Introduction

Inflammatory Bowel Diseases (IBD) are multifactorial diseases, including Ulcerative Colitis (UC) and Crohn's Disease (CD).
Up to 50% of IBD patients show a primary or secondary non-response to standard biological therapy. Therefore, Randomized Clinical Trials (RCTs) represent a significant therapeutic opportunity for them.

Aims & Methods

This study aims to compare the clinical characteristics of "real-life" IBD patients of our tertiary IBD centre to "trial patients" and to identify novel therapeutic targets from real-life populations to be considered for RCTs. We prospectively enrolled consecutive patients who started biologic therapy from August 2019 to August 2020 at Fondazione Policlinico Universitario "A. Gemelli" IRCCS, Rome. We divided patients into three sub-groups: (1) "real-life" patients, patients treated according to clinical practice with standard biologic therapy that did not meet the inclusion and exclusion criteria for RCTs, (2) patients "real-life suitable for trial", potentially eligible in RCTs, but treated with standard of care, and (3) "trial" patients". Trial patients were treated with drugs from 14 phases 2b and 3 studies actively recruiting in our centre.

Results

We enrolled 134 patients: 72 with UC and 62 with CD. "Real-life" patients were 41 in UC and 38 in CD, "real-life suitable for trial" were 6 in UC and 14 in CD, and "trial" were 25 in UC and 10 in CD.
According to the existing inclusion and exclusion criteria, the study showed that only 43% of UC patients and 39% of CD were suitable for a potential enrollment in RCTs. However, only 16% of CD was finally enrolled in RCTs. At the enrollment, UC patients were excluded due to topical therapy use, cancer, recent Clostridium difficile infection or laboratory abnormalities. In contrast, CD patients were excluded mainly for a low CDAI but also due to complications of the disease that might require surgery, previous total or subtotal colectomy or major abdominal surgery in the previous six months. Both "trial" UC and CD patients showed more extraintestinal manifestations of disease, especially the articular ones (p<0,05). Moreover, "trial" patients presented, both in CD and UC, a higher significant concomitant use of systemic corticosteroids at the dose < or = 20mg/die (p < 0,05). Percentages of patients treated previously with other biological drugs were superior in "trial" patients compared to "real-life" in UC (88% vs 29%); instead, in CD, there was no significant difference (60% vs 45%).
We observed different baseline clinical disease activity scores in CD: the mean HBI of "real-life" patients was 4.8, and the mean HBI of "trial" patients was 9.1.
In addition, patients enrolled in RCTs waited longer before accessing the proposed biological therapy.

Conclusion

This study highlights some differences between clinical practice and research, particularly regarding the criteria for starting biological therapy in CD.
Firstly, “trial patients” presented more complex diseases, significantly impacting their clinical status.
Furthermore, RCTs use clinical scores (CDAI) as determinants for enrollment and decision-making, although endoscopy and radiological imaging are more widely used in clinical practice for decision making.
These differences could cover the actual effectiveness of a new drug compared to the theoretical efficacy derived from registration RCTs.
Therefore, we believe that new therapeutic targets, such as mucosal or histological healing in UC and transmural healing or new radiological scores in CD, should be considered and perhaps used in RCTs.

Introduction

Endoscopic retrograde cholangiopancreatography (ERCP) remains the procedure with the highest complication rate underscoring the importance of high-quality training. Despite existing guidelines, real-world data regarding training conditions remain limited. This Pan-European survey aims to explore the perceptions surrounding ERCP training.

Aims & Methods

A survey was distributed through the friends of the Young United European Gastroenterologists (UEG) Talent Group network. Inclusion criteria were physicians working in a UEG member state and regularly performing ERCP. Likert-scales as well as single- and multiple-choice questions were utilized.

Results

649 of 1035 respondents from 35 countries were eligible for analysis. 228 were identified as trainees, 225 as trainers, and 196 regularly performed ERCP without being in either category. The mean age was 43 years with 72.0%, 27.6%, and 0.3% identifying as male, female, and non-binary, respectively. 73% of all participants agreed that a structured training regimen is desirable. However, 64% of participants reported not having such a structured program at their institution (71% of trainees and 55% of trainers). Likewise, 80% of participants did not have a mandatory self-assessment before training initiation.
Majority of trainees (73%) and trainers (81%) favored that training should be concentrated within centers meeting certain quality metrics. For this, 64% of all participants indicated that a threshold of 200 annual ERCPs should be used. Using this threshold revealed that 32% of trainees pursued training in centers with <200 annual ERCPs and that a low annual caseload of <50 was more frequent at lower volume centers vs. higher volume centers (86% vs. 63%, respectively). Furthermore, 71% of trainees performed <50 ERCPs/year in stark contrast to 95% of trainers performing >50 ERCPs/year. While 27% of trainees in higher volume centers were female, all trainees in lower volume centers were of female gender.

Conclusion

The first Pan-European survey investigating ERCP training conditions revealed that structured training and concentration of training efforts within European centers meeting specific quality metrics are desirable but exposed the low availability of structured training programs and that around 30% of trainees are practicing at lower volume centers. These data could be interpreted as motivation to further standardize ERCP training conditions and ultimately improve patient care throughout Europe.

Introduction

Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease in which muscle weakness develops insidiously. In advanced stages of the disease dysphagia, weight loss and malnutrition occur in the majority of patients. Thus, the consideration of Percutaneous Endoscopic Gastrostomy (PEG) tube placement is common. However, the optimal timing and setting for the procedure as well desired spirometry results are either are not stated in most guidelines or debated. Previous studies have suggested greater procedural risk for patients whose forced vital capacity (FVC) was less than 50% than predicted

Aims & Methods

The aim of this study was to assess the safety of our tertiary center simplified approach to PEG tube placement in patients with ALS.
The electronical records of patients who underwent percutaneous endoscopic gastrostomy (PEG) placement between 1.1.2020 to 1.1.2023 were retrospectively reviewed. All adult patients diagnosed with ALS who underwent ambulatory PEG tube placement were included. Patients who underwent PEG tube placement during hospitalization due to acute illness were excluded. The common practice of our center regarding PEG placement in patients with ALS is as follows: initial evaluation at the Gastroenterology clinic of patients referred by the neurology clinic, followed by ambulatory PEG placement under sedation by an anesthesiologist. We collected baseline information from the neurology and gastroenterology clinic visits regarding comorbidities and baseline FVC and the functional rating score [ALS-FRS, 0-48 points, lower score indicating a more severe disease). The data concerning the procedure and possible complications were collected from endoscopy reports, emergency department visits and clinic visits after the procedure.

Results

Six hundred and sixty patients had underwent PEG tube placement in our center during study period. Of them, 67 patients had an established diagnosis of ALS (10.2%). One patient was excluded due to PEG placement in the setting of an acute illness during admission, thus 66 patients were included (mean age at PEG placement 63.5±12.7 years and 37.8% were females). Mean ALS-FRS score was 23.6±8.81 and mean FVC was 49.8±19.45% of predicted. More than half (54.7%) of the patients were using non-invasive ventilation support device at home prior to the procedure and 12.1% of patients had underwent endotracheal tube placement prior to PEG tube placement.
The averaged duration of PEG tube placement was 11.96±5.3 minutes. All PEG procedures were completed successfully. Complications were documented during two PEG placements (3%): one patient suffered from aspiration and was hospitalized and later diagnosed with aspiration pneumonia and the second patient suffered from gastric perforation and peritonitis which was diagnosed a few hours after the procedure. During the 30 days following PEG placement, 3 (4.5%) complications were documented: Two patients (3%) suffered from PEG site infections and were treated with antibiotics and one (1.5%) was diagnosed with aspiration pneumonia. No deaths occurred during the procedure or during the 30 days that followed.

Conclusion

The simple straight forward approach to PEG placement in our institution was successful and safe in the vast majority of cases. The complications rate in our cohort is comparable to other studies, even though the patients in our cohort had significantly reduced lung function and functional scores then accepted in the common practice.

References

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