“A Ticking Time Bomb”: voice of the patient Report Emphasizes Burden of Lifelong Cardiac Late Effects in Pediatric Cancer Survivors
June 15, 2023: This just-released Voice of the Patient report stems from an Externally-led Patient-Focused Drug Development (EL-PFDD) meeting on reducing cardiac late effects in pediatric cancer survivors, held in September 2022. This virtual meeting was an opportunity for childhood cancer survivors and caregivers to speak to the Food and Drug Administration (FDA) and other experts about the need to develop new drugs that protect the heart and reduce cardiac late effects caused by harsh chemotherapy and radiation treatments.
View the report here as a PDF, or here as an e-book in your browser.
Appendix 1: Meeting Agenda & Discussion Questions
Appendix 2: Survivor and Caregiver Panel Participants
Appendix 3: Panelist Statements & Submitted Written Comments
Appendix 4: Meeting Polling Questions & Responses
Appendix 5: Meeting Recording
“We are so grateful to the many courageous survivors and caregivers who came forward to describe how these late effects impact their quality of life and provide their insights about why new treatments are needed,” said Steve Wosahla, Chief Executive Officer of Children’s Cancer Cause. “Many of the drugs used today were developed decades ago when we didn’t understand the harm they cause. We hope that this Voice of the Patient report will impact the way we think about this population's needs, encourage future research, and advance successful new treatments as survivors transition into adulthood and face a lifetime of significant health challenges.”
Among the report’s key messages:
Managing ongoing cardiac late effects can have devastating quality of life impacts and too often becomes central to a survivor’s daily life.
Survivors and caregivers feel that they lack information to prepare for the future. They express disappointment, frustration, and feelings of trauma. For parents, trauma is compounded by guilt and anxiety over the difficult decisions they’ve had to make regarding their child’s care.
Survivors rely on a wide range of medical interventions, from heart medications to control blood pressure and manage cholesterol to more significant interventions like pacemaker placement, valve replacement, and even heart transplant.
For survivors and caregivers, the most important potential new treatments are protective treatments to prevent heart effects from cancer treatments, as well as less toxic cancer treatments with reduced impact on heart health.
There needs to be even greater incorporation of the survivor and caregiver voice in drug development to prevent, mitigate, or improve treatment for these late effects.
The report includes survivor stories about challenges with everyday activities due to shortness of breath, racing heartbeat, fatigue, depression, and anxiety, in addition to testimony from parents whose children survived their cancer but later died from cardiac complications caused by the harsh, toxic treatments.
“Aging, as a survivor of childhood cancer, is terrifying. The treatments that saved my life now seems to be slowly stealing it,” said a participating survivor who was treated for Hodgkin’s lymphoma as a child and has spent subsequent decades suffering from multiple cardiac late effects. “On difficult days, I feel as if survivorship is a progressive terminal illness. The effects of childhood cancer last a lifetime.”
