Publications by authors named "Ambra Corti"

Article Synopsis
  • Mucopolysaccharidosis type I Hurler (MPSIH) causes severe skeletal issues that aren't fully improved by traditional stem cell transplants (HSCT), but autologous gene therapy (HSPC-GT) shows promise for better metabolic outcomes.
  • In a clinical trial with eight young patients (average age 1.9 years), those who underwent HSPC-GT were evaluated over four years for various growth and skeletal measures, showing significant improvements compared to those treated with HSCT.
  • After nearly four years, HSPC-GT patients demonstrated better growth, joint mobility, and reduced signs of hip dysplasia, indicating early positive impacts on skeletal health, but more long-term data is needed for con
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Adenosine deaminase (ADA) deficiency leads to severe combined immunodeficiency (SCID). Previous clinical trials showed that autologous CD34 cell gene therapy (GT) following busulfan reduced-intensity conditioning is a promising therapeutic approach for ADA-SCID, but long-term data are warranted. Here we report an analysis on long-term safety and efficacy data of 43 patients with ADA-SCID who received retroviral ex vivo bone marrow-derived hematopoietic stem cell GT.

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Background: Effective treatment for metachromatic leukodystrophy (MLD) remains a substantial unmet medical need. In this study we investigated the safety and efficacy of atidarsagene autotemcel (arsa-cel) in patients with MLD.

Methods: This study is an integrated analysis of results from a prospective, non-randomised, phase 1/2 clinical study and expanded-access frameworks.

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Background: Allogeneic hematopoietic stem-cell transplantation is the standard of care for Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). However, this treatment is only partially curative and is associated with complications.

Methods: We are conducting an ongoing study involving eight children with MPSIH.

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Article Synopsis
  • The study highlights the physical and psychological challenges faced by breast cancer patients that necessitate individualized rehabilitation programs post-treatment.
  • The analysis of 5,538 women in Italy revealed that a significant majority (81.7%) received outpatient care, primarily focused on pathology-related interventions, while only a small fraction engaged in inpatient or intensive outpatient programs.
  • The findings indicate a gap in adequate rehabilitation services, particularly concerning psychosocial and cognitive support, suggesting a need for improved access and resources for breast cancer patients.
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Microenvironment-related immune and inflammatory markers, when combined with established Ki-67 and morphology parameters, can improve prognostic prediction in gastro-entero-pancreatic neuroendocrine neoplasms (GEP-NENs). Therefore, we evaluated the prognostic value of microenvironment and tumor inflammatory features (MoTIFs) in GEP-NENs. For this purpose, formalin-fixed paraffin-embedded tissue sections from 350 patients were profiled by immunohistochemistry for immune, inflammatory, angiogenesis, proliferation, NEN-, and fibroblast-related markers.

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Background: In this pilot non-interference clinical study we evaluated possible interactions between Quercetin Phytosome® (Quercefit™), an innovative delivery form of quercetin, and antiplatelet agents, anticoagulants, and anti-diabetic therapy in otherwise healthy subjects.

Methods: Interaction with antiplatelet therapy was assessed through the bleeding time (BT) test in 30 patients treated with acetylsalicylic acid, ticlopidine or clopidogrel before and after 10 days of supplementation with Quercetin Phytosome®. Interaction with anticoagulants was evaluated by measuring the International Normalized Ratio (INR) in 20 patients using warfarin or dabigatran before and after 20 days of supplementation with Quercetin Phytosome®.

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Advanced Merkel cell carcinoma (MCC) is a very aggressive, rare neuroendocrine tumor of the skin with a high frequency of locoregional recurrence and metastasis, and a high mortality rate. Surgical resection, sentinel lymph node biopsy, and radiotherapy represent the gold standard of treatment in patients with localized disease, while chemotherapy has a significant role in the treatment of advanced disease. However, no definitive evidence on the survival impact of radiotherapy in the advanced stages has been provided to date, and response to chemotherapy remains brief in the majority of cases, indicating an urgent need for alternative approaches.

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Background: Strimvelis (autologous CD34+ cells transduced to express adenosine deaminase [ADA]) is the first ex vivo stem cell gene therapy approved by the European Medicines Agency (EMA), indicated as a single treatment for patients with ADA-severe combined immunodeficiency (ADA-SCID) who lack a suitable matched related bone marrow donor. Existing primary immunodeficiency registries are tailored to transplantation outcomes and do not capture the breadth of safety and efficacy endpoints required by the EMA for the long-term monitoring of gene therapies. Furthermore, for extended monitoring of Strimvelis, the young age of children treated, small patient numbers, and broad geographic distribution of patients all increase the risk of loss to follow-up before sufficient data have been collected.

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Background: Curcumin is a powerful anti-oxidant that can be used to treat inflammation and pain in chronic conditions such as osteoarthrosis (OA). Phytoproflex® is characterized by an innovative delivery system that improves bioavailability of curcuminoids and could be useful in the management of OA.

Methods: This 4-week registry included 56 patients with knee OA treated according to the best standard management for symptomatic OA.

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Curative treatments, including liver transplantation, surgical resection and percutaneous treatments, are the recommended therapies in BCLC-0 (Barcelona Clinic of Liver Cancer) or BCLC-A hepatocellular carcinoma (HCC). This review provides an overview of some issues of clinical importance concerning curative treatments in HCC.

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In the last decade trans-arterial radioembolization has given promising results in the treatment of patients with intermediate or advanced stage hepatocellular carcinoma (HCC), both in terms of disease control and tolerability profile. This technique consists of the selective intra-arterial administration of microspheres loaded with a radioactive compound (usually Yttrium(90)), and exerts its therapeutic effect through the radiation carried by these microspheres. A careful and meticulous selection of patients is crucial before performing the radioembolization to correctly perform the procedure and reduce the incidence of complications.

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