Neupogen; Luspatercept

[BeccaBoo]

They just put my high risk MDS Dad on 2 new meds, Neupogen and Luspatercept, and I'm freaking out, again. He went through 8 rounds of Vidaza and then added 2 rounds of Venetaclax, which disabled him entirely, I was freaking out, quit my job, moved back in, he needed 24 hour care, and it landed him in the hospital with neutropenic fever. Zero benefit, only harm. Now he's off the chemo, doing 90% better as far as quality of life goes, and now... these new meds. I can't wrap my head around it. I looked up neupogen for MDS, and not only is there no proven benefit for treating MDS... all I could find was a warning that is actually CAUSES MDS and AML! Why would they prescribe something for MDS that causes MDS? It can also speed up cancer and tumor growth along with many other risks. I look up Luspatercept, and find little information about MDS, only minimal debated info for low risk MDS, and info saying it has had zero benefit for high risk MDS, and a slew of risks come with it. The chemo was meant to destroy proteins. These new meds are meant to build proteins. They say it's only meant to do one thing, and that is to boost his counts and help lower the number of transfusions he needs. Since he stopped the chemo his counts have been all steadily going in the right directions. He hasn't had a single infection. His white count more than tripled! I can't find any studies showing this is what it will do! All I am finding is info saying it either doesn't help or they don't know, and they are still studying it. Has anyone had any experience with these before? Anyone had any good results? Or bad results? I just can't find anything that gives me any hope this is going to be helpful. I'm terrified they are just using my Dad to experiment on and are feeding him cocktails that have no studies to back it up. It appears he is the study!

[Neil Cuadra]

BeccaBoo,

I'm sorry that your Dad's treatment hasn't gone better and that it's been such a strain on you.

I'm not a medical professional but perhaps I can offer some information.

Do you go to your Dad's medical appointments? Have you asked his hematologist/oncologist why these treatments are being used? Is this a doctor who you trust? Sometimes doctors don't take enough time to explain their treatment approaches and the patient's status, so family members have to ask for explanations and speak up if something doesn't make sense to them.

When it comes to quality of life versus length of life, it's important for patients and their caregivers to speak up. These are very personal decisions.

Granulocyte colony-stimulating factors like Neupogen can boost white blood cell counts. If his white cell count is increasing, this may be why. Similarly, Luspatercept can increase hemoglobin, although as you say it's typical use is for lower-risk MDS. Research to find out when it can be beneficial is ongoing. Perhaps it will help, and if not the doctors may stop giving it to him.

I wouldn't worry that he's getting an MDS drug that causes MDS. They have to list all known side effects, even the ones that are rare. (For example, some antidepressants can cause depression.) The common side effects of Neupogen, like headaches, are usually tolerable or manageable, and the danger of a low white count is real. As with all drugs with side effects, doctors prescribe them when they think the benefits outweigh the risks, and with a serious disease like MDS all treatments have risks. But you and your Dad should have some say about these tradeoffs.

You mentioned proteins being destroyed or built up. It's not a contradiction. The chemo was intended to destroy "bad" MDS cells, while these agents are meant to build up "good" cells.

They aren't experimenting on your Dad unless he's on a clinical trial that he agreed to. When other treatments have failed, a trial can be the best option for a patient, even though the trial uses treatments that may or may not work and aren't yet approved for their condition. If your Dad's first-line Vidaza treatment didn't succeed, as it sounds, then unfortunately there are fewer choices for second-line treatment, but a lot of research has gone into identifying the best path forward.

In addition to asking questions here, I suggest that you contact the MDS Foundation and the Aplastic Anemia & MDS International Foundation, if you haven't already. They have information specialists and medical advisors who can address your concerns.

[BeccaBoo]

I guess I'm hoping to find other people who have taken these and what their results were, because I cannot find anything. Yes, I take Dad to all his appointments. I am his full time caregiver too. I do not trust his doctor, the oncologist, at all. She has abandoned him 3 times now, and I found out she has a home in Greece she goes to as she pleases. It's as if she is given a script to read, and she downplays all side effects and belittles our questions. When I asked her about the side effects for luspatercept she literally said "Oh well, small things, like you shouldn't breast feed while taking this". She and my Dad had a laugh, and I was livid. When Dad started chemo all she said was "he may be tired and get constipated". She was in complete denial that any of the side effects my father was having from chemo was from the chemo. She kept circling back and saying it was his disease. She said she'd know if the Vidaza was working within 2 cycles, and kept him on for 8. Venetaclax, she said she said she'd know in 1 cycle, and kept him on for 2. Everything I learned let me know this was extremely irresponsible. Even after it had zero positive effect after all that time, and it landed him in the hospital, she STILL suggested he stay on them, just at all lower dose. Thank goodness he said no!! With these new meds, I've so far learned that they should be seeing results within the first 1-2 weeks (10 day median). I know why they suggested them, and I know everyone I ask is going to tell me the same thing. Talk to his oncologist if I have concerns. It's always the same answers. These new meds are in hopes to bring his red and white count up so he is less transfusion dependent. The venetaclax made his white count tank. It's tripled since he stopped, but not because of the new medication. He just started the Neupogen yesterday, and I had no time to research it. It was brought up for the first time, and administered, yesterday, when went in for a regular blood draw. We had not heard of it or discussed it with the oncologist. He did not even know the oncologist had ordered it. Oncologist mentioned Luspartercept when we saw her last week, and we told her we wanted to research it first. I had about 15 minutes to research Neupogen on my phone while my Dad was in the transfusion chair before they gave it to him. We were both confused, but he wanted to go for it. Search "Neupogen for MDS" and the first thing that came up was that is caused MDS, and I still have found nothing saying it treats it. I went home to dig more and can't find anything, and certainly nothing good. Just that it may lower hospitalization days during severe neutropenia for some people. My father says "Yes" to everything his doctor suggests. My Dad did make an appointment for a 2nd opion about 3 weeks ago, while his oncologist was in Greece. That oncologist was awesome!!! He was so honest, thorough, and said everything, straight up, no beating around the bush. He said the chemo was doing absolutely nothing but harming him, and he didn't have anything else to suggest my Dad take. That was before these new meds were prescribed so we had no chance to bring it up. I wonder what he would say.... but my Dad doesn't want to bother making an appointment to ask him. The truth is, my father seems to feel better about everything when he is taking something. He feels like he is doing something, anything, everything he can about it. There has been no discussion about these medications being for trials, but they obviously are, and I know long ago my Dad said he was willing to be in trials. (I actually believe I found the exact Venetaclax trial he was in, 3rd phase, which included the antibiotic and antiviral he was on, specifically for high risk MDS patients who failed Vidaza). It just hasn't been mentioned. I support him all the way, even though I don't agree, even though I totally do not trust or even like his doctor, but I think it is foolish to blindly take drugs without understanding how likely they are to help, or harm. I just want to know what to look out for mostly. I am his full time caregiver now. After going through what we did with the venetaclax and vidaza, I am convinced no one is really looking out for him except his family. The transfusion people aren't supposed to suggest anything, but I could tell, even they were confused why nothing was being addressed as his health tanked so badly he became wheelchair bound and lost 100 pounds. They all just assumed it was the disease.... Oh boy. I'm terrified they are going to kill him in this process of playing with numbers for their study. Dad is an engineer and a gambler though, it's understood, he is going to keep trying.

[BeccaBoo]

I found some motivation.... oncologist gets paid for each study. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1123551/

[Sally C]

Hi Becca,
I am sorry you and your father are having to deal with this. My husband was treated at The NIH starting in 2009. They gave him Neulasta which is similar to Neupogen. He didn't have any side effects and it did bring his white cells up. He was at great risk for infection which could have killed him.
After over 125 blood/platelet transfusions, he entered a clinical trial at NIH as the first MDS patient to receive Promacta. He took it from spring 2011 until August 2012. He has been in remission since and has not needed a transfusion since Aug. 2011 which was for a colonoscopy. The first drug they gave him was Campath. One of the side effects listed was "death". As Neal said, they list any and everything. It was a roll of the dice for my husband but it certainly paid off. His situation was bad enough that he really didn't have a choice.
All the best,
Sally

[BeccaBoo]

Oh how wonderful! Thank you so much for sharing, and congratulations! After so many transfusions, wow!! To be free from it must be amazing. It's awesome to hear it's possible. Can you tell me how long it took for the Neulasta/neupogen to give results? Does he still take it? Promacta is for low platelets. My Dad isn't taking anything for platelets now, this may be worth bringing up to the doctors. Do you think the campath had a lot to do with his remission? See, I am already finding a lot of information on the drugs you mentioned. Good reviews, and studies done. It's disconcerting when I can't find information. I appreciate you taking the time to respond.

[BeccaBoo]

For the first time in 9 months my fathers WBC is back in the normal range!!! It's 2.8, up from .06 about 3 months ago. He has only needed 3 transfusions in the last 6 weeks, which is incredible. He had achey bones for the first 3 weeks after starting neupogen and luspatercept, but he doesn't seem to be having any other side effects that he can detect. We are pleased with the results so far