Plerixafor alone for the mobilization and transplantation of HLA-matched sibling donor hematopoietic stem cells.

Journal Article (Journal Article;Multicenter Study)

Plerixafor, a direct antagonist of CXCR4/stromal-derived factor 1, can safely and rapidly mobilize allografts without the use of granulocyte colony-stimulating factor (G-CSF). We conducted a phase 2, multicenter, prospective study of plerixafor-mobilized HLA-identical sibling allografts for allogeneic hematopoietic cell transplantation in recipients with hematological malignancies. Donors (n = 64) were treated with subcutaneous plerixafor (240 µg/kg) and started leukapheresis (LP) 4 hours later. The primary objective was to determine the proportion of donors who were successfully mobilized: defined as collection of ≥2.0 × 106 CD34+ cells per kilogram recipient weight in ≤2 LP sessions. Recipients subsequently received reduced intensity (RIC; n = 33) or myeloablative (MAC; n = 30) conditioning. Sixty-three of 64 (98%) donors achieved the primary objective. The median CD34+ cell dose per kilogram recipient weight collected within 2 days was 4.7 (0.9-9.6). Plerixafor was well tolerated with only grade 1 or 2 drug-related adverse events noted. Bone pain was not observed. Plerixafor-mobilized grafts engrafted promptly. One-year progression-free and overall survivals were 53% (95% confidence interval [CI], 36% to 71%) and 63% (95% CI, 46% to 79%) for MAC and 64% (95% CI, 47% to 79%) and 70% (95% CI, 53% to 84%) for RIC recipients, respectively. Donor toxicity was reduced relative to G-CSF mobilized related donors. This is the first multicenter trial to demonstrate that, as an alternative to G-CSF, plerixafor rapidly and safely mobilizes sufficient numbers of CD34+ cells from matched sibling donors for HCT. Engraftment was prompt, and outcomes in recipients were encouraging. This trial was registered at as #NCT01696461.

Full Text

Duke Authors

Cited Authors

  • Chen, Y-B; Le-Rademacher, J; Brazauskas, R; Kiefer, DM; Hamadani, M; DiPersio, JF; Litzow, MR; Craig, M; Horwitz, ME; Artz, AS; McClune, BL; Fernandez, HF; Duong, HK; Kobusingye, H; Proue, M; Drexler, RJ; Horowitz, MM; Shaw, BE; Miller, JP; Hosoba, S; Waller, EK; Devine, SM

Published Date

  • March 26, 2019

Published In

  • Blood Adv

Volume / Issue

  • 3 / 6

Start / End Page

  • 875 - 883

PubMed ID

  • 30890544

Pubmed Central ID

  • PMC6436017

Electronic International Standard Serial Number (EISSN)

  • 2473-9537

Digital Object Identifier (DOI)

  • 10.1182/bloodadvances.2018027599


  • eng

Conference Location

  • United States