Current therapeutic approaches in the management of hemophilia—a consensus view by the Romanian Society of Hematology

Abstract

Hemophilia A (HA) and hemophilia B (HB) are rare disorders, being caused by the total lack or under-expression of two factors from the coagulation cascade coded by genes of the X chromosome. Thus, in hemophilic patients, the blood does not clot properly. This results in spontaneous bleeding episodes after an injury or surgical intervention. A patient-centered regimen is considered optimal. Age, pharmacokinetics, bleeding phenotype, joint status, adherence, physical activity, personal goals are all factors that should be considered when individualizing therapy. In the past 10 years, many innovations in the diagnostic and treatment options were presented as being either approved or in development, thus helping clinicians to improve the standard-of-care for patients with hemophilia. Recombinant factors still remain the standard of care in hemophilia, however they pose a challenge to treatment adherence because they have short halflife, which where the extended half-life (EHL) factors come with the solution, increasing the half-life to 96 hours. Gene therapies have a promising future with proven beneficial effects in clinical trials. We present and critically analyze in the current manuscript the pros and cons of all the major discoveries in the diagnosis and treatment of HA and HB, as well as identify key areas of hemophilia research where improvements are needed.

Management of pregnant female with Haemophilia-A: A case report

“…Mechanical thromboprophylaxis is sufficient for carriers undergoing surgeries, thromboprophylaxis with LMWH is not recommended. [ 9 ] There is no contraindication to regional analgesia (effective method for labour analgesia) or anaesthesia, when the FVIII concentration are ≥50 IU/dl. Regional anaesthesia can be given safely after correction of FVIII level, Platelets (>80,000) and normal range INR (<1.5) for caesarean delivery.…”

Hemophilic Arthropathy: Barriers to Early Diagnosis and Management

“…Both types of hemophilia can be classified according to the plasma level of existing clotting factor: mild (5-40%), moderate (1-5%) or severe (<1%). 3 The severity of bleeding in patients with hemophilia is proportional to the amount of residual factor activity in the blood. In patients with severe hemophilia, bleeding may occur spontaneously or with minor trauma.…”

“…1 However, most patients with hemophilia worldwide can only access on-demand pharmacological treatment once bleeding has happened, with the consequent risk of joint damage. 3 The main complication related to the treatment of hemophilia is the development of antibodies to clotting factor concentrates. These inhibitors react against the administered substitution factor, increasing the risk of serious bleeding and accelerating the degenerative damage caused by the recurrence of hemarthrosis.…”

Expert Opinions on the Management of Hemophilia A in India: The Role of Emicizumab

“…The WFH recommends beginning with HA prophylaxis early in life. There are three types of prophylaxis based on when it is initiated and the treatment goals: primary, secondary, and tertiary [ 9 ]. Prophylaxis provides sustained hemostatic control, maintaining clotting factor levels within the therapeutic range, for preventing spontaneous and unpredictable bleeding in hemophilia.…”