# APL0406

Source: https://onco.cc/trials/apl0406/  
OnCo record `apl0406` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

APL0406 showed that acute promyelocytic leukaemia can be cured without conventional chemotherapy: retinoic acid plus arsenic trioxide did at least as well as, and in fact better than, the standard chemotherapy-based treatment.

## Summary

The Italian-German APL0406 trial randomised adults with newly diagnosed acute promyelocytic leukaemia and a white cell count of 10 x 10^9/L or less to all-trans retinoic acid plus arsenic trioxide for induction and consolidation, or to all-trans retinoic acid plus idarubicin induction followed by anthracycline-based consolidation and maintenance. The primary endpoint was event-free survival at two years, tested for non-inferiority.

The chemotherapy-free arm was not only non-inferior but superior for event-free and overall survival, with fewer infections and deaths in remission, and the advantage held in the extended analysis with more patients. Retinoic acid plus arsenic became the worldwide standard for non-high-risk disease, the first cure of a leukaemia without cytotoxic chemotherapy.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-17
- Also known as: GIMEMA-AMLSG-SAL APL0406
- Tags: subtype-trials
- Registry id: NCT00482833
- Phase: 3
- Setting: Newly diagnosed low- or intermediate-risk acute promyelocytic leukaemia: all-trans retinoic acid plus arsenic trioxide against all-trans retinoic acid plus idarubicin chemotherapy
- Sponsor: GIMEMA (with AMLSG and SAL)
- Enrolled: 276
- Result: All-trans retinoic acid plus arsenic trioxide was superior to retinoic acid plus chemotherapy for event-free and overall survival in non-high-risk APL.

## Sources

- ClinicalTrials.gov NCT00482833: https://clinicaltrials.gov/study/NCT00482833

## Connected records

- cancers: [Acute myeloid leukaemia](https://onco.cc/cancers/aml/), [Acute promyelocytic leukaemia](https://onco.cc/cancers/apl/)
- drugs: [Arsenic trioxide](https://onco.cc/drugs/arsenic-trioxide/), [Tretinoin (all-trans retinoic acid, ATRA)](https://onco.cc/drugs/tretinoin-atra/)
- people: [Francesco Lo-Coco](https://onco.cc/people/francesco-lo-coco/)
- key papers: [APL0406: retinoic acid plus arsenic trioxide without chemotherapy for low- and intermediate-risk acute promyelocytic leukaemia](https://onco.cc/key-papers/paper-apl0406-lo-coco-nejm-2013/)

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