Table 3.
Summary of the main clinical studies on recombinant factor IX (rFIX) concentrates
| Reference | Study design (patient population) | Main results |
|---|---|---|
| White et al34 | Double-blind, randomized, crossover (rFIX. n = 11; pdFIX, n = 11) | Significantly lower recovery for rFIX; safe and effective |
| Roth et al32 | Prospective PK, safety, and efficacy (rFIX, n = 56) | Low recovery; safe and effective |
| Poon et al25 | Retrospective observational (rFIX, n = 126; pdFIX, n = 75) | Significantly lower recovery for rFIX; in boys aged < 15 years, decreased recovery for both products; inhibitors, 2/244 (0.8%) |
| Ewenstein et al26 | Double-blind, two-period crossover (rFIX, n = 43; pdFIX, n = 43) | Wide product-related (decreased for rFIX) and patient-related variability in recovery |
| Kisker et al38 | Double-blind, two-period crossover (rFIX, n = 15; pdFIX, n = 15) | Decreased recovery for rFIX rFIX more expensive because of higher doses |
| Shapiro et al27 | Open-label, single-cohort (rFIX, n = 63) | Recovery depending on age; safe and effective |
| Lambert et al39 | Double-blind, randomized, PK crossover (rFIX, n = 34) | Recovery, safety, and efficacy of reformulated rFIX is comparable with original |
| Monahan et al40 | Prospective PK, safety, and efficacy (rFIX, n = 25) | One or two rFIX infusions per week as prophylaxis is well tolerated |
| Recht et al41 | Retrospective, safety (rFIX, n = 163; pdFIX, n = 88; rFIX and pdFIX, n = 71) | No difference in the frequency of allergic reactions or inhibitor development between pd- and rFIX concentrates |
| Berntorp et al33 | Prospective, observational cohort (rFIX, n = 218) | A low incidence of SAEs was detected (inhibitors 0.9%, thrombosis 0.5%, allergic events 3.7%) |
Abbreviations: pdFIX, plasma-derived factor IX; PK, pharmacokinetic; SAEs, serious adverse events.