Impact of trial design on the estimation of drug potency and power in clinical trials of haemophilia with inhibitors

[1]  N. Kotani,et al.  A Pharmacometric Approach to Substitute for a Conventional Dose-Finding Study in Rare Diseases: Example of Phase III Dose Selection for Emicizumab in Hemophilia A , 2017, Clinical Pharmacokinetics.

[2]  F. Leebeek,et al.  In silico evaluation of limited blood sampling strategies for individualized recombinant factor IX prophylaxis in hemophilia B patients , 2017, Journal of thrombosis and haemostasis : JTH.

[3]  L. Christrup,et al.  Analysis of opioid consumption in clinical trials: a simulation based analysis of power of four approaches , 2017, Journal of Pharmacokinetics and Pharmacodynamics.

[4]  S. Rasmussen,et al.  Repeated Time-to-event Analysis of Consecutive Analgesic Events in Postoperative Pain , 2015, Anesthesiology.

[5]  Thomas B. L. Kirkwood,et al.  Deciphering death: a commentary on Gompertz (1825) ‘On the nature of the function expressive of the law of human mortality, and on a new mode of determining the value of life contingencies’ , 2015, Philosophical Transactions of the Royal Society B: Biological Sciences.

[6]  V. Jiménez‐Yuste,et al.  The pharmacokinetics of a B‐domain truncated recombinant factor VIII, turoctocog alfa (NovoEight®), in patients with hemophilia A , 2015, Journal of thrombosis and haemostasis : JTH.

[7]  E. Berntorp,et al.  Methods for individualising factor VIII dosing in prophylaxis , 2014, European journal of haematology. Supplementum.

[8]  Michael Hay,et al.  Clinical development success rates for investigational drugs , 2014, Nature Biotechnology.

[9]  A. Čeponis,et al.  Rapid musculoskeletal ultrasound for painful episodes in adult haemophilia patients , 2013, Haemophilia : the official journal of the World Federation of Hemophilia.

[10]  S. Antunes,et al.  Randomized comparison of prophylaxis and on-demand regimens with FEIBA NF in the treatment of haemophilia A and B with inhibitors , 2013, Haemophilia : the official journal of the World Federation of Hemophilia.

[11]  P. Giangrande,et al.  40K glycoPEGylated, recombinant FVIIa: 3‐month, double‐blind, randomized trial of safety, pharmacokinetics and preliminary efficacy in hemophilia patients with inhibitors , 2013, Journal of thrombosis and haemostasis : JTH.

[12]  R J Keizer,et al.  Modeling and Simulation Workbench for NONMEM: Tutorial on Pirana, PsN, and Xpose , 2013, CPT: pharmacometrics & systems pharmacology.

[13]  N. Holford A Time to Event Tutorial for Pharmacometricians , 2013, CPT: pharmacometrics & systems pharmacology.

[14]  Erik Berntorp,et al.  Clinical trial design in haemophilia , 2012, Haemophilia : the official journal of the World Federation of Hemophilia.

[15]  Maria Blettner,et al.  On the proper use of the crossover design in clinical trials: part 18 of a series on evaluation of scientific publications. , 2012, Deutsches Arzteblatt international.

[16]  M. Karlsson,et al.  Transient Lower Esophageal Sphincter Relaxation Pharmacokinetic-Pharmacodynamic Modeling: Count Model and Repeated Time-To-Event Model , 2011, Journal of Pharmacology and Experimental Therapeutics.

[17]  N. Holford,et al.  Clinical Trial Simulation: A Review , 2010, Clinical pharmacology and therapeutics.

[18]  S. Björkman Limited blood sampling for pharmacokinetic dose tailoring of FVIII in the prophylactic treatment of haemophilia A , 2010, Haemophilia : the official journal of the World Federation of Hemophilia.

[19]  Mats O. Karlsson,et al.  Modelling overdispersion and Markovian features in count data , 2009, Journal of Pharmacokinetics and Pharmacodynamics.

[20]  William F Dunn,et al.  Simulation for clinical research trials: a theoretical outline. , 2009, Journal of critical care.

[21]  A. Shapiro Anti-hemophilic factor (recombinant), plasma/albumin-free method (octocog-alpha; ADVATE®) in the management of hemophilia A , 2007, Vascular health and risk management.

[22]  R. Rossaint,et al.  Pharmacokinetics of recombinant activated factor VII in trauma patients with severe bleeding , 2006, Critical care.

[23]  Mats O. Karlsson,et al.  Evaluation of Type I Error Rates When Modeling Ordered Categorical Data in NONMEM , 2004, Journal of Pharmacokinetics and Pharmacodynamics.

[24]  K. Pasi,et al.  Haemophilias A and B , 2003, The Lancet.

[25]  Mats O. Karlsson,et al.  Assessment of Type I Error Rates for the Statistical Sub-model in NONMEM , 2002, Journal of Pharmacokinetics and Pharmacodynamics.

[26]  Mats O. Karlsson,et al.  Assessment of Actual Significance Levels for Covariate Effects in NONMEM , 2001, Journal of Pharmacokinetics and Pharmacodynamics.

[27]  Lewis B. Sheiner,et al.  A Population Pharmacokinetic–Pharmacodynamic Analysis of Repeated Measures Time-to-Event Pharmacodynamic Responses: The Antiemetic Effect of Ondansetron , 1999, Journal of Pharmacokinetics and Biopharmaceutics.

[28]  D J Sargent,et al.  A general framework for random effects survival analysis in the Cox proportional hazards setting. , 1998, Biometrics.

[29]  K. Land,et al.  A Comparison of Poisson, Negative Binomial, and Semiparametric Mixed Poisson Regression Models , 1996 .

[30]  S. Shapiro,et al.  Efficacy of prothrombin-complex concentrates in hemophiliacs with antibodies to factor VIII: a multicenter therapeutic trial. , 1980, The New England journal of medicine.

[31]  M. Ozelo,et al.  Key issues in inhibitor management in patients with haemophilia. , 2014, Blood transfusion = Trasfusione del sangue.

[32]  L. Haan,et al.  Extreme value theory : an introduction , 2006 .

[33]  C. H. Evans,et al.  Small Clinical Trials: Issues and Challenges , 2001 .