Blood Research

Summary of clinical trials of IFNα products in patients with MPN.

Clinical trial Phase Characteristics Patient population IFN Comparator Clinical response (ORR/CHR) Molecular response (ORR/CMR) Grade III/IV AE/discontinuation rate



IFN Comparator IFN Comparator IFN Comparator
LOW-PV [41] (abstract) 2 Low-risk PV (diagnosed less than 3 yr prior) PV(N=100) Ropeg-IFNα-2b Phlebotomy 84%/NR 66%/NR NR NR 6%/NR 8%/NR
Proud-PV/continuation-PV [40] 3 Early stage PV (less than 3 years of previous hydroxyurea treatment) PV (N=257) Ropeg-IFNα-2b Hydroxyurea NR/43% NR/46% 34%/NR 42%/NR (1 yr f/u) 43%/8% 34%/4%
NR/71% NR/51% 66%/NR 27%/NR (3 yr f/u)
MPD-RC 112 [39] (abstract) 3 High risk (Treatment-naïv, hydroxyurea <3 mo) ET (N=81) Peg-IFNα-2a Hydroxyurea 78.0%/35.4% 69.8%/37.2% (1 y f/u) NR NR 46.3%/NR 27.5%/NR
PV (N=87) 59.6%/28.8% 40.7%/20.4% (2 y f/u)
MPD-RC 111 [38] 2 High risk (hydroxyurea intolerant or resistant) ET (N=65) Peg-IFNα-2a None 69.2%/43.1% (ET) CR patients (JAK2 allele burden: -6%) 30.7%/13.9%
PV(N=50) 60%/22% (PV)
COMBI [46] 2 IFN+JAK inhibitor PV (N=32) Peg-IFNα-2a+ruxo-litinib 31%/9% (PV) 41%/2% NR/31% (PV)
MF (N=18) 44%/28% (MF) NR/39% (MF)

Abbreviations: AE, adverse event; CHR, complete hematologic response; CMR, complete molecular response; ET, essential thrombocythemia; f/u, follow-up; IFNα, interferon alpha; MF, myelofibrosis; MPN, myeloproliferative neoplasm; NR, not reported; ORR, overall response rate; Peg-IFNα-2a, pegylated interferon alpha-2a; PV, polycythemia vera; Ropeg-IFNα-2b, ropeginterferon alpha-2b.

Blood Res 2021;56:S44~S50 https://doi.org/10.5045/br.2021.2020334
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