23 blood transfusions & counting

We visited St. Jude in Memphis, TN April 2025. This was a big milestone in Charlotte’s journey. It was filled with a lot of emotions and a lot of big things we have to process and make decisions on.

Our visit was filled with meeting many doctors, specialists and care team members. We met with genetics, the rare bone marrow failure team, the bone marrow transplant team, a social worker, and the anesthesia team. We had a few lovely nurse navigators that guided us through our visit and walked us to every appointment. Everyone at the hospital was so kind and helpful! We were able to focus on Charlotte and St. Jude had everything else covered. Despite what our family is going through, St. Jude has truly been the biggest blessing!

Charlotte had a blood transfusion during this visit. She also had a sedated FerriScan (MRI of her liver), sedated bone marrow biopsy and sedated auditory brainstem response (hearing test). Charlotte was an absolute rockstar and we could not be more proud of her! This was a hard visit on us, but we know it was even harder for her. 

We still have many pending results, but we do have the results of the FerriScan. Charlotte has severe iron overload from her blood transfusions. Her LIC (liver iron concentration) was 8 mg Fe/g dry liver (severe iron overload is categorized as 7-15 mg Fe/g dry liver). Charlotte is currently on a low dose of Jadenu (iron chelator) but we will start to titrate up on her dose to be a bit more aggressive.

Next steps for Charlotte:

• Continue blood transfusions every ~3 weeks
• Continue taking L-leucine and Jadenu 
• Await pending labs and discuss with St. Jude team in Memphis and our St. Jude clinic in Charlotte in ~4 weeks
• Re-trial steroids sometime in the next 6 months - 1 year
• Follow-up at St. Jude in Memphis, TN in one year for repeat FerriScan, Cardiac T2* (MRI of the heart, iron usually stores here after the liver), auditory brainstem response and to meet with the rare bone marrow failure team again

Charlotte is the strongest girl we know! Despite everything she is going through and will have to go through in the future, we are still choosing hope. Hope is what keeps us going and we know this will be just a small part of Charlotte’s story!