Ruxolitinib in the Treatment of Acute Corticosteroid-Refractory Graft-Versus-Host Disease: A Scoping Review

Abstract

Background: Graft-versus-Host Disease (GVHD) is a common complication following allogeneic hematopoietic stem cell transplantation (HSCT) and is considered one of the leading causes of post-transplant morbidity and mortality. Acute GVHD (aGVHD) primarily affects the skin, gastrointestinal tract, and liver. Despite advances in immunoprophylaxis, 20% to 80% of patients develop aGVHD, of whom only 60% respond to first-line treatment (e.g., methylprednisolone). Ruxolitinib, a selective inhibitor of Janus Kinase (JAK) 1 and 2, has been proposed as a treatment option following failure of first-line therapy.

Objective: The present study aims to map the current evidence regarding the use of ruxolitinib in the treatment of glucocorticoid-refractory aGVHD in patients with hematologic diseases undergoing allogeneic HSCT.

Study design: We performed a scoping review in accordance with the Joanna Briggs Institute guidelines. Systematic searches were performed in the PubMed and Scopus databases in December 2024, with no restrictions on year or language. Two independent reviewers carried out article selection and data extraction.

Results: A total of 1162 studies were screened, with 13 matching the inclusion criteria. The articles were published between 2016 and 2024 and originated from developed countries, with the majority from China (33.8%) and Germany (23.1%), being predominantly retrospective cohort studies (38.5%) and case reports or case series (38.5%). The main adverse effects reported included infections, viral reactivation, and pancytopenia. The overall response rates were 65.0% for combination therapy and 67.4% in comparison with other therapies. All patients who received monotherapy achieved a complete or partial response to treatment, however, the studies involved small sample sizes, limiting the ability to extrapolate these findings.

Conclusions: Ruxolitinib demonstrates therapeutic potential in this context; however, higher-level primary studies, such as randomized controlled trials, are necessary to achieve more robust conclusions.

Keywords: Graft vs Host Disease; Hematopoietic Stem Cell Transplantation; Janus Kinase Inhibitors; Scoping Review.