Selected clinical trials of IFN in PV
| Author . | Intervention . | Design . | Patient population . | Outcomes . |
|---|---|---|---|---|
| Kiladjian et al51 | peg-IFN-α2a | Single-arm, phase 2 multicenter study | 40 patients with PV, aged 18-65 y; no previous treatment, only phlebotomies, or cytoreductive treatment for <2 y | 100% ORR (CHR: 95%; PHR: 5%); 72% with complete or partial molecular response |
| Quintas-Cardama et al52 | peg-IFN-α2a | Single-arm, open-label, single center, phase 2 study | 40 patients with PV; newly diagnosed or previously treated | 80% hematologic response rate (CHR 70%); 54% molecular response rate (14% undetectable JAK2 V617F) |
| Masarova et al53 | peg-IFN-α2a | Single-arm, open-label, single center, phase 2 study | 43 patients with PV; newly diagnosed or previously treated | 84% ORR (CHR: 77%; PHR: 7%); 63% molecular response rate |
| Yacoub et al54 | peg-IFN-α2a | Single-arm, phase 2 multicenter study | 50 patients with PV; refractory or intolerant to hydroxyurea | 60% ORR (22% CR; 38% PR) |
| Gisslinger et al36 | ropeg-IFN-α2b vs hydroxyurea | Randomized, open-label, phase 3 trial | 257 patients with PV randomized (no, or <3 y of, cytoreductive treatment) | CHR higher with ropeg-IFN at 36 mo (53% vs 38%; P = .044); noninferiority of ropeg-IFN for hematologic response and normal spleen size not shown at 12 mo |
| Gisslinger et al55 | ropeg-IFN-α2b | Single-arm, open-label, multicenter phase 1/2 study | 51 patients with PV | ORR 90% (CR 47%, PR 43%); CMR 21%, PHR 47% |
| Barbui et al56 | ropeg-IFN-α2b vs phlebotomy | Randomized phase 2 trial | 127 patients with low-risk PV | Higher rates of hematologic response with ropeg-IFN (84% vs 60%; P = .0075) |
| Mascarenhas et al45 | peg-IFN-α2a vs hydroxyurea | Randomized, open-label, phase 3 trial | 87 patients with high-risk PV, randomized; no prior cytoreductive therapy except for up to 3 mo of hydroxyurea | CR rate at 12 mo comparable for IFN vs hydroxyurea (27.9% vs 29.5%; primary end point); ORR 86% vs 68% |
| Knudsen et al57 | peg-IFN-α2a or -2b vs hydroxyurea | Randomized, open-label, phase 3 trial | 90 patients with PV; newly diagnosed or previously treated | ORR: 68% for hydroxyurea (CHR, 16%; PHR, 53%), 42% (14/33) for IFN-α among patients aged ≤60 y (CHR, 9%; PHR, 33%), and 39% for IFN-α among patients aged >60 y (CHR, 9%; PHR, 30%) |
| Author . | Intervention . | Design . | Patient population . | Outcomes . |
|---|---|---|---|---|
| Kiladjian et al51 | peg-IFN-α2a | Single-arm, phase 2 multicenter study | 40 patients with PV, aged 18-65 y; no previous treatment, only phlebotomies, or cytoreductive treatment for <2 y | 100% ORR (CHR: 95%; PHR: 5%); 72% with complete or partial molecular response |
| Quintas-Cardama et al52 | peg-IFN-α2a | Single-arm, open-label, single center, phase 2 study | 40 patients with PV; newly diagnosed or previously treated | 80% hematologic response rate (CHR 70%); 54% molecular response rate (14% undetectable JAK2 V617F) |
| Masarova et al53 | peg-IFN-α2a | Single-arm, open-label, single center, phase 2 study | 43 patients with PV; newly diagnosed or previously treated | 84% ORR (CHR: 77%; PHR: 7%); 63% molecular response rate |
| Yacoub et al54 | peg-IFN-α2a | Single-arm, phase 2 multicenter study | 50 patients with PV; refractory or intolerant to hydroxyurea | 60% ORR (22% CR; 38% PR) |
| Gisslinger et al36 | ropeg-IFN-α2b vs hydroxyurea | Randomized, open-label, phase 3 trial | 257 patients with PV randomized (no, or <3 y of, cytoreductive treatment) | CHR higher with ropeg-IFN at 36 mo (53% vs 38%; P = .044); noninferiority of ropeg-IFN for hematologic response and normal spleen size not shown at 12 mo |
| Gisslinger et al55 | ropeg-IFN-α2b | Single-arm, open-label, multicenter phase 1/2 study | 51 patients with PV | ORR 90% (CR 47%, PR 43%); CMR 21%, PHR 47% |
| Barbui et al56 | ropeg-IFN-α2b vs phlebotomy | Randomized phase 2 trial | 127 patients with low-risk PV | Higher rates of hematologic response with ropeg-IFN (84% vs 60%; P = .0075) |
| Mascarenhas et al45 | peg-IFN-α2a vs hydroxyurea | Randomized, open-label, phase 3 trial | 87 patients with high-risk PV, randomized; no prior cytoreductive therapy except for up to 3 mo of hydroxyurea | CR rate at 12 mo comparable for IFN vs hydroxyurea (27.9% vs 29.5%; primary end point); ORR 86% vs 68% |
| Knudsen et al57 | peg-IFN-α2a or -2b vs hydroxyurea | Randomized, open-label, phase 3 trial | 90 patients with PV; newly diagnosed or previously treated | ORR: 68% for hydroxyurea (CHR, 16%; PHR, 53%), 42% (14/33) for IFN-α among patients aged ≤60 y (CHR, 9%; PHR, 33%), and 39% for IFN-α among patients aged >60 y (CHR, 9%; PHR, 30%) |
CMR, complete molecular response; ORR, overall response rate; PHR, partial hematologic remission; PMR, partial molecular response.