Efficacy and Safety of Luspatercept: a Study by Fondazione Italiana Sindromi Mielodisplastiche

Summary

Myelodysplastic syndromes (MDS) are a group of malignancies characterized by reduced differentiation and increased apoptosis of hematopoietic progenitor cells, leading to ineffective hematopoiesis. Treatment of MDS varies according to prognosis. Patients with low IPSS-R risk have a low probability of progression to acute myeloid leukemia (AML) and the treatment is aimed at controlling cytopenia and improving quality of life (QOL). Anemia is the most common disease feature, occurring in 80%-85% of low-risk patients, 40% of whom eventually become RBC transfusion-dependent (TD).

Luspatercept is a recombinant fusion protein that selectively binds to ligands belonging to the transforming growth factor-beta (TGF-beta) superfamily. Luspatercept binds to GDF11, GDF8, activin B, and other ligands. This binding leads to inhibition of Smad2/3 signaling, which is abnormally high in disease models of ineffective erythropoiesis such as MDS, resulting in erythroid maturation and differentiation.

Luspatercept is now approved for the treatment of adult patients with TD anemia due to very low-, low-, and intermediate-risk MDS with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.

FISiM (Fondazione Italiana Sindromi Mielodidplastiche) promotes a multicenter, retrospective observational study to collect information on the efficacy and safety of luspatercept in a real world Italian population of adult patients with transfusion-dependent anemia due to very low- and intermediate-risk MDS with ring sideroblasts

Conditions

• Myeloid Dysplasia

Interventions

DRUG

Luspatercept

Luspatercept treatment in adults with transfusion-dependent anaemia due to MDS

Sponsors & Collaborators

• Istituto Clinico Humanitas

  collaborator OTHER

• Fondazione Italiana Sindromi Mielodisplastiche-ETS

  lead OTHER

Principal Investigators

• Matteo Della Porta, MD · Istituto Clinico Humanitas

• Valeria Santini, MD · Università degli studi di Firenze - AOU Careggi

• Lorenza Borin, MD · ASST-MONZA

• Daniela Cilloni, MD · Aziena Ospedaliera Mauriziano - Torino

• Bruno Fattizzo, MD · Ospedale Policlinico di Milano

• Pellegrino Musto, MD · Policlinico di Bari

• Esther Oliva, MD · Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli di Reggio Calabria

• Marta Riva, MD · ASST Grande Ospedale Metropolitano Niguarda

• Prassede Salutari, MD · Presidio Ospedaliero Pescara

• Maria Teresa Voso, MD · Università Cattolica del Sacro Cuore - Roma

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2022-01-01

Primary Completion

2022-12-31

Completion

2023-01-31

Countries

• Italy

Study Locations