How we treat refractory chronic graft-versus-host disease

Stefanie Sarantopoulos, Adela R. Cardones and Keith M. Sullivan


Approximately 35-50% of patients otherwise cured of hematologic malignancies after allogeneic hematopoietic stem cell transplantation will develop the pleomorphic autoimmune-like syndrome known as chronic graft-versus-host disease (cGVHD). Starting in 2005, NIH consensus panels have proposed definitions and classifications of disease to standardize treatment trials. Recently, the first agent was approved by the Food and Drug Administration for steroid-refractory cGVHD. Despite these advances, most individuals fail to achieve durable resolution of disease activity with initial treatment. Moreover, standardized recommendations on how to best implement existing and novel immunomodulatory agents and how to best taper salvage agents are often lacking. Given the potential life-threatening nature of cGVHD, we employ in our practice templated patient assessments at each clinic visit to elucidate known prognostic indicators and 'red flags.' We find NIH scoring templates practical for ongoing assessments of these complex patients and for determination of when changes in immunosuppressive therapy are warranted. Patients not eligible or suitable for clinical trials have systemic and organ-directed adjunctive treatments crafted in a multi-disciplinary clinic. Herein, we review these treatment options and offer a management and monitoring scaffold for representative patients with cGVHD not responding to initial therapy.

  • Submitted April 23, 2018.
  • Accepted January 20, 2019.