Hemophagocytic lymphohistiocytosis (HLH) used to have a dismal prognosis and in 1983 the long-term survival was reported as 4%. Prompted by these poor results, in 1994 the Histiocyte Society developed a treatment strategy (HLH-94) which combined two previously reported regimens, chemotherapy and immunotherapy. HLH-94 is based on etoposide (VP-16), corticosteroids, cyclosporin A (CSA), and, in selected patients, IT MTX, prior to intended stem cell transplantation (SCT).
The Histiocyte Society launched the first international therapeutic study on HLH in 1994 (HLH-94). It started July 1, 1994, and closed for recruitment December 31, 2003.
OUTCOMES OF THE TRIAL:
Altogether 249 patients fulfilled inclusion criteria. At a median follow-up of 6.2 years, estimated 5-year probability-of-survival was 54±6%. Seventy-two patients (29%) died before SCT. In the 124 patients that underwent SCT, 5-year-survival was 66±9% with tendency to increased survival (p=0.064) in patients with non-active disease at SCT. Patients with familial disease had a 5-year-survival of 50%±13%. To conclude, HLH-94 chemo-immunotherapy considerably improved outcome in HLH.
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