# APL0406: retinoic acid plus arsenic trioxide without chemotherapy for low- and intermediate-risk acute promyelocytic leukaemia

Source: https://onco.cc/key-papers/paper-apl0406-lo-coco-nejm-2013/  
OnCo record `paper-apl0406-lo-coco-nejm-2013` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Combining all-trans retinoic acid with arsenic trioxide cured almost every patient with lower-risk acute promyelocytic leukaemia without any conventional chemotherapy, and did so with fewer complications than the chemotherapy-based standard.

## Summary

Phase 3 non-inferiority trial of 162 patients with non-high-risk APL randomised to all-trans retinoic acid plus arsenic trioxide or to retinoic acid plus idarubicin chemotherapy (AIDA).

Two-year event-free survival was 97 percent with the chemotherapy-free regimen against 86 percent with AIDA, and overall survival 99 versus 91 percent; the arsenic arm had less haematological toxicity and fewer infections but more liver enzyme rises and QT prolongation.

## Fields

- Kind: Key paper
- Last checked: 2026-09-17
- Journal: New England Journal of Medicine
- Year: 2013
- DOI: 10.1056/NEJMoa1300874
- Authors: Lo-Coco F, Avvisati G, Vignetti M, et al.
- Findings: Two-year event-free survival 97 percent vs 86 percent.; Two-year overall survival 99 percent vs 91 percent.
- What it means: Low- and intermediate-risk APL is now treated without cytotoxic chemotherapy. The regimen has become the standard worldwide and made APL the most curable adult acute leukaemia.
- Caveats: High-risk patients (white count over 10,000) were excluded and still receive added chemotherapy or gemtuzumab.; Differentiation syndrome remains a risk with either regimen.

## Sources

- N Engl J Med 2013: https://doi.org/10.1056/NEJMoa1300874
- PubMed: https://pubmed.ncbi.nlm.nih.gov/23841729/

## Connected records

- cancers: [Acute promyelocytic leukaemia](https://onco.cc/cancers/apl/)
- drugs: [Arsenic trioxide](https://onco.cc/drugs/arsenic-trioxide/)
- trials: [APL0406](https://onco.cc/trials/apl0406/)
- journals: [New England Journal of Medicine](https://onco.cc/journals/nejm/)

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