Real-world Single-center Experience Research Articles

Single Center Real World Experience of Talquetamab in Patients with Relapsed and Refractory Multiple Myeloma

Single Center Real World Experience of Talquetamab in Patients with Relapsed and Refractory Multiple Myeloma

Bridging Radiotherapy to Axicabtagene Ciloleucel (axi-cel) Is Associated with Favourable Outcomes for Large B-Cell Lymphoma (LBCL) in Third-Line and Beyond: An Australian Single-Centre Real-World Experience

Bridging Radiotherapy to Axicabtagene Ciloleucel (axi-cel) Is Associated with Favourable Outcomes for Large B-Cell Lymphoma (LBCL) in Third-Line and Beyond: An Australian Single-Centre Real-World Experience

Eculizumab in refractory myasthenia gravis: a real-world single-center experience.

Immunosuppressive treatment is effective in most Myasthenia gravis patients, but 10-15% of patients areconsidered refractory due to inadequate response or intolerance to therapy. Eculizumab, a humanized monoclonalantibody directed against C5 complement protein, was approved in Italy to treat Ab-AchR generalized refractoryMG (rMG) in October 2022. We aim to describe a real-world Italian experience in a population of refractory myasthenia gravis patients with oneyear follow up. A retrospective data analysis was conducted on patients with refractory generalized MG treated with eculizumabbetween November 2022 and May 2024. Clinical assessment through specific scales (MG ADL - QMG - MGFA -PIS), rescue, and background therapy was recorded after one, three, six, and twelve months. 21 rMG patients were treated with eculizumab with a medium follow up of 10.4 months and 14 patients had at leastone year follow up. A clinically meaningful reduction in total MG-ADL and QMG scores was achieved in the firstmonth. It was maintained throughout the first, third, sixth, and twelfth month along with concomitant reduction ofimmunosuppressive treatments. A drastic reduction of myasthenic exacerbations and crisis was observed duringfollow up and intravenous immunoglobulin treatment was discontinued in all patients except one. The total dailydose of prednisone was significantly reduced. This single-center real-world study confirmes safety and effectiveness of eculizumab. Eculizumab improved rapidlyall clinical outcome measures, leading to discontinuation of intravenous immunoglobulin treatment and remarkable immunosuppressant-sparing benefits.

Treatment of steroid-refractory acute/chronic graft versus host disease: A single-center real-world experience of ruxolitinib in combination with extracorporeal photopheresis in a high-risk population

Steroid-refractory acute and chronic graft-versus-host disease (SR-a/cGvHD) represents a potential life-threatening complication following allogeneic stem-cell transplantation (allo-SCT). The JAK1/2-inhibitor ruxolitinib and the extracorporeal photopheresis (ECP) have been shown to significantly improve the overall response rate (ORR) in this setting. However, about 30–40 % of high-risk patients do not respond to monotherapy and/or experience side effects. Considering the potential synergic mechanism of action of ruxolitinib and ECP and the good safety profile, we decided to investigate the role of a treatment strategy of ruxolitinib in combination with ECP in frail patients with high-risk SR-a/cGvHD. We conducted a retrospective single-center study comprising 47 patients who underwent allo-SCT from November 2018 to October 2023 and received treatment for SR-aGvHD (n=20) or SR-cGvHD (n=27) with ruxolitinib and ECP. In the SR-aGvHD group, 95 % of patients had a lower GI-tract involvement, with 80 % presenting with a grade III-IV SR-aGvHD. The ORR at day +28 was 65 %, with a 30 % CR rate. The 1-year overall survival (OS) for responders (PR and CR) was 33 % (95 % CI, 10 %-59 %). In the SR-cGvHD group, 55.6 % and 44.4 % had moderate and severe SR-cGvHD, respectively. The majority of patients (66.7 %) had a GI-involvement. The ORR at week 24 was 88 %, including 12 % CR and 76 % PR. The 1-year OS for responders was 76 % (95 % CI, 47 %-90 %). Our retrospective analysis shows that the treatment of ruxolitinib in combination with ECP has potential efficacy in patients with SR-a/cGvHD with a high-risk for transplantation-associated mortality.

Percutaneous decannulation of extracorporeal membrane oxygenation using MANTA device: A real-world single-center experience.

The MANTA vascular closure device (VCD) represents a novel approach to achieving hemostasis after large-bore femoral access procedures. Numerous clinical studies have evaluated the efficacy of the MANTA device across a range of patient populations undergoing different procedures. However, there is still a paucity of data available concerning the use of MANTA devices in aiding the decannulation of venoarterial extracorporeal membrane oxygenation (VA-ECMO). To present our single-center experience of utilizing the MANTA VCD in patients undergoing this procedure. This single-center study included all patients undergoing percutaneous decannulation of femoral VA-ECMO using the MANTA plug-based VCD between January 2021 and October 2023 at University Hospitals Cleveland Medical Center. Inclusion criteria were adult patients who required prolonged (> 24 hours) hemodynamic support with VA-ECMO. Outcomes included all-cause mortality, hemostasis, bleeding, limb ischemia, and site infection. This is a retrospective cohort study of 19 patients with a mean age of 56.8 years. Twelve of them were males with a mean body mass index of 29. The most common extracorporeal membrane oxygenation indication was acute coronary syndrome complicated by cardiogenic shock at 36.8%. The mean length of intensive care unit stay for these patients was 18.8 ± 8.42 days. Seventeen out of 19 patients survived to discharge. The MANTA device was successfully deployed in 19 patients, with 10 procedures conducted at the bedside and 9 in an operating room setting. Complete hemostasis was achieved within 5 minutes of MANTA deployment in 17 out of 19 patients. In 2 patients manual compression after Manta deployment was required to achieve adequate hemostasis. Additionally, acute lower extremity ischemia was noted in two patients, necessitating endovascular interventions. No infections were reported at the site of MANTA deployment. Overall, based on our experience and that of other centers, the MANTA VCD has proven to be a simple, safe, and effective percutaneous technique for facilitating in the OR, but most of all it opens the opportunity for bedside VA-ECMO decannulation. Post-decannulation ischemic complications are higher in this series of sick patients when compared with elective procedures like transcatheter aortic valve replacement and endovascular aneurysm repair. Additionally, operators should be mindful of the incidence of ischemic complications. Distal Doppler pulse signals should always be checked, to indicate bailout options when this occurs.

Silk vista baby flow diversion beyond the circle of Willis: A single-center experience with long-term outcomes.

There is a lack of evidence of flow diversion (FD) safety for aneurysms treatment beyond the circle of Willis. Therefore, we provide a single-center real-world experience with the Silk Vista Baby (SVB). A single-center database was retrospectively reviewed for aneurysms treated with SVB flow diverters. Demographic information, clinical presentation, radiographic characteristics, procedural complications, and outcomes were assessed. About 57 patients (66.7% female, mean age 54.3 ± 13.2) encompassing 57 aneurysms were included. Overall, 40.4% were ruptured: 68.4% saccular, 17.5% blister, 8.7% fusiform, and 5.3% dissecting. The majority were in the anterior circulation (68.4%), and in 48.2% of cases, the distal vessel diameter was inferior to 2 mm. The symptomatic ischemic rate was 5.2%, with one case due to in-stent thrombosis (1.8%). There were no hemorrhagic complications. Complication rates did not differ between ruptured and unruptured lesions (p = 0.356). There were no cases of delayed aneurysm rupture, and overall mortality was 1.8%. The median follow-up time was 18 ± 12 months. In-stent stenosis rate was 10.5% (6/57), all of which were asymptomatic. At the last follow-up, 70.2% of cases had an adequate occlusion (OKM C and D), and 96.5% had an mRS of 0-2. In our series, SVB was shown to be a safe device in the treatment of not only distal anterior circulation aneurysms but also in the management of complex posterior fossa and ruptured blister aneurysms. Multicenter studies are needed to confirm and generalize these results.

Low to intermediate grade lung neuroendocrine tumours. A single centre real world experience

IntroductionLung neuroendocrine tumours (LNETs) are a rare heterogenous group of tumours whose incidence has been increasing. We investigated the diagnosis, treatment, and survival patterns of patients with low to intermediate grade LNETs. MethodsA retrospective chart review of patients with low to intermediate grade LNETs, treated at a Canadian tertiary-level cancer centre was performed. ResultsWe identified 59 patients. Most were G1or G2 and well or moderately differentiated. Forty-seven patients presented with local or locally advanced disease, of which 57.4 % received curative intent surgery. The rest were treated with definitive radiation, radical chemoradiation with platinum and etoposide, palliative chemotherapy with doxorubicin, or supportive care. The five-year overall survival (OS) for those treated surgically was 83 % versus 44 % in the non-surgical group. Metastatic disease was seen in 24/59 patients, with a five-year OS in patients with stage IV disease of 39 %. Of those with advanced or unresectable disease (n = 32), 21 received palliative systemic treatment with up to three lines of therapy. First-line treatment was most commonly chemotherapy with platinum/etoposide combination or somatostatin analogue therapy. Second-line treatment involved chemotherapy or targeted everolimus. PRRT was used once as a first-line and once as second-line therapy. Third-line included lanreotide or chemotherapy with capecitabine/temozolomide combination. ConclusionOverall, patients with surgically resectable disease had a good five-year OS. However, inoperable or more advanced disease was associated with a poorer OS. Despite many treatment options, the sequence of treatments is poorly established. This highlights the need for further development and dissemination of evidence-based guidelines for LNET patients.

49533 308-nm excimer light is effective for palmoplantar pustulosis regardless of the presence or absence of focal infection: Single-center real-world experience of treatment for palmoplantar pustulosis

49533 308-nm excimer light is effective for palmoplantar pustulosis regardless of the presence or absence of focal infection: Single-center real-world experience of treatment for palmoplantar pustulosis

2105: Pregnancy in BRCA-carrier patients after breast cancer therapy: single-centre real-world experience

2105: Pregnancy in BRCA-carrier patients after breast cancer therapy: single-centre real-world experience

Aortic versus axillary artery cannulation for hemiarch replacement: single-centre real-world experience

BackgroundAortic arch disease is a major cause of acute dissections. Surgical replacement is the current curative treatment for aortic arch disease. While traditional aortic cannulation ensures lower body perfusion, axillary cannulation offers optimum cerebral perfusion.AimTo evaluate the outcomes of aortic and axillary cannulation methods in hemiarch replacements, focusing on postoperative perfusion and survival.Materials and methodsA retrospective analysis was conducted on 91 patients who underwent hemiarch replacement surgery between February 2007 and October 2016. Patients were divided into two groups based on the cannulation method: aortic cannulation (54 patients) and axillary cannulation (37 patients). Data regarding preoperative, intraoperative, and postoperative parameters were analyzed, including demographics, surgical outcomes, and complications.ResultsDemographic analysis showed comparable characteristics between the two groups, with notable differences in aortic disease severity and classification. Patients in the axillary group had a larger ascending aorta diameter (57.7 ± 10.8 mm vs. 51.8 ± 5.7 mm, p = 0.002) and a higher prevalence of acute dissections (27.0% (n = 10) vs. 3.7% (n = 2), p = 0.001). Cerebral protection methods varied significantly between the two groups (p < 0.001). Antegrade cerebral perfusion was used in 37.8% (n = 14) of the axillary group compared to 3.7% (n = 2) of the central group. The central cannulation group had a higher proportion of patients with temperatures under 20 °C (98.1% (n = 53) vs. 21.6% (n = 8), p < 0.001), whereas the axillary group maintained higher temperatures (24 –28 °C) in 68.6% (n = 23) of cases. AV repair/replacement was more frequent in the aortic cannulation group (48.2% (n = 26) vs. 18.9% (n = 7), p = 0.013). No significant disparities were observed in operative mortality or intraoperative complications. Statistical analysis showed no significant differences between the two groups in the in-hospital outcomes, but renal complications were more prevalent in the axillary cannulation group with 21.6% (n = 8) experiencing acute kidney injury compared to 9.3% (n = 5) in the central group (p = 0.098). The overall survival rate was slightly higher in the aortic cannulation group at various follow-up periods, yet no statistically significant difference was found between the two groups.ConclusionWe found no significant differences in safety and efficacy between axillary cannulation and aortic cannulation in hemiarch replacement procedures.

Incidence of Cytogenetic Abnormalities in Myelodysplastic Syndrome in the African American Population: A Decade-Long Single-Center Real-World Experience

Incidence of Cytogenetic Abnormalities in Myelodysplastic Syndrome in the African American Population: A Decade-Long Single-Center Real-World Experience

Clinical utility of plasma percent donor-derived cell-free DNA for lung allograft surveillance: A real-world single-center experience

BackgroundPlasma percent donor-derived cell-free DNA (%dd-cfDNA) has been investigated as a biomarker of allograft injury after lung transplantation (LT). We sought to determine the clinical utility of plasma percent donor-derived cell-free DNA (%dd-cfDNA) as a screening tool for acute cellular rejection (ACR) and respiratory infections (RIs) among LT recipients (LTRs). MethodsWe retrospectively analyzed %dd-cfDNA results from 95 plasma samples collected from 81 bilateral LTRs at our center with a paired transbronchial biopsy within 24 hours after sample collection. We calculated sensitivity, specificity, negative predictive value (NPV), and positive predictive value (PPV) of %dd-cfDNA to detect ACR and RIs and used a generalized estimating equation model to compare %dd-cfDNA between groups. ResultsA dd-cfDNA threshold of 0.5% had low sensitivity to detect ACR among LTRs >45 days after LT (41.67%), as did a 70% increase in %dd-cfDNA (50.00%). The NPV was high (88.89% and 87.50%, respectively) but driven by the low prevalence of ACR (12/95 [12.6%]). The area under the receiver operating characteristic curve (AUC) was 0.499 (95%CI [0.326-0.672]) and 0.360 (95%CI [0.132-0.588]) for the detection of ACR and ACR grade ≥A2, respectively. The adjusted mean %dd-cfDNA trended higher in LTRs with a definite or possible RI (1.218, 95%CI [0.671-2.212]) than in LTRs without microbial isolation (0.731, 95%CI [0.525-1.017], p=0.059), but was not significantly different from those with microbial colonization (0.873, 95%CI [0.538-1.415], p=0.390). There was no significant difference in the adjusted mean %dd-cfDNA between patients with microbial colonization and those without microbial isolation (p=0.464). %dd-cfDNA testing had an AUC of 0.573 (95%CI [0.431-0.716]) for the detection of lung allograft dysfunction due to ACR and/or infection. Conclusions%dd-cfDNA may have limited utility as a screening tool to detect ACR among LTRs.

Analysis of apheresis outcomes in a cohort of Chilean patients treated with autologous stem cell transplantation: A single center real-world experience

Adequate stem cell harvesting is required for autologous hematopoietic transplantation. In deficient mobilizer patients, the collection of stem cells can be challenging because of the impossibility of achieving satisfactory CD34 cell counts with GCSF + - chemotherapy. Plerixafor is a potent and expensive drug that promotes the release of stem cells from the medullary niche to the peripheral blood and allows satisfactory harvests. We performed a retrospective analysis of 370 patients with myeloma and lymphoma harvested at our institution. 99 % of patients achieved satisfactory apheresis using Plerixafor in 45 %. Satisfactory harvests were obtained in patients mobilized with GCSF or plerixafor. In patients who used plerixafor, it was necessary to perform fewer apheresis procedures (P = 0.05). In multivariate analysis, the only factor that predicted the need for plerixafor was the presence of less than 30,000 CD34 / ul on the day of apheresis (OR 0.3. p < 0.001). Since we adopted the plerixafor protocol guided by CD34 counts, the number of patients with harvest failure has decreased. In conclusion, the rational and standardized use of plerixafor favors satisfactory harvest in patients who require autologous transplantation in South-American patients.

The fate of nonscalp hair in alopecia areata universalis under baricitinib treatment: A real-world single-center experience

The fate of nonscalp hair in alopecia areata universalis under baricitinib treatment: A real-world single-center experience

Real-world single center experience with the wearable cardioverter defibrillator: sex differences in risk stratification for sudden cardiac death

Abstract Background Wearable cardioverter defibrillator (WCD) demonstrated an important role for prevention of sudden cardiac death in patients with advanced heart failure and reduced left ventricular ejection fraction. Although in randomized trial there are no gender difference in WCD use and defibrillator (ICD) implantation rate, real world data in female patients is lacking. Purposes: We aimed to describe the sex differences in WCD use, the rate of cardiac events detected with WCD and the rate of ICD implantation after WCD use in women in a real-world single center experience. nd reduced left ventricular ejection fraction or prior ventricular tachyarrhythmia. Methods and results According with the current guidelines indications, between August 2017 and June 2023 ninety-six consecutive patients (mean age 66.0±11.1 years) received a WCD at Piacenza Hospitals and were retrospectively included in this analysis. There were no differences in clinical characteristics between female (18 patients, 18,8%) and male: in particular baseline left ventricular ejection fraction (LV EF) were similar between the two groups (29.5±5.2 vs 27.9±7.9, p=0.23) and no differences were found in the rate of ischaemic cardiopathy (66,6% vs 69,2% p=0.51). After 3-months follow up with WCD use, a similar rate of ventricular arrhythmias were found in the two groups (5.5% vs 3.8%, p=0.48) and no ventricular fibrillation was detected. The WCD usage period and median daily wear time were not different between female and male (55.2±50.6 vs 56.2±34.8 days p=0.53, 21.0±6.4 vs 23.1±1.5 hours p=0.48, respectively). However women presented a better LV EF improvement (12.2±9.7 vs 7.2±6.8, p=0.01), with a significant lower ICD implantation rate at the end of WCD use (55.5% vs 43.8%; p&amp;lt; 0.01) Conclusions In this real word analysis women presented a nice compliance using WCD. In spite of a similar burden of ventricular arrhythmic events, women presented a better LV EF improvement with a significant lower ICD implantation rate.

The clinical effectiveness of Fidaxomicin compared to Vancomycin in the treatment of Clostridioides difficile infection, a single center real-world experience.

The use of fidaxomicin is recommended as first line therapy for all patients with Clostridioides difficile infection (CDI). However, real-world studies have shown conflicting evidence of superiority. We conducted a retrospective single center study of patients diagnosed with CDI between 2011-2021. A primary composite outcome of clinical failure, 30-day relapse or CDI-related death was used. A multivariable cause specific Cox proportional hazards model was used to evaluate fidaxomicin compared to vancomycin in preventing the composite outcome. A separate model was fit on a subset of patients with C. difficile ribotypes adjusting for ribotype. There were 598 patients included, of whom 84 received fidaxomicin. The primary outcome occurred in 8 (9.5%) in the fidaxomicin group compared to 111 (21.6%) in the vancomycin group. The adjusted multivariable model showed fidaxomicin was associated with 63% reduction in the risk of the composite outcome compared to vancomycin (HR = 0.37, 95% CI 0.17-0.80). In the 337 patients with ribotype data after adjusting for ribotype 027, the results showing superiority of fidaxomicin were maintained (HR = 0.19, 95% CI 0.05-0.77). In the treatment of CDI, we showed that real-world use of fidaxomicin is associated with lower risk of a composite endpoint of treatment failure.

Predicting of overall survival in non-small cell lung cancer patients receiving concurrent radiochemotherapy and adjuvant durvalumab – a Polish real-world single-center experience

Predicting of overall survival in non-small cell lung cancer patients receiving concurrent radiochemotherapy and adjuvant durvalumab – a Polish real-world single-center experience

REAL–WORLD SINGLE CENTER EXPERIENCE WITH THE WEARABLE CARDIOVERTER DEFIBRILLATOR: SEX DIFFERENCES IN RISK STRATIFICATION FOR SUDDEN CARDIAC DEATH

Abstract Background Wearable cardioverter defibrillator (WCD) demonstrated an important role for prevention of sudden cardiac death in patients with advanced heart failure and reduced left ventricular ejection fraction. Although in randomized trial there are no gender difference in WCD use and defibrillator (ICD) implantation rate, real world data in female patients is lacking. Purposes: We aimed to describe the sex differences in WCD use, the rate of cardiac events detected with WCD and the rate of ICD implantation after WCD use in women in a real–world single center experience with reduced left ventricular ejection fraction or prior ventricular tachyarrhythmia. Methods and Results According with the current guidelines indications, between August 2017 and June 2023 ninety–six consecutive patients (mean age 66.0±11.1 years) received a WCD at Piacenza Hospitals and were retrospectively included in this analysis. There were no differences in clinical characteristics between female (18 patients, 18,8%) and male: in particular baseline left ventricular ejection fraction (LV EF) were similar between the two groups (29.5±5.2 vs 27.9±7.9, p=0.23) and no differences were found in the rate of ischaemic cardiopathy (66,6% vs 69,2% p=0.51). After 3–months follow up with WCD use, a similar rate of ventricular arrhythmias were found in the two groups (5.5% vs 3.8%, p=0.48) and no ventricular fibrillation was detected. The WCD usage period and median daily wear time were not different between female and male (55.2±50.6 vs 56.2±34.8 days p=0.53, 21.0±6.4 vs 23.1±1.5 hours p=0.48, respectively). However women presented a better LV EF improvement (12.2±9.7 vs 7.2±6.8, p=0.01), with a significant lower ICD implantation rate at the end of WCD use (55.5% vs 43.8%; p&amp;lt; 0.01). Conclusions In this real word analysis women presented a nice compliance using WCD. In spite of a similar burden of ventricular arrhythmic events, women presented a better LV EF improvement with a significant lower ICD implantation rate.

Dabrafenib-trametinib in BRAF V600-mutated non-small-cell lung cancer: a single center real world experience.

Aim: We retrospectively evaluated the effect of dabrafenib/trametinib combination in patients with BRAF-mutated non-small-cell lung cancer (NSCLC) treated in a single center from 2017 to 2022.Patients: The response and safety data of 42 patients (27 treated in first-line and 15 as second/subsequent lines) were analyzed.Results: The objective response was 73.8%, with no differences between patients undergoing first- or second-line. A longer, statistically significant median progression-free survival (PFS) was observed in patients receiving the combination in first-line vs those in the second/subsequent lines (19.9 months [95% CI: 19.7-20]vs 13.1 months [95% CI: 8.6-17.6], respectively;p=0.012). The median overall survival (OS) was 29.9 months (95% CI: 14.1-45.7) for patients treated with the combination in first-line and 22.4months (95% CI: 14.6-30.2) for those treated in subsequent lines. The combination was well toleratedConclusion: We confirm the efficacy of dabrafenib/trametinib in BRAF-V600-mutated NSCLC.

Single suture-mediated closure system after transfemoral transcatheter aortic valve implantation: A single-center real-world experience.

Despite the use of two crossed Perclose ProGlide™ (Abbott Vascular Devices) is the most widespread technique to close the main arterial access in transfemoral transcatheter aortic valve implantation (TF-TAVI), the safest and most effective strategy still remains much debated. The aim of the present study was to evaluate the performance of a single Perclose ProGlide suture-mediated closure device to obtain femoral hemostasis after sheathless implantation of self-expanding transcatheter heart valves through their 14 F-equivalent fix delivery systems. This prospective observational study included 439 patients undergoing TF-TAVI at the "Montevergine" Clinic of Mercogliano, Italy. All patients underwent hemostasis of the large-bore access using a single Perclose ProGlide with preclose technique, after sheathless implantation of self-expanding transcatheter heart valves through 14 F-equivalent fix delivery systems. A multidetector computed tomography analysis of size, tortuosity, atherosclerotic, and calcification burdens of the ilio-femoral access route was made by a dedicated corelab. Vascular complications (VCs), percutaneous closure device (PCD) failure, and bleedings were adjudicated by a clinical events committee. A total of 81 different VCs were observed in 60 patients (13.7%); among these, 41 (5% of patients) were categorized as major. PCD failure occurred in 14 patients (3.2%). At the logistic regression analysis, no predictors of PCD failure have been identified. This registry suggests that the use of a single suture-mediated closure device could be considered a safe and efficient technique to achieve access site hemostasis in patients undergoing TF-TAVI through 14 F-equivalent fix delivery systems.