Pilot Study of Etoposide-based Therapy and Hematopoietic Cell Transplantation for Hemophagocytic Lymphohistiocytosis (HELA2012)

Inclusion Criteria:

• Patients whose clinical findings satisfy 5 or more criteria out of the following 8 ones

  1. Fever ≥ 38.5 ℃ for ≥ 7 days
  2. Splenomegaly ≥ 3 FB below left subcostal margin
  3. Cytopenias affecting ≥ 2 of 3 lineages in PB Hb < 9 g/L Platelet < 100 x 109 /L ANC < 1.0 x 109 /L
  4. Hypertriglyceridemia and/or hypofibrinogenemia (fasting triglycerides ≥ 265 mg/dL, fibrinogen ≤ 1.5 g/L)
  5. Hemophagocytosis in BM or spleen or LN
  6. Low or absent NK-cell activity ( according to local laboratory reference)
  7. Serum-ferritin ≥ 500 mcg/L
  8. Soluble CD25(sIL-2 receptor) ≥ 2,400 U/ml
• 18 years of age and over.
• All patients (or his/her family when the patient cannot sign the consent form because of his/her general conditions) give written informed consent according to guidelines at institution's committee on human research.

Exclusion Criteria:

• HLH from malignancy (such as lymphoma, myeloma, leukemia, and other solid tumor)
• HLH from rheumatic disorder (such as SLE, AOSD, antiphospholipid antibody syndrome)
• Patients with psychiatric disorder or mental deficiency severe as to make compliance with the treatment unlike, and making informed consent impossible
• Nursing women, pregnant women, women of childbearing potential who do not want adequate contraception
• Male patient who reject the methods of avoiding pregnancy via methods such as abstinence, barrier method (condom etc).
• Patients with a diagnosis of prior malignancy unless disease-free for at least 5 years following therapy with curative intent (except curatively treated nonmelanoma skin cancer, in situ carcinoma, or cervical intraepithelial neoplasia)