Sign-in to Ask A Doctor - 24x7
OR
Sign in with Google
What Does Worsening Of Aplastic Anemia While On Promacta Indicate?
Question:
Brief:
Moderate aplastic anemia progressed to severe aplastic anemia. Idiopathic. Evolved with trisomy 8 found later. Currently on Eltrombopag 75 mg daily and cyclosporine 50 mg twice per day.
Details
My niece (East Asian), now late teens, dx moderate AA early 2009;ATG in mid 2010 with limited response, RBC tx independent for one year then. Re-start RBC tx in early 2012. Re-start cyclosporine alone in April 2013, then tx free for 9 months. Re-start RBC tx in early 2014
Started Eltrombopag (Promacta) in Nov 2014, initially 25 mg daily for 1 month, then raised to 50 mg daily until end March 2015, then 75 mg daily until now, currently also on cyclosporine 50 mg x 2 times each day.
Before starting Eltrombopag , platelets range 12k to 17k, ANC about 0.8 to 1.4. No noticeable improvement after starting Promacta
Lab reports attached
Our doctors here basically have no experience in using this new drug for treating SAA. We really need expert advice from other places with more experience in using Promacta for treating AA or other bone marrow diseases, so that we can discuss with our doctors. I understand that Promacta has yielded success in clinical trial for AA patients and seems it is under trial for MDS patients. Questions –
1. We have started Promacta for nearly 8 months and stayed on 75 mg daily for more than 3 months already since late March but results are disappointing (please see CBC reports). Does it mean that Promacta has no effect on my niece? Shall we continue its use? Why or why not?
2. If continue, shall we stay on 75 mg daily or increase to 100 mg? My niece is an east asian, is 75 mg daily the maximum dose for East Asians?
3. How long should we continue to try in your opinion?
4. I recall that I have seen from articles/reports that chromosome changes were reported in some patients during clinical trial of Promacta for AA (some are more complex/serious chromosome 7 changes). What is the chance of chromosome changes? How does it relate to the duration of use of Promacta? We are concerned as my niece is on Promacta for nearly 8 months already.
5. If one does respond to Promacta, what is the dosage at which noticeable improvements started to be seen? Platelet is usually the first line to improve? How long should we wait before we declare Promacta has no positive effects on my niece?
6. My niece is currently on cyclosporine as well. Can cyclosporine taken along with Promacta yield better results?
7. My niece is very very scared of BMT and refused this option. Also, the best available MUD match we can find is only 7/8 at high resolution. Promacta seems give us a light of hope, but results are so discouraging. What are your suggested way forward and actions to be taken if you were in our place? What other advice do you have in relation to the use of Promacta
Would also appreciate any advice regarding her disease.
Best Regards,
Brief:
Moderate aplastic anemia progressed to severe aplastic anemia. Idiopathic. Evolved with trisomy 8 found later. Currently on Eltrombopag 75 mg daily and cyclosporine 50 mg twice per day.
Details
My niece (East Asian), now late teens, dx moderate AA early 2009;ATG in mid 2010 with limited response, RBC tx independent for one year then. Re-start RBC tx in early 2012. Re-start cyclosporine alone in April 2013, then tx free for 9 months. Re-start RBC tx in early 2014
Started Eltrombopag (Promacta) in Nov 2014, initially 25 mg daily for 1 month, then raised to 50 mg daily until end March 2015, then 75 mg daily until now, currently also on cyclosporine 50 mg x 2 times each day.
Before starting Eltrombopag , platelets range 12k to 17k, ANC about 0.8 to 1.4. No noticeable improvement after starting Promacta
Lab reports attached
Our doctors here basically have no experience in using this new drug for treating SAA. We really need expert advice from other places with more experience in using Promacta for treating AA or other bone marrow diseases, so that we can discuss with our doctors. I understand that Promacta has yielded success in clinical trial for AA patients and seems it is under trial for MDS patients. Questions –
1. We have started Promacta for nearly 8 months and stayed on 75 mg daily for more than 3 months already since late March but results are disappointing (please see CBC reports). Does it mean that Promacta has no effect on my niece? Shall we continue its use? Why or why not?
2. If continue, shall we stay on 75 mg daily or increase to 100 mg? My niece is an east asian, is 75 mg daily the maximum dose for East Asians?
3. How long should we continue to try in your opinion?
4. I recall that I have seen from articles/reports that chromosome changes were reported in some patients during clinical trial of Promacta for AA (some are more complex/serious chromosome 7 changes). What is the chance of chromosome changes? How does it relate to the duration of use of Promacta? We are concerned as my niece is on Promacta for nearly 8 months already.
5. If one does respond to Promacta, what is the dosage at which noticeable improvements started to be seen? Platelet is usually the first line to improve? How long should we wait before we declare Promacta has no positive effects on my niece?
6. My niece is currently on cyclosporine as well. Can cyclosporine taken along with Promacta yield better results?
7. My niece is very very scared of BMT and refused this option. Also, the best available MUD match we can find is only 7/8 at high resolution. Promacta seems give us a light of hope, but results are so discouraging. What are your suggested way forward and actions to be taken if you were in our place? What other advice do you have in relation to the use of Promacta
Would also appreciate any advice regarding her disease.
Best Regards,
Brief Answer:
Maximum dosage of promecta is 150 mg per day for aplastic anemia.
Detailed Answer:
Hi, dear
I have gone through your question. I can understand your concern.
Promecta is used in severe aplastic anemia if one of the immunosuppressive drugs failed.
She tried ATG with no response so use of promecta is justifiable.
First of all 75mg is not the maximum dosage for aplastic anemia. It is for immune thrombocytopenic purpura. Maximum dosage for aplastic anemia is 150 mg per day.
It should be started with 50 mg per day and it can be increased upto 150 mg day.
Approximately 40% patients show improvement within 12 weeks.
If no any improvement seen with maximum dosages then it should be discontinued after 18 weeks.
20% patients develop new genetic abnormalities by change in chromosome.
If patient develop new genetic abnormality then it should be stopped.
cyclosporin can be used along with promecta.
Your niece should take promecta with increased dosage up to 150 mg per day and if no response is seen in 18 weeks then it should be stopped.
It is strictly prescription based medicine so consult your treating doctor and take treatment accordingly.
Hope I have answered your question, if you have doubt then I will be happy to answer. Thanks for using health care magic. Wish you a very good health.
Maximum dosage of promecta is 150 mg per day for aplastic anemia.
Detailed Answer:
Hi, dear
I have gone through your question. I can understand your concern.
Promecta is used in severe aplastic anemia if one of the immunosuppressive drugs failed.
She tried ATG with no response so use of promecta is justifiable.
First of all 75mg is not the maximum dosage for aplastic anemia. It is for immune thrombocytopenic purpura. Maximum dosage for aplastic anemia is 150 mg per day.
It should be started with 50 mg per day and it can be increased upto 150 mg day.
Approximately 40% patients show improvement within 12 weeks.
If no any improvement seen with maximum dosages then it should be discontinued after 18 weeks.
20% patients develop new genetic abnormalities by change in chromosome.
If patient develop new genetic abnormality then it should be stopped.
cyclosporin can be used along with promecta.
Your niece should take promecta with increased dosage up to 150 mg per day and if no response is seen in 18 weeks then it should be stopped.
It is strictly prescription based medicine so consult your treating doctor and take treatment accordingly.
Hope I have answered your question, if you have doubt then I will be happy to answer. Thanks for using health care magic. Wish you a very good health.
Note: Do you have more questions on diagnosis or treatment of blood disorders? Ask An Expert/ Specialist Now
Above answer was peer-reviewed by :
Dr. Yogesh D
Answered by
Get personalised answers from verified doctor in minutes across 80+ specialties
