Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified June 2011 by Cancer and Leukemia Group B.
Recruitment status was  Active, not recruiting
Eastern Cooperative Oncology Group
Information provided by:
Cancer and Leukemia Group B Identifier:
First received: December 10, 2003
Last updated: June 21, 2011
Last verified: June 2011

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 Intervention Phase
Breast Cancer
Chronic Myeloproliferative Disorders
Gestational Trophoblastic Tumor
Graft Versus Host Disease
Multiple Myeloma
Plasma Cell Neoplasm
Myelodysplastic Syndromes
Ovarian Cancer
Testicular Germ Cell Tumor
Drug: pentostatin
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:

Drug Information available for: Pentostatin
Genetic and Rare Diseases Information Center resources: Acute Lymphoblastic Leukemia Acute Monoblastic Leukemia Acute Myeloblastic Leukemia Type 5 Acute Myelocytic Leukemia Acute Myeloid Leukemia, Adult Acute Non Lymphoblastic Leukemia Anaplastic Plasmacytoma B-cell Lymphomas Burkitt Lymphoma Chronic Graft Versus Host Disease Chronic Lymphocytic Leukemia Chronic Myeloid Leukemia Chronic Myeloproliferative Disorders Cutaneous T-cell Lymphoma Follicular Lymphoma Gestational Trophoblastic Tumor Hairy Cell Leukemia Hodgkin Lymphoma Homologous Wasting Disease Hydatidiform Mole Leukemia, B-cell, Chronic Leukemia, Myeloid Lymphoblastic Lymphoma Lymphoma, Large-cell Lymphoma, Large-cell, Immunoblastic Lymphoma, Small Cleaved-cell, Diffuse Lymphosarcoma Mantle Cell Lymphoma Multiple Myeloma Myelodysplastic Syndromes Myelofibrosis Neural Crest Tumor Neuroblastoma Neuroepithelioma Ovarian Cancer Ovarian Epithelial Cancer Ovarian Germ Cell Tumor Plasmablastic Lymphoma Small Non-cleaved Cell Lymphoma Squamous Cell Carcinoma of the Head and Neck Testicular Cancer
U.S. FDA Resources

Further study details as provided by Cancer and Leukemia Group B:

Primary Outcome Measures:
  • Response rate [ Time Frame: 3 months ] [ Designated as safety issue: No ]
    Both complete and partial response will be assessed

Secondary Outcome Measures:
  • Toxicity [ Time Frame: During tx, then at relapse or progression ] [ Designated as safety issue: Yes ]
  • Survival [ Time Frame: At 1 year and 2 years post treatment initiation ] [ Designated as safety issue: No ]
  • Pharmacokinetics [ Time Frame: At initiation of Tx and at 3 months ] [ Designated as safety issue: No ]

Enrollment: 39
Study Start Date: December 2003
Estimated Study Completion Date: March 2013
Primary Completion Date: August 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • Histologically confirmed chronic graft-versus-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation or donor lymphocyte infusion

    • Progressive, quiescent, or de novo onset
  • Extensive stage disease requiring systemic immunosuppressive therapy, defined according to Seattle criteria as 1 of the following:

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

      • Liver histology showing chronic progressive hepatitis, bridging necrosis, or cirrhosis
      • Eye involvement (i.e., Schirmer's test with less than 5 mm wetting)
      • Involvement of minor salivary glands or oral mucosa
      • Involvement of any other organ
  • Failed prior corticosteroid therapy, meeting 1 of the following criteria:

    • Progressive disease or less than a minor response in any organ system despite 2 weeks on steroid therapy at a dose of at least 1 mg/kg of methylprednisolone or equivalent
    • No response or minor response after at least 4 weeks of steroid therapy at a dose of least 0.5 mg/kg of methylprednisolone or equivalent
    • Less than a partial response after 8 weeks of steroid therapy at a dose of least 0.5 mg/kg of methylprednisolone or equivalent
    • Required a dose of least 0.5 mg/kg of methylprednisolone or equivalent after completion of at least 12 weeks of corticosteroid therapy in order to maintain a partial response or better
    • Required a dose of least 10 mg/kg of methylprednisolone or equivalent after completion of at least 18 weeks of corticosteroid therapy in order to maintain a partial response or better
    • Progressive extensive stage chronic GVHD after completion of at least 18 weeks of corticosteroid therapy and currently requiring reintroduction of corticosteroid therapy at a dose of least 10 mg/kg of methylprednisolone or equivalent OR an additional therapy (e.g., photopheresis or psoralen-ultraviolet-light [PUVA] therapy)
  • Established chronic GVHD either not improving or progressing on other immunosuppressive agents allowed provided steroid refractoriness has been previously established



  • 18 and over

Performance status

  • 0-3

Life expectancy

  • Not specified


  • Absolute neutrophil count greater than 1,000/mm^3
  • Platelet count greater than 50,000/mm^3 (without transfusion)


  • Not specified


  • Creatinine clearance at least 30 mL/min


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • HIV negative
  • No active infection


Biologic therapy

  • See Disease Characteristics


  • Not specified

Endocrine therapy

  • See Disease Characteristics
  • No concurrent corticosteroids as antiemetics


  • Not specified


  • Not specified


  • Concurrent continuation of other immunosuppressants administered during onset or progression of chronic GVHD is allowed
  • No concurrent mechanical ventilation
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00074035

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 Chicago Cancer Research Center
Chicago, Illinois, United States, 60637-1470
University of Illinois Cancer Center
Chicago, Illinois, United States, 60612-7243
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 MD, 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
Cancer and Leukemia Group B
Eastern Cooperative Oncology Group
Study Chair: Sherif S. Farag, MD, PhD Ohio State University
  More Information

Additional Information:
No publications provided

Responsible Party: Monica M Bertagnolli, MD, Cancer and Leukemia Group B Identifier: NCT00074035     History of Changes
Other Study ID Numbers: CDR0000341678, U10CA031946, CALGB-100101, ECOG-1010
Study First Received: December 10, 2003
Last Updated: June 21, 2011
Health Authority: United States: Federal Government

Keywords provided by Cancer and Leukemia Group B:
graft versus host disease
accelerated phase chronic myelogenous leukemia
adult acute lymphoblastic leukemia in remission
adult acute myeloid leukemia in remission
blastic phase chronic myelogenous leukemia
primary myelofibrosis
chronic phase chronic myelogenous leukemia
de novo myelodysplastic syndromes
disseminated neuroblastoma
meningeal chronic myelogenous leukemia
noncontiguous stage II adult diffuse large cell lymphoma
noncontiguous stage II adult diffuse mixed cell lymphoma
noncontiguous stage II adult Burkitt lymphoma
noncontiguous stage II adult immunoblastic large cell lymphoma
noncontiguous stage II adult lymphoblastic lymphoma
noncontiguous stage II grade 3 follicular lymphoma
noncontiguous stage II mantle cell lymphoma
poor prognosis metastatic gestational trophoblastic tumor
previously treated myelodysplastic syndromes
recurrent adult acute lymphoblastic leukemia
recurrent adult acute myeloid leukemia
recurrent adult diffuse large cell lymphoma
recurrent adult diffuse mixed cell lymphoma
recurrent adult diffuse small cleaved cell lymphoma
recurrent adult Burkitt lymphoma
recurrent adult Hodgkin lymphoma
recurrent adult immunoblastic large cell lymphoma
recurrent adult lymphoblastic lymphoma
recurrent cutaneous T-cell non-Hodgkin lymphoma
recurrent grade 1 follicular lymphoma

Additional relevant MeSH terms:
Breast Neoplasms
Gestational Trophoblastic Disease
Graft vs Host Disease
Multiple Myeloma
Myelodysplastic Syndromes
Myeloproliferative Disorders
Neoplasms, Germ Cell and Embryonal
Neoplasms, Plasma Cell
Testicular Neoplasms
Trophoblastic Neoplasms
Blood Protein Disorders
Bone Marrow Diseases
Breast Diseases
Cardiovascular Diseases
Endocrine Gland Neoplasms
Endocrine System Diseases
Genital Diseases, Male
Genital Neoplasms, Male
Gonadal Disorders
Hematologic Diseases
Hemorrhagic Disorders
Hemostatic Disorders
Immune System Diseases
Immunoproliferative Disorders
Lymphoproliferative Disorders
Neoplasms by Histologic Type processed this record on February 27, 2015