Patient characteristics studied by IHC
| Age . | Gender . | KD mutation . | Previous TKIs . | Prior years of therapy . | %FGF2 preponatinib . | %FGF2 postponatinib . | Δ . |
|---|---|---|---|---|---|---|---|
| 56 | M | None | IM, DAS | 3 | 49.8 | 35.1 | −14.7 |
| 67 | M | None | IM, DAS, NIL | 2 | 46.4 | 24.9 | −21.5 |
| 31 | M | None | IM, DAS | 3 | 55.4 | 43.8 | −11.7 |
| 54 | M | L248V | IM, DAS, NIL | 2 | 26.6 | 40.0 | 13.4 |
| 68 | M | F317L/F359V | IM, DAS, NIL | 2 | 43.0 | 46.9 | 3.9 |
| 62 | M | F359V/M244V | IM, DAS, NIL, BOS | 2 | 36.6 | 30.2 | −6.5 |
| 70 | F | F359C | IM, NIL | 4 | 40.1 | 37.6 | −2.5 |
| 47 | M | T315I | IM, DAS | 3 | 22.7 | 37.6 | 15.0 |
| 53 | M | T315I | IM, DAS | 4 | 41.3 | 36.8 | −4.5 |
| 45 | M | T315I | IM, DAS | 3 | 30.6 | 21.6 | −9.1 |
| 59 | F | T315I | IM, DAS | 4 | 49.8 | 40.0 | −9.8 |
| Nonresponder | |||||||
| 37 | F | None | IM, DAS, NIL | 4 | 19.2 | 19 | 0.2 |
| Age . | Gender . | KD mutation . | Previous TKIs . | Prior years of therapy . | %FGF2 preponatinib . | %FGF2 postponatinib . | Δ . |
|---|---|---|---|---|---|---|---|
| 56 | M | None | IM, DAS | 3 | 49.8 | 35.1 | −14.7 |
| 67 | M | None | IM, DAS, NIL | 2 | 46.4 | 24.9 | −21.5 |
| 31 | M | None | IM, DAS | 3 | 55.4 | 43.8 | −11.7 |
| 54 | M | L248V | IM, DAS, NIL | 2 | 26.6 | 40.0 | 13.4 |
| 68 | M | F317L/F359V | IM, DAS, NIL | 2 | 43.0 | 46.9 | 3.9 |
| 62 | M | F359V/M244V | IM, DAS, NIL, BOS | 2 | 36.6 | 30.2 | −6.5 |
| 70 | F | F359C | IM, NIL | 4 | 40.1 | 37.6 | −2.5 |
| 47 | M | T315I | IM, DAS | 3 | 22.7 | 37.6 | 15.0 |
| 53 | M | T315I | IM, DAS | 4 | 41.3 | 36.8 | −4.5 |
| 45 | M | T315I | IM, DAS | 3 | 30.6 | 21.6 | −9.1 |
| 59 | F | T315I | IM, DAS | 4 | 49.8 | 40.0 | −9.8 |
| Nonresponder | |||||||
| 37 | F | None | IM, DAS, NIL | 4 | 19.2 | 19 | 0.2 |
BOS, bosutinib; TKI, tyrosine kinase inhibitor.
Bone marrow biopsies from patients who responded to ponatinib were evaluated both before therapy and after response to ponatinib. A single patient who developed resistance to ponatinib within 6 months of therapy is shown for comparison. The clinical characteristics are listed as well as the quantitation of the FGF2 present in the marrow by Aperio ImageScope software (supplemental Figure 5).