Odds for pediatric cancer patients have improved, but not enough

March 23, 2013 | by Rachel Hall

Thirty years ago, children with cancer had only a 30 percent chance of surviving. Now, not only are more patients with pediatric cancers surviving, they're also living longer and healthier lives than in generations past.

The odds for children diagnosed with cancer have improved in recent decades, but much work remains to be done. The odds for children diagnosed with cancer have improved in recent decades, but much work remains to be done.

Research and care at City of Hope have helped make that possible. Here's a look at some of that work:

Ewing’s sarcoma: A new approach to treatment

Ewing’s sarcoma, the second most-common primary bone tumor in children and adolescents, represents 3 percent of all pediatric malignancies. Tumors usually develop in the bones of the arms, legs, pelvis or chest.

In rare cases, some tumors may develop in the skull. As many as 30 percent of children diagnosed with these tumors do not respond to conventional therapies and ultimately die.

Anna Pawlowska, M.D., Department of Pediatrics, seeks to improve survival outcomes for children faced with this devastating disease. She is studying the efficacy of a protocol currently being tested in clinical trials in Europe.

In this approach, European researchers are using whole-body MRI-guided radiation therapy. Patients with Ewing’s sarcoma then receive a combination of high-dose chemotherapy and autologous hematopoietic cell transplant (in which stem cells come from a patient's own body). So far, this approach has had promising results, with patients’ one-year survival rates increasing to 94 percent and two-year survival rates to 70 percent — significantly above national averages.

Pawlowska seeks to conduct a similar study at City of Hope, with the goal of determining the safety and feasibility of this new regimen. In the proposed five-year pilot trial, she aims to enroll 15 patients between the ages of 6 and 40 with relapsed, refractory and/or high-risk Ewing’s sarcoma.

City of Hope would be the first medical center in the United States to test this approach.

Keeping young adult patients connected

Jonathan Espenschied aims to identify the best ways to communicate with adolescents and young adults with cancer about their treatment, compliance and late effects. He is particularly interested in pinpointing the causes of memory, attention and concentration deficits.

To help patients better communicate with their physicians and family, Espenschied has created a touch-screen tool that allows patients to pinpoint the stressors they are experiencing. In his recently completed feasibility study, 52 patients used the touch-screen tool and gave feedback about the ease of using it.

Espenschied plans to analyze the data, publish the results and, with further funding, test the tool in a multi-institutional study and refine it for wider use.

Lifesaving protocols for children with hemophilia

Hemophilia is a condition that prevents a person’s blood from clotting. Children with this disease require a lifetime of care, including frequent infusions of clotting factors derived from donor blood. About 25 percent of children with hemophilia develop antibodies to Von Willebrand Factor (FVIII), the clotting factor essential in preventing and treating hemorrhages.

The Rescue Immune Tolerance Study (RESIST) is an international study that seeks to gain a broader understanding of this potentially deadly complication, and City of Hope researcher Nadia P. Ewing, M.D., director of the Hemophilia and Sickle Cell Program, is the RESIST North America principal investigator.

Children that develop antibodies to FVIII experience hemorrhages that are difficult to control, increased joint and muscle damage, bleeding at surgery, and potentially serious neurologic damage or death from bleeds in the brain. The specific treatment in RESIST involves daily intravenous injections of FVIII alone or FVIII combined with other clotting products in high doses over a period of several months.

Ewing has determined that successfully treated children respond to treatment of bleeding episodes rapidly and consistently each time they receive FVIII, thus decreasing joint damage and successfully managing surgical procedures, free of hemorrhagic complications.

They don't have to miss school because of painful and debilitating bleeds, and most important, have the opportunity to grow up to become productive and successful adults, as all other children.

Childhood cancer survivorship clinic

As we develop more effective treatments, more children are surviving their cancers and growing into adulthood. Smita Bhatia, M.D., M.P.H.,  the Ruth Ziegler Chair in Population Sciences, leads City of Hope’s efforts to monitor the health of hundreds of patients over many years and investigate the potential complications of cancer treatment, as well as the optimal follow-up care and education for cancer survivors.

In one study led by  Bhatia, also director of the Center for Cancer Survivorship, she and a team of researchers are studying the connection between anthracyclines, powerful drugs used in treatment for as many as 50 percent of childhood cancers, and risk for developing cardiomyopathy (weakening of the heart muscle).

After observing a marked difference in how children tolerated the drugs, researchers at City of Hope studied the genetic make-up of nearly 500 cancer survivors from 130 institutions across the country to identify factors contributing to the increased risk of anthracycline-related cardiomyopathy.

Researchers found that variations in two specific genes influence risk of this late effect, and also identified one genetic group, in particular, that causes the greatest risk. The team is now developing tests physicians can use to determine if children have these genes.

Physicians can use this knowledge to either prescribe different drug regimens to children at high risk, or give children additional drugs to protect the heart.

Edited from the Department of Pediatrics Impact Report, February 2013

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