When Cheryl Trocke’s nine-year old son Graham was diagnosed with melanoma four years ago, she quickly learned that when it comes to kids, there were no great treatment options and that care can vary greatly based on where your child is treated. After surgically removing the primary tumor, Graham’s doctors suggested a treatment plan of ‘wait and see.’
“I call it the ‘sitting duck plan’ because that’s what it is. We watch and wait and hope nothing comes up. Any research that has pediatric attached to it is important because clinicians just don’t know what to do because we just don’t have the research to back it up,” says Trocke.
That’s why last month’s approval of ipilimumab by the U.S. Food and Drug Administration (FDA) to treat pediatric patients 12 years and older with unresectable or metastatic melanoma is so encouraging. For the first time, immunotherapy—a game changer in the treatment of cancer among adults—is available for pediatric patients; giving patients and their families a new tool in the fight against melanoma.
Cancer of any kind is rare among children, but among skin cancers, melanoma is the most common. In fact, about 2% of all childhood cancers are melanoma. This year, more than 87,000 people will be diagnosed with new melanomas. Of this number 0.4%, or about 350, will be under 20 years old.
Early detection and care is key to the success of melanoma treatment. Unfortunately, early melanoma detection is complicated in kids. The signs of pediatric melanoma are frequently overlooked or misdiagnosed as other minor skin problems. In fact, in a 2013 study, 60% of children between the ages of 0 and 10 and 40% between 10 and 20 years old did not meet the ‘ABCDE’ melanoma detection criteria—Asymmetry, Border irregularity, Color variation, Diameter over six millimeters, and Evolution of the lesion.
“All of these things fall by the wayside for pediatric melanoma. When parents of kids with melanoma get together, and we talk about the primary tumor site, they are all different. Pediatric melanoma is regularly quite different and it can feel so scary,” says Trocke.
Until last month’s approval of ipilimumab as the first immunotherapy agent approved for children, metastatic pediatric melanoma did not have any FDA-approved therapies available. As for adult melanoma patients, the mainstay of care is surgical excision. Studies also show that children treated for melanoma should be closely monitored as they are at increased risk of recurrence later in life.
Ipilimumab’s approval was based on positive results from two clinical trials which demonstrated a consistent safety and efficacy profile across pediatric and adult patients. Of the 17 pediatric patients over the age of 12 studied, two experienced objective responses including one partial response that was sustained for 16 months. While generally safe, ipilimumab is required by the FDA to include a boxed warning that the drug is associated with severe to fatal immune-mediated adverse reactions. The drug remains unapproved for children under the age of 12, however numerous clinical trials are underway to examine other therapies and their suitability for pediatric patient use.
Today, Trocke is thrilled that Graham, now 13-years old, is still doing well and is happy with the care that Graham gets at the Mayo Clinic. She gives back by supporting other parents who are going through similar situations. Other parents email, call, or message her and the biggest piece of advice she offers is to “never settle and to get other opinions so that you understand what is out there in terms of options.”
“Now, knowing what I know, I’d go to the top cancer centers in the country. I would get all the info. Most parents don’t know they need to do that. If you get pneumonia, there is a treatment plan that is approved and anywhere in America you’ll get that treatment. Right now, for pediatric melanoma, we don’t have anything like that,” says Trocke.
The approval of ipilimumab as the first immunotherapy drug for pediatric use is a critical step to ensuring that all patients, regardless of age, have access to the highest quality medications possible.
“That feeling in your stomach when they tell you your child has cancer... it never goes away. Some days it is worse than others, but I will always feel the guilt, did I do enough? You just don’t know. I’m grateful that research is finally starting to catch up so that we have more options,” says Trocke.