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BackgroundDesmoid tumors are rare, locally aggressive, highly recurrent soft-tissue tumors without approved treatments.MethodsWe conducted a phase 3, international, double-blind, randomized, placebo-controlled trial of nirogacestat in adults with progressing desmoid tumors according to the Response Evaluation Criteria in Solid Tumors, version 1.1. Patients were assigned in a 1:1 ratio to receive the oral γ-secretase inhibitor nirogacestat (150 mg) or placebo twice daily. The primary end point was progression-free survival.ResultsFrom May 2019 through August 2020, a total of 70 patients were assigned to receive nirogacestat and 72 to receive placebo. Nirogacestat had a significant progression-free survival benefit over placebo (hazard ratio for disease progression or death, 0.29; 95% confidence interval, 0.15 to 0.55; P

PurposeOsteosarcoma is a rare cancer and a third of patients who have completed primary treatment will develop osteosarcoma recurrence. The Src pathway has been implicated in the metastatic behavior of osteosarcoma; about 95% of samples examined express Src or have evidence of downstream activation of this pathway. Saracatinib (AZD0530) is a potent and selective Src kinase inhibitor that was evaluated in adults in Phase 1 studies. The primary goal of this study was to determine if treatment with saracatinib could increase progression-free survival (PFS) for patients who have undergone complete resection of osteosarcoma lung metastases in a double-blinded, placebo-controlled trial. Patients and Methods. Subjects with recurrent osteosarcoma localized to lung and who had complete surgical removal of all lung nodules were randomized within six weeks after complete surgical resection. Saracatinib, or placebo, was administered at a dose of 175 mg orally, once daily, for up to thirteen 28-day cycles.ResultsThirty-seven subjects were included in the analyses; 18 subjects were randomized to receive saracatinib and 19 to receive placebo. Intent-to-treat analysis demonstrated a median PFS of 19.4 months in the saracatinib treatment group and 8.6 months in the placebo treatment group (p=0.47). Median OS was not reached in either arm.ConclusionsAlthough saracatinib was well tolerated in this patient population, there was no apparent impact of the drug in this double-blinded, placebo-controlled trial on OS, and Src inhibition alone may not be sufficient to suppress metastatic progression in osteosarcoma. There is a suggestion of potential clinical benefit as evidenced by longer PFS in patients randomized to saracatinib based on limited numbers of patients treated.

This cross-sectional study described prevalent body image (BI) concerns among adolescents and young adults (AYAs) with neoplasms who received treatment at a quaternary care children's hospital. Thirty-two AYAs, aged 15–39 years, completed questionnaires assessing BI within six months of diagnosis. The most frequently endorsed questionnaire items included the following: desire for increased physical fitness (62.5%), self-consciousness about hair (45.2%), weight dissatisfaction (40.6%), lack of strength (37.5%), wearing loose clothing to hide one's body (37.5%), decreased agility (34.4%), shape dissatisfaction (32.2%), and self-perception of too much body fat (31.3%). Awareness of AYA BI concerns during treatment may generate early intervention targeting this complex issue. [ABSTRACT FROM AUTHOR]

Small cell carcinoma of the ovary of hypercalcemic type (SCCOHT) is an extremely rare, aggressive cancer affecting children and young women. We identified germline and somatic inactivating mutations in the SWI/SNF chromatin-remodeling gene SMARCA4 in 75% (9/12) of SCCOHT cases in addition to SMARCA4 protein loss in 82% (14/17) of SCCOHT tumors but in only 0.4% (2/485) of other primary ovarian tumors. These data implicate SMARCA4 in SCCOHT oncogenesis.

Background: Technetium‐99 (99mTc) lymphoscintigraphy with blue dye injection is an accepted method for sentinel lymph node (SLN) mapping, but blue dye has known adverse effects, and injection of 99mTc may increase time under anesthesia for pediatric patients. Indocyanine green (ICG) may serve as an adjunct to assist with visibility and identification of SLNs. We hypothesized that sensitivity of ICG was similar to blue dye in SLN biopsies. Methods: Thirty patients (36 procedures with 96 total specimens) underwent preoperative intradermal injection of 99mTc, followed by intradermal injection of isosulfan blue and ICG. Test characteristics of blue dye, ICG, and 99mTc included sensitivity, specificity, positive predictive value (PPV), and negative predictive value (NPV). Results: ICG had a sensitivity of 87% and PPV of 83% for detection of 99mTc‐hot lymph nodes; blue dye had a sensitivity of 44% and PPV of 97%. For detection of pathologically confirmed lymph nodes, ICG had a sensitivity of 84% and a positive predictive value (PPV) of 91%. 99mTc had a sensitivity of 82% and a PPV of 94%. ICG had no significant difference in odds of being positive in pathology‐confirmed lymph nodes compared to 99mTc (odds ratio [OR], 0.818; 95% confidence interval [CI], 0.3–2.172; p =.823) and had higher odds than isosulfan blue (OR, 0.025, 95% CI, 0.001–0.148; p <.001). Conclusion: This study established the efficacy of ICG as an adjunct to SLNB in the pediatric and young adult population. ICG was safe, more efficacious than blue dye, and may obviate the need for lymphoscintigraphy in selected patients resulting in reduced time under anesthesia. This trial in the pediatric and young adult oncology population studied patients undergoing sentinel lymph node biopsy with the use of gold‐standard technetium‐99, isosulfan blue, and indocyanine green. Indocyanine green was found to be safe, more efficacious than blue dye, and noninferior to technetium‐99, potentially obviating the need for nuclear scanning in select patient populations, resulting in reduced time under anesthesia. [ABSTRACT FROM AUTHOR]