Transplantation of Umbilical Cord Blood From Related and Unrelated Donors
N/A
45 Years
Both
Chronic Myeloproliferative Disorders, Diamond-blackfan Anemia, Fanconi Anemia, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Trial Information
Transplantation of Umbilical Cord Blood From Related and Unrelated Donors
OBJECTIVES:
Primary
- Determine the engraftment potential of umbilical cord blood (UCB) in patients with
hematological cancers.
- Determine the safety of UCB transplantation in these patients.
Secondary
- Determine the rate of neutrophil and platelet recovery and the completeness of donor
cell engraftment.
- Determine the incidence and severity of acute and chronic graft-versus-host disease.
- Determine the incidence of relapse in patients with malignant disease.
- Determine the probabilities of survival and event-free survival (EFS) at 1 and 2 years
after UCB transplantation.
OUTLINE: Patients are stratified according to degree of HLA disparity (0-1 vs 2-3
disparities between donor and recipient), donor type (related vs unrelated), and the basis
of cell dose (< 2 vs ≥ 2 x 10^7 nucleated cells/kg recipient body weight). Patients are
assigned to 1 of 4 treatment groups according to disease*.
NOTE: *Patients with acute lymphocytic leukemia (ALL), secondary acute myeloid leukemia
(AML), severe combined immunodeficiency, familial erythrophagocytic lymphohistiocytosis
(FEL)/viral-associated hemophagocytic syndrome (VAHS), inborn errors of metabolism, aplastic
anemia, Fanconi's anemia, or Diamond-Blackfan anemia who have an unrelated umbilical cord
blood donor may proceed directly to transplantation.
- Preparative regimen:
- Group 1 (patients with chronic myelogenous leukemia, AML, myelodysplastic
syndromes, or ALL): Patients receive cyclophosphamide IV once daily on days -7 and
-6. Patients then undergo total-body irradiation (TBI) twice daily on days -4 to
-1. Patients undergoing unrelated allogeneic umbilical cord blood transplantation
(UCBT) also receive methylprednisone IV and anti-thymocyte globulin (ATG) IV twice
daily on days -2 and -1.
- Group 2 (patients with infant leukemia): Patients receive busulfan orally or IV
four times daily on days -9 to -6 and melphalan IV once daily on days -4 to -2.
Patients undergoing unrelated allogeneic UCBT also receive methylprednisolone IV
and ATG IV twice daily on days -2 and -1.
- Group 3 (patients with inborn errors of metabolism): Patients receive busulfan
orally or IV four times daily on days -9 to -6 and cyclophosphamide IV once daily
on days -5 to -2. Patients undergoing unrelated allogeneic UCBT also receive
methylprednisolone IV and ATG IV twice daily on days -2 and -1.
- Group 4 (patients with aplastic anemia): Patients receive cyclophosphamide IV once
daily on days -6 to -3 and ATG IV twice daily on days -5 and -3. Patients then
undergo TBI once on day -2.
- Allogeneic UCBT: Patients undergo UCBT on day 0. Patients with inborn errors of
metabolism receive methylprednisolone IV before and after UCBT on day 0.
- Graft-versus-host disease (GVHD) prophylaxis: Patients receive 1 of the following
regimens:
- Related donor UCBT: Patients receive cyclosporine IV over 2 hours or orally
beginning on day -3 and continuing until day 60.
- Unrelated donor UCBT and myeloablative preparative regimen: Patients receive
cyclosporine orally or IV over 2 hours twice daily beginning on day -3 and
continuing until day 180. Patients also receive methylprednisolone IV twice daily
on days 5-19.
- Unrelated donor UCBT and nonmyeloablative preparative regimen: Patients receive
cyclosporine IV over 2 hours or orally twice daily beginning on day -3 and
continuing until day 180. Patients also receive mycophenolate mofetil IV or orally
beginning on day 5 and continuing until day 30 or 7 days after active GVHD is
controlled.
All patients receive filgrastim (G-CSF) IV beginning on day 1 and continuing until blood
counts recover.
Patients are followed periodically for 2 years.
PROJECTED ACCRUAL: A total of 60 patients will be accrued for this study.
Inclusion Criteria
DISEASE CHARACTERISTICS:
- Diagnosis of 1 of the following:
- Leukemia including, but not limited to, the following subtypes:
- Chronic myelogenous leukemia
- Acute myeloid leukemia (primary or secondary)
- Acute lymphoblastic leukemia
- Lymphoma
- Myelodysplastic syndrome
- Aplastic anemia
- Fanconi's anemia
- Diamond-Blackfan anemia
- Inborn errors of metabolism (e.g., Hurler syndrome, Maroteaux-Lamy syndrome,
myelodysplastic syndrome VI, metachromatic leukodystrophy, adrenoleukodystrophy,
and globoid cell leukodystrophy)
- Immune deficiency disorders
- Patients must meet the eligibility requirements outlined in currently active
treatment protocols of the University of Minnesota Bone Marrow Transplant Program
- HLA-identical or 1, 2, or 3 antigen mismatched umbilical cord blood (UCB) donor with
at least one DRB1 match available
- Unrelated or related donor
- UCB specimen must contain ≥ 2.0 x 10^7 nucleated cells/kg patient body weight
PATIENT CHARACTERISTICS:
- See Disease Characteristics
- No active infection
- No history of HIV infection
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception
PRIOR CONCURRENT THERAPY:
- Not specified
Type of Study:
Interventional
Study Design:
Masking: Open Label, Primary Purpose: Treatment
Principal Investigator
John E. Wagner, MD
Investigator Role:
Study Chair
Investigator Affiliation:
Masonic Cancer Center, University of Minnesota
Authority:
United States: Federal Government
Study ID:
2000LS017
NCT ID:
NCT00290628
Start Date:
October 1999
Completion Date:
April 2007
Related Keywords:
- Chronic Myeloproliferative Disorders
- Diamond-Blackfan Anemia
- Fanconi Anemia
- Graft Versus Host Disease
- Leukemia
- Lymphoma
- Multiple Myeloma and Plasma Cell Neoplasm
- Myelodysplastic Syndromes
- Myelodysplastic/Myeloproliferative Diseases
- graft versus host disease
- 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)
- adult acute myeloid leukemia with t(8;21)(q22;q22)
- accelerated phase chronic myelogenous leukemia
- adult acute lymphoblastic leukemia in remission
- adult acute myeloid leukemia in remission
- atypical chronic myeloid leukemia
- blastic phase chronic myelogenous leukemia
- childhood acute lymphoblastic leukemia in remission
- childhood acute myeloid leukemia in remission
- childhood chronic myelogenous leukemia
- chronic eosinophilic leukemia
- chronic idiopathic myelofibrosis
- chronic myelomonocytic leukemia
- chronic neutrophilic leukemia
- chronic phase chronic myelogenous leukemia
- de novo myelodysplastic syndromes
- extranodal marginal zone B-cell lymphoma of mucosa-associated lymphoid tissue
- juvenile myelomonocytic leukemia
- myelodysplastic/myeloproliferative disease, unclassifiable
- nodal marginal zone B-cell lymphoma
- noncontiguous stage II adult Burkitt lymphoma
- noncontiguous stage II adult diffuse large cell lymphoma
- noncontiguous stage II adult diffuse mixed cell lymphoma
- noncontiguous stage II adult diffuse small cleaved cell lymphoma
- noncontiguous stage II adult immunoblastic large cell lymphoma
- noncontiguous stage II adult lymphoblastic lymphoma
- noncontiguous stage II grade 1 follicular lymphoma
- noncontiguous stage II grade 2 follicular lymphoma
- noncontiguous stage II grade 3 follicular lymphoma
- noncontiguous stage II mantle cell lymphoma
- noncontiguous stage II marginal zone lymphoma
- noncontiguous stage II small lymphocytic lymphoma
- previously treated myelodysplastic syndromes
- recurrent adult acute lymphoblastic leukemia
- recurrent adult acute myeloid leukemia
- recurrent adult Burkitt lymphoma
- recurrent adult Hodgkin lymphoma
- recurrent adult diffuse large cell lymphoma
- recurrent adult diffuse mixed cell lymphoma
- recurrent adult diffuse small cleaved cell lymphoma
- recurrent adult immunoblastic large cell lymphoma
- recurrent adult lymphoblastic lymphoma
- recurrent/refractory childhood Hodgkin lymphoma
- recurrent childhood acute lymphoblastic leukemia
- recurrent childhood acute myeloid leukemia
- recurrent childhood large cell lymphoma
- recurrent childhood lymphoblastic lymphoma
- recurrent childhood small noncleaved cell 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
- refractory chronic lymphocytic leukemia
- refractory hairy cell leukemia
- refractory multiple myeloma
- relapsing chronic myelogenous leukemia
- secondary acute myeloid leukemia
- secondary myelodysplastic syndromes
- splenic marginal zone lymphoma
- stage III adult Burkitt lymphoma
- stage III adult Hodgkin lymphoma
- stage III adult diffuse large cell lymphoma
- stage III adult diffuse mixed cell lymphoma
- stage III adult diffuse small cleaved cell lymphoma
- stage III adult immunoblastic large cell lymphoma
- stage III adult lymphoblastic lymphoma
- stage III chronic lymphocytic leukemia
- stage III grade 1 follicular lymphoma
- stage III grade 2 follicular lymphoma
- stage III grade 3 follicular lymphoma
- stage III mantle cell lymphoma
- stage III marginal zone lymphoma
- stage III small lymphocytic lymphoma
- stage IV adult Burkitt lymphoma
- stage IV adult Hodgkin lymphoma
- stage IV adult diffuse large cell lymphoma
- stage IV adult diffuse mixed cell lymphoma
- stage IV adult diffuse small cleaved cell lymphoma
- stage IV adult immunoblastic large cell lymphoma
- stage IV adult lymphoblastic lymphoma
- stage IV chronic lymphocytic leukemia
- stage IV grade 1 follicular lymphoma
- stage IV grade 2 follicular lymphoma
- stage IV grade 3 follicular lymphoma
- stage IV mantle cell lymphoma
- stage IV marginal zone lymphoma
- stage IV small lymphocytic lymphoma
- Diamond-Blackfan anemia
- Fanconi anemia
- childhood myelodysplastic syndromes
- Anemia
- Neoplasms
- Fanconi Anemia
- Fanconi Syndrome
- Graft vs Host Disease
- Leukemia
- Lymphoma
- Lymphoma, Non-Hodgkin
- Multiple Myeloma
- Neoplasms, Plasma Cell
- Plasmacytoma
- Myelodysplastic Syndromes
- Preleukemia
- Myeloproliferative Disorders
- Lymphoma, Large-Cell, Immunoblastic
- Anemia, Diamond-Blackfan
- Myelodysplastic-Myeloproliferative Diseases
Name | Location |
|---|---|
| University of Minnesota Cancer Center | Minneapolis, Minnesota 55455 |
