title: Treatment of myelodysplastic syndromes (MDS) and high leukaemic risk with low-dose cytosine arabinoside (LD-AraC) plus granulocyte-macrophage colony-stimulating factor (rh GM-CSF). The EORTC Leukaemia Group.
abstract: Symptomatic patients with myelodysplastic syndromes (MDS) and 10-30% blasts in the bone marrow were treated with low-dose AraC (2 x 10 mg/m2 subcutaneously (sc) days 1-14) and GM-CSF (fully glycosylated, Sandoz/Schering-Plough, 2 x 150 micrograms protein/day sc) given either subsequently (days 15-21) or simultaneously (days 8-14 and one week rest). Evaluations were carried out after three courses (nine weeks); responding patients could be continued for two further cycles. Eighty-two patients with refractory anaemia and excess of blasts (RAEB), with (RAEBt) or without transformation, were evaluable: 45 RAEB and 37 RAEBt, mean age 64 years (range 17-80 years). A complete remission was achieved in 14 cases (17%), 11 had a good response (13%), and 12 a partial response (15%). Stable disease was found in 21 cases (26%). There were 12 cases of toxic death (15%), progression was noted in eight patients (10%), and death due to disease in three (4%). No difference existed between the two treatment arms with respect to response. Major adverse events during treatment were haemorrhage (25%), infections (23%), and fever with GM-CSF (21%). GM-CSF did not induce leukaemia nor contribute to haemorrhage induced by AraC, but gave rise to an overall response rate of 46% which is high and relatively durable as compared to other treatments in this disease.
authors.affiliation: Medizinische Klinik III, Klinikum Grosshadern, München, Germany.
authors.forename: H H
authors.initials: HH
authors.lastname: Gerhartz
authors.forename: R
authors.initials: R
authors.lastname: Marcus
authors.forename: A
authors.initials: A
authors.lastname: Delmer
authors.forename: H
authors.initials: H
authors.lastname: Zwierzina
authors.forename: T
authors.initials: T
authors.lastname: de Witte
authors.forename: A
authors.initials: A
authors.lastname: Jacobs
authors.forename: G
authors.initials: G
authors.lastname: Visiani
authors.forename: D
authors.initials: D
authors.lastname: Fiere
authors.forename: P
authors.initials: P
authors.lastname: Sonneveld
authors.forename: B
authors.initials: B
authors.lastname: Labar
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000754:Anemia, Refractory, with Excess of Blasts / Q000188:drug therapy*; D001853:Bone Marrow / Q000187:drug effects*; D003561:Cytarabine / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D005260:Female; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D007279:Injections, Subcutaneous; D007938:Leukemia / Q000209:etiology; D008297:Male; D008875:Middle Aged; D012307:Risk Factors; D015996:Survival Rate
chemical_list: D003561:Cytarabine; D016178:Granulocyte-Macrophage Colony-Stimulating Factor