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Pentostatin in Treating Patients With Refractory Chronic Graft-Versus-Host Disease

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: NCT00074035
Recruitment Status : Completed
First Posted : December 11, 2003
Results First Posted : March 15, 2017
Last Update Posted : May 8, 2018
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Alliance for Clinical Trials in Oncology

Brief Summary:

RATIONALE: Pentostatin may be effective in treating chronic graft-versus-host disease by stopping the immune system from rejecting donor stem cells or donor white blood cells.

PURPOSE: This phase II trial is studying how well pentostatin works in treating patients with chronic graft-versus-host disease that is refractory (not responsive) to treatment with steroids.

Condition or disease Intervention/treatment Phase
Graft Versus Host Disease Drug: pentostatin Phase 2

Detailed Description:



  • Determine the response rate in patients with refractory chronic graft-versus-host disease treated with pentostatin.


  • Determine the time to next immunosuppressive agent (i.e., the time to progression from best response) in patients treated with this drug.
  • Determine the toxicity of this drug in these patients.
  • Determine the infection rate in patients treated with this drug.
  • Determine the pharmacokinetics of this drug in these patients.
  • Determine the changes in lymphocyte populations in patients treated with this drug.
  • Determine the survival of patients treated with this drug.

OUTLINE: This is a multicenter study.

Patients receive pentostatin IV over 20-30 minutes on day 1. Treatment repeats every 14 days for 6 courses in the absence of disease progression or unacceptable toxicity.

Patients who achieve a complete response after 6 courses receive 4 additional courses. Patients who achieve a partial response, minor response, or stable disease after 6 courses may receive up to 6 additional courses.

Patients are followed every 4 weeks for 1 year, every 3 months for 2 years, and then annually for 5 years.

PROJECTED ACCRUAL: Approximately 37 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 39 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
Official Title: A Phase II Trial Of Intravenous Pentostatin For The Treatment Of Patients With Refractory Chronic Graft-Versus-Host Disease
Actual Study Start Date : December 2003
Actual Primary Completion Date : August 2008
Actual Study Completion Date : November 1, 2014

Arm Intervention/treatment
Experimental: Pentostatin
treatment of pts with refractory graft vs host disease
Drug: pentostatin
4 mg/sq m IV infusion over 20-30 min q 2 weeks

Primary Outcome Measures :
  1. Response Rate [ Time Frame: 3 months ]

    Percentage of participants who had a complete or partial response defined by the Hopkins scoring system.

    A complete response is defined as the disappearance of signs and symptoms of chronic GVHD in all involved systems that is sustained for at lest 4 weeks. A partial response is an improvement by 2 or more points in at least one system score, which is sustained for at least 4 weeks, with no signs of worsening in others.

Secondary Outcome Measures :
  1. Grade 3 or Higher Non-hematologic Adverse Events [ Time Frame: Duration of treatment (up to 5 years) ]
    Number of participants experiencing a grade 3, 4 or 5 clinically significant non-hematologic adverse events, at least possibly related to treatment.

  2. Overall Survival At 1 Year [ Time Frame: 1 year ]
    Percentage of patients who were alive at 1 year.

  3. Overall Survival At 2 Years [ Time Frame: 2 year ]
    Percentage of patients who were alive at 2 years.

  4. Pharmacokinetics [ Time Frame: At initiation of Tx and at 3 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
  1. Histologic documentation of chronic GvHD following allogeneic HCT or donor lymphocyte infusion.
  2. Patients may have progressive, quiescent, or de novo onset chronic GvHD.
  3. Patients with extensive stage chronic GvHD requiring systemic immunosuppressive therapy are eligible. Patients with limited stage disease are excluded. Extensive stage is defined according to Seattle criteria (9) as either:

    • Generalized skin involvement or
    • Limited skin involvement or hepatic involvement with any one of the following:

      • Liver histology showing chronic progressive hepatitis, bridging necrosis or cirrhosis
      • Eye involvement (Schirmer's test with < 5 mm wetting)
      • Involvement of minor salivary glands or oral mucosa
      • Involvement of any other organ
  4. Patients must have failed treatment with, or experience progression after, prior corticosteroids for extensive stage chronic GvHD, as defined below.

    4.1 Patients will be considered to have failed corticosteroids if they have any one of the following criteria:

    • Progressive disease or less than a minor response in any organ system despite 2 weeks on corticosteroid treatment at least 1 mg/kg methylprednisolone or equivalent.
    • Failure to achieve at least a minor response after at least 4 weeks of treatment with a dose of ≥ 0.5 mg/kg methylprednisolone or equivalent.
    • Achievement of less than a partial response at 8 weeks of corticosteroid treatment despite use of a dose ≥ 0.5 mg/kg methylprednisolone or equivalent.
    • Requirement of ≥ 0.5 mg/kg methylprednisolone or equivalent to maintain a partial response or better at 12 weeks of corticosteroid treatment.
    • Requirement of > 10 mg/kg methylprednisolone or equivalent to maintain a partial response or better at 18 weeks of corticosteroid treatment.

    4.2 Patients with progression of extensive stage chronic GvHD after a prior history of treatment with at least 18 weeks of corticosteroids, now requiring the reintroduction of corticosteroids (> 10 mg/day methylprednisolone or equivalent) or an additional agent (including photopheresis, PUVA) for treatment.

  5. Patients with established chronic GvHD not improving or progressing on other immunosuppressive agents are also eligible if steroid refractoriness has been established previously.
  6. Age ≥ 18 years
  7. Performance Status 0-3
  8. Patients on mechanical ventilation are excluded.
  9. No active infection. Patients with active infection requiring antibiotic therapy are not eligible until infection is controlled.
  10. No HIV infection. Patients with HIV infection are excluded because of safety concerns in this patient population.
  11. Non-pregnant and non-nursing. Women and men of reproductive potential should agree to use an appropriate method of birth control throughout their participation in this study due to the teratogenic potential of the therapy utilized in this trial (although it is unlikely that successful pregnancy will occur in patients with chronic GvHD). Appropriate methods of birth control include oral contraceptives, implantable hormonal contraceptives (Norplant®), or double barrier method (diaphragm plus condom).
  12. Required Initial Laboratory Values:

    • Calc. Creatinine Clearance ≥ 30 mL/min/1.73 m^2
    • ANC > 1000/μL
    • Platelets > 50,000/μL without transfusion

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): NCT00074035

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United States, Delaware
Tunnell Cancer Center at Beebe Medical Center
Lewes, Delaware, United States, 19958
CCOP - Christiana Care Health Services
Newark, Delaware, United States, 19713
United States, Illinois
University of Illinois Cancer Center
Chicago, Illinois, United States, 60612-7243
University of Chicago Cancer Research Center
Chicago, Illinois, United States, 60637-1470
United States, Maryland
Greenebaum Cancer Center at University of Maryland Medical Center
Baltimore, Maryland, United States, 21201
Union Hospital Cancer Program at Union Hospital
Elkton, Maryland, United States, 21921
United States, Minnesota
Mayo Clinic Cancer Center
Rochester, Minnesota, United States, 55905
United States, New Jersey
Cancer Institute of New Jersey at Cooper - Voorhees
Voorhees, New Jersey, United States, 08043
United States, New York
New York Weill Cornell Cancer Center at Cornell University
New York, New York, United States, 10021
United States, North Carolina
Duke Comprehensive Cancer Center
Durham, North Carolina, United States, 27710
Wake Forest University Comprehensive Cancer Center
Winston-Salem, North Carolina, United States, 27157-1096
United States, Ohio
Arthur G. James Cancer Hospital and Solove Research Institute at Ohio State University Medical Center
Columbus, Ohio, United States, 43210-1240
United States, Pennsylvania
Abramson Cancer Center of the University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104-4283
Fox Chase Cancer Center - Philadelphia
Philadelphia, Pennsylvania, United States, 19111-2497
Western Pennsylvania Cancer Institute at Western Pennsylvania Hospital
Pittsburgh, Pennsylvania, United States, 15224-1791
United States, Virginia
Virginia Commonwealth University Massey Cancer Center
Richmond, Virginia, United States, 23298-0037
Sponsors and Collaborators
Alliance for Clinical Trials in Oncology
National Cancer Institute (NCI)
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Study Chair: Sherif S. Farag, MD, PhD Ohio State University
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Responsible Party: Alliance for Clinical Trials in Oncology Identifier: NCT00074035    
Other Study ID Numbers: CALGB-100101
U10CA031946 ( U.S. NIH Grant/Contract )
CDR0000341678 ( Registry Identifier: NCI Physician Data Query )
First Posted: December 11, 2003    Key Record Dates
Results First Posted: March 15, 2017
Last Update Posted: May 8, 2018
Last Verified: April 2018
Additional relevant MeSH terms:
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Graft vs Host Disease
Immune System Diseases
Antineoplastic Agents
Adenosine Deaminase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action