As reported in the Journal of Clinical Oncology by Holtan et al, the addition of cyclophosphamide to posttransplantation graft-vs-host disease (GVHD) prophylaxis with tacrolimus and mycophenolate mofetil was associated with improved patient-reported outcomes in the phase III BMT CTN 1703 trial in patients with hematologic cancers.
An update at 2 years showed maintained superiority of the cyclophosphamide group over the control group in GVHD-free relapse-free survival.
Study Details
In the U.S. multicenter trial, 431 patients with hematologic cancers undergoing allogeneic hematopoietic cell transplantation were randomly assigned to posttransplantation prophylaxis with cyclophosphamide plus tacrolimus and mycophenolate mofetil (n = 214) or tacrolimus and mycophenolate mofetil (n = 217). The initial report from the trial showed significantly better GVHD-free relapse-free survival in the cyclophosphamide group vs the control group. The current analysis focused on patient-reported outcomes within the first year after transplantation.
Key Findings
The cyclophosphamide group had significantly lower scores on the Lee Chronic GVHD Symptom Scale (P = .01), indicating lower symptom burden.
Lee Scale nutrition (P <.01) and mouth (P < .01) subscores were significantly better in the cyclophosphamide group.
No significant differences between groups were observed for hemorrhagic cystitis or for PROMIS scales of physical function, gastrointestinal symptoms, or satisfaction with social roles.
Overall, patients aged > 65 years reported better Lee Scale psychological subscores vs younger patients (P = .003).
At 2 years, the cyclophosphamide group maintained a significant benefit in GVHD-free relapse-free survival vs the control group (42.4% vs 28.8%, P = .001).
The investigators concluded, “In addition to improved [GVHD-free relapse-free survival], patients randomly assigned to the [cyclophosphamide group] reported lower symptom burden during the first year after transplant.”
Shernan G. Holtan, MD, of the Department of Medicine, Transplant and Cellular Therapy Program, Roswell Park Comprehensive Cancer Center, Buffalo, is the corresponding author for the Journal of Clinical Oncology article.
Disclosure: The study was supported by the National Heart, Lung, and Blood Institute; National Cancer Institute; and others. For full disclosures of the study authors, visit ascopubs.org.
