New Developments in Management of Oral Mucositis in Patients With Head and Neck Cancer or Receiving Targeted Anticancer Therapies

Edward Li; James A. Trovato


Am J Health Syst Pharm. 2012;69(12):1031-1037. 

In This Article

Abstract and Introduction


Purpose Issues surrounding the prevention and management of severe oral mucositis in patients with head and neck cancer and patients receiving targeted anticancer therapies are reviewed.
Summary Cancer therapy-related mucositis is associated with many negative and potentially life-threatening sequelae. Patients receiving chemoradiotherapy for head and neck cancer are at high risk for severe oral mucositis, but there are currently no definitive recommendations on pharmacologic preventive strategies. Recently published evidence on the use of palifermin to combat oral mucositis during chemoradiotherapy for head and neck cancer is encouraging, with two randomized controlled trials indicating an absolute risk reduction of about 15%; however, palifermin use was not associated with lower rates of mucositis-related treatment delays or chemotherapy dosage reductions, and concerns about optimal dosage and cost–benefit issues persist. Oral mucositis due to targeted therapies for renal cell carcinoma and other disorders (e.g., kinase inhibitors, mammalian target of rapamycin inhibitors) is generally less severe than mucositis caused by conventional cytotoxic chemotherapy. For both types of mucositis, recommended management strategies include good oral hygiene and optimal pain control. Research in this area continues to be complicated by investigators' use of varying terminology and mucositis classification schemes.
Conclusion Palifermin appears to reduce the frequency of oral mucositis in patients treated for head and neck cancer, but its place in therapy has not been determined. Although the oral complications of targeted therapies are clinically distinct from those of conventional cytotoxic therapy, the literature recommends similar palliative management strategies for both.


Oral mucositis as a result of chemotherapy or chemoradiotherapy is a common supportive care issue and is associated with many negative health and economic consequences.[1] The common clinical presentation includes the development of inflammation and ulceration of the oral mucosa, resulting in pain that varies in intensity depending on the severity of the condition. Severe mucositis can lead to other negative effects such as the inability to eat or drink, chemotherapy interruptions or dosage reductions, and infection; these sequelae, if severe enough, can potentially lead to death.[1,2]

Because of the potential for severe sequelae, the prevention and management of cancer treatment-related mucositis have been the focuses of considerable professional discussion. Recommendations and principles outlining strategies for preventing and managing mucositis have been established by many organizations. For instance, the Multinational Association of Supportive Care in Cancer (MASCC) and the International Society for Oral Oncology (ISOO) have reviewed the evidence on interventions for managing mucositis and first published joint practice guidelines in 2004.[3] An update to those guidelines was published in 2007.[4] The National Comprehensive Cancer Network (NCCN) does not produce guidelines on mucositis management, but the organization convened a task force to explore the topic, resulting in the publication of a task force report in 2008.[5] The American Society of Clinical Oncology also does not publish guidelines on managing mucositis, but some relevant information can be found in its guideline "Use of Chemotherapy and Radiation Therapy Protectants," which was updated in 2008.[6] Also in 2008, the Oncology Nursing Society (ONS) published a review of the evidence on various interventions for the management of mucositis.[7] More recently, in 2010, the European Society for Medical Oncology (ESMO) published clinical practice guidelines for the management of mucositis based on the 2007 MASCC–ISOO guidelines.[2]

From the aforementioned documents, common themes in the prevention and management of mucositis have emerged. Examples include good oral hygiene (to be promoted through the implementation of oral care protocols), the avoidance of mouthwash products containing alcohol, the use of palifermin in patients with hematologic malignancies receiving high-dose chemotherapy plus stem cell transplantation (with or without total body irradiation), and vigilance in addressing pain, nutrition, and concomitant infections. Additionally, most of the publications discuss therapies that should not be used for the management of mucositis, and there appears to be a consensus that sargramostim mouthwashes, chlorhexidine, and sucralfate are ineffective and should not be used.

Sometimes professional organizations issue conflicting recommendations regarding a specific therapy. For example, "magic mouthwash" preparations (various mouth rinse formulations containing topical anesthetics and antiinflammatory agents) are not recommended in the MASCC–ISOO guidelines or the ONS review, but they were mentioned as useful therapies in the NCCN task force report.[4–6]

The recommendations and position statements offered by professional organizations are the results of substantial efforts to collect, evaluate, and interpret the existing literature relevant to the prevention and management of mucositis in patients receiving treatment for cancer. Since the publication of the various documents mentioned above, new information has emerged to guide the prevention of mucositis in patients with head and neck cancer, as well as mucositis resulting from targeted therapies. This article specifically focuses on those two issues, since they are not discussed in the publications mentioned above. Therapies about which there are conflicting position statements (e.g., magic mouthwashes) and others lacking sufficient supportive evidence have been discussed in length elsewhere and will not be reviewed here because new and sufficiently meaningful evidence likely to change the direction of those discussions has not emerged in recent years.


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