Hematopoietic cell transplantation in pediatric EB virus-associated hemophagocytic lymphohistiocytosis.

BACKGROUND

Few reports exist on the transplant outcomes of Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis (EBV-HLH). This nationwide survey evaluated the clinical outcomes of pediatric patients with EBV-HLH who underwent allogeneic HCT.

METHODS

Data from 32 pediatric patients with EBV-HLH who underwent their first allogeneic HCT between 2000 and 2020 were reviewed retrospectively.

RESULTS

Of the 32 patients, 12 had a performance status of 3-4 and 8 had multiple organ dysfunction. The cumulative incidence of engraftment on day 30 was 53.1% (95% confidence interval [CI] 34.1-68.9). The 1-year overall survival (OS) rate of the entire cohort was 56.2% (37.6-71.3). Univariate analysis revealed that poor ECOG-PS at HCT and a reduced-intensity conditioning regimen were significant risk factors for OS. Furthermore, multivariate analysis revealed that only ECOG-PS was a significant risk factor for OS. Sixteen of the 32 (50.0%) patients died after HCT, and the main causes of death were HLH progression (n = 12) and infection (n = 2). Most notably, 12 of the 13 patients with primary graft failure died of HLH.

CONCLUSION

Good general condition prior to HCT, which is typically achieved through optimal disease control, is essential for stable engraftment and long-term survival following allogeneic HCT.