Table 1.
Patients and donor characteristics
| Patient 1 (P1) | Patient 2 (P2) | |
|---|---|---|
| Type of sickle Hb | HbSS | HbSS |
| Age at HSCT (years) | 10 | 5 |
|
Patient pretransplant morbidity and
indications for HSCT |
Recurrent VOC, Stroke, ACS, Liver fibrosis, Cholestasis: BT: 51.38 mg/d BD: 42 mg/d |
Abnormal TCD, recurrent VOC |
| Medical management before HSCT | Chronic RBC transfusions, Hydroxyurea, iron chelation |
Chronic RBC transfusions, Hydroxyurea, exchange transfusions, iron chelation |
| CD34 Dose Infused (106/kg) | 4.9 | 6.1 |
| Donor Hb electrophoresis | HbAS | Hb AA |
| Donor gender | Male | Female |
| Donor age (years) | 12 | 17 |
| Days to ANC recovery(>0.5 x 109/L) | 22 | 13 |
| Days to platelet recovery (>50x 109/L) | 22 | 14 |
| Donor Myeloid Chimerism at Day 30 | 93 % | 82% |
| Donor Myeloid Chimerism at Day 100 | N/A | 91% |
| Donor Myeloid Chimerism at Day 300 | 96 % | 100% |
| HbS last follow-up (%) | 30 | 0 |
| Status and follow-Up (days) | Cured/903 | Cured/348 |
Abbreviations: Hb, hemoglobin; HBSS, homozygous sickle cell anemia; HSCT, hematopoietic stem cell transplantation; VOC, vaso-occlusive crises; ACS, acute chest syndrome; BT,total bilirubin; BD, direct bilirubin; TCD, transcranial doppler ultrasonography; RBC, red blood cells; HbAS, Hb S trait; Hb AA, normal electrophoresis.