Alemtuzumab and Low-Dose Cyclosporine-A as Alternative Immunosuppressive Treatment for Severe Aplastic Anemia (SAA) and Single-Lineage Aplastic Patients
- Determine the safety of alemtuzumab and low-dose cyclosporine, as defined by occurrence
of adverse effects, in patients with severe aplastic anemia or single lineage acquired
- Determine the efficacy of this regimen, in terms of overall survival, hematological
response (partial and complete response, including time to response) and failure-free
survival (failure is defined as no response, chronic treatment-maintained response, or
relapse), in these patients.
- Evaluate the incidence of adverse effects after treatment.
- Evaluate the long-term safety of alemtuzumab treatment.
- Determine the time to achieve a complete hematological response.
- Determine the proportion of patients maintaining hematological response free of any
- Determine the incidence of relapse in responding patients.
- Determine the incidence of severe infections.
- Determine the requirement for IV antibiotics and antifungal therapy.
- Determine the requirement for red cell and platelet transfusion.
- Determine the incidence of CMV reactivation.
- Determine the kinetics of immune reconstitution.
- Determine the incidence of paroxysmal nocturnal hemoglobinuria clone (lymphoid or
- Determine the incidence of clonal evolution (i.e., karyotypic abnormalities or
OUTLINE: Patients receive alemtuzumab subcutaneously on days 1-5*. Patients also receive
oral cyclosporine beginning on day 7 and continuing for ≥ 180 days, followed by a taper
according to clinical condition.
NOTE: *Patients with single lineage aquired marrow failure receive alemtuzumab on days 1-4.
After completion of study therapy, patients will be followed up every 3 months for up to 2
Allocation: Non-Randomized, Masking: Open Label, Primary Purpose: Treatment
Safety, as defined by occurrence of adverse effects
Bruno Rotoli, MD
Federico II University