Table 2.
Selection of CML studies using HSCT including patients beyond frontline therapy
| Authors | Number of patients | HSCT y | Disease stage (patient %) | TKI used before HSCT (patient %) | TRM % | DFS/LFS/PFS/RI % | OS % | Study type |
|---|---|---|---|---|---|---|---|---|
| chP | ||||||||
| Chaudhury et al34 | 449 177 (<18 y) 272 (18-29 y) |
2001-2010 | chP1 (100) | TKI (60) <18 y TKI (48) 18-29 y |
13 (at 1 y), 18 (at 3 y), 20 (at 5 y) | LFS 59% 57 (<18 y) 60 (18-29 y) n.s. |
75% OS (at 5 y) 76% (<18 y) 74% (18-29 y) n.s. |
Retrospective, multicenter, children and young adults (CIBMTR) |
| Yassine et al35 | 199 (adults) 97 (children/adults) |
Meta- analysis | chP (100) | Meta-analysis | 20 (adults) 28 (children/adults) |
Adult DFS 66%, adults/children DFS 47%/PFS 82% |
Adults 84%, children 91%, adults/children 76% |
Meta-analysis resistant/intolerant chP to >1 TKI |
| AP and BC | ||||||||
| Jiang et al29 | 132 | 2001-2008 | AP (100) | Imatinib (66%) Imatinib + HSCT (34) |
11 | Low risk: imatinib 85% HSCT 95% (PFS at 6 y) High risk: imatinib 19% HSCT 100% (PFS at 5 y) |
Low risk: imatinib 100% HSCT 81% (OS at 6 y) High risk: imatinib 18% HSCT 100% (OS at 5 y) |
Prospective single-center study (imatinib vs HSCT) |
| Khoury et al57 | 449 | 1999-2004 | chP2 (41), AP (41), BC (18) | Imatinib (50) | chP2 33, AP 34, BC 46 (at 1 y) | chP2 27%, AP 37%, BC 10% (LFS at 3 y) |
chP2 36%, AP 43%, BC 14% (OS at 3 y) |
Retrospective multicenter study (CIBMTR) |
| Zheng et al36 | 32 | 2002-2011 | AP (59), BC (41) | Imatinib (53) | Cord 38, sib 12 (at 0.5 y) |
Cord 50% sib 40% (LFS at 5 y) |
Cord 62%, Sib 49% (OS at 5 y) |
Retrospective single-center study (cord vs sib) |
| Radujkovic et al33 | 170 | 2004-2016 | BC in 2 chP (56) BC active (44) |
1G-TKI (59), 2G-TKI (33), 3G-TKI (8) | 19.7 (at 1 y) 23.3 (at 3 y) Active BC: 27.1% 3 y BC in remission: 20.2% 3 y |
LFS 34.6, RI 45.7 (at 1 y). LFS 26.1, RI 50.7 (at 3 y) LFS: active BC: 11.6% 3 y BC in remission 33.8% 3 y RI: active BC: 56.4.6% 3 y BC in remission 45.9% 3 y |
57.5%(at 1 y) 38.5 (at 3 y) Active BC: 23.8% (3 y) BC in remission 51% (3 y) |
Retrospective EBMT study HSCT in treated vs untreated BC |
| Yang et al41 | 278 | 2002-2021 | de novo AP (100) | Imatinib (67) or 2G-TKI (33; nilotinib 24, dasatanib 7, flumatinib 1) | Censored at HSCT | TFS 89% (at 6 y) Low risk 95 (5 y) Interm 76 (5 y) High-risk 19 (5 y) No significance between 1G-TKI versus 2G for TFS |
OS 90% (at 6 y) Low risk 54 (5 y) Interm. 36 (5 y) High-risk 10 (5 y) No significance between 1G-TKI vs 2G for OS |
Comparison between imatinib and 2G-TKI before HSCT |
| Different disease phase | ||||||||
| Saussele et al24 | 84 | 2003-2008 | chP1 (TKI) (23), chP1 (TKI failure) (44), AP (4), BC (30) | Imatinib | 8 in chP (at 3 y) 18 in AdP (at 3 y) |
CMR at last PCR 88% | chP1 (elective) 88%, chP1 (imatinib failure) 94%, AP 59% (OS at 3 y) |
Prospective multicenter study (CML IV study) |
| Jabbour et al55 | 47 | 2004-2007 | chP1 (34), chP2 (21), AP (25), BC (19) | Imatinib failure (100) + 2G-TKI (62) |
13% (at 2 y) | 49% (EFS at 2 y) (mutation 36% vs no mutation 58%) | 63% (OS at 2y) (mutation 44% vs no mutation 76%) | Retrospective single-center study |
| Topcuoglu et al37 | 84 | 1989-2007 | chP1 (79), chP2 (6), AP (15) | NA | 7% RIC 14% MAC |
48% (LFS at 5 y) No difference RIC vs MAC | 56% (OS at 5 y) No difference RIC vs MAC | Retrospective single-center study (RIC vs MAC) |
| Oyekunle et al38 | 68 | 2002 – 2009 | chP1 (40), >chP1 (60) | Pre-HSCT TKI (71), post-HSCT TKI (29) |
NA | 54% (LFS at 2 y) | 63% (OS at 2 y) | Single institute HSCT in TKI era |
| Milojkovic et al51 | 5732 | 2000-2011 | Prior TKI: chP (51), chP >1 2(59), AP (14), BC (10), no-TKI: NR |
Prior TKI (22), no TKI (78) |
NA | Non-TKI 46% Prior TKI 42% (PFS at 5 y) |
Non-TKI 61% Prior TKI 59% (OS at 5 y) |
Retrospective multicenter study (EBMT) |
| Piekarska et al39 | 25 | 2008-2013 | chP1 (50), chP2/AP (29), BC (21) | Dasatinib (53), nilotinib (18), or both (29) | 7.1 (chP1), 12.5 (chP2/AP); 50 (BC) | RI: 29.6% RI: chP1 21.4% RI: chP2/AP 12.5% RI: BP 50% |
chP1 92.9%, chP2/AP 85.7% BP 0% (at 1 or 3 y) |
Prospective |
| Lubking et al23 | 118 | 2002-2017 | chP1 (47.5), chP >1 (40.7), AP/BC (11.9) | Imatinib (39.8), imatinib +2G-TKI (33.1), imatinib +2G-TKI +3G-TKI (5.1), 2G-TKI (13.6), 2G-TKI +3G-TKI (5.1), no TKI (3.4) | 11.6 in chP ≥1 (at 5 y) 23.1 in AP/BC (at 5 y) |
chP: 66% molec relapse (at 2 y) AP/BC: 71.4% progress to AP/BC 50% to AdP in chP >1 |
chP 96,3% 70.1% > chP1/AP 36.9% BP AP/BP to chP 70.1% chP TKI resist. 96.8% (all at 5 y) |
Swedish registry study |
| Hu et al40 | 1223 | 2001-2013 | chP1 (60), chP2 (21), AP (12), BC (7), progression to AP/BC (19) | TKI 1 (median, range 0–3) | 10–20 (at 1 y) | NA | chP 1 HSCT (vs non-HSCT inferior OS, HR 2.4) No difference chP2 and AP; BC trend favoring HSCT |
Life expectancy calculation in comparison to no HSCT (CIBMTR) |
| Niederwieser et al27 | 147 | 1990-2018 | Non-BC (75) BC (25) |
Prior TKI: 1G (27.2); 1G + 2G (13.6); 1G + 3G (0.7); 2G ± 3G (38.1); 3G (0.7) |
28 (at 15 y) 24 in BC (at 5 y) 24 non-BC (at 5 y) |
30% at 10 y and 26% at 15 y BP 24% 5 y Non-BP 31% 5 y |
OS 15 y 34% BP 30% 10 y; 30% 5 y non-BP 41% 10 y; 44% 5 y |
Bicentric retrospective study Long-term follow-up |
| Masouridi-Levrat et al50 | 383 | 2009-2013 | chP1 (38) AP > chP1 (45) BC (16) |
Prior TKI: dasatinib (40) or nilotinib (17) or sequential ± bosutinib/ponatinib (43) |
18 (at 1 y) 24% (at 5 y) No difference between 2G-TKIs |
RFS 40% (at 5 y) RI 29% (at 2 y) RI 36% (at 5 y) |
65% (at 2 y) 56% (at 5 y) chP1 67% AP/chP >1 57% BC 37% |
EBMT study, retrospective study |
CMR, complete molecular response; cord, cord blood; NA, not available; n.s., not significantly different; PCR, polymerase chain reaction; PFS, progression-free survival; RI, relapse incidence; sib, sibling; TFS, transformation-free survival.