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<p>Myelodysplastic syndromes (MDS) are clonal hematopoietic stem cell disorders characterised by dysplasia leading to cytopenias and a high probability of progression to acute myeloid leukemia (AML). Most strategies in lower-risk MDS (LR-MDS) (IPSS-R score ≤ 3.5) have focused on improving transfusion requirements or are only active in a small subset of patients. Hypomethylating agents like azacitidine are an emerging option in improving overall survival in lower risk MDS. To assess the responses of low dose azacitidine in patients of LR-MDS.This was a prospective study done on 20 LR-MDS cases over a study period of 18 months. All patients received subcutaneous azacitidine 75&#xa0;mg/m2 for three days in every four weeks for six cycles and were followed up for another six months.At the end of the study period, 11 patients (55%) showed an overall response. It was found that the mean deviation rise of hemoglobin and absolute neutrophil count at 6 months was higher compared to baseline and a better overall response was achieved among patients with a higher baseline platelet count (P value ≤ 0.05). After 6 cycles of low dose azacitidine therapy, 56% were transfusion-independent and 63% of the patients had improvement in ECOG status. Myelosuppression, thrombocytopenia and infections were observed in 35%, 20% and 25% of the patients, respectively. One-year overall survival was 95%.Low dose azacitidine can a safe and cost-effective treatment option as frontline therapy to achieve an overall response for transfusion-dependent LR-MDS with high EPO levels.</p>

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Outcome of Low Dose Azacitidine in Patients of Lower Risk Myelodysplastic Syndromes

  • 期刊论文

  • 发表日期：

    2025年9月3日

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  • Kaustav Ghosh,
  • Tuphan Kanti Dolai,
  • Shipla Roy,
  • Swapnodeep Sarkar,
  • Prakas Kumar Mandal

摘要

Myelodysplastic syndromes (MDS) are clonal hematopoietic stem cell disorders characterised by dysplasia leading to cytopenias and a high probability of progression to acute myeloid leukemia (AML). Most strategies in lower-risk MDS (LR-MDS) (IPSS-R score ≤ 3.5) have focused on improving transfusion requirements or are only active in a small subset of patients. Hypomethylating agents like azacitidine are an emerging option in improving overall survival in lower risk MDS. To assess the responses of low dose azacitidine in patients of LR-MDS.This was a prospective study done on 20 LR-MDS cases over a study period of 18 months. All patients received subcutaneous azacitidine 75 mg/m2 for three days in every four weeks for six cycles and were followed up for another six months.At the end of the study period, 11 patients (55%) showed an overall response. It was found that the mean deviation rise of hemoglobin and absolute neutrophil count at 6 months was higher compared to baseline and a better overall response was achieved among patients with a higher baseline platelet count (P value ≤ 0.05). After 6 cycles of low dose azacitidine therapy, 56% were transfusion-independent and 63% of the patients had improvement in ECOG status. Myelosuppression, thrombocytopenia and infections were observed in 35%, 20% and 25% of the patients, respectively. One-year overall survival was 95%.Low dose azacitidine can a safe and cost-effective treatment option as frontline therapy to achieve an overall response for transfusion-dependent LR-MDS with high EPO levels.