Moving forward with ATG and searching for unrelated BMT match.

[Shroob]

Hello all!

We spoke with Seattle Cancer Care Alliance today and it made my mom almost cry (happy tears) after the talk. Dr. Deeg gave my mom unparalleled attention to anything she's received before (no slight to her previous doctors as I know this is unfamiliar terrority for everyone with her kidney disease and AA) and she really feels she's in good hands now. After a sobering two hours, we are moving forward with inpatient ATG in a few weeks as soon as paperwork gets processed. They also want to try another BMB soon to check if there's any progression into MDS territory, as her counts are rapidly declining each week. She will come home and be monitored for 2-3 months to see if there's any improvement, and after that we will head back to Seattle for a BMT, unrelated as all of her 3 siblings either suffer from Polycystic Kidney Disease as well or Melanoma. He told us it would be a 4 month inpatient stay, which floored me and my dad, but explained there are a lot of complications (such as GVHD) to watch out for.

I know success rates are still low, and Dr. Deeg admitted my mom is a complicated case due to her transplanted kidney, but I am glad we are moving in a good treatment direction and anything to keep my mom stable helps.

If anyone has any advice it would be greatly appreciated. I have scoured the forums but did want to post an update here.

Thank you!

[Marlene]

Glad your mom has a plan in place and is happy with her new medical team. As you know, this disease will test your patience.

Hopefully the ATG will knock out the SAA quickly. I'm a bit confused as to why they are going to do a BMT later. If the ATG works for her, then why a BMT? Is it related to her kidney disease?

Wishing your family the best outcomes.

[Neil Cuadra]

Audrey,

You can't be in better hands than with Dr. Deeg involved. We've known him from many past conferences, and he's one of the most knowledgeable physicians in this field. Plus you have all of the accumulated knowledge from past patients on your side.

I'm surprised at the 4-month prediction, even with the complications involved, but if that's what it takes then it's important to know it ahead of time, with all the logistics of a long stay to plan.

Here's hoping that the biopsy results show no progression.

[Shroob]

Marlene & Neil,

Thank you for your replies! I am so glad to hear about your positive experience with Dr. Deeg as well, Neil.

As for planning a BMT after the ATG, I think Dr. Deeg just wants to get a search going ASAP for a non-related donor just in case the ATG isn't successful. Her hemoglobin is dropping weekly and she is now getting weekly blood & platelet transfusions so I think he wants to move rapidly (and most likely due to the kidney & avoiding iron overload). I think they're also nervous about the immunosuppressant therapy on the transplanted kidney and feel the BMT might even be "easier" for her. I could be completely wrong though. We just read his after care visit summary and he mentioned a small detection of PNH DN M T3a and that we have to be diligent in watching for MDS.

I was wrong about the 4 month inpatient stay--she will just have to stay nearby to the hospital in Seattle. She might be able to come home, but she doesn't want to be treated here in Idaho, and it did sound like Dr. Deeg wanted her close by there as well.

Really just trying to wrap my head around this. I am in no way versed in medical terminology so thank you for bearing with me and taking the time to reply to this thread. It means a lot to my mom and I.

Take care everyone.

[Hopeful]

Hi Shroob,

I'm glad that your mom is in good hands and that there is a PLAN in place. Having a small PNH clone is a sign that her disease may be immune mediated and may respond to the ATG. It is sometimes used as part of a pre-transplant regimen. So, it sounds like she will be getting the best of both worlds by doing the transplant search in parallel with ATG.

If she does have a transplant, don't be in a hurry to leave the security of being near the hospital for the first 100 days. You will appreciate the monitoring and quick access to experts should problem arise. 4 months is probably a worse case scenario.

Wishing her the best!

[Matthew42]

Originally Posted by Hopeful

Hi Shroob,

I'm glad that your mom is in good hands and that there is a PLAN in place. Having a small PNH clone is a sign that her disease may be immune mediated and may respond to the ATG. It is sometimes used as part of a pre-transplant regimen. So, it sounds like she will be getting the best of both worlds by doing the transplant search in parallel with ATG.

If she does have a transplant, don't be in a hurry to leave the security of being near the hospital for the first 100 days. You will appreciate the monitoring and quick access to experts should problem arise. 4 months is probably a worse case scenario.

Wishing her the best!

Yes, Hopeful: you are right about the small PNH clone. It is the greatest predictor of the disease being immune-mediated. I know this firsthand: Although my mother hasn't responded to ATG in a year (except for neutrophils), Dr. Young says that my mother's disease is highly immune-mediated because of her small PNH clone. He still wants to give her some more time (because of her age) before moving on to Campath. He suggests no rabbit-atg because it can cause DNA changes in the bone marrow, whereas Campath will not (he also believes that Campath is more likely to be effective). He still insists on immunosuppressant therapy for her because of the small PNH clone. Her aplastic anemia is highly autoimmune, he says. If my mother did not have a small PNH clone, Dr. Young and his team would have suggested a different treatment for her (Cytoxan?). I never knew any of this until rather recently.

@ Shroob: I wish your mother the best of luck. Please take care.

[Shroob]

@Hopeful,

I had no idea having a small PNH clone meant it could be immune mediated. That provides some hope in a rather scary time, thank you! My family and I are learning so much and are so thankful to have these forums as a source of help and information.
Also, my parents are definitely planning on sticking around as long as needed after the transplant. We are lucky to have lived close by to Seattle years ago and know many people we can reach out to if need be.

Thank you!!

@Matthew42,
I wish you and your mom the best of luck with her continued treatment, and am glad to see Dr. Young seems very invested in what's best for her. Thank you for the reply, it is comforting to hear more confirmation firsthand from others nearby to this disease. Please take care as well.

[Shroob]

So things have taken a weird turn. We were under the impression we’d be getting my mom to Seattle in a few weeks after the initial visit (we were told once the board meets and the paperwork goes through we’d hear back) but never heard anything. We called, were told to call the next week, and the next…we kept being told there were no orders and they’d look into it. Finally we found out Dr. Deeg wanted to schedule a follow up in SEPTEMBER! We were shocked. After a bit of prodding we were told they are full and basically the other patients take precedent over her disease right now? He said “you’re not going to like this but the cancer patients take priority” and it’s just disheartening and confusing.
I guess she’s doing okay? More energy being off the Cyclosporine. She’s getting red blood and platelet transfusions every week, but has energy. Is only on Tacrolimus right now. Guess we’re just in a wait and see period. Kind of feeling like no one is really giving us answers. Obviously you can live with low levels but I also feel like this is a type of disease to treat sooner than later? I don’t know. Trying to get ahold of her hematologist but he’s gone radio silent and was probably hoping Seattle would take on her care from now on.

[Hopeful]

Wow - I'm really saddened and surprised to read this. I've heard that some hospitals are overloaded because people were delaying care during the height of COVID.

Weekly transfusions are never a good thing.

Have you considered consulting with Dr Young at the NIH in parallel with the wait?

[Shroob]

Originally Posted by Hopeful

Wow - I'm really saddened and surprised to read this. I've heard that some hospitals are overloaded because people were delaying care during the height of COVID.

Weekly transfusions are never a good thing.

Have you considered consulting with Dr Young at the NIH in parallel with the wait?

Definitely understandable. Who knows when we'll actually be out of the crazy times, if ever.

I believe the doctor at Utah consulted with Dr. Young, but that visit was also very inconclusive. We haven't spoken with him ourselves though, besides the email I sent his team several months ago out of desperation (and understandably got no response from).

[mola-tecta]

Shroob, I am so sorry that the treatment course has made a turn for the worse like this. What a hassle!

I understand the need to place cancer patients first. But I also feel that leaves patients with other diseases like Aplastic Anemia kind of hung out to dry. Sometimes I get the same sort of sense from my mother's hematologist office.

Hopefully you can find some answers soon. I would try giving Dr. Young's office a call, from what I remember Matthew42 had gotten a better response from them this way.

I also found that the Cleveland Clinic will do a Virtual Second Opinion: https://my.clevelandclinic.org/depar...s#overview-tab

It's a bit pricey, but it also looks like if you call them or have your doctor refer you to their office, they have the option for virtual visits as well. Dr. Maciejewski is supposed to be another of the world's leading experts in AA.

[Matthew42]

Originally Posted by mola-tecta

Shroob, I am so sorry that the treatment course has made a turn for the worse like this. What a hassle!

I understand the need to place cancer patients first. But I also feel that leaves patients with other diseases like Aplastic Anemia kind of hung out to dry. Sometimes I get the same sort of sense from my mother's hematologist office.

Hopefully you can find some answers soon. I would try giving Dr. Young's office a call, from what I remember Matthew42 had gotten a better response from them this way.

I also found that the Cleveland Clinic will do a Virtual Second Opinion: https://my.clevelandclinic.org/depar...s#overview-tab

It's a bit pricey, but it also looks like if you call them or have your doctor refer you to their office, they have the option for virtual visits as well. Dr. Maciejewski is supposed to be another of the world's leading experts in AA.

Yes, you can call Dr. Young's office and have them consult with your current hematologist. He and his team wouldn't see my mother, but they told my mother's doctor what the next course of treatment should be if things don't improve (Campath). Also, he said that my mother did have some response to immuno-suppressant therapy, but that she should still wait a few more months before going on to Campath. Sometimes, a complete response to ATG can take well over a year, particularly in older people. He also said that my mother's condition is highly immune-mediated because of her PNH clone. In other words, it is safe to say that she very much has autoimmune aplastic anemia. That's all the info we got from him. He reviewed her blood tests and bone marrow biopsies.

I would very much recommend the Cleveland Clinic, too.

[Shroob]

Originally Posted by mola-tecta

Shroob, I am so sorry that the treatment course has made a turn for the worse like this. What a hassle!

I understand the need to place cancer patients first. But I also feel that leaves patients with other diseases like Aplastic Anemia kind of hung out to dry. Sometimes I get the same sort of sense from my mother's hematologist office.

Hopefully you can find some answers soon. I would try giving Dr. Young's office a call, from what I remember Matthew42 had gotten a better response from them this way.

I also found that the Cleveland Clinic will do a Virtual Second Opinion: https://my.clevelandclinic.org/depar...s#overview-tab

It's a bit pricey, but it also looks like if you call them or have your doctor refer you to their office, they have the option for virtual visits as well. Dr. Maciejewski is supposed to be another of the world's leading experts in AA.

mola-tecta,

Of course. I hold no animosity towards Seattle or the cancer patients, I just have more of a negative emotional response since it's my mom who is being turned away...OUR moms who are being turned away (I read your latest post, and I'm so sorry your mother's hematologist isn't treating her how he should), but it's no one's fault.

It's really funny actually --my mom just got off the phone with Cleveland and we're pushing through a referral from her hematologist here as soon as we can. I gave her Dr. Maciejewski's name after your reply so she can ask for him if possible.
We're hoping third times a charm...
Will definitely recommend her to call Dr. Young as well.

Thank you for all your help! Please take care of yourself.

[Shroob]

Originally Posted by Matthew42

Yes, you can call Dr. Young's office and have them consult with your current hematologist. He and his team wouldn't see my mother, but they told my mother's doctor what the next course of treatment should be if things don't improve (Campath). Also, he said that my mother did have some response to immuno-suppressant therapy, but that she should still wait a few more months before going on to Campath. Sometimes, a complete response to ATG can take well over a year, particularly in older people. He also said that my mother's condition is highly immune-mediated because of her PNH clone. In other words, it is safe to say that she very much has autoimmune aplastic anemia. That's all the info we got from him. He reviewed her blood tests and bone marrow biopsies.

I would very much recommend the Cleveland Clinic, too.

He sounds like a great resource, and I'm glad he was able to give some advice on treatment for your mother even while not being able to treat her himself.

Thank you for your reply! Take care, Matthew!

[Shroob]

Small update: Cleveland won't take my moms insurance so we're not going that route at the moment.
We spoke to her hematologist last week and he said that we need to get her the ATG treatment either here or in Utah, so we left it up to him to figure out if Utah could take her or if here would be sooner. While it would be nice to have her closer to home, Boise doesn't see as many aplastic anemia cases and my mom prefers to do ATG at a facility with more experience.
So far it's been a week and we haven't heard anything.

She had a 20 minute nose bleed yesterday and when she went in for her weekly blood draw today her platelets were at 6. Lowest they've ever been. That's what we get for going 2 weeks without platelets (last transfusion was 2 weeks ago Friday and they did a blood draw last Wednesday and her platelets were 12, so we held off until this week). Not sure what her hemoglobin was but not low enough yet for blood, I'm guessing next week she will get blood though as she's been getting it every 3 weeks now.

Hoping we hear back and get her in for ATG soon.

Edit: hemoglobin 7.9 this last visit.

[Shroob]

Hi all,

Thought I?d give an update. My mom just got back yesterday from ATG at the Huntsman Institute. She only had the usual side effects during treatment but now her blood sugars are all over the place because of the steroids. Craving fruit smoothies a lot with the insulin they?re giving her haha. Other than that, just a lot of transfusions/infusions the last few days. Magnesium today. Seems to be doing well, but we?re being careful.
She told me they?re most likely not going to pursue the BMT because of her previous kidney transplant, so ATG is our last line of defense.

Hope you?re all well and having a wonderful June.