Decrease of duration and symptoms in chemotherapy-induced oral mucositis by topical GM-CSF: results of a prospective randomised trial

We have conducted a prospective controlled randomised clinical study testing for the efficacy of topical GM-CSF (molgramostim), as compared to the combined topical use of an antiseptic agent (povidone-iodine) and amphotericin B (AA) in patients with chemotherapy-induced mucositis World Health Organization (WHO) grades I–III. 31 patients (17 females, 14 males) developing oral mucositis following the administration of 5-fluorouracil (5-FU)-based chemotherapy were entered into the present trial. 15 patients were randomised to receive GM-CSF mouthwashes, whereas 16 patients were randomised into the control arm to receive AA. Reported history (P=0.6109) and grading of oral mucositis (2.1±0.7, respectively; P=0.9867) were balanced and equally distributed between the two groups. The mean size of lesions of oral mucositis was 1.5±0.6 cm (range: 0.7–2.5 cm) in the GM-CSF group and 1.2±0.5 cm (range: 0.5–2.5 cm) in the AA group (P=0.08), respectively. The mean number of oral mucositis lesions was 1.9±1.1 (range: 1–4) in the GM-CSF group and 2.1±1.2 (range: 1–4) in the AA group (P=0.63), respectively. None of the patients had previously received colony stimulating factors either topically or systemically. Treatment for oral mucositis was initiated on day 2.7±1.2 (range: day 1–8) after onset of symptoms in the GM-CSF group and on day 1.8±1.4 (range: day 1–3; P=0.11) in the AA group. The topical application of GM-CSF resulted in a significantly shorter duration and quicker resolution of oral mucositis, as compared to AA including both, pretreatment plus treatment periods (5.3±2.5 versus 8.1±1.5 days; P=0.0008) as well as the necessary duration of treatment needed until complete remission of lesions (2.8±0.7 versus 6.3±1.1 days; P