{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,25]],"date-time":"2026-01-25T23:05:48Z","timestamp":1769382348540,"version":"3.49.0"},"reference-count":36,"publisher":"Georg Thieme Verlag KG","issue":"07","funder":[{"name":"Bayer HealthCare as a phase IV"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Thromb Haemost"],"published-print":{"date-parts":[[2015]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>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 On-demand Therapy Through Economic Report (POTTER) study (Clinical-Trials.gov NCT01159587) compared long-term late secondary prophylaxis (recombinant FVIII-FS 20\u201330 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 &gt; 5-year period (2004\u20132010). 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\u201325 and 26\u201355 years). Patients receiving prophylaxis experienced a significantly lower number of joint bleeding episodes vs the on\u2013demand 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&lt; 0.001), better orthopaedic (p=0.0019) and Pettersson (p=0.0177) scores, better HRQoL, and fewer days of everyday activities lost (p&lt; 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.<\/jats:p>","DOI":"10.1160\/th14-05-0407","type":"journal-article","created":{"date-parts":[[2015,4,9]],"date-time":"2015-04-09T08:40:01Z","timestamp":1428568801000},"page":"35-45","source":"Crossref","is-referenced-by-count":81,"title":["Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study"],"prefix":"10.1160","volume":"114","author":[{"given":"Giulio","family":"Feola","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Angelo Claudio","family":"Molinari","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cristina","family":"Santoro","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gianna 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