BAY 81-8973 prophylaxis and pharmacokinetics in haemophilia A

TY - JOUR

T1 - BAY 81-8973 prophylaxis and pharmacokinetics in haemophilia A

T2 - Interim results from the TAURUS study

AU - Santoro, Cristina

AU - Fuh, Beng

AU - Le, Phu Quoc

AU - Maes, Philip

AU - Berrueco, Rubén

AU - Mingot-Castellano, Eva Maria

AU - von Mackensen, Sylvia

AU - Solms, Alexander

AU - Wang, Michael

N1 - © 2020 The Authors. European Journal of Haematology published by John Wiley & Sons Ltd.

PY - 2020/8

Y1 - 2020/8

N2 - OBJECTIVES: To report interim data from TAURUS, a study assessing real-world prophylactic treatment with unmodified, full-length recombinant FVIII BAY 81-8973 (Kovaltry® ; Bayer) indicated for haemophilia A.METHODS: TAURUS (NCT02830477) is an international, open-label, prospective, non-interventional, single-arm study with a one-year observation period (target N = 350). Patients have moderate or severe haemophilia A (FVIII ≤5% or ≤1%) and ≥50 exposure days to any FVIII product. Clinician- and patient-reported outcomes are captured on previous product use, changes in prophylaxis dose and dosing frequency, FVIII consumption, reported bleeding rates, treatment satisfaction and adherence, pharmacokinetic (PK) data (if available) and safety data.RESULTS: At cut-off, baseline data were available from 160 patients (89 had ≥6 months of follow-up data). Most patients had severe haemophilia A (85%), infused BAY 81-8973 ≥ 3×/wk (59%) and experienced a median number of total bleeds of 2.0 (non-annualised; 246 days median documentation period). Good levels of treatment satisfaction (Hemo-SATA,P ) and adherence (VERITAS-Pro) were maintained. TAURUS demonstrated a favourable PK profile of BAY 81-8973 in comparison with other standard half-life rFVIIIs and supported the WAPPS PopPK model. No patients developed inhibitors.CONCLUSIONS: TAURUS data demonstrate effective prophylaxis with BAY 81-8973 in the real world without compromising patient satisfaction or adherence.

AB - OBJECTIVES: To report interim data from TAURUS, a study assessing real-world prophylactic treatment with unmodified, full-length recombinant FVIII BAY 81-8973 (Kovaltry® ; Bayer) indicated for haemophilia A.METHODS: TAURUS (NCT02830477) is an international, open-label, prospective, non-interventional, single-arm study with a one-year observation period (target N = 350). Patients have moderate or severe haemophilia A (FVIII ≤5% or ≤1%) and ≥50 exposure days to any FVIII product. Clinician- and patient-reported outcomes are captured on previous product use, changes in prophylaxis dose and dosing frequency, FVIII consumption, reported bleeding rates, treatment satisfaction and adherence, pharmacokinetic (PK) data (if available) and safety data.RESULTS: At cut-off, baseline data were available from 160 patients (89 had ≥6 months of follow-up data). Most patients had severe haemophilia A (85%), infused BAY 81-8973 ≥ 3×/wk (59%) and experienced a median number of total bleeds of 2.0 (non-annualised; 246 days median documentation period). Good levels of treatment satisfaction (Hemo-SATA,P ) and adherence (VERITAS-Pro) were maintained. TAURUS demonstrated a favourable PK profile of BAY 81-8973 in comparison with other standard half-life rFVIIIs and supported the WAPPS PopPK model. No patients developed inhibitors.CONCLUSIONS: TAURUS data demonstrate effective prophylaxis with BAY 81-8973 in the real world without compromising patient satisfaction or adherence.

U2 - 10.1111/ejh.13420

DO - 10.1111/ejh.13420

M3 - SCORING: Journal article

C2 - 32259334

VL - 105

SP - 164

EP - 172

JO - EUR J HAEMATOL

JF - EUR J HAEMATOL

SN - 0902-4441

IS - 2

ER -