Publications by authors named "R de Cristofaro"

Article Synopsis
  • iTTP is a serious blood disorder caused by antibodies that affect clotting, and caplacizumab is a new treatment approved for acute cases, used alongside plasma exchange and immunosuppression.
  • A study involving 38 iTTP patients across six Italian medical centers showed that caplacizumab led to rapid normalization of platelet counts, with a median recovery time of just 2 days.
  • The use of caplacizumab resulted in fewer exacerbations and relapses, shorter hospital stays, and no severe side effects, highlighting its effectiveness compared to traditional treatments.
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There is a need to address the evidence gap regarding the in-hospital administration of sacubitril/valsartan in acute myocardial infarction patients. After searching MEDLINE, Google Scholars and Scopus, a random-effects meta-analysis of randomized controlled trials comparing the in-hospital administration of the angiotensin receptor-neprilysin inhibitors (ARNis) versus the standard therapy in patients with reduced heart failure due to myocardial infarction was performed. The primary outcome was major adverse cardiovascular events.

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Haemophilia is associated with reduced bone mass and mineral density. Due to the rarity of the disease and the heterogeneity among the studies, the pathogenesis of bone loss is still under investigation. We studied the effects of coagulation factors on bone cells and characterized in a pilot study the osteoclastogenic potential of patients' osteoclast precursors.

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Adeno-associated virus-based gene therapy (valoctocogene roxaparvovec) is an attractive treatment for hemophilia A. Careful clinical management is required to minimize the risk of hepatotoxicity, including assessment of baseline liver condition to determine treatment eligibility and monitoring liver function after gene therapy. This article describes recommendations (developed by a group of hemophilia experts) on hepatic function monitoring before and after gene therapy.

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Article Synopsis
  • - Plasma-derived von Willebrand factor-containing factor VIII concentrates (pd-VWF/FVIII-C), specifically Fanhdi® and Alphanate®, are commonly used to treat von Willebrand disease (VWD) in Italy, and a study evaluated their real-world effectiveness and safety.
  • - The retrospective study involved 57 patients with different types of VWD, treating various bleeding episodes, surgical procedures, and long-term prophylaxis over a period from 2007 to 2019.
  • - Results showed that pd-VWF/FVIII-C had excellent or good efficacy in 85% of bleeding episodes, 98% of surgeries, and 100% of long-term prophylaxis cases, with no serious safety concerns reported. *
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