0000000000083407
AUTHOR
Maria Francesca Mansueto
Acquired Hemophilia A Associated with Venous Thrombosis and Very High Inhibitor Titer: A Challenging Scenario
Combined Point of Care Tools Are Able to Improve Treatment Adherence and Health-Related Quality of Life in Patients with Severe Hemophilia: An Observational Prospective Study
Introduction: Ultrasound (US) assessment of joints is an evolving point of care tool for the detection of early joint arthropathy (Napolitano M, Kessler CM. Hemophilia A and B. Consultative Hemostasis and Thrombosis, Kitchens, 4th edition); population pharmacokinetic (pop-PK) studies are adopted as a useful instrument to set the prophylaxis regimen for patients with hemophilia, they may improve adherence (Nagao A.et al. Thromb Res. 2019 Jan; 173:79-84) and reduce the annual bleeding rate (ABR). Adherence to continuous intravenous administrations of factor VIII or Factor IX products is challenging, thus patients may experience breakthrough bleedings while on prophylaxis. Repeated US examinat…
Laying the foundations for gene therapy in Italy for patients with haemophilia A: A Delphi consensus study
IntroductionCurrent treatment for haemophilia A involves factor VIII replacement or non-replacement (emicizumab) therapies, neither of which permanently normalise factor VIII levels. Gene therapy using adeno-associated viral (AAV) vectors is an emerging long-term treatment strategy for people with severe haemophilia A (PwSHA) that is likely to be available for clinical use in the near future. AimThis article proposes practical guidelines for the assessment, treatment, and follow-up of potential PwSHA candidates for AAV-based gene therapy. MethodUsing the Delphi method, a working group of Italian stakeholders with expertise in and knowledge of the care of adults with haemophilia A analysed l…
Personalized prophylaxis with simoctocog-alfa(human-cl rhFVIII):A real-life experience on children and moderate haemophilia A patients
Tailoring haemophilia A prophylaxis with BAY 81-8973: A case series
BAY 81-8973 is an unmodified, full-length third generation recombinant factor VIII (rFVIII) which offers a more favorable pharmacokinetic (PK) profile, compared to its predecessor sucrose-formulated rFVIII (rFVIII-FS). We here report on a retrospective case series of nine patients affected by hemophilia A (HA), with variable disease severity, bleeding phenotype and comorbidities, to underline our clinical practice on prophylaxis with a recently introduced standard hall-life recombinant Factor VIII. The current case series highlights how the current clinical management of hemophilia is able to personalize treatment in several specific conditions like concomitant illnesses with thrombotic ris…
Emotions and Opinions of Adult Patients with Haemophilia During the COVID-19 (Coronavirus Disease 2019) Pandemic Caused by SARS-CoV-2: A Monocentric Survey
Mariasanta Napolitano, 1 Maria Francesca Mansueto, 1 Davide Sirocchi, 1 Angelo Davide Contrino, 1 Simona Raso, 2 Sergio Siragusa 1 1Department of Health Promotion, Mother and Child Care, Internal Medicine and Medical Specialties (PROMISE), Haematology Unit, Reference Regional Centre for Thrombosis and Haemostasis, University of Palermo, Palermo, Italy; 2Division of Haematology, Department of Surgical, Oncological and Stomatological Disciplines (Di.Chir.On.S.), AOUP Paolo Giaccone, Palermo, ItalyCorrespondence: Mariasanta NapolitanoDepartment of Health Promotion, Mother and Child Care, Internal Medicine and Medical Specialties (PROMISE), Haematology Unit, Reference Regional Centre for Thromb…
Quality of Life in Patients With Cancer Under Prolonged Anticoagulation for High-Risk Deep Vein Thrombosis: a Long-Term Follow-Up
Current guidelines recommend to prolong anticoagulant treatment in patients with cancer with venous thromboembolism (VTE); only few studies evaluated other parameters than cancer itself for selecting patients at higher risk of recurrent VTE. Long-term management of VTE is thus challenged by several controversies mainly for patients compliance. We here report results of a long-term follow-up in patients with deep vein thrombosis under anticoagulant treatment with low-molecular-weight heparin (LMWH) for residual vein thrombosis (RVT) detected at compression ultrasonography (CUS), 6 months after standard anticoagulant treatment. Patients with RVT were deemed at high risk of recurrences and in…