Colin was diagnosed by doctors in Virginia with a rare and high-risk type of leukemia. The family faced an extremely short window of time to get to Children's Hospital of Philadelphia (CHOP) to meet a deadline for Colin to be enrolled in a clinical trial. Several months later, Colin had terrible pancreas complications from his chemotherapy that required complex care from a team with CHOP’s Division of Gastroenterology, Hepatology, and Nutrition (GI) and a partnership with interventional gastroenterologists at Penn Medicine, who successfully performed a procedure rarely seen in pediatrics.

In 2018, Colin, now 7, began having pains in his side and fevers. His mother, Allison, is a physician and associate professor of internal medicine at the University of Virginia (UVA). “Being a doctor, I could attribute it to a lot of different things,” she says. “In hindsight, it seems obvious it was leukemia.” Twelve days after UVA doctors diagnosed Colin with acute lymphoblastic leukemia (ALL), the results of genetic tests showed he had the rare Philadelphia-chromosome positive form (Ph+ ALL), which occurs in only about 3% of children with ALL.

More about ALL

As part of an international collaboration sponsored by the EsPhALL consortium and the Children’s Oncology Group, CHOP had just opened a brand-new phase 3 clinical trial for children with Ph+ ALL. “The trial is studying whether less-intensive chemotherapy in combination with a standard drug for Ph+ ALL can preserve the good clinical outcomes achieved with more intensive chemotherapy that is our current standard of care,” explains pediatric oncologist Sarah Tasian, MD. “We are also hoping to learn if children’s outcomes could even be improved due to a reduction in the toxic side effects of chemotherapy.” To participate in the study, a child with Ph+ ALL must be enrolled within 15 days of diagnosis. That gave Colin’s family three days from learning his Ph+ test results on a Friday to travel from Virginia to CHOP and get enrolled on the trial by Monday morning.

Allison quickly emailed Tasian and Stephen Hunger, MD, Chief of the Division of Oncology. “I got responses immediately,” says Allison. “They were so wonderful and gave me so much confidence.”

Colin was enrolled and randomly assigned to the group that receives less intensive chemotherapy. Tasian discussed with Colin’s family about eventually transferring Colin’s care back to UVA given the family’s distance from Philadelphia, but Allison and her husband, Chris, decided to keep his care at CHOP since the team had significant experience with and expertise in Ph+ ALL. At first weekly, then monthly, the family drove five-plus hours each way for Colin’s clinic appointments.

A pancreas complication and an agonizing wait

Throughout his chemotherapy regimen, despite being part of the less intensive treatment group, Colin had terrible nausea and vomiting. Then in July 2019, he had intense abdominal pain and was admitted to UVA. “He was miserable,” says Allison, with heartbreak in her voice. “We felt horrible. He couldn’t eat, had pain and fevers, and couldn’t keep down his chemotherapy pills to keep the leukemia under control. We felt completely stuck.” He was diagnosed with pancreatitis caused by one of the chemotherapy drugs. A few days later, the family drove to CHOP, worried enough that they had mapped out at which hospitals to stop along the way if Colin couldn’t tolerate the trip.

At CHOP, Colin was seen by Jefferson Brownell, MD, a pediatric gastroenterologist in the Pancreatic Disorders Program with expertise in pancreatic disease. Imaging showed fluid collection next to his pancreas, but Brownell and the family agreed to hold off on performing any procedures because intervention is much safer once the fluid collection has matured into what’s called a pseudocyst. “It was a nightmare,” says Allison. “We didn’t know how long it had been there, and you can’t drain it unless it’s been there for a while.”

Colin was put on medication to treat bacterial infections, and the family returned home. “We had a chart for keeping track of his pills and IVs,” says Allison. “He got a sticker for every pill he kept down. It was so hard — a kid shouldn’t have to go through that.”

‘Rare in pediatrics’

More about Pancreatic Pseudocysts

After the course of the medication was completed, however, the severe abdominal pain returned. Imaging indicated Colin did indeed have a pseudocyst. In fact, it had grown larger and was pushing on his stomach, causing pain and nausea. Brownell and the team thought that instead of the typical surgical intervention, which would require a drain to the skin, a prolonged hospital stay, and a higher risk of complications, the best option would be to drain it into the stomach with a specialized endoscopic procedure using a new piece of hardware called an Axios stent. “This is relatively rare procedure in pediatrics,” explains Brownell, “and Colin would be one of the smallest patients to ever have this stent placed.” They contacted interventional gastroenterologists at Penn Medicine, who performed the drainage. Colin’s pain and vomiting rapidly resolved after successful stent placement. Unfortunately, his pseudocyst recurred a few months later, which is a potential risk of endoscopic drainage. The team recommended the same stent with a modified technique to prevent recurrence. This time the pseudocyst has stayed away for good.

“Dr. Brownell’s coordination with his team and the doctors at Penn got Colin back to being a normal kid,” says Allison. “He began eating normally — vegetables and everything!”

Now, a year later, Colin recently finished his chemotherapy and is a happy and healthy second grader with a head full of curly hair. “He’s doing absolutely amazing,” Allison reports. “He’s going to virtual school. He loves to ride his bike, play soccer, and play with his little brother, Owen.” During treatment, while in the hospital or at home, he had long stretches of time to polish his Lego construction skills, and so to celebrate the end of chemotherapy, Allison and Chris got him a 6,000-piece Lego Hogwarts castle. “He finished it in two-and-a-half weeks, all by himself, while still doing schoolwork and playing for an hour outdoors.”

When speaking of the care Colin received at CHOP, Allison’s voice fills with emotion:

“Drs. Tasian and Brownell are such wonderful and kind people who take care of their patients like they are their own kids. I can’t speak highly enough of them and their teams.

She notes that people find themselves at CHOP because their children have complex conditions. “Through it all, they take such good care of the whole family.”