Uniform graft-versus-host disease prophylaxis with posttransplant cyclophosphamide, sirolimus, and mycophenolate mofetil following hematopoietic stem cell transplantation from haploidentical, matched sibling and unrelated donors

Autores de IIS La Fe

• Juan Bautista Montoro Gómez

  Autor

• José Luis Piñana Sánchez

  Autor

• Jose Ignacio Lorenzo Herrero

  Autor

• Manuel Nuno Direito De Morais Guerreiro

  Autor

• Aitana Balaguer Roselló

  Autor

• Guillermo Sanz Santillana

  Autor

• Carlos Carretero Marquez

  Autor

• Miguel Ángel Sanz Alonso

  Autor

• Jaime Sanz Caballer

  Autor

Participantes ajenos a IIS La Fe

• Hernández-Boluda J.C.
• Hernani R.
• Pérez A.
• Albert E.
• Navarro D.
• Solano C.

Grupos

• Hematología y Hemoterapia

Abstract

Following the success of posttransplant cyclophosphamide (PT-CY) as graft-versus-host disease (GVHD) prophylaxis in haploidentical transplantation, this prevention strategy has progressively been used for allogeneic hematopoietic stem cell transplantation (allo-HSCT) from HLA-matched sibling (MSD) and unrelated donor (MUD). We have introduced PT-CY plus sirolimus and micophenolate mofetil (PT-CY-Sir-MMF) as GVHD prophylaxis in allo-HSCT, irrespective of donor type. This study reports on the safety and efficacy of PT-CY-Sir-MMF in 158 consecutive allo-HSCT from MSD (n = 52), MUD (n = 64), and haploidentical (n = 42) donor. Median age was 53 years and 66% had acute leukemia or myelodysplastic syndrome. Cumulative incidences of acute GHVD grade II–IV, III–IV and moderate to severe cGVHD were 27%, 9% and 27%, respectively. The incidence of hepatic sinusoidal obstruction syndrome was 9.5%. The 1-year cumulative incidence of non-relapse mortality, relapse and event-free survival were 14%, 12% and 75%, respectively. Compared with MSD and MUD, haploidentical transplantation had a higher incidence of CMV DNAemia requiring therapy (34% vs 35% and 52%, respectively, p = 0.04) and was a risk factor for grade III–IV acute GVHD (RR 2.8, p = 0.05). Our study shows that PT-CY-Sir-MMF is not only feasible and effective in preventing GVHD after haploidentical HSCT, but also in allo-HSCT from MSD and MUD. © 2020, The Author(s), under exclusive licence to Springer Nature Limited.

Keywords

• cyclophosphamide; mycophenolate mofetil; rapamycin; virus DNA; acute leukemia; adult; age; Article; cancer recurrence; child; cohort analysis; Cytomegalovirus; disease severity; drug efficacy; drug safety; event free survival; graft versus host reaction; haploidentical donor; hematopoietic stem cell transplantation; HLA matching; human; incidence; liver venoocclusive disease; major clinical study; male; matched unrelated donor; mortality; myelodysplastic syndrome; observational study; postoperative care; priority journal; prophylaxis; risk factor; sibling donor; viremia