Improvement in clinical outcomes and replacement factor <scp>VIII</scp> use in patients with haemophilia A after factor <scp>VIII</scp> pharmacokinetic‐guided prophylaxis based on Bayesian models with my<scp>PKF</scp>iT<sup>®</sup>

Mingot‐Castellano, María Eva; Parra, Rafael; Núñez, Ramiro; Martorell, Marta

doi:10.1111/hae.13540

Cited by 32 publications

(36 citation statements)

References 18 publications

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“…14,16,22,23 PK studies of non-Chinese populations have also been conducted. 24,25 A PK study of Kogenate FS ® in Canada revealed that the mean t 1/2 , CL, and was nearly identical to ours (10.18 h), whereas the CL was lower than in the present study; this discrepancy may be related to inter-individual variability, age difference, and ethnicity-related differences. 25,26 Further research is needed to compare PK among populations.…”

Section: Discussionsupporting

confidence: 73%

Pharmacokinetic variability of factor VIII concentrates in Chinese pediatric patients with moderate or severe hemophilia A

Chen

Huang

et al. 2021

Pediatric Investigation

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Importance The use of factor VIII (FVIII) concentrates under pharmacokinetic (PK) guidance has become the main approach for treatment of hemophilia. However, limited PK research has been conducted in Chinese pediatric patients. Objective To investigate the PK parameters of various FVIII concentrates in Chinese pediatric patients. Methods Seventy‐nine patients were enrolled (28 treated with Kogenate FS ® , 23 treated with Advate ® , and 28 treated with GreenMono™). All enrolled patients participated in single‐dose PK analysis after at least a 3‐day washout period. Blood samples were collected predose, as well as at 1 h, 9 h, 24 h, and 48 h after infusion; FVIII levels were measured using a one‐stage clotting assay. von Willebrand Factor Antigen (VWF:Ag) levels and blood types were also determined. PK parameters were evaluated by WAPPS‐Hemo. Results Mean values of terminal elimination half‐life time (t 1/2 ) for the Kogenate FS ® , Advate ® , and GreenMono™ FVIII groups were 12.24 h, 10.18 h, and 9.62 h; median clearance values were 4.16, 6.23, and 5.11 mL·kg −1 ·h −1 ; and median in vivo recovery values were 1.97, 1.55, and 1.61 IU/dL per IU/kg. Longer t 1/2 , higher in vivo recovery, and lower clearance were observed in patients with higher VWF:Ag level who were treated with recombinant concentrates. Interpretation Chinese pediatric patients with hemophilia had FVIII PK characteristics similar to those previously observed in non‐Chinese children, including large variation among individuals. VWF:Ag level and FVIII brand were associated with differences in FVIII PK. Thus, PK‐guided dosing should be used to optimize individualized therapy in Chinese children.

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Section: Discussionsupporting

confidence: 73%

Pharmacokinetic variability of factor VIII concentrates in Chinese pediatric patients with moderate or severe hemophilia A

Chen

Huang

et al. 2021

Pediatric Investigation

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“…A number of recent reports have documented the potential benefits of individualized prophylaxis regimens based on measured PK profiles of subjects with hemophilia A. 7,[21][22][23] A challenge to the implementation of PK directed prophylaxis regimens in the past was that traditional PK studies in individuals with hemophilia were demanding as they required a long washout to achieve trough levels below the limit of quantitation of FVIII using conventional one-stage or chromogenic FVIII:C assays, followed by multiple post-infusion blood samples. Consequently, such PK studies were impractical from a clinical perspective.…”

Section: Discussionmentioning

confidence: 99%

A Practical, One-Clinic Visit Protocol for Pharmacokinetic Profile Generation with the ADVATE myPKFiT Dosing Tool in Severe Hemophilia A Subjects

et al. 2021

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Standard pharmacokinetic (PK) assessments are demanding for persons with hemophilia A, requiring a 72hr washout and 5-11 timed blood samples. A no-washout, single-clinic visit, sparse sampling population PK (PPK) protocol is an attractive alternative. Here, we compared PK parameters obtained with a traditional washout, 6-sampling time point PPK protocol with a no-washout, single-clinic visit, reverse 2-sampling time point PPK protocol in persons with severe hemophilia A (SHA) receiving ADVATE®. 39 inhibitor-negative males with SHA (FVIII:C<2%) were enrolled in a prospective sequential design PK study. Participants completed a washout, 6-sampling time point PPK protocol as well as a no-washout, reverse 2-sampling time point protocol, with samples taken during a single 3hr clinic visit 24hr-post home infusion of FVIII and then 3hr-post infusion in clinic. FVIII:C levels were analyzed by one-stage and chromogenic assays; blood group and VWF:Ag were determined; and PK parameters were analyzed using the ADVATE® myPKFiT® dosing tool. There was moderate to almost perfect agreement for the PK parameters obtained with the 2- and the 6-point PPK protocols using a one-stage FVIII:C assay and a substantial to almost perfect agreement using a chromogenic FVIII:C assay. Significant associations between specific PK parameters and blood group and VWF:Ag were observed. The no-washout, single-clinic visit, reverse 2-sampling time point PPK protocol can be used in the routine clinical setting since it demonstrates sufficient accuracy compared to the more demanding and less practical washout, 6-sampling time point PPK protocol in persons with SHA receiving ADVATE®.

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“…Recently, some authors have provided recommendations for performing the switch between different FVIII products, 6 including extended half-life FVIII (EHL). In our centre, we use extensively PopPK resources to tailor therapy, and consequently, it was also used to assess PK with rFVIII-FS, and therefore, with BAY 81-8973.…”

Section: Cross-sectional Comparative Study Of Pharmacokinetics and Efmentioning

confidence: 99%

Cross‐sectional comparative study of pharmacokinetics and efficacy between sucrose‐formulated recombinant factor VIII (Kogenate^®) and BAY 81‐8973 (Kovaltry^®) in patients with severe or moderate haemophilia A in prophylaxis

et al. 2019

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Improvement in clinical outcomes and replacement factor VIII use in patients with haemophilia A after factor VIII pharmacokinetic‐guided prophylaxis based on Bayesian models with myPKFiT^®

Abstract: Our results suggest that PK-guided prophylaxis using myPKFiT improved clinical outcomes and optimized FVIII consumption in the study population. This personalized approach may reduce bleeding rates without significantly increasing the overall cost of FVIII therapy.

Cited by 32 publications

References 18 publications

Pharmacokinetic variability of factor VIII concentrates in Chinese pediatric patients with moderate or severe hemophilia A

Pharmacokinetic variability of factor VIII concentrates in Chinese pediatric patients with moderate or severe hemophilia A

A Practical, One-Clinic Visit Protocol for Pharmacokinetic Profile Generation with the ADVATE myPKFiT Dosing Tool in Severe Hemophilia A Subjects

Cross‐sectional comparative study of pharmacokinetics and efficacy between sucrose‐formulated recombinant factor VIII (Kogenate^®) and BAY 81‐8973 (Kovaltry^®) in patients with severe or moderate haemophilia A in prophylaxis

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Improvement in clinical outcomes and replacement factor VIII use in patients with haemophilia A after factor VIII pharmacokinetic‐guided prophylaxis based on Bayesian models with myPKFiT®

Abstract: Our results suggest that PK-guided prophylaxis using myPKFiT improved clinical outcomes and optimized FVIII consumption in the study population. This personalized approach may reduce bleeding rates without significantly increasing the overall cost of FVIII therapy.

Cited by 32 publications

References 18 publications

Pharmacokinetic variability of factor VIII concentrates in Chinese pediatric patients with moderate or severe hemophilia A

Pharmacokinetic variability of factor VIII concentrates in Chinese pediatric patients with moderate or severe hemophilia A

A Practical, One-Clinic Visit Protocol for Pharmacokinetic Profile Generation with the ADVATE myPKFiT Dosing Tool in Severe Hemophilia A Subjects

Cross‐sectional comparative study of pharmacokinetics and efficacy between sucrose‐formulated recombinant factor VIII (Kogenate®) and BAY 81‐8973 (Kovaltry®) in patients with severe or moderate haemophilia A in prophylaxis

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Product

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About

Improvement in clinical outcomes and replacement factor VIII use in patients with haemophilia A after factor VIII pharmacokinetic‐guided prophylaxis based on Bayesian models with myPKFiT^®

Cross‐sectional comparative study of pharmacokinetics and efficacy between sucrose‐formulated recombinant factor VIII (Kogenate^®) and BAY 81‐8973 (Kovaltry^®) in patients with severe or moderate haemophilia A in prophylaxis