|
|
Ref |
Phase | No. of pts | Type of Tplt (no of pts) | Conditioning Regimen (no. of pts) | CD34 dose. Other cells if specified (×106 cells/kg, median (range)). | Other GVHD PPx | Type of MSC (no. of doses) | MSC dose (×106 cells/kg, median (range)) | Time to Tplt |
Failed
engraftment | Engraftment (days, median (range)) | aGVHD (no/total evaluable (percent)) | cGVHD (no/total evaluable (percent)) | Relapsed disease (no/total (percent)) | Infections (no/total (percent)) | Morbidity/
mortality
(percent (95% CI)) | Survival (percent (95% CI)) |
|
| [38] | I | 46 (adults) | PBSCT,
MF. | Bu/Cy,
Thiopeta/Cy,
BEAM,
Cy/TBI | 5.0 (3.2–15.8) | CSA & MTX
(day 1, 3, 6). | MF, same as SCT donor. | 2.5 (1–5) | 4 hrs before SCT | 0 | ANC: 14 (11–26).
Plt: 20.5 (15–36). | 23/46
(50%).
⩾ grade II: 13/46 (28.3%) | 22/36
(61%)
Extensive: 8/36
(22.2%) | 12/46
(26%) | NA | Est. 2-yr
PFS: 53%
(37–66%) | Est. 2-yr: 78%
(63–87%) |
|
| [39] | I/II | 14 (peds) | PBSCT,
haplo. | TBI-based (9),
chemo-based (5). | 21.5 (11.6–38.6);
CD3: 0.3 ±
0.3 × 105 cells/kg. | NA | Haplo, same as SCT donor. | 1.6 (1–3.3) | 4 hrs before SCT | 0 | ANC: 12 (10–17).
Plt: 10 (9–18). | 2/14 (14.3%).
Grade III-IV: 0. | 1/7 (14.3%).
Extensive: 0. | 2/14
(14.3%) | Viral reacti-vation: 7/14 (50%).
Fatal: 2/14 (14.3%) | Relapse-free survival at median 9 mo
FU: 82% | Est. 9 mo: 72% |
|
| [40] | Pilot | 10 (9 adults, 1 ped) | PBSCT (4),
BM (4),
PBSC &
BM (2).
All MF. | Cy/TBI (9),
Bu/Cy (1). | 5.14 (0.42–7.14) | CSA & MTX
(day 1, 3, 6). | MF, same as SCT donor. | 0.34 (0.03–1.53) | 4 hrs before SCT | 1 | ANC: 16 (12–21).
Plt > 50 × 109/L: 30
(16–45). | 4/9 (44.5%).
Grade III-IV: 0. | 1/7 (14.3%).
Extensive: 0. | 6/10
(60%) | 3/10 (30%) | Est. 3-yr
PFS: 30% | Est. 3-yr: 40% |
|
| [41] | Pilot/I | 12 (adults) | PBSCT,
MF. | Cy (2),
Cy/TBI (8),
Bu/Cy (2). | 4.34 (2.59–13.1) | CSA & MTX
(day 1, 3, 6, 11). | MF, same as SCT donor. | 1.48 (1.29–2.24) | 1 hr after SCT | 0 | ANC: 11 (7–15).
Plt: 13.5 (10–17). | 9/12 (75%).
⩾ grade II: 2/12 (16.7%). | 4/12 (33.3%).
Extensive: 2/12 (16.7%). | 4/12
(33.3%) | Viral reacti-vation: 4/12 (30%).
Fatal infection 1/12 (8.3%). | NA | Alive: 7/12, 58.3%,
range of FU 29 to >57 mo.
Dead: 5/12, 41.7%,
range 5–19 mo post-tplt. |
|
| [42] | Pilot | 7 (3 adults, 4 peds) | PBSCT (4):
MF (2),
MUD (2).
BM (2):
MF (1),
MM (1).
CB,
MM (1). | Cy/TBI (1),
Flu/ATG (2),
Bu/Cy/
ATG (2),
Flu/Mel/
ATG (1),
Cy/TBI/
ATG/Cam (1). | 7.2 (0.21–68.9) | CSA (2),
CSA & MTX (3),
CSA & Pred (1),
Tac & Rapamycin (1). | MF, same as SCT donor (3).
Haplo (4). | 1.0 | Within 4 hrs of SCT | 0 | ANC: 12 (10–28).
Plt > 50 × 109/L: 15
(11–45). | 6/7 (85.7%).
Grade II: 2/7 (28.6%).
Grade III-IV: 0. | 1/7 (14.3%),
extensive. | 0/7
(0%). | Severe infections: 2/7 (28.3%).
Fatal: 1/7 (14.3%). | PFS at 2-yr median
FU: 6/6 (100%). | 2-yr median
FU: 6/7 (85.7%). |
|
| [43] | I/II | 9 (adults) | CB & PBSCT:
PBSCT haplo (7),
MM (2). | Cy/TBI/ATG
/Flu (6), Bu/Cy
/Flu/ATG (3). | PBSCT: 2.61 (2.4–3.31).
CB: 0.12 (0.037–0.28).
CD3: 0.0036 (0.0001–0.0083). | CSA & Methyl-pred
(day-1 to 10–14). | same as SCT donors. | 1.2 (1.04–2.22) | CB, then PB-SCT,
then MSC within 24 hrs. | 0 | ANC: 12 (10–31).
Plt: 44 (27–98). | 5/9 (55.6%).
Grade II: 4/9 (44.4%).
Grade III-IV: 0. | 1/8 (12.5%),
limited. | 1/9
(11.1%) | Viral reacti-vation:
2/9 (22.2%). | 2-yr cumulative
TRM: 11% (2–71%) | OS at 22 mo:
8/9 (88.9%). |
|
| [44] | Pilot/I | 20 (adults) | PBSCT,
MM. | Flu/TBI | 4.8 (1.6–11.8).
CD3: 312 (120–540). | MMF & Tac | MM, third party different from SCT donors. | NA | 0.5–2 hrs before SCT | 1 | NA | 11/20 (55%).
Grade II: 5/20 (25%).
Grade III-IV: 4/20 (20%). | 15/20 (75%).
Extensive: 8/20 (40%). | 6/20 (30%) | Fatal: 1/20 (5%). | 1-yr
PFS: 60%. | 1-yr
OS: 80%. |
|
