AB
1Main
2Tefferi et al., New Engl. J. Med., 2011, 365, 1455-1457.
3Discussed 51 patients, out of 153, from Phase 1/2 study carried out at Mayo Clinic.
4Cited 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%)
5In comparison to a historic control of 410 patients, there was no meaningful survival benefit to drug.
6To do a meaningful OS study would take 5+ years.