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 GVHDâ€, Palmer et al suggest.
“Alternatively, worsening symptoms and quality of life may simply reflect declining health with its associated higher mortality ratesâ€, 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.â€
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