Cord Blood Stem Cell Transplantation, Hematologic Malignancies
Conditions
Keywords
Cord Blood Stem Cell Transplantation, Hematologic Malignancies, Leukemia, Myelodysplastic Syndrome
Brief summary
The hypothesis of the study is double unit umbilical cord blood transplantation in adults will be associated with a one year survival rate of at least 40%.
Interventions
PROCEDURECord blood transplantation
Myeloablative preparative regimen of chemotherapy and radiation followed by double unit umbilical cord blood transplantation
Sponsors
Center for International Blood and Marrow Transplant Research
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
22 Years to 50 Years
Healthy volunteers
No
Inclusion criteria
* Age 22 - 50 years * Patients will have one of the following hematological malignancies: * Acute myelogenous leukemia (AML): Complete first remission (CR1) at high risk for relapse or Complete second remission (CR2) * Acute lymphoblastic leukemia (ALL): Complete first remission (CR1) at high risk for relapse or Complete second remission (CR2) * Acute undifferentiated leukemia (AUL) or biphenotypic leukemia: CR1 or CR2 * Myelodysplastic Syndrome (MDS) with one of the following: Low and Intermediate-1 International Prognostic Scoring System (IPSS) score with Life-threatening neutropenia or thrombocytopenia; or Platelet transfusion dependence Intermediate-2 or High IPSS score Therapy-related disease: patient with history of chemotherapy and current evidence of MDS * Patients with adequate organ function and performance status criteria * Two Suitable Umbilical Cord Blood Units: a cryopreserved dose of at least 1.5 x 107 TNC/kg. If the unit contains red cells at time of cryopreservation, the cryopreserved dose must be at least 2.0 x 107 TNC/kg. Each unit must be at least 4/6 HLA-A and B antigen, and DRB1 allele matched with the recipient. Each unit must be at least 3/6 HLA-A, B DRB1 antigen matched to each other.
Exclusion criteria
* Patient with suitable related donor * AML, ALL, AUL, biphenotypic leukemia beyond CR2 * AML evolved from myelofibrosis * Any acute leukemia with: * Morphologic relapse or persistent disease in the BM * Active extra-medullary leukemia including active CNS leukemia * Requiring greater than two cycles of chemotherapy to obtain present remission status * Bone marrow aplasia (defined as BM cellularity \< 5% at transplant work-up) * MDS with 10% or greater bone marrow blasts at pre-transplant workup * Prior autologous or allogeneic HSC transplant at any time * Prior radiation therapy rendering patient ineligible for TBI * Any uncontrolled infection at time of study enrollment * Seropositive or NAT positive for HIV or HTLV1 * Females who are pregnant or breast feeding * Patient unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, follow-up, and research tests
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Measure overall survival of double unit UCBT in adult patients with hematologic malignancies | One year |
Secondary
| Measure | Time frame |
|---|---|
| Measure contribution of each unit to initial and sustained engraftment | 2 years |
| Measure incidence and severity of acute graft-versus-host disease | 100 Days |
| Measure incidence and severity of chronic GVHD | 1 year |
| Measure incidence of transplant-related mortality | 6 months |
| Measure incidence of donor-derived neutrophil and platelet recovery | 100 Days |
| Measure incidence of serious infectious complications | 1 year |
| Measure incidence of immune reconstitution | 2 years |
| Measure probability of overall and disease-free survival | 2 years |
| Measure incidence of malignant relapse | 2 years |
Countries
United States
Outcome results
None listed