Innovative Therapies for Acquired Hemophilia A

Abstract

Acquired hemophilia A (AHA) is a rare autoimmune bleeding disorder which can be life-threatening. AHA is due to autoantibodies against coagulation factor VIII. Disease onset may be idiopathic (approximately half of the cases) or triggered by autoimmune disorders, cancers, drugs, infections, or pregnancy. Besides treating the underlying disorder, specific AHA treatments include management of bleeding and inhibitor eradication. Various first-line and second-line hemostatic and immunosuppressive agents are currently available for the management of AHA. Recently, the hemostatic drug emicizumab and the immunosuppressive drug rituximab have been the object of intense research from investigators as innovative promising therapies for AHA. This narrative review will be focused on the current status of the clinical use of these two off-label therapeutic agents in AHA.

Genetics and Epigenetics in Acquired Hemophilia A: From Bench to Bedside

“…When rpFVIII is chosen as the initial treatment, a dose of 200 U/kg, followed by additional doses to sustain the levels of FVIII > 50% through the treatment, is used [87]. Studies are suggesting that, during therapy with rpFVIII, the activity of FVIII should be monitored [87,99]. Furthermore, de novo anti-rpFVIII inhibitors can be developed in these patients, leading to discontinuation of the treatment [100,101].…”

“…Plasma-deprived FVIIa/FX has been also evaluated in patients with AHA, with favorable outcomes without an increase in the thrombotic risk [ 94 ]. Emicizumab, a monoclonal antibody restoring the function of missing FVIII by connecting FIXa and FX, has been used off-label in AHA [ 95 , 96 , 97 , 98 , 99 ]. Figure 2 presents the initial clinical administration of the patient with AHA in the emergency room department.…”