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Promising results at 1 year follow-up following familial haploidentical (FHI) T-cell depleted (TCD) with CD34 enrichment and T-cell (CD3) addback allogeneic stem cell transplantation in patients with high-risk sickle cell disease (SCD)

key information

source: American Society of Hematology

year: 2017

authors: Julie-An Talano, Theodore B Moore, Carolyn A. Keever-Taylor, Shalini Shenoy, Mark C. Walters, Susan K Parsons, Allen J Dozor, Deborah Friedman, Qiuhu Shi, Suzanne Braniecki, Brenda J. Grossman, Rona Singer Weinberg, Elliott Vichinsky, Yaya Chu, Erin Morris, Sandra Fabricatore, Janet Ayello, Lee Ann Baxter-Lowe, Mitchell S. Cairo

summary/abstract:

AlloSCT from HLA-matched MSD has been successful for high-risk SCD, and is the only known curative therapy (Talano/Cairo et al EJH, 2014). We have recently demonstrated 100% EFS and absence of sickle cell symptoms following reduced toxicity conditioning in HLA matched sibling donor (MSD) or cord blood AlloSCT (Bhatia/Cairo et al BMT, 2014). However, 5 out of 6 children lack an HLA MSD without SCD. Only 19% of recipients of African descent will identify a well-matched unrelated donor (MUD) and results after unrelated UCBT are poor (Radhakrishnan/Cairo et al BBMT, 2013). Recent results utilizing matched unrelated donors in a multi-center trial showed unacceptable rates of cGVHD at 62% (95% CI 41-77) (Shenoy et al Blood, 2016). We demonstrated CD34+ selection followed by T-cell addback from MUD in pediatric recipients led to 100% engraftment with minimal aGVHD (Geyer/Cairo et al BJH, 2011). Limitations in the past of various T-cell depletion methods have included a higher incidence of graft failure, delayed immune reconstitution, opportunistic fungal infection and higher incidence of cGVHD. FHI TCD AlloSCT utilizing the approach of CD34+ enrichment and T-cell addback could expand the donor pool and improve outcomes for patients with high risk SCD and have similar outcomes to AlloSCT from HLA MSD.

organization: Medical College of Wisconsin, Milwaukee, WI; UCLA Division of Pediatric Hematology, Los Angeles, CA; Washington University Medical Center, Saint Louis, MO; UCSF Benioff Children’s Hospital, Oakland, CA; Tufts Medical Center, Boston, MA; New York Medical College, Valhalla, NY; New York Blood Center, New York, NY; Children’s Hospital Los Angeles, Los Angeles, CA

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