high-risk hematological patients
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 b
Sponsors
Erasmus MC, Universitair Medisch Centrum Rotterdam
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: - Age 18-70 years 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 =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 | — |
Countries
Netherlands
Outcome results
None listed