Clinical trial NCT03821727: SCT in Ph Positive Acute Lymphoblastic Leukemia
Official title: Outcome of Hematopoietic Stem Cell Transplantation in Philadelphia Positive Acute Lymphoblastic Leukemia in the Era of Tyrosine Kinase Inhibitors. A Registry-based Study of the Italian Blood and Marrow Transplantation Society (GITMO)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Philadelphia Positive Acute Lymphoblastic Leukemia
SCT
Lead sponsor: University Hospital, Udine, Italy
Sponsor class: OTHER
Collaborators: name: Gruppo Italiano Trapianto di Midollo Osseo
First posted: 2019-01-30
Enrollment: 441
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Observational model: CASE_ONLY
Time perspective: OTHER
Sampling method: PROBABILITY_SAMPLE
Study population: Data were extracted from the GITMO Registry (PROMISE Registry). All patients received a first allogeneic HSCT from any donor.

Brief summary: This study includes a registry-based, nationwide analysis to describe the clinical outcome of adult patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) who underwent an allogeneic hematopoietic stem cell transplantation (HSCT) after a Tyrosine Kinase Inhibitors (TKI)-based treatment.

Detailed description: This was a retrospective nationwide analysis based on registry data collected by GITMO. Inclusion criteria were: 1) diagnosis of Ph+ ALL; age ≥18 years at transplant; 2) patients receiving first allogeneic HSCT from any donor (HLA-identical sibling donor (MSD), unrelated donor (UD) or alternative donor (haploidentical or cord blood) between 2005 and 2016 in a GITMO center; 3) TKI-based treatment prior to HSCT; 4) patients with available pre-transplant MRD status, as well as complete clinical data and outcome. Data were extracted from the GITMO Registry (PROMISE Registry).

The endpoints of the study were: OS, progression-free survival (PFS), cumulative incidence of relapse (CIR), non-relapse mortality (NRM), cumulative incidence of extensive chronic graft versus host disease (cGVHD), rate of minimal residual disease (MRD) negativity and the rate of complete cytologic remission (CR) before and after transplant.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of Ph+ ALL; age ≥18 years at transplant.
* Patients receiving first allogeneic HSCT from any donor (HLA-identical sibling donor (MSD), unrelated donor (UD) or alternative donor (haploidentical or cord blood) between 2005 and 2016 in a GITMO center.
* TKI-based treatment prior to HSCT.
* Patients with available pre-transplant MRD status, as well as complete clinical data and outcome.

Exclusion Criteria:

* All cases without one or more of inclusion criteria.

Primary outcomes:
measure: Overall Survival (OS).
timeFrame: 5 years
Secondary outcomes:
measure: Rate of complete cytologic remission (CR) before and after transplant.
timeFrame: Baseline and 3 months after SCT
measure: Rate of minimal residual disease (MRD).
timeFrame: Baseline and 3 months post SCT
measure: Cumulative incidence of extensive chronic graft versus host disease (cGVHD).
timeFrame: 5 years
Other outcomes:
measure: Progression-free survival (PFS).
timeFrame: 5 years
measure: Cumulative incidence of relapse (CIR).
timeFrame: 5 years
measure: Non-relapse mortality (NRM).
timeFrame: 5 years
Locations:
city: Genova
country: Italy
facility: GITMO Centers and GITMO CLINICAL OFFICE-AOU-IRCSS San Martino-Ge
city: Udine
country: Italy
facility: University Hospital, Udine
Study officials:
affiliation: University of Udine
name: Renato Fanin, Professor
role: PRINCIPAL_INVESTIGATOR
References:
citation: Candoni A, Rambaldi A, Fanin R, Velardi A, Arcese W, Ciceri F, Lazzarotto D, Lussana F, Olivieri J, Grillo G, Parma M, Bruno B, Sora F, Bernasconi P, Saccardi R, Foa R, Sessa M, Bresciani P, Giglio F, Picardi A, Busca A, Sica S, Perruccio K, Zucchetti E, Diral E, Iori AP, Colombo AA, Tringali S, Santarone S, Irrera G, Mancini S, Zallio F, Malagola M, Albano F, Carella AM, Olivieri A, Tecchio C, Dominietto A, Vacca A, Sorasio R, Orciuolo E, Risitano AM, Leotta S, Cortelezzi A, Mammoliti S, Oldani E, Bonifazi F; GITMO. Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Adult Patients with Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia in the Era of Tyrosine Kinase Inhibitors: A Registry-Based Study of the Italian Blood and Marrow Transplantation Society (GITMO). Biol Blood Marrow Transplant. 2019 Dec;25(12):2388-2397. doi: 10.1016/j.bbmt.2019.07.037. Epub 2019 Aug 7.