Peripheral Blood Stem Cell Transplant vs Bone Marrow Transplant in Individuals With Hematologic Cancers (BMT CTN 0201)
Condition: Leukemia; Myeloproliferative Disorders; Myelodysplastic-Myeloproliferative Diseases
MeSH terms: Myeloproliferative Disorders; Myelodysplastic-Myeloproliferative Diseases
Intervention: Allogeneic bone marrow transplantation; Peripheral blood stem cell transplantation
Intervention description: Bone marrow transplant from HLA compatible unrelated donors.; Peripheral blood transplant from HLA compatible unrelated donors.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 551.0
Sex: All
Maximum age: 66 Years
Healthy volunteers: No

Summary:

      The study is designed as a Phase III, randomized, open label, multicenter, prospective,
      comparative trial of granulocyte colony stimulating factor (G-CSF)-mobilized peripheral blood
      stem cells (PBSC) versus marrow from unrelated donors for transplantation in patients with
      hematologic malignancies. Recipients will be stratified by transplant center and disease risk
      and will be randomized to either the PBSC or marrow arm in a 1:1 ratio.
    

Eligibility criteria:

        Patient Inclusion Criteria:

        One of the following diagnoses:

          -  Acute myelogenous leukemia at the following stages: first remission, second remission,
             third or subsequent remission, or not in remission

          -  Acute lymphoblastic leukemia at the following stages: first remission, second
             remission, third or subsequent remission, or not in remission

          -  Chronic myelogenous leukemia at the following stages: chronic phase, accelerated
             phase, or blast phase

          -  Myelodysplastic syndromes (MDS) at the following stages: refractory anemia; refractory
             anemia with ringed sideroblasts; refractory cytopenia with multilineage dysplasia;
             refractory cytopenia with multilineage dysplasia and ringed sideroblasts; refractory
             anemia with excess blasts-1 (5-10% blasts); refractory anemia with excess blasts-2
             (10-20% blasts); myelodysplastic syndrome, unclassified; or MDS associated with
             isolated del (5q)

          -  Myeloproliferative diseases: chronic myelomonocytic leukemia; agnogenic myeloid
             metaplasia with myelofibrosis (idiopathic myelofibrosis); juvenile myelomonocytic
             leukemia

          -  Therapy-related acute myelogenous leukemia (AML) or MDS with prior malignancy that has
             been in remission for at least 12 months. If the remission is less than 12 months,
             Medical Monitor or Protocol Chair approval is required for eligibility

        Patient Exclusion Criteria:

          -  Prior allogeneic or autologous transplants using any hematopoietic stem cell source;
             patients with secondary malignancies who have had a prior autologous transplant will
             be eligible; the prior autologous transplant must have been performed for the primary
             malignancy (such as lymphoma) and must have occurred 12 or more months prior to
             enrollment

          -  Lymphoma (11% of 2001 NMDP transplants), other malignant disorders (6%), and
             non-malignant disorders (9%)

        Donor Inclusion Criteria:

          -  Matched for HLA-A, B, and DRB1 antigens

               1. One antigen mismatch at HLA-A, B, or DRB1 is acceptable with or without mismatch
                  at HLA-C

               2. Typing is by DNA techniques: intermediate resolution for A, B, and C, and high
                  resolution for DRB1. HLA-C typing is mandatory but will not count in the match.

          -  Willing to undergo both bone marrow harvest and G-CSF administration with apheresis

          -  Willing to be randomly assigned to either marrow or PBSC collection

          -  Adequate peripheral venous access for leukapheresis or willing to undergo placement of
             a central catheter

          -  Donor center affiliation with NMDP

          -  Additional donor inclusion criteria can be found in the Donor Companion Manual

        Donor Exclusion Criteria:

          -  Pregnant (positive serum β-HCG) or uninterruptible breastfeeding

          -  Known allergy to G-CSF or to E. Coli-derived recombinant protein products

          -  History of autoimmune disorders

          -  History of deep vein thrombosis or venous thromboembolism

          -  History of iritis or episcleritis

          -  History of serious adverse reaction to anesthesia

          -  Thrombocytopenia (platelets less than 150,000 per mcL) at baseline evaluation

          -  Current treatment with lithium

          -  Presence of sickle hemoglobin as demonstrated by appropriate testing such as
             hemoglobin electrophoresis

          -  Receiving experimental therapy or investigational agents