Tag: pediatrics

Beyond Imagining: Pediatric HIV Research Faces the Future

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Originally published in the 2015 CHOP Research Annual Report.

I composed this original article based on multiple interviews and background literature research.

Excerpt:

The HIV epidemic in 2015 and beyond is a dramatically different one than ever seen or imagined during the height of the AIDS crisis in the 1980s and ‘90s.

“In the early days, up to a quarter of all infants born to women with HIV became infected. Now it’s less than one percent,” said Richard Rutstein, MD, an HIV clinical research leader and medical director of the Special Immunology Service at The Children’s Hospital of Philadelphia since its inception in 1989. “For those infected, HIV has changed from a rapidly fatal disease to a chronic illness.” Dr. Rutstein is also a professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania.

At the front line of this evolution, CHOP researchers are helping infected pregnant women, infants, children, and youth around the world live full, productive lives.

Palliative Care Experts Study Grieving Siblings’ Psychological Welfare

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Originally published in Bench to Bedside, the CHOP Research monthly publication

I composed this original article based on interviews with the investigators.

Excerpt:

The experience of grieving for a dying brother or sister during childhood is something that the surviving siblings carry with them for the rest of their lives. Research conducted at The Children’s Hospital of Philadelphia suggests that the experience itself is not a source of serious psychological problems, despite many parents’ concerns that their healthy children could suffer lasting harm from the loss of their sibling. .

“The study comprises a small number of children, but indicates that kids honestly do OK,” said first author Lisa Humphrey, MD, medical director of palliative care at Nationwide Children’s Hospital, who performed the study during her fellowship at CHOP. “When we share this information with parents, this gives them a little glimmer of hope.”

New CHOP Study to Understand Risk of Hospital Readmission

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Originally published in Bench to Bedside, the CHOP Research monthly publication

I composed this original article based on an interview with the investigator.

Excerpt:

One in 20 infants is admitted to the hospital during the first year of life. As frightening as it may be for families to have a child whose health condition requires hospitalization, in too many cases the experience gets worse when a relapse or problem managing the condition after discharge means their child must be re-admitted later. Babies born prematurely are among the groups of children are at highest risk of hospital readmission.

With a new grant from the U.S. Department of Health and Human Services’ Agency for Healthcare Research and Quality (AHRQ), researchers at The Children’s Hospital of Philadelphia are working to reduce the need for pediatric readmissions by taking a population-level look at clinical factors, and an up-close look at familial and social factors, that send high-risk groups of children back to the hospital.

“The long-term goal is to take this information and develop real-time predictions,” said study leader Scott Lorch, MD, MSCE, director of the Center for Perinatal and Pediatric Health Disparities Research at CHOP and associate professor of pediatrics at the Perelman School of Medicine at the University of Pennsylvania. “We are looking for things that can be fed back to the clinical team to say, based on these factors, this patient has a higher than usual risk of readmission.”

On the Trail of a Cancer Predisposition Syndrome

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Originally published in Bench to Bedside, the CHOP Research monthly publication

I composed this original article and complementary blog post based on interviews with the investigator and two parents of children with this syndrome.

Excerpt:

Inside the cells of a developing human embryo is a little piece of “Alice in Wonderland.” While most of our bodies’ genes are expressed from both our mothers’ and fathers’ chromosomes, there is a particular growth-regulating region of chromosome 11 where Dad’s genes make you grow bigger, and Mom’s genes make you small. As in Alice’s adventure, there is potential for some difficult situations to occur when that growth process is not handled with exacting care.

With new grants awarded by the St. Baldrick’s Foundation and the National Cancer Institute, attending physician and geneticist Jennifer M. Kalish, MD, PhD, at The Children’s Hospital of Philadelphia, is going down the rabbit hole to try to set things right. She aims to answer key questions about cancer while helping children with Beckwith-Wiedemann Syndrome (BWS), an overgrowth disorder that can result when epigenetic regulation of growth-regulating regions of chromosome 11 goes awry.

Five Fascinating Facets of Beckwith-Wiedemann Syndrome

Originally published on Cornerstone, the CHOP Research Blog

I composed this blog post as a complement to the above article about the new investigation into BWS.

Excerpt:

1. BWS is a mosaic condition. Some cells and organs or limbs grow unusually large in children with BWS, and some do not. Patterns of this overgrowth vary from child to child. This mosaicism happens because the changes in gene expression that cause BWS arise early when the developing human embryo has relatively few cells — and the changes occur in only some of them. Cells and organs descended from those dysregulated cells have unusual growth patterns, while cells and organs descended from normally developing cells continue to grow at normal rates. This results in a widely varying presentation of the syndrome in different children.

The mosaic nature of the condition makes it challenging to manage the elevated cancer risk that goes along with BWS. As Dr. Kalish noted, “I can see if a child’s arms are bigger. I cannot see what is going on in the liver or kidney.” Each child’s areas of overgrowth are variable, so all young children with BWS must undergo regular cancer screenings.

Cornering a Cancer-Connected Autoimmune Disease

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Originally published in Bench to Bedside, the CHOP Research monthly publication

I composed this original article and related behind-the-science human interest blog post based on interviews with the investigators.

Excerpt:

It is certainly not good news for children to get a double whammy of both cancer and autoimmune disease. Unfortunately, for a small subset of children with neuroblastoma, a common childhood cancer of the peripheral nervous system, an extremely rare autoimmune disorder called OpsoclonusMyoclonus Ataxia Syndrome (OMAS) comes along for the ride. The overactive immune response is believed to be triggered by the cancer.

But there is a twist.

“Patients with neuroblastoma who have OMAS have better outcomes, in terms of their tumor, than patients with neuroblastoma who don’t have OMAS,” said Jessica Panzer, MD, PhD, a pediatric neurologist and attending physician at The Children’s Hospital of Philadelphia who is studying this disease.

That pattern leads Dr. Panzer and other researchers to wonder: Is it possible that OMAS is a case of the body’s immune system finding a successful defense against cancer (but taking it a little too far against healthy cells)? And could we learn safe ways to harness its ability to help more children with neuroblastoma, or even other cancers?

These are among many long-term questions on the distant horizon for researchers who study this little-understood autoimmune disease. First, they need to understand the basics.

Dancing Eyes Brought a Research Team Together

Originally published on Cornerstone, the CHOP Research Blog

I composed this story as a complement to the above article highlighting the science of this team’s collaboration.

Excerpt:

It started at the end of a long day. Jessica Panzer, MD, PhD, then just a few weeks into her pediatric neurology residency at The Children’s Hospital of Philadelphia, was about to go home. Instead, she was called to the emergency room to consult on a 3-year-old girl who could barely walk. What happened then opened up new questions in her budding research career.

Not long after that, Miriam Rosenberg, PhD, started on a convergent path when her own 19-month-old daughter got sick. The toddler first developed problems with excessive drooling and stumbling while she walked. Within a few months, she had a sudden onset of more severe symptoms — unable to walk, severe tremor, unable to feed herself. Dr. Rosenberg and her husband brought their child to the nearest hospital.

CHOP-led Consortium Focuses on Patient-Reported Outcomes

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Originally published in Bench to Bedside, the CHOP Research monthly publication

I composed this original article based on an interview with the investigators.

Excerpt:

“How tired do you feel?” a doctor asks a child with a chronic disease. Or, “How well are you managing stress?”

The answers to questions like these are even more important, from many patients’ and families’ perspectives, than the particular numerical result of their lab test results.

But the answers are less useful to doctors than they could be. Doctors do not have validated tools to use such patient-reported outcomes to track progress managing a condition over time in the same way they can compare results of blood tests over time. In clinical research, they are unable to compare the answers across patients to ultimately show an experimental drug meaningfully improves fatigue or other patient-reported measures.

“Our vision is that patient-reported outcomes become like lab tests,” said Christopher Forrest, MD, PhD, a pediatrician and researcher at The Children’s Hospital of Philadelphia and professor of pediatrics at the Perelman School of Medicine at the University of Pennsylvania. “Soon doctors will use patient-reported outcomes to monitor patients’ clinical care in the same way they use lab tests or X-rays.”

Dr. Forrest and colleagues at CHOP and partner institutions received a new grant from the National Institutes of Health to advance the science of patient-reported outcome measures to one day achieve that vision.

No Rush for Eye Disease Screening in Most Children With Diabetes

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Originally published on Cornerstone, the CHOP Research Blog

I composed this blog post as a follow-up to a CHOP press release on the same study, based on an interview conducted by a colleague.

Excerpt:

Something surprising happened when Gil Binenbaum, MD, MSCE, and his colleagues in the Division of Ophthalmology at The Children’s Hospital of Philadelphia examined children with diabetes: They kept failing to find what they were looking for.

“We examined many kids for diabetic eye complications, and they didn’t have diabetic retinopathy,” said Dr. Binenbaum, an eye surgeon, who is also an assistant professor of ophthalmology at the Perelman School of Medicine at the University of Pennsylvania.

Diabetic retinopathy (DR) is one of many potential complications caused by diabetes’ effects on the body’s blood vessels. Although DR can damage retinal tissue and seriously impair vision, the severe form of the condition is quite rare in children, regardless of how long they have had diabetes or how well they control their blood glucose levels, Dr. Binenbaum noted.