Retinoic Acid for High-risk Neuroblastoma Patients after Autologous Stem Cell Transplantation – Cochrane Review ^[*]

Abstract

Background: Neuroblastoma is a rare malignant disease and patients with high-risk neuroblastoma have a poor prognosis. Retinoic acid has been shown to inhibit growth of human neuroblastoma cells and has been considered as a potential candidate for improving the outcome.

Methods: The objective was to evaluate effects of retinoic acid after consolidation with high-dose chemotherapy and bone marrow transplantation as compared to placebo or no further treatment in patients with high-risk neuroblastoma. We searched the databases CENTRAL, MEDLINE, and EMBASE from inception to 01 October 2014 and included randomized controlled trials.

Results: We identified one relevant randomized controlled trial with 50 participants receiving retinoic acid and 48 participants receiving no further therapy. There was no statistically significant difference between the treatment groups in overall survival (hazard ratio 0.87, 95% confidence interval 0.46–1.63, P=0.66) and event-free survival (hazard ratio 0.86, 95% confidence interval 0.50–1.49, P=0.59). We did not identify results for other outcomes, including toxicity.

Conclusion: The difference in overall and event-free survival between treatment alternatives was not statistically significantly different. Based on the currently available evidence, we are uncertain about the effects of retinoic acid after bone marrow transplantation in patients with high-risk neuroblastoma.

Zusammenfassung

Hintergrund: Neuroblastoma ist eine seltene maligne Krankheit, und Patienten mit einem Hochrisikotumor haben eine ungünstige Prognose. Retinsäure hemmt das Wachstum von menschlichen Neuroblastomzellen und könnte den Krankheitsverlauf günstig beeinflussen.

Methoden: Es sollte bei Patienten mit Hochrisikotumor nach der Konsolidierung mit Hochdosischemotherapie und Knochenmarktransplantation untersucht werden, ob sich die anschließende Therapie mit Retinsäure im Vergleich zu Plazebo oder keiner weiteren Therapie auf den Behandlungserfolg auswirkt. Wir suchten randomisierte Studien in den Datenbanken CENTRAL, MEDLINE und EMBASE, die dort jeweils bis zum 01. Oktober 2014 aufgenommen waren.

Ergebnisse: Wir identifizierten eine relevante randomisierte Studie: 50 Teilnehmer erhielten Retinsäure und 48 Teilnehmer erhielten statistisch signifikant keine weitere Therapie. Es zeigte sich kein statistisch signifikanter Unterschied zwischen beiden Behandlungsgruppen hinsichtlich Gesamtüberleben (Hazard-Ratio 0.87, 95% Konfidenzintervall 0.46–1.63, P=0.66) und ereignisfreiem Überleben (Hazard ratio 0.86, 95% Konfidenzinterval 0.50–1.49, P=0.59). Ergebnisse anderer Endpunkte, auch zur Toxizität, wurden nicht berichtet.

Schlussfolgerung: Der Unterschied des Gesamtüberlebens und des ereignisfreien Überlebens zwischen den Behandlungsalternativen war jeweils nicht statistisch signifikant. Ausgehend von den momentan verfügbaren Studiendaten besteht eine Unsicherheit über die Wirkungen der Retinsäure nach Knochenmarktransplantation in Patienten mit Hochrisiko-Neuroblastom.

Key words

neuroblastoma - tretinoin - bone marrow transplantation - systematic review

Schlüsselwörter

Neuroblastom - Tretinoin - Knochenmarktransplantation - Systematische Übersichtsarbeit

^* This article is based on a Cochrane Systematic Review published in the Cochrane Database of Systematic Reviews (CDSR) 2015, Issue 1. Art. No.: CD010685. DOI: 10.1002/14651858.CD010685.pub2. (see www.thecochranelibrary.com for information). Cochrane Systematic Reviews are regularly updated as new evidence emerges and in response to feedback, and the CDSR should be consulted for the most recent version of the review.

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