Bayesian approach to the assessment of the population-specific risk of inhibitors in hemophilia A patients: a case study

References

• W CollinsPChalmersEHartDDiagnosis and management of acquired coagulation inhibitors: a guideline from UKHCDOBr J Haematol2013162675877323889317
   PubMed Web of Science ®Google Scholar
• IorioAEpidemiology of inhibitors in hemophiliaLeeCABerntorpEEHootsWKTextbook of HemophliaOxfordJohn Wiley & Sons, Ltd20145358
   Google Scholar
• CaramCde SouzaRGde SousaJCThe long-term course of factor VIII inhibitors in patients with congenital haemophilia A without immune tolerance inductionThromb Haemost20111051596521057702
   PubMed Web of Science ®Google Scholar
• HayCRMPalmerBChalmersEUnited Kingdom Haemophilia Centre Doctors’ Organisation (UKHCDO)Incidence of factor VIII inhibitors throughout life in severe hemophilia A in the United KingdomBlood2011117236367637021471523
   PubMed Web of Science ®Google Scholar
• StieltjesNTorchetMFMisrahiLEpidemiological survey of haemophiliacs with inhibitors in France: orthopaedic status, quality of life and cost – the “Statut Orthopédique des Patients Hémophiles” avec Inhibiteur studyBlood Coagul Fibrinolysis200920141120527720
   PubMed Web of Science ®Google Scholar
• GringeriAMonziniMTagarielloGOccurrence of inhibitors in previously untreated or minimally treated patients with haemophilia A after exposure to a plasma-derived solvent-detergent factor VIII concentrateHaemophilia2006122128132
   Web of Science ®Google Scholar
• AstermarkJAltisentCBatorovaAEuropean Haemophilia Therapy Standardisation BoardNon-genetic risk factors and the development of inhibitors in haemophilia: a comprehensive review and consensus reportHaemophilia201016574776620398077
   PubMed Web of Science ®Google Scholar
• CoppolaASantoroCTagliaferriAFranchiniMDI MinnoGUnderstanding inhibitor development in haemophilia A: towards clinical prediction and prevention strategiesHaemophilia201016suppl 11319
   PubMed Web of Science ®Google Scholar
• IorioAPuccettiPMakrisMClotting factor concentrate switching and inhibitor development in hemophilia ABlood2012120472072722692511
   PubMed Web of Science ®Google Scholar
• LeeCALillicrapDAstermarkJInhibitor development in hemophiliacs: the roles of genetic versus environmental factorsSemin Thromb Hemost200632suppl 2101416804830
   PubMed Web of Science ®Google Scholar
• GouwSCvan den BergHMFischerKPedNet and Research of Determinants of INhibitor development (RODIN) Study GroupIntensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN studyBlood2013121204046405523553768
   PubMed Web of Science ®Google Scholar
• GouwSCvan den BergHMThe multifactorial etiology of inhibitor development in hemophilia: genetics and environmentSemin Thromb Hemost200935872373420169509
   PubMed Web of Science ®Google Scholar
• GouwSCvan der BomJGMarijke van den BergHTreatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort studyBlood2007109114648465417289808
   PubMed Web of Science ®Google Scholar
• PavlovaADelevDLacroix-DesmazesSImpact of polymorphisms of the major histocompatibility complex class II, interleukin-10, tumor necrosis factor-alpha and cytotoxic T-lymphocyte antigen-4 genes on inhibitor development in severe hemophilia AJ Thromb Haemost20097122006201519817985
   PubMed Web of Science ®Google Scholar
• AstermarkJLacroix-DesmazesSRedingMTInhibitor developmentHaemophilia200814suppl 33642
   PubMed Web of Science ®Google Scholar
• Saint-RemyJ-MReipertBMMonroeDMModels for assessing immunogenicity and efficacy of new therapeutics for the treatment of haemophiliaHaemophilia201218suppl 4434722726082
   PubMed Web of Science ®Google Scholar
• MatinoDLillicrapDAstermarkJSwitching clotting factor concentrates: considerations in estimating the risk of immunogenicityHaemophilia201420220020624533949
   PubMed Web of Science ®Google Scholar
• KesselheimASMyersJAAvornJCharacteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancerJAMA2011305222320232621642684
   PubMed Web of Science ®Google Scholar
• IorioAMarcucciMClinical trials and haemophilia : does the Bayesian approach make the ideal and desirable good friends ?Haemophilia200915490090319473413
   PubMed Web of Science ®Google Scholar
• ChowS-CChangMAdaptive design methods in clinical trials – a reviewOrphanet J Rare Dis200831118454853
   PubMed Web of Science ®Google Scholar
• HonkanenVESiegelAFSzalaiJPBergerVFeldmanBMSiegelJNA three-stage clinical trial design for rare disordersStat Med200120203009302111590629
   PubMed Web of Science ®Google Scholar
• LilfordRJThorntonJGBraunholtzDClinical trials and rare diseases: a way out of a conundrumBMJ19953117020162116258555809
   PubMed Web of Science ®Google Scholar
• BeheraMKumarASoaresHPSokolLDjulbegovicBEvidence-based medicine for rare diseases: implications for data interpretation and clinical trial designCancer Control200714216016617387301
   PubMedGoogle Scholar
• Committee on Strategies for Small-Number-Participant Clinical Research Trials, Board on Health Sciences Policy Committee on Strategies for Small-Number-Participant Clinical Research Trials B on HSPSmall Clinical Trials: Issues and ChallengesWashington, DCNational Academy Press2001
   Google Scholar
• HarrellFELeeKLMarkDBMultivariable prognostic models: issues in developing models, evaluating assumptions and adequacy, and measuring and reducing errorsStat Med19961543613878668867
   PubMed Web of Science ®Google Scholar
• VandenbrouckeJPWhen are observational studies as credible as randomised trials?Lancet200436394221728173115158638
   PubMed Web of Science ®Google Scholar
• HornbergerJWroneEWhen to base clinical policies on observational versus randomized trial dataAnn Intern Med19971278 pt 26977039382383
   PubMed Web of Science ®Google Scholar
• Von ElmEAltmanDGEggerMSTROBE InitiativeThe strengthening the reporting of observational studies in epidemiology (STROBE) statement: guidelines for reporting observational studiesEpidemiology200718680080418049194
   PubMed Web of Science ®Google Scholar
• KalilACSunJBayesian methodology for the design and interpretation of clinical trials in critical care medicineCrit Care Med2014422267227725226118
   PubMed Web of Science ®Google Scholar
• SpiegelhaterDJAbramsKRMylesJPBayesian Approaches to Clinical Trials and Health-Care Evaluation1st edChichester, West Sussex, EnglandJohn Wiley & Sons Ltd2004
   Google Scholar
• LunnDJThomasABestNSpiegelhalterDWinBUGS – a Bayesian modelling framework: concepts, structure, and extensibilityStat Comput200010325337
   Web of Science ®Google Scholar
• OldenburgJGoudemandJValentinoLPostauthorization safety surveillance of ADVATE [antihaemophilic factor (recombinant), plasma/albumin-free method] demonstrates efficacy, safety and low-risk for immunogenicity in routine clinical practiceHaemophilia201016686687720579113
   PubMed Web of Science ®Google Scholar
• IorioAMarcucciMChengJPatient data meta-analysis of post-authorization safety surveillance (PASS) studies of haemophilia A patients treated with rAHF-PFMHaemophilia201420677778325039809
   PubMed Web of Science ®Google Scholar
• RomanovVMarcucciMChengJThabaneLIorioAEvaluation of safety and effectiveness of factor VIII treatment in hemophilia A patients with low titer inhibitors or a personal history of inhibitorThromb Haemost201511335664
   Web of Science ®Google Scholar
• TarantinoMDCollinsPWHayCRMRAHF-PFM Clinical Study GroupClinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin-free method: pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia AHaemophilia200410542843715357767
   PubMed Web of Science ®Google Scholar
• XiMMakrisMMarcucciMSantagostinoEMannucciPMIorioAInhibitor development in previously treated hemophilia A patients: a systematic review, meta-analysis, and meta-regressionJ Thromb Haemost20131191655166223802542
   PubMed Web of Science ®Google Scholar
• FischerKLassilaRPeyvandiFEUHASS ParticipantsInhibitor development in haemophilia according to concentrate four-year results from the European haemophilia safety surveillance (EUHASS) projectThromb Haemost2015113596897525567324
   PubMed Web of Science ®Google Scholar
• DimicheleDMLacroix-DesmazesSPeyvandiFSrivastavaARosendaalFRDesign of clinical trials for new products in hemophilia: communication from the SSC of the ISTHJ Thromb Haemost201513587687925728341
   PubMed Web of Science ®Google Scholar
• AledortLMHarmonization of clinical trial guidelines for assessing the risk of inhibitor development in hemophilia A treatmentJ Thromb Haemost20119342342721143382
   PubMed Web of Science ®Google Scholar
• Department of Health and Human Services Food And Drug Administration, Center For Biologics Evaluation And Research, Lister Hill Center National Institutes of HealthWorkshop on Factor Viii InhibitorsNovember 21, 2003Miller Reporting Co., IncWashington DC
   Google Scholar
• SalpeterSRChengJThabaneLBuckleyNSSalpeterEEBayesian meta-analysis of hormone therapy and mortality in younger postmenopausal womenAm J Med2009122111016.e1022.e19854329
   PubMed Web of Science ®Google Scholar
• GreenlandSPooleCLiving with P valuesEpidemiology2013241626823232611
   PubMed Web of Science ®Google Scholar
• LindleyDVAn introduction to Bayesian inference and decisionJ Oper Res Soc1974252336337
   Google Scholar