Clinical trial NCT04801563: Allogeneic HSCT in MDS Patients Based on Risk According to R-IPSS
Official title: Allogeneic Transplantation in Patients With Myelodysplastic Syndrome Based on Risk According to Revised-International Prognostic Scoring System (R-IPSS)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: MDS and Allogeneic Stem Cell Transplantation
Lead sponsor: University Of Perugia
Sponsor class: OTHER
First posted: 2021-03-17
Enrollment: 80
Sex: ALL
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: overall survival
timeFrame: 4 years
Secondary outcomes:
Other outcomes:
Locations:
city: Perugia
country: Italy
facility: University of Perugia
state: PG
Study officials:
affiliation: University Of Perugia
name: Andrea Velardi, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Di Ianni M, Falzetti F, Carotti A, Terenzi A, Castellino F, Bonifacio E, Del Papa B, Zei T, Ostini RI, Cecchini D, Aloisi T, Perruccio K, Ruggeri L, Balucani C, Pierini A, Sportoletti P, Aristei C, Falini B, Reisner Y, Velardi A, Aversa F, Martelli MF. Tregs prevent GVHD and promote immune reconstitution in HLA-haploidentical transplantation. Blood. 2011 Apr 7;117(14):3921-8. doi: 10.1182/blood-2010-10-311894. Epub 2011 Feb 3.
citation: Martelli MF, Di Ianni M, Ruggeri L, Falzetti F, Carotti A, Terenzi A, Pierini A, Massei MS, Amico L, Urbani E, Del Papa B, Zei T, Iacucci Ostini R, Cecchini D, Tognellini R, Reisner Y, Aversa F, Falini B, Velardi A. HLA-haploidentical transplantation with regulatory and conventional T-cell adoptive immunotherapy prevents acute leukemia relapse. Blood. 2014 Jul 24;124(4):638-44. doi: 10.1182/blood-2014-03-564401. Epub 2014 Jun 12.