• Join Today!

Become a member and connect with:

  • An Active Online Community
  • Articles and Advice on SCD
  • Help Understanding Clinical Trials
abstracts & posters

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


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

read more

To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More information

The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.