Kaplan-Meier estimates of OS and EFS for all evaluated children, and for children with and without verified FHL
| Variable . | All children (N = 187), 5-y pSu (95% CI) (no. evaluated) (no. events) . | Children with FHL (n = 134), 5-y pSu (95% CI) (no. evaluated) (no. events) . | Children without verified FHL (n = 53), 5-y pSu (95% CI) (no. evaluated) (no. events) . | |||
|---|---|---|---|---|---|---|
| OS . | EFS . | OS . | EFS . | OS . | EFS . | |
| General | 66 (59-72) (187) (67) | 60 (52-67) (187) (78) | 71 (62-78) (134) (43) | 62 (54-70) (134) (54) | 52 (38-65) (53) (24) | 52 (38-65) (53) (24) |
| Female | 68 (57-76) (97) (32) | 62 (52-71) (97) (38) | 75 (63-83) (73) (19) | 68 (56-77) (73) (25) | 44 (24-63) (24) (13) | 44 (24-63) (24) (13) |
| Male | 64 (53-73) (90) 35) | 57 (46-66) (90) (40) | 66 (52-76) (61) (24) | 55 (41-67) (61) (29) | 61 (40-76) (29) (11) | 61 (40-76) (29) (11) |
| Complete response (ferritin <500 µg/L)* | 81 (65-90) (42) (10) | 63 (47-76) (42 (17) | 83 (64-93) (30) (7) | 59 (39-75) (30) (14) | 75 (41-91) (12) (3) | 75 (41-91) (12) (3) |
| Partial response (ferritin <500 µg/L)† | 59 (48-69) (87) (37) | 56 (44-65) (87) (40) | 64 (51-75) (65) (25) | 59 (47-70) (65) (28) | 43 (21-62) (22) (12) | 43 (21-62) (22) (12) |
| Complete response (ferritin <2000 µg/L)‡ | 76 (62-85) (55) (15) | 61 (47-73) (55) (23) | 82 (65-91) (38) (9) | 60 (43-74) (38) (17) | 64 (36-82) (17) (6) | 64 (36-82) (17) (6) |
| Partial response (ferritin <2000 µg/L)§ | 61 (49-71) (76) (31) | 59 (47-69) (76) (33) | 65 (51-76) (58) (22) | 62 (48-73) (58) (24) | 48 (24-69) (18) (9) | 48 (24-69) (18) (9) |
| No neurologic symptoms | 69 (59-76) (120) (38) | 61 (52-70) (120) (46) | 74 (63-82) (84) (23) | 63 (52-73) (84) (31) | 57 (39-71) (36) (15) | 57 (39-71) (36) (15) |
| Neurologic symptoms | 67 (47-80) (30) (13) | 60 (41-75) (30) (15) | 71 (48-85) (24) (10) | 63 (40-78) (24) (12) | 50 (11-80) (6) (3) | 50 (11-80) (6) (3) |
| CSF cells or protein normal‖ | 63 (44-78) (30) (11) | 60 (41-75) (30) (12) | 68 (43-84) (19) (6) | 63 (38-81) (19) (7) | 55 (23-78) (11) (5) | 55 (23-78) (11) (5) |
| CSF cells or protein pathologic‖ | 62 (40-78) (24) (11) | 49 (28-67) (24) (14) | 71 (43-87) (17) (7) | 53 (28-73) (17) (10) | 38 (6-72) (7) (4) | 38 (6-72) (7) (4) |
| Age at HSCT conditioning <1 y | 67 (56-76) (91) (32) | 62 (51-73) (91) 836) | 69 (58-78) (78) (26) | 64 (52-73) (78) (30) | 54 (25-76) (13) (6) | 54 (25-76) (13) (6) |
| Age at HSCT conditioning 1-2.99 y | 71 (57-81) (60) (19) | 59 (45-70) (60) (26) | 80 (63-90) (37) (9) | 60 (43-74) (37) (16) | 55 (33-73) (23) (10) | 55 (33-73) (23) (10) |
| Age at HSCT conditioning 3-5.99 y | 60 (32-80) (15) (6) | 60 (32-80) (15) (6) | 56 (20-81) (9) (4) | 56 (20-81) (9) (4) | 67 (20-90) (6) (2) | 67 (20-90) (6) (2) |
| Age at HSCT conditioning ≥6 y | 51 (28-70) (21) (10) | 51 (28-70) (21) (10) | 60 (25-83) (10) (4) | 60 (25-83) (10) (4) | 44 (15-70) (11) (6) | 44 (15-70) (11) (6) |
| Time to HSCT <6 mo | 69 (60-76) (120) (40) | 64 (55-72) (120) (45) | 70 (60-78) (94) (31) | 64 (54-73) (94) (36) | 65 (43-80) (26) (9) | 65 (43-80) (26) (9) |
| Time to HSCT ≥6 mo | 60 (47-71) (67) (27) | 51 (38-62) (67) (33) | 72 (55-83) (40) (12) | 57 (40-70) (40) (18) | 41 (22-59) (27) (15) | 41 (22-59) (27) (15) |
| HSCT time interval 2004-2006 | 70 (56-80) (54) (17) | 61 (46-72) (54) (22) | 73 (56-84) (37) (11) | 59 (42-73) (37) (16) | 63 (35-82) (17) (6) | 63 (35-82) (17) (6) |
| HSCT time interval 2007-2009 | 60 (48-70) 875) (32) | 55 (43-66) (75) (34) | 65 (51-76) (52) (20) | 59 (44-71) (52) 22) | 48 (27-66) (23) (12) | 48 (27-66) (23) (12) |
| HSCT time interval 2010-2012 | 70 (56-80) (58) (18) | 65 (51-76) (58) (22) | 75 (60-86) (45) (12) | 69 (53-80) (45) (16) | 50 (21-74) (13) (6) | 50 (21-74) (13) (6) |
| Donor: MRD | 58 (42-71) (44) (19) | 54 (38-67) (44) (21) | 64 (45-78) (31 (12) | 58 (38-73) (31) (14) | 44 (17-69) (13) (7) | 44 (17-69) (13) (7) |
| Donor: MUD | 73 (60-83) (60) (18) | 62 (48-73) (60) (24) | 78 (63-88) (45) (12) | 65 (48-77) (45) (18) | 55 (26-77) (15) (6) | 55 (26-77) (15) (6) |
| Donor: UCB | 60 (45-72) (53) (22) | 58 (44-70) (53) (23) | 69 (52-81) (39) (13) | 66 (49-79) (39) (14) | 38 (13-59) (14) (9) | 38 (13-59) (14) (9) |
| Conditioning busulfan-based | 63 (53-72) (99) (38) | 59 (48-68) (99) (42) | 70 (58-79) (69) (23) | 64 (51-74) (69) (27) | 46 (27-63) (30) (15) | 46 (27-63) (30) (15) |
| Conditioning fludarabine-based | 69 (51-81) (39) (13) | 50 (32-66) (39) (18) | 76 (55-89) (26) (7) | 50 (28-68) (26) (12) | 53 (23-75) (13) (6) | 53 (23-75) (13) (6) |
| Conditioning treosulfan-based | 80 (55-92) (20) (4) | 80 (55-92) (20) (5) | 78 (51-91) (18) (4) | 78 (51-91) (18) (5) | 100 (—) (2) (0) | 100 (—) (2) (0) |
| Variable . | All children (N = 187), 5-y pSu (95% CI) (no. evaluated) (no. events) . | Children with FHL (n = 134), 5-y pSu (95% CI) (no. evaluated) (no. events) . | Children without verified FHL (n = 53), 5-y pSu (95% CI) (no. evaluated) (no. events) . | |||
|---|---|---|---|---|---|---|
| OS . | EFS . | OS . | EFS . | OS . | EFS . | |
| General | 66 (59-72) (187) (67) | 60 (52-67) (187) (78) | 71 (62-78) (134) (43) | 62 (54-70) (134) (54) | 52 (38-65) (53) (24) | 52 (38-65) (53) (24) |
| Female | 68 (57-76) (97) (32) | 62 (52-71) (97) (38) | 75 (63-83) (73) (19) | 68 (56-77) (73) (25) | 44 (24-63) (24) (13) | 44 (24-63) (24) (13) |
| Male | 64 (53-73) (90) 35) | 57 (46-66) (90) (40) | 66 (52-76) (61) (24) | 55 (41-67) (61) (29) | 61 (40-76) (29) (11) | 61 (40-76) (29) (11) |
| Complete response (ferritin <500 µg/L)* | 81 (65-90) (42) (10) | 63 (47-76) (42 (17) | 83 (64-93) (30) (7) | 59 (39-75) (30) (14) | 75 (41-91) (12) (3) | 75 (41-91) (12) (3) |
| Partial response (ferritin <500 µg/L)† | 59 (48-69) (87) (37) | 56 (44-65) (87) (40) | 64 (51-75) (65) (25) | 59 (47-70) (65) (28) | 43 (21-62) (22) (12) | 43 (21-62) (22) (12) |
| Complete response (ferritin <2000 µg/L)‡ | 76 (62-85) (55) (15) | 61 (47-73) (55) (23) | 82 (65-91) (38) (9) | 60 (43-74) (38) (17) | 64 (36-82) (17) (6) | 64 (36-82) (17) (6) |
| Partial response (ferritin <2000 µg/L)§ | 61 (49-71) (76) (31) | 59 (47-69) (76) (33) | 65 (51-76) (58) (22) | 62 (48-73) (58) (24) | 48 (24-69) (18) (9) | 48 (24-69) (18) (9) |
| No neurologic symptoms | 69 (59-76) (120) (38) | 61 (52-70) (120) (46) | 74 (63-82) (84) (23) | 63 (52-73) (84) (31) | 57 (39-71) (36) (15) | 57 (39-71) (36) (15) |
| Neurologic symptoms | 67 (47-80) (30) (13) | 60 (41-75) (30) (15) | 71 (48-85) (24) (10) | 63 (40-78) (24) (12) | 50 (11-80) (6) (3) | 50 (11-80) (6) (3) |
| CSF cells or protein normal‖ | 63 (44-78) (30) (11) | 60 (41-75) (30) (12) | 68 (43-84) (19) (6) | 63 (38-81) (19) (7) | 55 (23-78) (11) (5) | 55 (23-78) (11) (5) |
| CSF cells or protein pathologic‖ | 62 (40-78) (24) (11) | 49 (28-67) (24) (14) | 71 (43-87) (17) (7) | 53 (28-73) (17) (10) | 38 (6-72) (7) (4) | 38 (6-72) (7) (4) |
| Age at HSCT conditioning <1 y | 67 (56-76) (91) (32) | 62 (51-73) (91) 836) | 69 (58-78) (78) (26) | 64 (52-73) (78) (30) | 54 (25-76) (13) (6) | 54 (25-76) (13) (6) |
| Age at HSCT conditioning 1-2.99 y | 71 (57-81) (60) (19) | 59 (45-70) (60) (26) | 80 (63-90) (37) (9) | 60 (43-74) (37) (16) | 55 (33-73) (23) (10) | 55 (33-73) (23) (10) |
| Age at HSCT conditioning 3-5.99 y | 60 (32-80) (15) (6) | 60 (32-80) (15) (6) | 56 (20-81) (9) (4) | 56 (20-81) (9) (4) | 67 (20-90) (6) (2) | 67 (20-90) (6) (2) |
| Age at HSCT conditioning ≥6 y | 51 (28-70) (21) (10) | 51 (28-70) (21) (10) | 60 (25-83) (10) (4) | 60 (25-83) (10) (4) | 44 (15-70) (11) (6) | 44 (15-70) (11) (6) |
| Time to HSCT <6 mo | 69 (60-76) (120) (40) | 64 (55-72) (120) (45) | 70 (60-78) (94) (31) | 64 (54-73) (94) (36) | 65 (43-80) (26) (9) | 65 (43-80) (26) (9) |
| Time to HSCT ≥6 mo | 60 (47-71) (67) (27) | 51 (38-62) (67) (33) | 72 (55-83) (40) (12) | 57 (40-70) (40) (18) | 41 (22-59) (27) (15) | 41 (22-59) (27) (15) |
| HSCT time interval 2004-2006 | 70 (56-80) (54) (17) | 61 (46-72) (54) (22) | 73 (56-84) (37) (11) | 59 (42-73) (37) (16) | 63 (35-82) (17) (6) | 63 (35-82) (17) (6) |
| HSCT time interval 2007-2009 | 60 (48-70) 875) (32) | 55 (43-66) (75) (34) | 65 (51-76) (52) (20) | 59 (44-71) (52) 22) | 48 (27-66) (23) (12) | 48 (27-66) (23) (12) |
| HSCT time interval 2010-2012 | 70 (56-80) (58) (18) | 65 (51-76) (58) (22) | 75 (60-86) (45) (12) | 69 (53-80) (45) (16) | 50 (21-74) (13) (6) | 50 (21-74) (13) (6) |
| Donor: MRD | 58 (42-71) (44) (19) | 54 (38-67) (44) (21) | 64 (45-78) (31 (12) | 58 (38-73) (31) (14) | 44 (17-69) (13) (7) | 44 (17-69) (13) (7) |
| Donor: MUD | 73 (60-83) (60) (18) | 62 (48-73) (60) (24) | 78 (63-88) (45) (12) | 65 (48-77) (45) (18) | 55 (26-77) (15) (6) | 55 (26-77) (15) (6) |
| Donor: UCB | 60 (45-72) (53) (22) | 58 (44-70) (53) (23) | 69 (52-81) (39) (13) | 66 (49-79) (39) (14) | 38 (13-59) (14) (9) | 38 (13-59) (14) (9) |
| Conditioning busulfan-based | 63 (53-72) (99) (38) | 59 (48-68) (99) (42) | 70 (58-79) (69) (23) | 64 (51-74) (69) (27) | 46 (27-63) (30) (15) | 46 (27-63) (30) (15) |
| Conditioning fludarabine-based | 69 (51-81) (39) (13) | 50 (32-66) (39) (18) | 76 (55-89) (26) (7) | 50 (28-68) (26) (12) | 53 (23-75) (13) (6) | 53 (23-75) (13) (6) |
| Conditioning treosulfan-based | 80 (55-92) (20) (4) | 80 (55-92) (20) (5) | 78 (51-91) (18) (4) | 78 (51-91) (18) (5) | 100 (—) (2) (0) | 100 (—) (2) (0) |
For OS and EFS, death, and death or second HSCT, were defined as events, respectively. All clinical parameters and laboratory values were analyzed at the time of HSCT conditioning.
—, cannot be calculated; CSF, cerebral spinal fluid; pSu, probability of survival.
Complete response was defined as no fever, no splenomegaly, ANC >1.0 × 109/L, platelets >100 × 109/L, fibrinogen >1.5 g/L, ferritin <500 µg/L; 6 of 6 or 5 of 5 (allowing missing information on 1 variable).
Partial response was defined as 3 to 5 of 6 normal parameters (fever, splenomegaly, ANC >1.0 × 109/L, platelets >100 × 109/L, fibrinogen >1.5 g/L, ferritin <500 µg/L), and the rest of the parameters abnormal.
Complete response was defined as no fever, no splenomegaly, ANC >1.0 × 109/L, platelets >100 × 109/L, fibrinogen >1.5 g/L, ferritin <2000 µg/L; 6 of 6 or 5 of 5 (allowing missing information on 1 variable).
Partial response was defined as 3 to 5 of 6 normal parameters (fever, splenomegaly, ANC >1.0 × 109/L, platelets >100 × 109/L, fibrinogen >1.5 g/L, ferritin <2000 µg/L), and the rest of the parameters abnormal.
Pathologic levels by age at HSCT conditioning.