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Veno-Occlusive Disease in Very Young Children: A Profile of High Morbidity With Low Mortality
Miki Nishitani1, Kyle Hebert2, Donna Neuberg2
1Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts, USA.
Insights
Veno-occlusive disease (VOD) in young children post-hematopoietic stem cell transplant (HCT) can be severe but has excellent early survival. Retrospective analysis revealed discrepancies between clinical diagnosis and established VOD criteria.
Area of Science:
- Pediatric Hematology Oncology
- Transplant Immunology
- Clinical Research
Background:
- Veno-occlusive disease (VOD) is a known complication of hematopoietic stem cell transplant (HCT).
- Younger age is a risk factor for VOD, but specific data on diagnostic criteria and outcomes in very young children are limited.
- This study addresses the gap in understanding VOD in children under 4 years old.
Purpose of the Study:
- To compare overall survival (OS) in very young children with and without a clinical diagnosis of VOD after HCT.
- To compare OS for patients with clinical VOD versus those who retrospectively met VOD criteria.
- To evaluate the diagnostic accuracy of clinical VOD assessment in this age group.
Main Methods:
- Retrospective analysis of 150 patients under 4 years old who received their first HCT between January 2016 and October 2021.
- Application of three established pediatric VOD criteria retrospectively to all subjects.
- Comparison of clinical VOD diagnosis against retrospective criteria fulfillment and assessment of patient outcomes.
Main Results:
- 18 patients (12%) were clinically diagnosed with VOD, often requiring intensive care, drainage procedures, and respiratory support.
- Despite high morbidity, all patients diagnosed with VOD survived to discharge and Day +100.
- Overall survival at Day +365, and rates of acute and chronic graft-versus-host disease were similar between VOD and non-VOD groups; however, VOD patients had a longer hospital stay.
- A higher number of patients met retrospective VOD criteria than were clinically diagnosed.
Conclusions:
- VOD in very young children undergoing HCT, while potentially severe, is associated with excellent early survival.
- There is a notable discrepancy between clinical VOD diagnosis and retrospective fulfillment of established criteria in this population.
- Further refinement of diagnostic approaches for VOD in very young pediatric HCT recipients may be warranted.
Background/Objectives:
Younger age is a recognized risk factor for veno-occlusive disease (VOD), yet no prior studies have focused on diagnostic criteria and outcomes in very young children. This retrospective study compared overall survival (OS) in very young children with and without a clinical diagnosis of VOD, in addition to comparing OS for those with clinical VOD versus those who retrospectively fulfilled VOD criteria.
Design/Methods:
Included were patients who received their first hematopoietic stem cell transplant (HCT) for any indication at less than 4 years of age between January 1, 2016 and October 31, 2021. Three pediatric VOD criteria were retrospectively applied to subjects.
Results:
Of 150 HCT recipients less than 4 years old, 18 were clinically diagnosed with VOD at a median of 14 days. More patients with VOD were admitted to the ICU [79% vs. 15%, p < 0.001]. Of those with VOD, 78% required peritoneal drainage, 17% pleural effusion drainage, and 56% respiratory failure. All patients with VOD survived to discharge and to Day +100. In allo-recipients, OS at Day +365, cumulative incidence of Grade II-IV acute graft-versus-host disease (aGVHD) at Day +100, and chronic graft-versus-host disease (cGVHD) at Day +365 were similar between those with and without VOD. Median length of stay was longer in those with VOD [60.5 vs. 38 days, p < 0.001]. More patients retrospectively met criteria for VOD than were clinically diagnosed.
Conclusion:
While VOD in very young children can be highly morbid, they have excellent early survival. Retrospective evaluation demonstrates that only some patients who fulfilled VOD criteria were clinically diagnosed.
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