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PNH Paroxysmal nocturnal hemoglobinuria

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  #1  
Old Sat Feb 1, 2014, 10:21 PM
ussoccer004 ussoccer004 is offline
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Newly Diagnosed PNH

After 8 years and being discharged from the military due to "Pancytopenia", I was admitted to the hospital 2 weeks ago and finally know exactly what I have. The hematologist finally diagnosed PNH, and while scared I am grateful to find this board.

The hematologist wants to start me on Soliris, but I have a question for those that are actually going through the treatments.

1. Does soliris help increase your white cells, neutrophils and platelets?

2. Will Soliris increase my Hemoglobin to the normal counts for a male I believe
12-14 range?

3. Is soliris a treatment we will have to be on for the remainder of one's life?

My recent Hemoglobin was 6.9, my Neutrophils were .5 and Platelets were 103K

I really appreciate any feedback, and sorry if the questions are a little off but I am really anxious and kinda depressed from reading up on the data behind PNH.
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  #2  
Old Sun Feb 2, 2014, 06:01 PM
Relentless Against SAA Relentless Against SAA is offline
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I am very sorry you have to go thru this. At the same time I am glad that you finally have a correct DX because now you can get a treatment that will help. I can tell you what a hematologist explained.

The PNH cells are missing the protection from a part of your immune system called Compliment. Without this protection, Compliment can break down those cells easily. We have been told this can really effect the Hgb but may also effect the ANC and Platelet count. Compliment is activated when you become ill or your body is placed under extra stress.

Solaris works by blocking Compliment. People who take Solaris feel so much better. One thing you should know.. People on Solaris are at a higher risk for Meningitis so you will need to be vaccinated every 5 years for this. Solaris blocks compliment at the level that Compliment would fight Meningitis.

Do you have to stay of this for your lifetime? Solaris does not stop the PNH. Actually the number of PNH cells you have will increase. Why? Because Solaris will stop Compliment from breaking down the PNH cells. If you go off Solaris, Compliment will be able to break down you blood cells again and your counts could drop pretty fast depending on the % of PNH cells you have. The break down of these cells is hard on the body, especially the kidneys which have to filter out all the waste. I have heard a Dr say that it is not impossible to come off Solaris, but it is tricky and people usually do not want to because they feels so good on it.

The only way to get rid of the PNH cells is a Bone Marrow Transplant. I heard a Dr say that with the development of Solaris, many people are not going for transplant. Solaris is given IV every 2 weeks.

I hope this info helps and you feel better soon. Please keep us informed of your progress. We are here for you.
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  #3  
Old Sun Feb 2, 2014, 07:33 PM
ussoccer004 ussoccer004 is offline
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So far that is what the hematologist is saying, I would have to stay on Soliris for the rest of my life. Currently diagnosed I am 26 years old, so hoping it is a long "normal" life. I had one hematologist say that I have PNH 1 and they are grouped by PNH 1, 2 or 3 stage?

Then being on Soliris would only stop the break down of the attack on my red cells, but Soliris wouldn't help block the attack on my White blood and platelets correct?

Once again I am thankful for this board and to have you answer my question. It is scary and to know that there are others with this rare disease makes it feel a little less lonely.
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  #4  
Old Sun Feb 2, 2014, 07:54 PM
Relentless Against SAA Relentless Against SAA is offline
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My understanding is that Solaris would help protect all the PNH cells therefore possibly increasing all 3 blood lines: RBC, Platelets, and your ANC. I have never heard of three stages. I have heard doc talk about percentage of cells effected by PNH.

From what I have heard, Solaris is amazing. PNH patients feel so much better. I have not heard of any bad side effects. The only down side is the cost- I've heard its the most expensive med on the market and you have to faithfully get the IV infusion every 2 weeks. Considering PNH was fatal not too many years ago, I am thankful for those who dedicated their lives to help people with this rare disease.

If you ever need a 2nd opinion: The doc that helped developed Solaris, Dr Maciejewski, is at Cleveland Clinic Ohio.
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  #5  
Old Sun Feb 2, 2014, 11:14 PM
Neil Cuadra Neil Cuadra is offline
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The PNH classification system that was proposed here defines PNH as Stage 1 when only cells in the bone marrow are affected, Stage 2 when leukocytes in circulating blood are affected as well, and Stage 3 when erythrocytes are also affected. Stage 3 is therefore the "classical" PNH that most patients are familiar with. Do you think that's what the hematologist was referring to?

Note that many PNH patients have chronic kidney disease (CKD), which is classified in stages.
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  #6  
Old Sun Feb 2, 2014, 11:27 PM
Relentless Against SAA Relentless Against SAA is offline
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Thanks Neil.
I am always impressed by your deep understanding of AA, MDS and PNH.
Do you think in his situation Solaris would help increase all three of the blood cell lines or just the RBC?
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  #7  
Old Sun Feb 2, 2014, 11:46 PM
ussoccer004 ussoccer004 is offline
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Quote:
Originally Posted by Neil Cuadra View Post
The PNH classification system that was proposed here defines PNH as Stage 1 when only cells in the bone marrow are affected, Stage 2 when leukocytes in circulating blood are affected as well, and Stage 3 when erythrocytes are also affected. Stage 3 is therefore the "classical" PNH that most patients are familiar with. Do you think that's what the hematologist was referring to?

Note that many PNH patients have chronic kidney disease (CKD), which is classified in stages.
I believe so. I am going to a second hematologist monday. This dr. Is the one who diagnosed me in the hospital, so I will write down the questions and answers also and report back. I was reading though that patients don't see improvements in their white or platelets. I have been placed on prednisone on and off for 8 years and it increases my blood counts but they come crashing down when I'm off them.
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  #8  
Old Mon Feb 3, 2014, 11:30 AM
mharrell mharrell is offline
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Please see a PNH specialist

My son was diagnosed with PNH 6 years ago and the local hematologist went from diagnosis straight to "You need a bone marrow transplant."

I researched and found this WONDERFUL support site: http://www.pnhdisease.org/index.php

The best advice we got from the members of the PNH support site was to see a PNH specialist. PNH is so rare that it's likely that your run-of-the-mill hematologist has never seen a patient with PNH.

The folks on that site can help with recommending a true PNH specialist. We had to travel to see one, as there are so few of them.

The specialist told us that the first thing we needed to address was the pancytopenia with ATG treatment. He also did not recommend Soliris for my son based on his clone size (% of defective blood cells). He also said that a BMT should be a last resort.

My son was 18 at the time. He had the ATG, and with the exception of platelets, which are still a bit low (130-135), all his counts are in normal range. His clone size went up after the ATG and has since decreased. He is doing very well now, and still does not need Soliris.
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  #9  
Old Mon Feb 3, 2014, 12:48 PM
Relentless Against SAA Relentless Against SAA is offline
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I would also like to emphasize the importance of seeing a PNH specialist. It is very important to see a doc that has had patients with this Dx. You can call the Aplastic Anemia Foundation. I believe they can give you names of PNH specialist. This is where I learned about Dr. Maciejewski. Even thou he helped develop Solaris, he does not" push" the med. He looks at counts and % of PNH. I know that he is also a firm believer in the success of ATG.
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  #10  
Old Mon Feb 3, 2014, 05:53 PM
NLJabbari NLJabbari is offline
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ussoccer 004, there are some great WEBinars on the AAMDS website that explain PNH a little better.

https://client.blueskybroadcast.com/...AMDSIF_072513/
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06/2004 my son was dx with SAA at the age of 10. No sibling BM match. He underwent ATG (H)/CsA. Relapsed 05/12 & dx'ed w/PNH. Currently in wait/see mode for Solaris as he is asymptomatic...
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  #11  
Old Mon Feb 3, 2014, 07:11 PM
Snuuze Snuuze is offline
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I think the Soliris should help all three blood types. However, the RBCs are the most likely to benefit from the Soliris. That's because they have thin skins and are large, thus they are the main target for the PNH complement in the first place. So if that's knocked out by the Soliris, then it stands to reason that the RBCs will be the first to recover. Not a very technical explanation, but that's my understanding.

Since I also have MDS, my blood cells have not all bounced back. However, with straight PNH, Soliris should lead you down the pathway to happiness and good health.

Sue
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Sue, age 72; Dx 6/2010 MDS Int-2. Revlimid unsuccessful, began Aranesp 10/2010; additionally Dx PNH 2/2011, Soliris added 3/2011. ATG 5/2011, Cyclosporine 5/2011. Nplate 10/2011 to 10/2012 . Exjade began 12/2013 due to high ferritin level, discontinued 3/2014 because of increase in creatinine.
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  #12  
Old Mon Feb 3, 2014, 10:04 PM
ussoccer004 ussoccer004 is offline
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Orlando Hematologist

Alright, so I had my consult with the hematologist who I thought diagnosed my condition with PNH, and turns out while I was in the hospital it was the internal doctor that ordered a CD (with some numbers) test and diagnosed me. The hematologist said it is a specific test that pin points PNH, she didn't see the Flow Cytometry test and said the HAM test is out dated and they don't use that test to diagnosed.

The hematologist did not know too much about PNH as I was led to believe. She also seemed distant and cold with me, like I was wasting her time. So what I will be doing is keeping my hematologist at the Veterans hospital because she has been a wealth of knowledge for me and has actually been supportive and treats me like a patient not a number or statistic.

I also found out that with the Soliris that it only helps the Hemoglobin, and if you do have an underlying issue with the bone marrow this has to be treated also. As many with PNH have AA or another issue going on.

I think at this point through the help with those on this board (and I thank everyone on here for the support you guys are great!) that I am going to see a specialist close to Orlando, FL which I believe is Dr. Carlos DeCastro for a consult and possibly see them there once a year and have them, along with my Hematologist keep in touch and hopefully be able to work together in this long journey.

I am excited also and lucky that NORD I believe it is, is having a conference about PNH here in Orlando, FL this week. I don't know if it was meant to be (Sarcastic) or what but I feel a little better. It is nice to know the support is out there with this board and NORD and other organizations.
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  #13  
Old Tue Feb 4, 2014, 12:14 PM
Neil Cuadra Neil Cuadra is offline
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Quote:
Originally Posted by ussoccer004 View Post
I am excited also and lucky that NORD I believe it is, is having a conference about PNH here in Orlando, FL this week. I don't know if it was meant to be (Sarcastic) or what but I feel a little better. It is nice to know the support is out there with this board and NORD and other organizations.
Here are the conference details.
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  #14  
Old Mon Feb 10, 2014, 08:36 PM
Relentless Against SAA Relentless Against SAA is offline
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Smile Soliris is saving lives

If anyone has any questions about Soliris, Alexion (the company that makes Soliris) encourages patients to call 888-765-4747.

The Alexion case manager said that Soliris could improve all three blood lines, but the main effect will be a rise in RBC.

Soliris Statistics from their patient info booklet
92% fewer blood clots
86% less hemolysis as measured by LDH
Reduced average number of RBC transfusions by 73%
Improved energy within 3 weeks of starting Soliris.

Thought this info is interesting. The case manager said they would love to talk with anyone who has any questions regarding this med.
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  #15  
Old Sat Feb 22, 2014, 12:48 PM
TLL414 TLL414 is offline
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USSOCCER004 -

since this was diagnosis in service you should see Veterans service representative eg. Disabled Veterans of America (DVA), Veterans of Foreign Wars (VFW), etc.

Reason being that they can help you file a disability claim and you could be eligible for tax free compensation... but even more important is you would be entitled to free medical care (including prescriptions). You don't have to switch hematology doctors if you don't want to, but you do need a VA primary care doctor (and you could continue to see your own private doctors). If your private doctor's prescribe medicine, you just bring the scripts to the VA PC doctor and they rewrite them to the VA Pharmacy. You can pick them up or have them mailed to you.

I am 100% disabled and receive VA compensation and SSDI... it certainly helps if unemployable. Also my dependents (spouse, children) are entitled to free medical insurance, USCAMPUS and education benefits. Some states have additional benefits so check that out too.

Please Google VBN, you'll learn a lot there regarding what you could be entitled to for serving in the military, it's a forum board for veterans and family members of veterans. And by the way... thank you for your service to the country!

My apologies to those that this message doesn't apply to, but I thought it to be important from one PNH veteran to another.
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  #16  
Old Mon Feb 24, 2014, 01:17 PM
ussoccer004 ussoccer004 is offline
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Originally Posted by TLL414 View Post
USSOCCER004 -

since this was diagnosis in service you should see Veterans service representative eg. Disabled Veterans of America (DVA), Veterans of Foreign Wars (VFW), etc.

Reason being that they can help you file a disability claim and you could be eligible for tax free compensation... but even more important is you would be entitled to free medical care (including prescriptions). You don't have to switch hematology doctors if you don't want to, but you do need a VA primary care doctor (and you could continue to see your own private doctors). If your private doctor's prescribe medicine, you just bring the scripts to the VA PC doctor and they rewrite them to the VA Pharmacy. You can pick them up or have them mailed to you.

I am 100% disabled and receive VA compensation and SSDI... it certainly helps if unemployable. Also my dependents (spouse, children) are entitled to free medical insurance, USCAMPUS and education benefits. Some states have additional benefits so check that out too.

Please Google VBN, you'll learn a lot there regarding what you could be entitled to for serving in the military, it's a forum board for veterans and family members of veterans. And by the way... thank you for your service to the country!

My apologies to those that this message doesn't apply to, but I thought it to be important from one PNH veteran to another.
I really appreciate your help, did you get service connected for Aplastic Anemia or PNH? Thank you so much for your help from one veteran to another.
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  #17  
Old Mon Feb 24, 2014, 02:01 PM
TLL414 TLL414 is offline
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Originally Posted by ussoccer004 View Post
I really appreciate your help, did you get service connected for Aplastic Anemia or PNH? Thank you so much for your help from one veteran to another.
I did not have an illness in-service so I would have to get a independent medical opinion siting research/experience that would connect my in-service event to my current disabling condition. I can suspect the fact I was exposed to hazardous chemicals (Benzene, PD-680, Agent Orange) but mere speculation will never support a claim. I'm not too upset because I am already 100% Total/Permanent due to other issues I have.

You on the other hand, you have service records that I would imagine any hematologist would concur that the issue you had in service is directly related to the issues you face today. As a matter of fact, at a minimum if they said it was just as likely as not - the veteran gets the benefit of the doubt.

If you need any further info and would like to discuss by Private Messages please feel free to do so.
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  #18  
Old Mon Apr 28, 2014, 01:26 PM
Rea Rea is offline
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I continued the Soliris treatment even though it did not help my hemoglobin, LDH or haptoglobin. Because it had been shown to reduce blood clots, my doctor thought it was worth it. 89% of my cells were PNH cells, my LDH was really high and my haptoglobin never registered on the readings (the last two are common in PNH patients). I had to rely on steroids and blood transfusions.

However, I did not have a blood clot, so while it's not a guarantee that Soliris prevents blood clots, it seemed to have helped me.
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