scholarly journals P416 Results of the first paediatric randomised controlled trial of faecal microbiota transplant for ulcerative colitis

2020 ◽  
Vol 14 (Supplement_1) ◽  
pp. S379-S380
Author(s):  
N Pai ◽  
J Popov ◽  
L Hill ◽  
E Hartung ◽  
K Grzywacz ◽  
...  
Keyword(s):  
Ulcerative Colitis ◽  
Clinical Response ◽  
Healthy Donor ◽  
Activity Index ◽  
Case Reports ◽  
Case Series ◽  
Faecal Microbiota ◽  
Open Label ◽  
Randomised Controlled

Abstract Background The role of faecal microbiota transplant (FMT) for the treatment of ulcerative colitis (UC) has been reported across 4 randomised-controlled trials (RCT) in adults. Promising data have emerged from small, open-label paediatric case series and case reports but a proper blinded, placebo-controlled RCT has not been described in children. We report results from the first multicentre RCT of FMT in paediatric UC patients, conducted over 36 months in Ontario and Quebec, Canada. Methods We enrolled 25 children, ages 4–17 years old with active UC across two tertiary IBD clinics. Patients had active inflammation and remained on stable doses of medication at entry. Blinded participants received enemas containing healthy donor stool (active) or normal saline (placebo), 2×/week for 6 weeks. Faecal calprotectin (fCal), C-reactive protein (CRP), and paediatric ulcerative colitis activity index (PUCAI) scores were compared between groups during intervention, and at four follow-up time points over 30 weeks. Donor and recipient stools were measured for 16s rRNA and metagenomics analyses. Results In intention-to-treat (ITT) analysis, FMT (n = 13) at 6 weeks was more likely to improve clinical response (OR 9.3, 95% CI [0.7, 122.6]), CRP (OR 4.7, 95% CI [0.8, 28.4]), and fCal (OR 13.3, 95% CI [1.1, 166.4]) from baseline compared with placebo (n = 12). FMT at 30 weeks was also more likely than placebo to improve clinical response, CRP, and fCal (Table 1). In ITT analysis of the open-label arm (n = 7), FMT at 6 weeks and 30 weeks decreased CRP (−42.9%, −28.6%), fCal (−28.6%, −42.9%), and PUCAI score (−14.3%, −42.9%) from baseline. Conclusion Serial FMT enemas containing healthy donor microbiota led to greater improvements in serum and stool inflammatory markers, and rates of clinical response, in paediatric patients with active UC compared with placebo. These improvements largely persisted beyond 6 months after final FMT treatment. This study offers the strongest preliminary evidence, from a blinded, placebo-controlled multicentre RCT for the role of FMT in the management of paediatric UC.

A systematic review of the efficacy and safety of second generation antipsychotics in the treatment of mania

2006 ◽  
Vol 21 (1) ◽  
pp. 1-9 ◽  
Author(s):  
R.M. Jones ◽  
C. Thompson ◽  
I. Bitter
Keyword(s):  
Systematic Review ◽  
Second Generation ◽  
Case Reports ◽  
Case Series ◽  
Antipsychotic Agents ◽  
Efficacy And Safety ◽  
Open Label ◽  
Second Generation Antipsychotics ◽  
Second Generation Antipsychotic ◽  
Randomised Controlled

AbstractSecond generation antipsychotic agents are increasingly used in the management of acute mania. A systematic review of the efficacy and safety of these agents, as both monotherapy and in combination with mood stabilisers, was performed to establish the evidence for their use. Randomised controlled trials (RCTs) were critically appraised in more detail than studies that presented lower levels of evidence such as case reports, case series and open label follow up studies. We found 11 RCTs reporting on patients treated with second generation antipsychotics for acute bipolar mania, of which three included randomisation between the second generation antipsychotic and placebo, and eight between a mood stabiliser combined with either the second generation antipsychotic or placebo. Data from non-randomised trials is also presented.

scholarly journals P375 Feasibility of the first paediatric randomised controlled pilot trial of faecal microbiota transplant for ulcerative colitis

2020 ◽  
Vol 14 (Supplement_1) ◽  
pp. S353-S354
Author(s):  
N PAI ◽  
J Popov ◽  
E Hartung ◽  
L Hill ◽  
K Grzywacz ◽  
...  
Keyword(s):  
Ulcerative Colitis ◽  
Adverse Events ◽  
Randomised Trial ◽  
Faecal Microbiota ◽  
Open Label ◽  
Serious Adverse Events ◽  
Paediatric Patients ◽  
Stool Samples ◽  
Randomised Controlled

Abstract Background The first multicentre paediatric randomised-controlled trial (RCT) of faecal microbiota transplant (FMT) in ulcerative colitis (UC) was recently completed in Ontario and Quebec, Canada. The use of FMT in paediatric UC treatment has been limited to small, open-label case series. Unique clinical and patient factors necessitated a paediatric-specific pilot RCT. Our aim was to assess key feasibility measures of conducting an FMT trial in paediatric patients to inform design, and feasibility of larger, future multicentre studies. Methods Patients were randomised (1:1) to receive rectal enemas 2×/week for 6 weeks, followed by a 6-month follow-up period. Enemas contained normal saline (placebo), or RBX2660 (Rebiotix; Roseville, USA), a faecal microbiota preparation of stool from healthy adult donors (active). Patients were eligible for the open-label extension following completion or withdrawal from the placebo arm. Study feasibility was assessed through patient eligibility, recruitment, completion of sample collections, adverse events, and hospitalisations. Results Over 36 months across two tertiary IBD clinics, 48 paediatric patients were screened for participation. 75.0% (36) met eligibility criteria for enrolment and 69.4% (25) participated in the randomised trial. 53.8% (7) of patients randomised to the placebo arm entered the open-label arm. We stratified completion of sample collections by intervention (Weeks 0–6), and follow-up phases of the trial (Weeks 6–30). 75.3% of required bloodwork was obtained during both phases; patient-provided stool samples declined from 76.0% (intervention) to 45% (follow-up). 83.0% (10) reported adverse events in the active arm, and 38.4% (5) in placebo; these were all minor and self-limited (muscle pain, cough, fatigue, etc.). No patients experienced adverse events related to enemas. Five patients experienced serious adverse events: IBD flare (2 active, 1 placebo), and reactivation of C. difficile infection (2 active) (Table 1). Conclusion In this first paediatric RCT of FMT for UC, we established important feasibility data regarding the design of FMT trials in children. Patient acceptance of FMT was high, and well-tolerated with minor, self-limited adverse events, and few serious adverse events. Return-rates of stool samples were low during follow-up; strategies to improve uptake are needed, particularly given the value of microbiome data for analyses. Overall, our study demonstrates strong interest and tolerance among paediatric patients for FMT-based trials. Future studies will build upon our protocol to enhance clinical data on the effects of microbiota-based therapeutics in children.

EVALUATION OF A DIET AND ANTIBIOTICS TARGETING THE MICROBIOTA FOR TREATMENT OF MILD TO MODETARE ACTIVE PEDIARTIC ULCERATIVE COLITIS: AN OPEN LABEL PILOT STUDY

2021 ◽  
Vol 27 (Supplement_1) ◽  
pp. S38-S38
Author(s):  
Chen Sarbagili-Shabat ◽  
Lindsey Albenberg ◽  
Johan Van Limbergen ◽  
Dror Weiner ◽  
Michal Yaakov ◽  
...  
Keyword(s):  
Ulcerative Colitis ◽  
Pilot Study ◽  
Dietary Intervention ◽  
Activity Index ◽  
Fecal Calprotectin ◽  
Dietary Therapy ◽  
The Novel ◽  
Open Label ◽  
Intention To Treat ◽  
Stable Maintenance

Abstract Background Newer strategies that target the microbiome may offer an alternative therapeutic approach for Ulcerative Colitis (UC). We developed a novel diet that targets changes in the microbiome and barrier function that have been reported in UC. The goal of the current study was to evaluate the efficacy of two sequential induction of remission strategies that target the microbiota: the novel diet termed the ulcerative colitis diet (UCD) and an antibiotics cocktail combination in dietary non responders. Methods This was a prospective, single arm, open label, pilot study in patients aged 8–19, with a pediatric UC activity index (PUCAI) scores >10 and ≤45 on stable maintenance therapy (5ASA or thiopurines). PUCAI score was assessed at week 3 and 6. Patients failing to enter remission or intolerant to dietary therapy could receive an open label 14-day course of Amoxycillin, Metronidazole and Doxycycline (AMD), and had PUCAI scored at day 21. Response was defined a decline in PUCAI ≥ 10 points, remission as PUCAI< 10. The primary endpoint was intention to treat (ITT) remission at week 6 with diet as the sole intervention. Results Twenty-three children mean age of 15.1±2.9 years were enrolled. Two patients (1 responder, 1 remission) withdrew by 3 weeks, four required additional therapy by week 3, all were considered failures by ITT. Mean PUCAI decreased at week 3 and 6 from 34.5±9.8 to 21.7±14.9 and 17.6±17.2 respectively (P=0.005, P=0.001) at ITT analysis including all patients. Sixteen out of twenty-three patients (69.6%) responded by week 6. Ten of twenty-three (43.5%) achieved remission by week 6, and nine (39.1%) had clinical remission at week 6. The median fecal calprotectin (FC) level decreased in patients (n=5) who achieved remission from 630 (IQR, 332–1586) μg/g at week 0 to 230 (75–1298) μg/g at week 6. Eight patients received treatment with antibiotics after failing diet, 4/8 (50.0%) subsequently entered remission. Conclusion A dietary intervention called the UC Diet appears to be effective for induction of remission in children with mild to moderate UC. Sequential use of diet, followed by antibiotic therapy in dietary non responders, needs further evaluation as a microbiome directed steroid sparing therapy in patient’s refractory to 5ASA and thiopurines.

Evidence-based management of epistaxis in hereditary haemorrhagic telangiectasia

2015 ◽  
Vol 129 (5) ◽  
pp. 410-415 ◽  
Author(s):  
I Syed ◽  
V S Sunkaraneni
Keyword(s):  
Assessment Tool ◽  
Case Reports ◽  
Treatment Algorithm ◽  
Case Series ◽  
Team Approach ◽  
Hereditary Haemorrhagic Telangiectasia ◽  
Questionnaire Studies ◽  
Randomised Controlled ◽  
Specific Management

AbstractBackground:There are currently no guidelines in the UK for the specific management of hereditary haemorrhagic telangiectasia related epistaxis. The authors aimed to review the literature and provide an algorithm for the management of hereditary haemorrhagic telangiectasia related epistaxis.Method:The Medline and Embase databases were interrogated on 15 November 2013 using the search items ‘hereditary haemorrhagic telangiectasia’ (title), ‘epistaxis’ (title) and ‘treatment’ (title and abstract), and limiting the search to articles published in English.Results:A total of 46 publications were identified, comprising 1 systematic review, 2 randomised, controlled trials, 27 case series, 9 case reports, 4 questionnaire studies and 3in vitrostudies.Conclusion:There is a lack of high-level evidence for the use of many of the available treatments for the specific management of epistaxis in hereditary haemorrhagic telangiectasia. Current management should be based on a multidisciplinary team approach involving both a hereditary haemorrhagic telangiectasia physician and an ENT surgeon, especially when systemic therapy is being considered. The suggested treatment algorithm considers that the severity of epistaxis merits intervention at different levels of the treatment ladder. The patient should be assessed using a reproducible validated assessment tool, for example an epistaxis severity score, to guide treatment. More research is required, particularly in the investigation of topical agents targeting the development and fragility of telangiectasiae in hereditary haemorrhagic telangiectasia.

scholarly journals Efficacy of 5-Aminosalicylic Acid Enemas in the Treatment of Distal Ulcerative Colitis

1990 ◽  
Vol 4 (7) ◽  
pp. 468-471 ◽  
Author(s):  
MG Robinson ◽  
DL Decktor
Keyword(s):  
Ulcerative Colitis ◽  
Disease Activity ◽  
Activity Index ◽  
Disease Activity Index ◽  
First Episode ◽  
High Dose ◽  
Aminosalicylic Acid ◽  
Open Label ◽  
Distal Ulcerative Colitis ◽  
The Mean

The efficacy of 4 g 5-aminosalicylic acid (5-ASA, mesalamine) enemas was assessed in 666 patients with distal ulcerative colitis. Patients were enrolled in an open-label compassionate use program. One 4 g 5-ASA enema was administered each night for a period of four weeks and the disease activity index was assessed at baseline and on days 14 and 28. On days 14 and 28, 78.0% and 88.1% of patients, respectively, demonstrated an improvement in disease activity index. The mean decline in disease activity index on day 14 was 40.7% (P=0.0001) and on day 28 it was 55.4% (P=0.0001). Efficacy was similar whether the disease was confined to or extended beyond 30 cm from the anus. There was no difference in efficacy in patients suffering their first episode of disease compared to patients suffering subsequent attacks. In conclusion, high dose 5-ASA enemas are a highly effective treatment for distal ulcerative colitis.

Overview of the Relevant Literature on the Possible Role of Acupuncture in Treating Male Sexual Dysfunction

2014 ◽  
Vol 32 (5) ◽  
pp. 406-410 ◽  
Author(s):  
Ming-Yen Tsai ◽  
Chun-Ting Liu ◽  
Cheng-Chieh Chang ◽  
Shih-Yu Chen ◽  
Sheng-Teng Huang
Keyword(s):  
Sexual Dysfunction ◽  
Premature Ejaculation ◽  
Relevant Literature ◽  
Case Series ◽  
Sham Acupuncture ◽  
Risk Of Bias ◽  
Inclusion Criteria ◽  
Male Sexual Dysfunction ◽  
Randomised Controlled

Objective To systematically review scientific reports on the effectiveness of acupuncture to treat male sexual dysfunction. Methods The Medline database was searched for published clinical trials of acupuncture for erectile dysfunction (ED) and premature ejaculation (PE) with English abstracts. Risk of bias was assessed for randomised controlled trials (RCTs). Results Seven studies on two conditions of male sexual dysfunction met the inclusion criteria. Three out of four RCTs were patient-blinded, but all had a high risk of bias. Three suggested that acupuncture has a therapeutic effect as compared with sham acupuncture. Comparisons with paroxetine were inconsistent. Other uncontrolled studies and case series suggested satisfactory improvements of ED and PE after acupuncture. Conclusions Acupuncture appears to have promise for treating male sexual dysfunction, but in view of the small number of studies and their variable quality, doubts remain about its effectiveness. Further studies are justified.

The Neuropathology of 1p36 Deletion Syndrome: An Autopsy Case Series

2021 ◽  
Author(s):  
Kyle S Conway ◽  
Fozia Ghafoor ◽  
Amy C Gottschalk ◽  
Joseph Laakman ◽  
Renee L Eigsti ◽  
...  
Keyword(s):  
Neuronal Migration ◽  
Single Case ◽  
Case Reports ◽  
Case Series ◽  
Deletion Syndrome ◽  
Cerebellar Hypoplasia ◽  
Autopsy Case ◽  
Single Case Report ◽  
Renal Abnormalities

Abstract 1p36 deletion syndrome is the most common terminal deletion syndrome, manifesting clinically as abnormal facies and developmental delay with frequent cardiac, skeletal, urogenital, and renal abnormalities. Limited autopsy case reports describe the neuropathology of 1p36 deletion syndrome. The most extensive single case report described a spectrum of abnormalities, mostly related to abnormal neuronal migration. We report the largest published series of 1p36 autopsy cases, with an emphasis on neuropathologic findings. Our series consists of 3 patients: 2 infants (5-hours old and 23-days old) and 1 older child (11 years). Our patients showed abnormal cortical gyration together with a spectrum of neuronal migration abnormalities, including heterotopias and hippocampal abnormalities, as well as cerebellar hypoplasia. Our findings thus support the role of neuronal migration defects in the pathogenesis of cognitive defects in 1p36 deletion syndrome and broaden the reported neuropathologic spectrum of this common syndrome.

scholarly journals Potential of Radiolabeled PSMA PET/CT or PET/MRI Diagnostic Procedures in Gliomas/Glioblastomas

2020 ◽  
Vol 13 (2) ◽  
pp. 94-98 ◽  
Author(s):  
Francesco Bertagna ◽  
Domenico Albano ◽  
Elisabetta Cerudelli ◽  
Maria Gazzilli ◽  
Raffaele Giubbini ◽  
...  
Keyword(s):  
Case Reports ◽  
Case Series ◽  
Whole Body ◽  
Cochrane Library ◽  
Whole Body Imaging ◽  
Mri Imaging ◽  
Psma Pet ◽  
Pet Ct ◽  
Diagnostic Role

Background: Radiolabeled prostate-specific membrane antigen PSMA-based PET/CT or PET/MRI is a whole-body imaging technique currently performed for the detection of prostate cancer lesions. PSMA has been also demonstrated to be expressed by the neovasculature of many other solid tumors. Objective: The aim of this review is to evaluate the possible diagnostic role of radiolabeled PSMA PET/CT or PET/MRI in patients with gliomas and glioblastomas, by summarizing the available literature data. Methods: A comprehensive literature search of the PubMed/MEDLINE, Scopus, Embase and Cochrane library databases was conducted to find relevant published articles about the diagnostic performance of radiolabeled PSMA binding agents in PET/CT or PET/MRI imaging of patients with suspected gliomas or glioblastomas. Results: Seven case reports or case series and 3 studies enrolling more than 10 patients showed that gliomas and glioblastoma are PSMA-avid tumors. Conclusion: Radiolabeled PSMA imaging seems to be useful in analyzing glioma/glioblastoma. Further studies enrolling a wider population are needed to clarify the real clinical and diagnostic role of radiolabeled PSMA in this setting and its possible position in the diagnostic flow-chart.

scholarly journals CURRENT THERAPEUTIC OPTIONS FOR CORONAVIRUS DISEASE-2019 – A PHARMACOLOGICAL REVIEW

2020 ◽  
pp. 42-50
Author(s):  
SASMI MB ◽  
MARIA JOSE ◽  
PRAVEENLAL KUTTICHIRA
Keyword(s):  
Case Reports ◽  
Treatment Options ◽  
Case Series ◽  
Standard Of Care ◽  
Therapeutic Options ◽  
Cochrane Library ◽  
Global Public Health ◽  
Medical Subject Headings ◽  
Published Evidence

Objectives: Coronavirus pandemic is currently a global public health emergency. With expanding knowledge of the virus and the disease, new therapeutic targets are emerging widely. There is limited evidence about the use of different treatment options in coronavirus disease-2019 (COVID-19). This review aims to summarize the available evidence regarding therapeutic options in treating coronavirus infection. Methods: We searched PubMed, Google Scholar, and Cochrane library using pre-specified Medical Subject Headings terms about the role of therapeutic options in COVID-19 patients. Results: The majority of the published evidence is either case reports or small observational studies. Antimalarial like hydroxychloroquine reported equivocal results with five studies got positive results and five without any added benefit compared with standard of care. Lopinavir/ ritonavir monotherapy does not show any significant role except in combination with other antiviral drugs but encouraging results are emerging with remdesivir. Studies with favipiravir are inconclusive with some exhibit benefit and others not. Limited case series have shown that tocilizumab and convalescent plasma to be useful as adjuvant therapy in critically ill patients. Conclusion: There is currently no strong evidence for the efficacy of different therapeutic agents in the treatment of COVID-19. More data from ongoing and future trials will add more insight into the role of various drugs.

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