Hydrauxia vs. new gene therapy vs chronic transfusion

Andy
My 18 month baby's hbl drops to level 7 in three month interval since last transfusion.  Looks like within a month I will go for third transfusion.

Is there any comparison on risk benefits analysis on
1 wait for trial medicine from celgene
2. Try hydrauxia or any other inducer
3. Regular transfusion with chelation
4. Transfuse only when it drops to 7  and monitor
5. Not transfuse and wait to symptoms and then transfuse

Any idea how thevmrdicine in trial is different than hydrauxia.  Btw.  My baby growth chat is ok and she has not shown any symptoms.  We have transfused in every three months so far

I would suggest keeping her Hb above 9 with transfusions to assure proper growth. If you have a doctor who is familiar with using hydroxyurea, I would suggest giving it a try, along with folate and wheatgrass.

I would not wait for the new med to be approved. Phase 2 trials showed it works better than hydroxyurea. Luspatercept can be tried once available, but for a growing child, growth and development must be the first concern, so regular transfusion should be considered for now.

Thanks Andy. Doctor is closely watching as we are drawing every week. As she is not sick and active they are deciding what's the baseline she maintains.  Before deciding chronic transfusion or irregular transfusion or as needed transfusion. 

Andy,
'Is there any relationship with bluebird research and Agio pharmaceutical? 
Agio was in the process of some medicine related to blood disorder and they discontinued stock down 20%.  Just wondering if blue bird research and recruiting is intact.

No, the companies are not related. bluebird just presented a very positive report at the ASH conference earlier this month.

Thank you!