From the Nursing Frontline of the Batten Disease Battle

When the neuroscience unit nurses at CHOC Children’s Hospital discovered that teen patient Maya loved to crack people up, they knew a joke book would be the perfect gift.

Compared to the life-changing treatment Maya is receiving at CHOC Children’s Hospital, a book is a very small token. But as partners in this journey, CHOC nurses say it’s the very least they can do.

Maya is among nine patients receiving a novel treatment for CLN2 disease, or late infantile Batten disease at CHOC. The group comes every two weeks for an infusion of Brineura, a drug that replaces a deficient enzyme.

The CHOC team has just celebrated the second anniversary of the first infusion, having clocked more than 300 procedures in the last 24 months. Since CHOC began offering the treatment in spring 2017, the number of patients has more than doubled, says Dr. Raymond Wang, a pediatric metabolic specialist and the program’s medical director.

Dr. Raymond Wang, medical director, and Susan See, Neuroscience nurse manager
Dr. Raymond Wang, medical director of the Batten disease treatment program, and Susan See, neuroscience unit nurse manager

Among the handful of institutions nationwide offering the treatment, CHOC’s program is the largest.

Despite the juggling act of placing patients, made especially tough during high wintertime patient census, CHOC nursing leadership has helped ensure these patients have never missed a procedure.

Nurses play an integral role in the procedure, assisting pediatric neurosurgeon Dr. Joffre Olaya in inserting a needle through the skin of the patient’s skull and into an Ommaya reservoir implanted beneath the flesh to reach the fluid surrounding the brain.

The procedure is conducted inside the patient’s room on CHOC’s neuroscience floor. During the procedure and afterward as they monitor the patients, the nursing team is in frequent, close contact with the children – and their parents.

“Because of this, we know how some parents take their coffee, which brand of diapers they prefer, and how the families gather to talk after treatments,” says Alison Cubacub, a clinical nurse II in CHOC’s neuroscience unit.

They also know how life-changing the treatment is for families.

Before Brineura clinical trials and its ultimate approval by the U.S. Food and Drug Administration, this small group of patients and their families endured a long odyssey of poring over Internet pages, making endless phone calls, and traveling the country in hopes of finding treatment for the devastating diagnosis that is Batten disease.

The rare and fatal neurologic condition typically begins with language delays and seizures before age 3, and rapidly progresses to dementia, blindness, loss of the ability to walk and talk, and death in childhood.

But Brineura has shown to slow the disease’s progression. Over a three-year period, patients treated during clinical trials showed no advancement of the disease, which was radically different from the disorder’s natural course. The medication improves quality of life and buys patients critical time as researchers continue to search for a cure.

Before, Southern California patients traveled every 10 days from their homes to Columbus, Ohio, to receive treatment as part of a clinical trial. Aside from the inconvenience, the process split apart families. But now, they need only drive a few miles to CHOC for a few hours of treatment.

And while it can’t compare to the relief felt by patients and families, CHOC’s neuroscience nursing team is invigorated by the professional growth that’s been afforded to them as a result of CHOC offering the treatment.

“The science and research that went into developing this treatment is fascinating,” says Melissa Rodriguez, RN, BSN, CPN, a clinical nurse III in CHOC’s neuroscience unit. “To assist in its administration, we participated in an in-depth study that included literature about the procedure and the condition, as well as extensive training in the sterile procedure required to infuse the medication.”

Nurses Week Q&A With CHOC’s Chief Nursing Officer Melanie Patterson

In honor of National Nurses Week (May 6-12), Melanie Patterson, DNP, MHA, RN, vice president, patient care services and chief nursing officer of CHOC Children’s Hospital, shared a few nursing highlights from the past year. CHOC nurses work in a variety of roles, from direct patient care to informatics. They partner with our pediatricians and pediatric specialists, as well as other staff, to advance care and safety for our patients and their families. Learn more about their most recent contributions in this Q&A with Melanie.

Melanie Patterson, DNP, MHA, RN, vice president, patient care services and chief nursing officer
Melanie Patterson, Chief Nursing Officer

CHOC received numerous accolades for patient care and safety; among them Magnet® recognition. What does this honor really mean?

This is our third time achieving Magnet recognition, reflecting our commitment to high-quality nursing practice. This is truly an honor and places us in an elite group of healthcare organizations; fewer than 500 (out of more than 6,300) hospitals in the country have achieved Magnet recognition.

Official green flag Magnet logo from the American Nurses Credentialing Center
CHOC received Magnet recognition for the third time

To earn this distinction, we passed a rigorous and lengthy process that required widespread participation from leadership and staff. We are a much better organization because of our Magnet recognition, which raised the bar for patient care and inspired every member of our team to achieve excellence every day.

CHOC has taken a leadership role in developing a pediatric system of mental health care in Orange County, with an inpatient center as its centerpiece. Do you have any updates to share?

Our Mental Health Inpatient Center opened last April, filling a gap in a fragmented system that left children younger than 12 with no inpatient services in Orange County. The innovative facility, which features a warm, healing environment, is the only one in the state to offer all private rooms and an option for parents to stay overnight with their child, as appropriate. We’ve admitted more than 600 children since we opened and achieved an 86 percent positive rating in our patient experience survey. In addition, our restraint and seclusion rates are far below the national average, as is nurse turnover. The Center’s team has worked hard to create the right combination of “people, place and practice” to promote the best outcomes for our patients and their families.

How has nursing contributed to evidence-based practices?

Evidence-based practice (EBP) is a dynamic process of integrating research, credible sources of knowledge, clinical expertise, and patient and family preferences and values to achieve optimal patient outcomes, enhance practice, and manage health care costs. We developed an EBP Scholars program that is a formal six-month long program funded in part by the Walden and Jean Young Shaw Foundation. Scholars receive education about the EBP process and guidance in developing their own projects focused on improving outcomes. Results have included an increased number of external poster and podium presentations, and nurse-led projects submitted for research consideration and peer-reviewed nursing publications. In addition, six nurse-led projects were awarded grants for projects that resulted in meaningful change, increased patient safety, and decreased cost of care.

A nurse and patient play with a stuffed animal.

How involved have nurses been in research projects?

We encourage a culture of inquiry among our nurses. Our Nursing Research and Innovation Council promotes the integration of research, and innovation into practice, and facilitates nurse involvement in related activities across the organization. Our Nursing Research Associate Training Program provides a meaningful and relatable approach to empower nurses, at all levels, to participate in multidisciplinary research. Participants attend classes and receive hands-on training with a clinical research coordinator.

One example of nurse-led research was focused on pupillary assessment (examining pupils’ reaction to light). This is important in neurological evaluations because changes in the size, equality and reactivity of the pupils can provide vital diagnostic information. Evidence suggests that the traditional pen-light pupil examination is subjective and has low precision and reproducibility. Automated hand-held pupillometers have recently been used to provide more objective measurements of pupillary size and reactivity. Studies suggest early detection of subtle changes using pupillometers may improve patient outcomes in adults, yet there is not much literature on its use in children.

Over the past few years, our nurses have established an organizational database of automated pupillometry to provide evidence for the use of pupillometers in children. Findings from a preliminary study were presented at the American Association of Neuroscience Nurses national conference in Denver, Colorado this past March and demonstrate a statistically significant inverse relationship between pupillometer readings and increased intracranial (inside the skull) pressure. We have since expanded the use of pupillometers in our emergency department and started additional multidisciplinary studies.

How have your nurses taken leadership roles in advancing patient safety throughout the hospital?

CHOC nurses have initiated and/or been asked to participate in numerous quality improvement projects focused on enhancing patient safety. I am especially proud of our team of safety coaches whose roles are to reinforce ― often through real-time feedback ― safe practices. This team now consists of 88 clinicians from more than 25 different areas across our entire healthcare system.

A group of CHOC nurses laugh while posing for a photo

Additionally, we recently created the role of discharge nurse navigator whose focus is on successfully transitioning patients from the hospital to their homes. This includes educating families on after-care instructions and any other follow-up recommended by their physicians. The ultimate goals are decreasing readmission rates and improving patient and family satisfaction. We’ve seen our seven and 30-day readmission rates decrease beyond our initial goals and patient/parent satisfaction regarding nurse communications increase.

What makes CHOC nurses so unique?

Obviously, I am biased and think our nurses are the best in the profession. Every day, I have the privilege of witnessing the countless ways they preserve the magic of childhood. They are compassionate advocates for our patients and families, and proactive in implementing positive change that improves the care and service we provide. They are also collaborative, partnering with our physicians, each other and staff from across the entire healthcare system. They are committed to nursing excellence in everything they do, and selflessly dedicated to advancing our mission and our vision. I am incredibly fortunate to lead such an amazing team.

Infant and Pediatric Feedings Book Receives Hermes Platinum Award

Caroline Steele, CHOC’s director of the Clinical Nutrition and Lactation Services Department, recently announced the book she co-authored with registered dietitian Katherine Bennett received a 2019 Hermes Creative Award. The book, Infant and Pediatric Feedings: Guidelines for Preparation of Human Milk and Formula in Health Care Facilities (third edition), was given Platinum award status—the highest distinction given by the group for excellence in concept, writing and design. We chatted with Caroline about the honor and about what makes this new edition of the book such a valuable read for providers.

Caroline Steele, MS, RD, CSP, IBCLC, FAND
Caroline Steele, MS, RD, CSP, IBCLC, FAND

Q: What inspired you to co-author a book on the topic of milk and formula preparation guidelines in healthcare facilities? 

A: Over the past 20 years, I have conducted and published many research studies specifically looking at patient safety and outcomes related to centralized handling of human milk and formula within the hospital setting. I was an author on the second edition of this publication. Therefore, when the opportunity arose to be the co-editor and author on the third edition, I jumped at it!

Katherine Bennett, Clinical Dietitian
Katherine Bennett, Clinical Dietitian

Q: What are some important themes providers can expect to learn about in the book? 

A: There are really three predominant themes in the publication. First is that the location of infant/pediatric feeding preparation is critical to prevent contamination. Second, use of dedicated technicians improves safety and accuracy. Third, the use of technology can reduce risk of human error during both preparation of feedings and administering feedings to the patient.

Q: Can you hint at some of the new research topics covered in the third edition?
 

A: In this edition, we included new chapters on lactoengineering and the use of blenderized tube feedings. We also provided expanded information on use of donor human milk and milk products as well as modular enteral ingredients.

Q: How does it feel to be recognized with this award? 

A: This award was a great honor and one outside my normal realm of nutrition! The award is granted as platinum, gold or honorable mention. We are thrilled that we received a platinum award, meaning that our book scored 90 to 100 on a scale of 100!

Q: Is there anything else you’d like to highlight about the book?
 

A: Infant and Pediatric Feedings is the most comprehensive resource for all things related to feeding preparation. I use my own copy all the time!

Q: How can people order a physical or digital copy?

A: This may be purchased from the Academy of Nutrition and Dietetics’ Eat Right Store website.   

CHOC Becomes SCID Referral Center

CHOC Children’s is pleased to have recently become a referral center for severe combined immune deficiency (SCID), filling a regional gap that once required Orange County infants to go outside the county for care.

Led by Drs. David Buchbinder, Wan-yin Chan, Diane Nugent and Jasjit Singh, the immunodeficiency program is a multidisciplinary effort crossing multiple specialties at CHOC including allergy and immunology, hematology and infectious disease

Though they appear healthy at birth, infants with this primary immunodeficiency disease lack T lymphocytes, one of the white blood cells that help fight infections. 

Babies with SCID cannot fight even the most innocuous infections and often die. The condition is considered by the medical community as a pediatric emergency.

“Prior to development of SCID newborn screening, the diagnosis would be delayed,” Dr. Chan says. “Often times these patients would not get sick until after 6 months of age. No one would know they were affected until the antibodies from their mother would wane. They end up with life threatening infections with serious complications often resulting in death.”

However, studies show that early bone marrow or stem cell transplants can improve outcomes significantly, Dr. Chan says.

Survival rates increase to 94 percent if administered to an affected infant by age 3 ½ months. However, if transplants occur after that age, survival rates increase to only 70 percent, underscoring the importance of early detection and intervention. 

To that end, California became one of the first states to add SCID to its list of recommended newborn screenings in 2010. In the years since, all states have followed suit. 

Under CHOC’s program, immunodeficiency team physicians review each case of Orange County babies who test positive in newborn screenings for SCID and ask parents to immediately seek a confirmatory blood test for the infant, Dr. Chan says.

If the additional tests confirm the diagnosis, patients are urgently admitted to CHOC for workup and treatment, Dr. Chan says.

Since CHOC’s center was formed in August, more than 20 patients have been flagged in the surrounding communities and each individual case has been reviewed by the immunodeficiency team in collaboration with local pediatricians.

Those urgent blood tests confirmed the presence of SCID or a SCID-like disorder in more than 25 percent of cases thus far. 

CHOC Children’s Doctors’ Day 2019 Celebration – April 5

CHOC Children’s physicians are invited to a special Doctors’ Day luncheon to celebrate their commitment and dedication to patients and families.

Festivities will be held from 11:30 a.m. to 2 p.m. April 5 in the physician dining room and adjacent outdoor patio on the second floor of the Bill Holmes Tower.

A recognition ceremony begins at 12:30 p.m., and a professional photographer will be available to take professional headshots from 11 a.m. to 2 p.m.

Please RSVP by March 25 at choc.org/doctorsday or call the CHOC business development team at 714-509-4291.