Hemophilia moderate

Deficiency of the VIIFC portion of the Factor VIII complex results in classic hemophilia or hemophilia A and is inherited as a sex-linked recessive disorder. Based on the degree of deficiency of the VIIFC molecule three different forms of hemophilia A are recognized. Less than 1% VIIFC activity equals severe hemophilia A. Two to 10% of normal VIIFC activity equals moderately severe hemophilia A. Ten to 25% of normal VIIFC activity equals minimal symptomatic disease. Deficiency of the VIIFvWFAg portion of the Factor VIII complex results in von WiUebrand disease. There are at present five principal types of von WiUebrand disease and numerous subtypes or variants. For the most part, von WiUebrand disease is inherited as an autosomal dominant, and a few subtypes may be inherited as an autosomal recessive trait. 

O Intravenous factor replacement with recombinant or plasma-derived products to treat or prevent bleeding is the primary treatment hemophilia. Primary prophylaxis is defined as the regular administration of factor concentrates with the intention of preventing joint bleeds.4 The rationale for primary prophylaxis is that individuals with factor levels of greater than 0.02 unit/mL (2 IU/dL) rarely suffer from spontaneous bleeds and arthropathy. Therefore, to maintain a trough level above this might convert severe hemophilia to moderate disease, with the abolition of joint bleeds and the associated arthropathy.5... 

Rash Mild to moderate rashes including urticarial rash, maculopapular rash, and possible photosensitivity have been reported in subjects receiving tipranavir/ritonavir. Hemophilia There have been reports of increased bleeding, including spontaneous skin hematomas and hemarthrosis in patients with hemophilia type A and B treated with Pis. 

At higher doses desmopressin also has significant hematological effects and can significantly boost concentrations of factor VIII and von Willebrand factor (VWF) in the blood. Desmopressin is therefore a valuable agent for the treatment of mild and moderate hemophilia A (congenital or acquired) and type 1 von Willebrand disease, in which the VWF protein structure is normal but... 

Gill JC, Ottum M, Schwartz B. Evaluation of high concentration intranasal and intravenous desmopressin in pediatric patients with mild hemophilia A or mild-to-moderate type 1 von Willebrand disease. J Pediatr 2002 140(5) 595-9. 

Schwarzinger I, Pabinger I, Korninger C, et al. Incidence of inhibitors in patients with severe and moderate hemophilia A treated with factor VIII concentrates. Am J Hematol 1987 24(3) 241-5. 

Two patients (aged 60 and 73 years) with mild and moderate hemophilia respectively, without a family history of inhibitor formation, were exposed to factor VIII for more than 50 days. An inhibitor developed in both patients after an intermediate factor VIII product was replaced by a high-purity factor VIII product, which was given by continuous infusion during a surgical procedure. 

Recombinant factor concentrates are usually first-line treatment for moderate and severe hemophilia because they have the lowest risk of infection. 

In the PTP Pivotal study. 111 patients aged > 10 years with moderately severe to severe hemophilia A (baseline FVllI <2%) and prior treatment with FVIIl concentrates... 

PUP Study this planned study is designed to determine the pharmacokinetics, efficacy, immunogenicity, and safety of rAHF-PFM in a minimum of 50 previously untreated patients, aged <6 years, with moderately severe to severe hemophilia A (baseline FVIII activity of <2%). 

Mild and moderate forms of hemophilia A and von Willebrand disease (39)... 

More recently, the ALN-AT3 (RNAi targeting antithrombin Sehgal et al, 2015) entered a phase I clinical trial with healthy volunteers (NCT02035605) and then patients with moderate or severe hemophilia A and B (NCT02554773). 

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