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

• Status: Terminated
• Trial end date: 2016-05-01
• Target enrollment: 0
• Participant gender: All

Summary

The investigators would like to propose a pilot study evaluating the efficacy of etoposide combined with immunosuppressive agents for adult secondary hemophagocytic lymphohistiocytosis (HLH), in order to prove out whether the modification of previous HLH-94 or HLH-2004 protocol for childhood patients can improve the outcome and decrease the toxicities. The results of this pilot study will be a base of a more-improved phase-2 protocol.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Asan Medical Center
• Treatments: BB 1101; Cyclosporine; Cyclosporins; Dexamethasone; Dexamethasone 21-phosphate; Dexamethasone acetate; Etoposide; Etoposide phosphate; gamma-Globulins

Criteria

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)