Treatment and Management of Pediatric Retinoblastoma

Retinoblastoma is a rare childhood cancer that requires a multidisciplinary clinical strategy for successful disease treatment and management. Until the 1990s, retinoblastoma treatment consisted of radiation therapy and, often, surgical removal of the eye. 

While these therapies cured patients, an increased likelihood of developing secondary cancers and the incursion of cosmetic deformities prompted investigators at CHOP’s Center for Childhood Cancer Research (CCCR), including Ann-Marie Leahey, MD, to evaluate alternative treatment regimens. 

Clinical studies pioneered at CHOP in partnership with the Wills Eye Institute in the early 1990s demonstrated that systemic chemotherapy using the anti-tumor drugs carboplatin, etoposide and vincristine (CEV) is safe, effective and well tolerated by children with retinoblastoma. In fact, systemic CEV chemotherapy has displaced radiation therapy as the first line of treatment for retinoblastoma and is now accepted as the global standard of care.

Today, much of the clinical research conducted at CHOP is directed toward optimizing CEV chemotherapy and investigating new strategies to better manage side effects and tolerability issues that can occur with CEV treatment.

Clinical investigation of new treatments for retinoblastoma are also underway at CHOP. In partnership with the Children’s Oncology Group, the Hospital was one of 10 institutions selected to evaluate the clinical effectiveness and safety of a new treatment for retinoblastoma called intra-arterial chemotherapy (IAC). 

Other clinical studies are in progress to determine whether or not IAC can be successfully used to treat retinoblastoma in patients when initial CEV chemotherapy has failed.  Ultimately, the goal of all of these clinical studies is to improve existing therapies and to evaluate novel new treatment regimens.

Our Collaborators