Risk Factors for and Impact of Pre‐Engraftment Syndrome on Outcomes Following Single‐Unit Cord Blood Transplantation in Adults

Pre-engraftment syndrome (PES) is a unique complication of cord blood transplantation (CBT) whose risk factors and impact on transplant outcomes remain controversial. Using a nationwide database in Japan, we analyzed a total of 3734 patients who underwent single-unit CBT. PES occurred in 18.3% of patients, and risk factors for PES included a higher hematopoietic cell transplantation-specific comorbidity index, first transplantation, myeloablative conditioning (MAC), lower total nucleated cell (TNC) dose, and graft-versus-host disease (GVHD) prophylaxis regimens excluding tacrolimus with methotrexate. Patients who developed PES had significantly higher incidences of grade II-IV acute GVHD (53.1% vs. 31.3%, p < 0.001) and chronic GVHD (27.2% vs. 21.7%, p = 0.002) compared to those without PES. Landmark analysis with multivariable adjustment revealed that PES was independently associated with increased non-relapse mortality (NRM, hazard ratio [HR] 1.46; 95% CI 1.22-1.75; p < 0.001), reduced relapse incidence (HR 0.78; 95% CI 0.63-0.96; p = 0.020), and a trend toward inferior overall survival (OS, HR 1.13; 95% CI 0.98-1.30; p = 0.088). Moreover, patients who experienced both PES and acute GVHD had the highest 2-year NRM (31.7%; 95% CI 26.0%-37.6%) and the lowest 2-year OS (55.9%; 95% CI 50.0%-62.4%), compared with those who experienced either PES (NRM 20.7%; OS 65.0%) or acute GVHD (NRM 19.5%; OS 62.8%) (p < 0.001), highlighting the combined effects of these complications. This study, the largest to date on PES, demonstrates its clinical significance as an early complication with lasting effects on transplant outcomes.

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Journal
American Journal of Hematology
Published
2025-09-26
DOI
https://doi.org/10.1002/ajh.70094
Citations
1
Primary Topic
Hematopoietic Stem Cell Transplantation
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article
Field-Weighted Citation Impact
1.77
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article

Risk Factors for and Impact of Pre‐Engraftment Syndrome on Outcomes Following Single‐Unit Cord Blood Transplantation in Adults

Masatsugu Tanaka, Shuichi Shirane, Yuta Katayama, Mamiko Sakata‐Yanagimoto et al.
1 citations
American Journal of Hematology
Hematopoietic Stem Cell Transplantation
1.77
article

Risk Factors for and Impact of Pre‐Engraftment Syndrome on Outcomes Following Single‐Unit Cord Blood Transplantation in Adults

Masatsugu Tanaka, Shuichi Shirane, Yuta Katayama, Mamiko Sakata‐Yanagimoto, Yuta Hasegawa, Shigesaburo Miyakoshi, Seitaro Terakura, Masatoshi Sakurai, Koji Kato, Tetsuya Nishida, Takuto Mori, Fumihiko Ishimaru, Hideki Nakasone, Shuichi Ota, Takehiko Mori, Norimichi Hattori, Junya Kanda, Makoto Onizuka, Hirotoshi Sakaguchi, Keisuke Kataoka, Kota Mizuno, Jun Kato, Masahito Tokunaga, Nobuyuki Takayama, Jun Ishikawa, Koichi Onodera, Masashi Sawa, Yoshiko Atsuta
article en
1 citations

Abstract

Pre-engraftment syndrome (PES) is a unique complication of cord blood transplantation (CBT) whose risk factors and impact on transplant outcomes remain controversial. Using a nationwide database in Japan, we analyzed a total of 3734 patients who underwent single-unit CBT. PES occurred in 18.3% of patients, and risk factors for PES included a higher hematopoietic cell transplantation-specific comorbidity index, first transplantation, myeloablative conditioning (MAC), lower total nucleated cell (TNC) dose, and graft-versus-host disease (GVHD) prophylaxis regimens excluding tacrolimus with methotrexate. Patients who developed PES had significantly higher incidences of grade II-IV acute GVHD (53.1% vs. 31.3%, p < 0.001) and chronic GVHD (27.2% vs. 21.7%, p = 0.002) compared to those without PES. Landmark analysis with multivariable adjustment revealed that PES was independently associated with increased non-relapse mortality (NRM, hazard ratio [HR] 1.46; 95% CI 1.22-1.75; p < 0.001), reduced relapse incidence (HR 0.78; 95% CI 0.63-0.96; p = 0.020), and a trend toward inferior overall survival (OS, HR 1.13; 95% CI 0.98-1.30; p = 0.088). Moreover, patients who experienced both PES and acute GVHD had the highest 2-year NRM (31.7%; 95% CI 26.0%-37.6%) and the lowest 2-year OS (55.9%; 95% CI 50.0%-62.4%), compared with those who experienced either PES (NRM 20.7%; OS 65.0%) or acute GVHD (NRM 19.5%; OS 62.8%) (p < 0.001), highlighting the combined effects of these complications. This study, the largest to date on PES, demonstrates its clinical significance as an early complication with lasting effects on transplant outcomes.

American Journal of HematologyVol. 100(12)
Tokyo Institute of Technology (JP), SHOWA Medical University (JP), Tokai University (JP), Jichi Medical University (JP), Aichi Medical University (JP), Keio University (JP), Kyoto University (JP), Japanese Red Cross Society, Japan (JP), Kanagawa Prefectural Hospital Organization (JP), Juntendo University (JP), Tokyo Institute of Psychiatry (JP), Tohoku University Hospital (JP), Hiroshima Red Cross Hospital & Atomic-bomb Survivors Hospital (JP), Sapporo Hokuyu Hospital (JP), University of Tsukuba Hospital (JP), Imamura Hospital (JP), Japanese Red Cross Nagoya Daiichi Hospital (JP), National Center For Child Health and Development (JP), Kyorin University Hospital (JP), Tokyo Metropolitan Institute of Gerontology (JP), Hokkaido University Hospital (JP), Anjo Kosei Hospital (JP), Osaka International Cancer Institute (JP), Tokyo Metropolitan Geriatric Hospital (JP), The Japanese Data Center for Hematopoietic Cell Transplantation (JP), Jichi Medical University Saitama Medical Center (JP), Kanagawa Cancer Center, Bank of Japan (JP), Nagoya University (JP)
Openalex Percentile: Top 12%
Hematopoietic Stem Cell Transplantation
1.77
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