Terms Of Efficacy Research Articles

Short term efficacy of subtalar arthroscopy combined with medial calcaneal-talar joint distraction in minimally invasive treatment of diabetic patients with calcaneal fractures: a retrospective study

BackgroundThe surgical treatment and management of postoperative soft tissue complications in diabetic patients with displaced calcaneal fractures are still controversial. We aimed to evaluate the short-term efficacy of percutaneous minimally invasive screw fixation in treatment of diabetic patients with Sanders II and III calcaneal fractures under subtalar arthroscopy assisted by preoperative musculoskeletal ultrasonic locating lateral calcaneal branch (LCB) of the sural nerve and calcaneal-talar joint distraction device.MethodsThe clinical data of 52 diabetic patients diagnosed with Sanders II or III calcaneal fractures from March 2016 to August 2020 were followed up and analyzed. There were 23 patients of type II and 29 patients of type III, 34 males and 18 females, with a mean age of 61.7 ± 14.5 years (range: 45–72 years). Preoperative musculoskeletal ultrasonography was routinely examined to locate LCB of the sural nerve. During surgery, we performed arthroscopic percutaneous prying reduction screw fixation assisted by medial calcaneal-talar joint distraction. Incision healing, local skin paraesthesia and other conditions were observed regularly at 3 days, 6, 12 months, and the last follow-up after surgery. Also, we measured the length, width, height, Böhler angle, and Gissane angle of the calcaneus on lateral and axial x-rays. Visual analogue pain scale (VAS), American Orthopedic Foot and Ankle Society (AOFAS) score and Maryland score were used to evaluate the efficacy.Results52 patients were followed up for 23.7 ± 3.2 months (range: 20–28 months) without incision-related complications. Calcaneal radiographic parameters (length, width, height, Böhler/Gissane angle) were improved after surgery, and the differences were all statistically significant (P<0.05). There was no difference between calcaneal radiographic parameters at 6,12 months and the last follow-up compared with 3 days after surgery without significant loss in overall morphology (P>0.05). Postoperative VAS, AOFAS scores, and Maryland scores were significantly improved compared with those before surgery (P<0.05).ConclusionsPreoperative ultrasonic locating LCB of the sural nerve and arthroscopic percutaneous minimally invasive screw fixation of Sanders II and III calcaneal fractures with the assistance of calcaneal-talar joint distraction have good short-term efficacy and clinical feasibility in diabetic patients.

Short term efficacy of biological treatment for moderate-to-severe plaque psoriasis: a systematic review and network meta-analysis.

Psoriasis is a chronic inflammatory disease that is debilitating, particularly in its more severe forms. Multiple systemic therapies are used in moderate-to-severe psoriasis, but the development of biological interventions has revolutionized its management and improved its outcomes. To compare the effectiveness and safety of the different biological interventions approved for use in moderate-to-severe plaque psoriasis. Multiple databases were searched for relevant articles and a prospectively planned network meta-analysis was conducted on randomized controlled trials that assessed biological treatments in moderate-to-severe psoriasis. The search yielded 84 trials that encompassed 39,798 patients. Infliximab 5mg/kg had the highest probability of achieving 75% reduction on PASI scale in comparison to placebo (RR = 18.76, 95% CI [12.31; 28.57], high certainty), while Ixekizumab 80mg and Brodalumab 210mg had the highest probability at achieving PASI90 and PASI100 (37.81, [28.57; 50.03] and 81.04, [26.16; 251.01], respectively, with moderate certainty) On the other hand, Risankizumab 150mg and Ustekinumab 90mg were the only regimens with significantly less withdrawal rates due to adverse events (0.41, [0.18-0.96], and 0.57, [0.35-0.91], respectively with High certainty) compared to placebo. Anti-IL17 and Infliximab were among the most effective in ameliorating the symptoms of psoriasis, however, anti-IL17 were better at achieving full or almost full improvement on the PASI scale. Real life decision-making is not so clear-cut and should remain patient centered, taking into consideration factors such as safety, comorbidities, biologic naivety, dosing preferences and insurance considerations.

S2095 Assessing Longer Term Efficacy of Duodenal Mucosal Resurfacing (DMR) on Glycemic Control and Weight Loss: An Updated Systematic Review and Meta-Analysis

S2095 Assessing Longer Term Efficacy of Duodenal Mucosal Resurfacing (DMR) on Glycemic Control and Weight Loss: An Updated Systematic Review and Meta-Analysis

Long-term efficacy of oblique lateral interbody fusion combined with vertebral screw fixation for the treatment of lumbar specific infection

To explore medium and long term efficacy of oblique lateral interbody fusion (OLIF) in treating lumbar specific infection. From October 2017 to January 2021, 24 patients with lumbar specific infection were treated by OLIF combined with vertebral screw internal fixation, including 15 males and 9 females, aged from 27 to 61 years old with an average of (43.0±15.0) years old;the courses of disease ranged from 6 to 24 months with an average of (14.0±7.0) months;7 patients with L2-L3, 12 patients with L3-L4 and 5 patients with L4-L5;19 patients with tuberculosis infection and 5 patients with brucella infection. The amount of intraoperative blood loss, operative time and complications were recorded, and erythrocyte sedimentation rate(ESR), C-reactive protein (CRP), visual analogue scale (VAS), Japanese Orthopaedic Association(JOA) score and American Spinal Injury Association (ASIA) rating were compared before and one month after opertaion. All patients were followed up from 9 to 24 months with an average of (13.0±6.0) months. Operative time was (132.5±21.4) min, and intraoperative blood loss was (227.3±43.1) ml. ESR and CRP were decreased from (82.34±18.62) mmol·h-1 and (53.08±21.84) mg·L-1 before operation to (33.52±17.31) mmol·h-1 and (15.48±8.36) mg·L-1 at one month after operation, respectively (P<0.05). VAS was decreased from (7.52±1.36) before opertaion to (1.74±0.87) at one month after operation (P<0.05). JOA was increased from (17.86±3.95) before operation to (24.72±3.19) at one month after operation (P<0.05). Four patients had neurological symptoms before operation, and were classified to grade D according to ASIA classification, who were recovered to grade E at 1 month after operation. One patient was suffered from psoas major muscle injury after operation, and returned to normal at 3 weeks. One patient was suffered from abdominal distension and difficulty in defecation, and relieved after gastrointestinal decompression and enema. No complications such as abdominal organ injury and poor wound healing occurred in all patients. OLIF combined with vertebral screw internal fixation is a new minimally invasive surgical method for the treatment of lumbar specific infection, especially the lesion located on the middle lumbar vertebra. It has advantages of less trauma, short operation time, less blood loss, convenient operation, complete removal of the lesion, safety and effectiveness, and has good medium-and long-term efficacy for lumbar specific infection.

Long-term Efficacy and Safety of Sodium Oxybate in Treating Alcohol Use Disorder: A Systematic Review and Meta-Analysis.

Worldwide, three million deaths each year are reported due to the harmful use of alcohol. To date, only a few drugs have been approved for the treatment of Alcohol Use Disorder (AUD). This systematic review and meta-analysis aim to assess the long-term efficacy and safety of sodium oxybate (SMO) treatment in patients with AUD. We followed the PRISMA statement guidelines and searched PubMed and ISI Web of Science to retrieve the studies of interest. In total, 13 studies on long-term (>12 weeks) SMO administra- tion in patients with AUD were included in this systematic review, and 7 were included in the meta- analysis. Overall, the abstinence rate after 12 weeks of treatment was similar in the SMO and placebo groups, while it was significantly in favour of SMO compared to Naltrexone (NTX). The completion rate was similar in all three conditions. Mean corpuscular volume (MCV) levels favoured SMO over NTX, while Alcohol Craving Scale (ACS) scores did not favour SMO. The incidence of adverse reactions varied widely between studies. SMO in the chronic treatment of patients with AUD showed no superiority to placebo in our analysis of published RCTs, although many observational studies reported its beneficial effect in the long term. On the contrary, SMO was superior to NTX treatment on abstinence. The rate of study completion was similar in the three groups. Safety was not an issue in any of the studies included. Further studies are needed to better assess SMO efficacy and safety in the long term.</p>.

123 Long Term Safety and Efficacy of Radiosurgery for High-Risk Brain Cavernomas

123 Long Term Safety and Efficacy of Radiosurgery for High-Risk Brain Cavernomas

290. EVALUATION OF THE POST-OPERATIVE GERD AND RESIDUAL CHEST PAIN AFTER POEM

Abstract Background Per-oral endoscopic myotomy (POEM) for esophageal achalasia has increasingly become a mainstream procedure worldwide, and its short and middle term efficacy and safety are more and more recognized. However, the long-term outcomes are still unclear and we sometimes experience the cases with severe post-operative GERD and residual chest pain. Methods We retrospectively reviewed 241 cases who were performed POEM in our center from Apr 2015 to Feb 2022 and evaluate their post-operative GERD and residual chest pain. Results Before POEM, 126 cases (54.8%) had chest pain. Residual chest pain 2 months after POEM was observed in 28 patients (23.1%). 78.2% had complete remission of chest pain after 1 year. Severe GERD (LA Grade C or D) 2 months and 1 year after POEM was observed in 5.1%, 8.2%, respectively. All of them except one patient were improved by PPIs. On univariable analysis, younger patients and pre-treatment were at risk for residual chest pain (p=0.02, p=0.04, respectively). Higher BMI was risk for severe GERD after 2 months (p=0.02), however it was not associate with that after 1 year (p=0.04). Conclusion Younger age and pre-treatment were at risk for residual chest pain after POEM and post-operative severe GERD was related to higher BMI, however almost all of them was controlled by PPIs. The number of cases was small, so it is necessary to accumulate more cases.

Safety and Efficacy of Bimekizumab in Patients with Psoriatic Arthritis: 2-Year Results from Two Phase 3 Studies.

Psoriatic arthritis (PsA) is a chronic inflammatory disease requiring long-term treatment. Bimekizumab, a monoclonal IgG1 antibody that selectively inhibits interleukin (IL)-17F in addition to IL-17A, has demonstrated tolerability and sustained clinical efficacy for up to 1year for patients with PsA. Here, we report the longer-‍term safety and efficacy of bimekizumab up to 2years. BE OPTIMAL (biologic disease-modifying antirheumatic drug [bDMARD]-naïve) and BE COMPLETE (prior inadequate response/intolerance to tumor necrosis factor inhibitors [TNFi-IR]) assessed subcutaneous bimekizumab 160mg every 4weeks in patients with PsA. BE OPTIMAL included a reference arm (adalimumab 40mg every 2weeks); patients switched to bimekizumab at week 52 with no washout between treatments. BE OPTIMAL week 52 and BE COMPLETE week 16 completers were eligible for the BE VITAL open-label extension. Efficacy outcomes are reported to week 104/100 (BE OPTIMAL/BE COMPLETE). A total of 710/852 (83.3%) bDMARD-naïve and 322/400 (80.5%) TNFi-IR patients completed week 104/100. Up to 104weeks, patients treated with bimekizumab in BE OPTIMAL and BE COMPLETE had treatment-emergent adverse event incidence rates (exposure-adjusted incidence rate/100 patient-years) of 179.9 (95% CI 166.9, 193.7) and 100.3 (89.2, ‍112.4), respectively. The proportion of patients achieving efficacy outcomes (≥ 50% improvement from baseline in American College of Rheumatology [ACR] response criteria, 100% improvement from baseline in Psorisis Area and Severity Index [PASI], minimal disease activity [MDA]) was sustained in all patients from week 52 to week 104/100. Bimekizumab was well tolerated for up to 2years of treatment and no new safety signals were observed. Sustained clinical efficacy was observed up to 2years in bDMARD-naïve and TNFi-IR patients with active PsA. Patients switching from adalimumab to bimekizumab demonstrated further improvement in skin and nail symptoms, and sustained efficacy in joint symptoms. BE OPTIMAL (NCT03895203), BE COMPLETE (NCT03896581), BE VITAL (NCT04009499).

Long term efficacy and safety of lerodalcibep in patients with atherosclerotic cardiovascular disease (liberate-CVD)

Long term efficacy and safety of lerodalcibep in patients with atherosclerotic cardiovascular disease (liberate-CVD)

Long term efficacy and safety of saroglitazar in metabolic-dysfunction associated steatotic liver disease including patients with advanced fibrosis: real world data of 96 week therapy

Long term efficacy and safety of saroglitazar in metabolic-dysfunction associated steatotic liver disease including patients with advanced fibrosis: real world data of 96 week therapy

2210 The adjustable Transobturator male system (ATOMS) for urinary incontinence: 9 year sub analysis within the geriatric population

Abstract Introduction The ongoing rise in prostate cancer rates and consequent prostatectomy have led to an increase in rates of male stress urinary incontinence. ATOMS is an adjustable sling requiring no manual input and suitable for frailer patients. We investigated the long term efficacy of the ATOMsin managing SUI and performed a sub-analysis within the geriatric population (aged 75+). Method 69 men (mean: 70.2, range 50–81) underwent an ATOMS insertion between 2015–2019. Follow up data for up to 9 years were analysed (mean: 5.8, range 5–9 years). Out of the 69 men in the original cohort, 19 were aged 75+ (mean: 76.5, range: 75–81). 17 had SUI post radical prostatectomy, 1 post TURP and 1 post AP resection. Results Out of the 19 men, 14 (74%) were dry post ATOMsimplant insertion (ie using maximum one pad per day for reassurance). This rate is lower compared with the original cohort (79.7%). The average number of top ups to achieve dryness was 3 (same as the initial cohort). Out of the 14 men who remained incontinent in the initial cohort, 5 were 75+. Of these, 2 reported a significant improvement in their incontinence without meeting the ‘dry’ criteria. 1 had his ATOMsdevice removed due to infection. 1 was switched to an artificial urinary sphincter. 1 remains incontinent and is being managed with botox injections. There were no cases of mechanical failure. Conclusions ATOMS appears to be an efficacious and safe procedure in the geriatric population, with only marginal difference in dry rates compared to the non-geriatric cohort. The main benefit over an artificial sphincter is that it exerts a passive effect to prevent incontinence and requires no patient input for every void. This is especially prudent as geriatric patients may lose hand dexterity or cognitive ability over time.

Long Term Safety, Feasibility and Efficacy of Left Bundle Branch Area Pacing in a Large Single Centre Study

Long Term Safety, Feasibility and Efficacy of Left Bundle Branch Area Pacing in a Large Single Centre Study

Psychoeducation group facilitation training for nurses: A quality improvement project.

According to the 2020-2021 National Survey of Children's Health, 23.3% of children and adolescents met criteria for one or more mental, emotional, developmental, or behavioral problems. However, the prevalence of depression, anxiety, and post traumatic stress disorder have risen dramatically since the COVID-19 pandemic, leading to an increase in suicidal ideations, completed suicides, and pediatric psychiatric hospitalizations. There is a critical need for high quality mental healthcare treatments, including psychoeducation. Inpatient psychiatric nurses are well positioned to lead psychoeducation groups, but few receive training. Nurses at a designated inpatient pediatric psychiatric unit expressed discomfort in facilitating psychoeducation groups. This is a one-group prepost and follow-up quality improvement study designed to improve the quality of psychoeducation group facilitation skills provided by nurses at a child and adolescent inpatient psychiatric unit. A convenience sample of nurses (N = 16) participated. Baseline (T1), post-test (T2), and follow-up (T3) data were collected regarding nurses' self-efficacy, evidence based-practice attitudes, and knowledge of psychoeducation group facilitation. Nurses received 45 min of psychoeducation group facilitation education and 15 min of skills simulation. The nurses' knowledge, self-efficacy, and attitudes toward evidence-based practice when facilitating psychoeducation groups increased significantly from the baseline to the post-test, which was sustained from the post-test to the follow-up period. Study findings indicate the importance of supporting nurses in their role of psychoeducation group facilitation through education and program structure. Further research is needed to assess longer term sustainability and efficacy in leading psychoeducation groups.

Stem cell therapy in diabetic men with erectile dysfunction: a 24-month follow-up of safety and efficacy of two intracavernous autologous bone marrow derived mesenchymal stem cells injections, an open label phase 2 clinical trial

BackgroundRecently we reported results of phase 1 pilot clinical trial of 2 consecutive intracavernous (IC) injection of autologous bone marrow-derived mesenchymal stem cells (BM-MSCs) for the first time in the treatment of diabetic patients with erectile dysfunction (DM-ED). In phase 2 of this study our aim is to evaluate long term safety and efficacy of IC injections of BM-MSC on additional eight patients with DM-ED.ResultsEach patient received 2 consecutive IC injections of BM-MSC and evaluated at 1, 3, 6, 12, and 24-month time points. Primary outcome was the tolerability and safety of stem cells therapy (SCT), while the secondary outcome was improvement of erectile function (EF) as assessed using the International Index of Erectile Function-5 (IIEF-5), Erection Hardness Score (EHS) questionnaires, and Color Duplex Doppler Ultrasound (CDDU). IC injections of BM-MSCs was safe and well-tolerated. Minor local and short-term adverse events related to the bone marrow aspiration and IC injections were observed and treated conservatively. There were significant improvement in mean IIEF-5, EHS, all over the follow-up time points in comparison to the baseline. At 24-month follow up there were significant decline in the mean IIEF-5, and EHS compared to the baseline. The mean basal and 20-min peak systolic velocity was significantly higher at 3-month after the IC injections compared to baseline.ConclusionsThis phase 2 clinical trial confirmed that IC injections of BM-MSC are safe and improve EF. The decline in EF over time suggests a need for assessing repeated injections.Clinical trial registrationNCT02945462

O-269 Long term efficacy of Linzagolix in women with endometriosis-associated pain

Abstract Study question Does once-daily linzagolix taken up to 12 months maintain pain reduction observed at 6 months in women with endometriosis? Summary answer Linzagolix alone or in combination with combined add-back therapy (ABT) for 12 months maintains improvements of endometriosis-associated pain (EAP) observed at 6 months. What is known already Linzagolix is an oral GnRH antagonist under development for EAP. In the EDELWEISS 3 study, the 200mg+ABT dose met its co-primary endpoints at 3 months by reducing dysmenorrhea (DYS) and non-menstrual pelvic pain (NMPP) and secondary endpoints such as DYS, NMPP, overall pelvic pain and dyschezia as well as interference of pain with daily activities at 6 months. Linzagolix 75mg significantly reduced dysmenorrhea but not NMPP at 3 months. Both doses of linzagolix were well tolerated over 6 months of treatment. Study design, size, duration EDELWEISS 6 was the extension study of EDELWEISS 3, a placebo-controlled Phase 3 study, investigating linzagolix 75mg and 200mg+ABT for up to 6 months in women with moderate to severe EAP. Subjects who completed EDELWEISS3 were invited to enter the extension study for an additional 6-month treatment on the same linzagolix dose; placebo subjects were randomized to either active dose. After end of treatment, subjects entered a drug-free post-treatment period of 6 months. Participants/materials, setting, methods Women participating to EDELWEISS 6 received linzagolix for up to 12 months consecutively. Key efficacy assessments included dysmenorrhea and non-menstrual pelvic pain (NMPP), dyspareunia (assessed with a 4-point VRS), overall pelvic pain and dyschezia (assessed with an 11-point NRS), and interference of pain with daily activities (assessed with the EHP-30 pain domain) at Month 12. We report the results for dysmenorrhea and NMPP. Main results and the role of chance Of 484 participants in EDELWEISS 3, 353 (72.9%) entered EDELWEISS 6 and were evaluated for efficacy. Demographics were similar to EDELWEISS 3 with a mean age of 35 years, a BMI of 24kg/m2 and 99% of subjects being White. Mean (SD) monthly baseline dysmenorrhea and NMPP pain scores on the VRS were 2.28 (0.41) and 1.76 (0.45), respectively. The threshold for a meaningful pain reduction was established at Month 3, being -1.10 and -0.80 for dysmenorrhea and NMPP, respectively. At Month 3, the proportion of subjects with a reduction of dysmenorrhea of 1.10 or greater, and stable or decreased use of analgesics was 45.2% for the 75mg group and 75.4% for the 200mg+ABT group, which increased to 49.6% and 84.7%, respectively, at Month 6, the end of EDELWEISS 3. At Month 12, the end of EDELWEISS 6 treatment, the proportion of subjects was further increased to 55.9% and 91.0%, respectively. For NMPP, the proportion of subjects with a reduction of 0.80 or greater and stable or decreased use of analgesics at Month 3 was 36.5% for 75 mg and 51.7% for 200mg+ABT, increased to 54% and 61% at Month 6, and increased to 59.5% and 67.6%, respectively, until Month 12. Limitations, reasons for caution Edelweiss 6 is the extension of the previously reported double blind, placebo controlled Edelweiss 3 study. The results confirm efficacy of 200mg + ABT at 12 months. Longer term data is required to further understand efficacy of GnRH antagonists in women with endometriosis associated pain. Wider implications of the findings The linzagolix 200mg+ABT group provided substantial and sustained improvement in EAP symptoms. The 75mg which did not meet the primary endpoint in EDELWEISS 3 still demonstrated marked improvements. There exists a substantial need for different treatment regimens in women suffering from endometriosis. Trial registration number NCT04335591

The challenge of running trials in advanced angiosarcoma: A systematic review of the literature from EORTC/STBSG to guide the development of angiosarcoma-specific trials

IntroductionWhile available systemic treatments have modest long term efficacy in advanced angiosarcoma, immunotherapy represents an interesting new therapeutic opportunity. To establish its benefit, it is required to conduct a clinical trial assessing its efficacy and toxicity compared to standard treatments. Material and methodsThis is a literature review from PubMed search. ResultsSeveral systemic treatments (chemotherapy and TKI) are currently used in advanced angiosarcoma with ORR ranging from 12.5 to 68 % and PFS from 2 to 7 months. However, few randomized trials, mainly phase II, has been conducted to compare these treatments. While most centers propose doxorubicin containing regimens or paclitaxel in 1st or 2nd line, a high heterogeneity of regimens administered in this setting is observed even across sarcoma specialized centers with no consensual standard treatment. Encouraging signals of immunotherapy activity have been reported in angiosarcoma from several retrospective and phase II studies assessing anti-PD1 either alone or in combination with anti CTLA4 or TKI. Although cutaneous and head and neck location seems to benefit more from immunotherapy, response may be observed in any angiosarcoma subtype. In sarcoma in general and AS in particular, no biomarker has been clearly established to predict the efficacy of immunotherapy: high tumor mutational burden and presence of tertiary lymphoid structures are under assessment. DiscussionEven essential, developing a randomized clinical trial in AS struggles with the heterogeneity of the disease, the lack of consensual standard regimen, the uncertainty on optimal immunotherapy administration and the absence of established predictive biomarkers. ConclusionInternational collaboration is essential to run randomized trial in advanced AS and asses the efficacy of immune therapy in this rare and heterogeneous disease.

Sjogren's Antibodies and Neonatal Lupus: A Scoping Review.

Sjogren's syndrome (SS) is an autoimmune disease characterized by inflammation of exocrine glands. The disorder predominantly affects middle-aged women. Autoantibodies, includinganti-SS-A/Ro and anti-SS-B/La antibodies, are present in most cases of SS. These antibodies can cross the placenta and likely play a role in pregnancy complications as well as the development of neonatal lupus, resulting in congenital heart block (CHB). It is essential to monitor the fetus for CHB during pregnancy. In particular, screening with echocardiography and monitoring heart rate at home are recommended practices. Regarding medical management, hydroxychloroquineand glucocorticoids have shown promise in reducing cardiac manifestations, but further research is needed to elucidate their longer term efficacy and safety. This scoping review analyzes literature from 2001 to 2024, focusing on pregnancy outcomes among women with SS, clinical manifestations of neonatal lupus, the role of anti-SS-A/Ro and anti-SS-B/La antibodies in the development of neonatal lupus and CHB, and emphasizes the need for future research efforts to refine treatment protocols and enhance clinical care strategies for pregnant women with SS.

Comparative Analysis of Standard Percutaneous Nephrolithotomy (PCNL) and Mini-Percutaneous Nephrolithotomy (Mini-PCNL) for Renal Stones Larger Than 2 cm at a Rural Hospital: A Study Protocol.

Background Percutaneous nephrolithotomy (PCNL) is considered a standard treatment option for large-size renal stones but is associated with drawbacks such as bleeding and prolonged recovery. Mini-PCNL offers a less invasive alternative, but its efficacy compared to standardPCNLfor renal stones larger than 2 cm remains under debate. This study aims to compare the efficacy and safety of standard PCNLversus mini-PCNLfor renal stone sizes more than2 cm. Methods This single-centre prospective interventional study willbe conductedat Acharya Vinoba Bhave Rural Hospital (AVBRH). The study will include patients 18 to 70 years of age undergoing unilateralPCNLfor renal calculi. Patients with renal stones larger than 2 cm will be enrolled. Data on stone-free rate (SFR), operative duration, hospital stay time, surgical site infection, haemoglobin (Hb) drop, need for blood transfusion, and post-surgery fever will be collected and compared between the two procedures.Statistical analysis of data willbe performedusingdescriptive and analytical statistics. Results The study aims to enrol a total of 32 patients.Data analysis willbe doneusing IBM SPSS Statistics for Windows, Version 24 (Released 2016; IBM Corp., Armonk, New York). Conclusion This study will provide valuable insights into thecomparative outcomein terms ofefficacy and safety of standard PCNL and mini-PCNLfor kidney stoneslarger than 2 cm.

Using serial 18F-FDG PET/CT PERCIST response to predict responses to immune check point inhibitors in patients with advanced cutaneous squamous cell carcinoma.

9548 Background: The role of serial 18F-FDG-PET/CT imaging in predicting long term efficacy of PD-1 targeted immune checkpoint inhibitor (ICI) in advanced cutaneous squamous cell carcinoma (cSCC) is unknown. This study aims to correlate PET PERCIST response with time to progressive disease. Methods: This was a single-centre retrospective study of patients treated with ICI for advanced cSCC who had baseline and serial FDG-PET/CT within 4-months of commencement of ICI. PERCST 1.0 was calculated by two radiologists independently and classified into complete metabolic response (CMR), partial metabolic response (PMR), stable metabolic disease (SMD), and progressive metabolic disease (PMD). The primary endpoint was time to progression from commencement of ICI. Progression was determined by a multidisciplinary team combining clinical and radiological assessments. Results: Of the 242 patients treated with ICI for advanced cSCC during 2018-2023, 83 (34%) patients had a serial FDG PET. Of these, 53 patients met the inclusion criteria of ≤4-months from ICI to second PET. CMR (54.7%), PMR (15.1%), SMD (11.3%) and PMD (18.9%) were seen as initial responses by PERCST 1.0. With a median follow-up of 8-months (1-55), progressive disease as determined by the multidisciplinary team was observed in 11 patients (21%). For these 11 patients, the 4-month PERCST 1.0 response was measured as CMR (n=1), PMR (N=1), SMD (N=2) and PMD (N=7). The median TTP was significantly higher in CMR/ PMR vs SMD/PMD (55 vs 4-Months, P&lt;0.001). At data cut off, 94.6% (N=35) of patients with a 4-month PET PERCST 1.0 CMR/ PMR assessment did not progress clinically. Clinical response was maintained at a minimum of 12-months in 94.7% (n=18) of those responding. Conclusions: Rate of CMR and PMR in advanced cSCC is high with ICI. Early PET CMR/ PMR appears to predict long term durable responses. Incorporation of PET/CT into the assessment of response in cSCC may help guide treatment decisions by early identification of those patients not responding to ICI.

#1327 mTOR inhibitors in kidney transplant recipients: long-term efficacy and discontinuation causes

Abstract Background and Aims mTOR inhibitors use in kidney transplantation showed conflicting results, but their use as add-on treatment to calcineurin inhibitors (CNI) is still considered a valuable option. High frequency of drug discontinuation hinders their use in clinical practice. We present a real-life study on long term efficacy of mTOR inhibitors use vs mycophenolic acid (MPA) and the causes of drug discontinuation. Method This is an observational retrospective monocentric study. We analyzed data from 773 patients that underwent kidney transplantation at Hospital Clinic between 2013 and 2019. Induction treatment was based either on basiliximab or a lymphocyte-depleting agent together with methylprednisolone. Maintenance treatment was based either on MPA or mTORi with a CNI. Baseline differences were accounted for with the Inverse Propensity of Treatment Weighting (IPTW). Outcomes explored were death with a functioning graft, graft loss or biopsy-proven rejection. Predictors of outcome were explored through logistic regression or Cox-regression as appropriate. Results 335/773 (43.3%) of the patients received MPA as maintenance treatment, whereas 438/773 (56.6%) received an mTOR inhibitor. Average follow-up was 3.36 years (SD ± 2.04 years). No outcome resulted statistically significant at the logistic regression weighted with IPTW in the subgroup mTORi vs MPA: odds ratio was 1.16 (0.75-1.63, p = 0.57) for death, 0.87 (0.54-1.41, p = 0.59) for graft loss and 1.15 (0.79-1.67, p = 0.45) for biopsy-proven acute rejection. The per-protocol analysis confirmed these results as not statistically significant. Strongest predictors of death resulted having a deceased donor of cardiac death (HR 2.17 (1.06-4.40, p = 0.03), having a delayed graft function (HR 1.9 (1.26-2.87, p = 0.002) and being readmitted within three months from the surgery (HR 1.76 (1.21-2.55, p = 0.003). Diabetic nephropathy as cause of kidney disease was significant as well (HR 2.01 (1.24-3.26, p = 0.004). Proportion of patients with a CMV infection were significantly lower in the mTORi than MPA subgroups (33.5% vs 41.5%, p = 0.02). Proportion of patients developing neoplasias were not significantly different (10% vs 11.3%, p = 0.55). A total of 307/773 (39.7%) of patients changed their immunosuppressive treatment at a median time of 110 days (26.5-375.5). In the MPA subgroup 77/335 (22.9%) changed treatment, whereas in the mTORi subgroup 230/438 (52.5%). The causes of change due to drug-related events were the most represented and related to mTOR treatment: lymphocele in 36/773 (4.6%), complications of the surgical wound in 31/773 (4.1%) and edemas in 23/773 (2.9%) were the most common. Of note, unspecific physician preference was cause of discontinuation in a total of 66/773 (8.5%) patients. Conclusion Our data from a tertiary-center, real-life experience show that mTOR inhibitors are a solid alternative to MPA based protocols, with effectiveness proven at the multivariate and weighted analysis. mTOR treatment has a high rate of discontinuation, with most of the causes represented by post-intervention complications; interestingly, physician preference represent a frequent cause. Further experience and research is needed to optimize mTORi treatment, in order to maintain a therapeutic option for transplanted patients.