2017 Clinical trials update: Innovations in hemophilia therapy

Hartmann, Jan; Croteau, Stacy E.

doi:10.1002/ajh.24543

Cited by 87 publications

(90 citation statements)

References 91 publications

(103 reference statements)

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“…These products are primarily helpful in the treatment of hemophilia B because of the significantly pronounced extension of the half-life of factor IX (FIX) compared to FVIII [11]. Other possible innovations in the hemophilia treatment might include disrupting anticoagulant proteins (Table 2), the according clinical trials are ongoing [12]. Novel treatment options might also include bispecific antibodies to mimic the coagulation function of FVIII or gene transfer for production of endogenous factor protein—these treatment options were highlighted during the ASH meeting 2016.…”

Section: Upcoming Treatment Options In Hemophiliamentioning

confidence: 99%

ASH Meeting 2016: developments in hemostaseology

Feistritzer

Mosheimer

2017

memo

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SummaryDuring the annual meeting of the American Society of Hematology (ASH) in San Diego/California, novel developments in the field of hemostaseology were presented. Alternative treatment strategies besides factor replacement were discussed for patients with hemophilia. One of the highlights of the meeting in this year’s plenary session was the presentation of successful adeno-associated virus mediated gene transfer in patients with hemophilia B leading to sustained elevation of factor IX:C (FIX:c). Other alternative treatment approaches in patients with hemophilia A may include bispecific antibodies mimicking factor VIIIa (FVIIIa) activity or disrupting anticoagulant proteins. Focusing on anticoagulation, data on the use of direct oral anticoagulants (DOACs) in cancer patients with atrial fibrillation as well as treatment of superficial vein thrombosis with rivaroxaban were presented. In this short review, we try to highlight the most important presentations during the ASH meeting 2016.

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Section: Upcoming Treatment Options In Hemophiliamentioning

confidence: 99%

ASH Meeting 2016: developments in hemostaseology

Feistritzer

Mosheimer

2017

memo

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“…In order to reduce the frequency of infusion, extended half-life products are becoming available with the first products currently licensed, and many others in the pipeline [55,56].…”

Section: Extended Half-life Productsmentioning

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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“…The significant annual cost of haemophilia therapy coupled with the expanding availability of newly designed factor concentrates 4 has created an opportunity for pharmacokinetics (PK) data to contribute to prophylaxis regimen decision-making. The significant annual cost of haemophilia therapy coupled with the expanding availability of newly designed factor concentrates 4 has created an opportunity for pharmacokinetics (PK) data to contribute to prophylaxis regimen decision-making.…”

Section: Introductionmentioning

confidence: 99%

Clinical application of Web Accessible Population Pharmacokinetic Service—Hemophilia (WAPPS‐Hemo): Patterns of blood sampling and patient characteristics among clinician users

et al. 2019

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Background: Use of population pharmacokinetics (PopPK) to facilitate PK-informed prophylaxis in clinical practice has gained momentum among haemophilia providers due to the accessibility of tools such as the Web Accessible Population Pharmacokinetic Service-Hemophilia (WAPPS-Hemo) and availability of extended half-life (EHL) factor concentrates. It is unknown how clinicians implement PopPK. Aim:To investigate the evolution of PopPK use in clinical practice by comparing blood sampling strategies, patient features, and factor group between initial and recent periods of WAPPS-Hemo availability.Methods: PK data for haemophilia A and haemophilia B patients from two time periods were extracted from the WAPPS-Hemo database: early availability (10/2015-09/2016) and recent use (10/2017-09/2018). We compared patient characteristics (age, body weight, haemophilia type), product type and dose, and blood sampling times between the time frames.Results: Over 1900 eligible infusions were submitted to WAPPS-Hemo during the periods studied, with 85% representing FVIII concentrates. In the recent cohort, PK profiles were requested for younger patients (median age 18 vs 26 years), with increased proportional EHL FVIII use (29% vs 14% of infusions). High-use centres generally submitted fewer blood samples per infusion than non-high-use centres, although the number of samples collected by non-high-use centres decreased significantly over time. During both periods, blood sample timing was generally consistent with ISTH recommended windows. Conclusion:The use of WAPPS-Hemo by haemophilia providers grew by over threefold between the time periods investigated. While sampling times have included key time points proposed first by Björkman since early WAPPS-Hemo usage, a trend towards minimizing sampling was observed. K E Y W O R D Sextended half-life, factor concentrate, haemophilia, pharmacokinetics, PopPK | 57McENENY-KING Et al.

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2017 Clinical trials update: Innovations in hemophilia therapy

Cited by 87 publications

References 91 publications

ASH Meeting 2016: developments in hemostaseology

ASH Meeting 2016: developments in hemostaseology

The current state of adverse event reporting in hemophilia

Clinical application of Web Accessible Population Pharmacokinetic Service—Hemophilia (WAPPS‐Hemo): Patterns of blood sampling and patient characteristics among clinician users

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