Extract: Type 1 diabetes (T1D) is an autoimmune disease in which insulin-producing beta-cells contained within the pancreatic islet of Langerhans are destroyed by autoreactive T cells. T1D patients are treated via insulin hormone replacement therapy by subcutaneous injection of recombinant insulin (produced by molecular engineering). Blood glucose levels must be monitored many times a day to determine the appropriate quantity of insulin to be injected in order to control blood glucose levels (glycemia). Under the insulin-based treatment, the large and sustained effort that a patient must make to strive for near optimal control of glycemia over many decades, frequently beginning in childhood, often make this approach impractical. As a result, T1D contributes substantially to the high rate of nephropathy, neuropathy, retinopathy, and generalized microvascular disease experienced by this population. Since insulin replacement therapy alone does not completely protect these individuals from severe complications, more appropriate treatments for curing T1D are needed. Transplantation of the whole pancreas or isolated pancreatic islets, have both been proposed in the aim of more effectively treating patients with complicated T1D. However, tempering the initial enthusiasm over transplantation has been the reported worsened survival rate for recipients of the pancreas alone, when compared with the survival of waiting-list patients receiving conventional insulin therapy, and the follow-up studies on islet recipients in which a gradual loss of islet function has been observed with time.
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Orphanet J Rare Dis
December 2024
Post Graduate School in Allergology and Internal Medicine "Guido Baccelli", Department of Precision and Regenerative Medicine and Ionian Area-(DiMePRe-J), School of Medicine, Aldo Moro University of Bari, Bari, 70124, Italy.
Background: Mucopolysaccharidosis (MPS) type 1 S and type 2 are rare lysosomal storage disorders characterized by impaired enzyme production, resulting in glycosaminoglycans accumulation within lysosomes. Enzyme Replacement Therapy (ERT) with laronidase and idursulfase are first line treatments, respectively. However, infusion-related hypersensitivity reactions (HR) may lead to ERT discontinuation.
View Article and Find Full Text PDFOrphanet J Rare Dis
December 2024
Department of Internal Medicine D, and Interdisciplinary Fabry Center (IFAZ), University Hospital Muenster, Muenster, Germany.
Background: The aim of our multicenter study was to investigate the implementation of the European Fabry guidelines on therapeutic recommendations in female patients with Fabry disease (FD) and to analyze the impact of enzyme replacement therapy (ERT) in treated and untreated females.
Results: Data from 3 consecutive visits of 159 female FD patients from 6 Fabry centers were retrospectively analyzed. According to their treatment, patients were separated in 3 groups (untreated, n = 71; newly ERT-treated, n = 47; long-term ERT-treated, n = 41).
BMC Musculoskelet Disord
December 2024
Department of Joint Surgery, The Affiliated Hospital of Qingdao University, No. 59, Haier Road, Laoshan District, Qingdao, 266100, Shandong, People's Republic of China.
Background: Venous thromboembolism (VTE) is a common complication after hip arthroplasty. Here, we investigated the clinical efficacy and safety of prophylactic aspirin vs. conventional therapy in hip arthroplasty for femoral neck fracture.
View Article and Find Full Text PDFBMC Anesthesiol
December 2024
Department of Anesthesiology, Clinical Oncology School of Fujian Medical University, Fujian Cancer Hospital, Fuzhou, 350014, Fujian Province, China.
Background: This study explores the correlation between nutritional status, as determined by the Geriatric Nutritional Risk Index (GNRI), and the incidence of postoperative delirium (POD) in patients undergoing gastric surgery.
Methods: Data were obtained from the MIMIC-IV 2.2 database for patients aged 18 years or older who underwent gastric surgery.
Oper Orthop Traumatol
December 2024
Department for Orthopaedic and Trauma Surgery, Lucerne Cantonal Hospital LUKS, Spitalstrasse, Lucerne, Switzerland.
Objective: To maximize local tumor control, stabilize affected bones, and preserve or replace joints with minimal interventional burden, thereby enhancing quality of life for empowered living.
Indications: Suitable for patients with bone metastases, particularly those with severe pain and/or fractures and appropriate life expectancy.
Contraindications: In primary bone tumors, refer to the sarcoma surgery team for evaluation of wide resection.
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