Haploidentical versus Cord Blood transplantation in pediatric AML. A retrospective outcome analysis on behalf of the pediatric subcommittee of GETH (Grupo Español de Trasplante Hematopoyético).

Fecha de publicación: 01/10/2024 Fecha Ahead of Print: 25/07/2024

Autores de IIS La Fe

Carolina Fuentes Socorro

Autor
José María Fernández Navarro

Autor

Participantes ajenos a IIS La Fe

Sisinni L
Monserrate GXA
Hurtado JMP
Panesso M
Molina B
Fuster JL
Verdu-Amoros J
Regueiro A
Palomo P
Beléndez C
Pascual A
Badell I
Mozo Y
Bueno D
Pérez-Martínez A
Vicent MG
de Heredia CD

Grupos

Investigación Clínica y Traslacional en Cáncer

Leader

José Gómez Codina

Abstract

Haploidentical stem cell transplantation (Haplo-SCT) and cord blood transplantation (CBT) are both effective alternative treatments in patients suffering from Acute Myeloid Leukemia (AML) and lacking a matched HLA donor. In the last years, many centers have abandoned CBT procedure mostly due to concern about poorer immune recovery compared to Haplo-SCT. We conducted a retrospective multicenter study comparing the outcomes using both alternative approaches in AML. A total of 122 transplants (86 Haplo-SCT and 36 CBT) from 12 Spanish centers were collected from 2007 to 2021. Median age at Hematopoietic stem cell transplantation (HSCT) was 7 years (0.4-20). Thirty-nine patients (31.9%) showed positive minimal residual disease (MRD) at HSCT and a previous HSCT was performed in 37 patients (30.3%). The median infused cellularity was 14.4?×?10^6/kg CD34+ cells (6-22.07) for haplo-SCT and 4.74?×?10^5/Kg CD34+ cells (0.8-9.4) for CBT. Median time to neutrophil engraftment was 14 days (7-44) for Haplo-SCT and 17 days (8-29) for CBT (p=0.03). The median time to platelet engraftment was 14 days (6-70) for Haplo-SCT and 43 days (10-151) for CBT (p<0.001). Graft rejection was observed in 13 Haplo-SCT (15%) and in 6 CBT (16%). The cumulative incidence of acute GvHD grade II-IV was 54% and 51% for Haplo-SCT and CBT respectively (p=0.5). The cumulative incidence of severe acute GvHD (grade III-IV) was 22% for Haplo-SCT and 25% for CBT (p= 0.9). There was a tendency to a higher risk of chronic GvHD in the Haplo-SCT group being the cumulative incidence of 30% for Haplo-SCT and 12 % for CBT (p= 0.09). The cumulative incidence of relapse was 28% and 20% for Haplo-SCT and CBT respectively (p= 0.6). We did not observe statistically significant differences in outcome measures between Haplo-SCT and CBT procedures: 5-year overall survival (OS) 64% vs 57% (p=0.5), 5-year disease-free survival (DFS) 58% vs 57% (p=0.8), GvHD-free and relapse-free survival (GFRFS) 41% vs 54% (p=0.3), and cumulative incidence of transplant-related mortality (TRM) 14% vs 15% (p=0.8), respectively. In the multivariate analysis MRD positivity and a disease status >CR1 at the time of HSCT were significantly associated with poorer outcomes (p<0.05). In conclusion, our study supports that both haploidentical and cord blood transplantation show comparable outcomes in pediatric AML patients. We obtained comparable survival rates, although CBT showed a trend to lower rates of chronic GvHD and higher GFRFS, demonstrating that it should still be considered a valuable option, particularly for pediatric patients.