Thoughts on the appendix

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biopearl123
Posts: 1665
Joined: Fri Jul 20, 2018 5:13 pm

Thoughts on the appendix

Post by biopearl123 » Wed Dec 18, 2019 12:23 am

No not that appendix, this one: https://www.nejm.org/doi/suppl/10.1056/ ... pendix.pdf

As part of the 2015 Tefferi article in the NEJM the 4 CR or PR patients were presented in the appendix. It is worth a read. And yes Bill, for unknown reasons Tefferi "damned with faint praise" Imetelstat with an off the cuff remark in Italy at a conference. I don't know why but I don't think is relationship with the company was at its best then. Had to know. Anyway Patient I: Had transfusion requirement every one to two weeks, palp spleen. 5 weeks post Rx becomes TI, eventually spleen resolves, bone marrow normalizes, no mutations detected, LDH and Hgb normalize. Patient II: 7 months into therapy leukoerythroblastosis disappears, LDH normalizes No fibrosis on bone marrow, JAK 2 burden drops to 1 %. Patient 3: Needed transfusion q 4 weeks--resolved,itching resolved, LDH normalizes. Pt 4: 3 weeks into therapy complete resolution of Luekoerythroblastosis, normalization of spleen and normalization of LDH. AFAIK these types of improvements NEVER occur spontaneously in MF.
These results were published in the NEJM as above. AFAIK, the validity of the study was never called into questions and Dr. Tefferi remains a preeminent physician in his field. I believe (correct me if I am wrong) the median duration of remission was 18 months. These are small numbers but much more experience with dosing exists today. The ongoing study of course looked only at R/R patients and showed impressive improvements in MOS using a RWD control (the potential weakness of this study). Given the bulk of the accumulated data I think a strong case will be made to the FDA.

rccola335
Posts: 305
Joined: Sat Sep 28, 2019 10:00 pm

Re: Thoughts on the appendix

Post by rccola335 » Wed Dec 18, 2019 3:00 am

thanks for the link - hopefully we get some good news - I am expecting approval for a phase 3 but hoping for early approval

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