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Alemtuzumab and Low-Dose Cyclosporine-A as Alternative Immunosuppressive Treatment for Severe Aplastic Anemia (SAA) and Single-Lineage Aplastic Patients

Phase 2
18 Years
Open (Enrolling)
Nonmalignant Neoplasm

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Trial Information

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
marrow failure.

- 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
myeloid) development.

- Determine the incidence of clonal evolution (i.e., karyotypic abnormalities or
secondary myelodysplasia/leukemia).

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

Inclusion Criteria


- Diagnosis of 1 of the following:

- Severe or very severe aplastic anemia, as defined by the following criteria:

- Meets ≥ 2 of the following criteria:

- Absolute neutrophil count < 0.5 x 10^9/L (severe) or < 0.2 x 10^9/L
(very severe)

- Platelet count < 20 x 10^9/L

- Reticulocyte count < 20 x 10^9/L

- Hypocellular bone marrow (< 30% cellularity) without evidence of fibrosis
or malignant cells

- Single lineage acquired marrow failure (e.g., pure red cell aplasia,
agranulocytosis, amegakaryocytic thrombocytopenia)

- Paroxysmal nocturnal hemoglobinuria clone allowed

- Failed first-line therapy with antithymocyte globulin (ATG) and cyclosporine OR not
eligible for ATG-based studies

- Failure is defined as lack of hematological response, requirement for chronic
immunosuppressive treatment to sustain response, or relapse

- Not eligible for a low-risk stem cell transplantation

- No evidence of risky myelodysplastic syndromes (i.e., IPSS 3-4), as defined by the
presence of marrow blast excess or karyotypic abnormalities, or other primitive
marrow disease

- No history of constitutional aplastic anemia (e.g., Fanconi anemia or dyskeratosis


- WHO performance status 0-2

- Not pregnant or nursing

- No active malignant tumor within the past 5 years

- Transaminases ≤ 3 times upper limit of normal (ULN)

- Albumin ≥ 1.5 g/L

- Creatinine ≤ 3 times ULN

- No CMV viremia, as defined by positive PCR or pp65 test

- No cardiac failure (i.e., ejection fraction < 35%)

- No other concurrent life-threatening disease (including HIV infection)


- No prior allogeneic stem cell transplantation

- At least 2 weeks since prior cyclosporine or filgrastim (G-CSF)

Type of Study:


Study Design:

Allocation: Non-Randomized, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Safety, as defined by occurrence of adverse effects

Safety Issue:


Principal Investigator

Bruno Rotoli, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Federico II University



Study ID:




Start Date:

June 2006

Completion Date:

Related Keywords:

  • Nonmalignant Neoplasm
  • aplastic anemia
  • Anemia
  • Anemia, Aplastic
  • Neoplasms