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NCT Number: NCT06764511

Evaluation of Response to Biosimilar Erythropoietin Alfa Therapy in Anemic Patients With Myelodysplastic Syndrome

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

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Key information

Conditions

Myelodysplastic Syndromes (MDS) Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Myelodysplastic Syndromes

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

IRCCS Azienda Ospedaliera -Universitaria di Bologna

Bologna, 40138, Italy

Location status: Recruiting

About this study

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.

Who can participate

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. Response and maintenance of ORR

    Time frame: 6 months from the start of the study

    response to treatment (ORR) (assessed according to the response criteria established by the International Working Group (23) in patients who initiated treatment with biosimilar EPO alfa

    • maintenance of ORR (assessed according to the response criteria established by the International Working Group (23) from the date of biosimilar drug initiation), in patients previously on treatment and responsive to "originator" EPO alfa who were subsequently treated with biosimilar EPO alfa

Secondary outcomes

  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

    • DOR to EPO alpha: to biosimilar drug only in patients who started treatment with biosimilar EPO alpha; to "originator" drug + biosimilar in patients already on treatment and responsive to "originator" EPO alpha who were subsequently treated with biosimilar EPO alpha ; 2) incidence of adverse events in year/person (in both patient groups) according to NCI-CTCAE v.5.0 ; 3) number of patients who discontinued treatment with the biosimilar drug due to intolerance, loss of response, disease progression (DP), evolution into AML, or death (both groups) ; 4) number of patients who resumed the originator drug due to intolerance or failure to respond to the biosimilar drug ; 5) ORR and incidence of adverse events of patients who resumed treatment with "originator" EPO alfa after discontinuation of the biosimilar drug ; 6) incidence of evolution into AML in year/person (both groups) ; 7) OS and causes of death (both groups)

Study contacts

Contact information is provided by the study sponsor or research team.

Stefania Paolini, MD

CONTACT

[email protected]

+390512143680

Sponsors and collaborators

Lead sponsor

IRCCS Azienda Ospedaliero-Universitaria di Bologna

Other

Registry information

Official study title

Valutazione Della Risposta Alla Terapia Con Eritropoietina Alfa Biosimilare Nei Pazienti Anemici Affetti da Sindrome Mielodisplastica

Important dates

Study start
2024
Primary completion
2025
Study completion
2025
First posted
Jan 8, 2025
Registry last updated
Jan 8, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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