Survivorship Matters Blog By CAC2 Individual Member Mary Beth Collins
“Your child has to be alive to experience side effects”
It is heard by every parent of a child with a high-risk pediatric cancer when reviewing treatment protocol: your child must endure and survive all of the therapies first, before a parent can afford to be concerned about side effects. It’s a pragmatic priority; the focus is on keeping your child alive and achieving No Evidence of Disease, or ”NED” as commonly referenced. With the most challenging cancers, it is spoken with earnest and delicate honesty. Today, according to the most recent information from NCI, with an overall survival rate of pediatric cancers 85% the childhood cancer community is doing a better job today than ever before to keep children alive, even when facing the high-risk cancers like neuroblastoma. Yet, as stated in the CAC2 Fact Library, 95% of those survivors experience at least one significant side effect by the time they are 45 years old. As a parent of a child who needed an extensive treatment regimen to survive, we quickly discovered that for those requiring more significant treatments, our survivors tend to experience a number of side effects. It is heart wrenching after such difficult times during treatment to then juggle side effects from the treatment. When cisplatin is part of the chemotherapeutic regimen, high frequency hearing loss is common. Recent studies suggest 75% of patients five years or younger needing cisplatin incur hearing loss. The impact of this loss on quality of life, for the rest of their lives, is significant.
New Hope with the Development of Pedmark
So often in the childhood cancer community, we talk often about wanting gentler treatment options to spare our children lifelong side effects. With the FDA’s recent approval of Pedmark (an injectable formulation of sodium thiosulfate (“STS”), children are protected from cisplatin’s ototoxicity. This development is substantial for children in treatment because it maintains their hearing, providing a better quality of life. This creates a new option for the childhood cancer community where development of new drugs and therapies is a long, exhaustive process: shield our children when giving harsh treatments so that they are spared side effects while fighting the cancer.
It has been a long road for Fennec Pharma who dedicated significant time and resources to evaluate and ensure that maintaining our children’s hearing did not protect the tumor. In 2011, the FDA held a Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee Meeting to troubleshoot this unique dilemma. The core challenge was being able to test a large enough sample of patients with the appropriate diagnosis, which are rare diseases warranting the platin drug, to measure impact. The realistic component of that challenge was having enough patients, who were sick enough to need the platin drug, survive the length of the study. The testing that continued after that important meeting was modified to address this issue. The trial process ran from 2007 – 2014, involving 52 sites from 11 countries. Results demonstrated that morbidity was consistent among both study groups, therefore proving that Pedmark did not shield the tumor while protecting the patient.
Hearing Loss and its Significant Impact
Any level of hearing loss is significant for younger children, influencing speech and language development, educational achievement, and social-emotional development. The earlier hearing loss occurs in a child’s life, the more serious the effects are on the child’s development. And as often expressed by audiological experts, “there is no such thing as insignificant mild hearing loss in a young child” because even low loss levels have been proven to be associated with academic challenges and behavioral problems. Hearing is tested during treatment to evaluate function when receiving drugs known to result in loss, as it often takes three years for hearing loss to stabilize after treatment is completed. In our case, my son initially encountered moderate-to-severe, high frequency hearing loss after his first dose of cisplatin, so all additional cisplatin doses were replaced with carboplatin to be a bit gentler on his auditory system.
Many are not aware that hearing tends to worsen over time. In puberty and transitioning to adulthood, children often experience an exasperation of loss. In the Externally-Led Patient Focused Drug Development (EL-PFDD) FDA workshop Chemotherapy-Induced Hearing Loss in Pediatrics, a polling of the attendants found 76% of those who had incurred loss experience a worsening impact over time, affecting home life, school, work, friendships, etc. This meeting involving 250 stakeholders was an incredible opportunity to appear before the FDA to allow patients, survivors, and parents to personalize the everyday impact of chemo-induced hearing loss, spotlighting the challenge across the spectrum of quality-of-life factors. Read the full impact report of this meeting here.
My son’s loss continued to progress and at 15 years old, it increased from moderate-to-severe to profound loss. His loss continued to increase every year afterwards, and eventually his audiologist recommended that he stop playing music entirely. The vibrations from the music would be so abrasive on his delicate and feeble hearing system, it threatened losing what little hearing he had left. With music being a cornerstone of his life when he was feeling well, this suggestion to stop a music meant so much more for my son. Music was more than just a meaningful activity that brought my son joy, it provided him an ability to cope (because of a lack of quality psychosocial support), supported his confidence when so many other side effects still prevailed and challenged him, and entertained when with friends and fellow musicians. Once again, as is so often experienced when dealing with side effects in survivorship, our children must make decisions between the worser of evils. In this case there simply wasn’t a good option; either decision would have a life-long negative impact on his life. Josh chose music, willing to sacrifice what remaining hearing he had to live the life he wanted.
The Significance of Loss to Children and Families Impacted by Cancer
Attempting to support your child with variable hearing challenges in survivorship becomes a way of life. For 23 years, my son and I have accommodated our lives with audiology appointments, IEP/504 meetings, and modification of things at home. It has been difficult over the years to watch the hearing issues grow from moderate-to-severe to profound loss. It is working with audiologists and neuropsychological evaluators to learn the extent of impact from the hearing, which often includes additional effects such as auditory processing disorder (affecting how the brain processes auditory input) and tinnitus (a chronic condition of ringing in the ears). The effect of these conditions on a child impacted by high frequency hearing loss was explained to me in simple terms: “Imagine the small amount of hearing your child has as a piece of cheese. With the influence of these conditions, now the ability to hear is more like a piece of Swiss cheese.” In essence, these co-occurring conditions can actually reduce your child’s auditory function even further.
Affording the support is often a challenge, as many insurance companies are extremely limited to providing hearing aids and assistive technology, should they provide any coverage at all.
We were told back in Josh’s first year after treatment that the annual economic burden for special education programs for children deaf or hard of hearing could amount to as high as $11,000 per child. We worked with our audiologist and a hearing therapist, who proved to be invaluable in teaching me what I needed to know to support my son at three years of age to navigate his world with this disability. More importantly, they taught me the critical importance of supporting his brain by filling it with language before the age of five. Our children are like sponges, absorbing words and filing them to serve as a repository for reference as they age. Young children with hearing loss lack a supportive repository, which affects so much in school, in communication with friends/family, and a sense of self. Hearing aids, for even mild loss, is critical and helps contribute to this repository. But acquiring the best quality hearing aids is expensive, often costing at a minimum $6000 a pair. Over the last 25 years, we’ve purchased eight pairs of hearing aids to give my son the highest level of support possible, a source of ongoing financial toxicity we’ve experienced in survivorship.
But even harder than challenges with learning, are the days when my son’s hearing loss causes pain and isolation. At 12 years old, soccer became problematic because teammates were unkind simply because he couldn’t hear them on the field. At 14 years old, a celebration of his younger brother finally being able to sit in the front seat of the car quickly down spiraled because Josh, teary-eyed in the back seat mumbled, “My life sucks. I can’t hear anything you two are saying up front.” I’ve attended countless teacher meetings where I would help them understand how hearing loss exponentially impacts the learning process, and creatively motivate them to partner with me to support my son. Not every teacher was willing to do what was needed to include him in class activities, leaving him isolated, frustrated, and exhausted at school. A parent of a child with hearing loss quickly learns that the removal of the hearing aids at home becomes the barometer of the difficulty of the day: stress, illness, or exhaustion results in the need for a break from the extra work required to use aids to stay connected with the world. Can you imagine how sick or tired a child must be to choose isolation over being connected with other people because he/she can no longer has the energy to do so? As our children age, a parent watches job considerations ranked by the ability to read other people’s lips while completing tasks. Most recently during the pandemic, a world full of masks completely isolated our hard-of-hearing children in public. Josh figured out that the best way to communicate with anyone in a store was to talk to the skinniest person. He was able to understand thin people easier because their masks were looser, and he could draw cues from the increase in nonverbal communication from a more exposed face.
Now at 25 years old, my son has worked hard to carve out a life for himself in music that draws from the hearing he has, instead of suffering from the hearing he has lost. He was not willing to sacrifice his love of music, where he is so gifted and skilled after many years of instruction and practice. Through an incredible music engineering program, spotlighting the lower-mid to upper-mid frequencies, Josh trained his ears within the sonic spectrum. He developed the sophistication to work within frequencies not affected by chemo-induced hearing loss. As so many of our survivors do, he accentuated what he can do, to overcompensate and accommodate what he cannot do because of long-term side effects. This is not an easy process and is a continual one as more challenges present themselves over time. It is what a survivor must do when insisting on a life of quality in survivorship.
A Brighter Future For Children Now With Pedmark
The development of Pedmark presents a brighter future for children diagnosed with cancer. Removing the possibility of hearing loss removes a long-term side effect that brings with it so much challenge, cost, and anxiety, often in children dealing with other significant side effects as well. Not only does Pedmark preserve a higher quality of life by maintaining a child’s hearing, but it also helps our children feel more “normal” when returning to their families, school, or work after treatment. With Pedmark, we are one giant step closer to the cure we all dream for our children while in treatment. Children can return to a life more normal because our children can be healthier and happier for the remainder of their lives. After treatment, Pedmark helps our children thrive in survivorship.