Table 1.
Overview of retro- and prospective studies in the TKI era including pediatric CML patients in BP.
| Study | Design | period | No. entire cohort | No. SCT | Donor type | Conditioning regimen | Stem Cell Source | Prior TKI therapy | Prior CT advanced phase | Disease status at transplant reported | Post-transplant TKI | GVHD | Outcome/Endpoints |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Sembill et al. [6] | r, m | 2007–2020 | BP (18) | BP (14) |
MUD (8) MRD (2) MMD (2) no data (2) |
TBI-based (5) Bu-based (4) others (3) no data (2) |
n.a. |
Y (12) N (2) |
Y (12) no data (2) |
Y | n.a. | GvHD (7) | OS |
| Hafez et al. [60] | r, s | 2007–2017 |
CML (43) BP (4) |
CML (43) BP (4) |
MRD (43) | Bu based (43) |
BM (32) PBSC (11) |
Y (37) N (6) |
Y (4) | Y | Y (4) |
aGvHD (5) cGvHD (19) |
OS EFS TRM |
| Meyran et al. [5] | r/p, s | 2001–2016 |
CML (339) Adv. P (19) |
Adv. P (16) |
MUD (7) MRD (8) MMD (1) |
TBI-based (7) Bu-based (3) no data (6) |
BM (10) PBSC (3) CB (2) No data (1) |
Y (16) |
Y (13) N (3) |
Y | Y (8) | n.a. | CHR, CCyR, MMR, OS |
| Millot et al. [7] | r/p, m | 2000–2017 |
CML (479) AP (19) BP (17) |
HSCT (17) AP (6) BP (11) |
MRD (8) MUD (7) MMD (2) |
n.a. | n.a. | Y (17) |
Y (13) N (4) |
Y | n.a. | n.a. | CHR, CCyR, MMR, OS |
| Suttorp et al. [24] | p, m | 2004–2017 |
CML (156) AP (3) BP (7) |
CML (28) AP (1) BP (4) |
n.a. | n.a. | n.a. | n.a. | n.a. | n.a. | n.a. | n.a. | CHR, CCyR, MMR, OS |
| Shulman et al. [67] | r, s | 2010–2013 |
Adv. P (5) AP (1) BC (4) |
Adv. P (5) AP (1) BC (4) |
MRD (1) MUD (4) |
TBI based (5) | BM (5) | Y (5) |
Y (4) N (1) |
Y | Y |
aGvHD (1) cGvHD (1) |
OS MMR |
| Suttorp et al. [58] | p, m | 1995–2004 |
CML (200) AP (17) BP (14) |
CML (176) AP (9) BP (9) |
MRD (41) MUD (71) MMD (55) |
Bu based (79) TBI-based (82) Others (15) |
BM (102) PBSC (64) CB (2) |
n.a. | Y | n.a. | n.a. |
aGvHD (62) cGvHD (36) |
TRM OS |
| Muramatsu et al. 2010 | r, m | 1993–2005 |
CML (125) Adv. P. (37) |
CML (125) Adv. P (37) |
UD (125) MRD (41) |
TBI-based (96) Others (29) |
BM (125) | Y (17) | n.a. | Y | n.a. |
aGvHD (77) cGvHD (51) |
OS TRM LFS |
| Unal et al. 2007 | r, s | 1997–2006 |
CML (14) AP (3) |
CML (14) AP (3) |
MRD (12) MUD (2) |
Bu based (13) Others (1) |
BM (14) |
Y (3) N (11) |
n.a. | n.a. | n.a. |
aGvHD (1) cGvHD (1) |
OS EFS |
| Millot et al. 2003 | r. m | 1982–1998 |
CML (76) Adv. P (29) |
CML (76) Adv. P (29) |
MRD (60) UD (16) |
TBI-based (53) Bu based (20) others (3) |
n.a. | n.a. | n.a. | n.a. | n.a. |
aGvHD (6) cGvHD (3) |
OS EFS |
Adv. P advanced phase, aGvHD acute GvHD, AP accelerated phase, BM bone marrow, BP blast-phase, Bu Busulfan, CB cord blood, CCyR complete cytogenetic remission, cGvHD chronic GvHD, CHR complete hematologic remission, EFS event free survival, Y Yes, LFS leukemia free survival, m multicenter, MMD mismatched donor, MMR major molecular response, MRD matched related donor, MUD matched unrelated donor, n.a. not available, N No, OS overall survival, p prospective, PBSC peripheral blood stem cells, r retrospective, s single-center, TBI total body irradiation, TRM treatment related mortality, UD unrelated donor.