Haploidentical Peripheral Blood Stem Cell Transplantation Demonstrates Stable Engraftment in Adults with Sickle Cell Disease

Santosh L. Saraf*, Annie L. Oh, Pritesh R. Patel, Karen Sweiss, Matthew Koshy, Sally Campbell-Lee, Michel Gowhari, Shivi Jain, David Peace, John G. Quigley, Irum Khan, Robert E. Molokie, Nadim Mahmud, Victor R. Gordeuk, Damiano Rondelli

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

39 Scopus citations


We report on the screening and development of haploidentical hematopoietic stem cell transplantation (HSCT) for adult patients with clinically aggressive sickle cell disease (SCD) at our institution. Of 50 adult SCD patients referred for HSCT between January 2014 and March 2017, 20% were denied by insurance. Of 41 patients initially screened, 10% lacked an available haploidentical donor, 29% had elevated donor-specific antibodies (DSAs), and 34% declined to proceed to HSCT. All 10 patients who were transplanted received peripheral blood stem cells. The initial 2 were conditioned with alemtuzumab/total body irradiation (TBI) 3 Gy followed by post-transplant cyclophosphamide and failed to engraft. The next 8 patients received the regimen developed at Johns Hopkins University with TBI 3 Gy. Granulocyte colony-stimulating factor was administered from day +12 in those with HbS < 30%. All 8 patients engrafted with a median time to neutrophil >.5 × 10 9 /L of 22 days (range, 18 to 23). One patient subsequently lost the graft, and 7 (87.5%) maintained >95% donor cell chimerism at 1-year post-HSCT. Two patients developed acute graft-versus-host disease (GVHD) of at least grade II. One had chronic GVHD and died >1 year after HSCT of unknown causes. With a median follow-up of 16 months (range, 11 to 29), 7 patients (87.5%) are alive. Our findings suggest that limited insurance coverage, high rate of DSAs, and patient declining HSCT may limit the availability of haploidentical HSCT in adult SCD patients. The modified Hopkins regimen used here demonstrates high engraftment and low morbidity rates and should be tested in larger, multicenter, prospective clinical trials.

Original languageEnglish (US)
Pages (from-to)1759-1765
Number of pages7
JournalBiology of Blood and Marrow Transplantation
Issue number8
StatePublished - Aug 2018


  • Donor specific antibody
  • G-CSF
  • Haploidentical
  • Sickle cell disease
  • Transplantation

ASJC Scopus subject areas

  • Hematology
  • Transplantation


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