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.

Základní údaje

Originální název

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

Autoři

BLANCHETTE, V. S. (garant), L. ZUNINO, V. GRASSMANN, C. BARNES, M. D. CARCAO, J. CURTIN, S. JACKSON, L. KHOO, Vladimir KOMRSKA (203 Česká republika), D. LILLICRAP, M. MORFINI, Gabriela ROMANOVÁ (203 Česká republika, domácí), D. STEPHENS, Ester ZAPOTOCKA (203 Česká republika), M. L. RAND a Jan BLATNÝ (203 Česká republika, domácí)

Vydání

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

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30205 Hematology

Stát vydavatele

Německo

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 6.681

Kód RIV

RIV/00216224:14110/21:00121731

Organizační jednotka

Lékařská fakulta

DOI

http://dx.doi.org/10.1055/a-1376-0970

UT WoS

000640018700001

Klíčová slova anglicky

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

Štítky

14110321, 14110616, rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 25. 2. 2022 10:56, Mgr. Tereza Miškechová

Anotace

V originále

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.

Citovat

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 a 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, roč. 121, č. 10, s. 1326-1336. ISSN 0340-6245. Dostupné z: https://dx.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 = {http://dx.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 a 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, roč.~121, č.~10, s.~1326-1336. ISSN~0340-6245. Dostupné z: https://dx.doi.org/10.1055/a-1376-0970.

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