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Haploidentical T-cell depleted hematopoietic stem cell transplant

Development stage
Discontinued
Lead developer
St. Jude Children's Research Hospital
Modality
Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

This therapeutic intervention refers to a specific protocol for haploidentical hematopoietic stem cell transplantation (HSCT) developed by St. Jude Children's Research Hospital for pediatric patients with severe sickle cell disease (SCD) who have experienced a stroke or have abnormal transcranial Doppler (TCD) results. The procedure utilizes stem cells from a partially HLA-matched family donor (haploidentical). To mitigate the high risk of graft-versus-host disease (GVHD) associated with mismatched donors, the graft is T-cell depleted (specifically CD34+ enrichment and CD3+ selection) using the CliniMACS device. The protocol employs a reduced-intensity conditioning (RIC) regimen consisting of busulfan, cyclophosphamide, thiotepa, and OKT3, preceded by a three-month course of hydroxyurea and azathioprine to facilitate engraftment and prevent rejection.

Other names
Haploidentical HSCTT-cell depleted haploidentical transplantHaploidentical stem cell transplantation

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