Clinical trial NCT01964625: Positron Emission Tomography - Computed Tomography (PET-CT) Scanning in Chronic Graft Versus Host Disease (cGvHD)
Official title: Assessment of Positron Emission Tomography - Computed Tomography (PET-CT) Scanning as a Potential Biomarker to Assess Disease Activity in Chronic Graft Versus Host Disease (GvHD): A Pilot Study
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Chronic Graft Versus Host Disease
Keywords: Chronic GvHD
Lead sponsor: University of Rochester
Sponsor class: OTHER
First posted: 2013-10-17
Enrollment: 1
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: DIAGNOSTIC

Brief summary: Graft versus Host Disease (GvHD), in both its acute and chronic forms, is the major intrinsic complication of allogeneic hematopoeitic stem cell transplant (allo-HSCT). Moreover, chronic GvHD may be regarded as a "late effect" of cancer therapy, and the severity of chronic GvHD is the chief determinant of long-term survival following allo-HSCT. Unfortunately, the investigators understanding (and thus management) of chronic GvHD is not optimal; a recent NIH Consensus Conference has defined inadequacies in virtually all facets of chronic GvHD management. Notably for this study, the lack of suitable biomarkers compromises diagnosis, staging and therapeutic response evaluation of chronic GvHD - and also hinders better understanding of the biology of this process.

In particular, the activity of chronic GvHD is often difficult to discern, potentially causing either undertreatment, with the risk of morbidity and/or mortality due to uncontrolled chronic GvHD, or possibly overtreatment, with potent ISTs causing unnecessary toxicity. Obviously, the development of reliable biomarkers of chronic GvHD activity would be a very useful advance in addressing this problem, as well as other facets of management not addressed due to certain limitations, as detailed herein.

Potentially, certain imaging technologies could address this problem. To date, imaging technology has been used only sporadically in chronic GvHD and is not an integral part of routine assessments. However, and despite its nonspecific nature, certain "inflammatory" features of some chronic GvHD cases, plus clinical similarity to certain autoimmune diseases in which functional imaging has been tested in research trials - (and perhaps notably), a limited experience in acute GvHD - the investigators postulate that Positron emission tomography - computed tomography (PET-CT) scans may be useful as a biomarker of disease activity in chronic GvHD. This protocol is an initial effort to that end.


Eligibility criteria: Inclusion Criteria:

* Prior allogeneic hematopoietic stem cell transplant (+/- DLI)
* As per current recommendations, no interval from allo HSCT will be required to differentiate acute from chronic Graft versus Host Disease (GvHD)
* Patients must have had negative routine restaging PET-CT scans
* Patients may not have had any more than two weeks' specific treatment for chronic GvHD. (It is recognized that some patients develop chronic GvHD while on immunosuppressive prophylaxis; such patients will remain eligible, even if dose adjustment of these prophylactic agents occurs. The "two week" interval pertains to the use of additional agents in this case.)
* Diagnosis of chronic GvHD must be confirmed. In the usual case, tissue biopsy will be required; however, some chronic GvHD patients do not require tissue biopsy for confirmation. The PI and co-investigator must agree on eligibility.
* Per patient approval by PI and one additional co-investigator
* Informed consent

Exclusion Criteria:

* Patients with a malignancy not in remission will be excluded.
* Negative routine pregnancy testing. Patients either pregnant or unwilling to use satisfactory contraception (if appropriate) will not be eligible. All female patients must use a highly effective birth control method or a combination of 2 additionally effective birth control methods while in this study. Examples of highly effective birth control are: a condom or a diaphragm with spermicidal jelly, oral, injectable, or implanted birth control, or abstinence.

Primary outcomes:
measure: To determine ability of PET-CT scans to delineate and stage initial chronic GvHD
timeFrame: At initial diagnosis of chronic GvHD
Secondary outcomes:
measure: To determine ability of PET-CT scans to document response to chronic GvHD therapy
timeFrame: PET-CT performed </= 6 months from study PET-CT #1
Other outcomes:
Locations:
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
Study officials:
affiliation: University of Rochester
name: Gordon L Phillips, II, MD
role: PRINCIPAL_INVESTIGATOR
References: