Rituxan questions

Have a 14 year old daughter, w/ chronic ITP, diagnosed 5/11, platelet levels bw 4-6k.
We have tried steroids, IVIG, and some natural remedies.
Now at a point where we are considering Rituxan.
We are worried about the safety of the Rituxan, considering it is a fairly new drug, I am worried about long term effects.
Anyone w/ personal experience or knnowledge about Rituxan, and its safety/effectiveness?
Also, anyone know the % of chronic childhood ITP patients that still resolve on their own after 12 months? We were told less than 1%, but thought we read otherwise?
Thanks

Rituxan has been around since 1998, used for ITP since 2001 or 2002. I first used it in 2003. It has a pretty good track record and so far, long term side effects have not been reported (other than PML).

It's a gamble. I know it's hard to determine benefit vs. risk, but that's what you have to do.

Having been around here for a very long time, I can tell you that the odds of your daughter's ITP going into spontaneous remission is better than 1%. Most do need a push though.

Just got back from the UK ITP Conference. Paediatric ITP doctor there said that she didn't think Rituxan should be used with children at all because it hasn't been trialled properly and the long term effects are not known. She said she would rather a child do a trial of one of the new TPOs than use Rituxan.

Oh and I can't remember the percentage of children who eventually remit. Ali might remember but it was way more than 1%, I seem to think it was something like 80% within 15 years.

Ann - I missed you AGAIN! - although I did see your name badge on the reception desk.

Yes, I think your figure is correct, and it matches with Dougie's consultant saying that he would expect around 10% of his chronic patients to spontaneously remit each year. So more than a 1% chance.

I went to the discussion group about childhood ITP and that doctor elaborated a bit on the rituximab in children issue. As I understood it, her argument was that children's immune systems are still developing and no-one knows what damage knocking a part of it out will do - whether it fully recovers, or whether you are left with a permanent deficit of some sort. She did however feel that the danger was greatest in children under 5 whose immune systems are still very under-developed.

There are two things that worry me about rituximab

a)I don't think it has ever been properly tested for ITP or for children

b)The odds aren't that great - it only works long term for about 1/3 of patients, but regardless of whether it works or not, it will knock out part of your immune system - I think for my son, I'd prefer to take the odds on a low platelet count.

Ali

Thank you for the replies. I knew I wasn't comfortable with the Rituxan, since there is no way of knowing long term effects yet, esp on children.
Now I have 2 more questions!
What is PML?
And the new TPOs?

Amazing how much wisdom, experience and knowledge is on this site!
Thank you!
Juliette

jmsteven,
I too have a daughter with ITP. She was diagnosed a month before her 13th birthday. She is now 15 and in remission due to Rituxan. We tried various meds for a year prior to trying Rituxan. It was a difficult decision for us but so far it has been the best decision. She is a very active athlet and plays select soccer. She had a difficult time giving up activities and did not like what the other medications did to her and how they made her feel (steroids, vincristine, methotrexate).
We were told by our doctor teen girls with ITP are less likely to remit then others. So we went for it and out of all the meds we tried it was the easiest with the less side effects.
I always worry about what the future may hold and if there maybe long term effects we don't know about, when I try to look into it I can't find any. So for now, we are blessed she is able to be a normal active teenager and enjoy high school like the rest of her friends.
It was the hardest decision we have ever had to make. I so wish we didn't have to and I wish you the best in what ever you decide.
Best of luck to you and your daughter.
Shauna

Thrombopoietin (TPO) receptor agonist - Nplate, Promacta. Basically drugs that stimulate the bone marrow to produce more platelets, so even though they are still getting destroyed, there are still enough to stop bleeding symptoms. My son is probably about to start a paediatric trial of eltrombopag (promacta)

Progressive multifocal leukoencephalopathy (PML), also known as progressive multifocal leukoencephalitis, is a rare and usually fatal viral disease that is characterized by progressive damage (-pathy) or inflammation of the white matter (leuko-) of the brain (-encephalo-) at multiple locations. My son's consultant says that it has been seen as a side effect of using rituximab, but only in cancer patients whose immune systems are otherwise very damaged. He says that it isn't worrying for use in ITP patients.

Hope this is helpful

Alison

My now 13 year old son has had ITP for a year. We took the watch and wait approach and his platelets rose up to 154k at the 6 month mark only to watch them fall again to single digits in February of this year. I wanted to stick my head in the sand and hope for the best but obviously that is not an option. I chose a 2nd opinion and went to see Dr B in NYC who recommended Rituxin and Dexamethasone together. While doing my due diligence and research, his platelt levels dropped to 2k for 2 weeks with lethargy, stomach pain and numerous large bruises and petechaie. Had IVIG after a night in the ER and platelets went up to 150K for 7 days before plummeting to 2k again. Though he has never had any wet bleeding, I had to treat him like the boy in the bubble..no sports, no bounce house birthday games etc so decided to go with the Rituxin/Dexamethasone treatment.

He started last week on a 4 week course of Rituxin with Dexamethasone every other week on weeks 1-3-5. He definetly had some tough days afterwards with stomach issues but Dr's thought was more from the steroids than the Rituxin. Of course I am concerned about putting these drugs in my son but after a lot of research, I went with my instincts in the hope that this would help. There are multiple articles that support Rituxin with apparent higher levels of sustained remission than other interventions. It is fairly new as Sandi cited so there are no long term studies but just last Tuesday 5/8/15 "Blood", the Journal of Hematology released an online article that further supports Rituxin.

I chose this tretament for him for a number of reasons - Rituxin targets B-cells where antibodies to Platelets are produced, % of remission is fairly decent in studies (~ 65% patial and complete remission) my son is 13 and also 5'8" 170 lbs and of course because his MD recommended it and he is internatioanlly known for ITP. Everyone has to make the decision that is right for them and my fingers are crossed that this will work out and allow my son to return to a more active lifestyle.

I will try and attach some of the articles here but the most well known research appears to be Zaja er al in the "Blood" Journal.

Blood April 8, 2010 vol. 115 no. 14 2755-2762

I hope this helps

The online article from last week 5/8/12 is only an abstract but I emailed the Journal of Hematology and they forwarded it to me. You can try this route but it should be published in next months "Blood" which is the Journal of Hematology

My son was diagnosed at age 5. At age 8, he crashed and nothing really worked well. With the years I had been on this site and reading alot of people experiences. The only treatment that I saw that got a remission response (besides spleenectomy - which was not an option for me for my young son) was Rituxan. Aaron is also seen by a cardiologist and everyone signed off on it. Aaron was part of the Rituxan study for children with ITP out of Boston's Children's Hospital. Though he did not get any response from it, he also did not get an negative response. But, I knew that all the good responses appeared to be in the teens and adults. But, I researched and we decided this was the best approach for him. You have to weigh all options. He went into remission at age 11 and just came out of remission just in time for his 16th birthday. I am a non-treater. He has been dropping slowly over months and he is doing well, no bleeding and he feels good. I feel that treatments raise the platelets up but they always seem to come down fast. I believe that it's the sudden drop that brings the major symptoms. When I read about kids that were refractory to treatments and lived with platelets less than 5-10K, I believe their bodies adjust. We are currently at 13K and they want me to treat at 10K, but I told them probably not if he is doing well and why. If he was to get really bad, I would consider Rituxan as a treatment option for him; especially since he is a teen and adult sized (hoping he might work better). It is very scary making these decisions for our kids. We research and make a gut decision. No one can tell you what is best for your situation (each person and situation is different - hence the frustration with ITP). We know our children best and we must advocate for them. Never feel pushed into something you don't feel comfortable with. I know this is much easier said than done. I have lost my footing before; been told I was hurting my child and have signed waivers not to treat. Good luck and I hope my story helps.

Hello, all!

Side effects and long term effects are always a concern, and after reading all we had to sign before Rituxan, we were!
And that was before they knew about PML! But I am here to tell you my daughter Caitlin had a few minor side effects at or after her 4 Rituxan infusions in Sept.-Oct. 2002, was followed closely by her hem-onc for five years with no negative findings, and now, 9 years, 8 months later, has still has had no apparent negative long term effects!
In fact, the only long term effects have been positive--as in no worries, far fewer med. bills, no restrictions! Her platelet count was still in the normal range at last count, in 2011.

She was 14 when diagnosed, WinRho and IVIG did not produce much effect, steroid pulses had worked, but were taking a real toll on her. ITP got in the way of her life, as she wanted to live it. She received Rituxan at 15.

And hasn't needed a blessed thing since!

We love Rituxan! And we found out about it here! Thank you, PDSA!

Peg, so nice to hear Aaron's gotten a break! And he's hip-deep in Scouting, even flying solo! Congrats! And nice to hear about Shauna's daughter, too! Race ya!