lower risk myelodysplastic syndrome
Conditions
Interventions
In the induction phase, there is no allocation. All the patients receive azacitidine 75mg/m2 for five consecutive days, and 21 days off. Repeat this treatment for six cycles. In the maintenance phas
Sponsors
National Research Group on Idiopathic Bone Marrow Failure Syndromes
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Cases that are diagnosed as MDS according to WHO 2008 criteria. Include therapy related MDS, but does not include MDS/MPN. 2) Cases in low/Int-1 according to IPSS criteria at both diagnostic and enrollment time. 3) Cases with at least one lineage cytopenia. Concretely, at least one of the following criteria are met. a) Hb<10g/dL and required RBC transfusions in the past 3 months. b) Plt<50000/mm3 or with bleeding tendency. c) Neu<1000/mm3 or with increased susceptibility to infection that require antibiotics. 4) Cases with expected life expectancy>1 year. 5) Aged 20 or older. 6) ECOG Performance Status 0~2 7) Cases with total bilirubin and serum creatinine level below 1.5xULN. 8) Cases with AST(GOT) or ALT(GPT) level below 3.0xULN. 9) Cases who can visit hospitals at the pre-defined schedule. 10) Cases with written informed consent.
Exclusion criteria
Exclusion criteria: 1) Cases with scheduled hematopoietic stem cell transplantation. We do not exclude patients who are judged as transplant candidate after entry of this study because of change of medical conditions. 2) Cases who received hematopoietic stem cell transplantation. 3) Cases who had already been enrolled into other clinical trials for MDS. 4) Cases with pregnancy, possibility of pregnancy, in breast-feeding, or with plant to bear a child 5) Cases with hypersensitivity to azacitidine. 6) Cases with other cancers than MDS that is invasive within 5 years. 7) Cases with complicating diseases that are severe or uncontrolled. 8) Cases with psychiatric diseases or psychiatric symptoms that preclude adequate entry to the study. 9) Cases with cognitive disorders. 10) Patients who are receiving successful treatment with other modalities, or who are expected to achieve better response with other treatments (such as with 5q- syndrome). 11) Cases that are considered to be inadequate to enroll this study by the attending physicians.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| This study has two primary endpoints for induction phase and maintenance phase, respectively. Induction phase: the ratio of cases in which hematological improvement is achieved with 6 course of azacitidine therapy. Maintenance phase: the ratio of patients who maintained hematological effectiveness achieved in the induction phase after one year with or without azacitidine maintenance therapy. | — |
Secondary
| Measure | Time frame |
|---|---|
| Induction phase: Achievement ratio of hematological improvement for each of hematological series. Cytogenetic response ratio. Transfusion dependence ratio at the end of induction phase. Sustainability of induction therapy. Incidence of Grade 3 side effects or higher. Incidence of infection that need intravenous antibiotics. Overall survival ratio. Progression free survival ratio to AML. Maintenance phase: the ratio of patients who maintained hematological effectiveness after two years. Ratio of AML development. Ratio of patients who received hematopoietic stem cell transplantation. Incidence of Grade 3 side effects or higher. Incidence of infection that need intravenous antibiotics. Overall survival ratio. Progression free survival ratio to AML. | — |
Countries
Japan
Contacts
Public ContactYasuhito Nannya
The University of Tokyo Hospital Department of Hematology & Oncology
Outcome results
None listed