Cryotherapy, Chemotherapy, and Clinical Trials: Treatment Options for Retinoblastoma
- Jyothi Dondero
- Nov 3
- 3 min read
Retinoblastoma is a very rare form of childhood cancer, affecting approximately fifty children in the U.K. every year, with similarly low numbers worldwide. Retinoblastoma attacks the retina of the eye and results in the formation of tumors in one or both eyes. There are two types of retinoblastoma — hereditary and non-hereditary. Approximately 40% of the incidences of childhood retinoblastoma are hereditary, meaning the child inherits the gene for the disease from their parents or develops the specific gene mutation independently in utero.
Children who have a family history of the disease often benefit from early detection through routine genetic screening, regular ophthalmic exams, and proactive treatment. However, for the majority of children suffering from retinoblastoma, the disease is often caught only after it has progressed into the later stages, when vision loss or visible eye abnormalities prompt medical attention.
Treatment Options
For families navigating this diagnosis, understanding the available therapies can make treatment decisions feel less overwhelming. A wide variety of treatment options are available today, depending on the size and number of tumors and the stage of the cancer. Smaller tumors often respond well to localized therapies such as cryotherapy, laser therapy, and thermotherapy. Cryotherapy uses intense cold to freeze and destroy cancer cells, while the latter two therapies use concentrated heat to achieve the same effect.
Small tumors may also respond well to plaque radiotherapy, a method in which a small radioactive plaque is attached directly to the tumor to deliver a precisely targeted radiation dose. Larger tumors typically require a combination of chemotherapy, radiotherapy, and surgery. Thermotherapy is often used alongside chemotherapy and radiotherapy to intensify their destructive effects on the tumor.
The Role of Clinical Trials
Clinical research trials are another treatment option to consider, especially in situations of late-stage cancer or when patients are not responding well to the standard treatments. Clinical trials allow researchers to closely study the mechanics of a disease and to test new treatment methods in a controlled environment. They are an effective treatment option for certain patients but are typically only entered into on a doctor’s recommendation and on a purely voluntary basis.
Clinical trials can focus on a variety of goals, such as evaluating new chemotherapy combinations, exploring targeted drug therapies, or testing precision treatments designed to spare healthy tissue and preserve vision. In some studies, researchers are investigating the use of gene-based therapies or immunotherapy to reduce long-term side effects and improve quality of life after treatment. For eligible families, participation in a clinical trial can provide access to cutting-edge care while also contributing valuable knowledge that helps advance treatment for future patients.
Prognosis and Outlook
Choosing the best treatment plan for a child with retinoblastoma depends on several factors, including tumor size, genetic profile, and disease stage. With the right care, however, the long-term prognosis is typically excellent. In high-income countries, survival rates for retinoblastoma exceed 95%, though outcomes can vary globally depending on access to early screening and advanced care.
Beyond survival, doctors now focus strongly on preserving vision and minimizing long-term side effects. Advances in targeted therapy, localized chemotherapy, and improved surgical techniques have greatly increased the chances of saving an affected eye while reducing complications such as secondary cancers or visual impairment. For many families, ongoing follow-up care, vision therapy, and genetic counseling are key components of recovery. With timely diagnosis, coordinated care, and consistent monitoring, most children go on to lead healthy, active lives after treatment.
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