Allogeneic HSCT in MDS Patients Based on Risk According to R-IPSS

University Of Perugia

Status: Enrolling

Conditions: MDS and Allogeneic Stem Cell Transplantation

Treatments: Other: MDS and AlloHSCT

Study type: Interventional

Funder types: Other

Identifiers

NCT04801563

03/19

Details and patient eligibility

About

An "intention-to-treat" study to evaluate the impact of allogeneic HSCT with Total Marrow and Lymphoid irradiation (TMLI), followed by Treg/Tcon adoptive immunotherapy, on overall survival in patients affected by Myelodysplastic Syndrome (MDS), according to IPSS-R.

Full description

MDS patients with Intermediate, High, or Very-High R-IPSS risk will be enrolled. Allogeneic stem cell transplantation with regulatory and conventional T cell adoptive immunotherapy will be proposed to eligible High and Very-High risk MDS patients. Intermediate risk MDS patients will be assessed for risk modification every six months and moved to transplant in case of increased risk. Intermediate risk MDS patients will be also evaluated for the presence of molecular alterations (TP53, ASXL1, RUNX1). Overall survival of the entire cohort will be assessed as primary endpoint. Incidence of Treatment Related Mortality, acute Graft versus Host Disease, chronic Graft versus Host Disease, Relapse will be also assessed.

Enrollment: 80 estimated patients

Sex: All

Ages: Under 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients affected by Myelodysplastic Syndrome with IPSS-R INT, HIGH or VERY HIGH;
• Age <71 years;
• Signature of the informed consent.

Exclusion criteria

• Patients affected by Myelodysplastic Syndrome with IPSS-R VERY LOW or LOW;
• Patients affected by Myelodysplastic Syndrome/Myeloproliferative Neoplasm;
• Age >70 years;
• No signature of the informed consent

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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80 participants in 1 patient group

SINGLE ARM

Other group

Treatment:

Other: MDS and AlloHSCT