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

Mitchell S Cairo; Julie-An Talano; Theodore B Moore; Qiuhu Shi; Rona Singer Weinberg; Brenda Grossman; Shalini Shenoy

doi:10.1001/jamapediatrics.2019.4715

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

JAMA Pediatr. 2020 Feb 1;174(2):195-197. doi: 10.1001/jamapediatrics.2019.4715.

Authors

Mitchell S Cairo^{1

2

3

4

5}, Julie-An Talano⁶, Theodore B Moore⁷, Qiuhu Shi⁸, Rona Singer Weinberg⁹, Brenda Grossman¹⁰, Shalini Shenoy¹¹

Affiliations

¹ Department of Pediatrics, New York Medical College, Valhalla.
² Department of Medicine, New York Medical College, Valhalla.
³ Department of Pathology, New York Medical College, Valhalla.
⁴ Department of Microbiology & Immunology, New York Medical College, Valhalla.
⁵ Department of Cell Biology & Anatomy, New York Medical College, Valhalla.
⁶ Department of Pediatrics, Medical College of Wisconsin, Milwaukee.
⁷ Department of Pediatrics, University of California, Los Angeles, Los Angeles.
⁸ Department of Epidemiology & Community Health, New York Medical College, Valhalla.
⁹ The New York Blood Center, New York.
¹⁰ Department of Pathology & Immunology, Washington University in St Louis, St Louis, Missouri.
¹¹ Department of Pediatrics, Washington University in St Louis, St Louis, Missouri.

Abstract

This phase 2 clinical trial in patients with high-risk sickle cell disease assesses the feasibility, safety, and 1-year event-free survival after myeloimmunoablative conditioning and familial haploidentical stem cell transplant.

Publication types

Clinical Trial, Phase II
Letter
Multicenter Study
Research Support, Non-U.S. Gov't
Research Support, U.S. Gov't, P.H.S.

MeSH terms

Adolescent
Anemia, Sickle Cell / surgery*
Child
Child, Preschool
Female
Humans
Male
Prospective Studies
Risk Assessment
Stem Cell Transplantation*
Young Adult

Grants and funding

R01 FD004090/FD/FDA HHS/United States