Bad news about AMG 531/Romiplostim/Nplate

[Birgitta-A]

Hi all,
Unfortunately the first of the new drugs for low platelets - AMG 531/Romiplostim/Nplate - can give dangerous adverse reactions. "FDA also expressed concerns about abnormal bone marrow growth and malignant tumors seen in some patients".
http://www.businessweek.com/ap/finan.../D8VANAG80.htm

The next drug in the race Eltrombopag/Promacta/Revolade is two months behind.
Kind regards
Birgitta-A
Last platelet count 48

[Marrowforums]

Nplate is the Amgen trade name for romiplostim, used to increase and sustain platelet counts and designed to treat chronic immune thrombocytopenic purpura (ITP).

Tomorrow the FDA will be getting advice from medical experts about whether to approve Nplate and for what conditions. The FDA is likely to follow their recommendations. Details are in the Advisory Committee Briefing Document (PDF, size 2.4MB).

According to news reports (one, two), Nplate's risks are a concern, but Nplate may still be approved based on Amgen's presentation of study data, its plan for controlling risks (by providing proper information to physicians and patients, doing follow-up studies, and not advertising directly to consumers), and because Nplate may still provide the best option for patients who have failed other treatments, including corticosteroids, immunoglobulins, and splenectomies.

Amgen's application to the FDA was submitted in October 2007 (see Marrowforums thread) and received priority review status. Amgen announced the results of it Phase 3 study in late January.

Current clinical trials for romiplostim include a study on romiplostim for MDS.

[Marrowforums]

According to wire service reports, the FDA advisory panel recommended today that the FDA approve Nplate, because the benefits outweigh the risks for treatment of ITP.

[Wendy Beltrami]

Oops, I started a new thread with the press release.

I am not discouraged with the reports of negative side effects. Seriously folks, are ANY of the drug treatments for AA free of side effects? Platelets are the ONE thing that hasn't had any kind of successful treatment and the ONE thing that has plagued us from day one. By far the worst aspect of AA for our son.

Wendy/mom to Grant
dx 12/4/98

[george peveto]

I took amg 531 Injection of 300 mg per week for 4 weeks-- It did improve the platelet counts from 27,000 up to 87,000 but after 4 weeks the blast increased in the peripheral blood and in the marrow--we stopped the drug for 4 weeks and then the dosage was reduced to 100 mg per week and again after 3 weeks the blast started to increase again in the peripheral blood so the drug was stopped
but again the platelets did improve to 80,000 or so. The platelets did remain in the 37,000 area for about 12 months.

[Wendy Beltrami]

George,
Is your diagnosis MDS or AA? I'm assuming you have MDS and got the AMG531 in a clinical trial.

Can you tell me about any other side effects? After stopping the drug, you mentioned that your platelets remained elevated for 12 months. Did your blast count return to pre-Nplate levels after stopping the drug?

Do you have any remaining negative effects from your experience with AMG 531?

Did the platelets increase within days? weeks? of starting the drug? Do you plan to try Eltrombopag, the oral platelet drug coming soon from GlaxoSmithKline?

Wendy/mom to Grant
dx 12/4/98 AA

[Trudy2008]

Hi, I'm new to this.
Has anyone taken romiplostin for low platelets recently. What is your experience with this drug.
I was diagosed with AA in 1997 and now have MDS with 20q deletion. My platelets are 13. Had ATG in '97 have gone in and out of remission twice. Am presently on cyclosporin and Eprex. I understand that romiplostin has been approved in the U.S., but not in Canada. Would like to try it before getting transfusions.

[Birgitta-A]

Hi Trudy,
Nplate (AMG 531) has given increased blast cells in MDS patients - don´t try that drug if you have MDS! Look at these lines:

"In a separate single-arm clinical study of 44 patients with myelodysplastic syndromes (MDS), 11 patients were reported as having possible disease progression, among whom 4 patients had confirmation of acute myelogenous leukemia (AML) during follow-up.

Nplate is not indicated for the treatment of thrombocytopenia due to MDS or any cause of thrombocytopenia other than chronic ITP."

http://www.drugs.com/nplate.html
Kind regards
Birgitta-A

[Trudy2008]

Hi Brigitta,
Thank you for your reply. Are there any other options available for low platelets besides transfusions? Is there anything in Europe that we don't have here? Would appreciate hearing from you.
Trudy
Victoria, B.C. Canada

[Birgitta-A]

Hi Trudy,
We have been waiting since July 2008 for FDA approval of GlaxoSmithKline´s drug for thrombocytopenia called Eltrombopag/Promacta/Revolade. The last info from FDA was that they should decide 19 Sept 2008 but then they have said that they have too much work to be able to keep the dead lines .

Eltrombopag has been effective in decreasing leukemia cells in tubes but is not in any trials for MDS only for low platelets in Immune Thrombopenic Purpura, hepatitis C (due to treatment with chemo) and other cancers like sarcoma.

I was hoping that we should be able to get Eltrombopag on license in Sweden as soon as it was approved by FDA. Now I have understood that they will be very careful when they give all kinds of growth factors off record because there have been so much problems with drugs for anemia like Aranesp.

There are many drugs for thrombocytopenia in trials. We will know more about them when the abstracts from the hematology conferens called ASH 2008 will be available .

We have no drugs for thrombocytopenia i EU.
Kind regards
Birgitta-A