Arsenic trioxide for treatment of myelodysplastic syndrome: An experience from India

Abstract

13156 Background: Myelodysplastic Syndrome (MDS) is a heterogeneous group of clonal diseases of the haematopoietic stem cells. The hallmark of the disease is ineffective haematopoiesis characterized by dysplasia with incomplete maturation and progressive increase in the percentage of myeloblast. No standard treatment is currently available for MDS. The early clinical experience has confirmed the activity of arsenic trioxide in MDS. The drug is able to induce differentiation and apoptosis and to inhibit cell proliferation or angiogenesis. It has the potential to be active in tumour models in MDS. The preliminary result of ongoing studies conducted in patients with MDS suggests that arsenic trioxide produces haematological improvement including durable transfusion independence in 30% of patients. The aim of our study was to see the response of MDS with arsenic trioxide and to see the toxicity profile of arsenic trioxide in Asian Indian population. Methods: During period from July 2005 to December 2005 we selected consecutive 10 patients of MDS in Refractory Anaemia, Refractory anaemia with ringed sideroblasts, Refractory anaemia with blast excess, Refractory anaemia with blast excess in transformations and chronic myelo monocytic leukemia phases. All patients had performance status more than 60%, some karyotypic abnormalities & in cytopenic phase. Median age of the patients 65 years (range 42 to 70 years). All patients were treated with arsenic trioxide 10mg (Alkem/India) daily for 2 hours infusion 28 days. In 15 days interval 3 courses were repeated. Response assessments were done by haematological, cytogenetic & quality of life assessment. All patients were evaluated after 3 courses of arsenic trioxide. Result: Sixty percent (6 patients) patients had shown major haematological response, forty percent minor & twenty percent has major cytogenetic response. Twenty percent of the patients has disease progression where as 20% has stable disease. The only mild adverse effects were seen in forms of nausea, vomiting, diarrhea, abdominal pain & dermatitis in 30% of patients. Only one patient (10%) had QT prolongation in ECG. Conclusion: We concluded that arsenic trioxide is very useful drug in myelodysplastic syndrome. It is also well tolerated in Asian Indian Population. No significant financial relationships to disclose.