Short-term Efficacy Research Articles

The short-term efficacy of neoadjuvant SOX versus SOX plus immune checkpoint inhibitor following laparoscopic gastrectomy for locally advanced gastric cancer: a multicenter retrospective cohort study in China

BackgroundThis study aims to evaluate the short-term efficacy for locally advanced gastric cancer (LAGC) who accepted laparoscopic gastrectomy (LG) after neoadjuvant SOX versus SOX plus immune checkpoint inhibitors (ICIs).MethodsLAGC patients who accepted LG after neoadjuvant SOX (SOX-LG, n = 169) and SOX plus ICIs (SOX + ICIs-LG, n = 140) in three medical centers between Jan 2020 and Mar 2024 were analyzed. We compared the tumor regression, treatment-related adverse events (TRAEs), perioperative safety between two groups, and explored the risk factors of postoperative complications (POCs) for LG after neoadjuvant therapy.ResultsThe baseline characteristics were comparable between two groups (P > 0.05). SOX + ICIs-LG group acquired a higher proportion of objective response (63.6% vs. 46.7%, P = 0.003), major pathological response (43.6% vs. 31.4%, P = 0.001), and pathological complete response (17.9% vs. 9.5%, P = 0.030). There were no significant differences in the TRAEs rates, operation time, R0 resection, retrieved lymph nodes, postoperative first flatus, and hospitalized days, overall and severe POCs between two groups (P > 0.05). Patients in the SOX-ICIs-LG group had lower estimated blood loss (EBL) compared with SOX-LG (P = 0.001). Multivariate analysis showed that more EBL (P = 0.003) and prognostic nutritional index (PNI) < 40 (P = 0.005) were independent risk factors of POCs for LG after neoadjuvant therapy.ConclusionNeoadjuvant SOX plus ICIs brings better tumor regression and similar TRAEs compared with SOX alone for LAGC. SOX + ICIs-LG is safe and feasible to conduct with less EBL. Surgeons should focus on the perioperative management to control POCs for patients with PNI < 40 and more EBL.

Comparison of the short-term efficacy of MIS-TLIF and Endo-LIF for the treatment of two-segment lumbar degenerative disease

BackgroundThis study retrospectively compared short-term clinical outcomes and complications of minimally invasive surgery transforaminal lumbar interbody fusion(MIS-TLIF)and endoscopic lumbar interbody fusion(Endo-LIF))for two-segmental lumbar degenerative disease, aiming to guide spine surgeons in selecting surgical approaches.MethodsFrom January 2019 to December 2023, 30 patients were enrolled,15 in the MIS-TLIF group and 15 in the Endo-LIF group. All patients were followed up for more than 3 months after surgery and the following information was recorded: (1)surgery time, difference in hemoglobin between preoperative and postoperative, surgical costs, first time out of bed after operation, postoperative hospitalization time, postoperative complication; (2) ODI score (The Oswestry Disability Index), leg and back VAS score (Visual Analogue Scale), and lumbar vertebra JOA score (Japanese Orthopaedic Association Scores); (3) MacNab score at final follow-up to assess clinical outcome, CT to evaluate lumbar fusion.ResultsThere were significant differences between the two groups regarding operation time and cost, with the MIS-TLIF group performing significantly better. Intraoperative bleeding was considerably less in the Endo-LIF group compared to the MIS-TLIF group. However, there were no significant differences in the time of the first postoperative ambulation, postoperative hospitalization time, and postoperative complications. There was no significant difference in preoperative VAS, ODI, and JOA between the two surgical groups There were no significant differences in VAS(leg), ODI, and JOA scores between the two groups before and at 1 day,7 days, 1 month, 3 months and final follow-up. However, at 1 day postoperatively, the VAS( back)score in the Endo-LIF group was lower than that in the MIS-TLIF group, and the difference was statistically significant. At the final follow-up, all patients achieved grade III and above according to the Bridwell criteria, and there was no significant difference between the two surgical groups compared to each other. According to the MacNab score at the final follow-up, the excellent rate was 80.00% in the Endo-LIF group and 73.33% in the MIS-TLIF group, with no significant difference between the two groups.ConclusionThere was no significant difference in short-term efficacy and safety between Endo-LIF and MIS-TLIF for two-segment degenerative lumbar diseases. MIS-TLIF has a shorter operative time and lower costs, while Endo-LIF causes less tissue damage, blood loss, and early postoperative pain, aiding long-term recovery. Both MIS-TLIF and Endo-LIF are promising for treating two-segment lumbar degenerative disease. The choice of a surgical procedure depends on the patient’s financial situation, their ability to tolerate surgery, and the surgeon’s expertise.

Short-term outcomes of totally robotic versus robotic-assisted distal gastrectomy for gastric cancer: a single-center retrospective study

BackgroundTotally robotic distal gastrectomy (TRDG) is being used more and more in gastric cancer (GC) patients. The study aims to evaluate the short-term efficacy of TRDG and robotic-assisted distal gastrectomy (RADG) in the treatment of GC.MethodsWe retrospectively collected the clinical data of patients who underwent TRDG or RADG, of which 60 patients were included in the study: 30 cases of totally robotic and 30 cases of robotic-assisted. The short-term efficacy of the two groups was compared.ResultsThere was no significant difference in the clinicopathological data between the two groups. Compared to RADG, TRDG had less intraoperative blood loss(P = 0.019), less postoperative abdominal drainage(P = 0.031), shorter time of exhaust( P = 0.001) and liquid diet(P = 0.001), shorter length of incision(P<0.01), shorter postoperative hospital stays(P = 0.033), lower postoperative C-reactive protein(CRP)(P = 0.024) and lower postoperative Visual Analogue Scale(VAS) scores(P = 0.048). However, no significant statistical differences were found in terms of total operation time(P = 0.108), number of lymph nodes retrieved(P = 0.307), time for anastomosis(P = 0.450), proximal resection margin(P = 0.210), distal resection margin(P = 0.202), postoperative complication(P = 0.506), total hospital cost(P = 0.286) and postoperative white blood cell(WBC)(P = 0.113).ConclusionsIn terms of security and technology, TRDG could serve as a better treatment method for GC.

Clinical-radiomics combination model for predicting the short-term efficacy of bipolar transurethral enucleation of the prostate in patients with benign prostatic hyperplasia

Abstract Background Bipolar transurethral enucleation of the prostate (B-TUEP) is a well-established surgical treatment for benign prostatic hyperplasia (BPH); however, its efficacy may vary depending on patient characteristics. Magnetic resonance imaging with radiomics analysis can offer comprehensive and quantitative information about prostate characteristics that may relate to surgical outcomes. This study aimed to explore the value of magnetic resonance imaging and radiomics analysis in predicting the short-term efficacy of B-TUEP for BPH. Materials and methods A total of 137 patients with BPH who underwent B-TUEP at 2 institutions were included. Radiological features were measured in the magnetic resonance images, and the radiomics score was developed from 1702 radiomics features extracted from the prostate and transitional zone regions of interest. Three prediction models were developed and validated based on clinical-radiological features, radiomic features, and their combinations. The models were evaluated using the area under the receiver operating characteristic curve, calibration curve, and decision curve analysis. Results The combination model exhibited the highest area under curve in both the training set (0.838) and the external validation set (0.802), indicating superior predictive performance and robustness. Furthermore, the combination model demonstrated good calibration (p &gt; 0.05) and optimal clinical utility. The combination model indicated that a higher maximum urine flow rate, lower transitional zone index, and higher radiomics score were associated with an increased risk of poor efficacy. Conclusions Magnetic resonance imaging with radiomic analysis can offer valuable insights for predicting the short-term efficacy of B-TUEP in patients with BPH. A combination model based on clinical and radiomics features can assist urologists in making more precise clinical decisions.

Comparison of short-term clinical efficacy between modified Kamikawa anastomosis and double tract anastomosis after laparoscopic proximal gastrectomy.

This study aimed to explore the short-term clinical efficacy of modified Kamikawa anastomosis and double tract anastomosis after laparoscopic proximal gastrectomy. A retrospective analysis was carried out by collecting the clinical and pathological data of 42 patients who underwent laparoscopic proximal gastrectomy after admission in our centre from May 2020 to October 2022. Among the 42 enrolled patients, 22 underwent modified Kamikawa anastomosis (modified Kamikawa group), and the other 20 underwent double tract anastomosis (double tract group). Outcome measures included intraoperative condition, postoperative recovery, postoperative quality of life, postoperative nutritional status and gastroesophageal reflux. The patients were followed up using outpatient examination and telephone interviews to identify their nutritional status, reflux esophagitis and anastomotic status. (1) Intraoperative condition: Compared with the double tract group, the modified Kamikawa group had significantly prolonged time for operation and digestive tract reconstruction. However, no statistically significant difference in intraoperative blood loss was found between the two groups. (2) Postoperative recovery: Compared with the double tract group, the modified Kamikawa group had a significantly shorter time for the first postoperative intake of fluids, drainage tube placement and postoperative hospital stay. No statistically significant difference in the time to first postoperative anal exhaust and postoperative complications was found between the two groups. (3) Postoperative quality of life: Compared with the double tract group, the modified Kamikawa group showed better quality of life at 12 months after surgery. (4) Postoperative nutritional status and gastroesophageal reflux: No statistically significant difference in hemoglobin, total serum albumin, albumin, body mass index, MUST score, PG-SGA score, grading of reflux esophagitis using the Los Angeles classification system and GERD score was found between the two groups at 6 and 12 months after surgery. All patients did not experience anastomotic stenosis and tumour recurrence or metastasis. Modified Kamikawa anastomosis is a safe and feasible treatment in laparoscopic proximal gastrectomy, which can ensure good postoperative anti-reflux effect and nutritional status. It has the advantage of better postoperative recorvery and quality of life compared with double tract anastomosis.

The Performance of Peroral Endoscopic Myotomy in Sigmoid-Type Achalasia

Objective: Sigmoid-type achalasia represents an advanced stage of achalasia characterized by significant dilation and tortuosity of the esophageal lumen. Considering the demonstrated efficacy of peroral endoscopic myotomy (POEM) in treating early-stage achalasia, this procedure may offer an alternative therapeutic approach for sigmoid-type achalasia. This study aimed to assess POEM’s feasibility and short-term efficacy in patients with sigmoid-type achalasia. Materials and Methods: We enrolled 16 consecutive patients with sigmoid-type achalasia (eight with type 1 and eight with type 2). The anticipated outcomes were symptom relief during the 12-month follow-up period (evaluated through a reduction in Eckardt symptom scores), an acceptable incidence of procedure-related adverse events, and a decrease in esophageal diameter and barium height. Results: POEM was successfully performed in all cases, with a median operative time of 118.50 minutes (range: 52–206 minutes). No serious complications associated with POEM were observed. During the 12-month follow-up period, the median Eckardt symptom score decreased from 6 (2-10) preoperatively to 1 (0-3) (P = 0.008). Complications were mucosal injuries (31.25% of cases), pneumoperitoneum (12.5%), and minor bleeding (6.25%), although no interventions were needed. Conclusion: POEM procedure has exhibited favorable treatment outcomes, showcasing a high clinical success rate in addressing sigmoid-type achalasia. Despite the occurrence of acceptable adverse events, the procedure remains a viable alternative treatment or bridging therapy for sigmoid-type achalasia. Nonetheless, it is crucial to acknowledge that this procedure presents greater challenges in comparison to the treatment of typical achalasia.

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.

575. PATHOLOGIC RESPONSE AND RESIDUAL LYMPH NODE PATTERNS IN ESOPHAGEAL SQUAMOUS CELL CARCINOMA PATIENTS UNDERGOING NEOADJUVANT IMMUNOCHEMOTHERAPY COMPARED TO NEOADJUVANT CHEMORADIOTHERAPY

Abstract Background Concurrent chemoradiotherapy is the cornerstone treatment for locally advanced esophageal cancer. Immunochemotherapy, established as the standard treatment for advanced esophageal cancer, concurrently exhibits excellent short-term efficacy in the neoadjuvant stage. Combining concurrent chemoradiotherapy with immunochemotherapy may enhance control over primary tumor and metastatic lymph nodes, potentially improving patients’ outcomes and survival. Therefore, comparing the pros and cons of these two treatment modalities in controlling primary lesions and lymph node metastasis is crucial for guiding clinical treatment. Methods Our study enrolled a total of 137 patients with esophageal squamous cell carcinoma (ESCC) who underwent neoadjuvant immunochemotherapy (NICT) and 151 ESCC patients who underwent neoadjuvant chemoradiotherapy (NCRT) between January 2019 and March 2022. We documented baseline patient characteristics, including clinical TNM stage, tumor location, and clinical metastatic lymph nodes, as well as pathologic outcomes such as pathological stage, pathologic complete response (pCR), tumor regression grade (TRG), and pathological metastatic lymph nodes. To balance baseline patient characteristics, we employed inverse probability of treatment weighting (IPTW) to adjust for confounding in different neoadjuvant treatment groups. Results After balancing baseline characteristics, there was no statistically significant difference in pathologic outcomes, including pCR rate, major pathologic response rate, TRG, yp-TNM stage, and R0 resection rate between NCRT and NICT. However, patients with clinical N2-3 stage (OR 2.66; 95% CI 1.05 to 6.79) or lower thoracic esophageal tumors (OR 2.31; 95% CI 1.05 to 5.04) had a higher chance of achieving pCR after NICT compared to NCRT. For patients undergoing NICT, the ypN0 rates for clinically N0 patients were 100.0% (5/5), while only 65.0% (13/20) of clinical N0 patients achieved ypN0 in the NCRT group. Even after adjusting for differences in clinical stage, tumor location, and baseline lymph node metastasis rates in various locations, the NICT group still demonstrated a lower rate of abdominal lymph node metastasis during surgery (p=0.030). Conclusion In comparison to concurrent chemoradiotherapy, immunochemotherapy exhibits comparable overall disease control for primary lesions and lymph node metastasis. Notably, patients with clinical N2-3 and lower thoracic esophageal cancer may derive greater benefits from NICT. The potential of immunochemotherapy in enhancing the effectiveness of chemoradiotherapy is evident in its ability to improve the control of occult metastatic lymph nodes and reduce abdominal lymph node metastasis.

271. EFFICACY OF NEOADJUVANT CF OR DCF, FLOT PLUS NIVOLUMAB FOR RESECTABLE LOCALLY ADVANCED ESOPHAGEAL SQUAMOUS CELL CARCINOMA (JCOG1804E: FRONTIER)

Abstract Background The standard neoadjuvant therapy for resectable locally advanced esophageal squamous cell carcinoma (ESCC) in Japan is DCF (docetaxel [DTX] + cisplatin [CDDP] + 5-fluorouracil [5-FU]). In addition, neoadjuvant immune checkpoint inhibitors have shown a survival benefit for metastatic ESCC and several other cancers. We previously reported that neoadjuvant CF (CDDP + 5-FU) + nivolumab (Nivo), DCF + Nivo, and FLOT (5-FU + l-leucovorin [l-LV] + oxaliplatin [L-OHP] + DTX) + Nivo are tolerable and show promising short-term efficacy for resectable locally advanced ESCC. However, the long-term efficacy of these neoadjuvant treatments remains unclear. Methods JCOG1804E (FRONTiER) is a multi-cohort phase I study designed to evaluate the safety and efficacy of Nivo combined with neoadjuvant CF (cohorts A and B [adding run-in Nivo]) or DCF (cohorts C and D [adding run-in Nivo]) or neoadjuvant FLOT (cohort E) for resectable locally advanced ESCC. The eligibility criteria were histologically proven ESCC staged as cT1N1-3M0 or cT2-3N0-3M0 (8th UICC TNM classification), age 20-75 years, performance status (PS) ≤1, and no prior cancer treatment. Secondary efficacy endpoints were pathological complete response (pCR), progression-free survival (PFS), and overall survival (OS) for each treatment. Results Thirty-seven patients were enrolled (median age [range]: 62 [31-75] years; PS 0/1: 32/5; clinical stage I/II/III/IVA: 7/9/20/1; PD-L1 TPS &amp;lt;1%/≥1%/unknown: 10/26/1). In CF-based treatment cohorts (n=13), median follow-up time (range), pCR, and 3-year PFS were 42.3 (20.9-50.1) months, 7.7%, and 66.6%, respectively. The respective values for the DCF-based treatment cohorts (n=12) were 32.2 (8.0-39.8) months, 33.3%, and 83.3% and for the FLOT-based treatment cohort (n=12) were 16.2 (5.3-20.6) months, 41.7%, and 91.7%. In the overall population (n=37), median follow-up time was 31.9 (5.3-50.1) months and 3-year OS was 82.4%. Conclusion Neoadjuvant CF or DCF or neoadjuvant FLOT + Nivo followed by surgery for resectable locally advanced ESCC showed promising short- and long-term efficacy. (NCT03914443)

931P Deep learning models for predicting short-term efficacy in locally advanced nasopharyngeal carcinoma

931P Deep learning models for predicting short-term efficacy in locally advanced nasopharyngeal carcinoma

Myosin ATPase inhibition fails to rescue the metabolically dysregulated proteome of nebulin-deficient muscle.

Nemaline myopathy (NM) is a genetic muscle disease, primarily caused by mutations in the NEB gene (NEB-NM) and with muscle myosin dysfunction as a major molecular pathogenic mechanism. Recently, we have observed that the myosin biochemical super-relaxed state was significantly impaired in NEB-NM, inducing an aberrant increase in ATP consumption and remodelling of the energy proteome in diseased muscle fibres. Because the small-molecule Mavacamten is known to promote the myosin super-relaxed state and reduce the ATP demand, we tested its potency in the context of NEB-NM. We first conducted in vitro experiments in isolated single myofibres from patients and found that Mavacamten successfully reversed the myosin ATP overconsumption. Following this, we assessed its short-term in vivo effects using the conditional nebulin knockout (cNeb KO) mouse model and subsequently performing global proteomics profiling in dissected soleus myofibres. After a 4week treatment period, we observed a remodelling of a large number of proteins in both cNeb KO mice and their wild-type siblings. Nevertheless, these changes were not related to the energy proteome, indicating that short-term Mavacamten treatment is not sufficient to properly counterbalance the metabolically dysregulated proteome of cNeb KO mice. Taken together, our findings emphasize Mavacamten potency in vitro but challenge its short-term efficacy in vivo. KEY POINTS: No cure exists for nemaline myopathy, a type of genetic skeletal muscle disease mainly derived from mutations in genes encoding myofilament proteins. Applying Mavacamten, a small molecule directly targeting the myofilaments, to isolated membrane-permeabilized muscle fibres from human patients restored myosin energetic disturbances. Treating a mouse model of nemaline myopathy in vivo with Mavacamten for 4weeks, remodelled the skeletal muscle fibre proteome without any noticeable effects on energetic proteins. Short-term Mavacamten treatment may not be sufficient to reverse the muscle phenotype in nemaline myopathy.

The efficacy and safety of single-incision plus one-port laparoscopic surgery vs. conventional five-port laparoscopic surgery for duodenum-preserving pancreatic head resection.

Single-incision plus one-port laparoscopic duodenum-preserving pancreatic head resection (SILDPPHR+1) is yet to be reported, and therefore, its safety and efficacy have yet to be established. This study aimed to assess the short-term efficacy of SILDPPHR+1 in comparison to conventional laparoscopic duodenum-preserving pancreatic head resection (cLDPPHR). Consecutive patients who underwent SILDPPHR+1 and cLDPPHR procedures were screened. An analysis of the intraoperative and postoperative data of all patients was carried out. Nineteen patients who underwent SILDPPHR+1 and 24 patients who underwent cLDPPHR at Sichuan Provincial People's Hospital from October 15, 2021, to October 30, 2023, were enrolled in this study. All baseline parameters of both groups were comparable. There was a statistically significant difference in the cosmetic score between the groups (P<0.001). No statistically significant differences were observed between the two groups regarding postoperative recovery, postoperative pancreatic fistula (POPF), bile leakage rate, delayed gastric emptying (DGE) rate, postpancreatectomy hemorrhage (PPH) rate, abdominal infection rate, or textbook outcomes. SILDPPHR+1 appears to be a reliable and safe procedure for certain patients, with no increase in the operating time or complications, similar to the results of cLDPPHR. Moreover, SILDPPHR+1 offers the added advantage of superior cosmetic results.

CT radiomics-based biomarkers can predict response to immunotherapy in hepatocellular carcinoma

Hepatocellular Carcinoma (HCC) remains a leading cause of cancer deaths. Despite the rise of immunotherapies, many HCC patients don't benefit. There's a clear need for biomarkers to guide treatment decisions. This research aims to identify such biomarkers by combining radiological data and machine learning. We analyzed clinical and CT imaging data of 54 HCC patients undergoing immunotherapy. Radiologic features were examined to develop a model predicting short-term immunotherapy effects. We utilized 9 machine learning and 2 ensemble learning techniques using RapidMiner for model construction. We conducted the validation of the above feature combination using 29 HCC patients who received immunotherapy from another hospital, and tested and validated it using XGBoost combined with a genetic algorithm. In 54 HCC patients, radiomics features varied significantly between those with partial response (PR) and stable disease (SD). Key features in Gray Level Run Length Matrix (GLRLM) and in adjacent tissues' Intensity Direct, Neighborhood Gray Tone Difference Matrix (NGTDM), and Shape correlated with short-term immunotherapy efficacy. Selected feature combinations of 15, 19, and 8/15 features yielded better outcomes. Deep learning, random forest, and naive bayes outperformed other methods, with Bagging being more reliable than Adaboost. In the validation set of 29 HCC patients, the mentioned feature combination also demonstrated favorable performance. Furthermore, we achieved improved results when employing XGBoost in conjunction with a genetic algorithm for testing and validation. The machine learning model built with CT image features derived from GLCM, GLRLM, IntensityDirect, NGTDM, and Shape can accurately forecast the short-term efficacy of immunotherapy for HCC.

The Short-Term Efficacy and Safety of Faricimab in Refractory Neovascular Age-Related Macular Degeneration: The Real-World Experience in Taiwan

Introduction: Anti-vascular endothelial growth factor (VEGF) treatment stands as the primary approach for neovascular age-related macular degeneration (nAMD). Faricimab has recently emerged as a novel anti-VEGF option for nAMD. This study aimed to assess the efficacy of faricimab in patients with refractory nAMD. Method: This retrospective study focused on refractory nAMD patients treated with faricimab at Taipei Veterans General Hospital from March 2023 to December 2023. Primary outcomes assessed the change in mean best-corrected visual acuity (BCVA) and central retinal thickness (CRT) over the first 4 months. Secondary outcomes included the presence of subretinal and intraretinal fluid (SRF and IRF) and changes in pigment epithelial detachment (PED). Subgroup analysis for the successful and unsuccessful treatment groups was conducted to identify potential confounding factors influencing treatment response. Result: This study included 42 eyes with refractory nAMD treated with faricimab. During a 6-month follow-up, no significant improvement in BCVA was observed, while CRT significantly decreased at all time points, except during the 5-month follow-up. Height PED showed significant reduction up to 5 months. The prevalence of SRF decreased significantly, while IRF remained lower but not significant. According to the treatment criteria, 67.4% successfully met the treatment goals. Subgroup analysis between successful and unsuccessful groups showed no significant differences in baseline characteristics, except a higher predominantly serous PED percentage in the successful group. Conclusion: Faricimab showed favorable outcomes in refractory nAMD patients. Further investigations are needed to understand the factors contributing to its efficacy.

Real-World Data in Children with Spinal Muscular Atrophy Type 1 on Long-Term Ventilation Receiving Gene Therapy: A Prospective Cohort Study.

Patients with spinal muscular atrophy type 1 (SMA-1) requiring invasive ventilation can be eligible for gene therapy if they tolerate at least 8 h off ventilation per day. We aimed to assess the short-term safety and efficacy of gene therapy (onasemnogene abeparvovec; Zolgensma) on respiratory function in SMA-1 patients ventilated via tracheostomy pre-gene therapy. A prospective cohort study included 22 patients. Patients were weaned off ventilation for at least 8 h daily by optimizing ventilator settings and duration, using cough augmentation, managing excessive airway secretions, enhancing nutrition, screening for respiratory bacterial colonization, and treating infections. Gene therapy was administered at a median age of 26 (Q1: 18, Q3: 43) months with a mean follow-up period of 7.64 (SD: 6.50) months. Gene therapy was safe and effective in resolving paradoxical breathing, improving cough ability, reducing airway secretions, and enhancing CHOP-INTEND scores. The clinical assessment and management implemented pre-gene therapy were effective in safely weaning patients for at least 8 h off ventilation daily. Gene therapy at a late age was safe and effective over the short-term period; however, long-term follow-up is recommended. In conjunction with gene therapy, high-quality clinical care is beneficial and should be paired with gene therapy.

EXPRESS: Short-term Efficacy of Moderate Intensity Rosuvastatin in COVID-19 Patients: A Randomized Clinical Trial.

As the COVID-19 pandemic persists, the exploration of adjunct therapies to mitigate disease severity remains a priority. Statins, known for their pleiotropic effects, have been under investigation for their potential role in managing COVID-19 complications. The study was conducted in a single referral hospital and adhered to Consolidated Standards of Reporting Trials (CONSORT) guidelines. Eligible participants were randomized in a 1:1 ratio into either the rosuvastatin group or the control group. Outcome measures included vital signs, laboratory data, clinical outcomes, and patient symptoms. Statistical analysis was performed using SPSS software. A total of 100 patients were enrolled. No significant differences were observed between the rosuvastatin and control groups in terms of baseline characteristics and laboratory parameters, except for the fact that rosuvastatin-treated patients showed lower levels of C-reactive protein (CRP) in comparison with the controls on both the first and fifth days (38.1 ± 16.3 vs. 50.5 ± 25.3) compared to the control group. Clinical outcomes, including hospital length of stay, Intensive Care Unit (ICU) admission, need for intubation, and 1-month mortality, did not differ significantly between the two groups. Symptom scales, as assessed by the Borg Rating of Perceived Exertion (Borg RPE) and Leicester Cough Questionnaire (LCQ), showed significant improvement in the rosuvastatin group compared to controls. Our study provides insights into the short-term efficacy of moderate-intensity rosuvastatin in COVID-19 patients. Further research is warranted to elucidate the long-term effects and optimal dosing of statins in COVID-19 management.

Effectiveness of the A3 robot on lower extremity motor function in stroke patients: A prospective, randomized controlled trial

BACKGROUND The results of existing lower extremity robotics studies are conflicting, and few relevant clinical trials have examined short-term efficacy. In addition, most of the outcome indicators in existing studies are scales, which are not objective enough. We used the combination of objective instrument measurement and scale to explore the short-term efficacy of the lower limb A3 robot, to provide a clinical reference. AIM To investigate the improvement of lower limb walking ability and balance in stroke treated by A3 lower limb robot. METHODS Sixty stroke patients were recruited prospectively in a hospital and randomized into the A3 group and the control group. They received 30 min of A3 robotics training and 30 min of floor walking training in addition to 30 min of regular rehabilitation training. The training was performed five times a week, once a day, for 2 wk. The t -test or non-parametric test was used to compare the three-dimensional gait parameters and balance between the two groups before and after treatment. RESULTS The scores of basic activities of daily living, Stroke-Specific Quality of Life Scale, FM balance meter, Fugl-Meyer Assessment scores, Rivermead Mobility Index, Stride speed, Stride length, and Time Up and Go test in the two groups were significantly better than before treatment (19.29 ± 12.15 vs 3.52 ± 4.34; 22.57 ± 17.99 vs 4.07 ± 2.51; 1.21 ± 0.83 vs 0.18 ± 0.40; 3.50 ± 3.80 vs 0.96 ± 2.08; 2.07 ± 1.21 vs 0.41 ± 0.57; 0.89 ± 0.63 vs 0.11 ± 0.32; 12.38 ± 9.00 vs 2.80 ± 3.43; 18.84 ± 11.24 vs 3.80 ± 10.83; 45.12 ± 69.41 vs 8.41 ± 10.20; 29.45 ± 16.62 vs 8.68 ± 10.74; P &lt; 0.05). All outcome indicators were significantly better in the A3 group than in the control group, except the area of the balance parameter. CONCLUSION For the short-term treatment of patients with subacute stroke, the addition of A3 robotic walking training to conventional physiotherapy appears to be more effective than the addition of ground-based walking training.

Assessment of the Short-Term Efficacy of a Wet-Cupping Therapy Session in Alleviating Symptoms of Fibromyalgia Syndrome: A Single-Arm Clinical Trial

Fibromyalgia holds significant importance in both primary care and physical treatment settings due to its high prevalence and the challenges associated with its diagnosis and management. This study aims to assess the short-term efficacy of a wet-cupping therapy session in alleviating symptoms of Fibromyalgia Syndrome (FMS). This study employed a prospective, single-arm intervention design. Baseline and post-treatment evaluations encompassed a comprehensive battery of assessment tools: Socio-Demographic Questionary, Visual Analog Scale (VAS) for Pain, Fatigue Severity Scale (FSS), Beck Depression Scale (BDS), Fibromyalgia Impact Questionnaire (FIQ). A total of 20 eligible individuals were enrolled in the study. Significant alterations in pain perception, as measured by the VAS, were evident both prior to treatment and at the end of the first week of treatment (p &lt; 0.001). Additionally, significant favorable effects of the treatment were observed on FSS and BDS levels during the initial week of therapy (p = 0.001, p = 0.01; respectively). Moreover, a marked improvement was identified in FIQ scores after treatment compared to baseline (p &lt; 0.001). Across all evaluated clinical indicators, cupping therapy exhibited a significant positive influence on FMS. In conclusion, wet cupping therapy yields favorable outcomes in terms of pain perception, fatigue reduction, mood enhancement, and the overall impact of fibromyalgia syndrome. Thus, it offers a promising avenue for therapeutic intervention among individuals afflicted by this condition.

Pulmonary artery banding in infants and young children with end-stage left ventricular dilated cardiomyopathy-Cohort Study.

Dilated cardiomyopathy (DCM) is the most common cardiomyopathy and 40-50% patients may die or need heart transplant in 5 years after diagnosis. Although heart transplantation being the most effective life-saving option of end-stage DCM, scarcity of donors and series of complications prevent many patients from receiving timely treatment. Pulmonary artery banding (PAB) is recently described as an alternative strategy for end-stage DCM, with low left ventricular function (LVEF) but preserved right ventricular function, may potentially restore heart function and delay the need for heart transplantation, but current clinical evidence is still insufficient. On the other hand, the medication treatment of DCM in pediatric patients is mostly based on the experience of adults. It remains unclear whether PAB combing medication treatment could benefit infants and young children patients. The aim of this study was to assess the short-term efficacy of PAB combined with medication therapy in infants and young children with end stage DCM, comparing with medication therapy alone. This is a retrospective analysis of 18 consecutive pediatric patients aged ranging from 1 month to 44 months old who diagnosed with end-stage DCM (LVEF30%) with preserved right ventricular function between 2019 and 2023 in our hospital. All patients had been treated with conventional medications for two months. Then they were divided in two groups: PAB surgery group (6/18), and non-surgery group (12/18). Regardless of whether surgery was performed, both groups continued to receive medication treatment. Recovery of ventricular function was primary endpoints. Secondary endpoints included 180-day mortality and severe heart failure (LVEF≤30%). We found there were no differences in age, weight, height, BMI, renal function, liver function, pulmonary hypertension, tricuspid valve regurgitation, mitral valve regurgitation and genetic abnormalities between those with and without PAB surgery. Comparing with non-surgery group, 5 patients in surgery group regain the normal cardiac ejection fraction (LVEF≥50%) (5/6, 83.3% vs. 4/12, 33.3%, P=0.131). A total of 3 patients had sudden death in non-surgery group, and there was no death in surgery group (P=0.180). 5 patients (5/12, 41.7%) still remain the low heart failure (LVEF≤40%) after 6 months of enrollment only given medical therapy, and none of patients present with LVEF≤40% in PAB surgery group (0/6, 0% vs. 8/12, 67.7%, P=0.034). Pulmonary artery banding is safe and effective in infants and young children with end-stage DCM with preserved right ventricular function. Combined with conventional heart failure medication therapy, it may provide short-term benefits post-operatively, decrease the cardiogenic shock, and act as a bridge to recovery and potentially reduce the need for heart transplantation. Long-term effects remain further observation, and larger randomized controlled trials would be more persuasive in validating its efficacy.

Efficacy and safety of hepatic arterial infusion chemotherapy combined with donafenib in the treatment of unresectable hepatocellular carcinoma.

This study aimed at ascertaining the efficacy and safety of hepatic arterial infusion chemotherapy (HAIC) combined with donafenib versus HAIC alone in the treatment of unresectable hepatocellular carcinoma (HCC). Seventy HCC patients were enrolled for our study, and they were randomized by simple randomization using computer-generated random numbers into two groups: control group and observation group. Regular follow-up reviews were conducted to assess the efficacy of treatments. The levels of apoptotic factors, the levels of hepatic fibrosis indices, the levels of serum tumor vascular factors and tumor markers, and the occurrence of adverse reactions in the two groups were recorded and compared. Disease control rate, objective response rate, and progression-free survival (PFS) of patients in the observation group were higher in contrast to the control group. After 12 weeks of treatment, lower mRNA expression of c-mesenchymal-epithelial transition factor, telomerase, and Fas Ligand and higher mRNA expression of Fas and Caspase-3 were observed in HCC tissues of the observation group versus the control group (p<0.05); lower detection values of serum laminin, hyaluronic acid, collage type IV, vascular endothelial growth factor receptor 2, and alpha-fetal protein (AFP) were noted in HCC patients of the observation group in comparison to the control group (p<0.05); there was no difference in the incidence of adverse reactions between the two groups. Donafenib combined with HAIC in the treatment of unresectable HCC patients can notably reduce serum AFP levels, improve hepatic fibrosis, enhance short-term efficacy, prolong PFS, and have a favorable safety profile.