Results Of Clinical Studies Research Articles

A real‑world study of clinical characteristics, treatment sequence and outcomes of patients with non-small cell lung cancer and EGFR exon 20 insertion mutations.

EGFR exon 20 insertion (EGFRex20ins) mutations are found in up to 4% of all patients with non-small cell lung cancer (NSCLC). These patients are often insensitive to EGFR-tyrosine kinase inhibitors (TKIs) and have worse prognosis than patients with more common EGFR mutations. In this multicenter, retrospective, real-world study, we sought to determine whether the administration of recently approved treatments that specifically target EGFRex20ins mutations could significantly improve outcomes in this patient population. We evaluated the clinical features of 41 patients diagnosed with NSCLC and EGFRex20ins mutations, their evolution, and response to treatments received across 7 hospitals in the Valencian Community, Spain, between 31st December 2012 and 31st December 2022. 32 patients (72%) developed metastatic disease, and 29 (71%) of them received oncological treatment. We found that administering a targeted therapy against EGFRex20ins mutations (amivantamab, mobocertinib and/or sunvozertinib) at some point during the course of treatment, significantly increased the median OS of metastatic patients from 8months (95% CI 0-21.7) to 30months (95% CI 11.1-48.8; Hazard ratio = 0.297, p = 0.02). Our findings contribute to the evolving standard of care for this specific population and highlight the clinical benefits of targeted cancer therapies.

Preoperative Immersive Virtual Reality (iVR) Applied to Percutaneous Nephrolithotomy: A Prospective Randomized Clinical Study of Surgical Planning and Clinical Outcomes.

Percutaneous nephrolithotomy (PCNL) is the procedure of choice for the management of complex or large renal stones. A major challenge for the surgeon, however, is the need to assimilate the nearly 2000 static images from a CT scan into a functional mental image to enable surgical planning. Accordingly, we investigated the potential of immersive virtual reality (iVR) to enhance surgical planning and its impact on the outcomes among patients undergoing PCNL. Between 2019-2023, 175 patients undergoing PCNL were pre-operatively randomized into a CT-only group (N=89) or a CT+iVR group (N=86). CT scans were rendered into iVR models that allowed the surgeon not only to visualize and manipulate each patient's relevant anatomy, but also simulate the percutaneous approach to the proposed calyx. Post-operative CT scans were defined as absolute stone-free, <2mm remnants or 2.1-4mm remnants. Pre-operative visualization of the iVR model resulted in a changed calyx of entry in 30% of cases. The CT+iVR group had a significant improvement in absolute stone-free rate (33.70% vs. 20.22%, p=0.043) and overall <4mm remnant rate (62.79% vs. 48.20%, p=0.044). Clavien-Dindo II+IIIa complications were less in the iVR group (3.48% vs. 12.30%, p=0.03). The results were independent of the surgeon's years of PCNL experience. Pre-operative iVR model visualization benefited surgeons and patients alike. From a surgical standpoint, viewing the iVR model resulted in a safer, more effective percutaneous stone removal procedure.

Treatment Patterns and Clinical Outcomes in Patients with Hodgkin Lymphoma from Saudi Arabia, Türkiye, and South Africa: Subgroup Analysis from the International, Multi-center, Retrospective BHOLISTIC Study.

B-HOLISTIC was a real-world, retrospective study of treatment patterns and clinical outcomes in Hodgkin lymphoma (HL) in regions outside Europe and North America. This subgroup analysis reports findings from Saudi Arabia, Türkiye, and South Africa. Patients aged ≥18 years and diagnosed with stage IIB-IV classical HL receiving frontline chemotherapy (frontline cHL) and/or relapsed/refractory HL (RRHL) from January 2010-December 2013 were assessed. The primary endpoint was progression-free survival (PFS) in patients with RRHL. Overall, 694 patients (RRHL: n=178; frontline cHL: n=653) were enrolled. Among patients with RRHL, >80% received first salvage chemotherapy. The most common first salvage regimens were etoposide, methylprednisolone, cytarabine, cisplatin (ESHAP) in Saudi Arabia (78.3%) and dexamethasone, cytarabine, cisplatin (DHAP) in Türkiye (36.1%) and South Africa (40%). Median PFS (95% confidence interval [CI]) in the RRHL group was 5.1 (3.0-15.9), 19.7 (7.5-not reached), and 5.2 (1.1- 10.1) months in Saudi Arabia, Türkiye, and South Africa, respectively. The 5-year PFS and overall survival (95% CI) rates in patients with RRHL were 33.2% (21.6-45.2) and 78.2% (65.9-86.5) in Saudi Arabia, 42.5% (29.5-54.9) and 79.4% (67.2-87.5) in Türkiye, and 13.1% (4.2-27.0) and 53% (35.5-67.8) in South Africa, respectively. This study showed that the clinical outcomes in Türkiye and Saudi Arabia were generally comparable with Western countries during the study period, although Saudi Arabia had lower PFS rates. Conversely, the clinical outcomes in South Africa were suboptimal, emphasizing the need for novel therapies and improved progression to stem cell transplantation. Additionally, these data may serve as a control group for future studies in these countries and inform clinical decision-making.

Long-term validation of AtherosSlerosis CardioVascular Disease (ASCVD) risk score and the role of aspirin for primary Cardiovascular prevention in Korean patients with over ASCVD 20% risk score

Abstract Background Atherosclerotic cardiovascular disease (ASCVD) is a leading cause of death worldwide. The ASCVD risk score can be a great tool for preventing ASCVD in the future. There is a mediation recommendation depending on calculated risk called United States Prevention Services Task Force (USPSTF) aspirin (ASA) recommendation. However, the validation studies of long-term clinical outcomes in Korean population using the ASCVD risk score and of the effect of ASA in Korean patients using USPSTF recommendation are limited. Methods A total of 7,249 patients with any chest pain who underwent coronary spasm provocation tests between January 2004 and December 2021 were enrolled. Patients were divided into four groups, based on the ASCVD risk score: &amp;lt; 5% group (Low Risk, n = 2,534), 5 - 10% group (Borderline Risk, n=1,681), 10 - 20% group (Intermediate Risk, n = 1,896) and ≥ 20% group (High Risk, n = 1,138). The primary endpoint was Major Adverse Cerebral and Cardiovascular Event (MACCE1), including stroke and MACE. The secondary endpoint was MACCE 2, defined as composite of MACE and recurrent angina requiring follow-up coronary angiography (CAG). The third endpoint was the effect of aspirin in patients who have over 20% ASCVD risk. Results Long term (10-year) ASCVD risk score was well-validated from the "Low Risk" group to the "High Risk" group by 1.87%, 7.44%, 14.09%, and 30.61%, respectively (P &amp;lt; 0.001). In baseline characteristics, the calculation factors of ASCVD risk score is also well-stratified, showing the more adverse clinical profiles in the "High Risk" group than the "Low Risk" group. In 10-year clinical outcomes, although MACCE1 and MACCE2 showed an increasing trend numerically, stroke was observed to significantly increase according to ASCVD risk score. There was no effect of ASA prescribed in accordance with USPSTF recommendation. The group with the highest rate of 20% or more failed to show the aspirin prevent effect on the occurrence of CV events, but also increased the incidence (MACCE1; HR: 1.647, P-Value: 0.283, 95% CI: 0.662 - 4.100, MACCE2; HR: 1.819, P-Value: 0.025, 95% CI: 1.078 - 3.068). Conclusion Long term ASCVD risk score was associated with higher risk of stroke. However, MACCE1 and MACCE2 only showed an increasing trend numerically. Furthermore, the effect of ASA prescribed based on the USPSTF ASA use recommendation statement were not observed in Korean population. Consequently, additional research is needed to ensure the utility of ASCVD risk score and the effect of ASA in high-risk patients to prevent future CV events in Korean population.

Long-term clinical outcome of a novel bilateral capsulotomy with focused ultrasound in refractory obsessive-compulsive disorder treatment.

Magnetic resonance-guided focused ultrasound (MRgFUS) capsulotomy is a promising treatment for refractory obsessive-compulsive disorder (OCD); however, long-term clinical outcome studies are lacking. We aimed to investigate the long-term efficacy and safety of MRgFUS capsulotomy in patients with refractory OCD. Ten of the eleven patients who underwent MRgFUS capsulotomy for treatment-resistant OCD between 2013 and 2014 were included in this study. Clinical outcomes were assessed after 10 years of follow-up post-MRgFUS capsulotomy using tools such as neuropsychological test, the Frontal Systems Behavior Scale (FrSBe), and a locally developed MRgFUS-patient-centered outcomes questionnaire. After 10 years of follow-up, there was a mean improvement of 52.3% in the Yale-Brown Obsessive-Compulsive Scale (Y-BOCS) score. Seven out of the ten participants responded fully (Y-BOCS reduction ≥35% + CGI-I 1 or 2) to the procedure, two of whom achieved remission (Y-BOCS score ≤12 and CGI-S 1 or 2). Obsessive-compulsive symptoms and overall functioning significantly reduced and improved, respectively (Y-BOCS = 20.7 after 2 years vs. 16.4 after 10 years, p = 0.012; Global Assessment of Functioning = 57.4 after 2 years vs. 69.0 after 10 years, p = 0.011). The patients experienced significantly improved frontal lobe-related functions (FrSBe Sum before 91.0 ± 17.6 vs. after 78.6 ± 17.7, p < 0.05). No adverse effects, including cases of suicide and neurological deficits, were reported. The majority of the respondents were generally satisfied with MRgFUS capsulotomy. MRgFUS capsulotomy is an effective and safe treatment option for the treatment of severe refractory OCD with sustained efficacy even after 10 years.

Study of clinical and functional outcome of malleoli fracture

Background: Ankle fractures are a common orthopedic injury, accounting for 9% of all fractures, with an increasing incidence among both young and elderly populations. These fractures often result from road traffic accidents, falls, and sports injuries. Malleolar fractures, involving the bony prominences of the ankle, are particularly prevalent and necessitate accurate reduction and stable internal fixation to prevent complications such as post-traumatic arthritis and restricted motion. This study aims to assess the functional outcomes and results of surgical treatment of malleolar fractures. Methods: The study utilized Lauge-Hansen’s classification to analyze the mechanism of injury in 32 patients with ankle fractures. Surgical techniques included the fixation of syndesmotic injuries with anatomical fibular plates, (AFP) posterior malleolus fractures with percutaneous screws or Ellis plates, and medial malleolus fractures with tension band wiring (TBW). The outcomes were measured in terms of union time, range of motion, and stability. Results: The most common injury mechanism was supination external rotation (SER) in 14 patients, followed by pronation external rotation (PER) in 7 patients, pronation abduction (PAB) in 6 patients, and supination adduction (SAD) in 5 patients. Syndesmotic injuries were found in 7 patients, all treated surgically except one. Posterior malleolus fractures were present in 3 patients and treated with screws or plates. Fixation of medial malleolus fractures with TBW showed better union rates and improved range of motion compared to screw fixation. Lateral malleolar fractures treated with AFP provided additional stability. The average union time was 6 to 8 weeks. Conclusions: Anatomical reduction and stable internal fixation are crucial for the successful treatment of malleolar fractures. TBW for medial malleolus fractures and AFP for lateral malleolar fractures provide favorable outcomes. However, a larger sample size and longer follow-up duration are required to further evaluate the outcomes of ankle injuries, particularly in older patients with osteoporosis and comorbidities.

Triple combination of vemurafenib, cobimetinib, and atezolizumab in real clinical practice in the Russian Federation: results of the A1 cohort of the ISABELLA study.

Among several treatment options for BRAF-mutant metastatic melanoma, a combination of BRAF inhibitor, MEK inhibitor, and anti-PDL1 antibody seems to be a new emergent approach recently registered in the Russian Federation. It is still not clear which patient population benefits more from this simultaneous use of three drugs instead of its sequencing. This study aimed to evaluate patients' characteristics treated in real practice in 14 Russian regions by triple combination and to analyze their outcomes depending on biomarkers (PD-L1 expression). This was a part (cohort A1) of a prospective non-interventional study of clinical outcomes and biomarkers in patients with skin melanoma. Patients were included in cohort A1 if combination treatment with vemurafenib (vem) + cobimetinib (cobi) + atezolizumab (atezo) was initiated no earlier than 12 weeks (84 days) prior to written informed consent to participate in this study. The index event was the initiation of therapy with all three drugs vem + cobi + atezo (i.e., triple combination). The primary efficacy endpoint of the study was the 24-month overall survival (OS), defined as the time from the index date to the date of death from any cause. If the patient did not experience an event, the OS will be censored at the date of the last contact. Objective response rate (ORR), duration of response (DoR), and progression-free survival (PFS) in the Intention to treat (ITT) population, in biomarker positive population, and in population with brain metastases were also evaluated. Quality of life questionnaires were pre-planned by protocol if it was a part of routine practice. Adverse events were also collected. Between March 2021 and May 2023, 59 patients were enrolled in 19 centers from 14 regions of Russia. Thirty-one of 59 (52.4%) patients had central nervous system metastases, and 18 of 31 (58.4%) were symptomatic. Forty of 59 patients (68%) received the triple combination as the first-line treatment. The median follow-up period was 16.83 [95% confidence interval (CI) 13.8-19.8] months. The mean duration of therapy with this regimen was 9.95 months (95% CI 7.48-13.8). ORR was 55.1%; progression as the best outcome was seen in 16.3%. The median DoR was 12.95 months (95% CI 11.0-14.8 months), with a median of 20.3 months (95% CI 9.1-31.5 months) when triple therapy was administered in the first-line treatment. In patients with brain metastases (N = 31), ORR was 45.1%; the median DoR was 12.95 (95% CI 11.0-14.8 months). The median PFS in the entire population was 13.6 months (95% CI 8.6-18.6); the 24-month PFS was 22%. The estimated median OS in the entire population was 15.8 months (95% CI NA); 24-month OS was 45% (95% CI 0.32-0.64). In multivariate Cox regression model, biomarkers of interest [lactate dehydrogenase, Programmed cell death ligand-1 (PD-L1)] did not have statistically significant impact on PFS, OS, or DoR probably due to high data missing rate. No unexpected adverse events were reported. Grades 3-4 AEs were seen in 23 of 59 patients (38%) with most common were skin and liver toxicity. Triple combination of atezolizumab, vemurafenib, and cobimetinib had proven its efficacy and tolerability in real settings. No impact of potential predictive biomarkers was seen (NCT05402059).

10-Year Study of Clinical Outcomes and Bowel Function Following Surgical Treatment in Hirschsprung?s Disease

10-Year Study of Clinical Outcomes and Bowel Function Following Surgical Treatment in Hirschsprung?s Disease

EP.11A.18 LUMINATE 101-Maccabi: Real-World Study of Treatment Patterns and Clinical Outcomes in Patients with Mnsclc in an Israeli HMO

EP.11A.18 LUMINATE 101-Maccabi: Real-World Study of Treatment Patterns and Clinical Outcomes in Patients with Mnsclc in an Israeli HMO

Approaches to the Diagnosis and Management of Paediatric Ovarian Tumours and Oncological Outcomes in a Single-centre Study. Evidence in Support Of IPSO Reccomendations

BackgroundApproaches to paediatric ovarian tumours vary. Cross-sectional imaging is reliable but not always performed. Laparoscopic tumourectomy, although popular, is not recommended by International Paediatric Surgical Oncology Society (IPSO); tumour spillage remains the main concern. We aimed to investigate the reliability of preoperative evaluation and identify perioperative factors associated with adverse outcomes. MethodsSingle-centre retrospective study (2015–2022) of clinical presentation, preoperative investigation, operative approaches and oncological outcomes in all females <18 years treated for ovarian tumours. Logistic regression was used to identify predictors of malignancy and of second disease events. Data are presented as median (IQR) or rates. ResultsSixty girls aged 12.5 (10–15) years with follow-up of 51 (30.5–76.3) months were included. Incidence of malignant and low malignant potential tumours was 19/60 (32%). Tumour size and consistency proved predictive of malignancy. Clinical assessement, tumour markers and cross-sectional imaging combined showed 75% sensitivity, 100% specificity and 7% misclassified malignancy rate. Second disease events (11/60, 18%) were associated with misclassified malignancy (2/11 vs. 1/49, p = 0.04) and positive margins (4/11 vs. 5/49, p = 0.02). Ovarian-sparing surgery did not influence second events (5/11 vs. 28/49, p = 0.48). There was no significant association between second events and laparoscopic tumourectomy (3/11 vs. 20/49, p = 0.40); spillage rate was however more frequent with laparoscopy (5/11 vs. 2/32, p = 0.008). ConclusionMisclassified malignancy, noted in 7% of patients undergoing optimal work-up, adversely impacted outcomes, highlighting the importance of oncological principles in both oophorectomy and ovarian-sparing surgery. Malignancy incidence and misclassification can be underestimated when tumours with malignant behaviour potential, such as immature teratomas and borderline tumours, are grouped as benign. There was no direct association between operative approaches and second events. Laparoscopy, however, failed to maintain oncological principles more frequently than open surgery and hence risks upstaging of paediatric ovarian pathology. Level of evidenceIII.

TCT-535 Watchman Left Atrial Appendage Occlusion Peri-Device Leaks With Elevated Mean Left Atrial Pressure Associated With Clinical Outcomes (WATCH-OUT) Study

TCT-535 Watchman Left Atrial Appendage Occlusion Peri-Device Leaks With Elevated Mean Left Atrial Pressure Associated With Clinical Outcomes (WATCH-OUT) Study

93P The International Clinical Outcome Study for Dysferlinopathy II: validation of motor outcome measures in a new patient cohort

93P The International Clinical Outcome Study for Dysferlinopathy II: validation of motor outcome measures in a new patient cohort

96P The impact of losing the ability to sit to stand on social participation in people with dysferlinopathy: clinical outcome study for dysferlinopathy

96P The impact of losing the ability to sit to stand on social participation in people with dysferlinopathy: clinical outcome study for dysferlinopathy

81P Minimal clinically important differences in dysferlinopathy from the 10-year, multicenter Jain Clinical Outcome Study

81P Minimal clinically important differences in dysferlinopathy from the 10-year, multicenter Jain Clinical Outcome Study

Correlation between clinical outcome and nerve conduction studies before and after surgery in patients with carpal tunnel syndrome

Correlation between clinical outcome and nerve conduction studies before and after surgery in patients with carpal tunnel syndrome

The study of clinical outcome in cases of placenta previa

The study of clinical outcome in cases of placenta previa

52632 Emerging Cat-related Cutaneous Sporotrichosis in Thailand: A Retrospective Cohort Study of Clinical Presentations, Antifungal Susceptibility, and Treatment Outcomes

52632 Emerging Cat-related Cutaneous Sporotrichosis in Thailand: A Retrospective Cohort Study of Clinical Presentations, Antifungal Susceptibility, and Treatment Outcomes

V-112.5: Covid-19 hospitalization among liver transplant recipients: Multicenter study of clinical outcomes in the United States.

V-112.5: Covid-19 hospitalization among liver transplant recipients: Multicenter study of clinical outcomes in the United States.

Nutritional Supplementation in Tuberculosis Treatment: A Mixed Methods Study of Clinical Outcomes and Patient Perceptions in Jamnagar, India.

Tuberculosis (TB) remains a significant global health challenge, with malnutrition being a key risk factor for poor outcomes. This study aimed to evaluate the impact of nutritional supplementation on treatment outcomes in drug-sensitive pulmonary tuberculosis (DS-PTB) patients and explore patient perspectives on nutrition during TB treatment. We conducted a mixed methods study in the Jamnagar district of Gujarat, India. The quantitative component was a retrospective cohort study comparing 645 DS-PTB patients who received nutritional supplements with 645 patients who did not. The primary outcomes were cure rates, mortality, and weight gain. Qualitative data were collected through in-depth interviews of 240 patients to explore their experiences and perceptions regarding nutrition during TB treatment. Patients receiving nutritional supplements had significantly higher cure rates (482/645, n=74.7% vs 328/645, n=50.9%, OR: 2.86, 95% CI: 2.26-3.61, p<0.001) and lower mortality (7/645, n=1.1% vs 37/645, n=5.7%, OR: 0.18, 95% CI: 0.08-0.41, p<0.001) compared to the non-supplemented group. The group that received nutritional supplementation showed greater weight gain over six months (6.5 kg vs 3.1 kg, p<0.001). Qualitative findings revealed that patients who received nutritional supplementation reported improved appetite, increased energy, and faster symptom resolution while control group participants faced financial constraints and reduced appetite as barriers to adequate nutrition. Nutritional supplementation significantly improved treatment outcomes in DS-PTB patients, including higher cure rates, reduced mortality, and enhanced weight gain. Patient perspectives highlighted the multifaceted impact of nutritional support. These findings suggest that integrating nutritional supplementation into standard TB care could substantially improve patient outcomes and experiences.

Catamaran SI Joint Fusion System(R) MAINSAILTM Study: a prospective, single-arm, multi-center, post-market study of six-month clinical outcomes and twelve-month radiographic findings

ABSTRACT Background Minimally invasive surgical techniques for sacroiliac joint (SIJ) fixation have the potential to reduce risk and improve patient outcomes, but evidence remains limited. This interim analysis presents initial findings from an ongoing prospective study evaluating the safety and efficacy of the Catamaran System. Methods The primary endpoint of success at 6 months was defined as a ≥20 mm improvement in SIJ pain (Visual Analog Scale, VAS), no neurologic worsening, absence of device-related serious adverse events (SAEs), and no surgical reintervention. Secondary endpoints included 6 month evaluation of the Oswestry Disability Index (ODI), patient satisfaction, and 12 month radiographic CT fusion, performed by an indpendent radiologist. Results Thirty-three consecutive patients (mean age: 58.9 years; %-females: 76%; Body Mass Index: 30.5) were treated across six U.S. clinical sites. At the primary endpoint of 6 months, 80% of patients met the criteria for success, with no device-related SAEs and no surgical reintervention reported. VASSIJ-Pain significantly decreased from preoperative levels (mean: 80.9 mm) to 6 months postoperatively (31.1 mm; p < 0.001). Mean ODI scores also showed a significant improvement from preoperative values (51.9%) to 6 months postoperatively (29.6%, p < 0.01). Patients reported high satisfaction rates throughout all follow-ups, with 93.3% of patients being satisfied at 6 months. Conclusion In patients diagnosed with chronic SIJ pain, minimally invasive inferior-posterior delivery of the Catamaran implant was safe and effective in relieving pain and reducing disability.