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Author Topic: Update [Luspatercept - Beyond Trial for NDT]  (Read 490 times)
dq
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« on: August 16, 2018, 05:11:43 PM »

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.
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Andy Battaglia
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« Reply #1 on: August 18, 2018, 03:11:35 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.
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Than2499
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« Reply #2 on: August 20, 2018, 12:34:04 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              
« Last Edit: August 20, 2018, 02:11:09 AM by Than2499 » Logged
dq
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« Reply #3 on: August 20, 2018, 06:56:31 AM »

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.

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.. 
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dq
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« Reply #4 on: August 21, 2018, 09:22:22 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              

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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Than2499
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« Reply #5 on: August 22, 2018, 12:44:44 AM »

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..!

1. Prior to starting Luspatercept, were you originally Transfusion Dependant, meaning did you have regular transfusions or non transfusion dependant?
--> My case is TD. I have had blood transfusion since 1 year old.
2. What was your haemoglobin before starting Luspatercept in general..? 7-8? or 13?
--> Before start Luspatercept my pre-transfusion is Hb 7 post-transfusion around Hb 8-9
3. Did you have an MRI prior to starting Luspatercept to see if you already had slight Extrameduallary Haemopoiesis (EMH)?
--> I have MRI at first step before take Luspatercept according to trail protocol and MRI on mid-way trail but i did't have any comment about EMH from my doctor until i have severe back pain and my legs starting numb.
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?
--> My doctor give me hyper-transfusion in order to reverse EMH by get enough blood (Hb13) my EMH in my spine was gone. My doctor told me i have maintain my Hb13 forever. 
5. I'm not too sure by what you mean your trial is finished then your trial is terminated by your doctor?
--> My trail is running to the last month of trail when i have EMH (month 11). The trial will disclose the label on month 12 then we have to blood collection in order to follow up after that all candidates in the program will receive real Luspatucept for 3 years with follow up schedule. My case i have to terminated on this point that i can't receive Luspatucept because it does not work for me and it may related about my EMH (we can not say Luspatucept is related or being the factor of EMH)     
6. I assume your Ferritin went up because of the heavy transfusions?
--> Yes, after i have hyper transfusion 13 units of blood during in the hospital, my SF 7,000 is jump to 12,000.
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?
--> First time only. My doctor said, other candidate in the trail seem the common complain about bone pain once they have Luspatucept in the first time and pain was relieved in a few days. 
9. Did you check your Uric Acid Levels? Did they decrease?
--> Yes i check every 2 week, during i have Luspatercept my Uric Acid is high(7-8) but when i stop it, Uric Acid become in normal range. I don't sure it will related to Luspatucept or not because i just take supplement/vitamin in last month and it grammatical lower my Liver (SGOT, SGPT) from SGOT 90-110 to 34-36 and SGPT 80-100 to 25 in 1 month (I start to take supplement/vitamin the same period of stop Luspatucept and got EMH )
10. Finally, what about your spleen? Did it decrease in size or was it normal size in the first place?
--> Just 1 month of Hyper-transfusion, right now i don't see significant decrees of spleen.

UPDATED
--> After EMH was gone on last month. A few days ago I feel the pain on my back on last Thursday Aug16, 2018. The pain symptom is very similar of  EMH so I went to see my doctor on last Friday Aug19 and found that my Hb was only 8.5 (maintain at Hb13) so I have 1 unit of transfusion. My back pain still existing so i came back to the hospital in Sunday (Emergency) and have 2 units of blood transfusion, my pre-transfusion on Sunday is only 8.9 (that weird, normally 1 unit of blood transfusion [220-300cc] may increased my Hb 1-1.5).
In Monday Aug20, my back pain still existing, so i went back to hospital again and have 2 units of transfusion, my pre-transfusion is Hb 10.5 (weird again. normally 2 units of blood 500-600cc may increased my Hb 2-2.5)

Today Aug22, my back pain still existing and i have doctor appointment on Friday Aug24, 2018. I plan to ask my doctor for MRI on my back.

   
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dq
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« Reply #6 on: August 23, 2018, 02:26:29 PM »

Hello my friend, thanks for getting back to us. Firstly I am so sorry to hear about your back pain. Are you sure you have not done anything recently to harm your back? Sometimes back pain can come from over exerting yourself. Just a quick response to your very helpful answers;

1) Ok, thanks.

2) Ok, thanks.

3) I am not a doctor but I think the reason that EMH may have occurred is because your body is use to having a higher hgb (8-9) and therefore when your hgb dropped to 7 for the trial your body needed to find a way to bring itself back up to its usual operating level. For this reason EMH may have developed. I don't think it is due to Luspatercept but again, I am not a doctor. If you can get your first MRI results you can check to see if you had slight EMH before starting Luspatercept.

4) Ok, thanks.

5) Ok, thanks.

6) Ok, thanks.

7) I believe you forgot to answer this question?   [What outstanding improvement did your friends see? 1hgb? 1.5hgb? How many doses did it take to work? How long did it last before it went down again?]

8) Ok, thanks

9) Your uric acid may have increased because you stopped having blood transfusions not because you stopped Luspatercept. The reason I think this is the case is because thalassaemia patients do not produce 'enough working haemoglobin' on their own therefore the body tries to compensate by producing even more red cells (to keep hgb up). The constant red cell creation and destruction causes uric acid levels to elevate because each red cell that dies releases uric acid into the blood. Once you started your transfusions again your body no longer needed to create so many red cells because the hgb increased therefore the uric acid levels may have reduced.

10) I doubt your spleen size would be large since you take blood transfusions so there aren't that many defective cells to remove from your blood (which is one of the reasons the spleen enlarges in NTDT thalassaemia).

Once again I am sorry to hear that you are experiencing these issues, please keep us updated on your condition.
BTW, where do you live?
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Andy Battaglia
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« Reply #7 on: August 25, 2018, 02:05:14 PM »

I am wondering if a low starting Hb level might interfere with the effects of Luspatercept. I think some questions won't be answered until we see full results from the trials and an accompanying analysis.
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Andy

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Than2499
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« Reply #8 on: August 27, 2018, 01:16:07 AM »

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?
--> please describe the question, I don't understand your question. Do you mean outstanding 1hgb or 1.5hgb of whome? Did you mean to my friend who have Luspatucept or meaning to me? 

I'am suspect my back pain may be cause from activities because last Friday Aug24, I went to have blood transfusion. I saw my post transfusion Hb is 13.8 that was the highest record of my entire life but at that time i still got my back pain and my doctor decided to gave me transfusion. I guessed my Hb could be reach 14 that very enough to stop development EMH completely. My back pain was better but not 100%gone. Today Aug27 I can feel a lit bit my back pain.

i was suspect because i usually lie down to my sofa it might cause of my back pain.

BTW, I have appointment MRI on Next Monday Sep3, 2018. I will update you once i found the cause.
 
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dq
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« Reply #9 on: August 27, 2018, 06:49:33 PM »

I am wondering if a low starting Hb level might interfere with the effects of Luspatercept. I think some questions won't be answered until we see full results from the trials and an accompanying analysis.

Hello Andy, good point - what brings that to mind..? Sounds like your heading towards something interesting...
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dq
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« Reply #10 on: August 27, 2018, 06:55:34 PM »

Hello Andy, good point - what brings that to mind..? Sounds like your heading towards something interesting...

Hello again, It's good news to see that your back pain might be something completely different.
Sometimes back pain and Abdominal pain can be linked to constipation I believe. Are you presently having troubles passing stools (going to the toilet)?

"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."
[You mentioned in your first post above that your friends saw an outstanding improvement while being on Luspatercept, so I was wondering how much in numbers (haemoglobin) was this improvement?]

p.s. Please let us know how your MRI goes.. Good luck.
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Andy Battaglia
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« Reply #11 on: September 01, 2018, 12:08:25 PM »

dq, another case where the patient was in the Luspatercept trial and didn't respond made me wonder. I have to follow up with her to see if her trial is finished and if she learned if she had the real drug or the placebo. She lives with a chronically low Hb because of a hard to match blood type, so I wondered if the low starting Hb was a factor.
I also want to hear what the developers of Luspatercept have to say to explain non-responders. I hate to speculate but wonder if individual genotypes make a difference.
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Andy

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Andy Battaglia
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« Reply #12 on: September 01, 2018, 12:10:13 PM »

Than, the pain could still be from EMH masses, as they will take time ti disappear once the Hb is kept at a high level and not allowed to drop. Obviously, time is required to see if this happens.
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Andy

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Than2499
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« Reply #13 on: September 10, 2018, 10:59:08 PM »

Hi dq and Andy,

My back pain is no more since I keep 13Hb for a while. I already have MRI on spine last week and I will go to see my doctor for MRI result in this Friday.
I will update my MRI result and my friends who are ongoing Luspatercept.

I don't have problem constipation. On the other hand I have problem with upset stomach. It happen every morning or 30 min after i have a breakfast (fist meal of the day).

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