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Update [Luspatercept - Beyond Trial for NDT]
dq:
Hi Andy,
So the Luspatercept trial for NDT is finally underway here in the UK.
I'm totally aware that the Placebo may be issued however I was wondering if you knew how many doses one needs to take before they see a hgb increase? Would hgb increases be generally seen after the first or second dose or does it take a few more before one sees any progress?
I'm having difficulty finding anything that shows 'dose to positive change' information.
Thanks mate.
Andy Battaglia:
Because of the way the results are reported at 12 week intervals, I can't tell you what to expect before then. I have to assume that there is a slow progression that starts early, but with enough change to make the 12 week measurement meaningful.
Than2499:
HI, I'm a Luspatercept candidate. Now, my trail is finished and the label was open, i've got "real" medicine. Unfortunately during a long the trail, my Hb does't increased or see any improvement. I've always believed i got placebo because my CBC seem not anything changed. After i take the trail, my Hb still low (pre transfusion Hb 7-8). According to protocol, if Hb not increased 4 months consecutive, dose will increase by 25% but still no any changed.
After 10-11 months of trail, i've got back pain problem. Finally i have MRI and found extramedullary hematopoiesis on my spine (T8), it compressed my spine and it cause lost of musculature strength on my lower half body. Right now i has been postpone the trail. My doctor told me i have terminated from the trail. It so sad to know the medicine does't work for me. T T ..sob sob
My doctor give me heavily blood transfusion until my Hb reached 13 and must keep this level, extramedullary hematopoiesis was gone and musculature strength is reversing. I have to have blood transfusion once a week (1-2units a week). My serum ferritin is crazy heavy loaded from 7,000 jump to 12,000 and even maximum dose of Exjade could not stop from flying up my SF.
I found my friends who got placebo once they have switched to real (open label), they have outstanding improve in Hb in just a few days. the common symptom is bone pain (hip) in a couple days and it gone after that Hb is jump up.
Hope my story may benefit you all
dq:
--- Quote from: Andy Battaglia on August 18, 2018, 08:11:35 PM ---Because of the way the results are reported at 12 week intervals, I can't tell you what to expect before then. I have to assume that there is a slow progression that starts early, but with enough change to make the 12 week measurement meaningful.
--- End quote ---
Hi Andy, thanks for the info mate. I will keep you updated either way. After dose 1 a slight increase was evident 6.8 to 7.4 however at dose 2 the level seemed to have gone back down to the original 6.8 number. The platelets have increased slightly. It was a little disappointing.. :(
dq:
--- Quote from: Than2499 on August 20, 2018, 05:34:04 AM ---HI, I'm a Luspatercept candidate. Now, my trail is finished and the label was open, i've got "real" medicine. Unfortunately during a long the trail, my Hb does't increased or see any improvement. I've always believed i got placebo because my CBC seem not anything changed. After i take the trail, my Hb still low (pre transfusion Hb 7-8). According to protocol, if Hb not increased 4 months consecutive, dose will increase by 25% but still no any changed.
After 10-11 months of trail, i've got back pain problem. Finally i have MRI and found extramedullary hematopoiesis on my spine (T8), it compressed my spine and it cause lost of musculature strength on my lower half body. Right now i has been postpone the trail. My doctor told me i have terminated from the trail. It so sad to know the medicine does't work for me. T T ..sob sob
My doctor give me heavily blood transfusion until my Hb reached 13 and must keep this level, extramedullary hematopoiesis was gone and musculature strength is reversing. I have to have blood transfusion once a week (1-2units a week). My serum ferritin is crazy heavy loaded from 7,000 jump to 12,000 and even maximum dose of Exjade could not stop from flying up my SF.
I found my friends who got placebo once they have switched to real (open label), they have outstanding improve in Hb in just a few days. the common symptom is bone pain (hip) in a couple days and it gone after that Hb is jump up.
Hope my story may benefit you all
--- End quote ---
Thank you so much for this information. Firstly, I'm sorry to Luspatercept didn't work out for you. Don't worry you have other options like Lentiglobin and in a few years maybe CRISPR. You also have Hydroxyurea.
I just wanted to ask you a few questions if you don't mind.
1. Prior to starting Luspatercept, were you originally Transfusion Dependant, meaning did you have regular transfusions or non transfusion dependant?
2. What was your haemoglobin before starting Luspatercept in general..? 7-8? or 13?
3. Did you have an MRI prior to starting Luspatercept to see if you already had slight Extrameduallary Haemopoiesis (EMH)?
4. Why did your doctor give you heavy transfusions? Was it temporary to relieve your EMH symptoms and then stopped to return to usual 7-8 level?
5. I'm not too sure by what you mean your trial is finished then your trial is terminated by your doctor?
6. I assume your Ferritin went up because of the heavy transfusions?
7. What outstanding improvement did they see? 1hgb? 1.5hgb? How many doses did it take to work? How long did it last before it went down again?
8. What do you mean hip pain in a couple of days? Do you mean after every dose or just once at the start of the trial?
9. Did you check your Uric Acid Levels? Did they decrease?
10. Finally, what about your spleen? Did it decrease in size or was it normal size in the first place?
Thank you so much for your help. This will help everyone on the trial..!
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