Patient characteristics and follow-up of patients with HCL treated with vemurafenib
| Patient . | Age, y . | Lines prior treatment . | Treatment duration, d . | Starting dose, mg . | Dose escalation . | Cumulative dose, mg . | Response . | Hairy cells after BRAFi in BM, % . | Spleen before BRAFi, cm . | Spleen after BRAFi, cm . | Side effects . | Follow-up, mo . | Comments . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Heidelberg 01 | 51 | 3 | 56 | 2 × 240 | 2 × 960 mg/d | 68 640 | CR | 0 | 25 | 13 | Arthralgia, elevated LFTs | 38 | Retreatment |
| Heidelberg 02 | 60 | 4 | 86 | 2 × 240 | 2 × 720 mg/d | 113 760 | PR | 10 | 17 | 15 | Arthralgia, elevated LFTs | 21 | Retreatment |
| Heidelberg 03 | 56 | 4 | 91 | 2 × 240 | No | 43 680 | PR | 40 | 22 | 10 | Squamous cell carcinoma | 26 | Retreatment |
| Heidelberg 04 | 69 | 3 | 89 | 2 × 240 | No | 42 720 | CR | 0 | 16 | 12 | No major | 17 | |
| Heidelberg 05 | 59 | 3 | 129 | 2 × 240 | No | 61 920 | NA | Not enlarged | Keratoacanthoma, arthralgia, skin phototoxicity | 14 | Retreatment | ||
| Heidelberg 06 | 45 | 1 | 88 | 2 × 240 | No | 42 240 | PR | 40 | 18 | 15 | No major | 11 | |
| Nice 01 | 74 | 2 | 90 | 2 × 240 | No | 43 200 | NA | NA | No major | 25 | Retreatment | ||
| Nice 02 | 49 | 0 | 57 | 2 × 240 | No | 27 360 | NA | 21 | 13 | No major | 15 | Retreatment | |
| Cambridge | 68 | 4 | 58 | 2 × 240 | No | 27 840 | NA | Splenectomy | Elevated LFTs, keratoacanthoma | 31 | Retreatment | ||
| Erfurt | 51 | 4 | 106 | 2 × 240 | No | 50 880 | PR | 40 | 6 cm BCM | Not palpable | No major | 17 | |
| Freiburg | 62 | 3 | 85 | 2 × 960 | No | 81 600 | CR | 0 | NA | No major | 9 | ||
| Lucerne | 50 | 5 | 56 | 2 × 240 | No | 26 880 | MR | 40 | Splenectomy | AML-M6 | 17 | Death | |
| Innsbruck 01 | 79 | 3 | 104 | 2 × 240 | 2 × 480 mg/d | 96 480 | CR | 0 | 18 | 12 | No major | 20 | |
| Innsbruck 02 | 69 | 0 | 142 | 2 × 480 | No | 68 160 | PR | 8 | 14 | 12 | Elevated LFTs, keratoacanthoma | 17 | Retreatment |
| Innsbruck 03 | 71 | 1 | 108 | 2 × 480 | No | 51 840 | CR | 0 | 12 | 12 | No major | 10 | |
| Innsbruck 04 | 67 | 12 | 112 | 1 × 240 | No | 26 880 | NA | 15 | 13 | Skin phototoxicity | 8 | Retreatment | |
| Cologne | 65 | 5 | 83 | 2 × 240 | No | 39 840 | PR | 5 | Splenectomy | No major | 10 | ||
| Leicester | 72 | 5 | 167 | 2 × 240 | No | 80 160 | PR | 4 | Splenectomy | Alopecia, squamous cell papilloma of the skin, skin phototoxicity | 10 | Death | |
| Schwabing 1 | 61 | 1 | 266 | 2 × 240 | 2 × 720 mg/d | 264 240 | CR | 0 | 18 | 12 | No major | 10 | |
| Schwabing 2 | 89 | 2 | 68 | 2 × 240 | No | 32 640 | NA | NA | Acute on chronic renal failure, exicosis | 3 | Death | ||
| London | 48 | 6 | 167 | 2 × 240 | 2 × 960 mg/d | 310 560 | PR | 10 | Splenectomy | Arthralgias, skin phototoxicity | 20 |
| Patient . | Age, y . | Lines prior treatment . | Treatment duration, d . | Starting dose, mg . | Dose escalation . | Cumulative dose, mg . | Response . | Hairy cells after BRAFi in BM, % . | Spleen before BRAFi, cm . | Spleen after BRAFi, cm . | Side effects . | Follow-up, mo . | Comments . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Heidelberg 01 | 51 | 3 | 56 | 2 × 240 | 2 × 960 mg/d | 68 640 | CR | 0 | 25 | 13 | Arthralgia, elevated LFTs | 38 | Retreatment |
| Heidelberg 02 | 60 | 4 | 86 | 2 × 240 | 2 × 720 mg/d | 113 760 | PR | 10 | 17 | 15 | Arthralgia, elevated LFTs | 21 | Retreatment |
| Heidelberg 03 | 56 | 4 | 91 | 2 × 240 | No | 43 680 | PR | 40 | 22 | 10 | Squamous cell carcinoma | 26 | Retreatment |
| Heidelberg 04 | 69 | 3 | 89 | 2 × 240 | No | 42 720 | CR | 0 | 16 | 12 | No major | 17 | |
| Heidelberg 05 | 59 | 3 | 129 | 2 × 240 | No | 61 920 | NA | Not enlarged | Keratoacanthoma, arthralgia, skin phototoxicity | 14 | Retreatment | ||
| Heidelberg 06 | 45 | 1 | 88 | 2 × 240 | No | 42 240 | PR | 40 | 18 | 15 | No major | 11 | |
| Nice 01 | 74 | 2 | 90 | 2 × 240 | No | 43 200 | NA | NA | No major | 25 | Retreatment | ||
| Nice 02 | 49 | 0 | 57 | 2 × 240 | No | 27 360 | NA | 21 | 13 | No major | 15 | Retreatment | |
| Cambridge | 68 | 4 | 58 | 2 × 240 | No | 27 840 | NA | Splenectomy | Elevated LFTs, keratoacanthoma | 31 | Retreatment | ||
| Erfurt | 51 | 4 | 106 | 2 × 240 | No | 50 880 | PR | 40 | 6 cm BCM | Not palpable | No major | 17 | |
| Freiburg | 62 | 3 | 85 | 2 × 960 | No | 81 600 | CR | 0 | NA | No major | 9 | ||
| Lucerne | 50 | 5 | 56 | 2 × 240 | No | 26 880 | MR | 40 | Splenectomy | AML-M6 | 17 | Death | |
| Innsbruck 01 | 79 | 3 | 104 | 2 × 240 | 2 × 480 mg/d | 96 480 | CR | 0 | 18 | 12 | No major | 20 | |
| Innsbruck 02 | 69 | 0 | 142 | 2 × 480 | No | 68 160 | PR | 8 | 14 | 12 | Elevated LFTs, keratoacanthoma | 17 | Retreatment |
| Innsbruck 03 | 71 | 1 | 108 | 2 × 480 | No | 51 840 | CR | 0 | 12 | 12 | No major | 10 | |
| Innsbruck 04 | 67 | 12 | 112 | 1 × 240 | No | 26 880 | NA | 15 | 13 | Skin phototoxicity | 8 | Retreatment | |
| Cologne | 65 | 5 | 83 | 2 × 240 | No | 39 840 | PR | 5 | Splenectomy | No major | 10 | ||
| Leicester | 72 | 5 | 167 | 2 × 240 | No | 80 160 | PR | 4 | Splenectomy | Alopecia, squamous cell papilloma of the skin, skin phototoxicity | 10 | Death | |
| Schwabing 1 | 61 | 1 | 266 | 2 × 240 | 2 × 720 mg/d | 264 240 | CR | 0 | 18 | 12 | No major | 10 | |
| Schwabing 2 | 89 | 2 | 68 | 2 × 240 | No | 32 640 | NA | NA | Acute on chronic renal failure, exicosis | 3 | Death | ||
| London | 48 | 6 | 167 | 2 × 240 | 2 × 960 mg/d | 310 560 | PR | 10 | Splenectomy | Arthralgias, skin phototoxicity | 20 |
Spleen sizes were measured by imaging studies and were not considered enlarged if the largest diameter was <13 cm. No imaging studies were performed in 1 patient, for whom spleen sizes are reported in centimeters BCM, or not palpable.
BCM, below costal margin; BM, bone marrow; LFTs, liver function tests; MR, minor response; NA, not available; PR, partial remission.