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Background: Optimal surgical margin width for patients with phyllodes tumors (PTs) of the breast remains debated. The aim of this study was to assess the influence of margin width on long-term local recurrence risk., Patients and Methods: This was a single-institution retrospective review of patients with confirmed PT treated from 2008-2015. Margins were defined as positive (ink on tumor), narrow (no tumor at inked margin but < 10mm), or widely free (>/= 10mm). LR rates were estimated by the Kaplan-Meier method., Results: Among 117 female patients, histology included 55 (47%) benign, 29 (25%) borderline, and 33 (28%) malignant PT. Final margins were positive in 16 (14%), narrow in 32 (27%), widely free in 64 (55%), and unknown in 5 (4%) patients. Compared with margins > 10 mm, patients with positive and narrow margins had a higher LR risk [HR 10.57 (95% CI 2.48-45.02) and HR 5.66 (95% CI 1.19-26.99), respectively]. Among benign PTs, the 10-year LR-free rates were 100%, 94%, and 66% for widely negative, narrow, and positive margins, respectively (p = 0.056). For borderline/malignant PT, the 10-year LR-free rates were 93% and 57% for widely negative and narrow margins, respectively (p = 0.02), with no difference in LR between narrow and positive margin groups (p = 1.00)., Conclusions: For benign PTs, a margin of no ink on tumor appears sufficient to optimize local control. In patients with borderline or malignant PTs, achieving a wide surgical margin may remain important as narrower margins were associated with LR rates comparable to those with positive margins., (© 2024. Society of Surgical Oncology.)

BACKGROUND. Treatment options are limited in patients with recurrent or metastatic disease after initial treatment of soft-tissue sarcoma (STS) by surgical resection, radiation, or systemic therapy. Percutaneous cryoablation may provide a complementary minimally invasive option in this setting. OBJECTIVE. The purpose of this article was to assess the safety and efficacy of percutaneous cryoablation performed for local control of treatment-refractory recurrent or metastatic STS. METHODS. This single-institution retrospective study included adult patients who underwent percutaneous cryoablation from January 2016 to April 2023 to achieve local control of recurrent or metastatic STS after earlier treatment (surgery, radiation, or chemotherapy). For each treated lesion, a single interventional radiologist rereviewed intraprocedural images to assess for adequate coverage by the ice ball of the entire lesion and a 5-mm or greater margin in all dimensions. Complications and outcomes were extracted from medical records. The primary end point for procedure efficacy was 1-year local progression-free survival. RESULTS. The study included 141 patients (median age, 66 years; 90 women, 51 men) who underwent 217 cryoablation procedures to treat 250 recurrent or metastatic STS lesions. The most common STS histologic types were leiomyosarcoma (56/141) and liposarcoma (39/141). Lesions had a mean long-axis diameter of 2.0 cm (range, 0.4-11.0 cm). Adequate ice-ball coverage was achieved for 82% (204/250) of lesions. The complication rate was 2% (4/217), including three major complications and one minor complication. Patients' median postablation follow-up was 25 months (range, 3-80 months). Local progression-free survival rate was 86% at 1 year and 80% at 2 years. The chemotherapy-free survival rate was 45% at 1 year and 31% at 2 years. The overall survival (OS) rate was 89% at 1 year and 80% at 2 years. In Kaplan-Meier analysis, leiomyosarcoma, in comparison with liposarcoma, had significantly higher local progression-free survival but no significant difference in OS. In multivariable analysis, factors independently associated with an increased risk for local progression included inadequate ice-ball coverage (HR = 7.34) and a lesion location of peritoneum (HR = 3.63) or retroperitoneum (HR = 3.71) relative to lung. CONCLUSION. Percutaneous cryoablation has a favorable safety and efficacy profile in patients with recurrent or metastatic STS after earlier treatments. CLINICAL IMPACT. Percutaneous cryoablation should be considered for local control of treatment-refractory STS.

Objective: We evaluated survival outcomes by primary tumor site in synovial sarcoma (SS) patients with localized and metastatic disease at diagnosis., Methods: We conducted a retrospective review of 504 SS patients diagnosed from 1974 to 2020. Kaplan-Meier method, log-rank test, and Cox-proportional hazards regression were used., Results: Among 504 patients, 401 (79.6%) presented with localized disease, and 103 (20.4%) with metastases. For patients with localized disease, (1) 5-year OS by tumor site was as follows: 80% (95% CI, 67%-89%) for head/neck, 30% (95% CI, 18%-42%) for intrathoracic, 51% (95% CI, 35%-65%) for abdomen/pelvis, 71% (95% CI, 62%-79%) for proximal-extremity, and 83% (71%, 91%) for distal-extremity. (2) On multivariable analysis, tumor site (compared with proximal-extremity: intrathoracic tumors [HR: 1.95; 95% CI, 1.22-3.16]; hand/foot [HR: 0.52; 95% CI, 0.28-0.97]), tumor size (compared with <5 cm, 5-10 cm [HR: 1.80; 95% CI, 1.14-2.85]; ≥10 cm [HR: 4.37; 95% CI, 2.69-7.11]), and use of neo/adjuvant radiation (HR: 0.54; 95% CI, 0.37-0.79) remained significantly associated with OS. For patients with metastatic disease, (1) 5-year OS was 12% (95% CI, 6%-21%) and (2) the only factor that remained significantly associated with OS on multivariable analysis was surgical resection for the primary tumor (HR: 0.14; 95% CI, 0.08-0.26)., Conclusions: The primary tumor location plays a significant role in predicting outcomes for patients with localized SS. Even though patients present with metastatic disease, surgical resection of the primary tumor improves their survival. These findings are critical for patient counseling and designing a personalized treatment plan that reflects the corresponding outcomes., Competing Interests: The authors report no conflicts of interest., (Copyright © 2024 Wolters Kluwer Health, Inc. All rights reserved.)

Background: Rhabdomyosarcomas are the most common soft tissue sarcoma in children, and pediatric alveolar rhabdomyosarcoma (ARMS) prognosis has improved based on cooperative studies. However, in adults, ARMS is significantly rarer, has poorer outcomes, and currently lacks optimal treatment strategies. Objective: This study aimed to evaluate the clinical outcome of an adult ARMS population with different front-line systemic chemotherapies and determine if any chemotherapy regimen is associated with improved survival. Materials and methods: This is a retrospective study of histologically confirmed fusion-positive ARMS patients over 18 years of age, who were treated at MD Anderson Cancer Center (MDACC) from 2004 to 2021 and received systemic chemotherapy. Descriptive clinical statistics were performed, including staging, front-line chemotherapy, multimodal therapy usage, response rates, and survival analyses. Results: 49 ARMS patients who received upfront chemotherapy were identified. Locoregional treatments included radiotherapy (RT) alone (29%, n = 14), surgery alone (10%, n = 5), or both (45%, n = 22). Median overall survival (OS) for the entire cohort was 3.6 years, and the overall response rate to systemic therapy was 89%. No chemotherapy regimen showed OS benefit, specifically analyzing the pediatric-based vincristine, actinomycin-D, cyclophosphamide (VAC) or adult-based vincristine, doxorubicin, ifosfamide (VDI) regimens, even when controlled for other clinical risk factors. Conclusion: In this single-center contemporary series, adult ARMS patient outcomes remain poor. There was no statistically significant OS difference in patients who did or did not receive adult or pediatric based ARMS regimens, although a high overall response rate to chemotherapy was seen across the entire cohort. Based on these observations, further randomized prospective studies are necessary to delineate which frontline chemotherapy regimen is most beneficial in this rare adult cancer., Competing Interests: The author(s) declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article., (© The Author(s) 2024.)

Purpose: To evaluate outcomes following percutaneous image-guided ablation of soft tissue sarcoma metastases to the liver., Materials and Methods: A single-institution retrospective analysis of patients with a diagnosis of metastatic soft tissue sarcoma who underwent percutaneous image-guided ablation of hepatic metastases between January 2011 and December 2021 was performed. Patients with less than 60 days of follow-up after ablation were excluded. The primary outcome was local tumor progression-free survival (LPFS). Secondary outcomes included overall survival, liver-specific progression-free survival. and chemotherapy-free survival., Results: Fifty-five patients who underwent percutaneous ablation for 84 metastatic liver lesions were included. The most common histopathological subtypes were leiomyosarcoma (23/55), followed by gastrointestinal stromal tumor (22/55). The median treated liver lesions was 2 (range, 1-8), whereas the median size of metastases were 1.8 cm (0.3-8.7 cm). Complete response at 2 months was achieved in 90.5% of the treated lesions. LPFS was 83% at 1 year and 80% at 2 years. Liver-specific progression-free survival was 66% at 1 year and 40% at 2 years. The overall survival at 1 and 2 years was 98% and 94%. The chemotherapy-free holiday from the start of ablation was 71.2% at 12 months. The complication rate was 3.6% (2/55); one of the complications was Common Terminology Criteria for Adverse Events grade 3 or higher. LPFS subgroup analysis for leiomyosarcoma versus gastrointestinal stromal tumor suggests histology-agnostic outcomes (2 years, 89% vs 82%, p = .35)., Conclusion: Percutaneous image-guided liver ablation of soft tissue sarcoma metastases is safe and efficacious., (© 2024 American Cancer Society.)

Kidney transplantation (KT) is the optimal form of renal replacement therapy for patients with end-stage renal diseases. However, this health service is not available to all patients, especially in developing countries. The deceased donor KT programs are mostly absent, and the living donor KT centers are scarce. Single-center studies presenting experiences from developing countries usually report a variety of challenges. This review addresses these challenges and the opposing strategies by reviewing the single-center experiences of developing countries. The financial challenges hamper the infrastructural and material availability, coverage of transplant costs, and qualification of medical personnel. The sociocultural challenges influence organ donation, equity of beneficence, and regular follow-up work. Low interests and motives for transplantation may result from high medicolegal responsibilities in KT practice, intense potential psychosocial burdens, complex qualification protocols, and low productivity or compensation for KT practice. Low medical literacy about KT advantages is prevalent among clinicians, patients, and the public. The inefficient organizational and regulatory oversight is translated into inefficient healthcare systems, absent national KT programs and registries, uncoordinated job descriptions and qualification protocols, uncoordinated on-site investigations with regulatory constraints, and the prevalence of commercial KT practices. These challenges resulted in noticeable differences between KT services in developed and developing countries. The coping strategies can be summarized in two main mechanisms: The first mechanism is maximizing the available resources by increasing the rates of living kidney donation, promoting the expertise of medical personnel, reducing material consumption, and supporting the establishment and maintenance of KT programs. The latter warrants the expansion of the public sector and the elimination of non-ethical KT practices. The second mechanism is recruiting external resources, including financial, experience, and training agreements., Competing Interests: Conflict-of-interest statement: All the authors report no relevant conflicts of interest for this article., (©The Author(s) 2024. Published by Baishideng Publishing Group Inc. All rights reserved.)

Background: Undifferentiated pleomorphic sarcomas (UPSs) are amongst the most common subtypes of soft-tissue sarcomas. Few real-world data on the use of immune checkpoint blockade (ICB) in UPS patients and other high-grade pleomorphic STS patients are available., Purpose: The purpose of our study is to describe the efficacy and toxicity of ICB in patients with advanced UPSs and other high-grade pleomorphic sarcomas treated at our institution., Methods: This is a retrospective, observational study of all patients with metastatic high-grade pleomorphic sarcomas treated with FDA-approved ICB at MD Anderson Cancer Center between 1 January 2015 and 1 January 2023. Patients included in trials for which results are not yet published were excluded., Results: Thirty-six patients with advanced/metastatic pleomorphic sarcomas were included. The median age was 52 years. A total of 26 patients (72%) had UPSs and 10 patients (28%) had other high-grade pleomorphic sarcomas. The median follow-up time was 8.8 months. The median PFS was 2.9 months. The 3-month PFS and 6-month PFS were 46% and 32%, respectively. The median OS was 12.9 months. The 12-month OS and 24-month OS were 53% and 29%, respectively. The best response, previous RT, and type of ICB treatment were significantly and independently associated with shorter PFS ( p = 0.0012, p = 0.0019 and p = 0.036, respectively). No new safety signal was identified, and the toxicity was overall manageable with no toxic deaths and only four patients (11%) stopping treatment due to toxicity., Conclusions: Real-world retrospective data are consistent with the published literature, with a promising 6-month PFS of 32%. Partial or stable responders to ICB treatment have significantly improved PFS compared to progressors.

Objective: The vast majority of gastrointestinal stromal tumors (GISTs) are driven by activating mutations in KIT , PDGFRA , or components of the succinate dehydrogenase (SDH) complex ( SDHA , SDHB , SDHC , and SDHD genes). A small fraction of GISTs lack alterations in KIT , PDGFRA , and SDH . We aimed to further characterize the clinical and genomic characteristics of these so-called "triple-negative" GISTs. Methods: We extracted clinical and genomic data from patients seen at MD Anderson Cancer Center with a diagnosis of GIST and available clinical next generation sequencing data to identify "triple-negative" patients. Results: Of the 20 patients identified, 11 (55.0%) had gastric, 8 (40.0%) had small intestinal, and 1 (5.0%) had rectal primary sites. In total, 18 patients (90.0%) eventually developed recurrent or metastatic disease, and 8 of these presented with de novo metastatic disease. For the 13 patients with evaluable response to imatinib (e.g., neoadjuvant treatment or for recurrent/metastatic disease), the median PFS with imatinib was 4.4 months (range 0.5-191.8 months). Outcomes varied widely, as some patients rapidly developed progressive disease while others had more indolent disease. Regarding potential genomic drivers, four patients were found to have alterations in the RAS/RAF/MAPK pathway: two with a BRAF V600E mutation and two with NF1 loss-of-function (LOF) mutations (one deletion and one splice site mutation). In addition, we identified two with TP53 LOF mutations, one with NTRK3 fusion ( ETV6-NTRK3 ), one with PTEN deletion, one with FGFR1 gain-of-function (GOF) mutation (K654E), one with CHEK2 LOF mutation (T367fs*), one with Aurora kinase A fusion ( AURKA-CSTF1 ), and one with FANCA deletion. Patients had better responses with molecularly targeted therapies than with imatinib. Conclusions: Triple-negative GISTs comprise a diverse cohort with different driver mutations. Compared to KIT / PDGFRA -mutant GIST, limited benefit was observed with imatinib in triple-negative GIST. In depth molecular profiling can be helpful in identifying driver mutations and guiding therapy.

Based on the demonstrated clinical activity of immune-checkpoint blockade (ICB) in advanced dedifferentiated liposarcoma (DDLPS) and undifferentiated pleomorphic sarcoma (UPS), we conducted a randomized, non-comparative phase 2 trial ( NCT03307616 ) of neoadjuvant nivolumab or nivolumab/ipilimumab in patients with resectable retroperitoneal DDLPS (n = 17) and extremity/truncal UPS (+ concurrent nivolumab/radiation therapy; n = 10). The primary end point of pathologic response (percent hyalinization) was a median of 8.8% in DDLPS and 89% in UPS. Secondary end points were the changes in immune infiltrate, radiographic response, 12- and 24-month relapse-free survival and overall survival. Lower densities of regulatory T cells before treatment were associated with a major pathologic response (hyalinization > 30%). Tumor infiltration by B cells was increased following neoadjuvant treatment and was associated with overall survival in DDLPS. B cell infiltration was associated with higher densities of regulatory T cells before treatment, which was lost upon ICB treatment. Our data demonstrate that neoadjuvant ICB is associated with complex immune changes within the tumor microenvironment in DDLPS and UPS and that neoadjuvant ICB with concurrent radiotherapy has significant efficacy in UPS., (© 2024. The Author(s), under exclusive licence to Springer Nature America, Inc.)

Purpose: The lungs are the most common site of metastasis for patients with soft tissue sarcoma. SABR is commonly employed to treat lung metastases among select patients with sarcoma with limited disease burden. We sought to evaluate outcomes and patterns of failure among patients with sarcoma treated with SABR for their lung metastases., Methods and Materials: We performed a retrospective review of patients treated at a tertiary cancer center between 2006 and 2020. Patient disease status at the time of SABR was categorized as either oligorecurrent or oligoprogressive. The Kaplan-Meier method was used to estimate disease outcomes. Uni- and multivariable analyses were conducted using the Cox proportional hazards model., Results: We identified 70 patients with soft tissue sarcoma treated with SABR to 98 metastatic lung lesions. Local recurrence-free survival after SABR treatment was 83% at 2 years. On univariable analysis, receipt of comprehensive SABR to all sites of pulmonary metastatic disease at the time of treatment was associated with improved progression-free survival (PFS; hazard ratio [HR], 0.51 [0.29-0.88]; P = .02). On multivariable analysis, only having systemic disease controlled at the time of SABR predicted improved PFS (median PFS, 14 vs 4 months; HR, 0.37 [0.20-0.69]; P = .002) and overall survival (median overall survival, 51 vs 14 months; HR, 0.17 [0.08-0.35]; P < .0001)., Conclusions: SABR provides durable long-term local control for sarcoma lung metastases. The most important predictor for improved outcomes was systemic disease control. Careful consideration of these factors should help guide decisions in a multidisciplinary setting to appropriately select the optimal candidates for SABR., (Copyright © 2023 Elsevier Inc. All rights reserved.)

Ripretinib and avapritinib have demonstrated activity in the late-line treatment of gastrointestinal stomal tumors (GISTs). We investigated whether patients previously treated with ripretinib benefit from avapritinib, and vice versa. Patients diagnosed with metastatic/unresectable GIST and treated with both drugs at two institutions in 2000-2021 were included. Patients were grouped by drug sequence: ripretinib-avapritinib (RA) or avapritinib-ripretinib (AR). Radiographic response was evaluated using RECIST 1.1. Kaplan-Meier and log-rank tests were used to compare time-to-progression (TTP) and overall survival (OS). Thirty-four patients (17 per group) were identified, with a median age of 48 years. The most common primary site was the small bowel (17/34, 50%), followed by the stomach (10/34, 29.4%). Baseline characteristics and tumor mutations were not significantly different between groups. Response rates (RRs) for ripretinib were 18% for RA and 12% for AR; RRs for avapritinib were 12% for AR and 18% for RA. Median TTPs for ripretinib were 3.65 months (95%CI 2-5.95) for RA and 4.73 months (1.87-15.84) for AR. Median TTPs for avapritinib were 5.39 months (2.86-18.99) for AR and 4.11 months (1.91-11.4) for RA. Median OS rates following RA or AR initiation were 29.63 (95%CI 13.8-50.53) and 33.7 (20.03-50.57) months, respectively. Both ripretinib and avapritinib were efficacious in the late-line treatment of GIST, with no evidence that efficacy depended on sequencing.

Background: Researches on the effect of hemodynamic stabilization on the implantation of conservative management for pediatric high-grade renal traumas are lacking. We aimed to assess the effect of maintaining the initial hemodynamic stability of pediatric patients with grades 3-5 renal trauma on the implementation of the conservative treatment and identify its defining factors., Methods: A prospective study was performed on pediatric patients with grade 3-5 renal traumas who presented to our hospital during July 2020-June 2022. Hemodynamically stable patients were compared with the unstable patients for clinical characteristics, hemodynamic stabilization, and rates of success of conservative treatment., Results: Forty-three patients were studied, including 26 boys and 17 girls. Of them, 28 (65.1%) patients presented with hemodynamic stability and 15 (34.9%) patients were unstable. Overall, 32 (74.4%) patients achieved and/or maintained hemodynamic stability for conservative management. There was a significant difference in blood pressure level at presentation (p < 0.001). The improvement of the hemodynamic parameters was significant per group and, in comparison (p < 0.001). The size of hematoma was significantly smaller in patients with hemodynamic stability (p = 0.023). Despite the longer (p = 0.033) hospital stay with conservative management, the rates of blood transfusion (p = 0.597) and hospital stay (p = 0.785) were not significantly different between both groups. The rates of nephrectomy and mortality were 14% and 0%, respectively. Blood pressure was independently associated with the achievement of maintained hemodynamic stability for conservative management (p = 0.022)., Conclusions: Hemodynamic stabilization seems to be effective and safe for implementing successful conservative management for pediatric patients with high-grade renal traumas. Blood pressure was the only independent factor of maintaining hemodynamic stability., (© 2023. The Author(s).)

Purpose: Chondrosarcomas are the most common primary bone tumor in adults. Isocitrate dehydrogenase 1 (IDH1) and IDH2 mutations are prevalent. We aimed to assess the clinico-genomic properties of IDH mutant versus IDH wild-type (WT) chondrosarcomas as well as alterations in other genes., Experimental Design: We included 93 patients with conventional and dedifferentiated chondrosarcoma for which there were available clinical next-generation sequencing data. Clinical and genomic data were extracted and compared between IDH mutant and IDH WT chondrosarcomas and between TP53 mutant and TP53 WT chondrosarcomas., Results: IDH1 and IDH2 mutations are prevalent in chondrosarcoma (50.5%), more common in chondrosarcomas arising in the extremities, associated with higher age at diagnosis, and more common in dedifferentiated chondrosarcomas compared with grades 1-3 conventional chondrosarcoma. There was no difference in survival based on IDH mutation in univariate and multivariate analyses. TP53 mutation was the next most prevalent (41.9%) and is associated with worse overall survival and metastasis-free survival in both univariate and multivariate analyses. TP53 mutation was also associated with higher risk of recurrence following curative-intent surgery and worse survival among patients that presented with de novo metastatic disease., Conclusions: IDH mutations are prevalent in chondrosarcoma though were not associated with survival outcomes in this cohort. TP53 mutations were the next most common alteration and were associated with worse outcomes., (©2023 American Association for Cancer Research.)

Background: Although the availability of related living donors (LDs) provides a better chance for receiving kidney transplantation (KT), the evaluation protocols for LD selection remain a safeguard for the LD's safety. These protocols are variable from one center to another, resulting in variable rates of decline of the potential LDs (PLDs). The decline of willing PLDs may occur at any stage of evaluation, starting from the initial contact and counseling to the day of operation., Aim: To identify the causes of the decline of PLDs, the predictors of PLD candidacy, and the effect on achieving LDKT., Methods: A retrospective study was performed on the willing PLDs who attended our outpatient clinic for kidney donation to their related potential recipients between October 2015 and December 2022. The variables influencing their candidacy rate and the fate of their potential recipients were studied. Two groups of PLDs were compared: Candidate PLDs after a completed evaluation vs non-candidate PLDs with a complete or incomplete evaluation. A multivariate logistic regression was performed to assess the factors contributing to the achievement of PLD candidacy., Results: Of 321 willing PLDs, 257 PLDs (80.1%) accessed the evaluation to variable extents for 212 potential recipients, with a mean age (range) of 40.5 ± 10.4 (18-65) years, including 169 females (65.8%). The remaining 64 PLDs (19.9%) did not access the evaluation. Only 58 PLDs (18.1%) succeeded in donating, but 199 PDLs (62.0%) were declined; exclusion occurred in 144 PLDs (56.0%) for immunological causes (37.5%), medical causes (54.9%), combined causes (9.7%), and financial causes (2.1%). Regression and release occurred in 55 PLDs (17.1%). The potential recipients with candidate PLDs were not significantly different from those with non-candidate PLDs, except in age ( P = 0.041), rates of completed evaluation, and exclusion of PLDs ( P < 0.001). There were no factors that independently influenced the rate of PLD candidacy. Most patients who failed to have KT after the decline of their PLDs remained on hemodialysis for 6 mo to 6 years., Conclusion: The rate of decline of willing related PLDs was high due to medical or immunological contraindications, release, or regression of PLDs. It reduced the chances of high percentages of potential recipients in LDKT., Competing Interests: Conflict-of-interest statement: The authors have no financial relationships to disclose., (©The Author(s) 2023. Published by Baishideng Publishing Group Inc. All rights reserved.)

Purpose: To compare the clinical results and patients' health-related quality of life (HR-QoL) of ileal conduit (IC) versus single stoma uretero-cutanoustomy (SSUC) after radical cystectomy in two groups of randomly selected patients ≥ 75 years., Methods: From January 2013 to March 2018, 100 patients ≥ 75 years with muscle invasive BCa underwent RCX and cutaneous diversion. Patients were divided in two groups; group I underwent IC (50 patients) and group II underwent SSUC (50 patients). Postoperative evaluation included clinical, laboratory, radiographic and HR-QoL. The latter was performed using the Functional Assessment of Cancer Therapy-Bladder Cancer (FACT-BL) after 12 months postoperatively., Results: Patients' characteristics were comparable between both groups. No intraoperative complications occurred. Early postoperative complications occurred in 27 patients [16 (35.5%) in group I and 11 (23.9%) in group II (p = 0.02)]. Delayed postoperative complications occurred in 26 patients [6 (13.3%) in group I and 20 (43.4%) in group II, (P = 0.002)]. No significant differences between both groups regarding the physical, social/family, emotional, functional and additional concerns scales of FACT-BL questionnaire were reported., Conclusion: SSUC is a good alternative to IC in elderly frail patients  ≥ 75 years and those with multiple comorbidities who require rapid surgery in terms of perioperative complications and HR-QoL. However, the stomal complications and the possibility of a frequent stent exchange are considered its drawbacks., (© 2023. The Author(s), under exclusive licence to Springer Nature B.V.)

Background: Preemptive living donor kidney transplantation (PLDKT) is recommended as the optimal treatment for end-stage renal disease., Aim: To assess the rate of PLDKT among patients who accessed KT in our center and review the status of PLDKT in Egypt., Methods: We performed a retrospective review of the patients who accessed KT in our center from November 2015 to November 2022. In addition, the PLDKT status in Egypt was reviewed relative to the literature., Results: Of the 304 patients who accessed KT, 32 patients (10.5%) had preemptive access to KT (PAKT). The means of age and estimated glomerular filtration rate were 31.7 ± 13 years and 12.8 ± 3.5 mL/min/1.73 m 2 , respectively. Fifty-nine patients had KT, including 3 PLDKTs only (5.1% of total KTs and 9.4% of PAKT). Twenty-nine patients (90.6%) failed to receive PLDKT due to donor unavailability (25%), exclusion (28.6%), regression from donation (3.6%), and patient regression on starting dialysis (39.3%). In multivariate analysis, known primary kidney disease ( P = 0.002), patient age ( P = 0.031) and sex ( P = 0.001) were independent predictors of achievement of KT in our center. However, PAKT was not significantly ( P = 0.065) associated with the achievement of KT. Review of the literature revealed lower rates of PLDKT in Egypt than those in the literature., Conclusion: Patient age, sex, and primary kidney disease are independent predictors of achieving living donor KT. Despite its non-significant effect, PAKT may enhance the low rates of PLDKT. The main causes of non-achievement of PLDKT were patient regression on starting regular dialysis and donor unavailability or exclusion., Competing Interests: Conflict-of-interest statement: The authors have no financial relationships to disclose., (©The Author(s) 2023. Published by Baishideng Publishing Group Inc. All rights reserved.)

Purpose: Developing new therapeutics for any of the more than 100 sarcoma subtypes presents a challenge. After progression from standard therapies, patients with sarcoma may be referred for enrollment in early-phase trials. This study aimed to investigate whether enrollment in biomarker-matched early-phase clinical trials leads to better outcomes for patients with advanced sarcoma., Experimental Design: In this retrospective analysis, investigational treatment characteristics and longitudinal survival outcomes were analyzed in patients with biopsy-confirmed sarcoma enrolled in early-phase trials at MD Anderson Cancer Center from May 2006 to July 2021., Results: Five hundred eighty-seven patients were included [405 soft tissue, 122 bone, 60 gastrointestinal stromal tumor (GIST); median of three prior lines of therapy]. Most common subtypes were leiomyosarcoma (17.2%), liposarcoma (14.0%), and GIST (10.2%). Molecular testing was available for 511 patients (87.1%); 221 patients (37.6%) were treated in matched trials. Overall response rate was 13.1% matched compared with 4.9% in unmatched (P < 0.001); the clinical benefit rate at 6 months was 43.9% vs. 19.9% (P < 0.001). Progression-free survival was longer for patients in matched trials (median, 5.5 vs. 2.4 months; P < 0.001), and overall survival was also superior for patients in matched trials (median, 21.5 vs. 12.3 months; P < 0.001). The benefit of enrollment in matched trials was maintained when patients with GIST were excluded from the analysis., Conclusions: Enrollment in biomarker-matched early-phase trials is associated with improved outcomes in heavily pretreated patients with metastatic sarcoma. Molecular testing of tumors from patients with advanced sarcoma and enrollment in matched trials is a reasonable therapeutic strategy., (©2023 The Authors; Published by the American Association for Cancer Research.)

Objective: Well-differentiated (WDLPS) and dedifferentiated liposarcoma (DDLPS) account for the majority of liposarcomas. Although gemcitabine-docetaxel is used as second-line treatment in soft tissue sarcomas, its efficacy in WDLPS/DDLPS is not established. This study retrospectively analyzed the efficacy of gemcitabine regimens in WDLPS/DDLPS., Methods: All patients with WDLPS or DDLPS who received gemcitabine-based chemotherapy at our institution between September 2002 and January 2021 were included. Response was evaluated by an independent radiologist using RECIST 1.1. The Kaplan-Meier method was used to estimate distributions of survival outcomes and log-rank tests were used to compare survival outcomes between subgroups., Results: Sixty-five WDLPS/DDLPS patients were included. Seven patients (10.8%) received a gemcitabine-based regimen more than once, totaling 72 treatments. The median age at the start of treatment was 66 years (range 32-80 years). Sixty-five (90.3%) regimens were gemcitabine-docetaxel, and 7 (9.7%) were gemcitabine alone. Majorities of treatments were for disease that was recurrent/metastatic (86.1%), was abdominal/retroperitoneal (83.3%), and had DDLPS components (88.9%), while 25.0% of treatments were for multifocal disease. The overall response rate was 9.7% (7/72). All responses were in patients with documented DDLPS. The median time to progression was 9.2 months (95% CI 5.3-12.3 months). The median overall survival from the start of therapy was 18.8 months (95% CI 13.1-32.4 months)., Conclusion: Gemcitabine-docetaxel is an efficacious second-line treatment for DDLPS. Though cross study comparisons are not advisable, response to gemcitabine-docetaxel compares favorably to current standard options trabectedin and eribulin. This combination is a valid comparator arm for future second-line trials in DDLPS., (© 2022 The Authors. Cancer Medicine published by John Wiley & Sons Ltd.)

Objectives: to assess the functional outcome of performing ureteric re-implantation to an angled short chimney in a modified spiral orthotopic ileal neobladder., Patients and Methods: From January 2018 to December 2020, 90 male patients with bladder cancer underwent radical cystectomy and spiral ileal neobladder reconstruction with a chimney. Patients were randomly divided in two groups according to the position of the chimney [straight and angled] to which the ureters will be implanted. Postoperative evaluation included clinical, laboratory, radiographic and urodynamic studies., Results: There were no perioperative deaths. The mean operative time for the procedure was 4.7 ± 1.2 h in group I and 4.9 ± 1.3 h in group II (p 0.456). No intraoperative complications occurred. Early postoperative complications occurred in 8 patients. In group I, according to the modified Clavien system, GII complication occurred in 2 (5%) patients in the form of DVT in 1 (2.5%) and surgical site infection in 1 (2.5%). GIIIa occurred in 3 (7.5%) patients in the form of wound dehiscence. In group II, GII occurred in 2 (4.8%) patients in the form of prolonged urinary leakage and myocardial infarction, each occurred in one patient. GIIIb occurred in 1 (2.4%) patient in the form of intestinal leak. Poucho-ureteral reflux occurred in 10 patients [3 (7.5%) in group I and 7 (16.8%) in group II (p 0.001)]., Conclusions: The preliminary results of the right sided angled chimney during neobladder reconstruction are safe, acceptable, without an extra time to develop an anti-reflux technique and without an increased incidence of reflux., Competing Interests: Declaration of Competing Interest None declared., (Copyright © 2022 Elsevier Ltd, BASO ~ The Association for Cancer Surgery, and the European Society of Surgical Oncology. All rights reserved.)

Background: Despite the documented effects of the coronavirus disease 2019 (COVID-19) on spermatogenesis, the reversibility of these effects is uncertain. We aimed to assess the changes of sperm quality between the infection and recovery phases of COVID-19 in reproductive-aged men. The semen quality of men with mild-to-moderated COVID-19 (defined by the degrees of symptoms and chest involvement on computed tomography) was studied during October, 2020-May, 2021 at our hospital. Two semen samples were analyzed at timings estimated to represent spermatogenic cycles during the infection and recovery phases of COVID-19., Results: A total of 100 patients were included with mean ± SD (range) age of 24.6 ± 3.3 (21-35) years. During infection, 33% of patients had abnormal semen quality. However, a significant reduction was found in this abnormality from 33 to 11% (P < 0.001) after recovery from infection. In a comparison of the two semen analyses, there were significant improvements in the mean values of sperm progressive motility (P =0.043) and normal morphology (P < 0.001). However, the mean sperm concentration showed a statistically insignificant increase (P = 0.844)., Conclusions: In reproductive-aged patients with mild-to-moderate COVID-19, the effects on seminal quality were recoverable, represented by significant improvements in the means of progressive sperm motility and normal morphology between the infection and recovery phases of COVID-19., Trial Registration: ClinicalTrials, NCT04595240 ., (© 2022. The Author(s).)

Purpose: Recently, the Connective Tissue Oncology Society published consensus guidelines for recognizing ultrarare sarcomas (URS), defined as sarcomas with an incidence ≤1 per 1,000,000. We assessed the outcomes of 56 patients with soft tissue, and 21 with bone sarcomas, enrolled in Phase 1 trials., Experimental Design: In this Sarcoma-Matched Biomarker Analysis (SAMBA-102 study), we reviewed records from patients on Phase 1 trials at the University of Texas MD Anderson Cancer Center between January 2013 and June 2021., Results: Among 587 sarcomas, 106 (18.1%) were classified as URS. Fifty (47%) were male, and the median age was 44.3 years (range, 19-82). The most common subtypes were alveolar soft part sarcoma (ASPS), chordoma, dedifferentiated chondrosarcoma, and sclerosing epithelioid fibrosarcoma. Compared with common sarcomas, median OS was similar 16.1 months [95% confidence interval (CI), 13.6-17.5] versus 16.1 (95% CI, 8.2-24.0) in URS (P = 0.359). Objective response to treatment was higher in URS 13.2% (n = 14/106) compared with common sarcomas 6.9% (n = 33/481; P = 0.029). Median OS for those treated on matched trials was 27.3 months (95% CI, 1.9-52.7) compared with 13.4 months (95% CI, 6.3-20.6) for those not treated on matched trials (P = 0.291). Eight of 33 (24%) molecularly matched treatments resulted in an objective response, whereas 6 of 73 unmatched treatments (8.2%) resulted in an objective response (P = 0.024). Clinical benefit rate was 36.4% (12/33) in matched trials versus 26.0% (19/73) in unmatched trials (P = 0.279)., Conclusions: The results demonstrate the benefit of genomic selection in Phase 1 trials to help identify molecular subsets likely to benefit from targeted therapy., (©2022 The Authors; Published by the American Association for Cancer Research.)

Background: Few standard treatment options are available for patients with metastatic sarcomas. We did this trial to evaluate the efficacy, safety, and changes in the tumour microenvironment for durvalumab, an anti-PD-L1 drug, and tremelimumab, an anti-CTLA-4 drug, across multiple sarcoma subtypes., Methods: In this single-centre phase 2 trial, done at The University of Texas MD Anderson Cancer Center (Houston, TX USA), patients aged 18 years or older with advanced or metastatic sarcoma with an Eastern Cooperative Oncology Group performance status of 0 or 1 who had received at least one previous line of systemic therapy were enrolled in disease subtype-specific groups (liposarcoma, leiomyosarcoma, angiosarcoma, undifferentiated pleomorphic sarcoma, synovial sarcoma, osteosarcoma, alveolar soft-part sarcoma, chordoma, and other sarcomas). Patients received 1500 mg intravenous durvalumab and 75 mg intravenous tremelimumab for four cycles, followed by durvalumab alone every 4 weeks for up to 12 months. The primary endpoint was progression-free survival at 12 weeks in the intention-to-treat population (all patients who received at least one dose of treatment). Safety was also analysed in the intention-to-treat population. This trial is registered with ClinicalTrials.gov, NCT02815995, and is completed., Findings: Between Aug 17, 2016, and April 9, 2018, 62 patients were enrolled, of whom 57 (92%) received treatment and were included in the intention-to-treat population. With a median follow-up of 37·2 months (IQR 1·8-10·1), progression-free survival at 12 weeks was 49% (95% CI 36-61). 21 grade 3-4 treatment-related adverse events were reported, the most common of which were increased lipase (four [7%] of 57 patients), colitis (three [5%] patients), and pneumonitis (three [5%] patients). Nine (16%) patients had a treatment related serious adverse event. One patient had grade 5 pneumonitis and colitis., Interpretation: The combination of durvalumab and tremelimumab is an active treatment regimen for advanced or metastatic sarcoma and merits evaluation in specific subsets in future trials., Funding: AstraZeneca., Competing Interests: Declaration of interests NS participated in the advisory board of Boehringer Ingelheim. APC reports consulting fees from Deciphera, Inhibrx, Bayer, and Genentech; and participated in the advisory board of Applied Clinical Intelligence. CH reports research funding from Dragonfly and Lovance; salary supported in part by National Institute for Health (NIH) grants (P30CA016672, P50CA221703, U24CA224285, and R01CA236905); consulting fees from Nanobiotix; honoraria from Society for Immunotherapy of Cancer; and stock options from Briacell. SS reports support for attending meetings and travel from Medtronic, and stocks from Inari. AJL reports research funding and consulting fees from, and advisory board membership for AstraZeneca. CLR reports research funding from Bristol Myers Squibb. AF reports research funding paid to institution from NIH (U01 CA224044-02). J-CS reports a leadership role as a fulltime employee at AstraZeneca from September, 2017, to December, 2019; a leadership role as a fulltime employee at Amgen since August, 2021; and is a stock owner from Relay Therapeutics and Gritstone Bio. IIW reports research funding to their institution from Genentech, HTG Molecular, Merck, Bristol Myers Squibb, MedImmune, Adaptive, Adaptimmune, EMD Serono, Pfizer, Takeda, Amgen, Karus, Johnson & Johnson, Bayer, Iovance, 4D, Novartis, and Akoya; scientific and financial support for the institutional Cancer Immune Monitoring and Analysis Center-Cancer Immunologic Data Commons Network through the National Cancer Institute of the NIH Cooperative Agreement U24CA224285); consulting fees from Roche, Bayer, Bristol Myers Squibb, AstraZeneca, Pfizer, HTG Molecular, Merck, GlaxoSmithKline, Guardant Health, Novartis, Flame, Sanofi, Janssen, Daiichi Sankyo, Oncocyte, Amgen, and MSD; and honoraria from Medscape, Roche, Pfizer, AstraZeneca, Platform Health, and Merck. PH reports advisory board membership for Dragonfly Scientific Advisory Board (SAB) and Immatics SAB. All other authors declare no competing interests., (Copyright © 2022 Elsevier Ltd. All rights reserved.)

Objective: The aim of this study is to compare the outcomes of right- and left-sided live donor nephrectomies using the inverted kidney transplantation technique for right live donor nephrectomy on transplantation., Material and Methods: A retrospective review was done for the cases of live donor nephrectomy, either as open donor nephrectomy or laparoscopic donor nephrectomy between 2004 and 2019. Inverted kidney transplantation was used with right-sided grafts. The variables of the right- and left-sided live donor nephrectomies were compared., Results: There were 202 live donor nephrectomies including 71 (35.1%) open donor nephrectomies and 131 (64.9%) laparoscopic donor nephrectomies with 4 cases of conversion to open donor nephrectomy. There were 119 (58.9%) right-sided and 83 (41.1%) left-sided live donor nephrectomies with insignificantly different mean operative time (123 and 127 minutes; P=.09), mean warm ischemia time (82.3 and 84.5 seconds; P=.32), and mean blood loss (73 and 78 mL; P=.18), respectively. Inverted kidney transplantation was performed for 86% of grafts from right live donor nephrectomies. Discharge from hospital was on an average of 4.3 days postoperatively. There were only 3 complications (1 in right live donor nephrectomy and 2 in left live donor nephrectomies) with grade 2 according to Clavien-Dindo Classification. Incidence of delayed graft function (P=.09), transplant vein thrombosis (1 case in each group), 1-year graft survival rate (93.2% vs. 94.8%; P=.12), and 1-year serum creatinine levels (1.4 ± 0.3 vs. 1.3 ± 0.2; P=.09) revealed statistically insignificant differences., Conclusion: Regardless of the surgical technique, the right live donor nephrectomy seems to be technically as safe as the left live donor nephrectomy for both the donors and the recipients. Using inverted kidney transplantation provided convenient extensions of graft's vessels to full length with no significant increased incidence of vascular thrombosis.

Palbociclib has been evaluated in early phase trials for well-differentiated liposarcoma (WDLPS) and dedifferentiated liposarcoma (DDLPS) patients, with reported median progression-free survival (PFS) of 18 weeks. Here, we report on real-world use and surgical outcomes associated with palbociclib treatment. We retrospectively reviewed 61 consecutive patients with retroperitoneal WDLPS (n = 14) or DDLPS (n = 47) treated with palbociclib monotherapy between 1 March 2016 and 28 February 2021 at The University of Texas MD Anderson Cancer Center. At palbociclib initiation, median age was 64 (interquartile range [IQR] 56-72). In WDLPS and DDLPS cohorts, the median number of prior systemic treatments was 0 (IQR 0-0) and 2 (IQR 0-4), respectively. Median number of prior surgeries was 2 (WDLPS IQR 1-2.75) and 2 (DDLPS IQR 1-3). Median PFS was 9.2 (WDLPS IQR 3.9-21.9) and 2.6 months (DDLPS IQR 2.0-6.1), with median time on treatment of 7.4 months (WDLPS IQR 3.5-14.2) and 2.7 months (DDLPS IQR 2.0-5.7). Twelve patients ultimately underwent surgical resection. Resections were macroscopically complete (R0/R1) in half (n = 6/12), among whom only one patient experienced relapse after resection (median follow-up 7.5 months). All patients who underwent macroscopically incomplete resections progressed after surgery with median time to progression of 3.3 months (IQR 2.3-4.4). Surgery after palbociclib treatment was not associated with improved overall survival. Efficacy of palbociclib monotherapy for patients with advanced WDLPS and DDLPS is disappointing. While palbociclib may have been used to delay surgery, there was no clear benefit from treatment and few patients achieved prolonged tumor control., (© 2022 UICC.)

Renal disorders are associated with Hepatitis E virus (HEV) infection. Progression to end-stage renal disease and acute kidney injury are complications associated with HEV infection. The mechanisms by which HEV mediates the glomerular diseases remain unclear. CD10 + /CD13 + primary proximal tubular (PT) epithelial cells, isolated from healthy donors, were infected with HEV. Inflammatory markers and kidney injury markers were assessed in the presence or absence of peripheral blood mononuclear cells (PBMCs) isolated from the same donors. HEV replicated efficiently in the PT cells as shown by the increase in HEV load over time and the expression of capsid Ag. In the absence of PBMCs, HEV was not nephrotoxic, with no direct effect on the transcription of chemokines (Cxcl-9, Cxcl-10, and Cxcl-11) nor the kidney injury markers (kidney injury molecule 1 (KIM-1), neutrophil gelatinase-associated lipocalin (NGAL), and interleukin 18 (lL-18)). While higher inflammatory responses, upregulation of chemokines and kidney injury markers expression, and signs of nephrotoxicity were recorded in HEV-infected PT cells cocultured with PBMCs. Interestingly, a significantly higher level of IFN-γ was released in the PBMCs-PT coculture compared to PT alone during HEV infection. In conclusion: The crosstalk between immune cells and renal epithelium and the signal axes IFN-γ/chemokines and IL-18 could be the immune-mediated mechanisms of HEV-induced renal disorder.

Background: The objective of this study was to evaluate treatment outcomes for patients with desmoid tumors (DTs) receiving local therapy with surgery alone, radiation therapy (RT) alone, or combined modality therapy (RT and surgery)., Methods: This was a cross-sectional cohort study of 412 patients with nonmesenteric DTs who received local therapy at the authors' institution between 1965 and 2018., Results: The median follow-up time was 95 months (range, 1-509 months). Local recurrence occurred in 127 patients (31%) at a median time of 21 months (interquartile range, 12-38 months). The 5-year local control (LC) rate was 67%. Patient or tumor factors that were significantly associated with poorer 5-year LC in a multivariable analysis included an age ≤ 30 years (57% vs 75% for an age > 30 years; hazard ratio [HR], 1.73; P = .004), an extremity location (57% vs 71% for a nonextremity location; HR, 1.77; P = .004), and large tumors (59% for >10 cm [HR, 2.17; P = .004] and 65% for 5.1-10 cm [HR, 1.71; P = .02] vs 76% for ≤5 cm). Subset analyses of these high-risk patients revealed no local therapy strategy to be superior for young patients ≤ 30 years old (HR for surgery, 1.42; P = .33; HR for RT, 1.36; P = .38) or for large tumors > 10 cm (HR for surgery, 1.55; P = .46; HR for RT, 0.91; P = .91). However, for patients with extremity tumors, surgery alone was significantly associated with inferior LC (HR for surgery, 5.15; P < .001; HR for RT, 1.51; P = .38)., Conclusions: Local therapy provides durable tumor control in the majority of patients with DTs. However, young patients, patients with an extremity location, and patients with large tumors are at increased risk of recurrence. When active treatment is indicated, systemic therapy should perhaps be considered as a first-line option in these high-risk subsets. Prospective multi-institutional studies evaluating this strategy are warranted., (© 2020 American Cancer Society.)

Overexpression of transcription factor 3 in alveolar soft part sarcoma(ASPS) results in upregulation of cell proliferation pathways. No standard treatment algorithm exists for ASPS; multikinase inhibitors[tyrosine kinase inhibitor (TKI)] and immune checkpoint inhibitors (ICI) have shown clinical benefit. To date, no studies have reported on management strategies or sequencing of therapy. We evaluated ASPS treatment patterns and responses in an experimental therapeutics clinic. Genomic and morphoproteomic analysis was performed to further elucidate novel targets. We retrospectively reviewed patients with ASPS treated on clinical trials. Demographic and clinical next-generation sequencing (NGS) profiles were collected. AACR GENIE database was queried to further evaluate aberrations in ASPS. Morphoproteomic analysis was carried out to better define the biology of ASPS with integration of genomic and proteomic findings. Eleven patients with ASPS were identified; 7 received NGS testing and mutations in CDKN2A ( n = 1) and hepatocyte growth factor ( n = 1) were present. Ten patients were treated with TKIs with stable disease as best response and 4 patients with ICI (three partial responses). Within GENIE, 20 patients were identified harboring 3 called pathogenic mutations. Tumor mutation burden was low in all samples. Morphoproteomic analysis confirmed the expression of phosphorylated c-Met. In addition, fatty acid synthase and phosphorylated-STAT3 were detected in tumor cell cytoplasm and nuclei. Patients with ASPS have a quiescent genome and derive clinical benefit from VEGF-targeting TKIs. Morphoproteomic analysis has provided both additional correlative pathways and angiogenic mechanisms that are targetable for patients with ASPS. Our study suggests that sequential therapy with TKIs and immune checkpoint inhibitors is a reasonable management strategy., (©2020 American Association for Cancer Research.)

Purpose: To update our experience with long-term outcomes in patients with desmoid fibromatosis treated with radiation therapy (RT) and to characterize factors associated with increased risk of local recurrence., Methods and Materials: We reviewed the records of 209 consecutive patients with desmoid fibromatosis treated with RT, either alone or as combined-modality therapy (CMT) with surgery, at our institution from 1965 to 2015., Results: Median follow-up time was 98 months (range, 1-509 months). The 5- and 10-year local control (LC) was 71% and 69%, respectively. Fifty-nine patients (28%) experienced a local recurrence at a median time of 23 months (interquartile range, 15-38 months). Among all patients, on multivariable analysis, adjusting for anatomic site, size, age, treatment era (>2005 vs ≤2005), treatment approach (RT alone vs CMT), and an interaction between age and treatment, we found only age ≤30 years (hazard ratio [HR], 2.94; P = .005; 95% confidence interval [CI], 1.38-6.27) and large tumor size >10 cm (HR, 2.51; P = .03; 95% CI, 1.09-5.78) to be correlated with poorer LC. Notably, for patients receiving RT alone, the 5-year LC was 43% for patients ≤30 years old versus 75% for >30 years old (P < .001). On multivariable analyses, for patients receiving RT alone, the only factor associated with inferior LC was age ≤30 years (HR, 2.87; P = .001; 95% CI, 1.51-5.47). The same was true for patients treated with CMT; age ≤30 years was the only factor associated with inferior LC (HR, 5.36; P = .01; 95% CI, 1.40-20.58)., Conclusions: Among all patients with desmoid fibromatosis, RT is an effective local therapy for tumor control. However, young patients ≤ 30 years have notably high rates of local recurrence regardless of treatment strategy, which requires further study. Treatment decisions should be risk-adapted by large referral centers with multidisciplinary expertise in desmoid management., (Copyright © 2018 Elsevier Inc. All rights reserved.)

Introduction: To prospectively compare the perioperative and functional outcomes of laparoscopic (LP) and open pyeloplasty (OP) in three academic institutions., Material and Methods: Between September 2012 and September 2016, 102 patients with primary uteropelvic junction obstruction (UPJO) underwent pyeloplasty (51 LP and OP for the other 51 patients). Demographic data, perioperative parameters, including operative time, estimated blood loss, complications, length of hospital stay, and functional outcome were compared, and SF-8 Health Survey scoring was recorded for each group.Patients were followed up by ultrasound (US) and /or intravenous urography (IVU) at 3, 6 and 12 months. A MAG-3 renal scan was performed at 3 months postoperatively., Results: The mean operative time was significantly shorter in the open group (153.2 ±42 min vs. 219.8 ±46 min; P <0.001). Compared to OP, the mean postoperative analgesia (Diclofenac) requirement was significantly less in the LP group (101.1 ±36 mg vs. 459.1 ±123 mg; P <0.001). The median hospital stay was significantly shorter for LP (2.7 ±1.8 days vs. 9.09 ±7.3 days; P <0.001). The median follow-up period was 19.7 months (12-28 months). The success rate was 96.1% in the OP group and 94.1% in the LP group., Conclusions: In spite of being a technically demanding procedure, LP offers faster recovery and higher patient satisfaction. In our hands, OP still has a shorter operative time and relatively lower retreatment rate.

Background: There are no clinical trials specifically addressing chemotherapy for adults with Ewing sarcoma (ES). Five-year event-free survival (EFS) of adults on pediatric studies of ES (44%-47%) is worse than that of children treated with the same therapy (69%). The object of this study was to review the results of therapy with vincristine, ifosfamide, and doxorubicin (VID) in the multidisciplinary treatment of adults with ES at our institution., Materials and Methods: Charts for adults treated for ES from 1995 to 2011 were retrospectively reviewed. Clinician-reported radiographic tumor response, type of local therapy, pathologic response, and survival data were collected., Results: Seventy-one patients were identified who received VID as initial therapy. The median age was 25 (range: 16-64). Forty-two patients (59%) presented with a localized disease and 29 patients (41%) presented with a distant metastasis. Of all patients treated with VID, 83.6% showed a radiological response. Patients who presented with a localized disease had a 5-year overall survival (OS) of 68% (median not reached), compared with 10.3% (median: 1.9 years) in those who presented with distant metastases. Five-year EFS was 67%. The nine patients with a pelvic primary tumor had inferior 5-year OS (42%) to the 33 with primary tumors at other sites (75%). The 5-year OS of those who had greater than or equal to 95% necrosis after neoadjuvant VID ( n  = 20; 5-year OS: 84%) was superior to those who had less than 95% necrosis ( n  = 13; 5-year OS: 53%)., Conclusion: In adults with primary ES, VID combined with an adjuvant strategy based on post-treatment percent necrosis has favorable outcomes compared with historical adult controls., Implications for Practice: Ewing sarcoma (ES) is a rare tumor in adults, and there are no dedicated clinical trials in the adult population. Most therapy is modeled after the published pediatric studies, although the small numbers of adult patients included on those studies did significantly worse than the children. We modeled our treatment on other adult sarcomas and reviewed the charts of 71 adult patients with ES treated with vincristine, ifosfamide, and doxorubicin (VID). In adults with primary ES, VID combined with an adjuvant strategy based on post-treatment percent necrosis has favorable outcomes compared with historical adult controls., Competing Interests: Disclosures of potential conflicts of interest may be found at the end of this article., (© AlphaMed Press 2017.)

Regorafenib was approved as third-line therapy for advanced Gastrointestinal Stromal Tumour (GIST) at a starting dose of 160 mg daily 3 weeks on, 1 week off, based on improvement in progression free survival over placebo (4.8 vs. 0.9 months), but the response rate was low at 4.5%. Given the high toxicity rate in GIST patients, there is variability in the post-marketing dosing of regorafenib. We aimed to summarize our experience regarding prescribing patterns, efficacy and toxicity of regorafenib and determine the role of response assessment by Choi criteria in GIST patients. We included 28 patients who received regorafenib from our pharmacy. Baseline patient characteristics and treatment outcomes were recorded and an independent radiologist assessed response using Choi and RECIST. Seventy-nine percent of patients started at a 120 mg continuous daily dosing schedule, different from the standard intermittent dosing schedule. Grade 3/4 adverse events were experienced by 43% of patients. Median progression-free survival was 8.7 months. Continuous dosing with regorafenib at 120 mg daily is the preferred prescribing pattern and appears to be better tolerated and with comparable efficacy to the current standard dose. Similar to imatinib, the partial response rate for regorafenib by Choi (29%) was higher compared to RECIST (4%).

Objectives: To report the findings and management of patients with persistent Müllerian duct syndrome (PMDS)., Patients and Methods: Nineteen phenotypically male patients (aged 8 months to 27 years) presented with testicular maldescent. All of them had normal male external genitalia. Two of them had had a previous diagnosis of persistent Müllerian structures. All patients were karyotyped, and had a hormonal profile, diagnostic laparoscopy, retrograde urethrocystogram, gonadal biopsies, and surgical management according to the findings. The follow-up was based on a clinical examination, abdominal ultrasonography (US) and scrotal colour-Doppler US at 3 and 6 months after surgery, and every 6 months thereafter., Results: Diagnostic laparoscopy showed the presence of persistent Müllerian structures in all 19 patients. All patients had a normal male karyotype (46XY). Ten patients had a laparoscopic excision of their Müllerian structures while the remaining nine patients had their Müllerian structures left in place. No malignant changes were found in the excised Müllerian tissues. Of the 37 gonadal biopsies taken, 31 (84%) indicated normal testes., Conclusions: The incidence and prevalence of PMDS are not well estimated. Müllerian structures should be removed whenever possible to avoid the risk of malignant transformation. The early diagnosis of PMDS makes possible the excision of Müllerian structures and a primary orchidopexy. A long-term follow-up is needed for patients with intact Müllerian structures and magnetic resonance imaging might be a better method than US for that purpose. Most of the patients had normal testicular histology, which might allow fertility.