|
| 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) |
|