Because sarcomas are so rare, symptoms are often overlooked. It’s always wise to investigate anything unusual, and to seek out experts
When Ted Kennedy Jr., son of the late senator Edward M. Kennedy, was 12 years old, he felt some pain and swelling in his right leg. In short order, he was diagnosed with a rare and aggressive form of sarcoma below the knee. Doctors needed to amputate his leg above the knee to ensure all the cancer was removed. They followed up with powerful chemotherapy drugs — still novel at the time — to eradicate any remaining cancer cells.
That was 1973. Options were limited back then, and as drastic as young Kennedy’s treatment was, it did save his life. But it did not spare him the challenges of living with a lost limb.
Cancers known as sarcomas, which can occur in bone or in soft tissue like muscle, nerves or fat, are exceedingly rare, with only about 15,000 new cases each year in the U.S., mostly in adults. However, in children, sarcoma is among the more common cancers, making up about 6% of total cancer cases.
Kennedy’s case occurred 50 years ago. While surgery remains a key tool for treating sarcoma in children, amputation is rarely needed these days. Tools and techniques have improved to the point where limb-sparing surgery has become the standard of care. In fact, it is now possible to do even more: Joints can be preserved or replaced, grafts can fill gaps left by surgery, bones can be lengthened and special prosthetic devices can expand as the child grows.
No one knows for sure what causes sarcomas in children, but there are theories.
“There are some very rare genetic abnormalities that predispose some children to developing sarcoma,” said Lee Zuckerman, M.D., associate clinical professor in the Division of Orthopaedic Surgery. Children with Maffucci syndrome or inherited Li-Fraumeni syndrome are at an increased risk for developing certain types of cancer, including sarcoma. In addition to this, children who receive radiation treatment for other types of cancer at a younger age may develop sarcoma later in life. Patients with Li-Fraumeni syndrome are especially sensitive to the cancer-causing effects of radiation.
Some forms of sarcoma may have a genetic component. For example, researchers have detected certain genetic mutations, or “translocations,” in the bone cancer known as Ewing’s sarcoma, and in a soft tissue variety that arises in muscle tissue called rhabdomyosarcoma.
For this reason, researchers are testing several targeted therapy drugs that would address those genetic anomalies. As yet there have been no “breakthrough” discoveries, which helps explain why overall survival rates haven’t changed much in 40 years since chemotherapy became standard.
Signs to Look for
Healthy, active kids typically experience all sorts of scrapes, tumbles, falls and injuries. Obviously, not every ache or pain is a sign of sarcoma. But both bone and soft tissue cancers often begin with innocent-looking symptoms. Ewing’s sarcoma, for example, may first appear as stiffness, pain, swelling or tenderness in or near the bone. A painless lump under the skin may be the initial sign of soft tissue sarcoma.
“Any mass or growth should get checked out, particularly if it’s growing,” said Zuckerman. “Same thing with ‘growing pains,’ where kids sometimes get pain around the knees. If the pain wakes the child up at night, is only on one side or requires pain medication, it should get checked out. Although sarcoma is rare, it’s better to have the area evaluated if the symptoms are lasting longer or worse than expected.”
Because sarcomas are so rare, parents, family physicians and even oncologists with limited sarcoma experience may attribute a child’s symptoms to something else. It’s always wise to investigate anything unusual, and to seek out experts.
If a biopsy is required, “it’s important to go to a sarcoma specialist,” Zuckerman said, “because someone without that training could cause problems,” even spread cancer cells beyond the tumor. “The most common reason we aren’t able to save someone’s arm or leg is due to a biopsy that isn’t performed properly.”
Most sarcomas in children are generally handled with a combination of surgery and chemotherapy, and is some cases radiation. Many times, chemotherapy and/or radiation are given before surgery to shrink the tumor, and afterward to ensure all cancer cells are eradicated. Treating children — who are still growing and developing — presents special challenges, and extra care needs to be taken.
Unfortunately, many of the treatments for sarcoma can cause other problems. “Radiation can cause scarring, stiffness, swelling, pain and cause damage to the bones and tissues,” Zuckerman said. “Chemotherapy can cause heart issues and infertility. The cancer and surgical treatment can cause issues with growth. Fortunately, we have become better at preventing and treating many of the issues that treating the cancer can cause, and we have programs, including the Childhood, Adolescent and Young Adult Survivorship Program,” which address long-term effects of cancer treatment for children.
Perhaps the best progress can be found in the operating room, where new surgical devices are making a profound difference. That’s certainly been the case for 18-year-old Adrian Lucatero. At age 9 he developed osteosarcoma: a tumor was lodged in his left femur, a situation that made him feel “overwhelmed. I was just a little kid. I never thought I could have cancer,” he recalled.
In his first surgery, Adrian’s left knee and most of his femur were removed and he had his knee and most of his femur replaced. He was fitted with a “child” prosthesis incorporating a spring loaded insert that could lengthen the replacement using an external electromagnet. The electromagnet activated the insert in the replacement and enabled the prosthesis to “grow” as Adrian grew, without needing any incisions.
When it came time for an adult prosthesis, Adrian had only a very small portion of his femur left and was at risk for needing his whole femur and hip replaced. In addition to this, the child prosthesis didn’t have enough length to make his legs the same length; his left leg was shorter than his right. Due to this, Zuckerman turned to a different device, which uses a motor driven by a magnet, designed to lengthen the small remaining segment of Adrian’s femur, near his hip. When the adult prosthesis was placed, a cut was made in the small portion of the bone that remained, and Zuckerman implanted the new device around it. The first step was to get Adrian's body to reform a portion of his femur, so that he wouldn’t need the entire bone replaced.
Three times a day, Adrian used an external magnet to activate a motor, gently separating the bone fragments, about a third of a millimeter at a time. This tricked his body into thinking that he had a broken bone that needed to heal. His body repeatedly formed new bone at the cut as the bone slowly moved, similar to how the body heals any broken bone. After four months, nearly 4 inches of new bone had formed, and Adrian still had his hip joint.
The final step will be to get his legs back to the same length, using the same process. Instead of just forming extra bone to recreate the femur, the device will be used to lengthen the femur. “I’m looking forward to getting back to normal,” said Adrian, who currently uses crutches to walk, although Zuckerman says he expects him to be able to walk normally once his leg fully heals. “Back to how I used to be.” He was so inspired by his experience, Adrian is now enrolled in college, studying to become an orthopedic nurse.
A Lifetime of Care
Pediatric cancer patients don’t “age out” at City of Hope, “because we are a cancer center, not a children’s hospital,” explained nurse practitioner Karla Wilson of the childhood, adolescent and young adult survivorship program. She recalls children who began treatment at 6 months old and continued their follow-up care through their college years and beyond. “We’re with the patient for life.”
That said, City of Hope’s Department of Pediatrics keeps the special needs of children in mind, with its extensive Child Life Services program offering age-appropriate explanations of procedures, psychological services, help for parents and more.
Pediatrics is a growing department at City of Hope; its pediatric musculoskeletal cancer and sarcoma program is one of the largest in California.
“We are persistent at City of Hope,” declared Mark Agulnik, M.D., a sarcoma specialist currently overseeing some 20 clinical trials. “We care for the patient at all stages.”