Histiocytosis Center

Patient Story | Johnny

Successful Drug Treatment Leads to Healthy Turnaround for Patient with LCH

Robinson Family Traveled 500 Miles so Johnny Could Receive Care from Ashish Kumar and Our Histiocytosis Center

Three-month-old Johnny Robinson was very sick when he first came to Cincinnati Children’s from his home in Virginia.

“He just seemed to be in so much pain and discomfort when we were in Richmond,” said his dad, Peter. “He wasn’t sleeping. He wasn’t eating. He could barely cry he was so weak. They had him on morphine he was so uncomfortable.” 

Two weeks later, his health dramatically improved. Johnny, who was born with Langerhans cell histiocytosis (LCH) and was in pain most of the time, had improved so much during his stay at Cincinnati Children’s that the family would be sent home the next day.

“It was shocking, the turnaround,” said Peter.  “Just two weeks later and he’s looking like a normal kid again – just smaller than he should be. It was astounding.”

An astounding outcome for the family, but not surprising to Johnny’s doctor, Ashish Kumar, MD, PhD, co-director of the Histiocytosis Center. In 2016 he first began using a new drug therapy to treat two patients who were not responding to traditional chemotherapy – the most common form of treatment for patients with LCH.

Both patients made a full recovery using the novel therapy, which involves an oral drug (trametinib) traditionally used to treat melanoma. Years later, Kumar and his team have seen a 100% success rate in treating over 40 current LCH patients with this therapy, including Johnny.

LCH is a rare, cancer-like condition that oftentimes affects a child’s skin and bones. When a person’s body makes too many immature Langerhans cells they can form tumors or damage tissue, bone and organs.

Searching for Answers, Finding the LCH Center

The Robinsons, fearful of the possible side effects of chemotherapy, weren’t looking for the most common treatment to help their son; they were searching for the most effective. So when they discovered the Histiocytosis Center through a combination of online research and help from friends, they didn’t waste any time making an appointment. Hours after arriving in Cincinnati, Johnny began the treatment.

“When he got to us, he was in such a bad state. He was dependent on IV nutrition and was requiring blood transfusions on pretty much a daily basis and running fevers every day,” said Kumar. “If the family had not taken the initiative, I don’t think we would’ve given Johnny the successful life back that he got. His condition would not have stabilized and he would've continued to suffer.”

Instead, Johnny slowly improved each day. A rash discovered at birth, which had quickly spread from his lower back to his entire body, would be gone and healed by the time they left the hospital. 

“The redness was pretty much gone. To see him start to improve was such a relief,” said Peter.

Confidence in Therapy, Confidence in Dr. Kumar

The two-week timeline of recovery – assuming all went well – is what Kumar and his team correctly predicted for Johnny. This despite earlier concerns about whether the therapy would be effective or if surgery would be required, due to apparent blockage issues in his abdomen. 

“We discuss openly with parents that the standard of care is chemotherapy and this is the newer option that we are offering. We are not saying they must do this or that. We share the data transparently with them and ask what is more reasonable to them,” said Kumar. “It’s with parents’ faith and confidence in us that we have been able to help children like Johnny.”

Unlike the doctors in Virginia, Kumar was confident the therapy would work and no surgeries to address blockage concerns or chemotherapy would be necessary. 

“I don’t have a crystal ball, it was an expectation,” said Kumar. “In other patients this is what we see, so I’m not surprised to see things turnaround pretty quickly. And it happened with Johnny within the first few days. His mother was in tears because it was the first time he had smiled at her.” 

Peter recalls these early discussions and how easy it was listening to Kumar explain Johnny’s treatment plan.

“He said, ‘[Johnny] needs to go on this drug. He needs to do it as soon as possible. We’re confident we can administer it to him because we’ve handled other cases similar to Johnny’s.’ It was just very reassuring knowing that was the right thing to do,” said Peter.

The Important Role Parents Play

Parents taking an active role in their child’s medical care is something Johnny’s parents and Kumar agree upon. 

“This is a day and age where more information is available than ever. It’s not necessarily easy to wade through and decipher all that information, but it’s important,” said Peter.

“It’s very easy to just rely on what your doctor is saying because you don’t want to make the people who are treating your child mad or angry by questioning them. But you can’t expect doctors to know everything. And doctors care about who they are treating. But they don’t care more about my son than I care about my son.”

Today, Johnny continues treatment in Virginia and makes periodic visits to Cincinnati. Doctors will keep watching for possible side effects but everything so far has been positive. He is doing very well and will continue therapy for another three to five years — possibly longer — or until the genetic mutation that is causing the LCH (BRAFV600E) goes away.

“What we don’t know is how long patients need to take the drug treatment. For some, we need to be prepared that this could be a lifelong therapy,” said Kumar, adding that he and his team will continue to regularly monitor Johnny and all of their patients who are on these new treatments.

The good news is Johnny is happy and healthy. He’s smiling, eating well, gaining weight and hitting various milestones. Everything the family hoped for before coming to Cincinnati Children’s has come to fruition.

And at Johnny’s one-year check-up, the family received the best news they could hope to hear.

“The doctor told us the only difference between Johnny and a complete healthy baby is he takes this drug to keep his symptoms of LCH at bay,” said Peter. “Other than that, he’s fine.”

(Published February 2021)