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Posted June 19, 2013
iPICS Enrolls First Patient in Groundbreaking Study

Dr. Neil Goldenberg, Angela Sanborn, and Frances Hamblin, RN
Angela Sanborn (center) with Dr. Neil Goldenberg and Frances Hamblin, RN.

It happened unexpectedly late last month, a simple question posed by the mother of a teenage girl recovering at All Children's from treatment to break up and dissolve a blood clot that extended from the deep veins of her calf to the inferior vena cava in her abdomen.

But the seemingly mundane inquiry represented a major milestone in the hospital's research mission.

Karen Sanborn's daughter, Angela (who, along with her mother, gave permission for their names to be used in this article), had just finished a follow-up examination by Dr. Neil Goldenberg, Director of Research at All Children's Hospital Johns Hopkins Medicine (ACH JHM) and of the Pediatric Thrombosis Program that spans the St. Petersburg and Baltimore campuses of Johns Hopkins Medicine. It had been a difficult and frightening ordeal for Angela and her family, and Karen wondered if her daughter's experience could somehow benefit others in the future.

"I asked Dr. Goldenberg if there was any research being done on the kind of thing Angela had," she recalled. "We'd love for other people in the field to learn from our experience."

The words were music to the ears of Dr. Goldenberg and Frances Hamblin, Manager of the new and innovative Clinical and Translational Research Organization at All Children's.

They had just opened the ground-breaking ACH JHM Institution - wide Prospective Inception Cohort Study (iPICS). iPICS is a one-of-a-kind study among children's hospitals and pediatric academic medical centers, designed to identify key predictors of outcome in children with a variety of acute and chronic health conditions, both medical and surgical. The information being collected includes both the clinical variety derived from routine care, as well as blood and other samples collected for the research - usually done when these samples are already being collected as part of usual care. The ultimate goal is for the new knowledge gained through the iPICS study to accelerate improvements in treatments and outcomes for these pediatric illnesses.

Completely unbeknownst to Karen, the study had just been opened, and was seeking its first eligible patient.

And suddenly, here she was.

Dr. Goldenberg was quick to reply that such a research opportunity did indeed exist, adding with a smile, "I can take the extra time, if you can - let's talk."

And so began a landmark step in what many leaders at ACH JHM believe will transform pediatric medicine and research. The Sanborns, who reside in East Lake, followed up with Hamblin in a full informed consent discussion and signed the papers on May 30. A week later, on June 7, Angela arrived with her mother for her follow-up appointment in Goldenberg's multidisciplinary Thrombosis Clinic at ACH JHM, and her first official study visit in iPICS.

Hamblin, who played a key role in putting the study together with Goldenberg and colleagues, met Angela and Karen in an examination room (father Scott was unable to attend). She couldn't get over the serendipitous timing of their participation and expressed excitement over the potential future findings of the initiative.

"The study doesn't determine how she's going to be treated, but we want to be able to collect information about how she's being treated with the current best approaches, and how she responds," she explained. "Then we'll enroll other kids like her and we can compare those children who had very good outcomes to those who unfortunately didn't do as well. This will allow us to determine what are the factors that determine the outcomes, so that we can modify them in future research... leading to better and better outcomes over time."

Dr. Goldenberg reflected on the momentous nature of the day. "It's important because of the impact of this study and the amount of diseases we'll be addressing," he said. "When we have this iPICS study well under our belts, and we're recruiting patients and collecting data and samples in a large variety of illnesses, we'll then embark on a second version of iPICS, which will focus on healthy children."

In the future companion study, "Healthy  iPICS", Dr. Goldenberg and his collaborators at both ACH JHM and Johns Hopkins University in Baltimore plan to discover risk key factors for developing illness among otherwise-healthy children, including risk factors for developing obesity. The goal of that study is to rapidly gain knowledge that will to better strategies of disease prevention in children.

"The current version of iPICS focuses on kids who have already developed conditions and we're looking at how to improve their outcomes," he said. "And the second piece will be really helpful as a step even earlier in the disease spectrum. To make the biggest impact on children's health, you need to address the full spectrum – from health to disease onset to long-term outcome of disease."

Being part of such a monumental program was the last thing on Angela's mind a month earlier. She was a normal, healthy 15-year-old attending East Lake High School and enjoyed workouts at the gym. But she began experiencing pain in her groin area, which doctors at a local hospital thought was a muscle strain.

"But it got worse and, within a week, I went to the hospital and they transferred me here to All Children's," she said.

A battery of "CAT" scans and ultrasounds followed. And that's when it was discovered that she was suffering from a blood clot ("deep vein thrombosis", or "DVT") that was extensive throughout the lower venous system of her body -from the level of her ankle to above her kidneys. By now, her leg was purple and swollen and the pain was excruciating. The clot was provoked by a medication she had taken for six weeks, but she was more susceptible to DVT due to certain preexisting anatomical conditions she hadn't even been aware of until arriving at ACH JHM.

"I was shocked and nervous at first and was like, 'Oh my gosh, I'm going to have to have to get my leg amputated, or I may die,' " she recalled. "I was so nervous, and anxious to find out how I was going to be recover." A first procedure was performed, but the clot returned. So a second procedure followed, involving a more aggressive approach with a few devices as well as clot-buster medicine and blood thinners, to eradicate the clot. The multi-modal treatment was a success, leaving Angela pain free and elated.

"I'm so glad they transferred me to All Children's," she said. "When I got here, all the nurses were all so nice to me. I don't feel  like it's a hassle to come back because I like coming here."

"Everyone has had such a great bedside manner," added her mother. "And Dr. Goldenberg is world-renowned. So we know we're in the right place."

She used a wheelchair to get around East Lake High in the final weeks of the school year and began walking on her own, wearing a compression stocking, only two days prior to her follow-up appointment in the Thrombosis Clinic and her enrollment as the first iPICS patient.

Angela, who just turned 16 and will be a high school junior in August, may still have blood clots in the future and issues with veins. "We don't know what's going to happen, what her body will do," Karen said.

Dr. Goldenberg, however, expressed cautious optimism. "Our experience has been that, with careful identification of underlying risk factors for thrombosis, and addressing those factors through treatment and prevention strategies, fewer and fewer children with even severe blot clots are developing second blood clots later on."

And there is no doubt her participation in iPICS will potentially help many other children in the future. She will help provide a basis of comparison for future patients dealing with similar problems. Yet thrombosis is only one of the diseases being explored in the IPICS program.

"We have different cohorts, different groups of patients," Hamblin explained. "Thrombosis is the one that Angela qualified for. Going forward, we'll enroll many patients who have all sorts of medical illnesses and surgical problems, taking the same approach of collecting information on how children are treated and what their long-term outcomes are, and then making the connections that we hope will lead to better outcomes in the future."

It is all part of the greater research mission that Dr. Goldenberg directs, and that has become a driving force at All Children's Hospital Johns Hopkins Medicine.

"This iPICs study, and its sister study ("Healthy iPICS") that we're currently designing, will have a huge impact on the research mission at ACH JHM," Dr. Goldenberg said.  "We don't envision ever stopping... these studies will be part of the ongoing process of achieving better and better outcomes for children through continuous advancement in knowledge. The medical field has been historically been slow to learn about how children respond to today's medical treatments...but we are committed to changing that."