Population Pharmacokinetics of Clotting Factor Concentrates and Desmopressin in Hemophilia

Preijers, Tim; Schütte, Lisette M.; Kruip, Marieke J.H.A.; Cnossen, Marjon H.; Leebeek, Frank W.G.; Hest, Reinier M. van; Mathôt, Ron A. A.

doi:10.1007/s40262-020-00936-5

Cited by 7 publications

(4 citation statements)

References 89 publications

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“…IV, intravenous; N/A, not applicable; ODT, orally disintegrating tablet. of pharmacokinetic-guided dosing of desmopressin since there is considerable pharmacokinetic variability (20,21). There is also variation in clinical response to desmopressin, which was recently demonstrated to be affected by VWF genetic variants (22).…”

Section: Results Of Literature Review and Discussion Of Findingsmentioning

confidence: 99%

“…In a retrospective study, half dose desmopressin (0.15 µg/kg IV) was also found to be effective in adult bleeding disorder patients undergoing low to moderate risk invasive procedures ( 19 ). There are ongoing studies looking at the feasibility of pharmacokinetic-guided dosing of desmopressin since there is considerable pharmacokinetic variability ( 20 , 21 ). There is also variation in clinical response to desmopressin, which was recently demonstrated to be affected by VWF genetic variants ( 22 ).…”

Section: Results Of Literature Review and Discussion Of Findingsmentioning

confidence: 99%

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Potential clinical applications of current and future oral forms of desmopressin (Review)

Everaert,

Holm‑larsen,

Bou Kheir

et al. 2024

Exp Ther Med

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Desmopressin is a synthetic analogue of vasopressin and a selective vasopressin receptor 2 agonist. It was first synthesised in 1967 and utilised for its antidiuretic properties. It is also used in bleeding disorders to enhance clotting. Other potential uses of the drug have been reported. The present review aims to provide a broad overview of the literature on potential further uses of oral forms of desmopressin. Key therapeutic areas of interest were identified based on known physiological activities/targets of desmopressin or reports of an effect of desmopressin in the literature. The feasibility of adequate dosing with oral forms of the drug was also considered. Systematic literature searches were carried out using the silvi.ai software for the identified areas, and summaries of available papers were included in tables and discussed. The results of the searches showed that desmopressin has been investigated for its efficacy in a number of areas, including bleeding control, renal colic, the central nervous system and oncology. Evidence suggests that oral desmopressin may have the potential to be of clinical benefit for renal colic and bleeding control in particular. However, further research is needed to clarify its effect in these areas, including randomised controlled studies and studies specifically of oral formulations (and doses). Further research may also yield findings for cancer, cognition and overactive bladder. Contents 1. Introduction 2. Methods 3. Results of literature review and discussion of findings 4. Conclusion

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Section: Results Of Literature Review and Discussion Of Findingsmentioning

confidence: 99%

Section: Results Of Literature Review and Discussion Of Findingsmentioning

confidence: 99%

Potential clinical applications of current and future oral forms of desmopressin (Review)

Everaert,

Holm‑larsen,

Bou Kheir

et al. 2024

Exp Ther Med

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“…The recommended dose is 0.3 µg/kg intravenously over 15-30 minutes or 300 mcg intranasally for adult patients weighing >50 kg [23]. Desmopressin is not a useful treatment for bleeding in patients with hemophilia B [24]. Clinical improvement, normal PTT and optimal factor VIII activity levels are good indicators of clinical response.…”

Section: Factor Replacementmentioning

confidence: 99%

Management of acute hemorrhage in patients with hemophilia or von Willebrand disease in the emergency department

2022

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Patients with hemophilia or von Willebrand disease may present to the emergency department (ED) with life-threatening bleeding, as severe hemorrhage can lead to hemodynamic instability and bleeding in the central nervous system, throat, and neck. Thus, inappropriate, or delayed management could lead to serious treatment-related complications or even death, and therefore, emergency medical staffs should be well-equipped with the latest knowledge to properly and timely treat these patients. The goal of treatment in emergency settings is to achieve hemostasis by replacing clotting factor levels, prevent hemodynamic instability, and prompt initiation of further specialized treatments. In this study, we searched Medline, PubMed, Embase and Google Scholar for papers addressing hemophilia- and Von Willebrand disease (VWD)-related bleeding and factors in emergency settings to determine evidence-based approaches for managing severe hemorrhage in these patients in the ED.

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“…Recently, Preijers et al discussed available population PK models for FVIII and FIX concentrates, focusing on the methods used to construct these models, key features, and established covariate relationships. 14 Our review aims to provide a detailed overview of the clinical and laboratory data used to construct available population PK models for both standard half-life (SHL) and EHL FVIII and FIX concentrates, as reported in literature. In this way, we aim to support physicians in their choices that model best suits each individual patient.…”

Section: Introductionmentioning

confidence: 99%

Population pharmacokinetic modeling of factor concentrates in hemophilia: an overview and evaluation of best practice

et al. 2021

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The accuracy of pharmacokinetic (PK)-guided dosing depends on the clinical and laboratory data used to construct a population PK model, as well as the patient's individual PK profile. This review provides a detailed overview of data used for published population PK models for factor VIII (FVIII) and factor IX (FIX) concentrates, to support physicians in their choices which model best suits each patient. Furthermore, to enhance detailed data collection and documentation, we do suggestions for best practice. A literature search was performed; publications describing prophylactic population PK models for FVIII and FIX concentrates based on original patient data and constructed using non-linear mixed-effect modeling were included. The following data were collected: detailed demographics, type of product, assessed and included covariates, laboratory specifications and validation of models. Included models were scored according to our recommendations for best practice, specifically scoring the quality of data documentation as reported. Respectively, twenty models for FVIII and seven for FIX concentrates were retrieved. Although most models (22/27) included pediatric patients, only four reported detailed demographics. The wide range of body weights suggested that overweight and obese adults were represented. Twenty-six models reported the assay applied to measure factor levels, whereas only 16 models named reagents used. Eight models were internally validated using a data subset. This overview presents detailed information on clinical and laboratory data used for published population PK models. We provide recommendations on data collection and documentation to increase the reliability of PK-guided prophylactic dosing of factor concentrates in hemophilia A and B.

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Population Pharmacokinetics of Clotting Factor Concentrates and Desmopressin in Hemophilia

Cited by 7 publications

References 89 publications

Potential clinical applications of current and future oral forms of desmopressin (Review)

Potential clinical applications of current and future oral forms of desmopressin (Review)

Management of acute hemorrhage in patients with hemophilia or von Willebrand disease in the emergency department

Population pharmacokinetic modeling of factor concentrates in hemophilia: an overview and evaluation of best practice

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