Sky Smith: Oh, Jude is just so fun. He's very active, very playful, just silly as can be.

Dennis Douda: Jude Smith has all the rambunctious energy a 3-year-old toddler should have.

Sam Smith: He loves trying new things. He loves getting active, getting outside.

Dennis Douda: But a year ago his typical playfulness raised some frightening red flags for his parents, Sky and Sam Smith.

Sky Smith: And we noticed a lot of bruises. They were in strange places, like on his stomach, all over his face.

Dennis Douda: Jude developed uncontrollable nosebleeds and red, rash-like outbreaks on his skin, called petechiae. Initially medical tests were inconclusive.

Sam Smith: Oh, it was terrifying. It was terrifying. I mean, it was very upsetting. It could be anything from leukemia to lymphoma to, we don't know what else.

Dennis Douda: Clues began to emerge from a closer look at Jude's blood.

Sky Smith: A normal platelet count is 150,000 to 400,000, and his platelets were only 10,000.

Behzad Bidadi, M.D. — Mayo Clinic Pediatric Hematology Oncology: They have low platelets and this increases their risk for bleeding because the job of the platelets is to stop bleeding.

Dennis Douda: Meaning a bump to the head and bleeding in the brain could be devastating.

Pediatric Hematology specialist Behzad Bidadi says figuring out what was making Jude sick was really a matter of eliminating all the other possibilities, including certain cancers. What was left was an autoimmune disorder — called ITP.

Behzad Bidadi, M.D.: We don't exactly know what causes ITP, also known as immune thrombocytopenic purpura. We believe that the immune system in the body mistakes the platelets for being foreign and attacks them.

Dennis Douda: Under a microscope it's easy to see just how drastically ITP reduces platelets in the blood.

To try to reverse the damage, doctors gave Jude multiple rounds of intravenous immunoglobulins to suppress his over-active immune system. When that ceased to be effective after a few months, they tried heavy doses of corticosteroids, then another immunotherapy drug made of antibodies; all proven treatments that work for the majority of patients.

Behzad Bidadi, M.D.: More than 80 percent of children diagnosed with ITP have the ITP actually resolves within a year and the patient's platelets counts go back to normal.

Dennis Douda: That was not the case for Jude, unfortunately. His ITP proved to be a chronic refractory variety, that would not stay in remission. The outlook became so uncertain, Jude was given a once in a lifetime trip, the kind often reserved for children who may not get another chance.

Sam Smith: It was a very emotional experience.

Sky Smith: Right, on one hand, it was so exciting. We have this incredible gift and then on the other, it was like I can't believe that my kid is going on a Make-A-Wish trip.

Sam Smith: His wish was to go to Disney World to meet Lightning McQueen. He's a huge fan of Cars and he's got this whole thing where when he would go to the hospital, every time he would go he would watch Cars on demand.

And he just ran right up to Lightning McQueen and gave him a big kiss, like, on the quarter panel.

Dennis Douda: Meanwhile, Jude's team of providers was planning its next approach for managing his ITP.

Sky Smith: The detective work has been incredible. I feel like we joke that his doctor's staying up late, researching things just for Jude.

Behzad Bidadi, M.D.: It's not just a destruction of the platelets by the immune system, but there also could be a problem with platelet production.

Dennis Douda: So, once a week, Jude gets an injection of Romiplostim, which basically stimulates the bone marrow to produce more platelets.

Sam Smith: It's been tried and tested in adults, but it's experimental and not FDA approved for children.

Dennis Douda: So far, it's working very well. Jude's platelets counts are nearly normal. So Sam and Sky can relax, well, at least as much as any parents with a rambunctious toddler.

Sky Smith: Jude's resilience has really carried us all through this.

Dec. 10, 2014