Unrelated Umbilical Cord Blood Transplantation for Sickle Cell Disease Following Reduced Intensity Conditioning: Results of a Phase I Trial.
Biol Blood Marrow Transplant. 2017 May 31;:
Authors: Abraham A, Cluster A, Jacobsohn D, Delgado D, Hulbert ML, Kukadiya D, Murray L, Shenoy S
Abstract
Hematopoietic stem cell transplantation from HLA-matched sibling donors results in disease free survival of >90% in patients with sickle cell disease (SCD) but only about 18% have suitable donors. Unrelated cord blood transplantation (UCBT) is one way to expand donor options for patients with severe SCD but historically has been associated with high graft rejection rates (50-62%). We hypothesized that the addition of thiotepa to a previously tested reduced intensity conditioning (RIC) regimen would support engraftment after UCBT in SCD patients. Nine children (3-10 years) with cerebrovascular complications of SCD underwent 5-6/6 HLA-matched (A, B and DRB1 loci) UCBT after conditioning with hydroxyurea, alemtuzumab, fludarabine, thiotepa and melphalan. A calcineurin inhibitor and mycophenolate mofetil were used for graft-versus-host-disease (GVHD) prophylaxis. With median follow up of 2.1 (range 1-4.2) years, seven patients had sustained donor cell engraftment and are free of SCD, two had autologous recovery. Acute GVHD (grade 2-4), mild, and moderate chronic GVHD developed in three, two, and one patient respectively. Of five patients >2 years post-UCBT, four have discontinued systemic immunosuppression. Seven patients had viral infections (CMV, EBV, RSV or Adenovirus) and recovered. The 1-year overall and disease free survival rates were 100% and 78% respectively. Thus, this RIC regimen was able to achieve donor engraftment in the majority. Future efforts will focus on further reducing acute GVHD and viral infection rates.
PMID: 28578010 [PubMed - as supplied by publisher]
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