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Seeking Ways to Improve Cancer Care for Children, Adolescents, and Young Adults

Eastman, Peggy

doi: 10.1097/01.COT.0000465162.19939.7a
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WASHINGTON—While children with pediatric cancers are surviving longer, there is a pressing need for improvements in their care, according to speakers at a workshop here hosted by the Institute of Medicine's National Cancer Policy Forum (NCPF) and the American Cancer Society. A summary of the meeting, titled “Comprehensive Cancer Care for Children and Their Families,” is expected to be published by National Academies Press.

Otis W. Brawley, MD, Chief Medical Officer of the ACS and a member of both the workshop planning committee and NCPF itself, quoted the well-known statistic that while less than three percent of adults with cancer enroll in clinical trials, 60 to 70 percent of children with cancer up to age 12 are treated on clinical trials. “That's a very good thing,” he said.

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But he and other speakers noted that about 2,000 American children still die of cancer each year. And, this is most likely an underestimate, because death certificates of childhood cancer survivors who die years later usually do not list cancer as the cause of death—even if the late effects of treatment contributed to the cancer patient's demise.

“Children are still dying of these diseases,” said Mary Jo Kupst, PhD, Professor Emeritus at Medical College of Wisconsin.

Childhood cancer care is considered a success story by many, since the U.S. five-year survival rate of children with cancer has risen to about 80 percent, according to the latest estimates by the American Childhood Cancer Organization.

But federal funding for the Children's Oncology Group (COG)—a National Cancer Institute-supported consortium of more than 220 childhood centers—has dropped by 30 percent in inflation-adjusted dollars over the last 10 years, hampering research advances in diagnosis, treatment, and psychosocial services, said COG Chair Peter C. Adamson, MD, who holds the Alan R. Cohen Endowed Chair in Pediatrics at the Children's Hospital of Philadelphia and Professor of Pediatrics and Pharmacology at the University of Pennsylvania School of Medicine.

“The idea that we can do more with less is nonsense,” he said.

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‘Disproportionate Hit’

Decrying the fact that there were no members of Congress at the IOM workshop, Adamson told OT that while federal cuts for cancer research have been detrimental for all of oncology, they have been especially devastating for childhood cancers. This disproportionate hit, he said, is because unlike adult cancer research, pediatric cancer research is almost entirely dependent on federal dollars. “We have been very adversely affected,” he said.

“We've got to change the dialogue that stops at presenting childhood cancer as a closed-chapter success story and convince Congress to increase funding for needed pediatric cancer research. The price our children pay is unacceptable.”

Workshop Planning Committee Chair Joanne Wolfe, MD, MPH, Chief of the Division of Pediatric Palliative Care at Dana-Farber Cancer Institute and Director of Palliative Care at Boston Children's Hospital, emphasized that children with cancer suffer physically, psychologically, and socially and the diagnosis is devastating to the entire family.

Also, she noted, children with cancer and their family members must cope with developmental changes while patients are going through treatment and its side effects, along with long-term health risks and late-stage effects.

“We don't even know how much better we can do—absolutely we need to raise the bar,” Wolfe said.

“These cancer treatments yield a steep cost in survival, including gross mental impairment, poor motor functioning, and highly compromised social well-being for decades of life after treatment.”

In addition, she cited the stress on parents of caring for pediatric cancer patients, making difficult treatment decisions with their oncologists; balancing jobs and the child's medical and psychological needs in what is likely an unfamiliar medical environment; helping the child navigate school and social issues; and tending to the needs of other healthy siblings.

“At all stages of the cancer, children and families experience distress and suffering,” agreed Lori Wiener, PhD, DCSW, LCSW-C, Head of the National Cancer Institute's Pediatric Psychosocial Support and Research Program and a psychologist in the Center for Cancer Research.

Like Wolfe, Wiener advocated integrating pediatric palliative care into cancer care for children starting at the time of diagnosis: “The recommendation is not to think of palliative care as an on-off switch,” she said.

“Families want access to palliative care. Emerging data suggests that palliative care improves outcomes.” Specifically, she said, when palliative care is integrated into the child's treatment plan from the beginning, children have shorter hospitalizations and fewer visits to the emergency department.

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Incentives for Pharmaceutical Companies

Childhood cancers are considered rare. At the meeting, several speakers stressed the need for incentives to encourage pharmaceutical companies to pursue clinical trials on drugs for pediatric cancer patients.

“There are a limited number of trials that can be done because of the limited number of patients,” said workshop planning committee member and immediate past chair of COG Gregory H. Reaman, MD, Associate Director for Oncology Sciences in the Center for Drug Evaluation and Research at the Food and Drug Administration and Professor of Pediatrics at George Washington University School of Medicine and Health Sciences.

Reaman noted that use of the term “rare” has a legislative basis—childhood cancers that affect fewer than 200,000 patients are considered orphan diseases. “Companies fear investing in a pediatric development plan that may flame out.” But despite the risk, several companies are pursuing drug research for pediatric cancer indications.

Indeed, while the IOM workshop was in session, the FDA approved dinutuximab (Unituxin)—whose manufacturer, United Therapeutics, had been granted priority review and orphan product designation—as part of first-line therapy for pediatric patients with high-risk neuroblastoma (OT 4/10/15 issue). This is the second rare pediatric disease priority review voucher granted by the FDA since inception of the rare pediatric disease review voucher program, which is designed to encourage development of new therapies for prevention and treatment of certain rare diseases in children.

Unituxin carries a Boxed Warning alert that the drug irritates nerve cells, causing severe pain that requires treatment with intravenous narcotics and that it can also cause nerve damage and life-threatening infusion reactions, including upper airway swelling, difficulty breathing, and low blood pressure, during or shortly after completion of the infusion. The drug can also cause other serious side effects including infections, eye problems, electrolyte abnormalities and bone marrow suppression.

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‘Robust Pediatric Development Plan’ at BMS

“We have a robust pediatric development plan distinct from that for adults,” said another speaker, Mary Brigid Bradley-Garelik, MD, MPH, Director of Oncology Global Clinical Research in Research and Development at Bristol-Myers Squibb.

Specifically, she cited studies on a pediatric drug formulation for the oral agent dasatinib, a Bcr-Abl tyrosine kinase inhibitor, for chronic myeloid leukemia; and pediatric studies of the immuno-oncology inhibitors ipilimumab and nivolumab. These drugs work by activating T cells and turning off the immune system's checkpoint.

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LLS's Therapy Acceleration Program

“We have invested heavily in immunotherapy,” said Lee Greenberger, PhD, Chief Scientific Officer of the Leukemia & Lymphoma Society (LLS), in describing that organization's efforts to translate research advances into bedside therapies for children and adults. He described the Therapy Acceleration Program (TAP), an LLS initiative that aims to bridge the “valley of death” in drug development, the period most prevalent in early-stage development when many compounds fail.

Greenberger said the LLS has invested about $1 billion in research since 1954, and cited the clout of public/private partnerships in advancing biomedical research. “I think we're making good progress toward cures for blood cancers. Overall, what's happening in the blood cancer space is truly remarkable.”

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Include PROs

Several speakers emphasized the need to include patient-reported outcomes (PROs) in pediatric cancer clinical trials. “Clinicians are not always good at picking up PROs,” said Lillian Sung, MD, PhD, Associate Professor and Scientist at the Hospital for Sick Children in Toronto and COG's Chair of Cancer Control and Supportive Care.

Health professionals and parents need to know the core symptoms they should look for in pediatric cancer patients in order to intervene early: “Is self-report always feasible? Absolutely not,” she said, citing the need for parents and guardians to serve as proxy reporters.

Sung recommended identifying PRO characteristics that can be included in early-phase pediatric cancer trials; clarifying the role of parents and guardians versus self-reporting in pediatric oncology; providing suggestions for core instruments and core symptoms, such as pain, mouth sores, and fatigue; identifying PROs that can be used in adolescent and young adult (AYA) cancer patients; identifying clinically important differences in PROs; implementing electronic PROs; focusing on data quality, completion of trials, and dissemination of knowledge; and bringing PROs to the young patient's bedside, which will improve patient/provider communication.

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Meeting Special Needs

Speakers also emphasized that AYA cancer patients have special needs that must be met, especially since the AYA years are fraught with intense emotions and peer pressure. In 2013 the IOM published a workshop summary, “Identifying and Addressing the Needs of Adolescents and Young Adults with Cancer” (OT 8/25/13 issue), which discussed the late and long-term effects of treatment; social and psychological issues; financial issues; lack of services such as support groups; and fears about developing a second cancer.

“The farther out patients are from their treatment, the more likely they are to have at least one serious health condition,” said Kevin Oeffinger, MD, Director of the Cancer Survivorship Center at Memorial Sloan Kettering Cancer Center, who was also a member of the IOM workshop on identifying the needs of AYA cancer patients. “The message is that we see these things unfold over time.”

He cited the Childhood Cancer Survivor Study, which found that almost half of childhood cancer survivors experience a serious or life-threatening condition or die between five and 30 years after diagnosis, and that almost three-quarters develop at least one chronic condition. “We need a risk-stratified, shared-care model for cancer survivors,” he said.

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Descriptions from Parents

Also at the IOM meeting, several parents described what it is like to have a child with a cancer diagnosis. Alyssa Sandler was diagnosed at age three with leukemia, successfully treated, and passed her five-year mark, said her father, Eric S. Sandler, MD, Associate Professor of Pediatrics at both the Mayo School of Medicine and the University of Florida, Chief of Pediatric Hematology/Oncology at Nemours Children's Clinic, and a member of the IOM workshop planning committee. “She had a recurrence after six years, which I tell people isn't supposed to happen.”

Alyssa was again successfully treated but “she was having a lot of issues in school,” Sandler said. “I think I'm a pretty sophisticated consumer, but we could not get the services we needed in the school district.” So Alyssa entered private school. Ironically, today Alyssa—who graduated from college and obtained a master's degree—teaches special education in the very school where she could not get the help she needed as a young cancer survivor.

Sandler told OT that some of Alyssa's issues were peer issues. “When her peers were going through their developmental changes, she was going through treatment,” he said. “She was primarily with adults.”

Another parent, Beth Anne Baber, PhD, MBA, described trying to find the least-toxic treatment regimen for her son Conor, who was diagnosed with high-risk neuroblastoma at about age one and a half when she worked at the Salk Institute: “We knew the treatments we would be forced to use would be toxic to normal cells. We tailored his treatment based on quality of life,” using genetic markers as opposed to relying solely on clinical pathology.

But, she said, it was difficult: “We were shocked that there were not decision support tools for doing this.” Today Conor is 10, cancer-free, and a devotee of extreme sports. Baber co-founded the Nicholas Conor Institute to accelerate, develop, and deploy innovative cancer treatments for children and AYAs.

The most harrowing stories at the IOM meeting came from parents whose children died of cancer. Gavin B. Lindberg's and his wife Wendy's only child, Evan, was diagnosed at age three with neuroblastoma and died at home at age seven, after experiencing pain, distress, and anxiety. “It's unbelievable what kids like Evan go through,” said Lindberg, Senior Director of Government Affairs and Patient Relations at Grifols, a global health care company. “We're here to speak for Evan and other kids like him.”

Lindberg described how home hospice care failed Evan; during this crucial period at home a nurse (whose experience was with adult patients) refused to give him IV pain medications. “He could have had good care at home but didn't,” said Lindberg, who has since founded the EVAN Foundation to support research on neuroblastoma. “If we can't get this right, then shame on us.”

Finally, Victoria Sardi-Brown, PhD, described trying to get the best care for her son Mattie, who died of osteosarcoma at the age of seven. “We must remember that cancer care is much more than medicine,” she said, relating how Mattie told her he was ugly and no one wanted to be his friend, and how he screamed in pain. “I was confronted with a health care system that wondered, is it really possible for Mattie to be in that much pain?”

Sardi-Brown and her husband Peter have now founded the Mattie Miracle Cancer Foundation to make sure children with cancer and their families receive the psychosocial support they need. The foundation is also supporting the Standards Project, a national initiative to develop standards for the psychosocial care of children with cancer and their families.

Copyright © 2015 Wolters Kluwer Health, Inc. All rights reserved.
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