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Published on: June 6, 2025
Amifostine treatment in patients with myelodysplastic syndrome
Abstract:
Myelodysplastic syndrome (MDS) is a clonal disorder that is characterized by peripheral cytopenia and the induction of apoptosis is thought to be partially responsible for pathological haematopoiesis in MDS. Amifostine is a cytoprotective and antioxidant agent, and it may prolong the survival of progenitor cells in MDS by delaying apoptosis. The study has been carried out with 9 MDS cases. Four of them were diagnosed as refractory anemia (MDS-RA), two as refractory anemia with ring sideroblasts (MDS-RARS) and the remaining three as refractory anemia with excess blasts (MDS-RAEB) according to the French-American-British (FAB) classification. Amifostine was given in a dose of 400 mg/m2, as an IV infusion administered in 5-6 minutes, three times a week for 4 consecutive weeks. Three of the cases (33.3%), two with MDS RARS and one with MDSRA, showed a significant improvement in the number of total leukocyte, neutrophil and reticulocyte counts and a decrease in the requirement of erithrocyte transfusions. In clinically responsive cases, all hematological parameters returned back to pre-treatment values two weeks after the cessation of therapy. We conclude that Amifostine can be used in a selected group of patients with MDS-RA and MDS-RARS.
Insights
Amifostine may improve blood counts and reduce transfusions in myelodysplastic syndromes (MDS), particularly in refractory anemia (MDS-RA) and refractory anemia with ring sideroblasts (MDS-RARS) subtypes. Further research is needed to confirm these findings.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Myelodysplastic syndromes (MDS) are clonal hematopoietic stem cell disorders.
- Peripheral cytopenia and pathological hematopoiesis are key features of MDS.
- Apoptosis induction is implicated in MDS pathogenesis.
Purpose of the Study:
- To investigate the efficacy of amifostine in treating myelodysplastic syndromes.
- To evaluate amifostine's potential to delay apoptosis and improve progenitor cell survival in MDS.
Main Methods:
- Nine patients with MDS (MDS-RA, MDS-RARS, MDS-RAEB) were treated with amifostine.
- Amifostine was administered intravenously at 400 mg/m2, three times weekly for 4 weeks.
- Hematological parameters and transfusion requirements were monitored.
Main Results:
- Three patients (33.3%) showed significant improvements in leukocyte, neutrophil, and reticulocyte counts.
- Two MDS-RARS and one MDS-RA patient experienced reduced red blood cell transfusion needs.
- Hematological parameters normalized within two weeks post-treatment in responsive cases.
Conclusions:
- Amifostine demonstrates potential therapeutic benefit in selected patients with MDS-RA and MDS-RARS.
- Amifostine may offer a novel treatment approach by mitigating apoptosis in MDS.
- Further clinical trials are warranted to validate these preliminary findings.
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