ALEMTUZUMAB AND LOW-DOSE CYCLOSPORINE-A AS ALTERNATIVE IMMUNOSUPPRESSIVE TREATMENT FOR SEVERE APLASTIC ANEMIA (SAA) AND SINGLE-LINEAGE APLASTIC PATIENTS. - Alesaa

Conditions: Aplastic anemia and single-lineage bone marrow failure
MedDRA version: 13.1Level: PTClassification code 10002965Term: Aplasia pure red cellSystem Organ Class: 10005329 - Blood and lymphatic system disorders
MedDRA version: 13.1Level: LLTClassification code 10047350Term: Very few granulocyte precursorsSystem Organ Class: 10005329 - Blood and lymphatic system disorders
MedDRA version: 13.1Level: PTClassification code 10002967Term: Aplastic anaemiaSystem Organ Class: 10005329 - Blood and lymphatic system disorders
MedDRA version: 13.1Level: PTClassification code 10001507Term: AgranulocytosisSystem Organ Class: 10005329 - Blood and lymphatic system disorders

Registration Number: EUCTR2008-001151-22-IT

Lead Sponsor: IVERSITA` DEGLI STUDI DI NAPOLI FEDERICO II

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 50

Inclusion Criteria

a.Diagnosis of severe or very severe aplastic anemia,22 defined by:
At least two of the following:
Absolute neutrophil counts <0.5 x 109/L (severe) or <0.2 x 109/L (very severe)
Platelet counts <20 x 109/L
Reticulocyte counts <20 x 109/L
Hypocellular bone marrow (<30% cellularity), without evidences of fibrosis or malignant cells
Or
Diagnosis of single lineage acquired marrow failure, such as:
Pure Red Cell Aplasia (PRCA
Agranucytosis
Amegakaryocytic thrombocytopenia

b.Failure of first line therapy with ATG+CsA, or lack of eligibily for ATG-based studies. Failure is defined as follows:
-lack of hematological response
-need of chronic IS treatment to sustain response
-relapse
c.Age ≥18 years
d.Written informed consent
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

a.Eligibility for a low-risk SCT procedure
b.Evidence of a risky myelodysplastic syndrome (IPSS 3-4), defined by the presence of marrow blast excess or karyotypic abnormalities, or other primitive marrow disease
c.History of constitutional aplastic anemia (i.e. Fanconi Anemia or Dyskeratosis Congenita)
d.History of malignant tumors with active disease within 5 years from enrollment
e.Previous history of allogeneic stem cell transplantation
f.Treatment with cyclosporin A <2 weeks before enrollment
g.Treatment with G-CSF <2 weeks before enrollment
h.CMV viremia, as defined by positive PCR or pp65 test
i.WHO performance status ≥3
j.Pregnant or breast feeding patients
k.Patients with hepatic (transaminases >UNLx3 or albumin <1,5 g/L), renal (creatinine >UNLx3) or cardiac failure (ejection fraction <35%), or any other life-threatening concurrent disease (including HIV infection).