Safe and Efficacious Allogeneic Bone Marrow Transplantation for Nonmalignant Disorders Using Partial T Cell Depletion and No Posttransplantation Graft-Versus-Host-Disease Prophylaxis

Ronit Elhasid*, Myriam Ben Arush, Irena Zaidman, Ronit Leiba, Ayelet Ben Barak, Sergey Postovsky, Nuhad Haddad, Tami Katz, Shimon Pollack, Ivanka Sami, Osnat Gidoni, Dina Rubin, Hanna Mandel, Dina Attias, Yair Reisner, Amos Etzioni, Jacob M. Rowe

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

15 Scopus citations

Abstract

In an attempt to abrogate the deleterious effects of graft-versus-host disease (GVHD), allogeneic transplantation for nonmalignant diseases was performed using high-dose CD34-cell infusion, partial T cell depletion, and no posttransplantation GVHD prophylaxis. Between 1998 and 2004, 16 patients with matched related donors were treated. Median age was 1.5 years (range, 5 months-18 years). The conditioning regimen consisted of busulphan 16 mg/kg, cyclophosphamide 200 mg/kg, antithymocyte globulin (ATG) 25 mg/kg, and fludarabine 200 mg/m2. No GVHD prophylaxis was given. High doses of CD34 cells, positively selected by immunomagnetic beads, were infused at a median dose of 10.7 × 106 CD34/kg (range, 7.4-50 × 106). A total of 1 × 105/kg T cells were given. All patients engrafted, with no graft rejections. All were alive and well at a median of 37 months posttransplantation (range, 18-89 months). Only 1 patient developed chronic GVHD. No episodes of severe infection occurred during or after transplantation. Immunologic reconstitution with CD3/CD4 T cells > 200/μL was observed at a median of 117 days and that with naive T cells (CD4/CD45RA) at a median of 188 days posttransplantation. Our findings suggest that allogeneic transplantation from a matched family donor for nonmalignant disorders can be successfully performed using high doses of CD34 cells, moderate T cell depletion, and no posttransplantation immunosuppression.

Original languageEnglish
Pages (from-to)329-338
Number of pages10
JournalBiology of Blood and Marrow Transplantation
Volume13
Issue number3
DOIs
StatePublished - Mar 2007
Externally publishedYes

Keywords

  • CD34 cells
  • Matched related donor
  • Nonmalignant disease
  • Peripheral stem cell transplantation
  • T cell depletion

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