Preparative regimens

A randomized trial that compared busulfan plus fludarabine with busulfan plus cyclophosphamide as a preparative regimen for AML in first CR demonstrated the following results:[89]

A randomized trial that compared busulfan plus fludarabine with busulfan plus cyclophosphamide as a preparative regimen for AML in first CR demonstrated the following results:[89]

The busulfan plus cyclophosphamide regimen was associated with less toxicity and produced a comparable DFS and OS.

A large prospective CIBMTR cohort study included children and adults with AML, myelodysplastic neoplasms (MDS), and chronic myeloid leukemia (CML).[90]

A large prospective CIBMTR cohort study included children and adults with AML, myelodysplastic neoplasms (MDS), and chronic myeloid leukemia (CML).[90]

Patients with early-stage disease (chronic-phase CML, first CR AML, and MDS-refractory anemia) had superior survival rates with busulfan-based regimens, compared with TBI.

A CIBMTR study of 624 children with de novo AML who underwent transplant between 2008 and 2016 and received either a TBI-based regimen (n = 199) or non-TBI–containing regimen (n = 425) demonstrated the following results:[91]

A CIBMTR study of 624 children with de novo AML who underwent transplant between 2008 and 2016 and received either a TBI-based regimen (n = 199) or non-TBI–containing regimen (n = 425) demonstrated the following results:[91]

TBI recipients had a higher nonrelapse mortality (P < .0001) with lower relapse (P < .0001), culminating in equivalent LFS and OS rates.

A CIBMTR study of 624 children with de novo AML who underwent transplant between 2008 and 2016 and received either a TBI-based regimen (n = 199) or non-TBI–containing regimen (n = 425) demonstrated the following results:[91]

TBI recipients experienced more grades 2 to 3 acute graft-versus-host disease (GVHD) (56% vs. 27%; P < .0001) but had equivalent chronic GVHD incidence.

A CIBMTR study of 624 children with de novo AML who underwent transplant between 2008 and 2016 and received either a TBI-based regimen (n = 199) or non-TBI–containing regimen (n = 425) demonstrated the following results:[91]

TBI recipient survivors had a greater incidence of gonadal or growth deficiency (24% vs. 8%; P < .0001), but there were no differences in pulmonary, cardiac, or renal impairment.

A CIBMTR study included 550 pediatric patients with AML who underwent HSCT between 2008 and 2016. The study compared the outcomes of those in first or second CR who had been CNS-positive versus CNS-negative and received TBI-based or non–TBI-containing preparative regimens.[87]

A CIBMTR study included 550 pediatric patients with AML who underwent HSCT between 2008 and 2016. The study compared the outcomes of those in first or second CR who had been CNS-positive versus CNS-negative and received TBI-based or non–TBI-containing preparative regimens.[87]

CNS involvement was more prevalent in patients aged 0 to 3 years, patients who were in second CR, and those receiving mismatched unrelated donor or umbilical cord blood transplants.

A CIBMTR study included 550 pediatric patients with AML who underwent HSCT between 2008 and 2016. The study compared the outcomes of those in first or second CR who had been CNS-positive versus CNS-negative and received TBI-based or non–TBI-containing preparative regimens.[87]

Patients with CNS-positive disease had a lower relapse rate (HR, 0.50; 95% CI, 0.33–0.76) than patients with CNS-negative disease, with comparable DFS and OS in the two cohorts.

A CIBMTR study included 550 pediatric patients with AML who underwent HSCT between 2008 and 2016. The study compared the outcomes of those in first or second CR who had been CNS-positive versus CNS-negative and received TBI-based or non–TBI-containing preparative regimens.[87]

Patients who received TBI had an increased risk of grades 2 to 4 acute GVHD and higher rates of bloodstream infection and endocrine dysfunction.

A CIBMTR study included 550 pediatric patients with AML who underwent HSCT between 2008 and 2016. The study compared the outcomes of those in first or second CR who had been CNS-positive versus CNS-negative and received TBI-based or non–TBI-containing preparative regimens.[87]

TBI use within the CNS-positive AML cohort was associated with a lower relapse rate, but these patients had increased risks of nonrelapse mortality and a trend toward higher grades 3 to 4 acute GVHD.

A CIBMTR study included 550 pediatric patients with AML who underwent HSCT between 2008 and 2016. The study compared the outcomes of those in first or second CR who had been CNS-positive versus CNS-negative and received TBI-based or non–TBI-containing preparative regimens.[87]

TBI regimens did not confer an advantage in DFS or OS, compared with non-TBI regimens, regardless of the patient's CNS-disease status.