[Skip to Content]
Access to paid content on this site is currently suspended due to excessive activity being detected from your IP address Please contact the publisher to request reinstatement.
[Skip to Content Landing]
Views 697
Citations 0
Research Letter
December 9, 2019

Familial Haploidentical Stem Cell Transplant in Children and Adolescents With High-Risk Sickle Cell Disease: A Phase 2 Clinical Trial

Author Affiliations
  • 1Department of Pediatrics, New York Medical College, Valhalla
  • 2Department of Medicine, New York Medical College, Valhalla
  • 3Department of Pathology, New York Medical College, Valhalla
  • 4Department of Microbiology & Immunology, New York Medical College, Valhalla
  • 5Department of Cell Biology & Anatomy, New York Medical College, Valhalla
  • 6Department of Pediatrics, Medical College of Wisconsin, Milwaukee
  • 7Department of Pediatrics, University of California, Los Angeles, Los Angeles
  • 8Department of Epidemiology & Community Health, New York Medical College, Valhalla
  • 9The New York Blood Center, New York
  • 10Department of Pathology & Immunology, Washington University in St Louis, St Louis, Missouri
  • 11Department of Pediatrics, Washington University in St Louis, St Louis, Missouri
JAMA Pediatr. 2020;174(2):195-197. doi:10.1001/jamapediatrics.2019.4715

Sickle cell disease (SCD) is an autosomal recessive disorder associated with cerebral vasculopathy, stroke, acute chest syndrome, pulmonary hypertension and/or frequent vaso-occlusive crises, and a high risk of early mortality.1,2 Studies have reported 90% to 100% event-free survival (EFS) following human leukocyte antigen matched sibling allogeneic stem cell transplant.3,4 Reported results have used unrelated allogeneic donor sources; however, a paucity of unrelated matched donors and a higher than expected rate of graft failure and chronic graft-vs-host disease (GVHD) were notable disadvantages.5 The CD34+ enrichment and mononuclear cell (MNC) addback (2 × 105 CD3 cells/kg of recipient body weight) have been reported following matched unrelated donor transplant that resulted in 100% engraftment and a low cumulative incidence of acute GVHD and chronic GVHD.6

Limit 200 characters
Limit 25 characters
Conflicts of Interest Disclosure

Identify all potential conflicts of interest that might be relevant to your comment.

Conflicts of interest comprise financial interests, activities, and relationships within the past 3 years including but not limited to employment, affiliation, grants or funding, consultancies, honoraria or payment, speaker's bureaus, stock ownership or options, expert testimony, royalties, donation of medical equipment, or patents planned, pending, or issued.

Err on the side of full disclosure.

If you have no conflicts of interest, check "No potential conflicts of interest" in the box below. The information will be posted with your response.

Not all submitted comments are published. Please see our commenting policy for details.

Limit 140 characters
Limit 3600 characters or approximately 600 words