A 64-year-old woman with hereditary hemorrhagic telangiectasia (HHT) characterized by a pathological variant in ACVRL1 presents to the clinic for follow-up. Manifestations of HHT include frequent epistaxis and gastrointestinal bleeding, leading to iron-deficiency anemia. Bevacizumab is initiated, with resolution of the anemia. While maintained on a regimen of bevacizumab every 6 weeks, she continues to report frequent epistaxis and has ongoing iron-deficiency requiring periodic iron infusions. She also finds the bevacizumab infusions inconvenient. She is interested in discussing other options for managing her disease.
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http://dx.doi.org/10.1182/hematology.2024000675 | DOI Listing |
Breast J
January 2025
Department of Chemotherapy Jiangsu Cancer Hospital Jiangsu Institute of Cancer Research The Affiliated Cancer Hospital of Nanjing Medical University, Nanjing, China.
Purpose: Endocrine therapy combined with cyclin-dependent kinase (CDK) 4/6 inhibitors (CDK4/6i) is the preferred treatment for hormone receptor-positive (HR+)/human epidermal growth factor receptor 2-negative (HER2-) metastatic breast cancer (MBC). However, there are currently no recommendations for therapeutic strategies after progression on CDK4/6i-based treatment. This study aimed to examine the efficacy and safety of anlotinib plus chemotherapy in HR+/HER2- MBC after progression on CDK4/6 inhibitors.
View Article and Find Full Text PDFSemin Thromb Hemost
December 2024
Gastroenterology Department, HHT European Reference Center, ASST Ospedale Maggiore Crema, Crema, Italy.
Hereditary hemorrhagic telangiectasia (HHT) is an inherited vascular disorder with highly variable penetrance, affecting up to 1 in 5,000 individuals. It is characterized by the presence of abnormal blood vessels that can lead to excessive bleeding-most frequently recurrent nosebleeds (epistaxis), skin and mucosal telangiectasias (small, dilated blood vessels), as well as arteriovenous malformations (AVMs) that can form in various organs, particularly the lungs, liver, and brain. HHT is caused by loss-of-function mutations in the BMP9-10/ENG/ALK1/SMAD4 signaling pathway, an important mediator of vascular quiescence.
View Article and Find Full Text PDFClin Appl Thromb Hemost
December 2024
Department of Pediatrics, Emory University School of Medicine, Atlanta, GA, USA.
Background: Prophylaxis with a von Willebrand factor (VWF) concentrate is recommended in von Willebrand disease (VWD) patients with a history of frequent and severe bleeds. Despite nosebleeds being a frequent manifestation of VWD, few studies have investigated the efficacy of factor prophylaxis in preventing nosebleeds in patients with severe VWD.
Methods: This post-hoc analysis of a prospective, 12-month, phase 3 study assessed the efficacy of wilate in the prevention of nosebleeds in 33 patients aged ≥6 years with severe type 1, type 2 or type 3 VWD.
Neurocirugia (Astur : Engl Ed)
December 2024
Departamento de Neurocirugía, Fundación Oftalmológica de Santander - Clínica Ardila Lülle, Floridablanca, Colombia. Electronic address:
The olfactory neuroblastoma is a rare malignant neoplasm derived from the olfactory neuroepithelium. It can metastasize to cervical lymph node chains and distant organs through hematogenous or lymphatic routes. Two clinical cases are presented: the first, a 56-year-old man with no pathological history, exhibited symptoms evolving over 2 months, characterized by persistent rhinorrhea with frequent epistaxis, ipsilateral proptosis, left hemicranial pain, anosmia, and dysgeusia.
View Article and Find Full Text PDFHematology Am Soc Hematol Educ Program
December 2024
Department of Medicine, Division of Hematology, University of North Carolina School of Medicine, Chapel Hill, NC.
A 64-year-old woman with hereditary hemorrhagic telangiectasia (HHT) characterized by a pathological variant in ACVRL1 presents to the clinic for follow-up. Manifestations of HHT include frequent epistaxis and gastrointestinal bleeding, leading to iron-deficiency anemia. Bevacizumab is initiated, with resolution of the anemia.
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