Itacitinib, Tacrolimus, and Sirolimus for the Prevention of GVHD in Patients With Acute Leukemia, Myelodysplastic Syndrome, or Myelofibrosis Undergoing Reduced Intensity Conditioning Donor Stem Cell Transplantation
Key claim: A phase IIa trial is evaluating whether adding itacitinib to standard tacrolimus and sirolimus prophylaxis can safely and effectively prevent GVHD in patients with acute leukemia, MDS, or myelofibrosis undergoing reduced-intensity conditioning allogeneic stem cell transplantation.
Abstract
This phase IIa trial studies the side effects of itacitinib when given together with standard treatment (tacrolimus and sirolimus), and to see how well it works in preventing graft-versus-host-disease (GVHD) in patients with acute leukemia, myelodysplastic syndrome or myelofibrosis who are undergoing reduced intensity conditioning donor stem cell transplantation. GVHD is a common complication after donor stem cell transplantation, resulting from donor immune cells recognizing recipients’ cells and attacking them. Adding itacitinib to tacrolimus and sirolimus may reduce the risk GVHD and ultimately improve overall outcome and survival after donor stem cell transplantation.
Why this matters
GVHD prophylaxis is a major determinant of allo-HSCT outcomes for myelofibrosis patients, and the field is rapidly diversifying beyond ruxolitinib-based JAK inhibition. This itacitinib trial probes whether JAK1 selectivity can prevent GVHD without the JAK2-mediated cytopenias that complicate ruxolitinib use peri-transplant, and its inclusion of MF alongside acute leukemia/MDS will inform whether the approach generalizes across disease contexts using RIC.