Mucosal toxicity in hematological malignancies: prevention, management, and novel therapeutic insights

Mucosal toxicities remain a longstanding and challenging concern in the treatment of hematopoietic malignancies (HM). In addition to the classic oral (OM) and gastrointestinal mucositis (GIM) induced by chemotherapy (CHT) and/or radiotherapy (RT), novel targeted treatments and immunotherapies may cause other forms of mucosal disorders.

This overview provides updated insights into the pathobiology and management strategies for mucosal toxicities induced by treatments for HMs. Additionally, it reappraises classic forms of mucositis and novel mucosal toxicities induced by new treatments for HMs.

Although significant progress has been made in the pathophysiologic pathways of conventional CHT/RT-associated OM, much remains to be discovered. Indeed, OM and GIM have a multifactorial etiopathogenesis that includes direct effects, oxidative injury, upregulation of immunologic molecules, and changes in the microbiome. Preventive measures remain the cornerstone of management, mainly palliative in clinically established mucositis. However, new therapeutic insights, primarily related to mesenchymal cells and cytokine inhibitors, are emerging, and ongoing research is critical for translating these new findings into clinical practice.