

Introduction: Atrophic acne scars are dermal depressions resulting from destruction of collagen after inflammatory acne which can lead to poor self-image and treating them can help improve patients’ self-esteem. Objectives: This study compared the efficacy and safety of combination of microneedling using dermaroller with topical application of autologous platelet rich plasma versus microneedling using dermaroller alone in facial atrophic acne scars. Methods: Patients with atrophic acne scars were randomized into 2 groups of 20 patients each and treated with either dermaroller alone or with a combination of dermaroller and topical application of autologous PRP at 4 weeks intervals for 16 weeks. Final response was evaluated at 4 weeks after the last sitting. Goodman and Baron’s qualitative acne scarring grading system, Goodman and Baron’s quantitative acne scar grading system, physician’s global assessment (PGA) and visual analogue scale were used to evaluate the treatment outcome in both the groups. Side effects were also noted at each visit. Results: At the end of treatment, improvement in Goodman and Baron’s qualitative acne scar grades, decrease in mean Goodman and Baron’s quantitative acne scar scores, percentage decrease in mean Goodman and Baron’s quantitative acne scar scores, physician global assessment and visual analogue scale were significantly higher in patients treated with dermaroller in combination with topical autologous PRP as compared to patients treated with dermaroller alone without much adverse effects. Conclusion: The combination approach using dermaroller and PRP was a better treatment option compared to dermaroller alone in atrophic acne scars with similar safety profile.

AB0773 THE INCIDENCE OF RESIDUAL DISEASE ACTIVITY FOLLOWING DIVERSE DISEASE ACTIVITY MEASUREMENTS FOR PSORIATIC ARTHRITIS

Background:Due to the complex nature of psoriatic arthritis (PsA), diverse disease activity measures have been developed, the most common of which include Disease Activity Score for Psoriatic Arthritis (DAPSA), Minimal Disease Activity (MDA) and Very Low Disease Activity (VLDA). Recently, new composite measures have been developed such as Psoriatic Arthritis Disease Activity Score (PASDAS) and GRACE index. Due to different domains and assessments, even though these measures may indicate remission or low disease activity, residual disease activity (RDA) may persist.Objectives:The aim of this study was to evaluate RDA in patients with PsA.Methods:A total of 148 patients (105 female, 43 male; mean age 47.5 (SD:12.6) years) who met the CASPAR criteria for PsA were recruited. Demographic and clinical characteristics of patients were recorded, including pain visual analog scale (VAS), joint VAS, patient global VAS, and tender and swollen joint counts. Evaluations included the Leeds Enthesitis Index (LEI), Psoriasis Area and Severity Index (PASI), Psoriatic Arthritis Quality of Life (PsAQoL), Short-Form 36 Health Survey (SF-36) and Health Assesment Questionnaire (HAQ). Disease activity and remission were assessed using the DAPSA, MDA, VLDA, PASDAS and GRACE Index. MDA was calculated with 5 and 6 positivity criteria separately. Low disease activity (LDA) was defined as follows: DAPSA≤14, PASDAS≤3.2 and GRACE Index≤2.3. RDA was defined as the presence of at least one of the following criteria despite remission or LDA: tender and/or swollen joints >1, dactilitis >1, LEI>1, HAQ>0.5, PASI>1, PtGA>20, physician VAS>20, or pain VAS>15.Results:The mean duration of disease was 68.2 (SD:80.2) months. DAPSA-LDA, PASDAS-LDA, GRACE-LDA, MDA and VLDA were observed in 48.6%, 14.6%, 14.9%, 23.6% and 2% of PsA patients, respectively. RDA as determined by at least one domain was identified in 91%, 95%, 86% and 86% of patients who were classified as having MDA, DAPSA-LDA, PASDAS-LDA and GRACE-LDA, respectively. Undetected RDA was most common with DAPSA, whereas VLDA completely ruled out RDA. PASDAS and GRACE resulted in similar rates of RDA (Table-1). With DAPSA-LDA, the incidence of RDA in the pain domain was significantly lower with older age. Female patients had higher rates of RDA with the LEI and HAQ (p<0.05).Conclusion:VLDA was the most and DAPSA was the least sensitive method to detect remission/LDA. RDA should be kept in mind in patients with PsA when using current measures to assess remission or LDA.

OP0290 EMAPALUMAB (ANTI-INTERFERON-GAMMA MONOCLONAL ANTIBODY) IN PATIENTS WITH MACROPHAGE ACTIVATION SYNDROME (MAS) COMPLICATING SYSTEMIC JUVENILE IDIOPATHIC ARTHRITIS (SJIA)

Background:MAS is a severe complication of rheumatic diseases and occurs most frequently in patients with sJIA. Data from animal models and from observational studies in patients suggest that interferon gamma (IFNy) is a driver of the hyperinflammation and hypercytokinemia observed in MAS.Objectives:To assess the pharmacokinetics, efficacy, and safety of intravenous (IV) infusions of emapalumab, a fully human anti-IFNγ monoclonal antibody, in patients with MAS in the context of sJIA.Methods:This ongoing, pilot, open-label, single-arm study (NCT03311854) includes patients with MAS (2016 ACR/EULAR criteria) on a background of confirmed, or high presumption of, sJIA, and with inadequate response to high-dose IV glucocorticoids. Emapalumab is initiated at 6 mg/kg (1 dose) and continued at 3 mg/kg twice weekly for a total of 4 weeks, or less upon achievement of complete response (CR). CR is defined as an absence of MAS clinical signs plus white blood cell and platelet counts above the lower limit of normal, LDH, AST and ALT <1.5 x upper limit of normal, fibrinogen >100 mg/dL, and ferritin decreased by ≥80% or to <2,000 ng/mL.Results:We report preliminary data from the first 9 patients (median age [range] 11.6 [2.1-25.3] years) enrolled (7 in Europe and 2 in the USA). All patients had failed high-dose methylprednisolone, of which there were prior treatment failures from cyclosporin A (n=4) and from anakinra (n=4). Treatment with emapalumab resulted in rapid IFNγ neutralization, as demonstrated by the decrease in CXCL9 levels (Figure 1), and subsequent deactivation of T cells, as indicated by the decrease in sIL-2R levels. CR was achieved in all patients after a median of 23 (12-56) days. A progressive improvement in all clinical and laboratory parameters of MAS was observed (Table 1 and Figure 2). Glucocorticoids were tapered in all patients (median % tapering -92%; range -45% to -98% at Week 8). Emapalumab infusions were well tolerated by all patients, with no discontinuation. CMV reactivation was reported in 1 patient as a serious event possibly related to emapalumab and resolved with antiviral treatment.Table 1.Time to response for key clinical and laboratory parameters.ParametersMedian baseline value (range)Median days of treatment (range)D-dimers to <1000 mg/L12,480 (550-89,552)15 (1-49)sIL-2R to <2000 ng/L4596 (1664-20,954)21 (6-37)Ferritin <500 mg/L29,240 (716-192,584)21 (9-42)Physician visual analog scale of MAS activity ≤19.0 (2-10)19 (9-56)All MAS laboratory parameters within range of CRNA21 (15-55)All MAS parameters within range of CRNA23 (12-56)Glucocorticoid tapering at ≤1 mg/kg prednisolone equivalent*NA42 (16-50)*Data incomplete for 1 patientFigure 1.Rapid neutralization of IFNy. Each line represents an individual patient (n=9).Figure 2.Ferritin levels and platelet counts over time.Conclusion:Emapalumab administration led to rapid neutralization of IFNy and was efficacious in controlling MAS with a favorable safety profile. These results support the pathogenic role of IFNγ in MAS/sJIA and the therapeutic value of IFNγ neutralization in MAS patients who have failed standard of care.Disclosure of Interests:Fabrizio De Benedetti Grant/research support from: AbbVie, Pfizer, Novartis, Novimmune, Sobi, Sanofi, Roche, Speakers bureau: AbbVie, Novartis, Roche, Sobi, Paul Brogan Grant/research support from: Sobi, Novartis, Roche, Chemocentryx, Consultant of: Roche, Sobi, Speakers bureau: Sobi, Roche, Novartis, UCB, Claudia Bracaglia: None declared, Manuela Pardeo: None declared, Giulia Marucci: None declared, Emanuela Sacco: None declared, Despina Eleftheriou Speakers bureau: Sobi, Charalampia Papadopoulou: None declared, Alexei Grom Grant/research support from: Novartis, AB2Bio, Consultant of: Novartis, Pierre Quartier Consultant of: AbbVie, Chugai-Roche, Lilly, Novartis, Sanofi, Sobi, Speakers bureau: AbbVie, BMS, Chugai-Roche, Novartis, Pfizer, Sobi, Rayfel Schneider Grant/research support from: Roche, Novartis, Sobi, Pfizer, Consultant of: Sobi, Novartis, Novimmune, Philippe Jacqmin Consultant of: Sobi, Rikke Frederiksen Employee of: Sobi, Maria Ballabio Employee of: Sobi, Cristina De Min Employee of: Sobi

Factors Associated With Response to Anorectal Biofeedback Therapy in Patients With Fecal Incontinence

Anorectal biofeedback (BF) is commonly used to treat patients with fecal incontinence (FI), but demand usually exceeds availability. It is therefore important to identify patients most likely to respond to BF treatment. We aimed to identify pre-treatment clinical or physiologic factors that might be used to predict completion and success of BF in women with FI. We analyzed data from 400 women with FI (mean age, 61 ± 14 y) undergoing instrumental BF in a tertiary care setting from 2003 through 2016. All patients completed questionnaires before BF, including Rome and the hospital anxiety and depression scale questionnaires. Histories of medication use, surgery, medical conditions, and bowel pattern were recorded, urge was assessed, and patients kept stool diaries. Before and after treatment (6 weekly sessions with a gastroenterologist-supervised nurse specialist, 4 involving instrumented anorectal biofeedback), patients were examined by a physician and fecal incontinence severity index and visual analogue scale scores were recorded. The main outcome measure was response to therapy, defined as improvement of 50% or more in weekly FI episodes at the end of BF compared with before BF. The BF treatment was completed by 363 women (91%); of these, 62 had low baseline symptom frequency (no FI episodes in the 2 weeks before BF). Younger age was associated with failure to complete treatment. Of the 301 patients remaining, 202 patients (67%) had a response to therapy; among these women, urge FI was associated with response at end of BF, but not at follow up (6 months after therapy). Baseline severity of symptom scores and quality of life measures were associated with greater improvement in the same variable at the end of BF and after 6 months. Patients with low baseline symptom frequency improved in all secondary outcome measures, similar to patients with higher baseline symptom frequency. In an analysis of 363 women with FI, approximately two-thirds had a response to BF treatment. Urge FI was the only baseline variable associated with response. Baseline severity of symptoms and quality of life measures were associated with greater improvement in the same variable, but not overall response. It is therefore a challenge to select treatment for patients with FI.

The burden of subclinical intra-articular inflammation in gout.

To assess the burden of subclinical intra-articular inflammation using ultrasound in people with gout. A pilot, prospective longitudinal cohort of 28 participants with gout were examined twice, once during a gout flare (n=25) and then during an inter-critical phase (n=27). At each visit, a 52 joint count was done followed by ultrasound examination for detection of intra-articular power Doppler (PD) signal. Clinically active joints were defined as tender and swollen. Data was collected on patient reported gout pain - visual analog scale (VAS) (painVAS), physician global VAS (physicianVAS), Health Assessment Questionnaire (HAQ), serum uric acid, erythrocyte sedimentation rate (ESR), and high-sensitivity C-reactive protein (HsCRP). At the flare visit, participants had a median of 1 clinically active joint (interquartile range [IQR] 1-2), and a median of 5 joints with a PD score≥2 (IQR 4-10, P<.001). At the inter-critical visit, participants reported an median of 0 clinically active joints (IQR 0-0), and a median of 4 joints with a PD score≥2 (IQR 3-7, P<.001). Physician VAS (5.69 vs 3.40, P<.001), painVAS (6 vs 0, P<.001), HAQ (0.75 vs 0.12, P=.032), and ESR (29 vs 13.5mm/h, P=.02) were higher at the acute visit, but HsCRP levels were similar (8.88 vs 5.15mg/L, P=.062). This pilot study established the presence of subclinical intra-articular inflammation in gout at both acute and inter-critical phases. Despite the apparent resolution of symptoms after an acute flare, a relatively high proportion of joints had subclinical inflammation in the inter-critical visit. The long-term implications of untreated subclinical joint inflammation are not clear.

Rejuvenation of periocular region in Koreans: A multimodal approach combining botulinum toxin, fillers, and micro-focused ultrasound with visualization for optimal results.

Periorbital aging involves all layers of the skin, fat, and the bony orbit. Therefore, a multimodal approach is necessary. Twenty Korean subjects who required periorbital rejuvenation were enrolled in this study. First, micro-focused ultrasound with visualization (MFU-V) was performed across the supra-brow area, lateral canthus, and the infraorbital area. Next, the filler was injected into the sunken upper eyelid, lateral eyebrow, and the infraorbital area. Lastly, botulinum toxin was administered into the forehead, glabella, crow's feet, and the lateral orbital rim below the brow on the same day. Photographs were taken at baseline; immediately after treatment; and at 1, 4, and 12 weeks of follow-up. The average eyebrow height was increased by 3.5 mm, and the maximum height was elevated by 3.9 mm at week 12. Physician visual analogue scale (VAS) scores regarding the whole periorbital area and eyebrow ptosis were continuously improved throughout the observation period. Physician VAS scores for infraorbital hollow and superior sulcus deformity improved immediately after the procedure and were maintained at week 12. Adverse events were mild and transient. We believe that a multimodal approach combining MFU-V, fillers, and botulinum toxin results in significant periocular rejuvenation.

Patient-physician discordance in assessment of adherence to inhaled controller medication: a cross-sectional analysis of two cohorts

ObjectiveWe aimed to compare patient’s and physician’s ratings of inhaled medication adherence and to identify predictors of patient-physician discordance.DesignBaseline data from two prospective multicentre observational studies.Setting29 allergy, pulmonology and paediatric...

Residual Disease Activity and Associated Factors in Psoriatic Arthritis.

Remission or low disease activity should be the treatment target of psoriatic arthritis (PsA). However, residual disease activity (RDA) in some domains could persist. The aim of this study was to assess RDA and its associated factors in a group of patients with PsA. Patients with PsA were enrolled if they satisfied ClASsification for Psoriatic ARthritis (CASPAR) criteria with > 6 months' followup and achieved a status of low disease activity (LDA), minimal disease activity (MDA), or remission [Disease Activity Index for PsA (DAPSA) remission or very low disease activity (VLDA)]. RDA was assessed by the percentage of patients who had, although in LDA or remission, tender and/or swollen joints > 1, Leeds Enthesitis Index > 1, Health Assessment Questionnaire > 0.5, Psoriasis Area Severity Index (PASI) > 1, patient's global assessment > 20, physician visual analog scale (VAS) > 20, and VAS pain > 15. Associated factors of RDA were also assessed. Of 113 enrolled patients, 78 (69%) were in MDA. Moreover, DAPSA remission was observed in 46 (40.7%) while VLDA only in 32 (28.3%) of patients with PsA. VLDA seems to be the most stringent criterion, with a minimal RDA only in the VAS physician in 1 patient (3.1%) and none in the different domains, while patients in MDA had RDA in tender joints (14.1%), VAS pain (29.4%) and PASI > 1 or body surface area (BSA) > 3% (17.9%). Of note, although patients in DAPSA remission show a very low rate of RDA in almost all domains, 12 (26%) of them show a PASI > 1 or BSA > 3%. Finally, LDA shows RDA in higher percentages, mainly in patient-reported outcomes, tender joints, and skin domain. RDA can be recognized in patients with PsA. VLDA seems to be the most stringent composite index to identify patients in the absence of RDA.

Updated version of Japanese Childhood Health Assessment Questionnaire (CHAQ)

Objectives: The Childhood Health Assessment Questionnaire (CHAQ) is one of the most widely used self- report questionnaires to measure functional status in Juvenile idiopathic arthritis (JIA). The Japanese version of the CHAQ (JCHAQ) has been revised to meet requirements of clinical international trials which need the same number of questions in each functional area of the CHAQ.Methods: The original JCHAQ consisted of 36 items, measuring eight functional areas. This was changed to 30 items of questionnaire so that each functional area has same number of questions as the original US English version. The revised version was professionally translated from English to Japanese, reviewed, and validated with Japanese JIA patients.Results: A total of 42 JIA patients were enrolled in the validation: seven systemic, 30 polyarticular/oligoarticular and five enthesis related. Most patients were well controlled and the median disability index (DI) scores was 0.0 [0–0.03]; however, significant correlation was seen with visual analog scale (VAS) of pain, VAS overall well-being, physician VAS, DAS (Disease Activity Score) 28-ESR, and JADAS (Juvenile Arthritis Disease Activity Score)-27. In comparison of two groups of disease activity, remission or inactive/low disease activity vs. moderate/high disease activity, both DAS28-ESR and JADAS-27 showed significant correlation with DI.Conclusion: The updated JCHAQ was a reliable and valid tool for the functional assessment of children with JIA. It is more suitable for international and transitional comparison.

The Impact of Cytokines on the Health-Related Quality of Life in Patients with Systemic Lupus Erythematosus.

Introduction: Systemic lupus erythematosus (SLE) reduces the health-related quality of life (HRQoL), even during periods of disease quiescence. We investigated whether subclinical inflammation as reflected by cytokine levels is linked with reduced HRQoL. Methods: A cross-sectional study of SLE patients (n = 52, mean age 47.3, 86.5% female) who completed a Short Form Health Survey-36 (SF-36) questionnaire. The clinical and demographic data, scores for the disease activity (SLEDAI-2K), organ damage (SDI), and laboratory data were collected simultaneously. The autoantibody and cytokine levels (IFN-γ, IL-1β, IL-4, IL-6, IL-10, IL-12, IL-17, BAFF, TNF-α, TGF-β1, MIP-1α, MIP-1β and MCP-1 (levels in pg/mL) were quantified by sandwich ELISA. The comparisons and associations were assessed non-parametrically, and a multiple regression determined the effect sizes (ES) of the variables on the SF-36 domain and summary scores. Results: The SF-36 summary and domain scores for SLE patients were significantly (20–40%) lower than in a comparable control group, with the exception of the Mental Health scores (p = 0.06). SLE patients had a normal body mass index (BMI) (median, 24.2 kg/m2), a high rate of smoking (69.2%), and usage of social security benefits (90.4%). TGF-β1 (ES 0.06), IL-12 (ES −0.11), IFN-γ (ES 0.07) and MCP-1 (ES 0.06) influenced the SF-36 domain scores; and MCP-1 (ES 0.04) influenced the Mental Health Summary Score (MCS). Obvious manifestations, including patient visual analogue scale (VAS) (ES −2.84 to −6.29), alopecia (ES −14.89), malar rash (ES −14.26), and analgesic requirement (ES −19.38), independently influenced the SF-36 items; however, the SF-36 scores were not reflected by the physician VAS or disease activity (SLEDAI-2K). Conclusions: Cytokines had a minimal impact on HRQoL in SLE patients, especially compared to visible skin manifestations, central nervous system (CNS) damage, and pain. Better tools are needed to capture HRQoL in measures of disease activity.

Nailfold capillary changes in adult new-onset dermatomyositis: a prospective cross-sectional study.

It is to prospectively analyze nailfold capillaroscopy (NC) findings in new-onset dermatomyositis (DM) and to correlate NC findings with serum angiogenic cytokines and DM clinical and laboratory features. Twenty-three patients with DM who experienced < 12months of symptoms were included in the study. To assess serum cytokine levels, 23 age-, sex-, and ethnicity-matched healthy volunteers were used. NC characteristics and DM activity parameters were analyzed. Significantly higher serum angiogenin (ANG) and vascular endothelial growth factor-1 (VEGF1) levels were observed in DM patients than in controls. Capillary density and avascular areas correlated positively and negatively, respectively, with serum levels of ANG. Moreover, the capillary density correlated inversely with the number of enlarged and giant capillaries and avascular areas. The number of enlarged capillaries correlated positively with patient and physician visual analogue scales (VAS), the presence of a facial rash, giant capillaries, and microhemorrhages. Giant capillaries had a positive correlation with physician and cutaneous VAS, enlarged capillaries, avascular areas, microhemorrhages and bushy capillaries, and a negative correlation with capillary density. Microhemorrhages correlated positively with the "V-neck" sign and physician VAS. VEGF1 showed no relationship with the NC parameters with DM-related clinical and laboratory features. Additionally, 15 out of 23 patients were assessed prospectively after 3.21years. All patients had a major clinical response with significant improvement in all NC parameters, except for enlarged and bushy capillaries. The NC may be a useful tool to assess disease activity in recent-onset DM, and it can also reinforce the role of ANG in the angiogenesis of this myopathy.

Innate Lymphoid Cells and T Cells Contribute to the Interleukin‐17A Signature Detected in the Synovial Fluid of Patients With Juvenile Idiopathic Arthritis

ObjectiveEvidence suggests that aberrant function of innate lymphoid cells (ILCs), whose functional and transcriptional profiles overlap with those of Th cell subsets, contributes to immune‐mediated pathologies. To date, analysis of juvenile idiopathic arthritis (JIA) immune pathology has concentrated on the contribution of CD4+ T cells; we have previously identified an expansion of Th17 cells within the synovial fluid (SF) of JIA patients. We undertook this study to extend this analysis to further investigate the role of ILCs and other interleukin‐17 (IL‐17)–producing T cell subsets in JIA.MethodsILCs and CD3+ T cell subsets were defined in peripheral blood mononuclear cells (PBMCs) from healthy adults, healthy children, and JIA patients and in SF mononuclear cells (SFMCs) from JIA patients using flow cytometry. Defined subsets in SFMCs were correlated with clinical measures including physician's global assessment of disease activity on a visual analog scale, number of joints with active disease, and erythrocyte sedimentation rate. Transcription factor and cytokine profiles of sorted ILCs were assessed by quantitative reverse transcriptase–polymerase chain reaction.ResultsGroup 1 ILCs (ILC1s), NKp44− group 3 ILCs (natural cytotoxicity receptor–negative [NCR−] ILC3s), and NKp44+ ILC3s (NCR+ ILC3s) were enriched in JIA SFMCs compared to PBMCs, which corresponded to an increase in transcripts for TBX21, IFNG, and IL17A. Of the ILC subsets, the frequency of NCR− ILC3s in JIA SFMCs displayed the strongest positive association with clinical measures, which was mirrored by an expansion in IL‐17A+CD4+, IL‐17A+CD8+, and IL‐17A+ γδ T cells.ConclusionWe demonstrate that the strength of the IL‐17A signature in JIA SFMCs is determined by multiple lymphoid cell types, including NCR− ILC3s and IL‐17A+CD4+, IL‐17A+CD8+, and IL‐17A+ γδ T cells. These observations may have important implications for the development of stratified therapeutics.

Responsiveness of clinical and ultrasound outcome measures in musculoskeletal systemic lupus erythematosus.

ObjectiveTo assess the responsiveness of clinical outcome measures in musculoskeletal SLE compared with US.MethodsA prospective pilot study was conducted in consecutive SLE patients with inflammatory musculoskeletal symptoms. Clinical assessments including SLEDAI, BILAG, 28 tender and swollen joint counts, physician and patient visual analogue scales (VAS), and US were performed at 0, 2 and 4 weeks following 120 mg i.m. methylprednisolone acetate. Responsiveness was analysed using changes and effect sizes using Cohen’s criteria.ResultsTwenty patients were recruited. Fifteen out of 20 had clinical swelling at baseline. All clinical and US parameters were significantly improved at week 4 (all P ⩽ 0.01). Musculoskeletal-BILAG score improved in 16/20. Musculoskeletal-SLEDAI improved in 7/20. SLE responder index 4 criteria were assessed in 19 patients with SLEDAI ⩾4 at baseline and were met in 9/19 at 4 weeks. Effect sizes at 4 weeks were large (>0.5) for US, physician VAS and BILAG, and medium (>0.3) for joint counts and SLEDAI. Large effect sizes for improvement in US grey-scale and power Doppler were observed in both SLE responder index 4 responders (r = −0.51 and −0.56, respectively) and non-responders (r = −0.62 and −0.59, respectively) at 4 weeks.ConclusionThis is the first study to measure the responsiveness of clinical outcome measures in musculoskeletal SLE against an objective inflammation measure. BILAG and physician VAS were the most responsive clinical instruments. US was highly responsive in musculoskeletal SLE, while SLEDAI and joint counts appeared suboptimal for detection of improvement. These results suggest that clinical trials based on the SLEDAI and SLE responder index 4 may underestimate the efficacy of therapy in SLE.

Preliminary Validation of the Digital Ulcer Clinical Assessment Score in Systemic Sclerosis.

To date, "healed/non-healed" and clinical judgment are the only available assessment tools for digital ulcers (DU) in patients with systemic sclerosis (SSc). The aim of our study is to examine a preliminary composite DU clinical assessment score (DUCAS) for SSc for face, content, and construct validity. Patients with SSc presenting at least 1 finger DU were enrolled and assessed with the Health Assessment Questionnaire-Disability Index, Cochin scale, visual analog scale (VAS) for DU-related pain, patient global DU status, and global assessment as patient-reported outcomes (PRO), and physician VAS for DU status (phyGDU) as an SSc-DU expert physician/nurse measure. The DUCAS included 7 DU-related variables selected by a committee of SSc DU experts and weighted on a clinical basis. Face validity was examined by consensus and partial construct validity was tested through convergent correlation with other measures of hand function, using Spearman's correlations. A range of patients with SSc was examined. A linear regression model with backward stepwise analysis was used to determine the relationship of individual variables with the primary clinical parameter, phyGDU. Forty-four patients with SSc (9 males, mean age 55 ± 15 yrs, mean disease duration 9.9 ± 5.8 yrs) were enrolled in the study. Overall DUCAS showed significant positive correlations with all abovementioned PRO (r > 0.4, p < 0.01). When all scores and scales were modeled, only DUCAS significantly predicted phyGDU (r = 0.59, R2 = 0.354, Akaike information criterion = 385.4). Preliminarily, we suggest that the DUCAS may be a new clinical score for SSc-related DU, having face and content validity and convergent/divergent correlations (construct validity). These early data suggest that this score deserves further evaluation.

Assessing Risk of Future Suicidality in Emergency Department Patients.

Emergency departments (ED) are the first line of evaluation for patients at risk and in crisis, with or without overt suicidality (ideation, attempts). Currently employed triage and assessments methods miss some of the individuals who subsequently become suicidal. The Convergent Functional Information for Suicidality (CFI-S) 22-item checklist of risk factors, which does not ask directly about suicidal ideation, has demonstrated good predictive ability for suicidality in previous studies in psychiatrict patients but has not been tested in the real-world setting of EDs. We administered CFI-S prospectively to a convenience sample of consecutive ED patients. Patients were also asked at triage about suicidal thoughts or intentions per standard ED suicide clinical screening (SCS), and the treating ED physician was asked to fill a physician gestalt visual analog scale (VAS) for likelihood of future suicidality spectrum events (SSE; ideation, preparatory acts, attempts, completed suicide). We performed structured chart review and telephone follow-up at 6 months post-index visit. The median time to complete the CFI-S was 3minutes (first to third quartile= 3-6 minutes). Of the 338 patients enrolled, 45 (13.3%) were positive on the initial SCS, and 32 (9.5%) experienced a SSE in the 6 months of follow-up. Overall, SCS had modest diagnostic accuracy sensitivity 14/32= 44%, (95% CI: 26-62%) and specificity 275/306= 90%, (86-93%). The physician VAS also had moderate overall diagnostic accuracy (AUC0.75, confidence interval [CI]= 0.66-0.85), and the CFI-S was best (AUC= 0.81, CI= 0.76-0.87). The top CFI-S differentiating items were psychiatric illness, perceived uselessness, and social isolation. Using CFI-S, or some of its items, in busy EDs may help improve the detection of patients at high risk for future suicidality.

Impact of subclinical synovitis in ankles and feet detected by ultrasonography in patients with rheumatoid arthritis.

To investigate the impact of subclinical synovitis detected by ultrasonography (US) on the ankles and feet of patients with rheumatoid arthritis. We retrospectively reviewed the data of patients (n=59) who underwent US. The functional ability and quality of life (QoL) of patients in the subclinical group were impaired. While the physician visual analog scale (VAS) scores significantly decreased in the subclinical group, patient and pain VAS scores significantly decreased only in patients without synovitis. US-detected subclinical foot and ankle synovitis considerably affected patient functional status and QoL; however, it was often unnoticed by physicians.

Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-Kinase δ Syndrome: The European Society for Immunodeficiencies-Activated Phosphoinositide 3-Kinase δ Syndrome Registry.

Activated phosphoinositide 3-kinase (PI3K) δ Syndrome (APDS), caused by autosomal dominant mutations in PIK3CD (APDS1) or PIK3R1 (APDS2), is a heterogeneous primary immunodeficiency. While initial cohort-descriptions summarized the spectrum of clinical and immunological manifestations, questions about long-term disease evolution and response to therapy remain. The prospective European Society for Immunodeficiencies (ESID)-APDS registry aims to characterize the disease course, identify outcome predictors, and evaluate treatment responses. So far, 77 patients have been recruited (51 APDS1, 26 APDS2). Analysis of disease evolution in the first 68 patients pinpoints the early occurrence of recurrent respiratory infections followed by chronic lymphoproliferation, gastrointestinal manifestations, and cytopenias. Although most manifestations occur by age 15, adult-onset and asymptomatic courses were documented. Bronchiectasis was observed in 24/40 APDS1 patients who received a CT-scan compared with 4/15 APDS2 patients. By age 20, half of the patients had received at least one immunosuppressant, but 2–3 lines of immunosuppressive therapy were not unusual before age 10. Response to rapamycin was rated by physician visual analog scale as good in 10, moderate in 9, and poor in 7. Lymphoproliferation showed the best response (8 complete, 11 partial, 6 no remission), while bowel inflammation (3 complete, 3 partial, 9 no remission) and cytopenia (3 complete, 2 partial, 9 no remission) responded less well. Hence, non-lymphoproliferative manifestations should be a key target for novel therapies. This report from the ESID-APDS registry provides comprehensive baseline documentation for a growing cohort that will be followed prospectively to establish prognostic factors and identify patients for treatment studies.

Using serum troponins to screen for cardiac involvement and assess disease activity in the idiopathic inflammatory myopathies.

ObjectivesLimitations in the methods available for identifying cardiac involvement and accurately quantifying disease activity in the idiopathic inflammatory myopathies (IIMs) may contribute to poor outcomes. We investigated the utility of different serum muscle damage markers [total creatine kinase (CK), cardiac troponin T (cTnT) and cardiac troponin I (cTnI)] to address these issues. MethodsWe assessed disease activity and cardiac involvement using the International Myositis Assessment and Clinical Studies Group core set measures in 123 participants with confirmed adult-onset IIM from the UK and Denmark. Total CK, cTnT and cTnI were measured. Associations were assessed using logistic regression and Spearman’s ranked correlation.ResultsCardiac involvement (n = 18) was associated with higher cTnI levels, independent of overall disease activity [adjusted odds ratio 1.03 (95% CI 1.01, 1.05); P = 0.002]. An abnormal cTnI had the highest specificity and positive predictive value for cardiac involvement (95% and 62%, respectively). In those with a normal CK but elevated cTnT or cTnI, an association with increased disease activity scores was observed. Serum cTnT correlated with the physician (ρ = 0.39) and patient-assessed (ρ = 0.28) global visual analogue scales and HAQ (ρ = 0.41) more strongly than CK or cTnI levels. cTnT was the only marker to correlate with manual muscle testing scores (ρ = −0.24).ConclusionSerum cTnI testing may have a role in screening for cardiac involvement in IIMs. Abnormal levels of serum cTnT and cTnI are associated with increased disease activity, including in those with a normal CK.

Repository corticotropin injection as adjunctive therapy in patients with rheumatoid arthritis who have failed previous therapies with at least three different modes of action.

ObjectiveMany types of treatment are available for patients with rheumatoid arthritis (RA), however, some patients fail to achieve remission. This report aims to determine the safety and efficacy of using repository corticotropin injection (RCI) as an adjunctive therapy in patients with RA refractory to at least three therapeutics with different mechanisms of action.MethodIn this open-label, interventional, single-group study, patients received 80 U RCI twice weekly via subcutaneous injection over 12 weeks. Changes in the Ritchie–Camp Articular Index and health assessment questionnaire scores were monitored for changes from baseline measures.ResultsEight patients were enrolled and consisted of seven females and one male with an average age of 64.6 years and disease duration of 20.9 years. Use of RCI resulted in significant improvement in swollen and tender joint counts. The disease activity score 28 and the physician and patient visual analog scale scores were significantly reduced at treatment week 12. The reduction in health assessment questionnaire scores did not reach statistical significance after RCI treatment. Once RCI therapy was discontinued, all improvements in disease activity score 28, physician and patient visual analog scale, and tender and swollen joint counts achieved during treatment were lost by the week 16 follow-up visit.ConclusionWhile larger clinical trials are necessary to further confirm the efficacy of RCI in patients with refractory RA, the response of patients with refractory RA in this study suggests that RCI can be an effective add-on therapy for patients who have exhausted several classes of treatments. Furthermore, this study suggests that RCI has an alternative mode of action, compared to other available antirheumatic drugs.