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

Primary outcomes

1. Clinical Response according to the 2014 criteria

   Time frame: Up to 6 months

   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.

2. Time to next systemic treatment

   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

   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.

3. Duration of treatment

   Time frame: Up to 3 months

   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.

4. 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

5. Non-relapse mortality

   Time frame: Up to 3 years

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

6. Patient-reported outcomes

   Time frame: Up to 3 years

   Will be assessed using Lee symptom scale and Patient Reported Outcomes Measurement Information System (PROMIS). The summary score of the Lee Symptom Scale and the PROMIS Global will be calculated according to the instructions of the developers. For analyses assessing change in quality of life, improvement or worsening of the Summary symptom score by 6 points or more or the PROMIS Physical or Mental Functioning scales by 5 points or more compared to baseline will be considered a clinically significant change.