The Pediatric Trials Network (PTN) has sent thank you cards to thousands of trial participants and their families this summer recognizing the critical role families play in advancing pediatric research. PTN has sent thank you notes domestically and internationally, translating the notes into foreign languages when appropriate.

The thank you note is customized for each study that PTN is coordinating. For example, if blood draws are a requirement of the study, then blood draws and the associated frequency are explicitly mentioned in the thank you note so that participants’ contributions are specifically acknowledged.

Researching the art of gratitude

The ability to send thank you notes did not happen overnight. In partnership with the BASE (Bioethics & Stakeholder Engagement) Lab, the Duke Clinical Research Institute’s (DCRI) Research Communications & Engagement team (RCE) conducted formative research in 2019 to understand how to make thank you cards and plain language results summaries an effective and meaningful engagement tactic for trial participants and families.

For this research, the RCE team developed three sample thank you notes, one with minimal text and graphics, and the other two with expanded text and images of children and families in the header. These notes were mailed to 24 caregivers and three adolescent participants in advance. Two trained interviewers from the BASE Lab followed up with one-on-one interviews.

The interviewees overwhelmingly preferred the expanded text/graphic thank you notes, with some saying that the version with the image of children and families made them feel like PTN acknowledged the whole family’s efforts in clinical trial participation. Some stated that an additional paragraph of text, which was missing from the other more streamlined version of the thank you note, did a better job expressing gratitude, recognizing participants’ time and effort, and making them feel that they were contributing to discoveries that would improve children’s health.

The added text was: “Your involvement in this study has been instrumental in helping us determine the safest and most effective dose of ampicillin for newborns. Without you and other volunteers like you, we could not conduct research that will improve medical care for our youngest patients. We are grateful to you for sacrificing your time and effort to help infants and children worldwide.”

Incorporating Gratitude into Studies

Ultimately, there are a variety of ways to make trial participants feel appreciated and important. Thank you notes are just one of many options and expressing sincere gratitude towards trial participants should always be a key focus of any trial design.  Other options could include using YouTube for thank you videos and disseminating them via social media or posting them on the study website. However, it is important to ensure that thank you messages and results summaries do not come across as promotional. Through the research process, the PTN team learned that it is important to remove any language that could be construed as publicity.

Danny Benjamin, Principal Investigator of the PTN, hopes to see wider adoption of thank you messages throughout more projects. “It’s just the right thing to do,” he said.

The 2020/2021 PTN Annual Report is now available. Click here to access.

A note from the PTN Steering Committee Chairs:

The COVID-19 pandemic has continued to challenge us in unparalleled ways and we are grateful for the commitment the Pediatric Trials Network (PTN) has exemplified in learning more about COVID as related to our youngest of patients. PTN’s Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children per Standard of Care (POP02) Study continues to make strides in supporting the treatment of younger COVID-19 patients and is currently evaluating several therapeutics to potentially treat COVID-19 pediatric patients younger than 21 years of age. In fact, POP02 has been engaged in a multi-institution, multi-study collaboration with National Institutes of Health (NIH) called The Collaboration to Assess Risk and Identify LoNG-term outcomes for Children with COVID (CARING for Children with COVID). This collaboration has worked to make de-identified data (or data that cannot be linked to a person’s identity) from children with COVID publicly available, in order to accelerate research on this important topic. The POP02 team was the first study to reach the important milestone of submitting these data and having them become available, and it is a testament to their tremendous effort and dedication. Click here for more on this. Additionally, this year PTN has released more than 20 publications and informed new label changes for clindamycin and diazepam. The Annual Review is recognition of all our hard-earned accomplishments over the past year. We look forward to all that is to come and am grateful for your commitment to determining the safest and most effective use of medications and devices in children.

The 2019/2020 PTN Annual Review is recognition of all the hard-earned accomplishments of the Network. We look forward to all that is to come in 2021.

Read the complete review here.

The PTN digoxin study has achieved more than 20% of participant enrollment goals and has activated all sites.

The study, which enrolled the first participant in the summer of 2019, will determine the pharmacokinetics (how a drug travels through the body) and safety of digoxin prescribed to infants with single ventricle congenital heart disease (CHD).

While digoxin has been approved by the U.S. Food and Drug Administration for the treatment of heart failure in children and adults, more information is needed, particularly in children with single ventricle CHD.

Each year 40,000 infants born in the U.S. are diagnosed with CHD. Single ventricle CHD is the most serious and complex form of the disease. Without intervention, CHD is fatal in the first week of life. Even with intervention, the risk of dying remains high. While three operations are included as part of the intervention phase, the highest risk of death occurs between the first and second surgeries.

“Unfortunately, there are no drugs currently proven to reduce death during this period. However, digoxin represents a promising drug to reduce mortality and is routinely used in this population,” said Dr. Karan Kumar, a PTN faculty member contributing to the digoxin study.

What is learned through this study will be shared with other researchers, healthcare providers, caregivers, and patients, with hope it will help infants in the future.

To review more about the digoxin study visit ClinicalTrials.gov.

Representatives from the International Children’s Advisory Network (iCAN), a worldwide consortium of children’s advisory groups that provide a voice for children and families in medicine and research, shared their insights at a recent PTN symposium held Sunday, April 28, at the annual Pediatric Academic Societies (PAS) Meeting in Baltimore, Md.

Reece Ohmer, a high school senior who is preparing to go to college in the fall, has lived with type 1 diabetes since she was 8 years old. She discussed the experience of “turning a negative into a positive” by using her voice to advocate for better research for young people.

She joined iCAN when she was 12, after an especially taxing doctor’s appointment. She had received upsetting lab results, but no one had engaged her in the conversation.

“The doctor was talking to my mom and my mom was talking to my doctor, and I pretended I wasn’t listening,” she said. “But I was.”

After the appointment, she tearfully shared her frustrations with her mother, who responded by asking her what she would do to make that experience better for another child. Reece eventually joined the hospital’s teen advisory council, which in 2014 became one of seven hospitals participating in iCAN.

Through the consortium, she has worked to advocate for better funding for pediatric research, ensure access for more children, provide input on assent and consent forms to make them more understandable, and verify that available research is actually the research children need.

“We’ve helped increase understanding within the medical community that in patient-centered care, even the youngest voice matters,” Reece said.

Building trust

iCAN representatives acknowledged that there is an element of distrust when researchers approach patients and families. Patients are often unsure where the researchers are coming from and whether they have their best interest at heart.

“They don’t know me and my life,” said Sophia Klaudt, an iCAN member and high school junior living with cystic fibrosis. “It’s hard to build that relationship with researchers when I’m just a case study to them.”

The iCAN representatives suggested that researchers may not be the best people to make the initial contact about research opportunities. “Have existing families who have already been through this experience help bridge that gap for you,” said Amy Ohmer, Reece’s mother and director of iCAN. “These patient ambassadors can share that empathy because they understand what it’s like to be in their shoes.”

When researchers need to approach patients directly, iCAN representatives suggested that a nurse or someone in a trusted position introduce them as a new member of the team who is coming from a genuine place of care and concern.

Encouraging adherence

The iCAN representatives acknowledged the challenges of getting adolescents, who enjoy more freedom and are less bound by their parents’ demands, to adhere to their medications.

To better incentivize patients, Reece encouraged clinicians to get to know them and where they are in their care journey. Treating a 7-year-old is completely different than treating a 17-year-old, she said, and each group has its own motives.

“When I was seven, candy would have motivated me, or a chart on my refrigerator,” she said. “Now things that would incentivize me would be being able to do things on my own independently of my family, which is something I had to work for.”

Sophia stressed the importance of compromise, and trusting adolescents to make responsible decisions. “Ultimately, I know it’s my health,” she said.

Amy Ohmer added that, as a parent, it is critical to stop scolding and start empathizing.

“We don’t say enough to our young people that what they’re doing is tough,” she said. “We need to remind them of what’s happening that’s positive in their life.”

Communicating with patients

Asked how researchers can better communicate with young patients, Sophia recommended that they tailor communications to the patient’s age and maturity level, and that they ensure the patient is informed throughout the process.

“I’ve been in studies where I felt informed at the beginning, but I wasn’t informed throughout,” she said. “[Patients] need to understand what’s going on throughout the whole process and be informed of the results.”

iCAN representatives reminded researchers to keep the patient first in mind when developing materials. They recommended putting the most important information front and center, providing clear timelines so patients get a better sense of the level of commitment involved, and thanking participants and informing them of the impact they have had. Finally, they encouraged the use of newer technologies to communicate with young people, meeting them where they are.

“We are the future, and we are going to be the biggest return on your clinical research investment,” Reece concluded.

Learn more about the 5th Annual iCAN Research & Advocacy Summit in Kansas City, Mo., June 24-28.

The Pediatric Trials Network (PTN) will be presenting its research at the Pediatric Academic Societies (PAS) Meeting May 5-8 at the Metro Toronto Convention Centre in Toronto, Canada.

The annual PAS Meeting brings together thousands of researchers, academics, and clinical care providers united by a common mission: to improve the health and well-being of children worldwide. It is supported by the American Pediatric Society, the Society for Pediatric Research, the Academic Pediatric Association, and the American Academy of Pediatrics.

During the annual meeting, PTN investigators will share information on a variety of studies involving antibiotic safety, treatment of hospital-associated and ventilator-acquired bacterial pneumonia, infant weight estimation, personal protective equipment, and the development of a data repository to aid in pediatric research. See a detailed schedule of PTN-related presentations below or view the online program guide on the PAS website.

Saturday, May 5

•  1:15 p.m. – Validation and Human Factors Evaluation of a New Device for Infant Weight Estimation – Dr. Susan Abdel-Rahman, Children’s Mercy Kansas City (Poster)
•  1:15 p.m. – The Use of PPE during Pediatric Resuscitation: Which Tasks are Most Affected? The Results of a Survey during a Simulation Trial – Dr. Aaron Donoghue, Children’s Hospital of Philadelphia (Poster)

Sunday, May 6

•  11 a.m. – The Impact of Personal Protective Equipment on Performing Critical Procedures for Pediatric Patients in the Pre-Hospital Setting – Dr. Maybelle Kou, Inova Fairfax Hospital (Platform)
•  3:45 p.m. – Population Pharmacokinetics of Milrinone in Infants and Children – Dr. Christoph Hornik, Duke University (Platform)
•  4:45 p.m. – Development of an Electronic Health Record Derived Multicenter Inpatient Pediatric Data Repository – Dr. Christoph Hornik, Duke University (Platform)
•  5:45 p.m. – Hospital Associated and Ventilator Acquired Bacterial Pneumonias in Infants and Children – Dr. Jessica Ericson, Penn State Milton S. Hershey Medical Center (Poster)
•  5:45 p.m. – Population Pharmacokinetics and Safety of Sildenafil in Premature Infants – Dr. Daniel Gonzalez, UNC Eshelman School of Pharmacy (Poster)

Monday, May 7

• 2 p.m. – Antibiotic Safety and Efficacy in Infants with Complicated Intra-Abdominal Infections (cIAIs) – Dr. Michael Smith, Duke University (Platform)
• 5:45 p.m. – Dosing-Safety Relationship for Acyclovir in the Treatment of Neonatal Herpes Simplex Virus Disease – Dr. Jessica Ericson, Penn State Milton S. Hershey Medical Center (Poster)
• 5:45 p.m. – Impact of Personal Protective Equipment on Pediatric Cardiopulmonary Resuscitation Performance – Dr. Aaron Donoghue, Children’s Hospital of Philadelphia (Poster)
• 5:45 p.m. – Weight Gain and Metabolic Syndrome in Children Exposed to Second-Generation Antipsychotic Medications – Dr. Angela Czaja, Children’s Hospital Colorado (Platform)

Tuesday, May 8

• 7:30 a.m. – Correlating Pulmonary Hypertension by Echocardiogram with Mortality in Premature Infants – Dr. Rachel Torok, Duke University (Platform)