Elsevier

Ophthalmology

Volume 119, Issue 3, March 2012, Pages 487-493
Ophthalmology

Original article
Validation of Measurement Scales in Ocular Graft-versus-Host Disease

Presented at: BMT Tandem Meeting in Honolulu, Hawaii, February, 2011.
https://doi.org/10.1016/j.ophtha.2011.08.040Get rights and content

Purpose

To validate measurement scales for rating ocular chronic graft-versus-host disease (GVHD) after allogeneic hematopoietic cell transplantation. Candidate scales were recommended for use in clinical trials by the National Institutes of Health (NIH) Chronic GVHD Consensus Conference or have been previously validated in dry eye syndromes.

Design

Prospective follow-up study.

Participants

Between August 2007 and June 2010, the study enrolled 387 patients with chronic GVHD in a multicenter, prospective, observational cohort.

Methods

Using anchor-based methods, we compared clinician or patient-reported changes in eye symptoms (8-point scale) with calculated changes in 5 candidate scales: The NIH eye score, patient-reported global rating of eye symptoms, Lee eye subscale, Ocular Surface Disease Index, and Schirmer test. Change was examined for 333 follow-up visits where both clinician and patient reported eye involvement at the previous visit. Linear mixed models were used to account for within-patient correlation.

Main Outcome Measures

An 8-point scale of clinician or patient-reported symptom change was used as an anchor to measure symptom changes at the follow-up visits.

Results

In serial evaluations, agreement regarding improvement, stability, or worsening between the clinician and patient was fair (weighted kappa = 0.34). Despite only fair agreement between evaluators, all scales except the Schirmer test correlated with both clinician-reported and patient-reported changes in ocular GVHD activity. Among all scales, changes in the NIH eye scores showed the greatest sensitivity to symptom change reported by clinicians or patients.

Conclusions

Our results support the use of the NIH eye score as a sensitive measure of eye symptom changes in clinical trials assessing treatment of chronic GVHD.

Financial Disclosure(s)

The authors have no proprietary or commercial interest in any materials discussed in this article.

Section snippets

Patients and Methods

Patients were enrolled in a multicenter, prospective, observational cohort in which all the measures recommended by the NIH chronic GVHD consensus conference were collected. The rationale and design of the cohort study is described in detail elsewhere.22 One of the specific aims of this cohort study was to validate the various measures for the different organ systems. The study protocol was approved by the institutional review board of each participating center, and all participants or their

Patient Characteristics

Between August 2007 and June 2010, the study enrolled 387 patients, including 14 children (4%). The OSDI and Schirmer tests were not evaluated in children. The median patient age at enrollment was 51 years (range, 2–79 years). The median time from transplant to enrollment was 12.3 months (range, 2.9–39.2 months). The cohort included 209 incident cases (54%) and 178 prevalent cases (46%). Other demographic characteristics of patients are summarized in Table 1 (available at http://aaojournal.org).

Discussion

We tested 3 recommended measures for response in ocular GVHD (the Schirmer test, patient-reported global rating of eye symptoms. and the Lee eye subscale), 1 experimental measure (the OSDI), and the NIH eye score. Our results suggested strong correlations of serial changes in all measurement scales with clinician- and patient-reported symptom changes, except for the Schirmer test. The sensitivity of each scale to symptom changes reported by clinicians or patients varied among the scales.

The NIH

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    Manuscript no. 2011-824.

    This work was supported in part by grants CA118953 and CA18029 from the National Cancer Institute. Y.I. is a recipient of the Banyu Fellowship Program from Banyu Life Science Foundation International and the JSPS Postdoctoral Fellowships for Research Abroad. The sponsor or funding organization had no role in the design or conduct of this research.

    Financial Disclosures: The authors have no proprietary or commercial interest in any of the materials discussed in this article.

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