Treatment of myelodysplastic syndromes with human granulocytic‐macrophage colony stimulating factor (GM‐CSF) or GM‐CSF combined with low‐dose cytosine arabinoside

Abstract

In a phase II study, 21 patients with MDS (RAEB, RAEBt, CMML and RA and RAS with severe cytopenia) were randomized to be treated with 3 courses of GM‐CSF (3 μg/kg/day s.c.) alone (11 patients) or in combination with AraC (20 mg/m2/d s.c.) (10 patients) for 14‐d periods, interrupted by 14‐d rest periods. Eight patients discontinued the treatment. In the GM‐CSF group a marked increase in WBC and neutrophil counts during each course of treatment administration were seen in most patients. Platelet counts decreased in 14 of 24 courses of treatment in the GM‐CSF plus AraC group but in none of the GM‐CSF group. Although the changes in the circulating blood cells were transient and the counts tended to return to the pretreatment levels during the rest periods, some more durable effects were seen. In 3/6 patients of the GM‐CSF group who completed the designed treatment, both WBC and neutrophils remained elevated above the pretreatment levels throughout the 3‐month period of treatment, while in one of them thrombocytopenia improved considerably. In the GM‐CSF plus AraC group, 4 out of the 7 patients who completed the treatment showed an improvement of neutropenia as well as anaemia. In these 4 patients the BM percentage of blasts was also decreased. In conclusion, the results of this study indicate that GM‐CSF given intermittently improves leukopenia in some patients with MDS. In addition, the administration of GM‐CSF seems to prevent granulocytopenia of concurrent AraC treatment and may be of benefit in the treatment of these diseases.

Diagnosis and treatment of primary myelodysplastic syndromes in adults: recommendations from the European LeukemiaNet

“…The evidence on the use of LDAC has been drawn from systematic reviews of the literature and randomized controlled trials. [178][179][180][181][182][183][184][185][186][187] One hundred forty-one patients with one of the FAB-defined subtypes of MDS were included in a combined Eastern Cooperative Oncology Group and Southwest Oncology Group randomized phase 3 study comparing LDAC (10 mg/m 2 subcutaneously twice daily for 21 consecutive days) vs supportive treatment. 183 The overall response rate to a single cycle of LDAC was 32%, with a median duration of response of 5.9 months (range, 1.4 to 33.5 months).…”