| | Study | Median age (years) | Number of pts | Type of disease (number of pts) | Type of relapse (number of pts) | Chemotherapy | HDCT (mg/m2; mg/kg) | Number of HDCT pts | Source of stem cell | Response (number of pts) | EFS | OS |
| | Rasper et al. 2014, registry study [14] | NL | 239 | Local (42)
Metastatic (197) | Local (42)
Distant (142)
Combined (30) | T/C
I/Te
If/E/carboplatin | C1: busulfan/melphalan (NL)
C2: treosulfan/melphalan (NL) | 73
C1 = 15
C2 = 38
COR = 20 (ONR) | NL | Prior to HDCT:
C1: CR (5)
PR (2)
C2: CR (19)
PR (8) | 2 yr HDCT: 44–47%
5 yr HDCT: 20–24% | 2 yr HDCT: 53–66%
5 yr HDCT: 40–42% |
| | McTiernan et al. 2006, retrospective [15] | 19 | 33 | Local (22)
Metastatic (11) | Local (11)
Distant (18)
Combined (4) | Ca
E
C
A
I
D
V | Busulfan/melphalan (600 mg/m2/140 mg/m2) (22 pts)
Melphalan/etoposide (130 mg/m2/60 mg/m2) (7 pts)
TBI
Melphalan/cytoxan (16 mg/kg/60 mg/kg) (1 pts)
Melphalan (150 mg/m2) (3 pts) | 33 | PBSC
BSC | Prior to HDCT:
CR (14)
PR (10)
Post HDCT:
CR (21)
PR (2) | 2 yr EFS 42.5%
5 yr EFS 38.5% (HDCT) | 2 yr OS 50.7%
5 yr OS 42.8% (HDCT) |
| | Barker et al. 2005, retrospective [16] | 13.5 | 55 | Local (30)
Metastatic (25) | Local (6)
Distant (39)
Combined (10) | VDC-IE
VACIME
VDC-A
VDC-A-M | Busulfan/melphalan/THIO (NL) | 13 | PBSC | Prior to HDCT
CR/PR (27) | 5 yr HDCT: 61% (HDCT) | 5 yr HDCT: 77% (HDCT) |
| | Ferrari et al. 2015, retrospective [17] | NL | 107 | NL | Local (11)
Distant (96) | High I
VDCAIE | Busulfan/melphalan (4 mg/kg and 140 mg/m2) | 20 | PBSC | NL | NL | 5 yr OS HDCT: 50% |
| | Palmerini et al. 2009, retrospective [18] | 17 | 72 | NL | Local (11) | NL | Busulfan/melphalan (4 mg/kg and 140 mg/m2)
Palliative | 24 | NL | NL | NL | 3 yr. HDCT: 33% |
| | Shankar et al. 2003, retrospective [19] | 14 | 64 | Local (64) | Local (11) | Ca/E
C/E/Ca
C/E | Melphalan (NL)
TBI | 7 | PBSC
BSC | NL | mRFS = 16 months | mOS = 49 months (HDCT) |
| | Bacci et al. 2003, retrospective [3] | 18 | 195 | NL | Local (57)
Distant (138) | None | Melphalan (NL)
Busulfan (NL) | 35
ONR = 2 | PBSC
BSC | NL | 5 yr with HDCT: 21.2% | mOS with HDCT: 27.1 m |
|
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TBI: total body irradiation, HDCT: high-dose chemotherapy, SCT: stem cell transplant, CR: complete response, PR: partial response, EFS: event-free survival, OS: overall survival, V: vincristine, E: etoposide, M: methotrexate, A: dactinomycin, If: ifosfamide, C: cyclophosphamide, D: doxorubicin, T: topotecan, I: irinotecan, Te: temozolomide, Ca: carboplatin, NL: not listed, high I: high-dose ifosfamide, mOS: median overall survival, mRFS: median relapse-free survival, COR: consortium of other regimens, and ONR: outcomes not reported.
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