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

Abstract

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<0.0001). A systemic effect of topical GM-CSF upon the number of peripheral blood leukocytes or granulocytes was excluded. We conclude that the topical application of GM-CSF by mouthwash significantly abbreviated the duration and relieved patients from symptoms of chemotherapy-induced mucositis and was superior to the topical application of AA.

• Authors: M Hejna, W J Köstler, M Raderer, G G Steger, T Brodowicz, W Scheithauer, C Wiltschke, C C Zielinski
• Journal: European journal of cancer (Oxford, England : 1990) (Eur J Cancer) Vol. 37 Issue 16 Pg. 1994-2002 (Nov 2001) ISSN: 0959-8049 [Print] England
• PMID: 11597376 (Publication Type: Clinical Trial, Journal Article, Randomized Controlled Trial)

Chemical References

• Antimetabolites, Antineoplastic
• Mouthwashes
• Protective Agents
• Recombinant Proteins
• Amphotericin B
• Granulocyte-Macrophage Colony-Stimulating Factor
• Povidone-Iodine
• molgramostim
• Fluorouracil

Topics

• Adult
• Aged
• Aged, 80 and over
• Amphotericin B (therapeutic use)
• Antimetabolites, Antineoplastic (adverse effects)
• Drug Administration Schedule
• Female
• Fluorouracil (adverse effects)
• Granulocyte-Macrophage Colony-Stimulating Factor (therapeutic use)
• Humans
• Leukocyte Count
• Male
• Middle Aged
• Mouth Mucosa
• Mouthwashes
• Povidone-Iodine (therapeutic use)
• Prospective Studies
• Protective Agents (therapeutic use)
• Recombinant Proteins (therapeutic use)
• Severity of Illness Index
• Single-Blind Method
• Stomatitis (chemically induced, drug therapy, pathology)
• Time Factors
• Treatment Outcome