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Trial record 8 of 21 for:    "Non-Langerhans-Cell Histiocytosis" | "Immunologic Factors"

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00334672
Recruitment Status : Unknown
Verified June 2009 by National Cancer Institute (NCI).
Recruitment status was:  Active, not recruiting
First Posted : June 8, 2006
Last Update Posted : September 17, 2013
Information provided by:
National Cancer Institute (NCI)

Brief 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.

Condition or disease Intervention/treatment Phase
Nonneoplastic Condition Biological: anti-thymocyte globulin Drug: busulfan Drug: cyclophosphamide Drug: cyclosporine Drug: dexamethasone Drug: etoposide Drug: methotrexate Drug: mycophenolate mofetil Drug: therapeutic hydrocortisone Other: laboratory biomarker analysis Procedure: allogeneic hematopoietic stem cell transplantation Procedure: biopsy Phase 3

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 288 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Hemophagocytic Lymphohistiocytosis
Study Start Date : March 2006
Estimated Primary Completion Date : November 2010

Primary Outcome Measures :
  1. Survival

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • 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


  • Not specified


  • No prior cytotoxic treatment for HLH
  • No prior cyclosporine treatment for HLH

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00334672

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United Kingdom
Birmingham Children's Hospital
Birmingham, England, United Kingdom, B4 6NH
Institute of Child Health at University of Bristol
Bristol, England, United Kingdom, BS2 8AE
Addenbrooke's Hospital
Cambridge, England, United Kingdom, CB2 2QQ
Watford General Hospital
Herts, England, United Kingdom, WD18 0HB
Leeds Cancer Centre at St. James's University Hospital
Leeds, England, United Kingdom, LS9 7TF
Royal Liverpool Children's Hospital, Alder Hey
Liverpool, England, United Kingdom, L12 2AP
Great Ormond Street Hospital for Children
London, England, United Kingdom, WC1N 3JH
Royal Manchester Children's Hospital
Manchester, England, United Kingdom, M27 4HA
Children's Hospital - Sheffield
Sheffield, England, United Kingdom, S10 2TH
Southampton General Hospital
Southampton, England, United Kingdom, SO16 6YD
Royal Aberdeen Children's Hospital
Aberdeen, Scotland, United Kingdom, AB25 2ZG
Royal Hospital for Sick Children
Edinburgh, Scotland, United Kingdom, EH9 1LF
Royal Hospital for Sick Children
Glasgow, Scotland, United Kingdom, G3 8SJ
Sponsors and Collaborators
Children's Cancer and Leukaemia Group
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Study Chair: Vasanta Nanduri, MD Watford General Hospital

Layout table for additonal information Identifier: NCT00334672     History of Changes
Other Study ID Numbers: CDR0000481605
First Posted: June 8, 2006    Key Record Dates
Last Update Posted: September 17, 2013
Last Verified: June 2009
Keywords provided by National Cancer Institute (NCI):
hemophagocytic lymphohistiocytosis
Additional relevant MeSH terms:
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Histiocytosis, Non-Langerhans-Cell
Immunologic Factors
Lymphohistiocytosis, Hemophagocytic
Lymphatic Diseases
Mycophenolic Acid
Hydrocortisone 17-butyrate 21-propionate
Hydrocortisone acetate
Hydrocortisone hemisuccinate
Antilymphocyte Serum
Anti-Inflammatory Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Immunosuppressive Agents