Plerixafor on demand in ten healthy family donors as a rescue strategy to achieve an adequate graft for stem cell transplantation

Transfusion. 2015 Aug;55(8):1993-2000. doi: 10.1111/trf.13059. Epub 2015 Feb 26.

Abstract

Background: In allogeneic hematopoietic stem cell (HSC) transplantation, the collection of an appropriate number of HSCs while maintaining a high level of safety for healthy donors is fundamental. Inadequate HSC mobilization can be seen with the standard use of granulocyte-colony-stimulating (G-CSF). Plerixafor (PL) is a chemokine receptor CXC Type 4-stromal-derived factor 1 inhibitor; its HSC-mobilizing properties are synergistic with G-CSF in poor mobilizing patients. The use of PL as adjuvant or alternative to G-CSF in healthy donors has shown a good safety profile but is so far off-label.

Study design and methods: We report 10 healthy HSC donors treated with PL because of insufficient response to G-CSF alone or contraindication to G-CSF. Eight donors did not mobilize enough CD34+ cells with G-CSF alone because poor mobilizers or because insufficient HSCs were harvested according to the clinical need of the patient; in two cases G-CSF administration and marrow harvest were unfeasible or contraindicated in the donor.

Results: The use of PL for mobilization increased the number of circulating CD34+ cells by 2.8-fold and the CD34+/kg collection by 3.0-fold. Only mild adverse events were reported (bone pain or discomfort) and not univocally attributable to PL. Rate of engraftment and graft-versus-host disease were similar to those seen in recipients of grafts from G-CSF only-mobilized donors.

Conclusion: We exposed 10 allogeneic donors to mobilization with PL. PL was well tolerated in all cases and ensured procurement of an adequate graft for transplantation resulting in a normal hematopoietic engraftment.

Publication types

  • Case Reports
  • Clinical Trial

MeSH terms

  • Adult
  • Aged
  • Allografts
  • Antigens, CD34 / blood
  • Benzylamines
  • Blood Cell Count
  • Carcinoma, Renal Cell / therapy
  • Colony-Forming Units Assay
  • Cyclams
  • Drug Synergism
  • Female
  • Graft Survival
  • Graft vs Host Disease / etiology
  • Granulocyte Colony-Stimulating Factor / pharmacology
  • Hematopoietic Stem Cell Mobilization / adverse effects
  • Hematopoietic Stem Cell Mobilization / methods*
  • Hematopoietic Stem Cells / chemistry
  • Hematopoietic Stem Cells / drug effects
  • Heterocyclic Compounds / adverse effects
  • Heterocyclic Compounds / pharmacology*
  • Humans
  • Kidney Neoplasms / therapy
  • Lenograstim
  • Leukapheresis
  • Leukemia, Myeloid, Acute / therapy
  • Living Donors
  • Male
  • Middle Aged
  • Pain / chemically induced
  • Parents
  • Peripheral Blood Stem Cell Transplantation / adverse effects
  • Peripheral Blood Stem Cell Transplantation / methods
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy
  • Recombinant Proteins / pharmacology
  • Siblings
  • Treatment Outcome

Substances

  • Antigens, CD34
  • Benzylamines
  • Cyclams
  • Heterocyclic Compounds
  • Recombinant Proteins
  • Granulocyte Colony-Stimulating Factor
  • Lenograstim
  • plerixafor