Oral Arsenic Trioxide Regimen Effective for Treating APL, Study Results Suggests


The 66th American Society for Hematology (ASH) Annual Meeting and Exhibition took place from December 7-10, 2023. During the meeting, researchers and other stakeholders shared insights into clinical trends, practices, and studies in the hematologic sphere. One study (Abstract 157) focused on the use of an oral arsenic trioxide regimen in treating people living with acute promyelocytic leukemia (APL). Normally, the treatment for APL includes ascorbic acid, all-trans retinoic acid, and intravenous (IV) arsenic trioxide. This study wanted to determine whether oral arsenic trioxide could improve outcomes. 

Study Findings 

According to reporting by Healio, the study evaluated how safe and effective oral arsenic trioxide is in five children (ages three to fifteen) and 116 adults with newly-diagnosed acute promyelocytic leukemia. Participants were divided into two groups: standard or high-risk. Those ages 65+ in both groups received oral arsenic trioxide as an induction therapy, with high-risk individuals also receiving daunorubicin. Altogether, the research analysis included data from 117 participants with APL over a 5.5-year period. 

Findings from the study show that: 

  • 56 participants received at least 2 years of oral AAA maintenance therapy during the median follow-up point of 29 months (roughly 2 years and 5 months). Out of these participants, only one experienced APL relapse. 
  • The overall survival rate was 99.1% after 3 years; the relapse-free survival rate was 97.9% for the same time period.
  • Researchers found oral AAA therapy to be safe and well-tolerated. However, some side effects did occur, such as headaches and an elevated level of liver enzymes called transaminases. 
  • 67 participants experienced APL differentiation syndrome and all were successfully treated with intravenous dexamethasone. 

Ultimately, the researchers found that oral arsenic trioxide is a promising and effective replacement for IV arsenic trioxide and may offer a lower cost and longer survival rates. 

About Acute Promyelocytic Leukemia (APL)

Acute promyelocytic leukemia is a rare and aggressive blood cancer. This subtype of acute myeloid leukemia (AML) is characterized by immature promyelocyte (a type of white blood cell) accumulation in the blood and bone marrow. These crowd out platelets and healthy blood cells. APL occurs due to a translocation between the PML gene on chromosome 15 and the RARA gene on chromosome 17. APL is most often seen in middle-aged adults, though it can be found in children. 

Symptoms relating to APL may include: 

  • Abnormal bleeding, such as bleeding from the gums or nosebleeds
  • Anemia (low red blood cell count) and related symptoms such as pale skin and fatigue 
  • Swollen gums 
  • Unintentional weight loss
  • Appetite loss
  • An enlarged spleen and/or liver
  • Fever
  • Pain in the affected area
  • Blood clots
  • Petechiae under the skin 
  • Night sweats

If you have APL and are looking for support, consider checking out one of the support groups offered by the Leukemia & Lymphoma Society.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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