Table 1 Patients’ characteristics and outcome of dual haplo-HCT and liver transplant for severe SCD
Patient #1 | Patient #2 | |
|---|---|---|
Age at HCT (yr.) | 17 | 25 |
Sex | Male | Male |
Disease | SCD and end-stage liver disease | SCD and end-stage liver disease |
HLA match of HCT graft (no. of alleles matched/total no.) | Haplo HLA 5/10 | Haplo HLA 5/10 |
Donor | Aunt | Brother |
ABO blood group and Rh type | ||
Donor | O+ | O+ |
Recipient | O− | O+ |
Donor age | 39 years | 27 years |
Donor sex | Female | Male |
Sex match | Unmatched | Matched |
Donor HBEP | Normal | Sickle cell trait |
Donor-specific antibodies (DSA) | No | No |
Donor HBEP | Normal | Sickle cell trait |
CMV IgG serology | Both seropositive | Both seropositive |
ABO alloimmunization | 0 | 0 |
Time from SOT | 8 months | 4 months |
Conditioning regimen | ATG/Thio/Flu/Cy/TBI | ATG/Thio/Flu/Cy/TBI |
GVHD prophylaxis | Sirolimus + PTCy + MMF | Sirolimus + PTCy + MMF |
Stem cell source | Bone marrow | Bone marrow |
Graft composition (cells/kg) | ||
TNC HSCs | 14.4*108 | 7.07 *108 |
CD 34+ HSCs | 5.79*106 | 7.01 *106 |
Neutrophil engraftment | Day +16 of HCT | Day +21 of HCT |
Platelet engraftment | Day +18 of HCT | Day +27 of HCT |
CMV reactivation after HCT or LDLT | Yes | Yes |
Chimerism post-HCT engraftment: | ||
Day +30, +100, +365, 2 years | 100% | 100% |
Myeloid | 100% | 100% |
Lymphoid | ||
Ferritin: | ||
Pre-HCT | 3943 µg/L | 850 µg/L |
Post-HCT | 137 µg/L | 98.4 µg/L |
Hemoglobin SS: | ||
Pre-HCT | 44.6% | 43.7% |
12 months post-HCT | 0.0%¶ | 24.0%¶ |
24 months post-HCT | 0.0% | 29.0% |
2-year OS | Alive | Alive |
