Absence of an HLA Identical Donor, Acquired Aplastic Anemia, Hereditary Diseases, Relapse
Conditions
Keywords
severe aplastic anemia, cord blood transplantation
Brief summary
This is a Prospective Phase II Study to evaluate Cord Blood Transplantation in Inherited or Acquired Severe Aplastic Anemia Refractory or in Relapse after Immunosuppressive Therapy in the absence of an HLA identical donor;
Detailed description
This is a Prospective Phase II Study to evaluate Cord Blood Transplantation in Inherited or Acquired Severe Aplastic Anemia Refractory or in Relapse after Immunosuppressive Therapy in the absence of an Human Leucocyte Antigen (HLA) identical donor; 26 patients will be recruited, according to a one step Fleming design
Interventions
PROCEDURECord Blood Transplantation
One or 2 cord blood units containing alone or both together more than 4x107 frozen nucleated cells/Kg with no more than 2 out of 6 HLA mismatches between them and with the patients for acquired Severe Aplastic Anemia
Sponsors
Assistance Publique - Hôpitaux de Paris
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 55 Years
Healthy volunteers
No
Inclusion criteria
* Age: 3-55 years old * inherited aplastic anemia (with severe aplastic anemia criteria) or acquired aplastic anemia in relapse or treatment failure after immunosuppressive therapy without clonal evolution * Karnovsky Index \>= 60% * Informed consent
Exclusion criteria
* HLA identical donor
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Overall survival | 1 year |
Secondary
| Measure | Time frame |
|---|---|
| Infectious episodes | 1 year |
| treatment related mortality | 100 days |
| Graft versus host disease | 2 years |
| adverse events | 1 year |
| karnofsky scale | 1 year |
| overall survival | 5 years |
Countries
France
Outcome results
None listed