Question about Retuximab for my son aged 13

Hi, Our son have been struggling with ITP for the last 6 years or so. He also has DiGeorge syndrome with a compromised immune system. His bone marrow produces lots of platelets and WBC etc. After numerous test we do know its his own immune system that is attacking the platelets and now recently his WBC. We have tried WinRHo, platelets, IGG and steroids. He never acts like others for side effects etc. On the steroid he had no side effects at all. Of course they also did not work like everything else. The next thing the docs want to try is Rituximab which we are very against. After this the next thing they will push for is spleen removal. He has also had heart surgery and has kidney stones related to his Digeorge syndrom. His platelet count bounces around 0 to 5000 and his WBC is 1.9. Yet he is rarely sick, minor bruises & a very occasional nose bleed.

Our question have many teenager had good luck with the Retuximab? Also any bad reactions to the med? Our parental instinct is telling us no and we really want to go down a more natural route. We canno find anyone with DiGeorge syndrome & ITP so it worries us to even try this drug.

Thank you,
Skilcal

Skil:

I don't know that any of us can give you the right answers since your son has other medical issues. We can only tell you what we have experienced with ITP.

Rituxan sounds very scary, but most people tolerate it very well with few, if any, side effects. Quite a few people do better with Rituxan than they do with IVIG. If a person responds and attains remission, it can last, on average, a year or more.

I would be concerned since your son already has a low white count since Rituxan can destroy B and T cells (lymphocytes). It is temporary though and they do regenerate. The splenectomy would also be a scary move for that reason. Tricky call.

The two most well known adverse side effects are serum sickness and PML. Both are rare. Sometimes there is a reaction during the infusion, but that can be easily controlled with IV medications and slowing down the drip.

Hi Skil--

I had a student with DiGeorge who had several surgeries and breathing and heart issues early in life, but she really has no limitations on her activities now (she's also 10). Still, I know her mom was dreading the prospect of further surgeries to deal with jaw alignment, etc. How has DiGeorge affected your son--esp. major organs? How has he handled the other treatments--any major side effects?

As a completely unmedical opinion, it sounds as if either option has risks for your son. The one minus the spleen would be a permanent elevated risk. The one with Rituxan puts his immune system at a temporary elevated risk. The splenectomy carries a hope for long-term fix--if it works for him. The Rituxan carries a smaller hope for a long-term fix (although my daughter's Rituxan remission is now 8 years, 1 month and counting) but it doesn't remove an organ, and can be repeated. And if it doesn't work, splenectomy is still an option.

He could have Rituxan AFTER a failed splenectomy, but then he'd have a doubly depressed immune system...

Caitlin received Rituxan at age 15, after a year of ITP. She has no other health issues, but her BP went up to the 180's during infusion, and she had achey lower legs for a few days after Rituxan. She developed pneumonia between doses 2 and 3, but received antibiotics and was good to go on dose 3. (Pneumonia was going around at school.) Other than that mild pneumonia, after Rituxan, she really didn't seem to get sick more often than the other kids. We are big Rituxan fans!

Good luck, whatever you decide! Ann, Caitlin's (23)Mom

Hi, Thanks for all the information. In terms of the Digeorge syndrome he has had more trouble with the ITP. From the DiGeorge he had succesfully heart surgery at 6 weeks old, ended up with kidney stones from mismanaged medication. He also has delayed speech but so far has not had any surgery. His immune system just does not act like everyone elses. A chickepox vacination put him in the hospital for 1 week. So we have become very wary of medication effects. When he first got ITP he was having problems with UTI's . The ITP showed up after many doses of antibiotics. Strangely now he has never had Urinary tract infection again. Most of the studies into Rituxan have not been in children. Seems like most people have a reaction during the first infusion. We are worried his reaction might not be normal or life threatening. His doctors want to go ahead and try Rituxan but because of his immune system we do not. His immune system is very agressive and attacks his Platelets and WBC. So going into this he would have a low WBC before he even takes the drug. His zero platelets counts only showed up this year since we started trying IVIG, Platlets and steriods. So for the moment we are in a holding pattern researching.

Skilcal