Busulfan, fludarabine, and melphalan are effective conditioning for pediatric and young adult patients with myeloid malignancies underdoing matched sibling or alternative donor transplantation

被引:1
作者
Truscott, Laurel [1 ,2 ,3 ]
Pariury, Holly [1 ,2 ,3 ]
Hanmod, Santosh [1 ,4 ]
Davini, Monica [1 ,2 ,3 ]
de la Maza, Michelina [1 ,2 ,3 ]
Sapp, Lauren N. [1 ,3 ]
Staples, Kyleigh [1 ,3 ]
Proytcheva, Maria [1 ,2 ,3 ,5 ]
Katsanis, Emmanuel [1 ,2 ,3 ,6 ,7 ,8 ]
机构
[1] Univ Arizona, Dept Pediat, Tucson, AZ USA
[2] Univ Arizona, Canc Ctr, Tucson, AZ USA
[3] Banner Univ, Med Ctr, Tucson, AZ USA
[4] Banner Desert Med Ctr, Mesa, AZ USA
[5] Univ Arizona, Dept Pathol, Tucson, AZ USA
[6] Univ Arizona, Dept Immunobiol, Tucson, AZ USA
[7] Univ Arizona, Dept Med, Tucson, AZ USA
[8] Dept Pediat, 1501N Campbell Ave,POBox245073, Tucson, AZ 85724 USA
关键词
alternative donor; graft-versus-host disease; myeloablative conditioning; myeloid leukemia; HEMATOPOIETIC-CELL TRANSPLANTATION; MYELOABLATIVE BUSULFAN; PLUS CYCLOPHOSPHAMIDE; CLINICAL-TRIALS; EUROPEAN GROUP; WORKING PARTY; LEUKEMIA AML; CHILDREN; SURVIVAL; REGIMEN;
D O I
10.1002/pbc.30102
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
BackgroundAllogeneic hematopoietic cell transplantation (allo-HCT) remains a curative option for patients with high-risk myeloid malignancies. ProcedureWe present our 10-year experience (October 2012 to October 2021) of consecutive allo-HCT in patients with myeloid malignancies treated on the pediatric HCT service and conditioned with myeloablative targeted dose-busulfan (BU), fludarabine (FLU), and melphalan (MEL). Twenty-three children, adolescents, and young adult patients (CAYA) (median age 15.4 years) with acute myeloid leukemia (AML, n = 17), myelodysplastic syndrome (MDS, n = 4), or chronic myeloid leukemia (CML, n = 2) underwent allo-HCT post-BU-FLU-MEL. Four patients had treatment-related AML/MDS. Donor/stem cell source was matched sibling donor (MSD) PBSC (n = 7), matched unrelated donor (MUD) PBSC (n = 2), umbilical cord blood (UCB) (n = 3), or haploidentical-BMT (n = 11). Risk stratification was low (n = 2), intermediate (n = 15), high (n = 3), and very high risk (n = 1). The two patients with CML had failed tyrosine kinase inhibitor therapies. ResultsWith a median follow-up of 41.6 months, the relapse rate is only 4.5% with an overall survival (OS) 100%, progression-free survival (PFS) 95.5%, and graft-versus-host-free-relapse-free survival (GRFS) 67.8%. The donor source and the acute graft-versus-host disease (GvHD) prophylaxis regimen significantly impacted grade II-IV aGvHD 66.7% versus 19.2% (p = .039) and chronic graft-versus-host-disease (cGvHD) 66.7% versus 0% (p = .002) in the patients receiving MSD or MUD PBSC compared to haplo-BMT, respectively, resulting in improved GRFS in haplo-BMT, 83.3% compared to 40% matched donor peripheral blood stem cell transplant (PBSCT) (p = .025). ConclusionsOur results demonstrate that BU-FLU-MEL is efficacious conditioning for disease control in young patients with myeloid malignancies undergoing MSD or alternative donor allo-HCT, but in the setting of PBSC grafts with cyclosporine A-methotrexate (CSA-MTX) GvHD prophylaxis, it results in an unacceptably high incidence of GvHD.
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