A Case of Acquired Hemophilia A in a Patient with Exposure to COVID-19.

Acquired Hemophilia A (AHA) is a very rare autoimmune condition involving immune-mediated depletion of Factor VIII, resulting in spontaneous hemorrhage. Failure to recognize AHA as a possible etiology of hemorrhage can result in delayed diagnosis and treatment. The COVID-19 pandemic has given rise to several hematologic conditions and complications, with a rare manifestation being Acquired Hemophilia A (AHA).

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

Background: In patients with severe hemophilia B, gene therapy that is mediated by a novel self-complementary adeno-associated virus serotype 8 (AAV8) vector has been shown to raise factor IX levels for periods of up to 16 months. We wanted to determine the durability of transgene expression, the vector dose-response relationship, and the level of persistent or late toxicity.

Methods: We evaluated the stability of transgene expression and long-term safety in 10 patients with severe hemophilia B: 6 patients who had been enrolled in an initial phase 1 dose-escalation trial, with 2 patients each receiving a low, intermediate, or high dose, and 4 additional patients who received the high dose (2×10(12) vector genomes per kilogram of body weight).

Newly diagnosed congenital factor VII deficiency and utilization of recombinant activated factor VII (NovoSeven(®)).

Unlabelled: This case report presents a newly diagnosed congenital factor VII deficiency treated with recombinant activated factor VII (rFVIIa). Congenital factor VII deficiency is a rare autosomal-recessive bleeding disorder that occurs in fewer than 1/500,000 persons. Its presentation can vary from epistaxis to hemarthroses and severe central nervous system bleeding, and correlates poorly with factor VII levels.

Reduced dose maintenance eculizumab in atypical hemolytic uremic syndrome (aHUS): an update on a previous case report.

Objective: To describe how maintenance eculizumab sustains improved renal function in severe atypical hemolytic uremic syndrome (aHUS).

Case Report: A previously described 50-year-old woman with aHUS had a remarkable recovery with eculizumab, which safely reversed profound neurologic damage and eliminated the need for dialysis. Her recovery has been sustained on long-term eculizumab treatment.

Eculizumab safely reverses neurologic impairment and eliminates need for dialysis in severe atypical hemolytic uremic syndrome.

This case report describes how eculizumab reversed neurologic impairment and improved renal damage in severe atypical hemolytic uremic syndrome. A 50-year-old female, after presenting with diarrhea and abdominal pain, developed pancolitis, acute renal failure, and thrombocytopenia. The patient underwent total abdominal colectomy.