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.

Potentiation of renal tumorigenicity by cyclosporine A in streptozotocin diabetic rats.

Male Wistar rats with streptozotocin induced diabetes were treated every third day with 10 mg/kg (13 rats) of cyclosporine A (CyA) for 20 weeks. At sacrifice, 7 of 13 rats (53.8%) demonstrated renal tumors.

A rare restriction enzyme site polymorphism in the breakpoint cluster region (bcr) of chromosome 22.

Detection of rearrangement of the breakpoint cluster region (bcr) of chromosome 22 by Southern blot analysis can be used for the routine diagnosis of CML. Restriction fragment length polymorphisms (RFLPs) in the bcr can potentially be confused with translocation since both alter the size of DNA fragments obtained. By digesting DNA with the restriction enzyme BamHl and analyzing with probes commonly used for identifying rearrangement of the bcr, we have observed a RFLP within the bcr.

Long-term marrow cultures from mice with busulfan-induced chronic latent aplasia.

Mice treated with busulfan develop chronic latent marrow aplasia characterized by near-normal peripheral leukocyte counts, hematocrits, and marrow cellularity but very reduced stem cell (CFU-S) and progenitor cell (CFU-GM) populations. To determine whether the lesion is primarily in the stem cells or whether there is also a microenvironmental component we have used the Dexter-type long-term marrow cultures. Normal marrow maintains hemopoiesis for several weeks to months in such cultures, whereas "busulfan-marrow" will not support long-term growth.

Granulopoiesis in long-term culture by marrow from mice with busulfan-induced chronic latent aplasia.

Mice given high-dose busulfan therapy develop a chronic latent marrow aplasia characterized by normal peripheral blood neutrophil numbers, hematocrits and marrow cellularity but reduced numbers of pluripotent hemopoietic stem cells (CFU-s) and granulocyte-monocyte progenitor cells (CFU-gm). To study the pathogenesis of this lesion, bone marrow was propagated in long-term marrow cultures (LTMC). Small amounts of normal marrow readily established and sustained long-term granulopoiesis in vitro.