Main characteristics and outcome of the 87 patients undergoing stem cell transplantation for sickle cell disease
| Characteristics . | SCT period . | |||
|---|---|---|---|---|
| 11/88-01/96 . | 02/96-01/00 . | 01/00-12/04 . | Total: 11/88-12/04 . | |
| Patients | ||||
| No. | 26* | 17 | 44 | 87 |
| Phenotype | 26 SS | 16 SS, 1 Sb0 | 43 SS, 1 Sb0 | 85 SS, 2 Sb0 |
| Sex, no. patients F/M | 13/13 | 11/6 | 16/28 | 40/47 (46% female) |
| Median age, y (range) | 8.8 (2.2-17.1) | 8.8 (3.2-20) | 9.9 (3.2-22) | 8.8 (2.2-22) |
| Age over 15 y, no. patients (%) | 2 | 3 | 5 (11.4) | 10 |
| Median no. of transfusions (range) | 21 (2-61) | 13 (3-41) | 15 (4-51) | 15 (2-61) |
| Median ferritin level, ng/mL (range) | 374 (13-3820) | 792 (340-2059) | 604 (31-3171) | 553 (13-3820) |
| Indications, no. patients | ||||
| Stroke (%) | 9 (34.6) | 13 (76.5) | 14 (31.8) | 36 (41.4) |
| Stenoses | 1 | 0 | 5 | 6 |
| Abnormal TCD without stenosis | 0 | 0 | 2 | 2 |
| Severe anemia and cognitive deficiency and/or silent infarcts | 2 | 0 | 11 | 13 |
| 3 or more VOC/ACSs per y | 14 | 4 | 10 | 28 |
| More than 2 red cell alloimmunizations | 0 | 0 | 2 | 2 |
| Preparation with ATG, no. patients (%) | 14 (54) | 13 (76.5) | 42 (95.5) | 69 (79.3) |
| Stem-cell source, no. patients | ||||
| Bone marrow (%) | 25 | 17 | 32 | 74 (85) |
| Cord blood (%) | 1 | 0 | 11 | 12 (14) |
| Peripheral blood cells (%) | 0 | 0 | 1 | 1 (1.1) |
| Donor mismatches, no. patients (%) | 0 | 4 | 0 | 4 (4.6) |
| Outcome, no. patients | ||||
| Deaths | 2 | 4 | 0 | 6 |
| Rejections/nonengraftment | 5 | 0 | 2 | 7 |
| aGVHD grade 2 or above (%) | 5 (19.2) | 7 (41) | 5 (11) | 17 (20) |
| cGVHD (%) | 4 | 5 | 2 | 11 (13) |
| Characteristics . | SCT period . | |||
|---|---|---|---|---|
| 11/88-01/96 . | 02/96-01/00 . | 01/00-12/04 . | Total: 11/88-12/04 . | |
| Patients | ||||
| No. | 26* | 17 | 44 | 87 |
| Phenotype | 26 SS | 16 SS, 1 Sb0 | 43 SS, 1 Sb0 | 85 SS, 2 Sb0 |
| Sex, no. patients F/M | 13/13 | 11/6 | 16/28 | 40/47 (46% female) |
| Median age, y (range) | 8.8 (2.2-17.1) | 8.8 (3.2-20) | 9.9 (3.2-22) | 8.8 (2.2-22) |
| Age over 15 y, no. patients (%) | 2 | 3 | 5 (11.4) | 10 |
| Median no. of transfusions (range) | 21 (2-61) | 13 (3-41) | 15 (4-51) | 15 (2-61) |
| Median ferritin level, ng/mL (range) | 374 (13-3820) | 792 (340-2059) | 604 (31-3171) | 553 (13-3820) |
| Indications, no. patients | ||||
| Stroke (%) | 9 (34.6) | 13 (76.5) | 14 (31.8) | 36 (41.4) |
| Stenoses | 1 | 0 | 5 | 6 |
| Abnormal TCD without stenosis | 0 | 0 | 2 | 2 |
| Severe anemia and cognitive deficiency and/or silent infarcts | 2 | 0 | 11 | 13 |
| 3 or more VOC/ACSs per y | 14 | 4 | 10 | 28 |
| More than 2 red cell alloimmunizations | 0 | 0 | 2 | 2 |
| Preparation with ATG, no. patients (%) | 14 (54) | 13 (76.5) | 42 (95.5) | 69 (79.3) |
| Stem-cell source, no. patients | ||||
| Bone marrow (%) | 25 | 17 | 32 | 74 (85) |
| Cord blood (%) | 1 | 0 | 11 | 12 (14) |
| Peripheral blood cells (%) | 0 | 0 | 1 | 1 (1.1) |
| Donor mismatches, no. patients (%) | 0 | 4 | 0 | 4 (4.6) |
| Outcome, no. patients | ||||
| Deaths | 2 | 4 | 0 | 6 |
| Rejections/nonengraftment | 5 | 0 | 2 | 7 |
| aGVHD grade 2 or above (%) | 5 (19.2) | 7 (41) | 5 (11) | 17 (20) |
| cGVHD (%) | 4 | 5 | 2 | 11 (13) |
Reported by Bernaudin et al.20