Related Experiment Videos
Arsenic trioxide in patients with myelodysplastic syndromes: a phase II multicenter study
Norbert Vey1, Andre Bosly, Agnes Guerci
1Institut Paoli-Calmettes, Marseille, France. veyn@marseille.fnclcc.fr
Purpose:
Evaluation of the safety and efficacy of arsenic trioxide in patients with myelodysplastic syndromes (MDS).
Patients And Methods:
MDS patients diagnosed according to standard French-American-British criteria received a loading dose of 0.3 mg/kg per day of arsenic trioxide for 5 days followed by a maintenance dose of 0.25 mg/kg arsenic trioxide twice weekly for 15 weeks. Patients were divided into two cohorts: lower-risk MDS (International Prognostic Scoring System risk category low or intermediate 1) and higher-risk MDS (International Prognostic Scoring System risk category intermediate 2 or high). Modified International Working Group criteria were used for response evaluation.
Results:
Of 115 patients enrolled and treated in the study, 67% of patients were transfusion dependent at baseline; median age was 68 years. Most treatment-related adverse events were mild to moderate. The overall rate of hematologic improvement (intent-to-treat) was 24 (19%) of 115, including one complete and one partial response in the higher-risk cohort. The hematologic response rates were 13 (26%) of 50 and 11 (17%) of 64 in patients with lower-risk and higher-risk MDS, respectively. Major responses were observed in all three hematologic lineages; 16% of RBC transfusion-dependent patients and 29% of platelet transfusion-dependent patients became transfusion independent. At data cut off, the median response duration was 3.4 months, with responses ongoing in nine patients.
Conclusion:
Arsenic trioxide treatment consisting of an initial loading dose followed by maintenance therapy has moderate activity in MDS, inducing hematologic responses in both lower- and higher-risk patients. This activity combined with a manageable adverse effect profile warrants the additional study of arsenic trioxide, particularly in combination therapy, for the treatment of patients with MDS.
Insights
Arsenic trioxide showed moderate activity in myelodysplastic syndromes (MDS), with hematologic improvement in 19% of patients. This treatment is safe and effective for both lower- and higher-risk MDS patients.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Myelodysplastic syndromes (MDS) are a group of clonal hematopoietic stem cell disorders.
- Current treatments for MDS have limitations, necessitating the exploration of novel therapeutic agents.
Purpose of the Study:
- To evaluate the safety and efficacy of arsenic trioxide in patients diagnosed with myelodysplastic syndromes (MDS).
Main Methods:
- 115 MDS patients received arsenic trioxide with a loading dose followed by maintenance therapy.
- Patients were stratified into lower-risk and higher-risk cohorts based on the International Prognostic Scoring System.
- Response was evaluated using modified International Working Group criteria.
Main Results:
- Overall hematologic improvement was observed in 19% of patients (24/115).
- Responses occurred across all three hematologic lineages, with some patients becoming transfusion independent.
- Adverse events were generally mild to moderate, indicating a manageable safety profile.
Conclusions:
- Arsenic trioxide demonstrates moderate activity in MDS, benefiting both lower- and higher-risk patient groups.
- The manageable safety profile supports further investigation of arsenic trioxide, particularly in combination therapies for MDS.
