Hematopoietic stem cell transplantation (HSCT) is a standard treatment for malignant and non-malignant conditions that lead to immune system failure, such as haematology malignancies, solid tumours and autoimmune diseases . Chemotherapy and/or radiotherapy-based conditioning regimen is administered prior to stem cell infusion to eradicate the underlying disease, to create space for engraftment and to provide immunosuppression . Patients undergoing autologous HSCT receive their own stem cells, while those undergoing allogeneic HSCT receive stem cells from related or unrelated donors. Stem cells may be harvested from bone marrow, peripheral blood or cord blood .
Oral mucositis (OM), a common toxic effect of the regimen drugs used, occurs in 70–99% of HSCT patients [4, 5], and the incidence of its severe forms range from 20% to over 75%, depending on the intensity of the conditioning regimen and on the patient’s predisposing factors [6, 7]. OM consists in the inflammation of the oral mucosal barrier accompanied by various signs and symptoms, such as erythema, ulcers, difficulty eating and/or drinking and pain . Severe cases of OM are associated with extreme discomfort and may affect patients’ quality of life (QoL) and transplant-related outcomes, including infection risk and procedure-related costs [9–11].
There is limited evidence in the literature on OM prevention, and treatment is frequently symptomatic and largely based on anecdotal evidence [12, 13]. Recent evidence is available regarding the topical application of natural products, including honey, aloe vera (AV), bovine colostrum (BC) and others [14, 15]. These composite agents contain a wide variety of biologically active substances, such as lactoferrin, lactoperoxidase, growth factors, immunoglobulins, cytokines, iron, folic acid, electrolytes and vitamins, which may interfere with the pathobiological pathway underlying OM [16–18]. The beneficial effects of these agents on mucosal healing have already been described as mainly attributable to their immune-modulatory, anti-inflammatory and antibacterial activity [14, 15, 19]. In addition, their topical formulas provide emollient, moisturizing and hydrating effects.
The impact of OM on clinical outcomes is generally underestimated, although patients often cite OM as one of the worst side effects of their treatment . For this reason, collecting patient-reported outcome measures (PROMs) as part of the patient’s clinical assessment is recommended in daily practice . Increased consideration of patients’ perspectives during anticancer treatments has allowed healthcare professionals (HCPs) to improve the quality of supportive care in oncology, and PROMs are commonly assessed in clinical trials . It has been shown that patient-reported assessment tools make it possible to detect symptom onset, peak and resolution earlier than does HCPs’ objective evaluation [23, 24].
Knowledge of the impact of OM on patient QoL during HSCT is still limited due to the tendency to consider QoL as a secondary outcome in clinical trials which are designed to explore the toxicity of cancer treatments. Furthermore, QoL is affected by various factors during HSCT, and the assessment of its relationship with OM can be very complex . The increase in the degree of OM has been associated with worse patient QoL; the adoption of strategies to prevent OM, such as low-level laser therapy (LLLT) and professional oral care, has led to improvements in QoL [11, 26].
A recent phase II study conducted by our group demonstrated that a BC–AV-based oral care protocol effectively and safely reduced the incidence of severe OM (sOM) in patients undergoing HSCT . In that study, both PROMs and healthcare professional-reported outcomes (HCP-ROs) were collected to give a true picture of toxicity and its effect on QoL.
This paper reports a secondary analysis on patient-reported data with the aim to describe the variations of QoL perceived by HSCT patients during their hospital stay and to assess the severity of patient-reported symptoms and functional impairments. Significant differences between patient- and HCP-reported assessment strategies were evaluated.