Six-month markers predict long-term cGVHD outcomes

Nov 27 2015

By Lynda Williams, Senior medwireNews Reporter

Researchers have identified measures early in the course of chronic graft-versus-host disease (cGVHD) that predict long-term prognosis in haematopoietic cell transplantation (HCT) recipients.

Jeanne Palmer, from Mayo Clinic in Phoenix, Arizona, USA, and co-authors write in Blood that identification of 6-month surrogate endpoints for overall survival (OS), failure-free survival (FFS) and non-relapse mortality (NRM) is “critical” for clinical trial design.

FFS has been proposed as an intermediate endpoint to measure treatment success, defined as disease-free survival without the need for a new systemic immunosuppressive therapy, they explain.

The team collated information for 575 cGVHD patients enrolled a median of 11.9 months after transplantation, 451 of whom were assessed at 6 months. Patients were followed-up for a median of 44 months, at over 2000 clinic visits, and 26% died during the study.

Analysis showed that 2014 National Institutes of Health (NIH) consensus criteria response measures at 6 months – used to assess changes in the skin, mouth, eye, lung, joints, gastrointestinal tract and liver – were significantly predictive of FFS.

And clinician-reported response, based on surveys used to classify patients as having a complete or partial response, or stable or progressive disease, were significantly predictive at 6 months for both FFS and OS.

In addition, FFS was also significantly predicted by a change in skin score on the 2005 NIH clinician-reported measure and a patient-reported itching score at 6 months, while OS significantly correlated with the Functional Assessment of Cancer Therapy–Bone Marrow Transplant score at 6 months. Also, the Lee skin symptom score at 6 months significantly correlated with both OS and NRM.

“These associations may be due to the increased immunosuppression given to patients who have advanced and symptomatic chronic GVHD”, Palmer et al suggest.

“Alternatively, worsening symptoms and quality of life may simply reflect declining health with its associated higher mortality rates”, they write.

The team concludes: “Based on these data, we recommend that for now, the 2014 NIH response measures, clinician-reported responses and patient-reported outcomes be collected in therapeutic trials of chronic GVHD to ensure that relevant data are available once the best algorithm to capture a meaningful objective response is determined.”

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