single cord blood transplantation stemregenin-1 expanded hematopoietic stem cells engraftment
Conditions
Interventions
Patients are treated with a reduced-intensity conditioning regimen, irrespective of patient age, followed by single UCBT, using one SR-1 expanded unit. Post grafting immunosuppression is performed by
Sponsors
Erasmus MC
Eligibility
Inclusion criteria
Inclusion criteria: - Age 18-70 years inclusive - Diagnosis of poor-risk hematological malignancy 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 ≤ 2 months in case of urgently needed alloSCT - Availability of 1 (≥5/6) matched UCB graft with a nuclear cell count > 2,7 x 107/kg (see paragraph 8.2). - Availability of an back-up autograft, harvested and frozen earlier in the course of treatment, (harvest according to local aphersis policies) - WHO performance status 0-2 - Written informed consent
Exclusion criteria
Exclusion criteria: - Bilirubin and/or transaminases > 2.5 x normal value - Creatinine clearance < 40 ml/min - Cardiac dysfunction as defined by: Reduced left ventricular function with an ejection fraction < 45% as measured by MUGA scan or echocardiogram (another method for measuring cardiac function is acceptable) Unstable angina Unstable cardiac arrhythmias - Pulmonary function test with VC, FEV1 and/ or DCO < 50% - Active, uncontrolled infection - History of high dose (≥ 8 Gy) total body irradiation - Pregnant or lactating females - HIV positivity
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Feasibility as defined by, and to be achieved in ≥ 80% of (evaluable) patients: 1. SR-1 mediated expansion, resulting in > 20-fold expansion of CD34+ cells, and 2. effective hematopoietic (neutrophils > 0.5 x 109/L) engraftment within 30 days upon transplantation | — |
Secondary
| Measure | Time frame |
|---|---|
|  Cumulative incidence of engraftment  Cumulative incidence of graft failure  Time to neutrophil recovery (> 0.5 x 109/L)  Time to lymphocyte (T-cells + subsets; B-cells; NK-cells) recovery  Time to platelet recovery ( > 20 x 109/L)  Time to red blood cell transfusion independence  Absolute number of CD3+CD4+, CD3+CD8+, CD19+ and CD3-CD16/56+ cells at 1,2, 3, 6, 12 and 24 months after UCBT  Incidence and grade of acute GVHD  Incidence of chronic GVHD  Incidence of infections  Incidence of CTC grade 3-4 adverse events  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. Patients still alive or lost to follow up are censored at the date they were last known to be alive | — |
Contacts
Public ContactJ.J. Cornelissen
Erasmus Medical Center, Daniel den Hoed Cancer Center, Department of Hematology, P.O. Box 5201
Outcome results
None listed