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1993 1
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Page 1
Clinical pharmacology of emicizumab for the treatment of hemophilia A.
Yoneyama K, Schmitt C, Portron A, Kiialainen A, Kotani N, Jaminion F, Retout S, Adamkewicz JI. Yoneyama K, et al. Expert Rev Clin Pharmacol. 2023 Jul-Dec;16(9):775-790. doi: 10.1080/17512433.2023.2243213. Epub 2023 Aug 21. Expert Rev Clin Pharmacol. 2023. PMID: 37529848 Review.
INTRODUCTION: Emicizumab is a humanized bispecific antibody approved for the routine prophylaxis of bleeding episodes in patients with hemophilia A (PwHA) regardless of the presence of factor VIII (FVIII) inhibitors. ...Once-weekly to every-4-week subcutaneous (SC) adminis …
INTRODUCTION: Emicizumab is a humanized bispecific antibody approved for the routine prophylaxis of bleeding episodes in patients with he
The molecular basis of hemophilia A: genotype-phenotype relationships and inhibitor development.
Goodeve AC, Peake IR. Goodeve AC, et al. Semin Thromb Hemost. 2003 Feb;29(1):23-30. doi: 10.1055/s-2003-37936. Semin Thromb Hemost. 2003. PMID: 12640561 Review.
Inhibitory antibodies that develop in a proportion of patients with hemophilia A following replacement therapy are now known to correlate with FVIII mutation type and location. This correlation is demonstrated, and a potential algorithm for predicting inhibitor deve …
Inhibitory antibodies that develop in a proportion of patients with hemophilia A following replacement therapy are now known t …
Inhibitors in childhood hemophilia A: genetic and treatment-related risk factors for development and eradication.
DiMichele DM. DiMichele DM. Pediatr Blood Cancer. 2013;60 Suppl 1:S30-3. doi: 10.1002/pbc.24338. Epub 2012 Oct 25. Pediatr Blood Cancer. 2013. PMID: 23109404 Review.
The development of neutralizing antibodies remains a serious complication of hemophilia replacement therapy. Factor VIII inhibiting antibodies (inhibitors) occur commonly following replacement therapy in hemophilia A, creating a significant burden of c …
The development of neutralizing antibodies remains a serious complication of hemophilia replacement therapy. Factor VIII inhib …
Treatment of inherited coagulation disorders.
Cohen AJ, Kessler CM. Cohen AJ, et al. Am J Med. 1995 Dec;99(6):675-82. doi: 10.1016/s0002-9343(99)80256-3. Am J Med. 1995. PMID: 7503092 Review.
Diagnostic evaluation with coagulation screening tests, followed by confirmation with coagulation factor assays, is essential for appropriate management. For moderate-to-severe hemophilia, treatment includes coagulation factor replacement with purified, plasma-derived coag …
Diagnostic evaluation with coagulation screening tests, followed by confirmation with coagulation factor assays, is essential for appropriat …
Molecular and clinical predictors of inhibitor risk and its prevention and treatment in mild hemophilia A.
Castaman G, Fijnvandraat K. Castaman G, et al. Blood. 2014 Oct 9;124(15):2333-6. doi: 10.1182/blood-2014-02-546127. Epub 2014 Aug 18. Blood. 2014. PMID: 25139352 Free PMC article. Review.
The risk for inhibitor development in mild hemophilia A (factor VIII levels between 5 and 40 U/dL) is larger than previously anticipated, continues throughout life, and is particularly associated with certain mutations in F8. ...
The risk for inhibitor development in mild hemophilia A (factor VIII levels between 5 and 40 U/dL) is larger than previously a …
International workshop on immune tolerance induction: consensus recommendations.
DiMichele DM, Hoots WK, Pipe SW, Rivard GE, Santagostino E. DiMichele DM, et al. Haemophilia. 2007 Jul;13 Suppl 1:1-22. doi: 10.1111/j.1365-2516.2007.01497.x. Haemophilia. 2007. PMID: 17593277 Free article.
Although immune tolerance induction (ITI) has been used for 30 years to eliminate inhibitors and restore normal factor pharmacokinetics in patients with hemophilia, there is a paucity of scientific evidence to guide therapeutic decision-making. In an effort to provi …
Although immune tolerance induction (ITI) has been used for 30 years to eliminate inhibitors and restore normal factor pharmacokinetics in p …
The von Willebrand factor-binding aptamer rondaptivon pegol as a treatment for severe and nonsevere hemophilia A.
Ay C, Kovacevic KD, Kraemmer D, Schoergenhofer C, Gelbenegger G, Firbas C, Quehenberger P, Jilma-Stohlawetz P, Gilbert JC, Zhu S, Beliveau M, Koenig F, Iorio A, Jilma B, Derhaschnig U, Pabinger I. Ay C, et al. Blood. 2023 Mar 9;141(10):1147-1158. doi: 10.1182/blood.2022016571. Blood. 2023. PMID: 36108308 Free PMC article.
This trial assessed its safety, pharmacokinetics, and pharmacodynamics in hemophilia A. Nineteen adult patients (ages 20-62 years, 4 women) with hemophilia A (8 mild, 2 moderate, and 9 severe) received subcutaneous injections of rondaptivon pegol. ...Median F …
This trial assessed its safety, pharmacokinetics, and pharmacodynamics in hemophilia A. Nineteen adult patients (ages 20-62 years, 4 …
Management and outcomes of mild hemophiliacs and hemophilia carriers during pregnancy and peripartum period: a hemophilia treatment center experience in the United States.
Lim HI, DeSancho MT. Lim HI, et al. J Matern Fetal Neonatal Med. 2022 Dec;35(25):9759-9764. doi: 10.1080/14767058.2022.2051699. Epub 2022 Apr 12. J Matern Fetal Neonatal Med. 2022. PMID: 35414333
AIM: To describe the management and outcomes of mild hemophiliacs and hemophilia carriers during assisted conception, pregnancy, peripartum and post-partum period at our hemophilia treatment center (HTC). ...RESULTS: There was a total of 18 pregnancies in 12 …
AIM: To describe the management and outcomes of mild hemophiliacs and hemophilia carriers during assisted conception, pregnanc …
Clinical efficacy and determinants of response to treatment with desmopressin in mild hemophilia a.
Di Perna C, Riccardi F, Franchini M, Rivolta GF, Pattacini C, Tagliaferri A. Di Perna C, et al. Semin Thromb Hemost. 2013 Oct;39(7):732-9. doi: 10.1055/s-0033-1354418. Epub 2013 Sep 12. Semin Thromb Hemost. 2013. PMID: 24030345
Although desmopressin (DDAVP) is considered as the treatment of choice for many patients with mild hemophilia A, several aspects of DDAVP therapy remain unclear, including the rate and type of response and the molecular determinants of its clinical efficacy. …
Although desmopressin (DDAVP) is considered as the treatment of choice for many patients with mild hemophilia A, several aspec …
17 results