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PNH Paroxysmal nocturnal hemoglobinuria

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  #1  
Old Thu Aug 1, 2013, 07:33 PM
topcow topcow is offline
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Question Son with PNH & MDS

Hello,

My 15 yr old son was diagnosed with MDS in 2011 and PNH just a few weeks ago. He suffered from viral encephalitis in 2004, and as a consequence of that he has seizures also. He has also had pneumonia a few times, and my question is about Soliris. One doctor we have seen has recommended it, and told us that most docs agree that they don't do BMT on a patient until they have tried Soliris. After reading everything I can find about BMT, PNH, and Soliris, I really do NOT want him to try the drug. Could some of you who have been on it let me know if I am being unreasonable about this?

My worry is that since he would have to have a port for Soliris infusions (his veins are shot) that it could get infected, or that the Soliris will make him more likely to get other infections. Infections are already something we deal with, and when he gets even a small one he starts having seizures. Any thoughts?
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Old Thu Aug 1, 2013, 08:20 PM
topcow topcow is offline
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I forgot to add that my son's LDH is 3,442 and he has >90% PNH clone size.
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  #3  
Old Thu Aug 1, 2013, 08:58 PM
Neil Cuadra Neil Cuadra is offline
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topcow,

Your son has a tough pair of health problems to cope with. I understand your concern and I would definitely get a couple of opinions from different doctors.

When you have a port, one issue is self-care. Some 15-year-olds would be more likely than others to be sufficiently careful with a port, keeping the area clean, not touching it, washing hands regularly, handling sterile supplies, cleaning the caps, flushing the port if you're in charge of doing that, and making daily checks at shower time to identify problems right away. It's worth considering where you think your son lands on the scale from hopelessly careless to dependably careful. Infections can't always be avoided but a lot depends on proper care of the port. Fifteen is an age when levels of responsibility vary widely.

If your son had a BMT so you know who the donor would be?

If the doctor decides to agree that it's medically appropriate to go straight to transplant then you might want to double-check if your son's medical insurance company is going to cover a transplant for PNH without prior treatment with Soliris. Presumably it should be covered but both choices are expensive (an insurance company's least favorite word) so finding out for sure might avoid problems later.
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Old Fri Aug 2, 2013, 12:54 AM
evansmom evansmom is offline
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My son had AA before his transplant, but with all the reading that I've done and continue to do about bone marrow failure diseases and PNH, if it were my son, I'd very seriously consider transplant even with his seizure disorder. His extremely high LDH indicates that he is having a lot of hemolysis. Plus the Soliris only addresses the PNH, not the MDS which is a pre-leukemic disease.

What are your son's other counts like (wbc, rbc, platelets, absolute neutrophils)?
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Nicole, mom to Evan (20); diagnosed SAA November 2007, hATG mid-November 2007, no response after 6 months, unrelated 9/10 BMT June 2008, no GVH, health completely restored thanks to our beloved donor Bryan from Tennessee.

www.caringbridge.org/visit/evanmacneil
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Old Fri Aug 2, 2013, 11:30 AM
topcow topcow is offline
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Thank you so much for the replies!

It is funny that your name is Evansmom, because my son's name is Evan too! The encephalitis in 2004 left him with mild brain damage, so he is more like a 7 year old than a 15 year old. So taking care of a port would be my responsibility.

We are having our other 2 sons tested next week to see if they are a match for Evan. His last blood work was done on July 22 and his counts were :WBC 3.64, HGB 10.4, platelets 194, HCT 33.1, and Retic 5.2

These numbers are higher than usual, because he was hospitalized in April and received his first blood transfusion (2 units). He sometimes aspirates during a seizure, chocking on his vomit, and it causes infection in his lungs. I can not imagine how much worse these infections would be if he was on Soliris and his immune system was even weaker!

We are getting another opinion from Dr. Dipersio at Siteman Cancer Center in St. Louis soon. Just waiting for an apt date right now.
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Old Tue Aug 20, 2013, 05:57 PM
Neil Cuadra Neil Cuadra is offline
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topcow,

I suggest that if possible you attend the AA&MDSIF Online Leaning Center webinar tomorrow on PNH. Click "Upcoming Webinars" and you'll see "Blood Cell Production vs. Blood Cell Destruction: PNH and Other Bone Marrow Failures" listed with a Sign Up button.
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  #7  
Old Thu Feb 19, 2015, 07:02 PM
GoodDay5150 GoodDay5150 is offline
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I am sorry to hear that your son has PNH/ MDS. I had a trans for PNH only abt 3 yrs ago and I am mostly doing well. My HMO/ spec clinic at the time advised me that I would need a trans; I had PNH for 8-10 yrs w/o knowing it or being diagnosed. The HMO spec. was going to treat me w/ Soliris but then changed his mind since he himself had nvr treated anyone w/ the drug and had some concerns. He did have a patient that was part of the trial b4 it was approved. I have been told that some insurance providers will advise patients that a trans is in order due to the high cost of Soliris. But a trans and all of the associated costs and follow up treatment and meds is also xpnsve. I wish you all luck and mp prayers are w/ you.
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  #8  
Old Thu Feb 19, 2015, 10:18 PM
amyangel amyangel is offline
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Hi TOPCOW

Hi Topcow ,
My daughter also had MDS and seizures ,
We found that if her Potassium levels were at a good level and he Magnesium also She didn't have any seizure on her seizure meds. she was on Phenobarbital , vimpat and onfi... Prayers to you this was by far one of the toughest journeys I had ever taken with her .

Amy went home to be with her heavenly father on February 11, 2015 she was 27
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Mother of a daughter 27 with MDS . Cognitively delayed at birth 1987 , seizures 5 days old . pancytopenia 2006, AIHA 2013, EVANS 2013 , CVID 2013, ALPS 2014. MDS 2014
8/18/2014
WBC .3 , hemoglobin 7.3 , hematocrit 2, platelets 60 , neutrophil .21 Mag 1.6, Potassium 4.6
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