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Children’s National Hospital ranked #5 in the nation on U.S. News & World Report’s Best Children’s Hospitals Honor Roll

U.S. News BadgesChildren’s National Hospital in Washington, D.C., was ranked #5 in the nation on the U.S. News & World Report 2023-24 Best Children’s Hospitals annual rankings. This marks the seventh straight year Children’s National has made the Honor Roll list. The Honor Roll is a distinction awarded to only 10 children’s hospitals nationwide.

For the thirteenth straight year, Children’s National also ranked in all 10 specialty services, with eight specialties ranked in the top 10 nationally. In addition, the hospital was ranked best in the Mid-Atlantic for neonatology, cancer, neurology and neurosurgery.

“Even from a team that is now a fixture on the list of the very best children’s hospitals in the nation, these results are phenomenal,” said Kurt Newman, M.D., president and chief executive officer of Children’s National. “It takes a ton of dedication and sacrifice to provide the best care anywhere and I could not be prouder of the team. Their commitment to excellence is in their DNA and will continue long after I retire as CEO later this month.”

“Congratulations to the entire Children’s National team on these truly incredible results. They leave me further humbled by the opportunity to lead this exceptional organization and contribute to its continued success,” said Michelle Riley-Brown, MHA, FACHE, who becomes the new president and CEO of Children’s National on July 1. “I am deeply committed to fostering a culture of collaboration, empowering our talented teams and charting a bold path forward to provide best in class pediatric care. Our focus will always remain on the kids.”

“I am incredibly proud of Kurt and the entire team. These rankings help families know that when they come to Children’s National, they’re receiving the best care available in the country,” said Horacio Rozanski, chair of the board of directors of Children’s National. “I’m confident that the organization’s next leader, Michelle Riley-Brown, will continue to ensure Children’s National is always a destination for excellent care.”

The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals and recognizes the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News.

“For 17 years, U.S. News has provided information to help parents of sick children and their doctors find the best children’s hospital to treat their illness or condition,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “Children’s hospitals that are on the Honor Roll transcend in providing exceptional specialized care.”

The bulk of the score for each specialty service is based on quality and outcomes data. The process includes a survey of relevant specialists across the country, who are asked to list hospitals they believe provide the best care for patients with the most complex conditions.

The eight Children’s National specialty services that U.S. News ranked in the top 10 nationally are:

The other two specialties ranked among the top 50 were cardiology and heart surgery, and urology.

illustration of diseased kidneys

The future of Wilms tumor therapies: Q&A with Jeffrey Dome, M.D., Ph.D.

illustration of diseased kidneys

Dr. Dome’s mission is to come up with other therapies to treat Wilms tumor.

Conducting Wilms tumor research has placed Children’s National Hospital as the leader in this space. Jeffrey Dome, M.D., Ph.D., talks about the future of Wilms tumor therapies and what excites him about being able to offer different treatment options to this group of patients.

Q: What’s next for Wilms tumor therapy treatments?

A: Something we’ve learned is that we’re reaching the maximum benefit that we can achieve with conventional chemotherapy and have hit a plateau with some of the high-risk Wilms tumors.

If you look at the history of Wilms tumor, we’ve ratcheted up the cure rate by adding more and more chemotherapy agents. We’re now at a point in which patients can’t tolerate having more toxic chemotherapy added to their regimen. Our mission is to come up with other therapies that work differently.

Q: What have you learned that can lead to new therapy options?

A: I was a co-investigator on a National Cancer Institute-funded project called the TARGET study, which entailed sequencing several hundred Wilms tumors to identify genetic mutations that could be druggable. While a wealth of knowledge was gained on the nature of the biology of Wilms tumor, only a minority of Wilms tumors have targetable mutations. We understand what the mutations are, but most are not mutations that lend themselves to drug therapy.

Therefore, we must think about other forms of therapy for Wilms tumor, such as immunotherapy. That’s where the work with the T cells targeting the WT1 protein Wilms tumor cells come into play.

Q: What do the new therapies look like?

A: I think the future of Wilms tumor therapy will be combining the classic chemotherapy and radiation therapy that we’ve used for many years but also adding another component for the patients with the highest risk of relapse, such as immunotherapy.

Up until now, the studies that we’ve conducted using immunotherapy have introduced this treatment very late as a last resort after everything else has failed. We have seen early signals of efficacy and if we can corroborate this in current studies, I believe the future will be to introduce immunotherapy earlier in the treatment course.

There has been success using immunotherapy in other pediatric solid tumors such as neuroblastoma. I believe the prospects for Wilms tumor are also exciting.

 

histological image of Wilms Tumor

Leading Wilms tumor research nationwide: Q&A with Jeffrey Dome, M.D., Ph.D.

histological image of Wilms Tumor

Children’s National has become a resource for patients and families with Wilms tumor.

During the past year, Children’s National Hospital saw nearly 100 patients with Wilms tumor and other less common kidney cancers of childhood, far more than most centers in the country. This is largely due to the reputation the hospital has established for specializing in these diseases. While most patients with Wilms tumor have excellent outcomes, a significant minority of children with kidney cancer do not fare well. Children’s National has become a resource for patients and families with these challenging cancers.

Behind this reputation is Jeffrey Dome, M.D., Ph.D., senior vice president of the Center for Cancer and Blood Disorders and division chief of Oncology at Children’s National, and the team of researchers he leads. For over a decade, he chaired the Children’s Oncology Group Renal Tumor Committee, an opportunity that gave him and his work great exposure.

Dr. Dome shares more on how Children’s National is leading in this space and what the future holds for new, exciting Wilms tumor treatment options.

Q: How is Children’s National leading in this space?

A: The good news is that for the most common type of childhood kidney cancer, Wilms tumor with “favorable histology,” the survival rate is more than 90%, which is an incredible success story. But approximately 25% of children and teens with other types of Wilms tumor and other kidney cancers do not fare as well. We specialize in kidney cancers that are harder to treat, such as anaplastic Wilms tumor, relapsed favorable histology Wilms tumor, bilateral Wilms tumor, clear cell sarcoma of the kidney, malignant rhabdoid tumor and renal cell carcinoma. Because we see a relatively large number of patients, we can draw on our prior experience and observations to recommend the best treatment options.

Q: What’s unique about this research?

A: We have several early-phase clinical trials that are of interest for children with relapsed kidney tumors. Some of these trials are part of research consortia, such as the National Cancer Institute-funded Pediatric Early Phase Clinical Trials Network (PEP-CTN). Other studies have been developed in-house at Children’s National, including a couple of studies using T cells to target pediatric solid tumors. The T cells that have been engineered by the Children’s National Cellular Therapy Laboratory are of particular interest for Wilms tumor because they target a protein called WT1, which is expressed in most Wilms tumors. In fact, WT1 was named after Wilms tumor. We have now had more than 25 patients with relapsed Wilms tumor come from around the country to participate in these studies. Based on early successes, we are continuing this line of research and trying to improve the technology in the current generation of studies.

Jeffrey Dome

Jeffrey Dome, M.D.: Making strides in the fight against pediatric cancer

Jeffrey DomeJeffrey Dome, M.D., Ph.D., senior vice president of the Center for Cancer and Blood Disorders and chief of the Division of Oncology (ranked number 6 in the nation by U.S. News & World Report 2022-23 Best Children’s Hospitals annual rankings) at Children’s National Hospital in Washington, D.C., is an internationally recognized expert on pediatric solid tumors, with an emphasis on kidney tumors and sarcomas. He chaired the Children’s Oncology Group (COG) Renal Tumor Committee, which oversees clinical research on kidney tumors at more than 200 children’s hospitals around the world for more than 10 years. Dr. Dome is currently the Continental President of North America for the International Society of Paediatric Oncology (SIOP) and serves on several medical advisory boards for cancer centers and foundations.

“This is a remarkably exciting time to be in the field of pediatric oncology, with an explosion of knowledge on cancer biology and genetics and the availability of new treatment modalities including molecularly targeted therapy, immunotherapy and devices to improve drug delivery and local control,” says Dome. “I am proud of the multidisciplinary and cross-center collaborations at Children’s National to deliver the latest innovative therapies.”

The team at Children’s National is making strides across all programs to benefit patients with pediatric cancer. A few highlights include:

  • The Brain Tumor lnstitute is one of the most active clinical and translational research programs in the country. Collaborating with other leading institutions, the Brain Tumor Institute is supported by a robust brain tumor bench research program with focused laboratories in medulloblastoma, high-grade glioma, midline diffuse glioma, diffuse intrinsic pontine glioma, low-grade glioma and immunotherapy. The Brain Tumor Institute is leading two national studies, both funded through the Moon Shot lnitiative. In addition, it works closely with the Virginia Tech brain tumor laboratories on the new Children’s National Research & Innovation Campus.
  • Children’s National is the first children’s hospital in the United States with a Focused Ultrasound Program. This pediatric dedicated program includes high-intensity (HIFU) and low-intensity focused ultrasound (LIFU), offering minimally invasive surgical options for children with extra-cranial solid tumors, low-grade brain tumors and novel, potentially life-saving therapy with LIFU-mediated blood-brain barrier disruptions for diffuse intrinsic pontine gliomas.
  • Children’s National has developed multi-antigen specific T cells that have shown success in early phase clinical trials for leukemias, solid tumors and brain tumors. This promising area of research earned a major boost in the form of a $25 million dollar grant from Cancer Grand Challenges, founded in 2020 by Cancer Research UK and the National Cancer Institute in the U.S. This award supported the foundation of NexTGen, a team of scientists and clinicians with expertise in immunology, proteomics, mathematics and more, across eight institutions in the U.S., U.K. and France. The Center for Cancer and Immunology Research at Children’s National is one of the leaders of this effort.
  • The Blood and Marrow Transplantation team, one of the only dedicated pediatric bone marrow transplant programs in the greater Washington, D.C., region, is celebrating its 35th anniversary, with a history of clinical and research accomplishments for both malignant and non-malignant disorders. This program has seen tremendous success in their day 100 transplant-related mortality (TRM). Recently, for the first time, the day 100 TRM average was 0%, meaning that the program did not lose a patient due to transplant complications in the first 100 days – a remarkable achievement in the world of transplantation.
  • The Cancer Genetics Program has grown tremendously in the past few years, reflecting recognition that approximately 10% of childhood cancers have an underlying cancer predisposition. Despite COVID-19, during the past fiscal year, there were 282 patient visits which is a 40% increase from the prior year. The team has developed a collaboration with researchers in the Rare Disease Institute and now can offer studies for patients with Beckwith-Wiedemann syndrome, children with previously undiagnosed developmental delay and children with undiagnosed syndromes. Further, the team was awarded a grant from the Children’s Cancer Foundation to allow testing for those without insurance coverage.
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Children’s National named to U.S. News & World Report’s Best Children’s Hospitals Honor Roll

US News BadgesChildren’s National Hospital in Washington, D.C., was ranked No. 5 nationally in the U.S. News & World Report 2022-23 Best Children’s Hospitals annual rankings. This marks the sixth straight year Children’s National has made the list, which ranks the top 10 children’s hospitals nationwide. In addition, its neonatology program, which provides newborn intensive care, ranked No.1 among all children’s hospitals for the sixth year in a row.

For the twelfth straight year, Children’s National also ranked in all 10 specialty services, with seven specialties ranked in the top 10.

“In any year, it would take an incredible team to earn a number 5 in the nation ranking. This year, our team performed at the very highest levels, all while facing incredible challenges, including the ongoing pandemic, national workforce shortages and enormous stress,” said Kurt Newman, M.D., president and chief executive officer of Children’s National. “I could not be prouder of every member of our organization who maintained a commitment to our mission. Through their resilience, Children’s National continued to provide outstanding care families.”

“Choosing the right hospital for a sick child is a critical decision for many parents,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “The Best Children’s Hospitals rankings spotlight hospitals that excel in specialized care.”

The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals and recognizes the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News.

The bulk of the score for each specialty service is based on quality and outcomes data. The process includes a survey of relevant specialists across the country, who are asked to list hospitals they believe provide the best care for patients with the most complex conditions.

The seven Children’s National specialty services that U.S. News ranked in the top 10 nationally are:

The other three specialties ranked among the top 50 were cardiology and heart surgery, gastroenterology and gastro-intestinal surgery, and urology.

Jeffrey Dome

Q&A with Dr. Jeffrey Dome on his new role as Continental President of SIOP-North America

Jeffrey Dome

In March 2021, Jeffrey Dome, M.D., Ph.D., senior vice president of the Center for Cancer and Blood Disorders at Children’s National Hospital, was elected as the International Society of Paediatric Oncology’s (SIOP) Continental President of North America.

In March 2021, Jeffrey Dome, M.D., Ph.D., senior vice president of the Center for Cancer and Blood Disorders at Children’s National Hospital, was elected as the International Society of Paediatric Oncology’s (SIOP) Continental President of North America.

On October 21-24, the society will hold its 53rd SIOP Annual Congress virtually. During the congress, Dr. Dome will begin his 3-year term as SIOP continental president of North America and will also chair and speak at an educational symposium on current approaches to the treatment of recurrent Wilms tumor.

Dr. Dome attended his very first SIOP meeting in 2005 and was captivated by how regional context influences pediatric cancer treatment. In 2017, he was chair of the local organizing committee for the 49th annual congress in Washington, D.C., and served on the SIOP Board of Directors.  After 15 years of involvement and attending many of the annual meetings, Dr. Dome shares what he looks forward to while serving as continental president of SIOP North America and the legacy he hopes to leave behind.

  1. What does it mean to you to be elected SIOP continental president of North America?

I’m very excited about this role. There are several important societies and organizations in North America that have made a mark on the field of pediatric oncology, but SIOP is unique in its sole focus on childhood cancer and global approach to improving outcomes, as encapsulated by its vision statement: No child should die of cancer: cure for more, care for all.

  1. What excites you most about this role?

In an eye-opening statistic, North America has only about 10% of the global burden of childhood cancer and less than 2% of worldwide childhood cancer deaths.  Although we relentlessly strive to improve childhood cancer outcomes in the United States, what we experience here is just the tip of the iceberg of the worldwide problem. SIOP seeks to make a difference on the international level by improving education, research and access to care for children with cancer around the world. And I’m excited to have a platform to lead North American ambassadors to do that.

Even though North America has a relatively small fraction of the overall childhood cancer cases, we are one of the most well-resourced continents. The question is, how can we use our knowledge, technology and resources to help the rest of the world.  A big part of this role is to make connections and liaisons to move the needle on improving outcomes.

The other thing we’ve learned from a research standpoint is that pediatric cancers are relatively uncommon and are becoming even rarer through molecular classification, which divides cancers into small genetically defined subgroups.  While these advances are tremendously exciting, they require international collaboration to amass a sufficient number of patients to evaluate novel treatment strategies.  My vision for SIOP North America is to be a convener of researchers and connect people around the world to facilitate that work.

  1. What are some of your goals while serving as continental president?

We recently sent a survey to more than 450 SIOP North America members and had a nearly 45% response rate, which I’m told is superb.  This speaks to an excellent level of engagement in SIOP’s mission, with many members volunteering to participate in committees related to research, advocacy and global health. The majority of the respondents to the survey were physicians but improving childhood cancer treatment takes a holistic approach.  One of my main goals is to increase SIOP North American membership to grow the number of nurses, pharmacists, scientists, psychologists, other behavioral health specialists and clinical research coordinators onboard.

I’d like to also identify two to three very specific projects that will impact pediatric cancer care on a global level. There are different ways to do that. We could improve education in different areas around the world (nursing education that we provide to areas that are lacking nursing support, for example). It could be research education and database education for regions of the world that would like to develop more robust research programs. It can also be medical support and developing medical guidelines for oncologists around the world that are adjusted to different levels of resources that are available.

The other goal would be to enhance supportive care and education for cancer care delivery on the global level.

  1. Why is this work important for you?

One of my mentors from when I was a junior faculty member advised me that to be a well-rounded oncologist, one must be familiar with how childhood cancer is treated around the world because different regions have different approaches. There is something to be learned from everyone.  I took that advice to heart and have tried to look beyond the North American approaches.  I think it’s very important to have a global exchange of ideas and serving as continental president of SIOP-North America will enable more to facilitate this dialogue.

  1. What’s the legacy or impact you hope to leave behind?

SIOP North America has a strong and devoted membership but has largely functioned at the level of the individual members.  I hope to bring more structure to the organization to tackle the global challenges of childhood cancer treatment.

Once this structure is in place, I hope to complete two or three SIOP-North America initiatives that have a measurable impact on improving childhood cancer care delivery or outcomes.  The specific projects have yet to be defined but will likely be in the categories of enhancing education, supportive care and facilitating research infrastructure. There’s so much to tackle that if you just look at the overall problem of childhood cancer, it’s overwhelming.  We’re not going to be able to solve everything in three years, but if we could have a few victories and be able to move the needle in some areas, I think that would be a huge success.

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For fifth year in a row, Children’s National Hospital nationally ranked a top 10 children’s hospital

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Children’s National Hospital in Washington, D.C., was ranked in the top 10 nationally in the U.S. News & World Report 2021-22 Best Children’s Hospitals annual rankings. This marks the fifth straight year Children’s National has made the Honor Roll list, which ranks the top 10 children’s hospitals nationwide. In addition, its neonatology program, which provides newborn intensive care, ranked No.1 among all children’s hospitals for the fifth year in a row.

For the eleventh straight year, Children’s National also ranked in all 10 specialty services, with seven specialties ranked in the top 10.

“It is always spectacular to be named one of the nation’s best children’s hospitals, but this year more than ever,” says Kurt Newman, M.D., president and CEO of Children’s National. “Every member of our organization helped us achieve this level of excellence, and they did it while sacrificing so much in order to help our country respond to and recover from the COVID-19 pandemic.”

“When choosing a hospital for a sick child, many parents want specialized expertise, convenience and caring medical professionals,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “The Best Children’s Hospitals rankings have always highlighted hospitals that excel in specialized care. As the pandemic continues to affect travel, finding high-quality care close to home has never been more important.”

The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals. The rankings recognize the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News. The top 10 scorers are awarded a distinction called the Honor Roll.

The bulk of the score for each specialty service is based on quality and outcomes data. The process includes a survey of relevant specialists across the country, who are asked to list hospitals they believe provide the best care for patients with the most complex conditions.

Below are links to the seven Children’s National specialty services that U.S. News ranked in the top 10 nationally:

The other three specialties ranked among the top 50 were cardiology and heart surgery, gastroenterology and gastro-intestinal surgery, and urology.

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Jeffrey Dome, M.D., elected SIOP Continental President of North America

Jeffrey Dome

“I’m honored to have been elected as president of a society that is a leader in propelling treatment and advocacy for childhood cancer,” Dr. Dome said. “I look forward to working alongside peers who are committed to efforts to improve outcomes for children with cancer globally.”

Jeffrey Dome, M.D., Ph.D., vice president of the Center for Cancer and Blood Disorders at Children’s National Hospital, has been elected as the International Society of Paediatric Oncology’s (SIOP) Continental President of North America.

“I’m honored to have been elected as president of a society that is a leader in propelling treatment and advocacy for childhood cancer,” Dr. Dome said. “I look forward to working alongside peers who are committed to efforts to improve outcomes for children with cancer globally.”

SIOP is the only global multidisciplinary society devoted to pediatric and adolescent cancer. With over 2,600 members worldwide – including doctors, nurses, other health-care professionals, scientists and researchers – the society is dedicated to increasing knowledge about all aspects of childhood cancer.

SIOP will officially welcome Dr. Dome to the position at its Annual Business Meeting in October.

light micrograph of wilms tumor

Evolution of risk stratification for Wilms tumor

light micrograph of wilms tumor

Light micrograph of Wilms tumor.

Wilms tumor is a rare kidney cancer that primarily affects children. Also known as nephroblastoma, it is the most common malignant renal tumor in children. Advances in the treatment of Wilms tumor are some of the great achievements in the field of oncology, improving survival to 90% and decreasing the burden of therapy.

A key factor in the success of Wilms tumor treatment has been improved risk stratification, enabling augmentation or reduction of therapy depending on a patient’s risk of relapse. In a review article in Current Opinion in Pediatrics, Jeffrey Dome, M.D., Ph.D., vice president of the Center for Cancer and Blood Disorders at Children’s National Hospital, Marie V. Nelson, M.D., assistant professor of pediatrics in the Division of Oncology, and their colleagues look at the evolution of clinical and biological factors that have been adopted for Wilms tumor.

The authors found that the original National Wilms Tumor Study Group (NWTSG) and International Society of Pediatric Oncology (SIOP) studies relied solely on tumor stage to define treatment. Over time, however, additional factors were incorporated into the risk stratification schema, allowing for a multifactorial precision medicine approach.

The authors conclude that “the application of new clinical and biological prognostic factors has created unprecedented ability to tailor therapy for Wilms tumor, accompanied with improved outcomes. Current and future trials will continue to enhance precision medicine for Wilms tumor.”

Read the full study in Current Opinion in Pediatrics.

illustration of cancer cells attacking kidneys

Renal cell carcinoma study shows excellent short-term outcomes without adjuvant therapy

illustration of cancer cells attacking kidneys

Researchers found that patients with localized pediatric renal cell carcinoma have excellent short-term outcomes without adjuvant therapy.

Although renal cell carcinoma (RCC) is the second most common kidney cancer diagnosed in children and adolescents, guidance regarding its clinical management has been confined to retrospective case series, which were limited by reporting bias, varied treatment approaches and a lack of central pathology review to confirm the diagnosis.

Research conducted by the Children’s Oncology Group (COG) and led by Jeffrey Dome, M.D., Ph.D., vice president of the Center for Cancer and Blood Disorders at Children’s National Hospital, found that patients with localized pediatric RCC have excellent short-term outcomes without adjuvant therapy with 4-year overall survival estimates of 96% for patients with stage I disease, 100% for patients with stage II disease and 88% for patients with stage III disease.

“The results of this study provide important practical insights into the management of pediatric RCC,” said Dr. Dome. “Oncologists now have validation that a surgery-only approach is appropriate management for the majority of children and adolescents with RCC.”

Jeffrey Dome

“The results of this study provide important practical insights into the management of pediatric RCC,” said Jeffrey Dome, M.D., Ph.D.

The excellent survival in patients with stage III disease held up even in those with tumor involvement of regional lymph nodes, a finding that differs from adult RCC. However, patients with metastatic disease (stage IV), had a 4-year overall survival estimate of only 29%, demonstrating the need to find active treatments for this group. Outcomes varied according to tumor histological subtype. Nearly all recurrences occurred in patients with the translocation histology and renal medullary carcinoma; recurrences were rare in other subtypes.

A follow-up study called AREN1721 is now open in the Children’s Oncology Group and adult cancer cooperative groups that participate in the National Clinical Trials Network. This study involves a comparison of two treatment regimens for metastatic or unresectable “translocation” renal cell carcinoma, the most common subtype of renal cell carcinoma in children, adolescents and young adults. The treatment regimens will include nivolumab, a PD1 immune checkpoint inhibitor, with or without axitinib, a tyrosine kinase inhibitor that targets vascular endothelial growth factor receptor (VEGFR).

Jeffrey Dome

Treating Wilms Tumor with vincristine and irinotecan

Jeffrey Dome

“The study was impactful because it established the activity of vincristine/irinotecan against Wilms tumor. Based on these findings, this chemotherapy combination will be applied more broadly in the treatment of Wilms tumor,” says study leader Jeffrey S. Dome, M.D, Ph.D.

Wilms tumor, the most common kidney cancer of childhood, may be classified into different subtypes based on its appearance under the microscope. The “favorable histology” subtype is associated with an excellent survival rate of approximately 90%, whereas the “diffuse anaplastic” subtype is associated with survival rates of only 55% for patients with stage II-IV disease.

The Children’s Oncology Group AREN0321 study, led by Jeffrey S. Dome, M.D, Ph.D., vice president of the Center for Cancer and Blood Disorders at Children’s National Hospital, tested the anti-tumor activity of the chemotherapy combination vincristine and irinotecan in patients with metastatic diffuse anaplastic Wilms tumor.

The study also evaluated whether a new treatment regimen containing carboplatin in addition to the currently used agents (vincristine, doxorubicin, cyclophosphamide and etoposide) would improve patient outcomes. The results, published in the March 5th issue of the Journal of Clinical Oncology, showed that the vincristine/irinotecan combination is highly active. Out of the group, 78% of patients who received this combination had an objective tumor response.

The study also demonstrated that additional chemotherapy drugs can reduce the rate of relapse, but it is likely that we have reached the limit of what children can tolerate. “Future gains will likely be made by using agents with novel mechanisms of action, such as immunotherapy and new drugs that target the molecular abnormalities of Wilms tumor cells,” says Dr. Dome.

Moreover, the additional chemotherapy agents improved cancer-free survival rates to levels unprecedented for diffuse anaplastic Wilms tumor. However, the decrease in relapse rate came at the cost of increased toxicity.

“The study was impactful because it established the activity of vincristine/irinotecan against Wilms tumor. Based on these findings, this chemotherapy combination will be applied more broadly in the treatment of Wilms tumor,” Dr. Dome added.

proton center

Johns Hopkins Proton Therapy Center opens in collaboration with Children’s National

proton center

The Center at Sibley offers state-of-the-art, pencil beam proton therapy equipment, as well as next-generation imaging technologies such as dual energy CT-guided treatment that reduces the range of error, and the latest innovation in biomatrix magnetic resonance imaging designed to target moving tumors in organs like the lung and liver.

Pediatric cancer patients in the Greater Washington region now have access to one of the most advanced, lifesaving proton therapy technologies offered in the U.S. The Johns Hopkins Proton Therapy Center opened Oct.28, 2019, at Sibley Memorial Hospital in collaboration with Children’s National Hospital.

The proton collaboration with Children’s National expands an existing collaboration between Children’s National and Johns Hopkins Medicine that established the pediatric radiation oncology program at Sibley, which treats a wide range of children’s cancer. Now, Sibley will offer the only proton center in the Washington D.C. region with a dedicated pediatric team, staff who are trained in pediatrics instead of adult providers who also treat children.

“This collaboration allows us to bring the latest technology to the region and offer the most advanced cancer treatment to help children live better lives,” says Kurt Newman, M.D., president and CEO at Children’s National. “As one of the Top 10 children’s hospitals in the nation, our goal is to ensure that patients and families are receiving the best care possible.”

The Center at Sibley offers state-of-the-art, pencil beam proton therapy equipment, as well as next-generation imaging technologies such as dual energy CT-guided treatment that reduces the range of error, and the latest innovation in biomatrix magnetic resonance imaging designed to target moving tumors in organs like the lung and liver. A large mechanical arm called a gantry can move the beam 360 degrees around the patient, treating the tumor from several angles as it destroys tumor cells layer by layer.

“Proton therapy is an advanced technology that allows radiation to be delivered precisely to cancer tissue,” says Jeffrey Dome, M.D., Ph.D., vice president for Cancer and Blood Disorders at Children’s National. “This provides a significant advantage compared with conventional radiation therapy, especially in children, where sparing the healthy tissue that surrounds the tumor may be critical for normal growth and development. Proton therapy shows great promise to reduce long-term side effects of radiation treatment.”

The Center at Sibley will have a fully integrated research room, which will allow clinical, basic science and medical physics faculty to advance clinical trial research, translational research and technology development research in proton therapy. Leading experts and oncologists will study proton outcomes for sarcoma, gynecological tumors, pancreatic and liver tumors, lymph node cancers and tumors located near the heart and major blood vessels, such as lung or breast cancers. In addition, the researchers will examine how the proton energy that kills cancer cells interacts with non-cancerous cells and tissue surrounding the tumors.

The JJohns Hopkins Proton Therapy Center opens in phases. The first treatment room opened October 2019. The second room is scheduled to open in spring 2020, and the third room and fixed beam research room are scheduled to open in fall 2020.

Chromosomes

Precision medicine for Wilms tumor patients

Chromosomes

Previously, researchers discovered that loss of heterozygosity (LOH) on chromosomes 1 and 16 is associated with a significantly increased risk of relapse of Wilms tumor.

About 650 children are diagnosed each year in the U.S. with Wilms tumor, the most common pediatric kidney cancer. The vast majority of patients respond well to the current standard of care involving a combination of surgery, chemotherapy and radiation. However, approximately 20% of patients with “favorable histology” Wilms tumor experience recurrence.

Previously, researchers discovered that loss of heterozygosity (LOH) on chromosomes 1 and 16 is associated with a significantly increased risk of relapse. A research team in the Children’s Oncology Group (COG), led by Jeffrey Dome, M.D., Ph.D., vice president of the Center for Cancer and Blood Disorders at Children’s National Hospital, sought to determine whether an augmented chemotherapy regimen can overcome the negative effect of LOH.

More than 2,500 patients with Wilms tumor were enrolled in the biology and classification study over a 7–year period. Tumor tissue was tested for LOH and patients with LOH at both chromosomes 1 and 16 received more intensive chemotherapy regimens compared to the standard approach. The results showed that the increased treatment provided a statistically significant benefit in the 4-year event-free survival, with trends toward improved overall survival. For low-stage disease (stage I-II), the four-year event-free survival was 87.3%, compared to a historical rate of 68.8%. Similarly, for advanced stage disease (stage III/IV) four-year event-free survival was 90.2%, compared with 61.3% historically.

Although the new regimens involved additional chemotherapy agents compared to the standard regimens, the short-term toxicities were expected and manageable. There is an increased risk of long-term toxicity including infertility and second malignancies, which requires careful discussion with families. Future studies will seek to mitigate these risks with newer chemotherapy agents.

By better understanding which patients might benefit from more intensive treatment regimens through precision medicine, doctors can tailor therapy according to the risk of relapse, Dr. Dome says.

“This study represents a significant milestone in the treatment of Wilms tumor because it is the first to demonstrate that patient outcome can be improved using a molecular biomarker to guide treatment,” he explains. “We have entered the age of precision medicine for Wilms tumor.”

Children’s National ranked No. 6 overall and No. 1 for newborn care by U.S. News

Children’s National in Washington, D.C., is the nation’s No. 6 children’s hospital and, for the third year in a row, its neonatology program is No.1 among all children’s hospitals providing newborn intensive care, according to the U.S. News Best Children’s Hospitals annual rankings for 2019-20.

This is also the third year in a row that Children’s National has been in the top 10 of these national rankings. It is the ninth straight year it has ranked in all 10 specialty services, with five specialty service areas ranked among the top 10.

“I’m proud that our rankings continue to cement our standing as among the best children’s hospitals in the nation,” says Kurt Newman, M.D., President and CEO for Children’s National. “In addition to these service lines, today’s recognition honors countless specialists and support staff who provide unparalleled, multidisciplinary patient care. Quality care is a function of every team member performing their role well, so I credit every member of the Children’s National team for this continued high performance.”

The annual rankings recognize the nation’s top 50 pediatric facilities based on a scoring system developed by U.S. News. The top 10 scorers are awarded a distinction called the Honor Roll.

“The top 10 pediatric centers on this year’s Best Children’s Hospitals Honor Roll deliver outstanding care across a range of specialties and deserve to be nationally recognized,” says Ben Harder, chief of health analysis at U.S. News. “According to our analysis, these Honor Roll hospitals provide state-of-the-art medical expertise to children with rare or complex conditions. Their rankings reflect U.S. News’ assessment of their commitment to providing high-quality, compassionate care to young patients and their families day in and day out.”

The bulk of the score for each specialty is based on quality and outcomes data. The process also includes a survey of relevant specialists across the country, who are asked to list hospitals they believe provide the best care for patients with challenging conditions.

Below are links to the five specialty services that U.S. News ranked in the top 10 nationally:

The other five specialties ranked among the top 50 were cardiology and heart surgery, diabetes and endocrinology, gastroenterology and gastro-intestinal surgery, orthopedics, and urology.

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Children’s National physicians attend the International Society of Paediatric Oncology in Japan

SIOP logo

From November 16 to 19, medical professionals, clinicians, nurses and oncology patients and families from around the globe gathered for the International Society of Paediatric Oncology (SIOP) in Kyoto, Japan. Pediatric experts in their respective fields Jeffrey Dome, M.D., Ph.D., AeRang Kim, M.D., Ph.D., Steven Hardy, Ph.D., and Karun Sharma, M.D., attended SIOP representing Children’s National. The four-day scientific programme engaged those in pediatric oncology with educational lectures, keynote speakers, tailored sessions for survivors, families and support organizations, free paper sessions, specialist sessions and Meet the Expert talks.

Dr. Kim, an oncologist with the Center for Cancer and Blood Disorders and a member of the solid tumor faculty at Children’s National, presented with Dr. Sharma, director of Interventional Radiology at Children’s, on “Interventional Radiology: Technology and Opportunities” in Meet the Expert talks on both Saturday and Sunday of the programme. They discussed background information, preclinical studies, current, ongoing studies of high-intensity focused ultrasound (HIFU), HIFU in combination with heat sensitive formulated chemotherapy and future directions. In 2017, Children’s National was the first U.S. children’s hospital to successfully use MR-HIFU to treat osteoid osteoma, and is currently accruing on early phase studies evaluating HIFU ablation and HIFU in combination with lyso-thermosensitive liposomal doxorubicin for pediatric patients with refractory/recurrent solid tumors.

Dr. Hardy, a pediatric psychologist in the Center for Cancer and Blood Disorders at Children’s, presented on “Brief Psychosocial Screening to Identify Patients in Need of a Mental Health Treatment Referral in a Childhood Cancer Survivorship Clinic.” In his educational lecture, Dr. Hardy described findings that show a brief mental and behavioral health questionnaire given to patients in the Children’s National survivorship clinic is a sensitive screening tool that can identify patients in need of more formal psychosocial evaluation and treatment. He also presented data supporting the use of a lower threshold of psychological symptoms necessary to trigger discussions about mental health treatment compared to previous reports. The key implication of Dr. Hardy’s work is that survivorship clinics lacking embedded psychology support could adopt this questionnaire, which is publically available and translated into 86 languages, to help identify survivors with mental and behavioral health concerns and ensure appropriate referrals are made.

Dr. Dome, Vice President of the Center for Cancer and Blood Disorders, served on the SIOP Scientific Programme Advisory Committee, which selected the topics for presentation.

SIOP provides an international forum for the sharing of new research and ideas related to pediatric oncology. The annual conference furthers the efforts made towards developing new treatments and cures and opens the conversation, encouraging innovation and collaboration with experts from around the world. Children’s National has taken part in SIOP for many years, most recently hosting the meeting in Washington, D.C., in 2017.

Jeffrey Dome

The impact of surveillance imaging to detect relapse in Wilms tumor patients

Jeffrey Dome

Dr. Jeffrey Dome, M.D., Ph.D., vice president, Center for Cancer and Blood Disorders.

The Children’s Oncology Group published an article in the Journal of Clinical Oncology looking at the impact that surveillance imaging has on patients with Wilms tumor (WT), the most common kidney cancer in children.

Despite the risks and costs, the use of computed tomography (CT) for routine surveillance to detect recurrence in patients with WT has increased in recent years. The rationale for using CT scans rather than chest x-rays (CXR) and abdominal ultrasounds (US) is that CT scans are more sensitive, thereby enabling recurrences to be detected earlier.

In this study, led by Jeffrey S. Dome, M.D., Ph.D, vice president of the Center for Cancer and Blood Disorders at Children’s National Health System, researchers conducted a retrospective analysis of patients enrolled in the fifth National Wilms Tumor Study (NWTS-5) who experienced relapse to determine if relapse detection with CT scan correlates with improved overall survival compared with relapse detection by CXR or abdominal US.

A total of 281 patients with favorable-histology WT (FHWT) were included in the analysis. The key findings of the study were that:

  • Among patients with relapse after completion of therapy, outcome was improved in patients whose relapse was detected by surveillance imaging rather after signs and symptoms developed.
  • A higher disease burden at relapse, defined by the diameter of the relapsed tumor and the number of sites of relapse, was associated with inferior survival.
  • Relapses detected by CT scan were detected earlier and were smaller on average than relapses detected by CXR or US.
  • However, there was no difference in survival between patients whose relapse was detected by CT versus CXR or US.

An analysis of radiation exposure levels showed that surveillance regimes including CT scans have about seven times the radiation exposure compared to regimens including only CXR and US. Moreover, the cost to detect each recurrence reduced by 50 percent when CXR and US are used for surveillance.

“The results of this study will be practice changing,” said Dr. Dome, one of the doctors leading the clinical trial. “The extra sensitivity that CT scans provide compared to CXR and US do not translate to improved survival and are associated with the downsides of extra radiation exposure, cost and false-positive results that can lead to unnecessary stress and medical interventions,” he added. “Although counter-intuitive, the more sensitive technology is not necessarily better for patients.”

In conclusion, the doctors found that the elimination of CT scans from surveillance programs for unilateral favorable histology Wilms tumor is unlikely to compromise survival. However, it could result in substantially less radiation exposure and lower health care costs. Overall, the risk-benefit ratio associated with imaging modalities should be considered and formally studied for all pediatric cancers.

Learn more about this research in a podcast from the Journal of Clinical Oncology.

Affiliations

Elizabeth A. Mullen, Dana-Farber Cancer Institute/Boston Children’s Cancer and Blood Disorders Center, Boston, MA; Yueh-Yun Chi and Emily Hibbitts, University of Florida, Gainesville, FL; James R. Anderson, Merck Research Laboratories, North Wales, PA; Katarina J. Steacy, University of Maryland Medical Center, Baltimore, MD; James I. Geller, Cincinnati Children’s Hospital Medical Centre, Cincinnati, OH; Daniel M. Green, St Jude Children’s Research Hospital, Memphis, TN; Geetika Khanna, Washington University School of Medicine, St Louis, MO; Marcio H. Malogolowkin, University of California at Davis Comprehensive Cancer Center, Sacramento, CA; Paul E. Grundy, Stollery Children’s Hospital, University of Alberta, Alberta; Conrad V. Fernandez, University, Halifax, Nova Scotia, Canada; and Jeffrey S. Dome, Children’s National Health System, George Washington University School of Medicine and Health Sciences, Washington, D.C.

Making the grade: Children’s National is nation’s Top 5 children’s hospital

Children’s National rose in rankings to become the nation’s Top 5 children’s hospital according to the 2018-19 Best Children’s Hospitals Honor Roll released June 26, 2018, by U.S. News & World Report. Additionally, for the second straight year, Children’s Neonatology division led by Billie Lou Short, M.D., ranked No. 1 among 50 neonatal intensive care units ranked across the nation.

Children’s National also ranked in the Top 10 in six additional services:

For the eighth year running, Children’s National ranked in all 10 specialty services, which underscores its unwavering commitment to excellence, continuous quality improvement and unmatched pediatric expertise throughout the organization.

“It’s a distinct honor for Children’s physicians, nurses and employees to be recognized as the nation’s Top 5 pediatric hospital. Children’s National provides the nation’s best care for kids and our dedicated physicians, neonatologists, surgeons, neuroscientists and other specialists, nurses and other clinical support teams are the reason why,” says Kurt Newman, M.D., Children’s President and CEO. “All of the Children’s staff is committed to ensuring that our kids and families enjoy the very best health outcomes today and for the rest of their lives.”

The excellence of Children’s care is made possible by our research insights and clinical innovations. In addition to being named to the U.S. News Honor Roll, a distinction awarded to just 10 children’s centers around the nation, Children’s National is a two-time Magnet® designated hospital for excellence in nursing and is a Leapfrog Group Top Hospital. Children’s ranks seventh among pediatric hospitals in funding from the National Institutes of Health, with a combined $40 million in direct and indirect funding, and transfers the latest research insights from the bench to patients’ bedsides.

“The 10 pediatric centers on this year’s Best Children’s Hospitals Honor Roll deliver exceptional care across a range of specialties and deserve to be highlighted,” says Ben Harder, chief of health analysis at U.S. News. “Day after day, these hospitals provide state-of-the-art medical expertise to children with complex conditions. Their U.S. News’ rankings reflect their commitment to providing high-quality care.”

The 12th annual rankings recognize the top 50 pediatric facilities across the U.S. in 10 pediatric specialties: cancer, cardiology and heart surgery, diabetes and endocrinology, gastroenterology and gastrointestinal surgery, neonatology, nephrology, neurology and neurosurgery, orthopedics, pulmonology and urology. Hospitals received points for being ranked in a specialty, and higher-ranking hospitals receive more points. The Best Children’s Hospitals Honor Roll recognizes the 10 hospitals that received the most points overall.

This year’s rankings will be published in the U.S. News & World Report’s “Best Hospitals 2019” guidebook, available for purchase in late September.

pill bottles and pills

New treatment approach shows promise for patients with stage IV Wilms tumor

The study assessed the benefit of adding two additional chemotherapy agents, cyclophosphamide and etoposide, to the treatment regimen for patients with incomplete lung nodule response or tumor loss of heterozygosity (LOH) at chromosomes 1p and 16q, both associated with interior outcomes in previous studies.

Wilms tumor, which first develops in the kidneys, is the fifth most common cancer in children under 15 years old. While overall outcomes for patients with Wilms tumor are excellent, patients with metastatic disease, with the lung as the most common site of spread, fare worse than patients with localized disease. That’s why a new study showing significantly improved survival rates for patients with stage IV Wilms tumors with lung metastases is making waves in the pediatric oncology community.

The study, “Treatment of Stage IV Favorable Histology Wilms Tumor With Lung Metastases: A Report From the Children’s Oncology Group AREN0533 Study” – recently published in the Journal of Clinical Oncology with Jeffrey Dome, M.D., Ph.D., vice president for the Center for Cancer and Blood Disorders at Children’s National Health System, as the senior author – assessed whether lung radiation therapy, part of the standard treatment in combination with chemotherapy drugs, can be avoided for patients with complete lung nodule response after six weeks of chemotherapy. Conversely, the study assessed the benefit of adding two additional chemotherapy agents, cyclophosphamide and etoposide, to the treatment regimen for patients with incomplete lung nodule response or tumor loss of heterozygosity (LOH) at chromosomes 1p and 16q, both associated with interior outcomes in previous studies. The results show that:

  • The new approach to therapy resulted in a 4-year overall survival rate of 96 percent, compared to 84 percent on the predecessor study.
  • About 40 percent of patients with Wilms tumor and lung metastases can be spared initial upfront lung radiation and still have outstanding survival. This will decrease the long-term risk of heart toxicity and breast cancer.
  • Patients with incomplete lung nodule response after six weeks of therapy with cyclophosphamide and etoposide had significantly better 4-year event-free survival: 89 percent compared with 75 percent that was expected based on historical data.
  • Intensification of therapy for patients with LOH at 1p and 16q was highly effective: 4-year event-free survival rate improved from 66 percent on the previous study to 100 percent.

“These findings will change clinical practice and improve survival for patients with Wilms tumor whose cancer has spread to the lungs” said Dr. Dome. “The risk-adapted approach to treatment based on tumor biology and tumor response provides a framework for future studies as we come one step closer to achieving 100 percent survival without treatment-associated side effects.”

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Advancing cures for pediatric cancer: Highlights from leading Children’s National experts at SIOP 2017

In mid-October 2017, nearly 2,000 clinicians, scientists, nurses, health care professionals and cancer patients and survivors gathered in Washington, D.C., for SIOP 2017, the Annual Congress of the International Society of Paediatric Oncology. For four days, attendees heard from world-renowned experts while exchanging ideas and information, all in the name of advancing cures for childhood cancer.

Hosted in the hometown of Children’s National Health System and chaired by Jeffrey Dome, M.D., Ph.D., Vice President of the Center for Cancer and Blood Disorders and Chief of Oncology at Children’s National Health System, more than 20 doctors and nurses from Children’s National made an impact on participants through a series of widely attended sessions and addresses, including:

  • Symposium lecture on the latest approaches in anti-viral T-cell therapy to improve patient outcomes, given by Catherine Bollard, M.D., M.B.Ch.B.
  • Keynote lecture on DICER1 mutations in pediatric cancer, given by Ashley Hill, M.D., whose study of a rare childhood lung cancer and gene mutations set the stage for a better understanding of microRNA processing gene mutations in the development of pediatric cancer.
  • Education session on new therapies for sarcomas, led by AeRang Kim, M.D., Ph.D., and Karun Sharma, M.D., Ph.D., sharing research on new approaches for local control of sarcomas, such as surgery, radiation and other ablative measures.
  • Education session on new therapies for gliomas, led by Roger J. Packer, M.D., with presentations on immunotherapy from Eugene Hwang, M.D., and targeted therapy by Lindsay Kilburn, M.D.
  • Podium paper presentation on a new method to measure cancer treatment toxicities as reported by the child by Pamela Hinds, Ph.D., RN, FAAN, as well as an education session on advanced care planning, led by Hinds with a presentation from Maureen E. Lyon, Ph.D.

“These sessions and lectures provided a glimpse into the groundbreaking work by SIOP attendees from around the world,” says Dr. Dome. “Children’s National is proud to play an active role in the development of life-saving treatments for children with cancer and our clinicians look forward to another year of revolutionary developments.”

For more on this year’s SIOP, see the Children’s National press release.

  • Jeffrey Dome, M.D., Ph.D., addresses a group of international colleagues at a reception at Children’s National.

    Jeffrey Dome SIOP

  • Catherine Bollard, M.D., M.B.Ch.B., addresses a group of international colleagues at a reception at Children’s National.

    Catherine-Bollard-SIOP

  • Lindsay Kilburn, M.D., engages with peers from around the world at a reception at Children’s National.

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little girl with cancer

New approach improves pediatric kidney cancer outcomes

little girl with cancerWilms tumor, also known as nephroblastoma, is the most common pediatric kidney cancer, typically seen in children ages three to four. Compared to patients with unilateral Wilms tumors, children with bilateral Wilms tumors (BWT) have poorer event-free survival (EFS) and are at higher risk for later effects such as renal failure. The treatment of BWT is challenging because it involves surgical removal of the cancer, while preserving as much healthy kidney tissue as possible to avoid the need for an organ transplant.

A new Children’s Oncology Group (COG) study published in the September issue of the Annals of Surgery demonstrated an exciting new approach to treating children diagnosed with BWT that significantly improved EFS and overall survival (OS) rates after four years when compared to historical rates. Jeffrey Dome, M.D., Ph.D., Vice President of the Center for Cancer and Blood Disorders at Children’s National Health System, was co-senior author of this first-ever, multi-institutional prospective study of children with BWT.

Historically, patients with BWT have had poor outcomes, especially if they have tumors with unfavorable histology. In this study, Dr. Dome and 18 other clinical researchers followed a new treatment approach consisting of three chemotherapy drugs before surgery rather than the standard two drug regimen, surgical removal of cancerous tissue within 12 weeks of diagnosis, and postoperative chemotherapy that was adjusted based on histology.

The study found that preoperative chemotherapy expedited surgical treatment, with 84 percent of patients having surgery within 12 weeks of diagnosis. The new treatment approach also vastly improved EFS and OS rates for patients participating in the study. The four-year EFS rate was 82.1 percent, compared to 56 percent on the predecessor National Wilms Tumor Study-5 (NWTS-5) study. The four-year OS rate was 94.9 percent, compared to 80.8 percent on NWTS-5.

“I am very encouraged by these results, which I believe will serve as a benchmark for future studies and lead to additional treatment improvements, giving more children the chance to overcome this diagnosis while sparing kidney tissue,” says Dr. Dome.

A total of 189 patients at children’s hospitals, universities and cancer centers in the United States and Canada participated in this study. These patients will continue to be followed for 10 years to track kidney failure rates. This study was funded by grants from the National Institutes of Health to the Children’s Oncology Group.