Evaluation of Response to Biosimilar Erythropoietin Alfa Therapy in Anemic Patients With Myelodysplastic Syndrome
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Myelodysplastic Syndromes (MDS). Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Italy
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Valutazione Della Risposta Alla Terapia Con Eritropoietina Alfa Biosimilare Nei Pazienti Anemici Affetti da Sindrome Mielodisplastica
Overview
The primary objective of this study is to describe the response to treatment with biosimilar EPO alpha in MDS patients who had already been treated with "originator" EPO alpha and were responsive, and in patients who started treatment with biosimilar EPO alpha
Detailed description
To date, therapy with erythropoiesis-stimulating agents (ESAs) has been shown to be effective in the treatment of symptomatic anemia in patients with Myelodysplastic Syndrome ( MDS) at low to intermediate risk, with the percentage of responsive patients ranging from 15 to 63%, depending on the characteristics of the patients treated, and averaging 45%.
Significant improvement in survival and quality of life was observed in treatment-responsive patients compared with non-responsive patients.
Recently, biosimilar ESAs have been introduced into clinical practice, and in patients with renal failure anemia and anemia associated with antineoplastic chemotherapy, their efficacy and safety have been demonstrated.
However, data on the efficacy and safety of biosimilar ESAs in patients with MDS are scarce to date, and efficacy and safety data for the purpose of their approval have been mainly extrapolated from studies conducted in patients with anemia from renal failure and anemia from antineoplastic chemotherapy. In these conditions bone marrow erythropoiesis is reduced but not qualitatively impaired as in MDS.
These considerations may justify a retrospective study examining the efficacy and safety of biosimilar EPO alpha in a real-life setting in anemic MDS patients.
Primary outcome measures
- Response and maintenance of ORR [Time frame: 6 months from the start of the study]
Secondary outcome measures (1)
- A measure of the frequency of major clinical outcomes in two groups of patients who had different modes of treatment administration with biosimilar EPO alpha [Time frame: 6 months from the start of the study]
Eligibility criteria
Inclusion criteria
MDS patients with symptomatic anemia (pre-treatment hemoglobin < 10g/dL) who started treatment with biosimilar EPO alpha, diagnosed according to the WHO 2016 classification, and characterized by very low, low, or intermediate IPSS-R risk and a pre-treatment serum EPO level < 500 U/L, who started treatment with biosimilar EPO alpha at the U. O. of Hematology of IRCCS AOUBO during the period from 01/06/2018 to 31/12/2021.
- Age ≥ 18 years at the time of enrollment
- Acquisition of informed consent to study participation and data processing
Exclusion criteria
- \- Presence of other possible contributory causes of anemia (e.g., anemia from chronic inflammatory disease, hemolysis, hemorrhage)
- Poor compliance with treatment
- Very impaired general clinical condition (ECOG performance status > 3)
- Concomitant treatment with antineoplastic cytotoxic drugs
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Study design
- Observational model
- Cohort
Study locations
Italy · 1 center
- IRCCS Azienda Ospedaliera -Universitaria di Bologna — Bologna
Identifiers
NCT: NCT06764511 · EPO BIOSIM