Successful treatment of metastatic retinoblastoma with high-dose chemotherapy and autologous stem cell rescue in South America

Palma, Julia; Sasso, D; Dufort, Gustavo; Koop, Klaas; Sampor, Claudia; Díez, Bárbara Cancela; Richard, L; Castillo, Luis; Chantada, Guillermo

doi:10.1038/bmt.2011.108

Cited by 44 publications

(27 citation statements)

References 31 publications

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“…Therapeutic options for most patients who progress or relapse are limited; however, some patients with relapsed or metastatic disease can be effectively treated with autologous stem cell transplantation (13,14). At baseline, PET/CT can identify patients at high risk for relapse, and similarly during response assessment PET/CT can identify disease progression before it manifests clinically.…”

Section: Discussionmentioning

confidence: 99%

Role of PET/CT in Staging and Evaluation of Treatment Response After 3 Cycles of Chemotherapy in Locally Advanced Retinoblastoma: A Prospective Study

Radhakrishnan¹,

Kumar²,

Malhotra³

et al. 2012

J Nucl Med

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The present study prospectively evaluated the role of 18 F-FDG PET/CT for staging, neoadjuvant chemotherapy response evaluation, and final outcome assessment in International Retinoblastoma Staging System (IRSS) stage III retinoblastoma. Methods: Twenty-five consecutive IRSS stage III patients, with a median age of 3 y, were prospectively enrolled after ethics approval. All patients received neoadjuvant chemotherapy followed by enucleation, radiotherapy, and adjuvant chemotherapy. PET/CT was performed at baseline (PET/CT-1) and after 3 cycles of neoadjuvant chemotherapy (PET/CT-2). All 25 patients underwent PET/CT-1, and 21 of 25 patients underwent PET/CT-2. PET/CT-1 was compared with routine staging, and response on PET/CT-2 was assessed by criteria from the European Organization for Research and Treatment of Cancer response. Eventfree survival (EFS) and overall survival (OS) were calculated using Kaplan-Meier survival analysis, and differences between the groups were compared using log-rank test. A P value of 0.05 or less was considered significant. Results: Increased 18 F-FDG uptake was noted in primary extraocular tumor in all patients, except 5 with bilateral retinoblastoma (one eye with advanced and the other eye with intraocular disease) in whom the intraocular tumor did not show 18 F-FDG uptake. Five of 22 IRSS stage IIIA patients with clinically negative cervical lymph node involvement were found to have uptake in cervical lymph nodes on PET/CT-1, and 2 of 3 IRSS stage IIIB patients with pathologically confirmed cervical lymph node involvement did not show any uptake in the involved lymph nodes. No significant difference in EFS and OS was seen between IRSS stage IIIA and IIIB patients using routine staging or PET/CT staging (P $ 0.05); however, there was a trend toward better OS in patients with IRSS stage IIIB disease on PET/CT (P 5 0.065). There was no significant discordance between routine staging and PET/CT staging (P 5 0.256). The 8 patients with optic nerve uptake had lower EFS (P 5 0.0001) and OS (P 5 0.0009) than did 17 patients without optic nerve uptake on PET/CT-1. The 17 patients with complete response or partial response had better EFS (P 5 0.042) and OS (P 5 0.026) than did the 4 patients with progressive disease on PET/CT-2. Conclusion: Optic nerve uptake at baseline on PET/CT and response after neoadjuvant chemotherapy according to criteria from the European Organization for Research and Treatment of Cancer are strong predictors of EFS and OS in IRSS stage III retinoblastoma.

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Section: Discussionmentioning

confidence: 99%

Role of PET/CT in Staging and Evaluation of Treatment Response After 3 Cycles of Chemotherapy in Locally Advanced Retinoblastoma: A Prospective Study

Radhakrishnan¹,

Kumar²,

Malhotra³

et al. 2012

J Nucl Med

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“…In a similar study, 11 patients with metastatic disease were treated with high-dose chemotherapy and autologous hematopoietic stem cell rescue therapy, with disease-free survival reported in 7 patients after 39 months. 52 Four patients with CNS involvement died due to recurrence occurring an average of 7 months later.…”

Section: Introductionmentioning

confidence: 99%

Chemotherapy in Retinoblastoma: Current Approaches

Yanık

Günduz

Yavuz

et al. 2015

tjo

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Retinoblastoma (RB) is the most common childhood malignant intraocular tumor. Although enucleation and external beam radiotherapy have been historically used, today the most commonly used eye-sparing approach is chemotherapy. Chemotherapy can be used in both intraocular and extraocular RB cases. Chemotherapeutic agents may be applied in different ways, including systemic, subconjunctival, intra-arterial and intravitreal routes. The main purposes of application of systemic therapy are to reduce the tumor size for local treatment (chemoreduction), or to reduce the risk of metastasis after enucleation surgery (adjuvant therapy). Intra-arterial chemotherapy with the current name “super-selective intra-arterial infusion therapy” could be applied as primary therapy in tumors confined to the retina or as a secondary method in tumor recurrence. The most important advantage of intra-arterial therapy is the prevention of systemic chemotherapy complications. Intravitreal chemotherapy is administered in the presence of persistent or recurrent vitreous seeding. The term “extraocular RB” includes orbital invasion and metastatic disease. Current treatment for orbital invasion is neoadjuvant chemotherapy followed by surgical enucleation and adjuvant chemotherapy and radiotherapy after surgery. In metastatic disease, regional lymph node involvement, distant metastases, and/or central nervous system (CNS) involvement may occur. Among them, CNS involvement has the worst prognosis, remaining at almost 100% mortality. In metastatic disease, high-dose salvage chemotherapy and autologous hematopoietic stem cell rescue therapy are the possible treatment options; radiotherapy could also be added to the protocol according to the side of involvement.

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“…Most recently, South American investigators reported 11 children with stage 4a or 4b disease and noted that 7 were disease-free with a median follow-up of 39 months, indicating that this strategy may also be effective in middle-income countries [ 22 ].…”

Section: Confi Rmatory Case Seriesmentioning

confidence: 98%