Abstract
BackgroundSalivary gland hypofunction and xerostomia are major complications following radiotherapy for head and neck cancer and may lead to debilitating oral disorders and impaired quality of life. Currently, only symptomatic treatment is available. However, mesenchymal stem cell (MSC) therapy has shown promising results in preclinical studies. Objectives are to assess safety and efficacy in a first-in-man trial on adipose-derived MSC therapy (ASC) for radiation-induced xerostomia.MethodsThis is a single-center, phase I/II, randomized, placebo-controlled, double-blinded clinical trial. A total of 30 patients are randomized in a 1:1 ratio to receive ultrasound-guided, administered ASC or placebo to the submandibular glands. The primary outcome is change in unstimulated whole salivary flow rate. The secondary outcomes are safety, efficacy, change in quality of life, qualitative and quantitative measurements of saliva, as well as submandibular gland size, vascularization, fibrosis, and secretory tissue evaluation based on contrast-induced magnetic resonance imaging (MRI) and core-needle samples. The assessments are performed at baseline (1 month prior to treatment) and 1 and 4 months following investigational intervention.DiscussionThe trial is the first attempt to evaluate the safety and efficacy of adipose-derived MSCs (ASCs) in patients with radiation-induced xerostomia. The results may provide evidence for the effectiveness of ASC in patients with salivary gland hypofunction and xerostomia and deliver valuable information for the design of subsequent trials.Trial registrationEudraCT, Identifier: 2014-004349-29. Registered on 1 April 2015.ClinicalTrials.gov, Identifier: NCT02513238. First received on 2 July 2015.The trial is prospectively registered.
Highlights
Salivary gland hypofunction and xerostomia are major complications following radiotherapy for head and neck cancer and may lead to debilitating oral disorders and impaired quality of life
With regards to cancer chemotherapy, studies suggest that in some patients it may induce temporary salivary gland hypofunction and xerostomia during and following treatment, while other patients are not affected to a noticeable extent; no firm conclusions can be drawn from the medical literature [2]
Justification for patient population In order to standardize the participant population, we choose to only include participants treated for a human papilloma virus (HPV)-positive oropharyngeal squamous cell carcinoma, and to exclude patients with severe salivary gland hypofunction, as this population most likely will not benefit from this treatment
Summary
Primary outcome Primary outcome is change in unstimulated whole salivary flow rate. Secondary outcomes Secondary outcomes are: Safety, including adverse events (AEs) and severe adverse events (SAEs). Justification for patient population In order to standardize the participant population, we choose to only include participants treated for a human papilloma virus (HPV)-positive oropharyngeal squamous cell carcinoma, and to exclude patients with severe salivary gland hypofunction (hyposalivation, e.g., whole saliva flow rate
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