Real-world effectiveness of combining arsenic trioxide with all-trans retinoic acid in patients with acute promyelocytic leukemia.

This study described the real-world effectiveness and safety of arsenic trioxide (ATO; Trisenox) combined with all-trans retinoic acid (ATRA; Vesanoid), without chemotherapy, in patients with newly diagnosed acute promyelocytic leukemia (APL). The authors concluded that a combination of ATO and ATRA showed good results for the treatment of APL, even without chemotherapy.

APL is a type of fast-growing, acute myeloid leukemia (AML) in which many immature blood-forming cells called promyelocytes are produced. It is caused by a genetic abnormality (mutation). Targeted therapy using ATRA and anthracycline-based chemotherapy has been used for these patients. Although highly effective, many patients develop important side effects such as second cancers. Therefore, less intensive options with similar effectiveness are needed.

ATO has been used alone for treating patients that have relapsed after treatment with ATRA and chemotherapy. Recently, good results have been shown in clinical trials for ATO and ATRA treatment combination. However, the safety and effectiveness of ATO and ATRA in a real-world setting are still unknown.

This study involved 23 newly diagnosed patients with APL. All patients received ATO and ATRA induction (first-line) and consolidation (treatment meant to kill any cancer cells that might be left after initial therapy) treatment. The average follow-up for patients was 48 months.

Molecular remission was achieved for all patients after an average time of 3 months. This means that no leukemia cells were found in the bone marrow even with very sensitive tests. Remission was maintained on an average of 44 months, with the exception of 1 patient who relapsed after 11 months.

11 patients needed short-term interruptions of treatment due to side effects. Side effects included fever, liver damage, infections, and headache. Most side effects were managed in the outpatient ward. 

This study indicated that ATO and ATRA treatment for APL, without chemotherapy, provided an effective and well-tolerated option for patients in a real-world setting.

This study was based on medical records. Information might have been incomplete. Also, there was no comparison group with ATRA and chemotherapy. Further studies are needed.