A Study of NeoRecormon (Epoetin Beta), CellCept (Mycophenolate Mofetil) and Prednisone in Patients With Low or Intermediate Myelodysplastic Syndromes.

Phase 2

Completed

Interventions: Drug: Mycophenolate mofetil
Drug: Prednisone
Drug: Erythropoietin Beta

Brief Summary: This single arm study will evaluate the efficacy and safety of a combination of NeoRecormon, CellCept and prednisone in patients with low or moderate risk myelodysplastic syndromes (MDS). In the first phase of the study, patients will receive CellCept (1g p.o. twice daily) plus prednisone. After 3 months, if patients have not responded to treatment, NeoRecormon (30000 IU/week, s.c.) will be added to the treatment regimen. If there is no response to NeoRecormon after 6 weeks, the dose will be increased to 60000 IU/week. The anticipated time on study treatment is 3-12 months, and the target sample size is \<100 individuals.

Recruitment & Eligibility

Inclusion Criteria

adult patients, >=18 years of age;
diagnosis of MDS, according to International Prognostic Scoring System (IPSS) criteria;
low or intermediate risk, who are not candidates for treatment with growth factors, or who have not responded to these treatments.

Exclusion Criteria

previous treatment with CellCept, or any erythropoietin-stimulating drug;
diagnosis of proliferative chronic myelomonocytic leukemia;
prior or concomitant malignancies other than MDS, with the exception of basocellular, spinocellular or adequately treated in situ cervical cancer, in the past 3 years;
biological antitumor and myelosuppressive treatment within 28 days before start of study;
bone marrow precursor cell transplantation previous to study.

Arm && Interventions

Group	Intervention	Description
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta	Erythropoietin Beta	Mycophenolate mofetil (MMF) 1 gm twice daily orally and prednisone 10 mg/day orally until the end of the study. Recombinant human erythropoietin beta 30,000 IU/week, subcutaneously for 6 weeks was added in case of no significant response at Week 12.
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta	Mycophenolate mofetil	Mycophenolate mofetil (MMF) 1 gm twice daily orally and prednisone 10 mg/day orally until the end of the study. Recombinant human erythropoietin beta 30,000 IU/week, subcutaneously for 6 weeks was added in case of no significant response at Week 12.
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta	Prednisone	Mycophenolate mofetil (MMF) 1 gm twice daily orally and prednisone 10 mg/day orally until the end of the study. Recombinant human erythropoietin beta 30,000 IU/week, subcutaneously for 6 weeks was added in case of no significant response at Week 12.

Primary Outcome Measures

Name	Time	Method
Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement	Up to approximately 2 years	International Working Group (IWG) criteria for hematological improvement was defined as having hemoglobin (Hgb) \<11 g/dL (pretreatment) and an increase in Hgb ≥1.5 g/dL after ≥8 weeks of treatment.
Mean Number of Blood Transfusions Per Visit	Up to approximately 2 years

Secondary Outcome Measures

Name	Time	Method
Percentage of Participants With at Least One Adverse Event (AE)	Up to approximately 2 years	An AE was considered any unfavorable and unintended sign, symptom, or disease associated with the use of the study drug, whether or not considered related to the study drug. Preexisting conditions that worsened during the study and laboratory or clinical tests that resulted in a change in treatment or discontinuation from study drug were reported as adverse events.