The National Comprehensive Cancer Network® (NCCN®) has released the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Pediatric Acute Lymphoblastic Leukemia (ALL)—the most comprehensive and up-to-date evidence-based, consensus-driven guidelines for treating children with cancer outside of a clinical trial setting. These new NCCN Guidelines® are the first of several planned to address various pediatric cancers.
"We are publishing the NCCN Guidelines for Pediatric ALL in response to a growing global need for protocols that ensure children with cancer receive the best care possible, no matter where they're treated," said Robert W. Carlson, MD, Chief Executive Officer, NCCN. "Thankfully, in recent years pediatric ALL has reached a very high cure rate, with almost all low-risk patients achieving long-term survival. However, that means fewer patients are being treated within the context of a clinical trial, which creates the need for treatment guidelines that set sufficient and appropriate standards for care. NCCN's evidence- and expert-consensus-based approach, proven track record for improving outcomes, and ability to provide rapid updates, allows us to fill this void."
ALL is a blood cancer that impacts about 2,900 people aged 21 years and younger in the United States every year. It is the most common children's cancer, accounting for 35% of all cancers in that age group.1 Treatment typically involves multi-agent chemotherapy regimens, lasts for two-to-three years, and is comprised of four components: remission induction, consolidation, maintenance, and central nervous system-directed therapy.2 Pediatric ALL is also at the forefront for innovative new approaches like targeted therapy and immunotherapy, including the first Food & Drug Administration (FDA) approval for chimeric antigen receptor (CAR) T-cell therapy.3
"The cure rate for pediatric ALL in the U.S. has risen from 0% in the 1960s to nearly 90% today," said Patrick Brown, MD, Associate Professor of Oncology and Pediatrics, Director, Pediatric Leukemia Program, The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Chair, NCCN Guidelines for Adult and Pediatric ALL. "This is among the most profound medical success stories in history. The NCCN Guidelines assemble today's 'best practices' into one comprehensive, consensus document, based on input from experts across medical disciplines, including pediatric oncologists, radiation oncologists, bone marrow transplant physicians, infectious disease experts, and molecular pathologists."
"These guidelines ensure that every child gets the highest quality treatment, even outside of a clinical trial," said Hiroto Inaba, MD, PhD, Member, St. Jude Children's Research Hospital, Vice Chair, NCCN Guidelines for Pediatric ALL. "Our recommendations include a strong focus on supportive care, so hopefully patients will not experience severe side effects. Pediatric ALL survivors live a long time; we have to consider long-term effects as well."
The NCCN Guidelines for Pediatric ALL span birth through adolescence and into young adulthood. They were designed to overlap with the NCCN Guidelines for Adult ALL, and harmonize treatment approaches for patients in the overlapping age rage. The recommendations are categorized by risk level, which can also be age-related; with the highest risk associated with those diagnosed within the first 12 months of life or between the ages 10 and 21 years old.
"One unique and useful component of these guidelines is the identification of vulnerable populations, and assembling the best supportive care advice for them," explained Dr. Brown. "Patients with Down syndrome and very young infants, for example, face particular challenges that we address."
NCCN plans to continue growing the library of NCCN Guidelines for pediatric cancers, until they address at least 90% of all incident childhood cancers. Up next will be NCCN Guidelines for Pediatric Burkitt Lymphoma, which will be published before the end of 2019. NCCN has also begun assembling panels for Pediatric Wilms Tumor and Pediatric Hodgkin Lymphoma.
There are also plans to adapt the NCCN Guidelines for Pediatric ALL into NCCN Harmonized Guidelines™ and NCCN Framework for Resource Stratification of NCCN Guidelines (NCCN Framework™) for low- and middle-income countries to provide direction for effective management even when resources are limited. In these settings, having established care standards can make the difference between life and death.
"We know that many, many children can be cured with inexpensive and widely-available therapies," said Dr. Brown. "With the increasing global reach of the NCCN Guidelines, we can really pave the way for increasing the cure rates throughout the world."
"In places where resources are limited, oncologists need to know they are using the available options optimally. Our plans for resource-stratified versions of the NCCN Guidelines for Pediatric ALL will provide the information they need to make the right call," said Dr. Carlson. "Experts from among the best children's hospitals in the world are represented on our panels. Families can be reassured that children who are treated according to the NCCN Guidelines are getting the very best care available."
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New NCCN guidelines released for treating children with acute lymphoblastic leukemia. Appl Rad Oncol.