Roberta Palla
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View article: Gynecologic and obstetric complications in women with congenital fibrinogen disorders: insights from the Prospective Rare Bleeding Disorders Database
Gynecologic and obstetric complications in women with congenital fibrinogen disorders: insights from the Prospective Rare Bleeding Disorders Database Open
The high rate of obstetric complications in women with CFDs highlights the need for early diagnosis and the potential need for prophylaxis, as pregnancy may also pose risks in asymptomatic cases. Hotspot variants do not appear to increase …
View article: Predicting inhibitor development using a random peptide phage-display library approach in the SIPPET cohort
Predicting inhibitor development using a random peptide phage-display library approach in the SIPPET cohort Open
Inhibitor development is the most severe complication of hemophilia A (HA) care and is associated with increased morbidity and mortality. This study aimed to use a novel immunoglobulin G epitope mapping method to explore the factor VIII (F…
View article: Predictive parameters for spontaneous joint bleeding during emicizumab prophylaxis
Predictive parameters for spontaneous joint bleeding during emicizumab prophylaxis Open
Emicizumab is approved for prophylaxis of patients with hemophilia A (HA). Despite its efficacy in reducing bleeding, some patients on emicizumab still experience hemarthrosis, but no tool is yet available to identify those at a higher ris…
View article: Congenital fibrinogen disorders: a retrospective clinical and genetic analysis of the Prospective Rare Bleeding Disorders Database
Congenital fibrinogen disorders: a retrospective clinical and genetic analysis of the Prospective Rare Bleeding Disorders Database Open
Congenital fibrinogen deficiency (CFD) is a rare bleeding disorder caused by mutations in FGA, FGB, and FGG. We sought to comprehensively characterize patients with CFD using PRO-RBDD (Prospective Rare Bleeding Disorders Database). Clinica…
View article: Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders Open
Centralization and expansion of expertise and resources, flexible innovative research and regulatory approaches, and inclusion of all people with ultra-rare inherited BDs and their health care professionals will be essential to capitalize …
View article: Diagnostic utility of bleeding assessment tools in congenital fibrinogen deficiencies
Diagnostic utility of bleeding assessment tools in congenital fibrinogen deficiencies Open
Background The assessment of clinical history is crucial before referring a patient for further laboratory testing. Bleeding assessment tools (BAT) are developed to standardize clinical evaluation. A small number of patients with congenita…
View article: Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders Open
Ultra-rare inherited bleeding disorders (BDs) present important challenges for generating a strong evidence foundation for optimal diagnosis and management. Without disorder-appropriate treatment, affected individuals potentially face life…
View article: Performance of a clinical risk prediction model for inhibitor formation in severe haemophilia A
Performance of a clinical risk prediction model for inhibitor formation in severe haemophilia A Open
Background There is a need to identify patients with haemophilia who have a very low or high risk of developing inhibitors. These patients could be candidates for personalized treatment strategies. Aims The aim of this study was to externa…
View article: IgG subclasses as biomarkers for persistence of factor VIII inhibitors in previously untreated patients with severe haemophilia A
IgG subclasses as biomarkers for persistence of factor VIII inhibitors in previously untreated patients with severe haemophilia A Open
Summary We investigated longitudinally the behaviour of anti‐factor VIII (anti‐FVIII) IgG subclasses for 6 months from inhibitor development in 43 patients from the Survey of Inhibitors in Plasma‐Products Exposed Toddlers (SIPPET) trial wh…
View article: Hemostatic alterations in COVID-19
Hemostatic alterations in COVID-19 Open
Hemostatic alterations in COVID-19Coronavirus disease 2019 (COVID-19) is caused by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), 1,2 that, first identified in China, has spread globally.A coagulopathy is common, particu…
View article: An international registry of patients with plasminogen deficiency (HISTORY)
An international registry of patients with plasminogen deficiency (HISTORY) Open
Plasminogen deficiency is an ultra-rare multisystem disorder characterized by the development of fibrin-rich pseudomembranes on mucous membranes. Ligneous conjunctivitis, which can result in vision impairment or loss, is the most frequent …
View article: Role of factor VIII-binding capacity of endogenous von Willebrand factor in the development of factor VIII inhibitors in patients with severe hemophilia A
Role of factor VIII-binding capacity of endogenous von Willebrand factor in the development of factor VIII inhibitors in patients with severe hemophilia A Open
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View article: High-titre inhibitors in previously untreated patients with severe haemophilia A receiving recombinant or plasma-derived factor VIII: a budget-impact analysis.
High-titre inhibitors in previously untreated patients with severe haemophilia A receiving recombinant or plasma-derived factor VIII: a budget-impact analysis. Open
The SIPPET (Survey of Inhibitors in Plasma-Products Exposed Toddlers) trial1 provided evidence that, in previously untreated patients with severe haemophilia A, recombinant factor VIII increases the risk of developing high-titre inhibitors…
View article: Genetic risk stratification to reduce inhibitor development in the early treatment of hemophilia A: a SIPPET analysis
Genetic risk stratification to reduce inhibitor development in the early treatment of hemophilia A: a SIPPET analysis Open
Key Points It has been suggested that rFVIII, which is more immunogenic than plasma-derived FVIII (pdFVIII), can be safely used in low-risk patients. Among 235 participants in a randomized trial, genetic risk stratification did not identif…
View article: Minimal factor XIII activity level to prevent major spontaneous bleeds
Minimal factor XIII activity level to prevent major spontaneous bleeds Open
Background Congenital factor XIII (FXIII) deficiency is a rare bleeding disorder associated with significant bleeding manifestations. The European Network of Rare Bleeding Disorders (EN-RBD) study, performed from 2007 to 2010, showed a str…
View article: Nonneutralizing antibodies against factor VIII and risk of inhibitor development in severe hemophilia A
Nonneutralizing antibodies against factor VIII and risk of inhibitor development in severe hemophilia A Open
Key Points Nonneutralizing antibodies against FVIII are detected in untreated or minimally treated patients with hemophilia A. The presence of nonneutralizing antibodies is associated with a substantially increased risk of inhibitor develo…