Acyclovir Prophylaxis Reduces Oral Mucositis Incidence in HSCT Recipients


Adding prophylactic acyclovir therapy to the treatment regimen for patients undergoing a stem cell transplant reduced the rates of chemotherapy-induced oral mucositis, a common and significant adverse effect of the treatment.

Multipotent stem cells in the bone marrow: © Juan Gärtner -

Multipotent stem cells in the bone marrow: © Juan Gärtner -

Prophylactic use of the oral antiviral acyclovir (Sitavig, Zovirax) in patients with hematologic malignancies undergoing autologous hematopoietic stem cell transplant (HSCT) reduced the incidence of chemotherapy-induced oral mucositis, a significant adverse effect in this patient population.1

In a phase 2 study (KCT0003885), 54 patients were randomized to the acyclovir or control arms. In the acyclovir arm, 16.0% of patients (n = 4) experienced oral mucositis, while the condition was reported in 58.6% of patients (n = 17) in the control arm (P =.001). All incidences of oral mucositis in the acyclovir arm were grade 1, and 3 patients in the control group experienced grade 2 mucositis.

Moreover, patients in the acyclovir group had lower rates of HSV-1 positivity compared with the control group (4.3% vs 46.2%; P =.001). A total of 13 patients experienced HSV-1 reactivation, and 12 developed oral mucositis, reconfirming an association between HSV-1 reactivation and chemotherapy-induced oral mucositis.

Patients in the acyclovir arm were administered 400 mg of the agent twice daily, a dosage in line with recommendations from the Infectious Diseases Working Party of the German Society for Hematology and Medical Oncology and the European Society for Blood and Marrow Transplant.

“Our study clearly and concisely shows that acyclovir prophylaxis effectively reduces the incidence of HSV-1 reactivation and [chemotherapy-induced oral mucositis] in HSV-seropositive autologous HSCT recipients during the neutropenic phase,” study authors wrote.

“HSCT recipients have the highest incidence of [chemotherapy-induced oral mucositis] among patients with [hematologic malignancies] …These high incidences suggest that [chemotherapy-induced oral mucositis] needs to be actively managed in autologous HSCT recipients, thus supporting the role of acyclovir prophylaxis,” the authors continued.

Mucositis is commonly seen in up to 90% of populations affected with cancer,2 and it is estimated that half of autologous HSCT patients experience it.1 Not only can it be an uncomfortable patient experience, but severe cases can lead to infection, dehydration, and bleeding, potentially disrupting anticancer treatments and worsening patient outcomes.2

Currently, standard-of-care preventive measures for chemotherapy-induced oral mucositis include prophylactic dental care, ice chip therapy, and mucoadhesive hydrogel and calcium phosphate rinses.2 Introducing a prophylactic antiviral to the standard-of-care regiment could present a path forward for management of adverse effects from chemotherapy regimens.

1. Hong J, Park HK, Chang SH et al. A randomized phase II study of acyclovir for the prevention of chemotherapy-induced oral mucositis in patients undergoing autologous hematopoietic stem cell transplantation. BMC Oral Health . 2023;23(1):1008. doi:10.1186/s12903-023-03623-6
2. Brown T, et al. Management of cancer therapy–associated oral mucositis. JCO Oncol Pract. 2020;16(3):103-109. doi:10.1200/JOP.19.00652

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