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Background & Aims: Clinical and radiologic variables associated with perianal fistula (PAF) outcomes are poorly understood. We developed prediction models for anti-tumor necrosis factor (TNF) treatment failure in patients with Crohn's disease-related PAF., Methods: In a multicenter retrospective study between 2005 and 2022 we included biologic-naive adults (>17 years) who initiated their first anti-TNF therapy for PAF after pelvic magnetic resonance imaging (MRI). Pretreatment MRI studies were prospectively reread centrally by blinded radiologists. We developed and internally validated a prediction model based on clinical and radiologic parameters to predict the likelihood of anti-TNF treatment failure, clinically, at 6 months. We compared our model and a simplified version of MRI parameters alone with existing imaging-based PAF activity indices (MAGNIFI-CD and modified Van Assche MRI scores) by De Long statistical test., Results: We included 221 patients: 32 ± 14 years, 60% males, 76% complex fistulas; 68% treated with infliximab and 32% treated with adalimumab. Treatment failure occurred in 102 (46%) patients. Our prediction model included age at PAF diagnosis, time to initiate anti-TNF treatment, and smoking and 8 MRI characteristics (supra/extrasphincteric anatomy, fistula length >4.3 cm, primary tracts >1, secondary tracts >1, external openings >1, tract hyperintensity on T1-weighted imaging, horseshoe anatomy, and collections >1.3 cm). Our full and simplified MRI models had fair discriminatory capacity for anti-TNF treatment failure (concordance statistic, 0.67 and 0.65, respectively) and outperformed MAGNIFI-CD (P = .002 and < .0005) and modified Van Assche MRI scores (P < .0001 and < .0001), respectively., Conclusions: Our risk prediction models consisting of clinical and/or radiologic variables accurately predict treatment failure in patients with PAF., (Copyright © 2024 AGA Institute. Published by Elsevier Inc. All rights reserved.)

Objective/hypothesis: To review the imaging findings of laryngeal amyloidosis and to identify radiological findings suggestive of this disease., Study Design: Retrospective case series., Methods: A retrospective chart review of patients with pathologically confirmed laryngeal amyloidosis was performed from 2009 to 2022. Clinical and demographic factors were collected. A fellowship-trained head and neck radiologist reviewed all computed tomography (CT) scans and magnetic resonance imaging (MRI) findings within this cohort., Results: 12 patients were identified and a total of 36 imaging studies analyzed. Localized amyloidosis was found in the supraglottic region (n = 6), glottic region (n = 7), and subglottic region (n = 5); six patients had disease spanning two subsites. The most common finding on the CT scan was a homogeneous and well-defined submucosal soft tissue mass. Punctate calcifications were present in three cases. The presence of contrast enhancement was identified in the majority of patients who underwent MRI (4/5). MRI showed consistent signal intensity, hypointense, or isointense on both T1-weighted and T2-weighted images. Diffusion-weighted sequences were obtained in every patient and did not demonstrate diffusion restriction., Conclusion: This is the largest series searching for unifying imaging characteristics of laryngeal amyloidosis. This research suggests that characteristics from CT and MR provide both similar and unique features of laryngeal amyloidosis on imaging. Both modalities identify a submucosal mass. CT is the preferred modality to demonstrate punctate calcifications, while MRI identifies enhancement and altered signal characteristics. The main benefit of serial imaging is the correlation with patient symptoms, identification of the extent of disease, and assisting in delineating appropriate timing for surgery., Competing Interests: Declaration of Competing Interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Dr Karen M. Kost discloses she is a consultant for Pentax Medical., (Copyright © 2023. Published by Elsevier Inc.)

Background: Patients with HER2+ breast cancer (BC) frequently develop leptomeningeal metastases (LM). While HER2-targeted therapies have demonstrated efficacy in the neoadjuvant, adjuvant, and metastatic settings, including for parenchymal brain metastases, their efficacy for patients with LM has not been studied in a randomized controlled trial. However, several single-armed prospective studies, case series and case reports have studied oral, intravenous, or intrathecally administered HER2-targeted therapy regimens for patients with HER2+ BC LM., Methods: We conducted a systematic review and meta-analysis of individual patient data to evaluate the efficacy of HER2-targeted therapies in HER2+ BC LM in accordance with PRISMA guidelines. Targeted therapies evaluated were trastuzumab (intrathecal or intravenous), pertuzumab, lapatinib, neratinib, tucatinib, trastuzumab-emtansine and trastuzumab-deruxtecan. The primary endpoint was overall survival (OS), with CNS-specific progression-free survival (PFS) as a secondary endpoint., Results: 7780 abstracts were screened, identifying 45 publications with 208 patients, corresponding to 275 lines of HER2-targeted therapy for BC LM which met inclusion criteria. In univariable and multivariable analyses, we observed no significant difference in OS and CNS-specific PFS between intrathecal trastuzumab compared to oral or intravenous administration of HER2-targeted therapy. Anti-HER2 monoclonal antibody-based regimens did not demonstrate superiority over HER2 tyrosine kinase inhibitors. In a cohort of 15 patients, treatment with trastuzumab-deruxtecan was associated with prolonged OS compared to other HER2-targeted therapies and compared to trastuzumab-emtansine., Conclusions: The results of this meta-analysis, comprising the limited data available, suggest that intrathecal administration of HER2-targeted therapy for patients with HER2+ BC LM confers no additional benefit over oral and/or IV treatment regimens. Although the number of patients receiving trastuzumab deruxtecan in this cohort is small, this novel agent offers promise for this patient population and requires further investigation in prospective studies., Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2023 The Authors. Published by Elsevier Ltd.. All rights reserved.)

Brain metastases (BM) are associated with significant morbidity and mortality in patients with advanced cancer. Despite significant advances in surgical, radiation, and systemic therapy in recent years, the median overall survival of patients with BM is less than 1 year. The acquisition of medical images, such as computed tomography (CT) and magnetic resonance imaging (MRI), is critical for the diagnosis and stratification of patients to appropriate treatments. Radiomic analyses have the potential to improve the standard of care for patients with BM by applying artificial intelligence (AI) with already acquired medical images to predict clinical outcomes and direct the personalized care of BM patients. Herein, we outline the existing literature applying radiomics for the clinical management of BM. This includes predicting patient response to radiotherapy and identifying radiation necrosis, performing virtual biopsies to predict tumor mutation status, and determining the cancer of origin in brain tumors identified via imaging. With further development, radiomics has the potential to aid in BM patient stratification while circumventing the need for invasive tissue sampling, particularly for patients not eligible for surgical resection., (© The Author(s) 2022. Published by Oxford University Press, the Society for Neuro-Oncology and the European Association of Neuro-Oncology.)

Background: There is increasing evidence for the use of multilevel epidural catheter-guided blood patches to treat spontaneous cerebrospinal fluid leaks in adults. Yet, there are scarce data for their use in children. Furthermore, higher level epidural blood patches are uncommon in both adult and pediatric populations., Case Presentation: An adolescent boy with multilevel cerebrospinal fluid leaks associated with status migrainosus failed conservative pain management treatment. As he remained severely symptomatic, epidural blood patches were required to mitigate his symptoms. Given his multilevel leaks, a catheter-guided blood patch approach was used to treat multiple cervicothoracic and thoracolumbar leaks. After three separate uneventful blood patch procedures, the patient was free of symptoms. Other than some rebound intracranial hypertension, the patient maintained full neurological capacity without further complications. Following the resolution of treatment, he has not sought help for pain since his last blood patch over a year ago., Conclusions: Multilevel catheter-guided blood patches have the potential to be administered to higher level spinal regions to the pediatric population suffering from multiple spontaneous cerebrospinal fluid leaks., Competing Interests: Competing interests: None declared., (© American Society of Regional Anesthesia & Pain Medicine 2022. No commercial re-use. See rights and permissions. Published by BMJ.)

Background and Aims: Homozygous familial hypercholesterolemia (HoFH) is an orphan disease, most often caused by bi-allelic mutations of the LDLR gene. Patients with HoFH have elevated LDL-C levels >13 mmol/L, tendinous xanthomata and severe, premature atherosclerotic cardiovascular disease (ASCVD). Untreated, most HoFH patients die of ASCVD in youth. New therapeutic modalities include lomitapide, an inhibitor of microsomal triglyceride transfer protein that lowers hepatic LDL-C production. We have recently identified 79 Canadian patients with HoFH. Here, we describe our experience with lomitapide in the province of Quebec, a geographic area known to have a high prevalence of HoFH., Methods: This is a retrospective case series of 12 HoFH patients followed at three lipidology centers in the province of Quebec., Results: Mean age of the patients was 44 ± 18 years; age at time of HoFH diagnosis ranged from 2 to 59 years. All patients were on a statin and ezetimibe 10 mg/day and five patients were treated with LDL apheresis. Treatment with lomitapide reduced LDL-C levels by 38% (intention-to-treat). Intolerable gastrointestinal side effects were observed in 3/12 patients and were the main reason for treatment discontinuation. Three patients tolerated lomitapide at doses ranging between 5 and 30 mg/day without major side effects. Downwards drug titration was necessary in the 6 remaining patients because of gastrointestinal side effects (n = 5) and elevated liver enzymes (n = 1), and 2 of them finally discontinued treatment., Conclusions: Lomitapide may be used to further decrease LDL-C in HoFH patients; gastrointestinal side effects and hepatic toxicity may limit adherence., (Copyright © 2020 Elsevier B.V. All rights reserved.)