Immunoreactivity to WT1 peptide vaccine is associated with prognosis in elderly patients with acute myeloid leukemia: follow-up study of randomized phase II trial of OCV-501, an HLA class II-binding WT1 polypeptide

Tomoki Naoe; Akiko Saito; Nahoko Hosono; Senji Kasahara; Hideharu Muto; Kaoru Hatano; Mizuki Ogura; Taro Masunari; Masatsugu Tanaka; Kensuke Usuki; Yuichi Ishikawa; Koji Ando; Yukio Kondo; Yusuke Takagi; Satoru Takada; Maho Ishikawa; Ilseung Choi; Akihiro Sano; Hirokazu Nagai

doi:10.1007/s00262-023-03432-4

Immunoreactivity to WT1 peptide vaccine is associated with prognosis in elderly patients with acute myeloid leukemia: follow-up study of randomized phase II trial of OCV-501, an HLA class II-binding WT1 polypeptide

Cancer Immunol Immunother. 2023 Aug;72(8):2865-2871. doi: 10.1007/s00262-023-03432-4. Epub 2023 Apr 24.

Authors

Tomoki Naoe¹, Akiko Saito^{2

3}, Nahoko Hosono⁴, Senji Kasahara⁵, Hideharu Muto⁶, Kaoru Hatano⁷, Mizuki Ogura⁸, Taro Masunari⁹, Masatsugu Tanaka¹⁰, Kensuke Usuki¹¹, Yuichi Ishikawa¹², Koji Ando¹³, Yukio Kondo¹⁴, Yusuke Takagi¹⁵, Satoru Takada¹⁶, Maho Ishikawa¹⁷, Ilseung Choi¹⁸, Akihiro Sano², Hirokazu Nagai¹⁹

Affiliations

¹ National Hospital Organization Nagoya Medical Center, 4-1-1 San-No-Maru, Naka-Ku, Nagoya, Japan. naoe.tomoki.wx@mail.hosp.go.jp.
² Clinical Research Center, National Hospital Organization Nagoya Medical Center, Nagoya, Japan.
³ Clinical Research Center, National Hospital Organization Headquarters, Tokyo, Japan.
⁴ Department of Hematology and Oncology, University of Fukui, Fukui, Japan.
⁵ Department of Hematology, Gifu Municipal Hospital, Gifu, Japan.
⁶ Department of Blood Transfusion, Tokyo Metropolitan Otsuka Hospital, Tokyo, Japan.
⁷ Department of Hematology, Jichi Medical University Hospital, Shimotsuke, Japan.
⁸ Department of Hematology, Japanese Red Cross Medical Center, Tokyo, Japan.
⁹ Department of Hematology/Infectious Diseases, Chugoku Central Hospital, Fukuyama, Japan.
¹⁰ Department of Hematology, Kanagawa Cancer Center, Yokohama, Japan.
¹¹ Department of Hematology, NTT Medical Center Tokyo, Tokyo, Japan.
¹² Department Hematology and Oncology, Nagoya University Graduate School of Medicine, Nagoya, Japan.
¹³ Department of Hematology, Nagasaki University Hospital, Nagasaki, Japan.
¹⁴ Department of Hematology, Toyama Prefectural Central Hospital, Toyama, Japan.
¹⁵ Department of Hematology, Ogaki Municipal Hospital, Ogaki, Japan.
¹⁶ Leukemia Research Center, Saiseikai Maebashi Hospital, Maebashi, Japan.
¹⁷ Department of Hemato-Oncology, Saitama Medical University International Medical Center, Hidaka, Japan.
¹⁸ Department of Hematology and Cell Therapy, National Hospital Organization Kyushu Cancer Center, Fukuoka, Japan.
¹⁹ Department of Hematology, National Hospital Organization Nagoya Medical Center, Nagoya, Japan.

Abstract

We previously conducted a randomized phase II trial of OCV-501, a WT1 peptide presented by helper T cells, in elderly AML (acute myeloid leukemia) patients in first remission, indicating no difference in 2-year disease-free survival (DSF) between the OCV-501 and placebo groups. Here, we analyzed 5-year outcome and biomarkers. Five-year DFS was 36.0% in the OCV-501 group (N = 52) and 33.7% in the placebo group (N = 53), with no significant difference (p = 0.74). The peripheral WT1 mRNA levels were marginally suppressed in the OCV-501 group compared with the placebo group. Enhanced anti-OCV-501 IgG response by the 25th week was an independent favorable prognostic factor. Anti-OCV-501 IFNγ responses were less frequent than the IgG reactions. These findings suggest that host immunoreactivity has a significant impact on the prognosis of AML and that further improvement of the WT1 peptide vaccine is needed.

Keywords: Acute myeloid leukemia (AML); Biomarker; Immunotherapy; Peptide vaccine; WT1.

Publication types

Randomized Controlled Trial
Clinical Trial, Phase II

MeSH terms

Aged
Follow-Up Studies
Humans
Immunoglobulin G
Leukemia, Myeloid, Acute* / genetics
Prognosis
Vaccines, Subunit / therapeutic use
WT1 Proteins*

Substances

WT1 Proteins
Vaccines, Subunit
Immunoglobulin G
WT1 protein, human