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Randomized Controlled Trial
. 2021 Jun;27(6):483.e1-483.e6.
doi: 10.1016/j.jtct.2021.02.031. Epub 2021 Feb 26.

Myeloablative versus Reduced-Intensity Conditioning for Hematopoietic Cell Transplantation in Acute Myelogenous Leukemia and Myelodysplastic Syndromes-Long-Term Follow-Up of the BMT CTN 0901 Clinical Trial

Bart L Scott  1 , Marcelo C Pasquini  2 , Mingwei Fei  2 , Raphael Fraser  2 , Juan Wu  3 , Steve M Devine  4 , David L Porter  5 , Richard T Maziarz  6 , Erica Warlick  7 , Hugo F Fernandez  8 , Robert J Soiffer  9 , Edwin Alyea  10 , Mehdi Hamadani  2 , Asad Bashey  11 , Sergio Giralt  12 , Nancy L Geller  13 , Eric Leifer  13 , Christopher S Hourigan  13 , Gege Gui  13 , Adam Mendizabal  3 , Mary M Horowitz  2 , H Joachim Deeg  14 , Mitchell E Horwitz  10
Affiliations
Randomized Controlled Trial

Myeloablative versus Reduced-Intensity Conditioning for Hematopoietic Cell Transplantation in Acute Myelogenous Leukemia and Myelodysplastic Syndromes-Long-Term Follow-Up of the BMT CTN 0901 Clinical Trial

Bart L Scott et al. Transplant Cell Ther. 2021 Jun.

Abstract

Several prospective randomized trials comparing conditioning intensity before allogeneic hematopoietic cell transplantation (HCT) have been performed, with conflicting results. Although reduced-intensity conditioning (RIC) leads to lower treatment-related mortality (TRM), this is offset by higher rates of relapse. Long-term follow-up of randomized comparative trials are limited. Here we present long-term follow-up of a randomized comparison of myeloablative conditioning (MAC) compared with RIC before HCT for acute myelogenous leukemia (AML) or myelodysplasia (MDS). Long-term comparative analyses of overall survival, relapse, and relapse-free survival were performed. Patients age 18 to 65 years with <5% marrow myeloblasts were randomized to receive MAC (n = 135) or RIC (n = 137), followed by HCT from an HLA-matched donor. The primary endpoint of the trial was an 18-month pointwise comparison of overall survival. The analyses were performed using a proportional hazards model. The median follow-up of the entire cohort was 51 months. At 4 years, the transplant-related mortality (TRM) was 25.1% for MAC, compared with 9.9% for RIC (P < .001). Patients who received RIC had a significantly higher risk of relapse compared to those who received MAC (hazard ratio [HR], 4.06; 95% CI, 2.59 to 6.35; P < 0.001). Among the patients who relapsed after HCT, postrelapse survival was similar at 3 years (24% for MAC and 26% for RIC). Overall survival was superior for patients who received MAC compared to those who received RIC (HR, 1.54; 95% CI, 1.07 to 2.2; P = .03). Our data show that patients who received MAC were at higher risk of late TRM compared with those who received RIC; however, because of the exceedingly high rates of relapse in the RIC arm, overall survival remained significantly better for patients who received MAC. Among patients with MDS or AML eligible for either MAC or RIC regimens, long-term follow up demonstrates a survival advantage for patients who received MAC.

Keywords: Acute myelogenous leukemia; Conditioning intensity; Hematopoietic cell transplantation; Myelodysplastic syndrome.

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Conflict of interest statement

Conflict of interest statement: There are no conflicts of interest to report.

Figures

Figure 1.
Figure 1.
OS.
Figure 2.
Figure 2.
RFS.
Figure 3.
Figure 3.
Relapse.
Figure 4.
Figure 4.
TRM.
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