Alemtuzumab and Low-Dose Cyclosporine in Treating Patients With Severe Aplastic Anemia or Acquired Marrow Failure

• Status: Unknown status
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

Summary

RATIONALE: Immunosuppressive therapies, such as alemtuzumab and cyclosporine, may improve bone marrow function and increase blood cell counts. Giving alemtuzumab together with cyclosporine may be an effective treatment for severe aplastic anemia or acquired marrow failure.

PURPOSE: This phase II trial is studying the side effects of giving alemtuzumab together with cyclosporine and to see how well it works in treating patients with severe aplastic anemia or acquired marrow failure.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Federico II University
• Treatments: Alemtuzumab; Cyclosporine; Cyclosporins

Criteria

DISEASE CHARACTERISTICS:

• 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 congenita)

PATIENT CHARACTERISTICS:

• 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)

PRIOR CONCURRENT THERAPY:

• No prior allogeneic stem cell transplantation

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