Amifostine in the treatment of low-risk myelodysplastic syndromes.
OBJECTIVE
The phosphorylated aminothiol agent amifostine (Ethyol) protects bone marrow and other tissues from toxicity due to ionizing radiation and antineoplastic drugs, and stimulates progenitors from normal and myelodysplastic bone marrow. Contrasting results have been published so far on the effectiveness of amifostine in correcting cytopenia in patients with myelodysplastic syndromes (MDS).


METHODS
In a pilot phase II study we treated 26 patients with low risk MDS (13 RA, 2 RARS, 2 CMML, 9 RAEB with blasts < 10%) with amifostine (200 mg/m(2 )x 3/week for 4 weeks).


RESULTS
Hemoglobin concentration, reticulocyte, neutrophil and platelet counts increased respectively in 6 (23%), 11 (42%), 13 (50%) and 9 (34%) of patients. Red cell transfusions were reduced (> 50%) in 4/26 patients and abolished in 1/26. Unexpectedly a significant decrease in soluble transferrin receptor level at week 4 of therapy, compared to the basal level (p<0.04), was observed in the whole population of patients.


CONCLUSIONS
Amifostine can ameliorate cytopenia in some patients with MDS, with few and mild side effects. Neutropenia is more likely to be corrected than anemia or thrombocytopenia. Mechanisms underlying this biological effect remain to be clarified.
Grossi
A
A
Division of Hematology, Policlinico Careggi, viale Morgagni 85, Florence, Italy. a.grossi@dfc.unifi.it.
Fabbri
A
A
Santini
V
V
Leoni
F
F
Nozzoli
C
C
Longo
G
G
Pagliai
G
G
Ciolli
S
S
Rossi Ferrini
P
P
D000368:Aged; D000369:Aged, 80 and over; D004999:Amifostine / Q000008:administration & dosage* / Q000633:toxicity; D001772:Blood Cell Count / Q000187:drug effects; D004921:Erythropoietin / Q000097:blood; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000097:blood / Q000188:drug therapy*; D010198:Pancytopenia / Q000188:drug therapy; D010865:Pilot Projects; D011837:Radiation-Protective Agents / Q000008:administration & dosage* / Q000633:toxicity; D011990:Receptors, Transferrin / Q000097:blood; D013926:Thrombopoietin / Q000097:blood
D011837:Radiation-Protective Agents; D011990:Receptors, Transferrin; D004921:Erythropoietin; D013926:Thrombopoietin; D004999:Amifostine