Longitudinal observation and outcome of nonfamilial childhood haemophagocytic syndrome receiving etoposide-containing regimens

The long-term outcome of 22 children treated with etoposide-containing regi mens for haemophagocytic syndrome (HS) were longitudinally studied; none of them had a family history of the disease. All patients received etoposide- containing (150 mg/m(2)/d) regimens, combined, in 16 cases, with intravenou s immunoglobulin (IVIG) and prednisolone, Complete remission (CR) was achie ved in 12 patients, partial remission in seven, and early mortality occurre d in three. Of the 11 CR patients, only four remain alive and disease-free, with a median follow-up of 47.4 months: one CR patient died due to infecti on and the remaining seven had relapsed diseases. Three patients with a par tial response or with relapsed disease progressed to T-cell lymphoma, chara cterized, in the two cases tested, by clonal chromosomal abnormalities. Eps tein-Barr virus (EBV) infection was implicated in disease pathogenesis in 1 5/22 patients. The overall survival was 45.5%, 40.9% and 40.9% at 1, 3 and 5 years, respectively, and disease-free survival for CR patients at these s ame times was 45.5%, 36.4% and 36.4%. The etoposide-containing regimen woul d appear to be an effective initial therapeutic option for childhood HS. Ho wever, in view of the frequency of partial remissions and relapsed disease, a more intensive chemotherapy or bone marrow transplantation should be app lied. The progression to EBV-containing T-cell lymphoma in three patients i s consistent with the previous observation that EBV-associated IIS is a pot entially malignant disease.