Predicting the Quality of Response to Specific Treatments in Patients With cGVHD, PQRST Study

Clinical Response according to the 2014 criteria

At the assessments, the 9 provider-reported National Institute of Health (NIH) organ severity scores (skin, eye, mouth, esophagus, upper gastrointestinal \[GI\], lower GI, liver, lung, and joint manifestations) will be collected reflecting disease activity in the past week. All scored items are single 4-7 point Likert scales. Based on past work, we anticipate it will take approximately 8 minutes to conduct the physical exam and record the relevant results. Pulmonary function testing results will be collected if available. Response will be assessed according to the recommendations of the 2014 NIH response measures publication or any applicable updates.

Time frame: Up to 6 months

Time to next systemic treatment

Any addition of another systemic cGVHD treatment for medical reasons will be considered a failure, whether added because of a new or worsening manifestation of cGVHD, used as a "steroid sparing agent," or substituted due to toxicity.

Time frame: From the start of the index medication until the addition of another systemic chronic graft versus host disease (cGVHD) treatment with death and treated recurrent malignancy considered competing events, assessed up to 3 years

Duration of treatment

Duration of treatment is defined as the time until discontinuation of therapeutic systemic immunosuppression (adrenal replacement and topical/local therapies are allowed) without resumption for at least 3 months.

Time frame: Up to 3 months

Survival

Time frame: From the start of the index medication to death with patients lost to follow up or alive at the conclusion of the study censored, assessed up to 3 years

Non-relapse mortality

Non-relapse mortality is defined as death in remission, and relapse is considered a competing risk.

Time frame: Up to 3 years

Patient-reported outcomes