Figure 1.

Overview of the HLH-94 treatment protocol. BMT: Patients with familial or persistent disease were recommended to go to HSCT as soon as an acceptable donor was available, preferably when the disease was nonactive. The patients without familial or persistent disease were recommended to stop therapy after the initial therapy, and restart in case of reactivation. Dexa: Daily dexamethasone (10 mg/m² for 2 weeks followed by 5 mg/m² for 2 weeks, 2.5 mg/m² for 2 weeks, 1.25 mg/m² for 1 week, and 1 week of tapering; pulses were 3 days, 10 mg/m² daily). VP-16: Etoposide 150 mg/m² IV. IT therapy: Intrathecal methotrexate in patients with progressive neurological symptoms and/or persisting abnormal cerebrospinal fluid findings. CSA: Cyclosporin A aiming at blood levels of 200 μg/L (trough value).