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| Tefferi et al., New Engl. J. Med., 2011, 365, 1455-1457. |
| Discussed 51 patients, out of 153, from Phase 1/2 study carried out at Mayo Clinic. |
| Cited discontinuation as an issue: Rates of treatment discontinuation at 1, 2, and 3 years were 51%, 72%, and 89%, respectively. Reasons for treatment discontinuation included disease progression or loss or lack of response (40%) and toxicity with or without disease progression or lack of response (34%) |
| In comparison to a historic control of 410 patients, there was no meaningful survival benefit to drug. |
| To do a meaningful OS study would take 5+ years. |