TY - JOUR
T1 - Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A
T2 - The POTTER study
AU - Tagliaferri, Annarita
AU - Feola, Giulio
AU - Molinari, Angelo Claudio
AU - Santoro, Cristina
AU - Rivolta, Gianna Franca
AU - Cultrera, Dorina Bianca
AU - Gagliano, Fabio
AU - Zanon, Ezio
AU - Mancuso, Maria Elisa
AU - Valdrè, Lelia
AU - Mameli, Luciana
AU - Amoresano, Susanna
AU - Mathew, Prasad
AU - Coppola, Antonio
AU - Tagliaferri, A.
AU - Rivolta, G. F.
AU - Di Perna, C.
AU - Feola, G.
AU - Molinari, A. C.
AU - Banov, L.
AU - Santoro, C.
AU - Mazzucconi, M. G.
AU - Cultrera, D. B.
AU - Gagliano, F.
AU - Zanon, E.
AU - Mancuso, M. E.
AU - Santagostino, E.
AU - Valdrè, L.
AU - Rodorigo, G.
AU - Mameli, L.
AU - Piseddu, G.
AU - Coppola, A.
AU - Di Minno, G.
AU - Negri, N.
AU - Amoresano, S.
AU - Mathew, P.
PY - 2015
Y1 - 2015
N2 - Rigorous evidence is lacking on long-term outcomes of factor VIII (FVIII) prophylaxis initiated in adolescent or adult patients with severe haemophilia A. The prospective, open-label Prophylaxis versus Ondemand Therapy Through Economic Report (POTTER) study (Clinical- Trials.gov NCT01159587) compared long-term late secondary prophylaxis (recombinant FVIII-FS 20–30 IU/kg thrice weekly) with on-demand treatment in patients aged 12 to 55 years with severe haemophilia A. The annual number of joint bleeding episodes (primary endpoint), total bleeding episodes, orthopaedic and radiologic (Pettersson) scores, health-related quality of life (HRQoL), pharmacoeconomic impact, and safety were evaluated over a > 5-year period (2004–2010). Fifty-eight patients were enrolled at 11 centres in Italy; 53 (27 prophylaxis, 26 on demand) were evaluated and stratified into 2 age subgroups (12–25 and 26–55 years). Patients receiving prophylaxis experienced a significantly lower number of joint bleeding episodes vs the on–demand group (annualised bleeding rate, 1.97 vs 16.80 and 2.46 vs 16.71 in younger and older patients, respectively; p=0.0043). Results were similar for total bleeding episodes. Prophylaxis was associated with significantly fewer target joints (p<0.001), better orthopaedic (p=0.0019) and Pettersson (p=0.0177) scores, better HRQoL, and fewer days of everyday activities lost (p<0.0001) but required significantly higher FVIII product consumption. The POTTER study is the first prospective, controlled trial documenting long-term benefits of late secondary prophylaxis in adolescents and adults with severe haemophilia A. The benefits of reduced bleeding frequency, improved joint status, and HRQoL may offset the higher FVIII consumption and costs.
AB - Rigorous evidence is lacking on long-term outcomes of factor VIII (FVIII) prophylaxis initiated in adolescent or adult patients with severe haemophilia A. The prospective, open-label Prophylaxis versus Ondemand Therapy Through Economic Report (POTTER) study (Clinical- Trials.gov NCT01159587) compared long-term late secondary prophylaxis (recombinant FVIII-FS 20–30 IU/kg thrice weekly) with on-demand treatment in patients aged 12 to 55 years with severe haemophilia A. The annual number of joint bleeding episodes (primary endpoint), total bleeding episodes, orthopaedic and radiologic (Pettersson) scores, health-related quality of life (HRQoL), pharmacoeconomic impact, and safety were evaluated over a > 5-year period (2004–2010). Fifty-eight patients were enrolled at 11 centres in Italy; 53 (27 prophylaxis, 26 on demand) were evaluated and stratified into 2 age subgroups (12–25 and 26–55 years). Patients receiving prophylaxis experienced a significantly lower number of joint bleeding episodes vs the on–demand group (annualised bleeding rate, 1.97 vs 16.80 and 2.46 vs 16.71 in younger and older patients, respectively; p=0.0043). Results were similar for total bleeding episodes. Prophylaxis was associated with significantly fewer target joints (p<0.001), better orthopaedic (p=0.0019) and Pettersson (p=0.0177) scores, better HRQoL, and fewer days of everyday activities lost (p<0.0001) but required significantly higher FVIII product consumption. The POTTER study is the first prospective, controlled trial documenting long-term benefits of late secondary prophylaxis in adolescents and adults with severe haemophilia A. The benefits of reduced bleeding frequency, improved joint status, and HRQoL may offset the higher FVIII consumption and costs.
KW - Bleeding
KW - Haemophilia
KW - Haemophilic arthropathy
KW - Health-related quality of life
KW - Prophylaxis
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U2 - 10.1160/TH14-05-0407
DO - 10.1160/TH14-05-0407
M3 - Article
C2 - 25855376
AN - SCOPUS:84934783282
VL - 114
SP - 35
EP - 45
JO - Thrombosis and Haemostasis
JF - Thrombosis and Haemostasis
SN - 0340-6245
IS - 1
ER -