Abstract
Amaç: Hemofili A hastalarında kanamaların önlenmesi ve kas-iskelet sistemi fonksiyonlarının korunması amacıyla faktör VIII (FVIII) ile profilaksi önerilmektedir. Farmakokinetik (FK) verilerle düzenlenen profilaksi, kanamalardan korunmada ve tedavi maliyetinin azaltılmasında günümüzdeki en başarılı stratejidir.
Gereç ve Yöntemler: Bu çalışmada Bayesian yöntemi kullanan, myPKFiT web erişimli program aracılığı ile FVIII FK verileri hesaplanan 17 ağır hemofili A (ortanca yaş 11.5 yıl, ortanca kullandıkları faktör miktarı 25 U/kg) hastası sunuldu.
Bulgular: Çalışmaya dahil edilen hastaların ortalama FVIII yarılanma ömrü 9.1 saat (min-max: 6.8-11.4), ortalama FVIII klerensi 4.7 ml/saat/kg (min-max: 2.6-6.8), ortalama FVIII’in <%1’e kadar geçen süre ise 47 saat (min-max: 33-61) olarak bulundu. Hasta yaşlarının artmasıyla FVIII yarılanma ömrünün arttığı görüldü. Hastaların hedef FVIII çukur değeri %1 üzeri olacak şekilde haftalık doz ve takvim simulasyonu uygulandığında 3 hastanın kullandığı FVIII dozunun yeterli olduğu, 5 hastada FVIII dozunun yüksek olduğu, 9 hastanın ise kullandığı FVIII dozunun yeterli olmadığı görüldü.
Sonuç: Çalışmamızda FVIII FK verileri ile profilaksi doz ve sıklıkları düzenlendi.
References
- Petrini P, Valentino LA, Gringeri A, Re WM, Ewenstein B. Individualizing prophylaxis in hemophilia: a review. Expert Rev Hematol. 2015;8:237-46
- Pasca S, Milan M, Sarolo L, Zanon E. PK-driven prophylaxis versus standard prophylaxis: When a tailored treatment may be a real and achievable cost-saving approach in children with severe hemophilia A. Thromb Res. 2017;157:58-63.
- Megías-Vericat JE, Bonanad S, Haya S, Cid AR, Marqués MR, Monte E, Pérez-Alenda S, Bosch P, Querol F, Poveda JL. Bayesian pharmacokinetic-guided prophylaxis with recombinant factor VIII in severe or moderate haemophilia A. Thromb Res. 2019;174:151-162.
- McEneny-King A, Iorio A, Foster G, Edginton AN. The use of pharmacokinetics in dose individualization of factor VIII in the treatment of hemophilia A. Expert Opin Drug Metab Toxicol. 2016;12:1313-1321.
- Björkman S, Oh M, Spotts G, Schroth P, Fritsch S, Ewenstein BM, Casey K, Fischer K, Blanchette VS, Collins PW. Population pharmacokinetics of recombinant factor VIII: the relationships of pharmacokinetics to age and body weight. Blood. 2012;119:612-8.
- Björkman S. Limited blood sampling for pharmacokinetic dose tailoring of FVIII in the prophylactic treatment of haemophilia A. Haemophilia. 2010;16:597-605.
- Valentino LA, Mamonov V, Hellmann A, Quon DV, Chybicka A, Schroth P, Patrone L, Wong WY; Prophylaxis Study Group. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. J Thromb Haemost. 2012;10:359-67.
- Preijers T, van Moort I, Fijnvandraat K, Leebeek FWG, Cnossen MH, Mathôt RAA; ‘OPTI-CLOT’ Study Group. Cross-evaluation of Pharmacokinetic-Guided Dosing Tools for Factor VIII. Thromb Haemost. 2018;118:514-525.
- Pasca S, Zanon E. Savings without changing: How to use the MyPKfit® device to improve treatment strategies in a cohort of patients with haemophilia A. Thromb Res. 2019;183:1-3.
- Álvarez-Román MT, Fernandez-Bello I, de la Corte-Rodríguez H, Hernández-Moreno AL, Martín-Salces M, Butta-Coll N, Rivas-Pollmar MI, Rivas-Muñoz S, Jiménez-uste V. Experience of tailoring prophylaxis using factor VIII pharmacokinetic parameters estimated with myPKFiT(®) in patients with severe haemophilia A without inhibitors. Haemophilia. 2017;23:e50-e54.
- Collins PW, Blanchette VS, Fischer K, et al. Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb Haemost. 2009;7:413‐420.
- Nagao A, Yeung CHT, Germini F, Suzuki T. Clinical outcomes in hemophilia A patients undergoing tailoring of prophylaxis based on population-based pharmacokinetic dosing. Thromb Res. 2019;173:79-84.
- Mingot-Castellano ME, Parra R, Núñez R, Martorell M. 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(®). Haemophilia. 2018;24:e338-e343.
Abstract
References
- Petrini P, Valentino LA, Gringeri A, Re WM, Ewenstein B. Individualizing prophylaxis in hemophilia: a review. Expert Rev Hematol. 2015;8:237-46
- Pasca S, Milan M, Sarolo L, Zanon E. PK-driven prophylaxis versus standard prophylaxis: When a tailored treatment may be a real and achievable cost-saving approach in children with severe hemophilia A. Thromb Res. 2017;157:58-63.
- Megías-Vericat JE, Bonanad S, Haya S, Cid AR, Marqués MR, Monte E, Pérez-Alenda S, Bosch P, Querol F, Poveda JL. Bayesian pharmacokinetic-guided prophylaxis with recombinant factor VIII in severe or moderate haemophilia A. Thromb Res. 2019;174:151-162.
- McEneny-King A, Iorio A, Foster G, Edginton AN. The use of pharmacokinetics in dose individualization of factor VIII in the treatment of hemophilia A. Expert Opin Drug Metab Toxicol. 2016;12:1313-1321.
- Björkman S, Oh M, Spotts G, Schroth P, Fritsch S, Ewenstein BM, Casey K, Fischer K, Blanchette VS, Collins PW. Population pharmacokinetics of recombinant factor VIII: the relationships of pharmacokinetics to age and body weight. Blood. 2012;119:612-8.
- Björkman S. Limited blood sampling for pharmacokinetic dose tailoring of FVIII in the prophylactic treatment of haemophilia A. Haemophilia. 2010;16:597-605.
- Valentino LA, Mamonov V, Hellmann A, Quon DV, Chybicka A, Schroth P, Patrone L, Wong WY; Prophylaxis Study Group. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. J Thromb Haemost. 2012;10:359-67.
- Preijers T, van Moort I, Fijnvandraat K, Leebeek FWG, Cnossen MH, Mathôt RAA; ‘OPTI-CLOT’ Study Group. Cross-evaluation of Pharmacokinetic-Guided Dosing Tools for Factor VIII. Thromb Haemost. 2018;118:514-525.
- Pasca S, Zanon E. Savings without changing: How to use the MyPKfit® device to improve treatment strategies in a cohort of patients with haemophilia A. Thromb Res. 2019;183:1-3.
- Álvarez-Román MT, Fernandez-Bello I, de la Corte-Rodríguez H, Hernández-Moreno AL, Martín-Salces M, Butta-Coll N, Rivas-Pollmar MI, Rivas-Muñoz S, Jiménez-uste V. Experience of tailoring prophylaxis using factor VIII pharmacokinetic parameters estimated with myPKFiT(®) in patients with severe haemophilia A without inhibitors. Haemophilia. 2017;23:e50-e54.
- Collins PW, Blanchette VS, Fischer K, et al. Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb Haemost. 2009;7:413‐420.
- Nagao A, Yeung CHT, Germini F, Suzuki T. Clinical outcomes in hemophilia A patients undergoing tailoring of prophylaxis based on population-based pharmacokinetic dosing. Thromb Res. 2019;173:79-84.
- Mingot-Castellano ME, Parra R, Núñez R, Martorell M. 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(®). Haemophilia. 2018;24:e338-e343.
Details
| Primary Language | Turkish |
|---|---|
| Subjects | Clinical Sciences |
| Journal Section | ORIGINAL ARTICLES |
| Authors | |
| Publication Date | September 29, 2020 |
| Submission Date | June 27, 2020 |
| Published in Issue | Year 2020 Volume: 14 Issue: 5 |
The publication language of Turkish Journal of Pediatric Disease is English.
Manuscripts submitted to the Turkish Journal of Pediatric Disease will go through a double-blind peer-review process. Each submission will be reviewed by at least two external, independent peer reviewers who are experts in the field, in order to ensure an unbiased evaluation process. The editorial board will invite an external and independent editor to manage the evaluation processes of manuscripts submitted by editors or by the editorial board members of the journal. The Editor in Chief is the final authority in the decision-making process for all submissions. Articles accepted for publication in the Turkish Journal of Pediatrics are put in the order of publication, with at least 10 original articles in each issue, taking into account the acceptance dates. If the articles sent to the reviewers for evaluation are assessed as a senior for publication by the reviewers, the section editor and the editor considering all aspects (originality, high scientific quality and citation potential), it receives publication priority in addition to the articles assigned for the next issue.
The aim of the Turkish Journal of Pediatrics is to publish high-quality original research articles that will contribute to the international literature in the field of general pediatric health and diseases and its sub-branches. It also publishes editorial opinions, letters to the editor, reviews, case reports, book reviews, comments on previously published articles, meeting and conference proceedings, announcements, and biography. In addition to the field of child health and diseases, the journal also includes articles prepared in fields such as surgery, dentistry, public health, nutrition and dietetics, social services, human genetics, basic sciences, psychology, psychiatry, educational sciences, sociology and nursing, provided that they are related to this field. can be published.