Similar bleeding phenotype in young children with haemophilia A or B: a cohort study

Clausen, Niels; Petrini, Pia; Claeyssens-Donadel, Ségolène; Gouw, Samantha C.; Liesner, Ri; PedNet,

doi:10.1111/hae.12470

Cited by 40 publications

(37 citation statements)

References 14 publications

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“…However, for separating haemophilic patients from healthy volunteers, peak seems to be a more sensitive parameter than ETP, as already described . No significant difference in ETP or peak was found between HA and HB patients with the same degree of disease severity, corroborating the finding that bleeding phenotype does not differ between HA and HB patients …”

Section: Discussionsupporting

confidence: 83%

Tissue factor pathway inhibitor is the main determinant of thrombin generation in haemophilic patients

et al. 2019

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The thrombin generation (TG) assay evaluates haemostatic balance, which is influenced by the levels of many coagulation factors and inhibitors. Our objective was to identify the determinant factors of TG in haemophilia A (HA) and haemophilia B (HB) patients and to compare them to those in healthy controls. Coagulation factor and inhibitor levels, and TG, were measured in platelet‐poor plasma from 40 patients with HA, 32 patients with HB and 40 healthy subjects. Data were analysed using multiple regression models. In HA patients, factor VIII was a positive determinant of endogenous thrombin potential (ETP) and peak, whereas tissue factor pathway inhibitor (TFPI) and factor V were negative determinants of ETP and peak. In HB patients, FIX was a positive determinant of ETP and peak, FVII being a positive determinant of peak. Antithrombin and protein S (PS) were negative determinants of ETP while FX was a negative determinant of peak. Above all, in HB patients, TFPI was a negative determinant of ETP and peak. In healthy subjects, FVIII was a positive determinant of ETP and peak, whereas FX and protein S were negative determinants of these parameters. TFPI was not a negative determinant of either peak or ETP. In haemophilic patients, the determinant factors of TG are all implicated in FXa generation and inhibition, the crucial determinant factor being TFPI whatever the type of haemophilia, A or B. These findings contribute to the rationale that recently place TFPI as a target for innovative therapies of haemophilia.

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Section: Discussionsupporting

confidence: 83%

Tissue factor pathway inhibitor is the main determinant of thrombin generation in haemophilic patients

et al. 2019

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“…Another question being discussed is whether patients with haemophilia B have the same need for prophylaxis as patients with haemophilia A. It has recently been shown that there is no difference in the bleeding phenotype in young children with haemophilia A compared to those with haemophilia B (Clausen et al , ). Using the PedNet Registry, Clausen et al () found no difference in children between the bleeding phenotype of severe or moderate haemophilia A and B using parameters such as the age at diagnosis, the age at first bleed or first joint bleed, the time from first to second joint bleed or the age at first treatment.…”

Section: Prophylactic Treatment Is Superior To On‐demandmentioning

confidence: 99%

The current status of prophylactic replacement therapy in children and adults with haemophilia

Ljung

Gretenkort

2015

Br J Haematol

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SummaryInitiating prophylactic treatment at an early age is considered to be the optimal form of therapy for a child with haemophilia A or B. The pioneering long term experiences of prophylactic treatment from Sweden and The Netherlands demonstrated the benefit of prophylaxis in retrospective and observational studies. Decades later, these benefits were confirmed in a randomized controlled study in USA. We review the current status of prophylactic replacement therapy of haemophilia in children, adolescents, adults and the elderly. Prophylaxis should begin at an early age and there are arguments for continuing it into adulthood. The dose of prophylaxis is dependent on the goal of treatment, economic resources and venous access and should be tailored individually. Starting the first exposures to clotting factor concentrates as prophylactic treatment, instead of on-demand in response to a bleed, may decrease the frequency of inhibitors in patients with haemophilia A. Novel longer-acting products are being introduced that could be helpful for patients with difficult venous access and enable higher trough levels.

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“…The participating centers are mainly in Europe with some centers in Canada and Israel. Among the important publications from this study were a report suggesting that one second generation recombinant FVIII concentrate was associated with a higher rate of inhibitors [18,43], and papers on the risk factors for inhibitor development in PUPs [44,45]. In terms of adverse events the RODIN study collects data only on inhibitor development.…”

Section: Specific Multicenter Schemes A) Rodinmentioning

confidence: 99%

The current state of adverse event reporting in hemophilia

Vulpen

Saccullo

Iorio

et al. 2016

Expert Review of Hematology

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Text word count: 3827; abstract word count: 187; number of tables: 5; number of references: 73.2 Abstract Introduction: Replacement of the missing clotting factor is the mainstay of haemophilia treatment. Whilst historically many haemophilia patients were infected with blood-borne viruses transmitted via plasma-derived products, nowadays the formation of alloantibodies against the missing clotting factor is the main adverse event of treatment.Areas covered: This paper provides an overview of the current national and international adverse event reporting systems, what these surveillance schemes taught us about side effects of the products presently in use, and elaborates on how to adapt these systems to the challenges we face with the changing treatment landscape.Expert commentary: Treatment of inherited bleeding disorders was accompanied by severe complications in the past, resulting in major morbidity and mortality. Current products are much safer, but still require monitoring via efficient safety surveillance systems. Adverse events are reported in national and international systems. With many new products entering the market, as well as non-factor replacement therapies, new safety issues may arise. It is important to identify potential adverse events early by making surveillance systems suitable to pick up unknown or unexpected effects, and to recognize and communicate patterns of adverse events rapidly.

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Similar bleeding phenotype in young children with haemophilia A or B: a cohort study

Cited by 40 publications

References 14 publications

Tissue factor pathway inhibitor is the main determinant of thrombin generation in haemophilic patients

Tissue factor pathway inhibitor is the main determinant of thrombin generation in haemophilic patients

The current status of prophylactic replacement therapy in children and adults with haemophilia

The current state of adverse event reporting in hemophilia

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