Table 1.

Patient characteristics and outcomes

PatientAge/SexDonor typeConditioning regimenStem cell (CD34 cells) dose per kg of recipientPost-HSCT outcome
3 mo/male IFN-γ Haplo Flu/Treo/PTCy 5 million Graft rejection/alive 
5 years/male IFN-γ MFD Flu/Treo/TT 5 million Graft rejection/dead 
3 years/female IFN-γ MSD Flu/Treo/TT 10 million Complete donor chimerism/alive 
2.5 years/female IFN-γ Haplo Flu/Treo/TT TCRαβ depletion 10 million Complete donor chimerism/alive 
6 years/male (second HSCT) IFN-γ Haplo Flu/Treo/TT TCRαβ depletion 10 million Mixed chimerism—improved after graded whole blood DLI/alive 
5 years/male IL-12 MFD Flu/Treo 7 million Complete donor chimerism/alive 
10 years/male IL-12 Haplo Flu/Treo/2 Gy TBI/PTCy 8 million Complete donor chimerism/alive 
6 years/female IL-12 Haplo Flu/Treo/PTCy 8 million Mixed chimerism—improved after graded whole blood DLI/alive 

MSD, matched sibling donor; TBI, total body irradiation.

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