2021
Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B
Nolan B, Klukowska A, Shapiro A, Rauch A, Recht M, Ragni M, Curtin J, Gunawardena S, Mukhopadhyay S, Jayawardene D, Winding B, Fischer K, Liesner R. Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B. Blood Advances 2021, 5: 2732-2739. PMID: 34242387, PMCID: PMC8288672, DOI: 10.1182/bloodadvances.2020004085.Peer-Reviewed Original ResearchConceptsRecombinant factor IX Fc fusion proteinHemophilia BUntreated patientsBleeding episodesFamily history of inhibitorsExposure daysTreatment of bleeding episodesLow-titer inhibitorsMedian dosing intervalHigh-titer inhibitorsAverage weekly doseHistory of inhibitorsSecondary end pointsPhase 3 studyAdverse event profilePrimary end pointMedian ABRBleed resolutionOpen-labelWeekly dosesDosing intervalEvent profileAdverse eventsBleeding rateEvaluate safety
2012
Experience of Advate rAHF-PFM in previously untreated patients and minimally treated patients with haemophilia A
Auerswald G, Thompson A, Recht M, Brown D, Liesner R, Guzmán-Becerra N, Dyck-Jones J, Ewenstein B, Abbuehl B. Experience of Advate rAHF-PFM in previously untreated patients and minimally treated patients with haemophilia A. Thrombosis And Haemostasis 2012, 107: 1072-1082. PMID: 22476554, PMCID: PMC6292131, DOI: 10.1160/th11-09-0642.Peer-Reviewed Original ResearchMeSH KeywordsAntibodiesDrug Administration ScheduleEthnicityEuropeFactor VIIIFemaleHemophilia AHemorrhageHemostasisHemostaticsHumansInfantInfant, NewbornLinear ModelsLogistic ModelsMaleNorth AmericaOdds RatioProspective StudiesRisk AssessmentRisk FactorsSeverity of Illness IndexTime FactorsTreatment OutcomeConceptsMinimally treated patientsFamily history of inhibitorsHistory of inhibitorsRAHF-PFMBleeding episodesNon-Caucasian ethnicityUntreated patientsHaemostatic efficacyHemophilia AAdverse eventsHigh risk of inhibitor developmentFamily historySevere/moderately severe hemophilia ARisk of inhibitor developmentTreat bleeding episodesSevere hemophilia AAssociated with higher riskSevere hemophilia A.Prophylactic treatment regimensExposure daysInhibitor developmentPerioperative coverageRating surgeriesProspective trialsTreatment regimens
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