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Rasburicase in Preventing Graft-Versus-Host Disease in Patients With Hematologic Cancer or Other Disease Undergoing Donor Stem Cell Transplant

This study has been completed.
Information provided by (Responsible Party):
Bimalangshu Dey, Massachusetts General Hospital Identifier:
First received: August 6, 2007
Last updated: May 8, 2015
Last verified: May 2015

RATIONALE: Rasburicase may be an effective treatment for graft-versus-host disease caused by a donor stem cell transplant.

PURPOSE: This clinical trial is studying how well rasburicase works in preventing graft-versus-host disease in patients with hematologic cancer or other disease undergoing donor stem cell transplant.

Condition Intervention
Chronic Myeloproliferative Disorders
Graft Versus Host Disease
Multiple Myeloma and Plasma Cell Neoplasm
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Neoplasms
Drug: busulfan
Drug: cyclophosphamide
Drug: cyclosporine
Drug: etoposide
Drug: methotrexate
Drug: rasburicase
Drug: sirolimus
Drug: tacrolimus
Other: diagnostic laboratory biomarker analysis
Other: flow cytometry
Other: immunologic technique
Procedure: allogeneic hematopoietic stem cell transplantation
Procedure: peripheral blood stem cell transplantation
Radiation: total-body irradiation

Study Type: Interventional
Study Design: Masking: Open Label
Primary Purpose: Supportive Care
Official Title: Rasburicase to Prevent Graft -Versus-Host Disease

Resource links provided by NLM:

Further study details as provided by Massachusetts General Hospital:

Primary Outcome Measures:
  • Incidence and severity of acute graft-vs-host disease

Secondary Outcome Measures:
  • Efficacy (in terms of reduction of uric acid levels) and safety
  • Graft-versus-host and host-versus-graft immune responses

Estimated Enrollment: 25
Study Start Date: January 2008
Study Completion Date: March 2013
Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)
Detailed Description:



  • To evaluate the incidence and severity of acute graft-vs-host disease (GVHD) in rasburicase-treated patients who will undergo myeloablative HLA-matched related or unrelated donor allogeneic peripheral blood hematopoietic stem cell transplantation (SCT) for hematologic malignancies and compare these outcomes with those of historical controls.


  • To evaluate the efficacy (in terms of reduction of uric acid levels) and safety of rasburicase in patients undergoing myeloablative allogeneic SCT.
  • To evaluate the graft-versus-host and host-versus-graft immune responses in rasburicase-treated patients.

OUTLINE: This is a multicenter study.

Patients receive a conventional myeloablative conditioning regimen consisting of high doses of cyclophosphamide, busulfan, and etoposide, with or without total-body irradiation. Depending on the preparative regimen selected, the conditioning of recipients will take a total of 6 to 7 days. On day 0, patients will receive filgrastim (G-CSF)-mobilized HLA-matched, related, or unrelated donor allogeneic peripheral blood stem cells (unmanipulated). Patients will receive standard graft-vs-host disease prophylaxis consisting of cyclosporine or tacrolimus and methotrexate or sirolimus. Patients will receive rasburicase IV over 30 minutes, beginning on the first day of conditioning therapy, for 5 consecutive days. If after 5 days of rasburicase the patient's uric acid plasma level remains above 5 mg/dL, rasburicase may be continued for up to 7 days in total.

Blood is obtained on day 0 and then at 14, 28, and 42 days post-transplant for immunologic studies, including quantitative analysis to follow the recovery of T cells, B cells, natural killer cells, dendritic cells (DC), and monocytes using flow cytometry (FCM); phenotypic analysis of T cells, DC and monocytes by FCM; lymphocyte activation analysis: CD3, CD4, CD8, CD25 2. CD3, CD8, CD71, CD69; DC analysis: CD45, CD14, DR, CD86, CD80 2. CD45, CD14, CD40, CD11c; and in vitro functional studies such as mixed lymphocyte reaction (MLR) and cell-mediated lysis (CML) to assess for the graft-versus-host and host-versus-graft responses. Peripheral blood is collected for chimerism studies on days 28 and 100 post-transplant.

After completion of study treatment, patients are followed periodically.


Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Patients with hematologic malignancies for whom conventional myeloablative allogeneic stem cell transplantation is deemed clinically appropriate and who are eligible for conventional myeloablative allogeneic stem cell transplantation on treatment plans/protocols, including any of the following:

    • Non-Hodgkin lymphoma or Hodgkin lymphoma (relapsed or refractory disease)
    • Chronic lymphocytic leukemia (received more than one previous treatment regimen)
    • Acute myelogenous or lymphoblastic leukemia (AML/ALL) (high-risk disease, in first complete remission [CR1] or subsequent remission, or primary refractory disease)
    • Chronic myelogenous leukemia in tyrosine-kinase resistant chronic phase, accelerated or blast phase, or primary refractory disease
    • Myelodysplastic syndromes in IPSS (International Prognostic Scoring System) high-intermediate or high-risk groups
    • Other hematologic disorders for which allogeneic stem cell transplantation is appropriate (e.g., myelofibrosis)
  • Patients who have relapsed after standard autologous and/or allogeneic bone marrow transplant are eligible
  • Must be receiving filgrastim (G-CSF)-mobilized related or unrelated donor allogeneic peripheral blood stem cells

    • Patients receiving hematopoietic stem cells of any other sources such as a marrow graft or umbilical cord blood will not be eligible for this study
  • Donor must be HLA-genotypically or phenotypically 6 of 6 antigen matched (at the A, B, DR loci) related or unrelated


Inclusion criteria:

  • Patients with a "currently active" second malignancy other than non-melanoma skin cancers can only be registered if survival from the second malignancy is expected to be more than 1 year
  • Ejection fraction ≥ 45% by either radioisotope MUGA scan or ECHO
  • Lung DLCO ≥ 50% of predicted with no symptomatic pulmonary disease
  • Mini Mental Status Exam Score ≥ 20
  • Patients must have an expected life expectancy of at least 3 months
  • Patients with symptomatic visceral, blood stream or nervous system opportunistic infection are eligible if the infection has been appropriately treated and controlled

    • Patients with a fungal infection must have had treatment for at least one month and must have proof of regression of the infection prior to enrollment
    • Patients may be on antibiotics at the time of transplant

Exclusion criteria:

  • HIV infection
  • Uncontrolled diabetes mellitus
  • Active congestive heart failure from any cause

    • Previous history of congestive heart failure allowed
  • Active angina pectoris
  • Oxygen-dependent obstructive pulmonary disease
  • Failure to demonstrate adequate compliance with medical therapy and follow-up
  • Known history of G6PD deficiency or history of hemolysis indicative of G6PD deficiency


  • See Disease Characteristics
  Contacts and Locations
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Please refer to this study by its identifier: NCT00513474

United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114-2617
Sponsors and Collaborators
Massachusetts General Hospital
Principal Investigator: Bimalangshu R. Dey, MD, PhD Massachusetts General Hospital
  More Information

Responsible Party: Bimalangshu Dey, Associate Professor of Medicine, Massachusetts General Hospital Identifier: NCT00513474     History of Changes
Other Study ID Numbers: CDR0000558480
Study First Received: August 6, 2007
Last Updated: May 8, 2015

Keywords provided by Massachusetts General Hospital:
graft versus host disease
anaplastic large cell lymphoma
angioimmunoblastic T-cell lymphoma
extranodal marginal zone B-cell lymphoma of mucosa-associated lymphoid tissue
nodal marginal zone B-cell lymphoma
recurrent adult Burkitt lymphoma
recurrent adult diffuse large cell lymphoma
recurrent adult diffuse mixed cell lymphoma
recurrent adult diffuse small cleaved cell lymphoma
recurrent adult Hodgkin lymphoma
recurrent adult immunoblastic large cell lymphoma
recurrent adult lymphoblastic lymphoma
recurrent adult T-cell leukemia/lymphoma
recurrent cutaneous T-cell non-Hodgkin lymphoma
recurrent grade 1 follicular lymphoma
recurrent grade 2 follicular lymphoma
recurrent grade 3 follicular lymphoma
recurrent mantle cell lymphoma
recurrent marginal zone lymphoma
recurrent mycosis fungoides/Sezary syndrome
recurrent small lymphocytic lymphoma
splenic marginal zone lymphoma
Waldenström macroglobulinemia
recurrent adult acute lymphoblastic leukemia
refractory chronic lymphocytic leukemia
recurrent adult acute myeloid leukemia
adult acute myeloid leukemia with 11q23 (MLL) abnormalities
adult acute myeloid leukemia with inv(16)(p13;q22)
adult acute myeloid leukemia with t(15;17)(q22;q12)
adult acute myeloid leukemia with t(16;16)(p13;q22)

Additional relevant MeSH terms:
Multiple Myeloma
Neoplasms, Plasma Cell
Myelodysplastic Syndromes
Graft vs Host Disease
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Lymphatic Diseases
Pathologic Processes
Bone Marrow Diseases
Precancerous Conditions
Methotrexate processed this record on March 23, 2017