Table 1:
Baseline characteristics
| Original | Expansion | Total | |
|---|---|---|---|
| N=35 | N=50 | N=85 | |
| Median age (IQR), years | 55 (9.5) | 54 (7) | 55 (8) |
| Men, n (%) | 25 (71) | 31 (62) | 56 (65·9) |
| Women | 10 (29) | 19 (38) | 29 (34·1) |
| ECOG performance status, n (%) | |||
| 0 | 23 (66) | 27 (54) | 50 (58·8) |
| 1 | 12 (34) | 23 (46) | 35 (41·2) |
| Rai stage, n (%) | |||
| 0 | 1 (2·9) | 0 (0) | 1 (1·2) |
| 1 | 6 (17·1) | 14 (28) | 20 (23·5) |
| 2 | 8 (22·9) | 13 (26) | 21 (24·7) |
| 3 | 4 (11·4) | 7 (14) | 11 (12·9) |
| 4 | 16 (45·7) | 13 (26) | 29 (34·1) |
| Median WBC (IQR), × 109/L | 78 (146·7) | 115 (136·7) | 109 (146·5) |
| Median haemoglobin (IQR), g/dL | 12 (3·2) | 11 (3·3) | 12 (3·3) |
| Median haematocrit (IQR), % | 37 (9·6) | 35 (9·2) | 37 (9·7) |
| Median platelets (IQR), × 109/L | 141 (92) | 127 (74) | 127 (79) |
| Median BM involvement (IQR), % | 47 (37·5) | 77 (31·3) | 60 (40) |
| Median IgG (IQR), mg/dL | 753 (481) | 631 (425) | 676 (463·3) |
| Median IgA (IQR), mg/dL | 74 (52·8) | 79 (47) | 77 (53·5) |
| Median β2-microglobulin (IQR), mg/L | 4 (1·9) | 2 (1·9) | 3·0 (2·2) |
| IGHV unmutated | 21/32 (65·6) | 25/47 (53·2) | 46/79 (58·2%) |
| ZAP-70 positive | 21/33 (63·6) | 22/43 (51·1) | 43/76 (56·6%) |
| del(17p) | 4/33 (12·1) | 0/50 (0) | 4/83 (4·8%) |
| del(11q) | 9/33 (27·3) | 8/50 (16) | 17/83 (20·5%) |
| del(13q) | 17/33 (51·5) | 28/50 (56) | 45/83 (54·2%) |
| Normal FISH | 5/33 (15·2) | 9/50 (18) | 14/82 (17·1%) |
| Trisomy 12 | 6/33 (18·2) | 8/50 (16) | 14/83 (16·9%) |
| 6q detected | 1/33 (3·0) | 1/50 (2) | 2/82 (2·4%) |
| Complex cytogenetics | 14/83 (16·9%) | ||
| Including del(17p) | 3/33 (9·1) | 1/50 (2) | 4/83 (4·8%) |
| Without del(17p) | 4/33 (12·1) | 6/50 (12) | 10/83 (12·1%) |
| TP53 mutation | 2/33 (6·1) | 1/48(2·1) | 3/81 (3·7%) |
| NOTCH1 mutation | 2/27 (7·4) | 3/31 (9·7) | 5/58 (8·6%) |
BM=bone marrow. ECOG=Eastern Cooperative Oncology Group. FISH=fluorescence in situ hybridisation, non-hierarchical. Ig=immunoglobulin. IGHV=immunoglobulin heavy-chain variable region. WBC=white blood cell.