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

Study Overview
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Brief Summary
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This trial collects clinical data and blood samples to predict the quality of response to specific treatments in patients with chronic graft-versus-host disease (cGVHD) who are about to start initial or second-line therapy. Collecting and analyzing clinical data and blood samples from patients with cGVHD before and after treatment initiation may help doctors identify changes that may predict treatment response.

Detailed Description
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OUTLINE: Patients complete questionnaires over 10 minutes about physical symptoms, activity level, and emotional well-being and have their medical records reviewed at baseline, 1, 3, and 6 months after starting index treatment, and at start of a new systemic treatment. Patients also undergo collection of blood samples over 1-2 minutes at baseline and at 1 month after starting index treatment, or at a treatment change visit if new therapy has not started.

Official Title
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Predicting the Quality of Response to Specific Treatments (PQRST)

Conditions
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Chronic Graft Versus Host Disease

Intervention / Treatment
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* Procedure: Biospecimen Collection
* Other: Medical Chart Review
* Other: Quality-of-Life Assessment
* Other: Questionnaire Administration


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Prior allogeneic stem cell transplant, with any graft source, donor type, and GVHD prophylaxis No evidence of persistent or progressive malignancy at the time of enrollment Agrees to be evaluated at the transplant center before initial or second-line treatment is started (may be concurrent with the enrollment visit), and later between 2-6 weeks, 3 months and 6 months after treatment is started or if a new therapy is started before 6 months Signed, informed consent Exclusion Criteria: Inability to comply with study procedures Uncontrolled psychiatric disorder Anticipated survival < 6 months

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Observational (questionnaire, biospecimen, chart review)<br>Patients complete questionnaires over 10 minutes about physical symptoms, activity level, and emotional well-being and have their medical records reviewed at baseline, 1, 3, and 6 months after starting index treatment, and at start of a new systemic treatment. Patients also undergo collection of blood samples over 1-2 minutes at baseline and at 1 month after starting index treatment, or at a treatment change visit if new therapy has not started. | Procedure: Biospecimen Collection<br>* Undergo collection of blood sample<br>Other: Medical Chart Review<br>* Review of medical chart<br>* Other names: Chart Review;Other: Quality-of-Life Assessment<br>* Ancillary studies<br>* Other names: Quality of Life Assessment;Other: Questionnaire Administration<br>* Complete questionnaire<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 otherwise portable spirometry will be performed. Response will be assessed according to the recommendations of the 2014 NIH response measures publication or any applicable updates. | 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. | 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. | Up to 3 months | 
| Survival |  | 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. | Up to 3 years | 
| Patient-reported outcomes | 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. | Up to 3 years |