That black box warning

I have written before about how hard it is to give my sweet Hannah Joy a medication with a black box warning. Any child receiving chronic transfusions has to receive some type of medication to remove the consequences of those transfusions. Otherwise, the consequences will eventually kill. We essentially have two medications to choose from. One is a pump, worn for 10 hours (while sleeping, in Mia's case) infusing the medication by needle. The other is the medication which is crushed and put into food, which carries the black box warning. There is one other medication recently approved by the FDA, but our hematologist does not recommend it. This is because it requires weekly blood counts due to a side effect which can make the person prone to serious infection and death.

The medication with the black box warning has that warning because it causes very serious side effects, sometimes leading to death. Side effects like kidney damage, including kidney failure; liver failure; GI hemorrhage. Over the past few years, I have come into contact with parents whose children were hospitalized due to side effects from the black box warning medication. One mother told me how her daughter almost died from multi organ failure. I heard this before Mia even came home and I would need to make that decision about which medication to try. In Mia's case, it was easy. We tried the black box warning medication, which did nothing to help her. Our hematologist said that this is the case in 1/3 of patients, so Mia is just one of them. She had to switch to the pump. There was no choice. Mia handled the switch to the pump beautifully. She tells anyone who asks she prefers the pump. She hated the nasty tasting black box medication, and would prefer having a needle inserted into her thigh for 10 hours each night. Other parents' children have not had multi organ failure, but have had hospitalizations. Some for vomiting blood after the medication had eaten away at the child's GI tract. Others for severe abdominal pain. Some for kidney issues. All of these parents had to stop the black box medication and change their children to the pump.

Hannah Joy's hematologist told me that they carefully monitor for any signs of side effects, which brings a tiny degree of comfort. I have also learned that there have been children, and adults, who have taken the black box medication for 8-9 years (it was only approved by the FDA 9 years ago) and have had NO side effects at all. None. There is some comfort in that too.

Hannah Joy hates needles. She has been traumatized by insertion of her IV's, and now expects multiple pokes even when the IV is inserted on the first try for a transfusion. She tries so hard not to cry. But it hurts her. The thought of needing to poke her myself, every night, to wear the infusion pump is a thought that no mother should have to entertain. I am supposed to comfort her when she is hurt. Not cause the hurt. And yet, could I be causing harm when I crush up that medication and feed it to her in her ranch dressing or ketchup?

There is no clear cut answer for this one. No easy answer. Without a doubt we need more medications to choose from. Medications which are both safe and effective. Medications, which don't carry that black box warning or require parents to traumatize their very young children. Even better would be a cure for thalassemia, and other conditions requiring regular blood transfusions.