Recent Advancements Shape the Future of Sarcoma Care

The rarity of sarcomas and their large number of diverse histologies have made this group of cancers very challenging to manage. At the Hyundai Cancer Institute at CHOC, collaborative research, experimental treatment protocols and surgical advancements are setting the stage to change that.

“The sarcoma and solid tumor field hasn’t advanced much in the last 50 to 60 years, unfortunately,” says Dr. Elyssa Rubin, pediatric oncologist at CHOC. “But we’re making exciting progress that will hopefully improve the care pediatric patients with bone and soft tissue sarcomas receive in the future.”

Dr. Elyssa Rubin, pediatric oncologist at CHOC

As a member of the Children’s Oncology Group (COG), CHOC is involved in a number of clinical trials that seek to improve the outcome for children with cancer. The international collaboration allows for the compilation of larger data sets that can be used to improve both research and treatments.

“Currently, we are working with COG to harmonize all the clinical trial research from the last 50 to 60 years and creating common data dictionaries so we’re all speaking the same language and being consistent with our methods,” says Dr. Rubin. “Sharing data and having this larger database gives us a better understanding of what’s working, what isn’t and what to target with our treatments. Our ultimate goal is to have our collective data in one central location so we can work together and hopefully, make more advances. This collaboration is what’s needed if we’re really going to make progress.”

Besides her role as principal investigator in clinical trials, Dr. Rubin is researching an experimental maintenance therapy protocol for sarcoma patients.

“I’ve been fascinated by applying the advancements made in leukemia treatment to my sarcoma patients,” Dr. Rubin says. “Leukemia patients are treated with aggressive therapy upfront and then they go into a maintenance phase where they’re taking their medicine over an extended period of time. Over the last seven or eight years, I’ve used a similar approach with my high-risk sarcoma patients, which isn’t the standard of care. The encouraging trend I’ve noticed is a change in the pattern of their relapse and a longer extension of time until they relapse, which tells me this protocol is working to keep their disease under control.”

While it’s still early and more research is needed, Dr. Rubin says her protocol is picking up interest within the bone and tumor committee, and larger studies will be conducted that will have patients follow this protocol for at least six months.

As Dr. Rubin continues her research and the further investigation of her maintenance therapy protocol, other advances are being utilized at CHOC today, particularly in surgical technologies for the treatment of bone and soft tissue sarcomas.

“Recent advances in surgical options help us achieve our goal of preserving as much function as possible so kids can get back to being kids and doing what they love to do,” says Dr. Amir Misaghi, pediatric orthopaedic oncology surgeon at CHOC. “With advances in growing-type prostheses for limb salvage and restoration, we are able to meet this goal now more than in the past.”

Dr. Amir Misaghi, pediatric orthopaedic oncology surgeon at CHOC

3D printing is also revolutionizing the field of orthopaedic oncology, allowing surgeons to print custom bone models for surgical planning.

“When you do so much preoperative planning, the actual surgical time can be minimized,” says Dr. Misaghi. “We’re also using 3D-printed custom cutting guides to help make the surgery as precise as possible, which helps preserve as much of the patient’s native tissue as possible.”

When it comes to the bone and soft tissue sarcoma program at CHOC, Dr. Rubin and Dr. Misaghi emphasize the robust team and comprehensive capabilities.

“Between oncology, orthopaedic surgery, plastic surgery and radiology, as well as general surgery and pathology, we really have the full package here at CHOC,” Dr. Misaghi says. “We are fully equipped to take care of all benign and malignant bone and soft tissue tumors, and we all focus specifically on pediatrics.”

Our Care and Commitment to Children Has Been Recognized

CHOC Children’s Hospital was named one of the nation’s best children’s hospitals by U.S. News & World Report in its 2020-21 Best Children’s Hospitals rankings and ranked in the cancer specialty.

Learn how CHOC’s pediatric oncology treatments, expertise and support programs preserve childhood for children in Orange County, Calif., and beyond.

Related posts:

Research Shows Endoscopic Strip Craniectomy is Strong Alternative to Open Approaches for Craniosynostosis

Minimally invasive endoscopic strip craniectomy offers a strong alternative for infants with craniosynostosis, according to a growing body of research in pediatric neurosurgery.

“The data has been clear that this is a very effective surgery with excellent results, and it’s less invasive than the traditional open approaches for treating craniosynostosis,” says Dr. Suresh Magge, medical director of neurosurgery at CHOC and co-medical director of the CHOC Neuroscience Institute. “A lot of the research that we and other groups have done shows that results are either as good or can even be better in certain aspects of facial growth compared to open vault reconstruction.”

Dr. Suresh Magge
Dr. Suresh Magge, medical director of neurosurgery at CHOC and co-medical director of the CHOC Neuroscience Institute.

Dr. Magge’s research on the topic has included:

Traditional surgery for craniosynostosis is an open cranial vault reconstruction, in which a surgical team takes apart the skull in order to reshape the skull plates. If craniosynostosis is diagnosed early enough – preferably before four months of age – minimally invasive endoscopic surgery can correct this condition. Small incisions are made and, using a camera, the fused portions of the skull are removed.

While both the traditional cranial vault reconstruction and the minimally invasive surgery can offer excellent surgical results, the minimally invasive approach generally involves less blood loss and swelling, smaller incisions, reduced need for blood transfusions, less time under anesthesia and shorter hospital stays. Pediatric patients usually go home the day after surgery. Once surgery is completed, the patient is fitted for a cranial molding helmet he or she must wear for a few months that helps guide the skull correction over time.

“We have an outstanding craniofacial team, including neurosurgeons, plastic surgeons and maxillofacial surgeons, here at CHOC, and we strive to offer an individualized approach to each patient,” Dr. Magge says. “Physicians need to know that craniosynostosis requires an early diagnosis so that parents have the option of the minimally invasive surgery. At the same time, we want to give parents different options when it comes to surgery.”

Dr. Magge recently joined CHOC after an 11-year tenure as a pediatric neurosurgeon at Children’s National Hospital in Washington, D.C., where he started the medical center’s minimally invasive craniosynostosis program and was the director of neurosurgery fellowship training. He completed his neurosurgery residency training at the University of Pennsylvania and his pediatric neurosurgery fellowship training at Boston Children’s Hospital.

While Dr. Magge has a wide-ranging clinical practice, his special clinical and research interests include craniosynostosis, brain and spinal tumors, especially diffuse intrinsic pontine gliomas, and pediatric neurovascular disease, including arteriovenous malformation and Moyamoya disease. In his new role at CHOC, Dr. Magge looks forward to contributing to the growth of CHOC’s neurosurgical programs, including the brain tumor program, neurovascular program, epilepsy program, robotic surgeries and more. 

“It’s such an exciting time here at CHOC, from how we’re building and growing our programs to driving clinical innovation, as well as training the next generation of pediatric neurosurgeons through our affiliation with the University of California, Irvine,” Dr. Magge says. “We’re always asking ourselves, ‘What’s the best thing we can do for each child in the least invasive manner with the least amount of pain?’ and then doing so in a compassionate manner.”

Our Care and Commitment to Children Has Been Recognized

CHOC Children’s Hospital was named one of the nation’s best children’s hospitals by U.S. News & World Report in its 2020-21 Best Children’s Hospitals rankings and ranked in the neurology/neurosurgery specialty.

USNWR Neurology and Neurosurgery award

Learn how CHOC’s neuroscience expertise, coordinated care, innovative programs and specialized treatments preserve childhood for children in Orange County, Calif., and beyond.

Related posts:

Seminal study of COVID-19 infections at Orange County schools nears completion

Two doctors – one a rising star in infectious disease research, the other a nationally known pediatric pulmonologist – are nearing completion of a seminal study on COVID-19 transmission at four Orange County schools and how closely students and staff are following mitigation procedures such as wearing face coverings.

The doctors believe the study is the first of its kind in the country, and that it could shed light on such critical issues such as the role that kids play in the spread of SARS-CoV-2, the novel coronavirus that has led to the deaths of some 300,000 Americans and counting.

“We’ll never understand COVID-19 disease until we understand it in children,” says Dr. Dan Cooper, who treats kids with lung conditions at CHOC and who serves as director of UC Irvine’s Institute for Clinical & Translational Science.

Dr. Cooper was reiterating a comment made by UCI colleague Dr. Donald Forthal, chief of the Division of Infectious Diseases at the university, in the early stages of the pandemic.

Dr. Cooper is a multiple principal investigator of the O.C. school study along with Dr. E.R. Chulie Ulloa, a CHOC infectious disease specialist and an assistant professor in the Department of Pediatrics at the UCI School of Medicine.

Drs. Coopers and Ulloa provided an update on their novel study of four O.C. middle and high schools during a recent presentation on Research Day. The study, a partnership of CHOC, UCI Health and the Orange County Health Care Agency, began at the end of October and will conclude before Christmas.

Results are expected during the first quarter of 2021.

“We understand little about the immune responses to COVID-19 in children, but hopefully we’ll be able to shed some light with this study,” Dr. Ulloa says.

Vaccinations in adult U.S. healthcare workers began in mid-December at a time when the country remains in the grips of a terrible increase in COVID-19 infections and deaths – the dreaded “second surge” healthcare experts predicted would happen this winter.

“We’re just beginning to understand and accumulate the knowledge we need to eventually make definitive statements about SARS-CoV-2 transmission in children,” Dr. Cooper says. “This information will ultimately inform decisions around vaccinations in the pediatric population.”

PRELIMINARY RESULTS

Although the numbers continue to increase as the study nears completion, as of mid-November — when Drs. Cooper and Ulloa provided their update on Research Day — 187 students and 100 staff members at four demographically diverse schools had undergone nasal-swab tests, with none testing positive for COVID-19, Dr. Ulloa reported.

Additionally, nearly 90 percent of those students agreed to have their blood drawn to check for antibodies that showed they had been exposed to the novel coronavirus. The positivity rate for those tests came back at 8.5 percent.

This number is encouraging, Dr. Ulloa says. She explains that nearly all these COVID-19 antibody-positive students were completely asymptomatic or mildly symptomatic.

“We’re not sure yet if this positivity rate shows that children do indeed have protection (against COVID-19), but it’s certainly a positive sign,” Dr. Ulloa says. “We will conduct more research in the lab to see if those antibodies are able to neutralize or inhibit the replication of the virus.”

The preliminary results of the study – which involves students from a charter school and a public school in Santa Ana, a charter school in another city that primarily serves students with special needs and a private school elsewhere whose students come from middle- to upper-class families – suggests that children likely are not super-spreaders of COVID-19, Dr. Cooper says.

“We still don’t know why that appears to be the case,” he says.

In addition to testing for COVID-19 with the nasal swabs, the school project team – which includes a total of nearly two-dozen people – also is testing for 21 other respiratory infections, such as the common cold, in the students. The thinking is that these additional tests will help determine how well students are following COVID-19 mitigation measures such as social distancing and frequent washing of hands – which should help prevent them from catching many viral diseases.

“We hope to be able to see if there’s a relationship between how well the school is able to put into place mitigation procedures and the degree of viral infection,” Dr. Cooper notes.

The pool of students involved in the research project has expanded to kids ages 7 to 17, from ages 10-15 at the onset. Dr. Cooper points out although data are accumulating through public health agencies on SARS-CoV-2 in school-aged children, there are few, if any studies like this one that involves students and school staff as research volunteers and who must consent to participate. These types of studies can often add to our knowledge of disease to a much greater extent than simple descriptions of ongoing data, he says.

“In addition, we’re capturing data from an age group that hasn’t been involved in vaccine clinical trials before,” Dr. Ulloa adds, “and these data may shed light on efforts to test COVID-19 vaccines in the future.”

Drs. Cooper and Ulloa both praised the cooperation of school officials and students in allowing them to carry out this seminal study, in which participants also receive free of charge a complete blood count to check for anemia and a full lipid screening to check the amount of cholesterol and triglycerides in their blood.

“In order to pull this off,” Dr. Cooper says, “we had to have very close relationships with the schools, with the parents, and with the kids.”

Dr. Ulloa’s fluency in Spanish and leadership has helped a lot, he says. So has her serving as a strong pediatrician-scientist role model for the children.

“We’ve really been active in the community for a while, which has helped establish trust,” Dr. Ulloa says.

Dr. Cooper notes that the school research team already is talking to the National Institutes of Health about putting together a project to follow middle- and high-school kids who are infected with the coronavirus over the next five years to determine the long-term effects on such things as cognitive development and the role of exercise in keeping the disease in check.

Virtual pediatric lecture series: Minimally invasive surgery for craniosynostosis

CHOC’s virtual pediatric lecture series continues with “Minimally invasive surgery for craniosynostosis.

This online discussion will be held Thursday, Jan. 14 from 12:30 to 1:30 p.m. and is designed for general practitioners, family practitioners and other healthcare providers.

Dr. Suresh Magge, medical director of neurosurgery and co-medical director of the CHOC Neuroscience Institute, will discuss several topics, including:

  • Diagnosing craniosynostosis and differentiating craniosynostosis from plagiocephaly.
  • Recognizing the early signs of craniosynostosis and understanding when to refer a patient to a neurosurgeon.
  • Differentiating open versus minimally invasive treatment of craniosynostosis.
Dr. Suresh Magge, medical director of neurosurgery and co-medical director of the CHOC Neuroscience Institute

This virtual lecture is part of a series provided by CHOC that aims to bring the latest, most relevant news to community providers. You can register here.

CHOC is accredited by the California Medical Association (CMA) to provide continuing medical education for physicians and has designated this live activity for a maximum of one AMA PRA Category 1 Credit™. Continuing Medical Education is also acceptable for meeting RN continuing education requirements, as long as the course is Category 1, and has been taken within the appropriate time frames.

Please contact CHOC Business Development at 714-509-4291 or BDINFO@choc.org with any questions.

New physician scientist already conducting pioneering research in neonatology

As a recently recruited young physician scientist on CHOC’s neonatal intensive care unit (NICU), Dr. Grant Shafer is maintaining a busy clinical schedule while settling into a new life in Southern California.

And Dr. Shafer, who joined CHOC on Sept. 1 after finishing a fellowship at Texas Children’s Hospital (TCH), arrives here with some pioneering result already under his belt – with more to come.

Dr. Grant Shafer, CHOC neonatologist

In one of the first such large studies of its kind in neonatology, Dr. Shafer is researching the prevalence of diagnostic errors and the ethical responsibilities of providers to disclose such errors to families of impacted NICU patients. 

“Studying and quantitating diagnostic errors is a relative new science in the field of neonatology,” said Dr. Vijay Dhar, medical director of CHOC’s NICU, and division chief, Neonatology, at CHOC/UCI. “Grant has been an outstanding addition to our growing young faculty in the division.”

In March 2020, Dr. Shafer, with a TCH colleague, authored the paper “The Ethics of Disclosing Diagnostic Errors: What is the Researcher’s Duty?” that was published in JAMA Pediatrics, a monthly peer-reviewed medical journal of the American Medical Association.

And in late October, Dr. Shafer was one of eight former distinguished fellows who spoke on a panel before a global international audience at the Society to Improve Diagnosis in Medicine’s (SIDM) Diagnosing Errors in Medicine 13th Annual International Conference.

“To me, this research is interesting because it’s about how we provide the care we provide,” Dr. Shafer said. “It’s the kind of research that I really enjoy. Some people enjoy benchwork, some people enjoy working in the lab, some enjoy clinical studies.

“I really enjoy research that looks at the systems in which we practice medicine and how that impacts the care we provide, and diagnostic errors encapsulates all of that,” he added. “But it’s a field that we really haven’t studied yet. There’s just not a lot of information out there. All the data we’re finding is new to everyone.”

Earned a master’s in English Literature before becoming a doctor

Dr. Shafer, whose parents are from Hawaii, grew up in Denver. His mother, Andrea, is a retired school administrator and his father, Duane, worked in finance. He has a younger sister who runs a CrossFit gym in Kansas City with her husband.

Dr. Shafer earned bachelor’s and master’s degrees in English Literature from Wayne State University in Detroit. He went on to earn his medical degree from the University of Cincinnati College of Medicine and completed his pediatrics residency at University of Texas Southwestern.

At Baylor College of Medicine at TCH, Dr. Shafer completed a neonatal-perinatal medicine fellowship and, over the last year, also completed a second research fellowship in diagnostic excellence through SIDM.

In his JAMA Pediatrics paper, Dr. Shafer and co-author Dr. Frank X. Placencia probed the duty of a researcher performing retrospective medical reviews to disclose diagnostic errors. They concluded that because researchers are outside the patient-clinician relationship, the researcher is not ethically obligated to disclose a diagnostic error directly to a patient with whom they have no formal relationship.

However, Drs. Shafer and Placencia concluded, there is potentially a responsibility to discuss the error with the treating clinician, who then assumes the responsibility of contacting the patient.

Elaborating on this ethical framework during his SIDM panel presentation, Dr. Shafer noted many researchers feel uncertain how to proceed when they come across a diagnostic error that potentially could cause harm to a patient. Because of the sensitive nature of the information, Dr. Shafer recommended that it be delivered to the clinician in a structured setting.

Dr. Shafer said with hard numbers about diagnostic errors in NICUs still years away, a lot of research remains to be done.

“It’s widely acknowledged that diagnostic errors occur in the NICU, but we don’t know how often or how much harm they are causing, which means we can’t try to make things better,” he said.

And making things better – building on CHOC’s already sterling reputation in the field of neonatology — is the whole point, he added.

“I think this is the right place and right time to really push this research forward,” Dr. Shafer said. “I’m humbled and excited to have the opportunity to provide clinical care to babies here in the NICU at CHOC as well as research how we can continue to improve the diagnostic care we provide moving forward.”

Related posts: