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CASE REPORT article

Front. Pediatr.
Sec. Pediatric Oncology
Volume 12 - 2024 | doi: 10.3389/fped.2024.1509645
This article is part of the Research Topic Novel Molecular Targets and Therapies for Pediatric Extracranial Solid Tumors View all 5 articles

Successful treatment of metastatic retinoblastoma with CNS involvement with anti-GD2 immunotherapy, intrathecal topotecan and reduced systemic chemotherapy

Provisionally accepted
Cristina Larrosa Cristina Larrosa 1Margarida Simao Margarida Simao 1Noelia Salvador Noelia Salvador 2Juan Pablo Muñoz Juan Pablo Muñoz 3Guillermo Chantada Guillermo Chantada 1Jaume Mora Jaume Mora 4*
  • 1 Oncology Department, SJD Pediatric Cancer Center Barcelona, Hospital Sant Joan de Deu, Barcelona, Spain
  • 2 Molecular Diagnostics Laboratory, SJD Pediatric Cancer Center Barcelona, Hospital Sant Joan de Deu, barcelona, Spain
  • 3 Oncology Department, SJD Pediatric Cancer Center Barcelona, Hospital Sant Joan de Deu, Barcelona, Spain, Barcelona, Balearic Islands, Spain
  • 4 Scientific Director, Laboratori de Tumors del Desenvolupament, Institut de Recerca Sant Joan de Deu, Barcelona, Spain

The final, formatted version of the article will be published soon.

    High-dose chemotherapy with autologous stem cell rescue has improved outcomes in patients with metastatic retinoblastoma (RB). Despite significant advances, acute and long-term side-effects, particularly in visually impaired and cancer-predisposed patients, underscore the need for additional treatment options. Monoclonal antibodies (mAbs) directed against the tumor-associated antigen GD2 are of considerable interest. Additional lines of RB research include tracking minimal disseminated disease (MDD) to permit timely intervention in patients with CNS metastasis. We present two cases of bilateral, metastatic RB, managed with the anti-GD2 mAb naxitamab following reduced intensity myeloablative chemotherapy and autologous stem cell transplant (ASCT) with intrathecal topotecan for MDD detected in the CSF. The patients remain disease-free 10 and 9 years after initial diagnosis. While additional studies are needed, the results suggest anti-GD2 mAbs and CNS-directed chemotherapy may improve long-term outcomes and reduce cytotoxicity for high-risk patients with RB.

    Keywords: retinoblastoma1, immunotherapy2, naxitamab3, GD24, topotecan5

    Received: 11 Oct 2024; Accepted: 18 Dec 2024.

    Copyright: © 2024 Larrosa, Simao, Salvador, Muñoz, Chantada and Mora. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

    * Correspondence: Jaume Mora, Scientific Director, Laboratori de Tumors del Desenvolupament, Institut de Recerca Sant Joan de Deu, Barcelona, Spain

    Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.