A win ratio approach for comparing graft‐versus‐host disease‐free, relapse‐free survival among alternative donors

The optimal alternative donor type for patients lacking human leucocyte antigen (HLA)-matched related or unrelated donors remains unclear. In comparative studies evaluating donor types, graft-versus-host disease (GVHD)-free, relapse-free survival (GRFS) represents a well-established end-point but has limitations. The win ratio approach addresses these limitations by analysing multiple end-points with varying severities to account for the relative component priorities. We compared HLA-mismatched unrelated donors, haploidentical donors and cord blood using both the hazard ratio (HR) of GRFS and the win ratio related to GRFS. The haploidentical donor group had a similar HR of GRFS (HR: 1.01, 95% confidence interval [CI]: 0.85-1.19, p = 0.916) and win ratio (win ratio: 0.86, 95% CI: 0.72-1.02, p = 0.081) to HLA-mismatched unrelated donors. Cord blood transplantation showed similar GRFS compared to HLA-mismatched unrelated donors in the Cox proportional model (HR: 1.14, 95% CI: 0.98-1.32, p = 0.085), significantly lower win ratio (win ratio: 0.80, 95% CI: 0.68-0.93, p = 0.004) and similar outcomes to haploidentical donors. HLA-mismatched unrelated donor transplantation showed comparable to superior outcomes among alternative donor types. Our results indicate the need to present the win ratio alongside conventional methods to assess the end-point robustly.

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Journal
British Journal of Haematology
Published
2025-07-16
DOI
https://doi.org/10.1111/bjh.70008
Primary Topic
Hematopoietic Stem Cell Transplantation
Type
article
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article

A win ratio approach for comparing graft‐versus‐host disease‐free, relapse‐free survival among alternative donors

Masatsugu Tanaka, Naoyuki Uchida, Yoshinobu Kanda, Mamiko Sakata‐Yanagimoto et al.
British Journal of Haematology
Hematopoietic Stem Cell Transplantation
article

A win ratio approach for comparing graft‐versus‐host disease‐free, relapse‐free survival among alternative donors

Masatsugu Tanaka, Naoyuki Uchida, Yoshinobu Kanda, Mamiko Sakata‐Yanagimoto, Yuta Hasegawa, Kazuya Ishiwata, Yoshiaki Usui, Yoshihiro Inamoto, Noriko Doki, Tetsuhisa Kitamura, Kazuki Yoshimura, Hideki Nakasone, Toshiro Kawakita, Yoshimitsu Shimomura, Yu Akahoshi, Junya Kanda, Sho Komukai, Makoto Onizuka, Makoto Yoshimitsu, Hirohisa Nakamae, Yachiyo Kuwatsuka, Marie Ohbiki, Takahiro Fukuda, Masashi Sawa, Yuta Katayama
article en

Abstract

The optimal alternative donor type for patients lacking human leucocyte antigen (HLA)-matched related or unrelated donors remains unclear. In comparative studies evaluating donor types, graft-versus-host disease (GVHD)-free, relapse-free survival (GRFS) represents a well-established end-point but has limitations. The win ratio approach addresses these limitations by analysing multiple end-points with varying severities to account for the relative component priorities. We compared HLA-mismatched unrelated donors, haploidentical donors and cord blood using both the hazard ratio (HR) of GRFS and the win ratio related to GRFS. The haploidentical donor group had a similar HR of GRFS (HR: 1.01, 95% confidence interval [CI]: 0.85-1.19, p = 0.916) and win ratio (win ratio: 0.86, 95% CI: 0.72-1.02, p = 0.081) to HLA-mismatched unrelated donors. Cord blood transplantation showed similar GRFS compared to HLA-mismatched unrelated donors in the Cox proportional model (HR: 1.14, 95% CI: 0.98-1.32, p = 0.085), significantly lower win ratio (win ratio: 0.80, 95% CI: 0.68-0.93, p = 0.004) and similar outcomes to haploidentical donors. HLA-mismatched unrelated donor transplantation showed comparable to superior outcomes among alternative donor types. Our results indicate the need to present the win ratio alongside conventional methods to assess the end-point robustly.

British Journal of HaematologyVol. 207(3)
Kagoshima University (JP), Tokai University (JP), Fujita Health University (JP), Jichi Medical University (JP), Aichi Medical University (JP), Kyoto University (JP), Kanagawa Prefectural Hospital Organization (JP), Tokyo Metropolitan Komagome Hospital (JP), Tokyo Health Care University (JP), Nagoya University Hospital (JP), Hiroshima Red Cross Hospital & Atomic-bomb Survivors Hospital (JP), University of Tsukuba Hospital (JP), Hokkaido University Hospital (JP), Anjo Kosei Hospital (JP), RIKEN Center for Integrative Medical Sciences (JP), The Japanese Data Center for Hematopoietic Cell Transplantation (JP), Kobe City Medical Center General Hospital (JP), Kumamoto Medical Center (JP), Osaka City University Hospital (JP), Jichi Medical University Saitama Medical Center (JP), Toranomon Hospital (JP), Kanagawa Cancer Center, Osaka Metropolitan University (JP), Nagoya University (JP), The University of Osaka (JP)
Good health and well-being
Openalex Percentile: Top 78%
Hematopoietic Stem Cell Transplantation
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