scholarly journals Colchicine Effectiveness and Safety in Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Adenitis

2021 ◽  
Vol 9 ◽  
Author(s):  
Tatjana Welzel ◽  
Maren Ellinghaus ◽  
Anna L. Wildermuth ◽  
Norbert Deschner ◽  
Susanne M. Benseler ◽  
...  
Keyword(s):  
Abdominal Pain ◽  
Liver Enzyme ◽  
Primary Outcome ◽  
Periodic Fever ◽  
Gastrointestinal Symptoms ◽  
Aphthous Stomatitis ◽  
Elevated Liver Enzymes ◽  
Secondary Outcomes ◽  
Flare Frequency

Introduction: Periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) is the most common fever syndrome in childhood. High disease activity (DA) dramatically impacts the health-related quality of life. Thus, effective and safe treatment is crucial. Colchicine might be effective, but data are still lacking. Study aimed to assess colchicine safety and effectiveness in PFAPA.Methods: This single center study was conducted between 03/2012 and 05/2021 in PFAPA patients without variants in genetic panel testing aged ≤ 18 years fulfilling Marshall criteria and classification criteria of Gattorno et al. Exclusion criteria were elevated liver enzymes, impaired kidney function, celiac disease, lactose intolerance, previous/ongoing biologics, known colchicine-intolerance. Demographics, clinical characteristics, treatment, DA, colchicine effectiveness and safety were recorded at baseline, first and last visit. Colchicine was started at 0.5–1.0 mg/day. DA was captured by physician (PGA) and patient/parent (PPGA) global assessment on a 10 cm visual analog scale, categorized as mild (<2), moderate (2–4), and high (≥5). Adverse event (AE) monitoring included gastrointestinal symptoms, liver enzyme/creatinine elevation, leukopenia, neutropenia. Primary outcome included response (R; composite of PPGA + PGA decrease ≥2) at last follow-up. Secondary outcomes were partial response (PR; PGA decrease = 1 + PPGA decrease ≥1), no response (NR; unchanged/worsened PGA/PPGA), colchicine safety, flare characteristics.Results: Twenty-seven PFAPA patients were included, 52% were female, median age was 5.8 years (1–10.75), median follow-up time was 13 months. At baseline, median PPGA was high; median PGA moderate. All patients had febrile flares. Median flare frequency was every 4–5 weeks; median duration 5–6 days. Nine patients were pre-treated with corticosteroids, increasing flare frequency in 8/9. Primary Outcome: 17 patients (63%) were responders. Secondary outcomes: PR was achieved in 15%; NR in 22% at last follow-up. DA decreased significantly (p <0.0001). At last follow-up, 52% reported no flares, median flare duration decreased to 1–2 days. At first follow-up, 22% reported mild abdominal pain/diarrhea. Moderate abdominal pain/diarrhea occurred with ≥1 mg/day. Mild asymptomatic liver enzyme elevation or leucopenia were rare; no severe AE or colchicine discontinuation were observed.Conclusion: Colchicine seems to be safe, well-tolerated, and effective in PFAPA patients. It can be considered in children with moderate/high DA even those without corticosteroid-benefit.

scholarly journals The Probiotic VSL#3® Does Not Seem to Be Efficacious for the Treatment of Gastrointestinal Symptomatology of Patients with Fibromyalgia: A Randomized, Double-Blind, Placebo-Controlled Clinical Trial

2021 ◽  
Vol 14 (10) ◽  
pp. 1063
Author(s):  
Elena P. Calandre ◽  
Javier Hidalgo-Tallon ◽  
Rocio Molina-Barea ◽  
Fernando Rico-Villademoros ◽  
Cristina Molina-Hidalgo ◽  
...  
Keyword(s):  
Abdominal Pain ◽  
Primary Outcome ◽  
Controlled Trial ◽  
Gastrointestinal Symptoms ◽  
Specific Treatment ◽  
Response To Treatment ◽  
Secondary Outcome ◽  
Outcome Variable ◽  
Controlled Clinical Trial

Background: Gastrointestinal symptomatology is frequent among patients with fibromyalgia, which increases disease burden and lacks specific treatment, either pharmacological or non-pharmacological. We aimed to evaluate the efficacy and tolerability of a multi-strain probiotic, VSL#3®, for the treatment of fibromyalgia-associated gastrointestinal manifestations. Methods: This randomized, placebo-controlled trial included 12 weeks of probiotic or placebo treatment followed by 12 weeks of follow up. The primary outcome variable was the mean change from the baseline to the endpoint in the composite severity score of the three main gastrointestinal symptoms reported by patients with fibromyalgia (abdominal pain, abdominal bloating and meteorism). Secondary outcome variables were the severity of additional gastrointestinal symptoms, fibromyalgia severity, depression, sleep disturbance, health-related quality of life and patients’ overall impression of improvement. Results: No differences were found between VSL#3® (n = 54) and the placebo (n = 56) in the primary outcome (estimated treatment difference: 1.1; 95% confidence interval [CI]: −2.1, 4.2; p = 0.501), or in any of the secondary outcomes. However, responders to VSL#3 were more likely to maintain any improvement during the follow-up period compared to responders in the placebo arm. Overall, VSL#3 tolerability was good. Conclusion: Our data could not demonstrate any beneficial effects of VSL#3® either on the composite score of severity of abdominal pain, bloating and meteorism or in any of the secondary outcome variables. More research is needed to elucidate specific factors that may predict a favourable response to treatment in patients with fibromyalgia.

Efficacy of intravitreal conbercept injection in the treatment of retinopathy of prematurity

2018 ◽  
Vol 103 (4) ◽  
pp. 494-498 ◽  
Author(s):  
Yichen Bai ◽  
Huanjie Nie ◽  
Shiyu Wei ◽  
Xiaohe Lu ◽  
Xiaoyun Ke ◽  
...  
Keyword(s):  
Retinopathy Of Prematurity ◽  
Laser Photocoagulation ◽  
Primary Outcome ◽  
Safety And Efficacy ◽  
Postmenstrual Age ◽  
Zone Ii ◽  
The Mean ◽  
Secondary Outcomes ◽  
Number Of Injections

BackgroundTo evaluate the safety and efficacy of intravitreal conbercept (IVC) injection in the treatment of retinopathy of prematurity (ROP).MethodsPatients with ROP who underwent IVC injection in Zhujiang Hospital from June 2015 to July 2016 were studied retrospectively. The primary outcome was defined as the regression of plus disease. The secondary outcomes were defined as the presence of recurrence, number of injections and the final regression of disease.ResultsA total of 48 eyes of 24 patients with ROP were included. Among them, 9 eyes of 5 patients had zone I ROP, 35 eyes of 18 patients had zone II ROP and 4 eyes of 2 patients had aggressive posterior ROP. The mean gestational age was 28.5±1.6 weeks, the mean birth weight was 1209.6±228.6 g, the mean postmenstrual age of first injection was 34.2±1.9 weeks and the mean follow-up period was 31.0±4.7 weeks. Forty of 48 eyes (83.3%) received IVC only once, and the regression of plus disease occurred at an average of 3.5±1.5 weeks after the first injection of conbercept. For eight recurrent eyes (16.7%), four eyes received a second IVC and the remaining four eyes received laser photocoagulation, and the regression of plus disease occurred in 3 weeks. No lens opacity, vitreous haemorrhage, entophthalmia or retinal detachment was observed during follow-up.ConclusionIVC injection is an effective treatment for ROP.

scholarly journals A Virtual Reality-Based Self-Help Intervention for Dealing with the Psychological Distress Associated with the COVID-19 Lockdown: An Effectiveness Study with a Two-Week Follow-Up

2021 ◽  
Vol 18 (15) ◽  
pp. 8188
Author(s):  
Giuseppe Riva ◽  
Luca Bernardelli ◽  
Gianluca Castelnuovo ◽  
Daniele Di Lernia ◽  
Cosimo Tuena ◽  
...  
Keyword(s):  
Virtual Reality ◽  
Psychological Distress ◽  
Outcome Measures ◽  
Perceived Stress ◽  
Social Connectedness ◽  
Primary Outcome ◽  
Post Intervention ◽  
Stress Levels ◽  
Secondary Outcomes

The aim of this study is to investigate the effectiveness of a novel self-administered at-home daily virtual reality (VR)-based intervention (COVID Feel Good) for reducing the psychological burden experienced during the COVID-19 lockdown in Italy. A total of 40 individuals who had experienced at least two months of strict social distancing measures followed COVID Feel Good between June and July 2020 for one week. Primary outcome measures were depression, anxiety, and stress symptoms, perceived stress levels, and hopelessness. Secondary outcomes were the experienced social connectedness and the level of fear experienced during the COVID-19 pandemic. Linear mixed-effects models were fitted to evaluate the effectiveness of the intervention. Additionally, we also performed a clinical change analysis on primary outcome measures. As concerning primary outcome measures, participants exhibited improvements from baseline to post-intervention for depression levels, stress levels, general distress, and perceived stress (all p < 0.05) but not for the perceived hopelessness (p = 0.110). Results for the secondary outcomes indicated an increase in social connectedness from T0 to T1 (p = 0.033) but not a significant reduction in the perceived fear of coronavirus (p = 0.412). Among these study variables, these significant improvements were maintained from post-intervention to the 2-week follow-up (p > 0.05). Results indicated that the intervention was associated with good clinical outcomes, low-to-no risks for the treatment, and no adverse effects or risks. Globally, evidence suggests a beneficial effect of the proposed protocol and its current availability in 12 different languages makes COVID Feel Good a free choice for helping individuals worldwide to cope with the psychological distress associated with the COVID-19 crisis, although large scale trials are needed to evaluate its efficacy.

scholarly journals Preventing sickness absence among employees with common mental disorders or stress-related symptoms at work: a cluster randomised controlled trial of a problem-solving-based intervention conducted by the Occupational Health Services

2020 ◽  
Vol 77 (7) ◽  
pp. 454-461 ◽  
Author(s):  
Marijke Keus van de Poll ◽  
Lotta Nybergh ◽  
Caroline Lornudd ◽  
Jan Hagberg ◽  
Lennart Bodin ◽  
...  
Keyword(s):  
Mental Health ◽  
Problem Solving ◽  
Mental Disorders ◽  
Occupational Health ◽  
Sickness Absence ◽  
Primary Outcome ◽  
Common Mental Disorders ◽  
Significant Group ◽  
Secondary Outcomes

ObjectivesCommon mental disorders (CMDs) are among the main causes of sickness absence and can lead to suffering and high costs for individuals, employers and the society. The occupational health service (OHS) can offer work-directed interventions to support employers and employees. The aim of this study was to evaluate the effect on sickness absence and health of a work-directed intervention given by the OHS to employees with CMDs or stress-related symptoms.MethodsRandomisation was conducted at the OHS consultant level and each consultant was allocated into either giving a brief problem-solving intervention (PSI) or care as usual (CAU). The study group consisted of 100 employees with stress symptoms or CMDs. PSI was highly structured and used a participatory approach, involving both the employee and the employee’s manager. CAU was also work-directed but not based on the same theoretical concepts as PSI. Outcomes were assessed at baseline, at 6 and at 12 months. Primary outcome was registered sickness absence during the 1-year follow-up period. Among the secondary outcomes were self-registered sickness absence, return to work (RTW) and mental health.ResultsA statistical interaction for group × time was found on the primary outcome (p=0.033) and PSI had almost 15 days less sickness absence during follow-up compared with CAU. Concerning the secondary outcomes, PSI showed an earlier partial RTW and the mental health improved in both groups without significant group differences.ConclusionPSI was effective in reducing sickness absence which was the primary outcome in this study.

scholarly journals Cardiac and Kidney Benefits of Empagliflozin in Heart Failure Across the Spectrum of Kidney Function: Insights from the EMPEROR-Reduced Trial

Circulation ◽  
2020 ◽  
Author(s):  
Faiez Zannad ◽  
João Pedro Ferreira ◽  
Stuart J. Pocock ◽  
Cordula Zeller ◽  
Stefan D. Anker ◽  
...  
Keyword(s):  
Kidney Function ◽  
Primary Outcome ◽  
Cardiovascular Death ◽  
Unique Identifier ◽  
Clinical Trial Registration ◽  
Reduced Ejection Fraction ◽  
Secondary Outcomes ◽  
Kidney Impairment ◽  
Kidney Function Decline

Background: In EMPEROR-Reduced, empagliflozin reduced cardiovascular death or HF hospitalization, total HF hospitalizations, and slowed the progressive decline in kidney function in patients with HF and a reduced ejection fraction (HFrEF), with and without diabetes. We aim to study the effect of empagliflozin on cardiovascular and kidney outcomes across the spectrum of kidney function. Methods: In this pre-specified analysis, patients were categorized by the presence or absence of CKD at baseline (eGFR<60ml/min/1.73m 2 or UACR>300mg/g). The primary and key secondary outcomes were (1) a composite of cardiovascular death or HF hospitalization (primary outcome); (2) total HF hospitalizations, and (3) eGFR slope. The direct impact on kidney events was investigated by a prespecified composite kidney outcome (defined as a sustained profound decline in eGFR, chronic dialysis or transplant). The median follow-up was 16 months. Results: 3730 patients were randomized to empagliflozin or placebo, of whom 1978 (53%) had CKD. Empagliflozin reduced the primary outcome and total HF hospitalizations in patients with and without CKD: primary outcome HR=0.78 (95%CI=0.65-0.93) and HR=0.72 (95%CI=0.58-0.90), respectively; interaction P=0.63. Empagliflozin slowed the slope of eGFR decline by 1.11 (0.23-1.98) ml/min/1.73m 2 /year in patients with CKD and by 2.41 (1.49-3.32) ml/min/1.73m2/year in patients without CKD. The risk of the composite kidney outcome was reduced similarly in patients with and without CKD: HR=0.53 (95%CI=0.31-0.91) and HR=0.46 (95%CI=0.22-0.99), respectively. The effect of empagliflozin on the primary composite outcome and the key secondary outcomes was consistent across a broad range of baseline kidney function, measured by clinically relevant eGFR subgroups or by albuminuria, including patients with eGFR as low as 20 ml/min/1.73m 2 . Empagliflozin was well tolerated in CKD patients. Conclusions: In EMPEROR-reduced, empagliflozin had a beneficial effect on the key efficacy outcomes and slowed the rate of kidney function decline in patients with and without CKD and regardless of the severity of kidney impairment at baseline. Clinical Trial Registration: URL: https://clinicaltrials.gov Unique Identifier: NCT03057977

Long-Term Follow-Up of Children with Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Cervical Adenitis Syndrome

2011 ◽  
Vol 159 (6) ◽  
pp. 958-964 ◽  
Author(s):  
Victoria M. Wurster ◽  
James G. Carlucci ◽  
Henry M. Feder ◽  
Kathryn M. Edwards
Keyword(s):  
Periodic Fever ◽  
Aphthous Stomatitis ◽  
Cervical Adenitis ◽  
Long Term Follow Up

Ruthenium-106 brachytherapy for iris and iridociliary melanomas

2017 ◽  
Vol 102 (8) ◽  
pp. 1154-1159 ◽  
Author(s):  
Marina Marinkovic ◽  
Nanda Horeweg ◽  
Mirjam S Laman ◽  
Jaco C Bleeker ◽  
Martijn Ketelaars ◽  
...  
Keyword(s):  
Visual Acuity ◽  
Local Control ◽  
Primary Outcome ◽  
Low Vision ◽  
Local Failure ◽  
Dry Eyes ◽  
Free Survival ◽  
Secondary Outcomes ◽  
Preservation Rate

Background and purposeTo evaluate ruthenium-106 (Ru106) brachytherapy as eye-conserving treatment of iris melanomas (IMs) and iridociliary melanomas (ICMs).Materials and methodsEighty-eight patients received Ru106 brachytherapy between 2006 and 2016. Primary outcome was local control, and secondary outcomes were metastasis, survival, eye preservation, complications and visual acuity (VA).ResultsOverall median follow-up was 36 months. Of 88 patients, 58 (65.9%) had IM and 30 (34.1%) had ICM. ICM were on average larger and more advanced than IM. Local failure-free survival at 3years was 98.9% (SE 1.2%). Metastasis-free survival was 98.2% (SE 1.8%) at 3years; no deaths due to melanoma occurred during follow-up. Eye preservation rate was 97.7%. Treatment-related toxicities were mostly mild and observed in 80.7% of the patients. Common toxicities were worsening of pre-existing or new cataract (51.1%), dry eyes (29.5%) and glaucoma (20.5%). VA was not affected by Ru106 brachytherapy, with only 2.3% having VA <0.33 (low vision) at follow-up.ConclusionsRu106 for IM and ICM yielded excellent local control (98.9%) and eye preservation (97.7%). Toxicities were common, but mostly mild and transient. Moreover, Ru106 did not affect visual acuity.

scholarly journals A Case of Multisystem Inflammatory Syndrome in Children Presenting as Acute Appendicitis and Pancreatitis

PRILOZI ◽  
2021 ◽  
Vol 42 (2) ◽  
pp. 95-101
Author(s):  
Lidija Kareva ◽  
Katarina Stavrik ◽  
Kristina Mironska ◽  
Arjeta Hasani ◽  
Sonja Bojadzieva ◽  
...  
Keyword(s):  
Abdominal Pain ◽  
Pediatric Patients ◽  
Close Contact ◽  
Multiorgan Failure ◽  
Gastrointestinal Symptoms ◽  
Gastrointestinal Diseases ◽  
Serological Testing ◽  
Inflammatory Syndrome ◽  
Pcr Test

Abstract Multisystem Inflammatory Syndrome in Children (MIS-C) is characterized by an inflammation with fever, elevated inflammatory markers, conjunctivitis, rash, impaired coagulation, gastrointestinal symptoms and cardiac abnormalities that may progress to multiorgan failure. The presence of a positive COVID-19 antigen via a PCR test, serological testing for antibodies or close contact with a person diagnosed with COVID-19 helps differentiate MIS-C from other diseases. Gastrointestinal symptoms are recognized to be associated with COVID-19 infection or MIS-C in children, presenting as abdominal pain, gastrointestinal infection with watery stools, appendicitis, ileitis, pancreatitis and hepatitis, confusing the diagnosis with other gastrointestinal diseases. In this case report, we describe an 11 year old boy with MIS-C, who presents acute phlegmona of the appendix for which he undergoes appendectomy, accompanied with acute pancreatitis. These manifestations of MIS-C in our patient resolved without additional complications after a 2 month follow up. We call attention to MIS-C presenting in pediatric patients with fever and abdominal pain which might be caused by appendicitis and pancreatitis, and we recommend abdominal imaging and additional laboratory investigation to promote earlier diagnosis.

scholarly journals 490 Right ventricular free wall longitudinal strain negatively impacts prognosis of patients with functional mitral regurgitation undergoing transcatheter edge-to-edge mitral valve repair

2021 ◽  
Vol 23 (Supplement_G) ◽  
Author(s):  
Giuseppe Iuliano ◽  
Angelo Silverio ◽  
Cesare Baldi ◽  
Michele Bellino ◽  
Luca Esposito ◽  
...  
Keyword(s):  
Mitral Valve ◽  
Cardiac Death ◽  
Longitudinal Strain ◽  
Primary Outcome ◽  
P Value ◽  
Free Wall ◽  
Composite Outcome ◽  
Valve Repair ◽  
Secondary Outcomes

Abstract Aims Although right ventricular (RV) systolic dysfunction seems to be associated with adverse outcome after transcatheter edge-to-edge mitral valve repair (TEER) with the MitraClip system, the prognostic value of RV free wall longitudinal strain (RVFWLS) in this setting has not been yet investigated. The aim of this study is to evaluate RVFWLS as predictor of outcome in patients with severe or moderate-to-severe functional MR undergoing TEER and its prognostic role compared with tricuspid annular plane systolic excursion (TAPSE). Methods and results 102 patients [73 (IQR: 66.8–77.0) years, 73 males (71.6%)] were retrospectively selected from March 2012 to February 2021. Echocardiograms were performed by using General Electric machines. RVFWLS was assessed through RV modified apical 4-chamber view, setting the region of interest to minimum size. Values of RVFWLS &gt; −20% were recognized as abnormal. We considered a composite endpoint including rehospitalization for heart failure (HF) and overall death as primary outcome. Secondary outcomes were overall death, cardiac death and rehospitalization for HF. All patients were assessed at the longest available follow-up [median 22.1 (IQR: 9.7–49.3) months]. Baseline clinical and echocardiographic characteristics are listed in Table 1. Primary outcome was found in 60 (58.8%) patients, while secondary outcomes, i.e. overall death, cardiac death and rehospitalization for HF, were found respectively in 50 (50.0%), 31 (30.4%), and 36 (35.3%) patients. Mean TAPSE was 16.7 ± 4.0 mm and mean RVFWLS was −16.9 ± 6.0%. At univariable analysis both TAPSE (HR: 0.907, CI: 0.848–0.970, P-value: 0.004) and RVFWLS (HR: 0.937, CI: 0.897–0.979, P-value: 0.004) were significantly associated with the primary outcome. Kaplan–Meier survival curves showed that patients with TAPSE &lt;17 mm had a lower survival free from the composite outcome compared with those with TAPSE ≥17 mm (Log-Rank = 0.030); patients with RVFWLS value &gt; −20% also showed a lower survival free from the composite outcome compared with patients with RVFWLS ≤ −20% (Log Rank 0.004). Among patients with preserved RV systolic longitudinal function as indicated by TAPSE ≥ 17 mm, subjects with RVFWLS &gt; −20% had a significantly higher incidence of the composite outcome compare with those with RVFWLS ≤ −20% (Log-Rank = 0.008). Conversely, no difference was found among patients with TAPSE &lt;17 mm. Conclusions RV dysfunction assessed either by TAPSE and RVFWLS is associated with poorer outcome in patients with severe or moderate-to-severe functional MR undergoing TEER. Compared with TAPSE, RVFWLS seems to be superior in identifying patients at higher risk of adverse events during follow-up. Our data encourage the use of this speckle tracking-derived echocardiographic parameter in routinely evaluation of patients with functional MR candidate for TEER.

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