Malignant retinal tumors in children, called retinoblastomas, which have spread to the bones, bone marrow and soft tissue can be successfully treated by a combination of high-dose chemotherapy, radiation therapy and transplantation of blood-producing stem cells.
In the study, conducted at St. Jude Children's Hospital in Memphis, Tennessee, four pediatric patients were treated with intensive chemotherapy – a combination of several very-high-dose anti-cancer drugs – radiation therapy, and the transplantation of the patients' own blood-producing stem cells. The blood stem cells were harvested after two to four courses of chemotherapy.
In all cases, the bone marrow disease went into complete remission after two courses of chemotherapy, which was followed by radiation and stem-cell therapy. Of the four patients, two survived free of disease longer than six years. In the other two patients, disease recurred in the central nervous system and they did not survive. Disease of the central nervous system continues to be the major obstacle to improved outcomes for patients with metastatic retinoblastomas.
Carlos Rodriguez-Galindo, MD, lead author of the study, said, "Our results show that retinal tumors that metastasize to other areas of the body can be cured. Chemotherapy by itself only results in transient improvement, with all patients dying of disease progression eventually, whereas chemotherapy in combination with radiation and stem-cell therapies can actually save patients' lives. We believe more than 90 percent of patients can be cured with surgery if the disease is detected early enough, before the disease spreads."
Dr. Rodriguez-Galindo and colleagues are working with Ira J. Dunkel, MD, at Memorial Sloan Kettering Cancer Center in New York and with Eric F. Grabowski, MD, ScD, at Massachusetts General Hospital for Children to develop a national protocol that will apply these principles of therapy to all children with this disease.
Based on data from the National Cancer Institute's Surveillance, Epidemiology, and End Results (SEER) program (http://seer.cancer.gov), Dr. Rodriguez-Galindo estimates the number of annual retinoblastoma cases in the United States to be between 300 and 350. Of these, approximately 15, or five percent, develop retinal tumors that spread outside of the eye. "However," he said, "in developing countries, close to 40 to 50 percent of retinoblastoma patients develop metastatic disease."
Academy spokesperson William Mieler, MD, said, "This study, along with comparable studies conducted by Drs. Dunkel and Grabowski, provides encouraging evidence that a successful approach to treating this advanced, devastating disease is close at hand. Once the protocol is further established and tested on additional patients, we can hope to see further documentation of effective treatment for patients with advanced metastatic retinoblastoma."
The American Academy of Ophthalmology is the world's largest association of eye physicians and surgeons--Eye M.D.s--with more than 27,000 members worldwide. For more information about eye health care, visit the Academy's partner Web site, the Medem Network, at http://www.medem.com/eyemd. To find an Eye M.D. in your area, visit the Academy's Web site at http://www.aao.org.
Journal
Ophthalmology