poor-risk hematological malignancy
Conditions
Interventions
Patients are treated with a reduced-intensity conditioning regimen,
irrespective of patient age, followed by double UCBT. Post grafting
immunosuppression is performed by mycophenolate mofetil (30 da
Sponsors
HOVON
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: - Age 18-70years inclusive - Diagnosis of poor-risk hematological malignancy or (V)SAA relapsing after or failing immunosuppressive therapy and meeting the criteria for a MUD allo SCT - Lacking a sufficiently matched volunteer unrelated donor or lacking such a donor within the required time period of =4/6) matched UCB grafts with a total nuclear cell count > 4 x 107/kg (see paragraph 8.2). - WHO performance status 0-2 - Written informed consent
Exclusion criteria
Exclusion criteria: - Bilirubin and/or transaminases > 2.5 x normal value - Creatinine clearance
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| - The proportion of patients with activated class II-specific T-cells (aTCs), defined as: the number of patients with aTCs, divided by the number of patients with class II mismatches for which there are tests available (defined as evaluable patients). - Transplant related mortality (TRM; defined as non-relapse mortality) | — |
Secondary
| Measure | Time frame |
|---|---|
| - Cumulative incidence of engraftment - Cumulative incidence of graft failure - Time to neutrophil recovery - Time to lymphocyte recovery - Time to platelet recovery - Time to red blood cell transfusion independence - Count of total CD3+, CD4+ and CD8+ cells and CD3-CD16/56+ cells at 3, 6, 12 and 24 months after UCBT - Incidence and grade of acute GVHD - Incidence of chronic GVHD - Incidence of infections - Progression free survival (PFS, i.e. time from transplantation until progression/relapse or death from any cause, whichever comes first) - Overall survival (OS) calculated from transplantation. | — |
Countries
Netherlands
Outcome results
None listed