PEDIATRICS

Increasing Awareness Broadens Access for Childhood Cancer and Sickle Cell Patients

By Ramamoorthy Nagasubramanian, MD

Treatment of childhood cancer is one of the great success stories of modern medicine. Often touted as an example of medical advances made possible through research, a pediatric cancer diagnosis that once meant a fatal prognosis is now survivable for 80 percent of patients. Despite these advancements, cancer remains the leading cause of death by disease among children. What’s more, all pediatric cancers combined receive only 4 percent of U.S. federal cancer research funding. This past September was Childhood Cancer Awareness Month and Sickle Cell Awareness Month, reminding us to take stock of our current situation and to raise awareness of these conditions.

IMPROVING ACCESS TO ADVANCE HEALTH Having access to care for any medical need is important. But for patients who need the pediatric-focused, specialized treatment that childhood cancer requires, it can take on even deeper significance. When one considers that society’s most underserved children — from racial and ethnic minority groups — remain more likely to die of childhood cancer than their non-minority peers due to lack of access to care, this is unacceptable. At Nemours Children’s Health, our Hematology-Oncology team has found that by working together we can better impact two common goals: To find a cure and to reduce the side effects of childhood cancers and sickle cell disease to help all children not just live, but live longer and with a better quality of life — today and for many tomorrows. HELPING TO HEAL THOUSANDS EACH YEAR Helping all children live longer and with a better quality of life requires that we deliver equitable care. Our program treats 6,000 pediatric cancer patients in Florida and Delaware each year, and many enroll in the latest clinical trials sponsored by the National Cancer Institute to help children with conditions like: • solid tumors

• sarcomas • neuroblastomas • blood disease and blood cancers The volume of patients, coupled with decades of experience, means that more kids are receiving life-saving care and having better outcomes. Today, our program boasts the nation’s second largest pediatric clinical trial program of its kind and is one of only two pediatric programs nationwide to be recognized as a National Cancer Institute Community Oncology Research Program (NCORP). REDUCING DISPARITIES IN CLINICAL TRIAL ACCESS AND RESEARCH PARTICIPATION To create a more equitable care environment for patients, we have worked intentionally to identify and minimize all barriers to research and clinical trial participation. To measure the impact of these efforts, our program tracks and reports weekly on three essential data elements: 1. Total clinical trial enrollments 2. Clinical trial enrollments across racial and ethnic groups and compared to population estimates 3. Screen failures (eligible but not enrolled) across racial and ethnic groups, as well as primary language spoken Our objective is to enroll all eligible patients on available clinical trials, inclusive of Therapeutic and Non-Therapeutic trials (e.g., symptom management and cancer prevention trials). We measure our program against national benchmarks published each year in the Children’s Oncology Group Report Card. What we’ve learned along the way is that by understanding the factors that contribute to underrepresentation in research and clinical trials, clinicians can create targeted strategies to increase engagement. For example, by translating our research consent forms into different languages common in our catchment areas, we have been able to eliminate patient screening failures due to language barriers. This is especially relevant considering that half of our clinical trial enrollments identify as Hispanic or non-white, a significant proportion of whom required consent in a language other than English. SPECIAL FOCUS ON SICKLE CELL TREATMENT Another area related to the delivery of equitable pediatric care pertains to sickle cell disease, a genetic condition affecting the red blood cells that disproportionately impacts Black children and families. Roughly 3,000 children are born with sickle cell disease in the U.S. each year. Since sickle trait usually doesn’t cause illness, many parents are unaware that they have sickle cell trait. If two parents both have sickle cell trait, then there is a 25 percent chance that their children can be born with sickle cell disease. Our Hematology-Oncology program is poised to lead the field of sickle cell care and research. For our work, we’ve been awarded a $10.6-million, 5-year grant from the Center of Biomedical Research Excellence (COBRE).