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

J 2021

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

BLANCHETTE, V. S.; L. ZUNINO; V. GRASSMANN; C. BARNES; M. D. CARCAO et al.

Basic information

Original name

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

Authors

Edition

THROMBOSIS AND HAEMOSTASIS, STUTTGART, GEORG THIEME VERLAG KG, 2021, 0340-6245

Other information

Language

English

Type of outcome

Article in a journal

Field of Study

30205 Hematology

Country of publisher

Germany

Confidentiality degree

is not subject to a state or trade secret

References:

URL

Impact factor

Impact factor: 6.830

Marked to be transferred to RIV

Yes

RIV identification code

RIV/00216224:14110/21:00121731

Organization unit

Faculty of Medicine

Keywords in English

factor VIII; hemophilia A; pharmacokinetic; observational study; population PK

Abstract

In the original language

Standard pharmacokinetic (PK) assessments are demanding for persons with hemophilia A, requiring a 72-hour washout and 5 to 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. A total of 39 inhibitor-negative males with SHA (factor VIII activity [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 3-hour clinic visit 24hours post home infusion of FVIII and then 3hours post infusion in clinic. FVIII:C levels were analyzed by one-stage and chromogenic assays; blood group and von Willebrand factor antigen (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 with the more demanding and less practical washout, 6-sampling time point PPK protocol in persons with SHA receiving ADVATE.

Cite

BLANCHETTE, V. S.; L. ZUNINO; V. GRASSMANN; C. BARNES; M. D. CARCAO; J. CURTIN; S. JACKSON; L. KHOO; Vladimir KOMRSKA; D. LILLICRAP; M. MORFINI; Gabriela ROMANOVÁ; D. STEPHENS; Ester ZAPOTOCKA; M. L. RAND and Jan BLATNÝ. A Practical, One-Clinic Visit Protocol for Pharmacokinetic Profile Generation with the ADVATE myPKFiT Dosing Tool in Severe Hemophilia A Subjects. THROMBOSIS AND HAEMOSTASIS. STUTTGART: GEORG THIEME VERLAG KG, 2021, vol. 121, No 10, p. 1326-1336. ISSN 0340-6245. Available from: https://doi.org/10.1055/a-1376-0970.

@article{1774697,
   author = {Blanchette, V. S. and Zunino, L. and Grassmann, V. and Barnes, C. and Carcao, M. D. and Curtin, J. and Jackson, S. and Khoo, L. and Komrska, Vladimir and Lillicrap, D. and Morfini, M. and Romanová, Gabriela and Stephens, D. and Zapotocka, Ester and Rand, M. L. and Blatný, Jan},
   article_location = {STUTTGART},
   article_number = {10},
   doi = {https://doi.org/10.1055/a-1376-0970},
   keywords = {factor VIII; hemophilia A; pharmacokinetic; observational study; population PK},
   language = {eng},
   issn = {0340-6245},
   journal = {THROMBOSIS AND HAEMOSTASIS},
   title = {A Practical, One-Clinic Visit Protocol for Pharmacokinetic Profile Generation with the ADVATE myPKFiT Dosing Tool in Severe Hemophilia A Subjects},
   url = {https://www.thieme-connect.de/products/ejournals/abstract/10.1055/a-1376-0970},
   volume = {121},
   year = {2021}
}

TY  - JOUR
ID  - 1774697
AU  - Blanchette, V. S. - Zunino, L. - Grassmann, V. - Barnes, C. - Carcao, M. D. - Curtin, J. - Jackson, S. - Khoo, L. - Komrska, Vladimir - Lillicrap, D. - Morfini, M. - Romanová, Gabriela - Stephens, D. - Zapotocka, Ester - Rand, M. L. - Blatný, Jan
PY  - 2021
TI  - A Practical, One-Clinic Visit Protocol for Pharmacokinetic Profile Generation with the ADVATE myPKFiT Dosing Tool in Severe Hemophilia A Subjects
JF  - THROMBOSIS AND HAEMOSTASIS
VL  - 121
IS  - 10
SP  - 1326-1336
EP  - 1326-1336
PB  - GEORG THIEME VERLAG KG
SN  - 03406245
KW  - factor VIII
KW  - hemophilia A
KW  - pharmacokinetic
KW  - observational study
KW  - population PK
UR  - https://www.thieme-connect.de/products/ejournals/abstract/10.1055/a-1376-0970
N2  - Standard pharmacokinetic (PK) assessments are demanding for persons with hemophilia A, requiring a 72-hour washout and 5 to 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. A total of 39 inhibitor-negative males with SHA (factor VIII activity [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 3-hour clinic visit 24hours post home infusion of FVIII and then 3hours post infusion in clinic. FVIII:C levels were analyzed by one-stage and chromogenic assays; blood group and von Willebrand factor antigen (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 with the more demanding and less practical washout, 6-sampling time point PPK protocol in persons with SHA receiving ADVATE.
ER  -

BLANCHETTE, V. S.; L. ZUNINO; V. GRASSMANN; C. BARNES; M. D. CARCAO; J. CURTIN; S. JACKSON; L. KHOO; Vladimir KOMRSKA; D. LILLICRAP; M. MORFINI; Gabriela ROMANOVÁ; D. STEPHENS; Ester ZAPOTOCKA; M. L. RAND and Jan BLATNÝ. A Practical, One-Clinic Visit Protocol for Pharmacokinetic Profile Generation with the ADVATE myPKFiT Dosing Tool in Severe Hemophilia A Subjects. \textit{THROMBOSIS AND HAEMOSTASIS}. STUTTGART: GEORG THIEME VERLAG KG, 2021, vol.~121, No~10, p.~1326-1336. ISSN~0340-6245. Available from: https://doi.org/10.1055/a-1376-0970.

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