Table 1.
Summary of data: pegloticase and immunomodulation
| Author | Type of studya | IMT and run-in (time frame)b | Responders,n/N (%) |
|---|---|---|---|
| Berhanu et al. [9] | Case report | Azathioprine (2 weeks) | 1/1 (100) |
| Freyne [11] | Case report | Mycophenolate mofetil and cyclosporine (background therapy) | 1/1 (100) |
| Botson et al. [14••] | Proof-of-concept case series | Methotrexate (4 weeks) | 10/10 (100) |
| Albert et al. [8] | Case series | Methotrexate (14–35 days)c | 8/10 (80) |
| Bessen et al. [10] | Case series | Methotrexate, azathioprine, or cyclosporine (at first infusion) | 7/7 (100) |
| Masri et al. [12] | Retrospective case study | Leflunomide (variable) | 4/6 (67) |
| Botson et al. [15] | Open-label pilot study (MIRROR) | Methotrexate (4 weeks) | 11/14 (79) |
| Rainey et al. [13] | Open-label pilot study (TRIPLE) | Azathioprine (2 weeks) | 6/10 (60)d |
| Khanna et al. [40••] | Randomized controlled trial (RECIPE) | Mycophenolate mofetil (2 weeks) | 19/22 (86) |
aStudies presented here are not head-to-head trials designed or statistically powered to compare the efficacy or safety of pegloticase alone or in combination with immunomodulation
bAll patients received a biweekly infusion of pegloticase 8 mg in combination with the immunomodulatory therapy shown
cOne patient received oral methotrexate 14 days after the initial pegloticase infusion
dExcludes 2 patients for whom treatment is ongoing
IMT immunomodulatory therapy