A multidisciplinary treatment strategy that includes high-dose chemotherapy for metastatic retinoblastoma without CNS involvement

Abstract

Summary:The prognosis of patients with metastatic retinoblastoma is poor with conventional chemotherapy and radiation. Since retinoblastoma is highly chemosensitive, doseescalation of chemotherapeutic agents with stem cell support should be promising. We report our experience with high-dose chemotherapy (HDC) and autologous stem cell transplantation (SCT) in patients with metastatic retinoblastoma. Five patients with metastatic retinoblastoma underwent HDC with autologous SCT following conventional chemotherapy and local radiation therapy. Stem cells (bone marrow in four and peripheral blood stem cells in one) were collected after marrow involvement was cleared. Melphalan was a key drug in all patients, and was administered in combination with other agents such as cisplatin, cyclophosphamide, carboplatin or thiotepa. Three patients are currently alive disease-free at 113, 107 and 38 months, respectively, from the time of SCT. They had no central nervous system (CNS) involvement. The two patients who died of disease had CNS involvement. No long-term sequelae of HDC have been noted. Our treatment strategy using HDC appears to be effective for treating metastatic retinoblastoma without CNS involvement.

Tandem high-dose chemotherapy and autologous stem cell rescue in children with bilateral advanced retinoblastoma

“…Although there have been several studies that employed HDCT/ASCR for the treatment of metastatic or relapsed retinoblastoma, [13][14][15][16] this is the first study of HDCT/ASCR in patients with newly diagnosed bilateral advanced retinoblastoma without extraocular metastasis. Vitreous seeds and subretinal fluid at diagnosis are known to be associated with a high chemoreduction failure rate.…”

An extremely rare case of metastatic retinoblastoma of parotids presenting as a massive swelling in a child

“…Chemoreduction before RT not only cause reduction in metastatic tumor bulk, controls micrometastasis and but also reduces the possibility of RT induced complications like growth deformities of skull and orbits, second malignancies like osteosarcoma, malignant melanoma, leukemia and cataract (1). The use of autologous stem cell rescue combined with high dose chemotherapeutic agents like carboplatin, etoposide, cyclophosphamide, melphalan and thiotepa with conformal RT has proved to improve survival up to 50% to 75% in patients with bulky metastatic disease without central nervous system involvement (15,16).…”

Treatment of Invasive Retinoblastoma in a Murine Model Using an Oncolytic Picornavirus

“…Retinoblastoma can spread peripherally by invading the ocular coats or metastasize to the brain by invading the optic nerve (4,5). No successful therapy is available for patients with central nervous system (CNS) metastasis, whereas only intensive chemotherapy followed by rescue bone marrow transplantation has been reported to successfully treat some patients with peripheral metastasis (6)(7)(8)(9). Therefore, finding a successful, low-toxicity therapy to prevent and treat metastatic retinoblastoma is important for children with this complication.…”