Randomized study between thymoglobulin and posttransplant cyclophosphamide in patients with chronic myeloid neoplasms undergoing unrelated allogeneic stem cell transplantation
Cellular Therapy and Transplantation
Рандомизированное исследование тимоглобулина и посттрансплантационного циклофосфана при аллогенной неродственной трансплантации у взрослых с хроническими миелоидными неоплазиями
Randomized study between thymoglobulin and posttransplant cyclophosphamide in patients with chronic myeloid neoplasms undergoing unrelated allogeneic stem cell transplantation Summary Despite growing evidence on the use of posttransplantation cyclophosphamide (PTCY) in matched unrelated transplantation in acute leukemia patients, prospective evidence to support this type of prophylaxis in myelodysplastic syndrome (MDS), chronic myeloproliferative neoplasms and chronic myeloid leukemia (CML)
... leukemia (CML) patients is lacking. In this patient population we conducted a prospective randomized study of PTCY vs thymoglobulin for graft -versus-host disease (GvHD) prophylaxis in the setting of 10/10 HLA-matched unrelated transplantation (NCT02627573, clinicaltrials.gov). Th e strata for randomization was pretransplant assessment of mortality score. Th e study was terminated prematurely due to poor recruitment, but 33 patients were enrolled, 17 in the PTCY and 16 in the thymoglobulin group. Median follow up was 29 months. Th ere was no diff erence between groups in the incidence primary graft failure (12.50% vs 11.8%, p=0.9), acute GvHD grade II-IV (23% vs 6%, p=0.2), moderate and severe chronic GvHD (25% vs 23%, p=0.4) in the thymoglobulin and PTCY arms, respectively. However there was a signifi cantly higher overall survival (82% vs 30%, p=0.0126), event-free survival (61% vs 26%, p=0.0335), and GvHD-relapse free survival (61% vs 16%, p=0.0072) in the PTCY group due to reduced late infection-related mortality. No diff erences was observed in terms of toxicity. In conclusion, despite incomplete recruitment, the study created the basis for the use of PTCY in unrelated transplantations from fully matched unrelated donors in CML and MDS. Future studies are required to confi rm if there is a benefi t of PTCY-based prophylaxis over thymoglobulin. Keywords Posttransplant cyclophosphamide, myelodysplastic syndrome, chronic myeloid leukemia, chronic myeloproliferative neoplasm, matched unrelated donor. Citation: Morozova EV, Moiseev IS, Vlasova YY et al. Randomized study between thymoglobulin and posttransplant cyclophosphamide in patients with chronic myeloid neoplasms undergoing unrelated allogeneic stem cell transplantation. Cell Th er Transplant 2020; 9(1): 53-59.