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. 2024 Dec;71(12):e31331.
doi: 10.1002/pbc.31331. Epub 2024 Sep 17.

Early defibrotide therapy and risk factors for post-transplant veno-occlusive disease/sinusoidal obstruction syndrome in childhood

Hironori Goto  1   2 , Utako Oba  1 , Tamaki Ueda  1 , Shunsuke Yamamoto  1 , Masataka Inoue  1 , Yu Shimo  1 , Satomi Yokoyama  3 , Yusuke Takase  4 , Wakako Kato  1 , Souichi Suenobu  2   5 , Kenji Ihara  2 , Yuhki Koga  1   6 , Shouichi Ohga  1
Affiliations
Free article

Early defibrotide therapy and risk factors for post-transplant veno-occlusive disease/sinusoidal obstruction syndrome in childhood

Hironori Goto et al. Pediatr Blood Cancer. 2024 Dec.
Free article

Abstract

Background: Veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), is a life-threatening complications of hematopoietic cell transplantation (HCT).

Methods: We studied the impact of early defibrotide (DF) therapy on the outcomes of pediatric patients with VOD/SOS after transplantation, focusing on recent immunotherapies. A total of 111 pediatric patients who underwent HCT for malignant disease between February 2017 and March 2023 at Kyushu University Hospital were included.

Results: Among 111 patients of less than 20 years of age who underwent HCT for malignancy at a single institution between 2017 and 2023, VOD/SOS occurred in 25 (23%) patients. VOD/SOS developed more frequently in the post-DF era (2020-2023, n = 58) than in the pre-DF era (31% vs. 13%, p = .04). The proportion of patients with relapsed/refractory acute lymphoblastic leukemia (ALL) was higher in the post-DF era than in the pre-DF era (44% vs. 8%, p = .04). Early DF therapy that was started at two European Society for Blood and Marrow Transplantation diagnostic criteria reduced the severity of VOD/SOS (p < .01) in comparison to non-early therapy started at less than two criteria. A multivariate analysis indicated that a history of cytokine release syndrome (odds ratio [OR] = 10.4, p = .01) and juvenile myelomonocytic leukemia (OR = 8.98, p = .04), but not an endothelial activation and stress index (EASIX) score of greater than 0.85, were independent risk factors for VOD/SOS.

Conclusions: Early DF therapy improves the severity and survival outcomes of post-transplant VOD/SOS in children. However, its incidence is increasing in the era of immunotherapy for progressive diseases.

Keywords: VOD/SOS; cytokine release syndrome; defibrotide; immunotherapy.

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References

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