5-Azacitidine in Low-risk Myelodysplastic Syndromes (MDSs)

• Status: Completed
• Trial end date: 2014-12-01
• Target enrollment: 0
• Participant gender: All

Summary

Azacitidine will be given at a dose of 75 mg/sqm (s.c) daily for 5 consecutive days every 28 days (every month) for a total of 8 courses to low risk MDSs according to IPSS scoring system. In fact, several studies produced high rates of trilineage responses, reduces the risk of progression to acute myeloid leukemia (AML) in high-risk MDS and improves the quality of life (QoL). The use of 5-Aza in the earlier phases of MDS could reduce the proliferative advantage of MDS clone and favour the regrowth of normal hematopoiesis.

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

Details

• Lead Sponsor: Università degli Studi di Brescia
• Collaborators: Cremona; Mantova; University of Bologna; University of Genova; University of Siena; University of Udine
• Treatments: Azacitidine

Criteria

Inclusion Criteria:

• Patients with low-risk (IPSS 0-INT1) MDS according to WHO classification, presenting one or more of the followings:

• Symptomatic anemia requiring RBC transfusion supportive therapy previously unresponsive to EPO or not expected to respond to EPO

• Thrombocytopenia requiring platelet transfusion with or without muco-cutaneous haemorrhagic syndrome

• Persistent (> 3 months) absolute neutrophil count less then 1,5 x 109/L, with or without infections, requiring or not myeloid growth factor therapy

• ≥ 18 years old.

• Life expectancy ≥ 3 months.

• ECOG performance Status Grade 0-2.

• Serum bilirubin levels ≤ 1.5 upper limit of the normal (ULN)

• Serum GOT and GPT levels ≤ 2x UNL.

• Creatinine levels ≤ 1.5x UNL.

• Negative serum β-human chorionic gonadotropin (β-HCG) pregnancy test 24 hours prior to beginning of therapy with 5-AZA, for fertile women.

• Written informed consent.

Exclusion Criteria:

• Patients with MDS according to WHO classification with INT-2 or high IPSS risk.

• Life expectancy < 3 months.

• ECOG performance Status Grade > 2.

• Serum bilirubin levels >1.5 upper limit of the normal (ULN).

• Serum GOT and GPT levels > 2 x UNL.

• Creatinine levels >1.5 x UNL.

• Pregnancy or breast feeding.

• Insulin-dependent diabetes and uncontrolled non insulin-dependent diabetes.

• Severe cardiac or pulmonary disease incompatible with the conduction of the protocol.

• Patient with a clear indication to receive long-term anticoagulant therapy.

• Other active hematologic or solid tumors.

• Severe CNS disease.

• Malignant hepatic tumors.

• Hypersensitivity to mannitol or azacitidine.

• No written informed consent.