Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: The POTTER study
| Autor: | Tagliaferri, Annarita, Feola, Giulio, Molinari, Angelo Claudio, Santoro, Cristina, Rivolta, Gianna Franca, Cultrera, Dorina Bianca, Gagliano, Fabio, Zanon, Ezio, Mancuso, Maria Elisa, Valdrè, Lelia, Mameli, Luciana, Amoresano, Susanna, Mathew, Prasad, Tagliaferri, A., Rivolta, G. F., Di Perna, C., Feola, G., Molinari, A. C., Banov, L., Santoro, C., Mazzucconi, M. G., Cultrera, D. B., Gagliano, F., Zanon, E., Mancuso, M. E., Santagostino, E., Valdrè, L., Rodorigo, G., Mameli, L., Piseddu, G., Coppola, A., Negri, N., Amoresano, S., Mathew, P., COPPOLA, ANTONIO, DI MINNO, GIOVANNI |
|---|---|
| Přispěvatelé: | Tagliaferri, Annarita, Feola, Giulio, Molinari, Angelo Claudio, Santoro, Cristina, Rivolta, Gianna Franca, Cultrera, Dorina Bianca, Gagliano, Fabio, Zanon, Ezio, Mancuso, Maria Elisa, Valdrè, Lelia, Mameli, Luciana, Amoresano, Susanna, Mathew, Prasad, Coppola, Antonio, Tagliaferri, A., Rivolta, G. F., Di Perna, C., Feola, G., Molinari, A. C., Banov, L., Santoro, C., Mazzucconi, M. G., Cultrera, D. B., Gagliano, F., Zanon, E., Mancuso, M. E., Santagostino, E., Valdrè, L., Rodorigo, G., Mameli, L., Piseddu, G., Coppola, A., DI MINNO, Giovanni, Negri, N., Amoresano, S., Mathew, P. |
| Jazyk: | angličtina |
| Rok vydání: | 2015 |
| Předmět: |
Adult
Male Pediatrics medicine.medical_specialty Haemophilia Time Factors Adolescent Cost-Benefit Analysis Health-related quality of life 030204 cardiovascular system & hematology Hemophilia A Severity of Illness Index Drug Administration Schedule Drug Costs Hemostatics 03 medical and health sciences Young Adult 0302 clinical medicine Quality of life Haemophilic arthropathy On demand Hemarthrosis Clinical endpoint Medicine Humans Prospective Studies Prophylaxi Child Bleeding episodes Factor VIII business.industry Bleeding Age Factors Secondary prophylaxis Hematology Middle Aged medicine.disease On demand treatment Treatment Outcome Italy Quality of Life Severe haemophilia A business 030215 immunology |
| Popis: | SummaryRigorous 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 On-demand 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. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|