Detailed information on treatment and outcome for the entire HLH-2004 cohort, for subgroups of the HLH-2004 cohort, and for comparison data from HLH-94 study
| . | HLH-2004 . | . | ||||
|---|---|---|---|---|---|---|
| . | n = 369 . | Verified FHL,* n = 168 . | Not verified FHL, n = 201 . | HLH-94 criteria,† n = 240 . | HLH-2004 criteria only,‡ n = 129 . | HLH-94 n = 232 . |
| Clinical status at 2 mo | ||||||
| Dead at 2 mo (% of all) | 50 (14) | 19 (11) | 31 (15) | 27 (11) | 23 (18) | 33 (14) |
| HSCT during the first 2 mo (% of all) | 15 (4) | 12 (7) | 3 (1) | 12 (5) | 3 (2) | 3 (1) |
| Achieved a resolution, had reactivation (% of all)§ | 19 (5) | 8 (5) | 11 (5) | 15 (6) | 4 (3) | 18 (8) |
| Treatment according to protocol|| | ||||||
| Etoposide according to plan weeks 1-2 | ||||||
| Yes | 254 (83) | 114 (84) | 140 (81) | 166 (81) | 88 (85) | 176 (86) |
| No | 53 (17) | 21 (16) | 32 (19) | 38 (19) | 15 (15) | 29 (14) |
| Missing | 62 (17) | 33 (20) | 29 (14) | 36 (15) | 26 (20) | 27 (12) |
| Etoposide according to plan weeks 3-8 | ||||||
| Yes | 214 (72) | 93 (73) | 121 (72) | 140 (70) | 74 (78) | 148 (74) |
| No | 82 (28) | 35 (27) | 47 (28) | 61(30) | 21 (22) | 52 (26) |
| Missing | 73 (20) | 40 (24) | 33 (16) | 39 (16) | 34 (26) | 32 (14) |
| Dexamethasone according to plan weeks 1-8 | ||||||
| Yes | 254 (83) | 109 (81) | 145 (85) | 163 (80) | 91 (89) | 171 (85) |
| No | 52 (17) | 26 (19) | 26 (15) | 41 (20) | 11 (11) | 31 (15) |
| Missing | 63 (17) | 33 (20) | 30 (15) | 36 (15) | 27 (21) | 30 (13) |
| CSA according to plan weeks 1-8 | ||||||
| Yes | 261 (85) | 114 (84) | 147 (85) | 170 (83) | 91 (88) | N.A. |
| No | 46 (15) | 21 (16) | 25 (15) | 34 (17) | 12 (12) | N.A. |
| Missing | 62 (17) | 33 (20) | 29 (14) | 36 (15) | 26 (20) | N.A. |
| IT therapy administrated 0-2 mo | ||||||
| Yes | 71 (24) | 52 (39) | 19 (12) | 57 (29) | 14 (14) | 58 (28) |
| No | 227 (76) | 81 (61) | 146 (88) | 141 (71) | 86 (86) | 147 (72) |
| Missing | 71 (19) | 35 (21) | 36 (18) | 42 (18) | 29 (22) | 27 (12) |
| . | HLH-2004 . | . | ||||
|---|---|---|---|---|---|---|
| . | n = 369 . | Verified FHL,* n = 168 . | Not verified FHL, n = 201 . | HLH-94 criteria,† n = 240 . | HLH-2004 criteria only,‡ n = 129 . | HLH-94 n = 232 . |
| Clinical status at 2 mo | ||||||
| Dead at 2 mo (% of all) | 50 (14) | 19 (11) | 31 (15) | 27 (11) | 23 (18) | 33 (14) |
| HSCT during the first 2 mo (% of all) | 15 (4) | 12 (7) | 3 (1) | 12 (5) | 3 (2) | 3 (1) |
| Achieved a resolution, had reactivation (% of all)§ | 19 (5) | 8 (5) | 11 (5) | 15 (6) | 4 (3) | 18 (8) |
| Treatment according to protocol|| | ||||||
| Etoposide according to plan weeks 1-2 | ||||||
| Yes | 254 (83) | 114 (84) | 140 (81) | 166 (81) | 88 (85) | 176 (86) |
| No | 53 (17) | 21 (16) | 32 (19) | 38 (19) | 15 (15) | 29 (14) |
| Missing | 62 (17) | 33 (20) | 29 (14) | 36 (15) | 26 (20) | 27 (12) |
| Etoposide according to plan weeks 3-8 | ||||||
| Yes | 214 (72) | 93 (73) | 121 (72) | 140 (70) | 74 (78) | 148 (74) |
| No | 82 (28) | 35 (27) | 47 (28) | 61(30) | 21 (22) | 52 (26) |
| Missing | 73 (20) | 40 (24) | 33 (16) | 39 (16) | 34 (26) | 32 (14) |
| Dexamethasone according to plan weeks 1-8 | ||||||
| Yes | 254 (83) | 109 (81) | 145 (85) | 163 (80) | 91 (89) | 171 (85) |
| No | 52 (17) | 26 (19) | 26 (15) | 41 (20) | 11 (11) | 31 (15) |
| Missing | 63 (17) | 33 (20) | 30 (15) | 36 (15) | 27 (21) | 30 (13) |
| CSA according to plan weeks 1-8 | ||||||
| Yes | 261 (85) | 114 (84) | 147 (85) | 170 (83) | 91 (88) | N.A. |
| No | 46 (15) | 21 (16) | 25 (15) | 34 (17) | 12 (12) | N.A. |
| Missing | 62 (17) | 33 (20) | 29 (14) | 36 (15) | 26 (20) | N.A. |
| IT therapy administrated 0-2 mo | ||||||
| Yes | 71 (24) | 52 (39) | 19 (12) | 57 (29) | 14 (14) | 58 (28) |
| No | 227 (76) | 81 (61) | 146 (88) | 141 (71) | 86 (86) | 147 (72) |
| Missing | 71 (19) | 35 (21) | 36 (18) | 42 (18) | 29 (22) | 27 (12) |
In HLH-2004, verified FHL was defined as having either biallelic mutations in FHL-causative genes (PRF1, UNC13D, STX11, and STXBP2) or an affected sibling.
Patients within the HLH-2004 study who fulfilled the HLH-94 criteria (5 of 5).
Patients within the HLH-2004 who only fulfilled the HLH-2004 criteria (5 of 8) but not the HLH-94 criteria (5 of 5).
As reported by the treating physicians.
For the yes/no columns, the percentage is given without including patients with missing data or not applicable patients. For the missing/not applicable columns, the percentage is given for the whole population. Regarding “Etoposide according to plan weeks 3-8,” missing data includes the not applicable data on patients who died weeks 1 to 2.