Lack of complete response pretransplant is not associated with inferior overall survival for stage 4a metastatic retinoblastoma

Abstract

Background Stage 4a metastatic retinoblastoma (RB) is curable with intensive multimodality therapy including myeloablative chemotherapy with autologous stem cell transplant (HDC‐ASCT) and involved field radiation therapy (IFRT). To our knowledge, no data exist on the impact of (a) pre‐ASCT disease status, and (b) IFRT to sites of metastatic disease post ASCT on survival. Procedure We retrospectively reviewed patients with stage 4a metastatic RB who underwent induction chemotherapy followed by HDC‐ASCT, with or without IFRT, to residual tumor sites at Memorial Sloan Kettering Cancer Center (MSKCC) (n = 24). Results The degree of postinduction response prior to ASCT did not affect outcome, with 5‐year overall survival (OS) of 68% and 86% in patients who achieved complete response (CR) and very good partial response (VGPR)/partial response (PR) prior to ASCT, respectively. IFRT administered post ASCT in patients with possible residual bony metastatic disease increases the likelihood of developing osteosarcoma in the radiation field. Conclusion OS for patients with stage 4a metastatic RB treated with ASCT with VGPR or PR to pretransplant chemotherapy was not significantly different from patients with CR. In addition, IFRT does not seem to be required for bony disease control and increased the likelihood of developing osteosarcoma.

Autologous hematopoietic stem cell transplantation followed by quadruple immunotherapy with dinutuximab beta, sargramostim, aldesleukin, and spironolactone for relapsed metastatic retinoblastoma

“…Involved-field radiotherapy is not required for bony disease control and would increase the likelihood of developing osteosarcoma. 12 Overall survival for patients with stage 4a metastatic RB treated with ASCT with very good partial remission or partial remission to pretransplant chemotherapy was not significantly different from patients with complete remission. 12 GD2 is a cell surface glycolipid highly expressed on cancer cells of neuroectodermal origin, including neuroblastoma and RB, and is therefore useful for disease monitoring.…”

“…12 Overall survival for patients with stage 4a metastatic RB treated with ASCT with very good partial remission or partial remission to pretransplant chemotherapy was not significantly different from patients with complete remission. 12 GD2 is a cell surface glycolipid highly expressed on cancer cells of neuroectodermal origin, including neuroblastoma and RB, and is therefore useful for disease monitoring. Serum GD2 level was found to be significantly higher in RB-bearing patients than normal individuals.…”