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Clinical Trial
. 2021 Jan 21;137(3):420-428.
doi: 10.1182/blood.2020007535.

Double unrelated umbilical cord blood vs HLA-haploidentical bone marrow transplantation: the BMT CTN 1101 trial

Ephraim J Fuchs  1 , Paul V O'Donnell  2 , Mary Eapen  3 , Brent Logan  4 , Joseph H Antin  5 , Peter Dawson  6 , Steven Devine  7 , Mary M Horowitz  3 , Mitchell E Horwitz  8 , Chatchada Karanes  9 , Eric Leifer  10 , John M Magenau  11 , Joseph P McGuirk  12 , Lawrence E Morris  13 , Andrew R Rezvani  14 , Richard J Jones  1 , Claudio G Brunstein  15
Affiliations
Clinical Trial

Double unrelated umbilical cord blood vs HLA-haploidentical bone marrow transplantation: the BMT CTN 1101 trial

Ephraim J Fuchs et al. Blood. .

Abstract

Results of 2 parallel phase 2 trials of transplantation of unrelated umbilical cord blood (UCB) or bone marrow (BM) from HLA-haploidentical relatives provided equipoise for direct comparison of these donor sources. Between June 2012 and June 2018, 368 patients aged 18 to 70 years with chemotherapy-sensitive lymphoma or acute leukemia in remission were randomly assigned to undergo UCB (n = 186) or haploidentical (n = 182) transplant. Reduced-intensity conditioning comprised total-body irradiation with cyclophosphamide and fludarabine for both donor types. Graft-versus-host disease prophylaxis for UCB transplantation was cyclosporine and mycophenolate mofetil (MMF) and for haploidentical transplantation, posttransplant cyclophosphamide, tacrolimus, and MMF. The primary end point was 2-year progression-free survival (PFS). Treatment groups had similar age, sex, self-reported ethnic origin, performance status, disease, and disease status at randomization. Two-year PFS was 35% (95% confidence interval [CI], 28% to 42%) compared with 41% (95% CI, 34% to 48%) after UCB and haploidentical transplants, respectively (P = .41). Prespecified analysis of secondary end points recorded higher 2-year nonrelapse mortality after UCB, 18% (95% CI, 13% to 24%), compared with haploidentical transplantation, 11% (95% CI, 6% to 16%), P = .04. This led to lower 2-year overall survival (OS) after UCB compared with haploidentical transplantation, 46% (95% CI, 38-53) and 57% (95% CI 49% to 64%), respectively (P = .04). The trial did not demonstrate a statistically significant difference in the primary end point, 2-year PFS, between the donor sources. Although both donor sources extend access to reduced-intensity transplantation, analyses of secondary end points, including OS, favor haploidentical BM donors. This trial was registered at www.clinicaltrials.gov as #NCT01597778.

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Conflict of interest statement

Conflict-of-interest disclosure: The authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Randomization and treatment. dUCB, double umbilical cord blood; haplo-BM, HLA-haploidentical bone marrow.
Figure 2.
Figure 2.
Treatment schema. For patients receiving umbilical cord blood (UCB) (A) or HLA-haploidentical bone marrow (B). BMT, blood or marrow transplantation; Cy, cyclophosphamide; G-CSF, granulocyte colony-stimulating factor; MMF, mycophenolate mofetil; TBI, total-body irradiation; tid, thrice daily.
Figure 3.
Figure 3.
Cumulative incidences of recovery. (A) Neutrophil recovery. (B) Platelet recovery. ANC, absolute neutrophil count; Conf. Interval, confidence interval; Haplo, haploidentical; PLT, platelet.
Figure 4.
Figure 4.
Cumulative incidences of acute GVHD. (A) Acute grades II-IV GVHD. (B) Acute grades III-IV GVHD. aGVHD, acute GVHD.
Figure 5.
Figure 5.
Cumulative incidence of chronic GVHD. cGVHD, chronic GVHD.
Figure 6.
Figure 6.
Outcomes of transplantation according to graft source. (A) PFS. (B) Relapse. (C) Nonrelapse mortality. (D) OS.
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References

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