Effects of PK-guided prophylaxis on clinical outcomes and FVIII consumption for patients with moderate to severe Haemophilia A.

Introduction: In recent years, there has been increased focus on individualizing treatment for persons with hemophilia including pharmacokinetic-guided (PK) dosing.

Aims: In this retrospective study clinical outcomes before and after PK-guided prophylaxis were examined.

Materials And Methods: Eight Haemophilia Treatment Centres from the United States participated in the study and included 132 patients classified into two cohorts: those undergoing a PK-assessment for product switch (switchers) or to optimize treatment (non-switchers).

Treatment of Congenital Afibrinogenemia in a Neonate With Critical Pulmonary Stenosis.

Fibrinogen deficiencies in neonates can lead to bleeding complications. In this report, we describe a case of congenital afibrinogenemia in a newborn with critical pulmonary stenosis who presented with bilateral cephalohematomas after an uncomplicated delivery. The initial use of cryoprecipitate was followed by administration of fibrinogen concentrate.

SARS-CoV-2 Infection Presenting as Acute Chest Syndrome in a Child With Hemoglobin SD-Los Angeles Disease: A Case Report and Review of Literature.

Hemoglobin D-Los Angeles is a variant of hemoglobin that can polymerize in the deoxygenated state. When co-inherited with Hemoglobin S (HbSD-Los Angeles disease) a severe sickling syndrome similar to HbSS can result. Corona virus infectious disease 2019 (COVID-19) is caused by the severe acute respiratory syndrome-corona virus-2.

Real-world study of rurioctocog alfa pegol and emicizumab in US clinical practice among patients with hemophilia A.

Background: FVIII replacement is standard treatment for hemophilia A without inhibitors, but non-factor therapies, such as emicizumab, are changing the treatment landscape. We explore the ramifications of switching treatment.

Methods: Pharmacy database data (July 2017-May 2020) from patients with hemophilia A without inhibitors who switched to rurioctocog alfa pegol or emicizumab prophylaxis after ≥6 months' prophylaxis with another FVIII product were analyzed for total mean weekly consumption, dosing frequency, product wastage, and ABR.

Unusually High Prevalence of Stroke and Cerebral Vasculopathy in Hemoglobin SC Disease: A Retrospective Single Institution Study.

Introduction: Unlike homozygous hemoglobin SS (HbSS) disease, stroke is a rare complication in hemoglobin SC (HbSC) disease. However, recent studies have demonstrated a high prevalence of silent stroke in HbSC disease. The factors associated with stroke and cerebral vasculopathy in the HbSC population are unknown.

A systematic review evaluating the efficacy and factor consumption of long-acting recombinant factor VIII products for the prophylactic treatment of hemophilia A.

Aims: Long-acting (LA) recombinant FVIII (rFVIII) products with extended dosing intervals have been developed for the treatment of hemophilia A; however, no direct head-to-head trial has been conducted to compare the efficacy of these products.

Materials And Methods: A systematic literature search was conducted to identify published Phase III clinical trials of prophylactic LA rFVIII treatment in previously treated patients aged ≥12 years, with moderate-to-severe hemophilia A (endogenous FVIII levels ≤2%). Studies that did not meet these criteria, or did not report the included outcomes, were excluded.

Development and Validation of a Population-Pharmacokinetic Model for Rurioctacog Alfa Pegol (Adynovate): A Report on Behalf of the WAPPS-Hemo Investigators Ad Hoc Subgroup.

Background And Objective: Rurioctacog alfa pegol (Adynovate) is a modified recombinant factor VIII concentrate used for treating hemophilia A. Aiming to improve treatment tailoring on the Web-Accessible Population Pharmacokinetic Service-Hemophilia (WAPPS-Hemo) platform for patients of all ages treated with Adynovate, we have developed and evaluated a population pharmacokinetic (PopPK) model. On the platform, PopPK models are used as priors for Bayesian forecasting that derive individual PK of hemophilia patients and are subsequently used for personalized dose regimen design.

Switching patients in the age of long-acting recombinant products?

: Prophylaxis with factor replacement therapy is the gold standard for the treatment of hemophilia, but this often requires frequent infusions. A number of long-acting factor products have been developed to reduce the burden on patients. : This is an overview of information presented at two symposia held at the World Federation of Hemophilia and International Society on Thrombosis and Haemostasis - Scientific and Standardization Committee annual meetings.

Transfusion practices and complications in thalassemia.

Background: The severe forms of thalassemia are the most common inherited anemias managed with regular blood transfusion therapy. Transfusion policies and complications are critical to quality of life and survival, but there is a lack of standardized care.

Study Design And Methods: A survey of 58 items was completed in 2016 by 11 centers in California, Washington, Oregon, Nevada, and Arizona providing long-term care for thalassemia.

Disparity in the management of iron overload between patients with sickle cell disease and thalassemia who received transfusions.

Background: Transfusion therapy is frequently used to prevent morbidity in sickle cell disease (SCD), and subsequent iron overload is common. The objective of this study was to evaluate the current standard of care in monitoring iron overload and related complications in patients with SCD compared to thalassemia (Thal).

Study Design And Methods: A cross-sectional study was conducted at 31 hematology clinics in the United States, Canada, or the United Kingdom.

New anticoagulants: a pediatric perspective.

Unfractionated heparin and vitamin K antagonists such as warfarin have been used as the anticoagulants of choice for over five decades. Subsequently, low molecular weight heparins (LMWHs) became widely available and have provided several advantages, especially in infants and children. The field of anticoagulation, however, has undergone a major revolution with better understanding of the structure of coagulation proteins and the development of a host of new drugs with highly specific actions.

Legg-Calve-Perthes disease and thrombophilia.

Background: Thrombophilia has previously been identified as a potential etiologic factor in Legg-Calve-Perthes disease. We prospectively studied the association between Legg-Calve-Perthes disease and coagulation abnormalities by comparing seventy-two children who had the disease with 197 healthy controls.

Methods: A nonselected, consecutive series of seventy-two patients with Legg-Calve-Perthes disease (mean age [and standard deviation], 6.

Hyperhomocysteinemia is associated with low plasma pyridoxine levels in children with sickle cell disease.

Elevated plasma homocysteine levels have been shown to be a risk factor for endothelial cell damage and thrombosis, which are implicated in sickle cell disease (SCD)-related vaso-occlusion. The aim of this study was to determine the prevalence of hyperhomocysteinemia in SCD. Fasting and postmethionine load (PML) homocysteine, red cell folate, and the MTHFR C677T mutation were determined in 77 patients with SCD and 110 African-American controls.