Clinical and molecular characteristics of the distinct clinical phenotypic groups of ALK-positive histiocytosis and the overall cohort
| . | Group 1A (n = 6) . | Group 1B (n = 10) . | Group 2 (n = 23) . | Overall (n = 39) . |
|---|---|---|---|---|
| Age at presentation | ||||
| Median age (range) | 1.5 mo (0-5 mo) | 14.5 y (0-41 y) | 7 y (0-41 y) | 3 y (0-41 y) |
| Child | 6 (100%) | 5 (50%) | 20 (87%) | 31 (79%) |
| Adult | 5 (50%) | 3 (13%) | 8 (21%) | |
| Sex | ||||
| Female | 4 (67%) | 7 (70%) | 13 (57%) | 24 (62%) |
| Male | 2 (33%) | 3 (30%) | 10 (43%) | 15 (38%) |
| Organ involvement | ||||
| Nervous system | 7 (70%) | 12 (52%) | 19 (49%) | |
| Liver | 6 (100%) | 5 (50%) | 11 (28%) | |
| Lung | 1 (17%) | 7 (70%) | 1 (4%) | 9 (23%) |
| Bone | 7 (70%) | 2 (9%) | 9 (23%) | |
| Skin | 1 (17%) | 4 (40%) | 4 (17%) | 9 (23%) |
| Soft tissue | 3 (30%) | 4 (17%) | 7 (18%) | |
| Hematopoietic system | 6 (100%) | 6 (15%) | ||
| Spleen | 5 (83%) | 5 (13%) | ||
| Kidney | 2 (33%) | 2 (20%) | 4 (10%) | |
| Lymph node | 4 (40%) | 4 (10%) | ||
| Breast | 2 (20%) | 2 (5%) | ||
| Pancreas | 2 (20%) | 2 (5%) | ||
| Other* | 2 (20%) | 2 (5%) | ||
| ALK rearrangement | ||||
| KIF5B-ALK | 1 (17%) | 7 (70%) | 19 (83%) | 27 (69%) |
| CLTC-ALK | 1 (17%) | 1 (3%) | ||
| TPM3-ALK | 1 (10%) | 1 (3%) | ||
| TFG-ALK | 1 (10%) | 1 (3%) | ||
| EML4-ALK | 1 (4%) | 1 (3%) | ||
| DCTN1-ALK | 1 (4%) | 1 (3%) | ||
| ALK-FISH+ | 2 (33%) | 1 (10%) | 2 (9%) | 5 (13%) |
| Not confirmed | 2 (33%) | 2 (5%) | ||
| Median follow-up (range) | 3.75 y (0-7 y) | 2 y (0-6 y) | 14 mo (0-18 y) | 21 mo (0-18 y) |
| Status at last follow-up | ||||
| Alive with | ||||
| no evidence of disease | 4 (67%) | 5 (50%) | 14 (61%) | 23 (59%) |
| regressive disease | 4 (40%) | 5 (22%) | 9 (23%) | |
| stable disease | 1 (4%) | 1 (3%) | ||
| recent diagnosis | 1 (4%) | 1 (3%) | ||
| Dead | 1 (17%) | 1 (10%) | 2 (5%) | |
| Lost to follow-up | 1 (17%) | 2 (9%) | 3 (8%) |
| . | Group 1A (n = 6) . | Group 1B (n = 10) . | Group 2 (n = 23) . | Overall (n = 39) . |
|---|---|---|---|---|
| Age at presentation | ||||
| Median age (range) | 1.5 mo (0-5 mo) | 14.5 y (0-41 y) | 7 y (0-41 y) | 3 y (0-41 y) |
| Child | 6 (100%) | 5 (50%) | 20 (87%) | 31 (79%) |
| Adult | 5 (50%) | 3 (13%) | 8 (21%) | |
| Sex | ||||
| Female | 4 (67%) | 7 (70%) | 13 (57%) | 24 (62%) |
| Male | 2 (33%) | 3 (30%) | 10 (43%) | 15 (38%) |
| Organ involvement | ||||
| Nervous system | 7 (70%) | 12 (52%) | 19 (49%) | |
| Liver | 6 (100%) | 5 (50%) | 11 (28%) | |
| Lung | 1 (17%) | 7 (70%) | 1 (4%) | 9 (23%) |
| Bone | 7 (70%) | 2 (9%) | 9 (23%) | |
| Skin | 1 (17%) | 4 (40%) | 4 (17%) | 9 (23%) |
| Soft tissue | 3 (30%) | 4 (17%) | 7 (18%) | |
| Hematopoietic system | 6 (100%) | 6 (15%) | ||
| Spleen | 5 (83%) | 5 (13%) | ||
| Kidney | 2 (33%) | 2 (20%) | 4 (10%) | |
| Lymph node | 4 (40%) | 4 (10%) | ||
| Breast | 2 (20%) | 2 (5%) | ||
| Pancreas | 2 (20%) | 2 (5%) | ||
| Other* | 2 (20%) | 2 (5%) | ||
| ALK rearrangement | ||||
| KIF5B-ALK | 1 (17%) | 7 (70%) | 19 (83%) | 27 (69%) |
| CLTC-ALK | 1 (17%) | 1 (3%) | ||
| TPM3-ALK | 1 (10%) | 1 (3%) | ||
| TFG-ALK | 1 (10%) | 1 (3%) | ||
| EML4-ALK | 1 (4%) | 1 (3%) | ||
| DCTN1-ALK | 1 (4%) | 1 (3%) | ||
| ALK-FISH+ | 2 (33%) | 1 (10%) | 2 (9%) | 5 (13%) |
| Not confirmed | 2 (33%) | 2 (5%) | ||
| Median follow-up (range) | 3.75 y (0-7 y) | 2 y (0-6 y) | 14 mo (0-18 y) | 21 mo (0-18 y) |
| Status at last follow-up | ||||
| Alive with | ||||
| no evidence of disease | 4 (67%) | 5 (50%) | 14 (61%) | 23 (59%) |
| regressive disease | 4 (40%) | 5 (22%) | 9 (23%) | |
| stable disease | 1 (4%) | 1 (3%) | ||
| recent diagnosis | 1 (4%) | 1 (3%) | ||
| Dead | 1 (17%) | 1 (10%) | 2 (5%) | |
| Lost to follow-up | 1 (17%) | 2 (9%) | 3 (8%) |
Cervix, thyroid, salivary glands, and colorectum.