Diagnosis and Treatment of Hemophilia
A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Hematology".
Deadline for manuscript submissions: closed (31 March 2021) | Viewed by 2916
Special Issue Editors
2. Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy
Interests: rare bleeding disorders; hemophilia A and B; patient treatment; inhibitor complication; diagnosis; novel treatment; gene therapy; thrombotic thrombocytopenic purpura (TTP); ADAMTS13
Interests: hemophilia A and B; genetic diagnosis; invasive and non-invasive prenatal diagnosis; preimplantation genetic testing; inhibitor; gene therapy
Special Issue Information
Dear Colleagues,
Over the past 30 years, the diagnosis and treatment of patients with hemophilia A and B has improved considerably. In recent years, the implementation of new technologies, such as next-generation sequencing or droplet PCR, has modernized the detection of genetic defects in hemophilia patients and in female relatives who might be carriers. Prenatal diagnosis has also significantly progressed during the last two decades. Non-invasive methods have replaced invasive procedures, and preimplantation genetic testing (PGT) has become an important complement to the approaches currently available in the prevention of genetic diseases.
At present, patients with hemophilia A and B can choose different effective and safe treatments, including novel extended-half-life products, non-replacement therapies, and even gene therapy. The shift from standard FVIII and FIX concentrates to the extended-half-life products has significantly changed the management of hemophilia patients, making treatment less distressing and improving their quality of life. Subcutaneous administration of non-replacement therapies such as emicizumab have simplified prophylaxis in patients with hemophilia A with and without inhibitors. In addition, gene therapy is becoming a promising therapeutic choice.
With this background, this Special Issue will try to highlight how new diagnostic and therapeutic approaches have advanced to improve patients' quality of life.
Prof. Flora Peyvandi
Dr. Isabella Garagiola
Guest Editors
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Keywords
- coagulation assays
- global assays
- genetic testing in hemophilia A and B
- invasive and non-invasive procedures in prenatal diagnosis
- preimplantation genetic testing in hemophilia
- new treatment strategies (extended-half-life treatment and new non-replacement therapy)
- gene therapy
- safety concerns and surveillance
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