A Study of Epoetin Beta Treatment in Anemic Participants With Myelodysplastic Syndrome (MDS)

Phase 4

Completed

Brief Summary: This is a Phase IV, prospective, multi-center, open-label study to assess the effectiveness and safety profile of epoetin beta (Recormon®) for treatment of symptomatic anemia in adult participants associated with low/intermediate-1-risk MDS. After screening, eligible participants will be treated with epoetin beta as recommended in the approved label and international guidelines for the use of epoetin in MDS participants and the dosage will be adjusted on the basis of erythroid response.

Recruitment & Eligibility

Inclusion Criteria

Adult participants with low or intermediate-1 risk MDS
No previous treatment with hematopoietic growth factors within 3 months prior to screening
Symptomatic anemia (hemoglobin <10 g/dL) as determined by investigator
Serum erythropoietin <500 milliunits/milliliter (mU/mL) within 14 days prior to the first dose of study treatment
Require no red blood cell transfusion or dependent on <4 units within 8 weeks prior to screening
Clinically stable for at least 1 month prior to entry into the study
For female participants of childbearing potential and male participants with partners of childbearing potential, agreement (by participants and/or partner) to use highly effective form(s) of contraception

Exclusion Criteria

Contraindications and/or known hypersensitivity to the active substance and/or any of the excipients of epoetin beta treatment
Poorly controlled hypertension as assessed by the investigator
History of Acute Myeloid Leukemia (AML) or high risk for AML
Administration of another investigational drug within 1 month before screening or planned during the study period
Previously documented evidence of Pure Red Cell Aplasia (PRCA)

Arm && Interventions

Group	Intervention	Description
Epoetin Beta	Epoetin beta	Participants will receive epoetin beta at an initial dose of 30,000 International Units (IU) per week administered subcutaneously (SC). Response will be firstly evaluated at Week 4 and the subsequent dose will be based on the response: if hemoglobin level reaches greater than or equal to (\>/=)12 grams per deciliter (g/dL) at any time, epoetin beta will be discontinued until hemoglobin levels are less than or equal to (\</=) 10 g/dL; if the hemoglobin level increases less than (\<) 1 g/dL from screening level and hemoglobin level ˂12 g/dL, a 60,000 IU per week epoetin beta will be administered SC until Week 12; if the hemoglobin level increases \>/=1 g/dL from screening level and hemoglobin level ˂12 g/dL, a 30,000 IU per week epoetin beta will be continued until Week 12.

Primary Outcome Measures

Name	Time	Method
Proportion of Participants Achieving Erythroid Response at Week 12 as Assessed by International Working Group (IWG) 2006 Response Criteria	Week 12	Erythroid response at Week 12 according to IWG 2006 criteria was defined as a hemoglobin (Hb) increase of \>/= 1.5 grams/deciliter (g/dL), and a reduction of units of red blood cell (RBC) transfusions by at least 4 transfusions/8 weeks compared with the pre-treatment transfusion number in the previous 8 weeks. Only RBC transfusions given for an Hb of \</= 9.0 g/dL pre-treatment were counted in the RBC transfusion response evaluation.

Secondary Outcome Measures

Name	Time	Method
Percentage of Participants With Neutrophil Response (in Participants With Pre-Treatment Neutrophil <1.0*10^9 Per Liter) at Week 12 as Assessed by IWG 2006 Response Criteria	Week 12	Neutrophil response according to IWG 2006 criteria was defined as at least 100% increase and an absolute increase of \>0.5x10\^9/L.
Percentage of Participants With Adverse Events	From signing of informed consent up to 4 weeks after last dose (up to 18 weeks)
Percentage of Participants With Platelet Response (in Participants With Pre-Treatment Platelets <100*10^9 Per Liter) at Week 12 as Assessed by IWG 2006 Response Criteria	Week 12	Platelet response according to IWG 2006 criteria was defined as an absolute increase of \>/= 30x10\^9/L for participants starting with \>20x10\^9/L platelets.

Locations (10): Siriraj Hospital; Division of Hematology, Department of Medicine
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Bangkok, Thailand
Thammasart Chalermprakiert Hospital, Thammasart Uni; Hematology
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Pathumthani, Thailand
Srinagarind Hospital, Khon Kaen Uni ; Dept of Medicine
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Khon Kaen, Thailand
King Chulalongkorn Memorial Hospital; Division of Hematology, Department of Medicine
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Bangkok, Thailand
Ramathibodi Hospital; Division of Hematology, Department of Medicine
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Bangkok, Thailand
Rajavithi Hospital; Medicine
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Bangkok, Thailand
Chiang Mai Uni Hospital; Division of Hematology,Dept of Medicine,Faculty of Medicine
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Chiang Mai, Thailand
Khonkean Regional Hospital; Medicine
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Khon Kean, Thailand
Sapprasitthiprasong Hospital
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Ubon Ratchathani, Thailand
Naresaun University hospital
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Phitsanulok, Thailand