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Clinical Trial
. 2025 Aug 1;157(3):526-533.
doi: 10.1002/ijc.35411. Epub 2025 Mar 15.

Hematologic and molecular responses to ropeginterferon alfa-2b therapy of polycythemia vera: 48-week results from a prospective study

Seug Yun Yoon  1 , Sung-Soo Yoon  2 , Deok-Hwan Yang  3 , Gyeong-Won Lee  4 , Sang Kyun Sohn  5 , Ho-Jin Shin  6 , Sung Hwa Bae  7 , Chul Won Choi  8 , Eun-Ji Choi  9 , June-Won Cheong  10 , Soo-Mee Bang  11 , Joon Seong Park  12 , Suk Joong Oh  13 , Yong Park  14 , Young Hoon Park  15 , Sung-Eun Lee  16
Affiliations
Clinical Trial

Hematologic and molecular responses to ropeginterferon alfa-2b therapy of polycythemia vera: 48-week results from a prospective study

Seug Yun Yoon et al. Int J Cancer. .

Abstract

To prevent thrombosis in patients with polycythemia vera (PV), achieving a complete hematologic response (CHR) is highly recommended in practice. In addition, a reduced JAK2 V617F mutation burden is expected to have a disease-modifying effect, and its molecular response (MR) is currently of significant interest. This study aimed to assess the association between CHR and MR in patients with PV following treatment with ropeginterferon alfa-2b. This phase 2, single-arm, open-label, investigator-initiated trial was conducted at 16 sites in South Korea. Ninety-nine patients were treated with ropeginterferon alfa-2b subcutaneously every 2 weeks, at doses of 250 μg (week 1), 350 μg (week 3), and 500 μg (week 5), until week 48. CHRs were 27% (25/94), 46% (40/87), 56% (47/84), and 63% (51/81) at 12, 24, 36, and 48 weeks, respectively. The MR rates were 32% (28/88), 36% (29/81), 49% (38/77), and 57% (42/74) at 12, 24, 36, and 48 weeks, respectively. The Phi Coefficient for the association between CHR and MR was 0.6146 (p < .0001) at 48 weeks. In the subgroup analysis, patients with hydroxyurea resistance or intolerance, and those who were hydroxyurea-naïve, had similar results in terms of the CHR. In conclusion, CHR and MR were observed to be associated in patients with PV treated with ropeginterferon.

Keywords: CHR; MR; association; polycythemia vera; ropeginterferon alfa‐2b.

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Conflict of interest statement

SYL's institution has received research funding from PharmaEssentia. The other authors declare no conflicts of interest.

Figures

FIGURE 1
FIGURE 1
Complete hematology and molecular response. (A) Sankey‐style patient flow chart for Complete Hematology Response (CHR) rate by assessment visit. (B) Time to reach 1st CHR after the 1st injection of ropeginterferon alfa‐2b. (C) Sankey‐style patient flow chart for molecular response (MR) rate by assessment visit. (D) Time to reach 1st MR after the 1st injection of ropeginterferon alfa‐2b.
FIGURE 2
FIGURE 2
Waterfall plot of percentage of change from baseline in JAK2 V617F allele burden in patients at 48 weeks based on response to CHR.
See this image and copyright information in PMC

References

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