Combination Chemotherapy Followed By Donor Stem Cell Transplant in Treating Patients With Hemophagocytic Lymphohistiocytosis

• Status: Unknown status
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

Summary

RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of hemophagocytic lymphohistiocytosis cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more hemophagocytic lymphohistiocytosis cells. A donor stem cell transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Cyclosporine and methotrexate may stop this from happening.

PURPOSE: This phase III trial is studying how well combination chemotherapy followed by a donor stem cell transplant works in treating patients with hemophagocytic lymphohistiocytosis.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Cancer and Leukaemia Group
• Treatments: Antilymphocyte Serum; Busulfan; Cortisol succinate; Cyclophosphamide; Cyclosporine; Cyclosporins; Dexamethasone; Etoposide; Hydrocortisone; Hydrocortisone 17-butyrate 21-propionate; Hydrocortisone acetate; Hydrocortisone hemisuccinate; Methotrexate; Mycophenolate mofetil; Mycophenolic Acid

Criteria

DISEASE CHARACTERISTICS:

• Newly diagnosed hemophagocytic lymphohistiocytosis (HLH) meeting 1 of the following criteria*:

• Diagnosis by molecular/genetic methods

• Diagnosis by meeting 5 out of 8 of the following criteria:

• Clinical criteria:

• Fever

• Splenomegaly

• Laboratory criteria:

• Cytopenias affecting ≥ 2 of 3 lineages in the peripheral blood, including the following:

• Hemoglobin < 9.0 g/dL (< 10.0 g/dL in infants < 4 weeks of age)

• Platelet count < 100,000/mm^3

• Neutrophil count < 1,000/mm^3

• Hypertriglyceridemia and/or hypofibrinogenemia:

• Fasting triglycerides ≥ 3.0 mmol/L (i.e., ≥ 265 mg/dL)

• Fibrinogen ≤ 1.5 g/L

• Histopathologic criteria:

• Hemophagocytosis in bone marrow, spleen, or lymph nodes

• No evidence of malignancy

• New diagnostic criteria:

• Low or absent natural killer (NK) cell activity

• Ferritin ≥ 500 mcg/L

• Soluble CD25 (i.e., soluble interleukin-2 receptor) ≥ 2,400 U/mL NOTE:

*Patients who do not meet the diagnostic criteria for HLH but who have a strong clinical suspicion of HLH may be eligible at the discretion of the investigator

• Primary HLH (i.e., familial hemophagocytic lymphohistiocytosis [FLH]) OR secondary HLH (i.e., severe acquired form of HLH)

• Acceptable donor meeting 1 of the following criteria:

• HLA-identical related donor

• Matched unrelated donor

• Mismatched unrelated donor

• Familial haploidentical donor

PATIENT CHARACTERISTICS:

• Not specified

PRIOR CONCURRENT THERAPY:

• No prior cytotoxic treatment for HLH

• No prior cyclosporine treatment for HLH