Original articleValidation of Measurement Scales in Ocular Graft-versus-Host Disease
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
References (37)
- et al.
Impact of chronic graft-versus-host disease on the health status of hematopoietic cell transplantation survivors: a report from the Bone Marrow Transplant Survivor Study
Blood
(2006) - et al.
Quality of life after allogeneic hematopoietic cell transplantation
Blood
(2009) - et al.
Patient reported quality of life is associated with severity of chronic graft-versus-host disease as measured by NIH criteria: report on baseline data from the Chronic GVHD Consortium
Blood
(2011) - et al.
Chronic graft-versus-host disease
Biol Blood Marrow Transplant
(2003) - et al.
Comparison of chronic graft-versus-host disease after transplantation of peripheral blood stem cells versus bone marrow in allogeneic recipients: long-term follow-up of a randomized trial
Blood
(2002) - et al.
Ancillary therapy and supportive care of chronic graft-versus-host disease: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: VAncillary Therapy and Supportive Care Working Group report
Biol Blood Marrow Transplant
(2006) - et al.
Consensus conference on clinical practice in chronic graft-versus-host disease (GVHD): first-line and topical treatment of chronic GVHD
Biol Blood Marrow Transplant
(2010) - et al.
National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: IDiagnosis and Staging Working Group report
Biol Blood Marrow Transplant
(2005) - et al.
Measuring therapeutic response in chronic graft-versus-host disease: National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: IVResponse Criteria Working Group report
Biol Blood Marrow Transplant
(2006) - et al.
Incorporating the patient's perspective into drug development and communication: an ad hoc task force report of the Patient-Reported Outcomes (PRO) Harmonization Group meeting at the Food and Drug Administration, February 16, 2001
Value Health
(2003)
Development and validation of a scale to measure symptoms of chronic graft-versus-host disease
Biol Blood Marrow Transplant
Methods to explain the clinical significance of health status measures
Mayo Clin Proc
Use of fluid-ventilated, gas-permeable scleral lens for management of severe keratoconjunctivitis sicca secondary to chronic graft-versus-host disease
Biol Blood Marrow Transplant
A multicenter pilot evaluation of the National Institutes of Health chronic graft-versus-host disease (cGVHD) therapeutic response measures: feasibility, interrater reliability, and minimum detectable change
Biol Blood Marrow Transplant
Thomas' hematopoietic cell transplantation
Long-term survival and late deaths after allogeneic bone marrow transplantation
N Engl J Med
Recovery after stem-cell transplantation for hematologic diseases
J Clin Oncol
Long-term medical outcomes and quality-of-life assessment of patients with chronic myeloid leukemia followed at least 10 years after allogeneic bone marrow transplantation
J Clin Oncol
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Efficacy and Safety of 1% Progesterone Gel to the Forehead for Ocular Chronic Graft-versus-Host Disease
2021, Transplantation and Cellular TherapySystematic Review of Patient-Reported Outcome Measures in Graft-versus-Host Disease
2020, Biology of Blood and Marrow TransplantationCitation Excerpt :Curtis et al. [85] (n = 210; United States) reported a statistically significantly higher score in patients with ocular cGVHD compared with those without (P < .001) and patients with active disease compared with those with nonactive disease (P = .0023). Inamoto et al. [72] (n = 387; United States) reported that the score showed slight correlation with the clinician-assessed NIH ocular score (kappa = .18). This study also reported that the score was reasonably responsive to clinician- (P = .03) and patient-reported symptom change (P < .001).
Organ Changes Associated with Provider-Assessed Responses in Patients with Chronic Graft-versus-Host Disease
2019, Biology of Blood and Marrow TransplantationCitation Excerpt :Strict application of these definitions means that anyone who has ever required esophageal dilation cannot have a score of 2 or less, and once punctual plugs are placed, an eye score of 1 or less is impossible, even if symptoms resolve. Also, the esophageal and upper gastrointestinal tract severity scores have never been empirically validated according to provider and patient assessments, as has been done for skin, mouth, eye, gastrointestinal, liver, and joint scores [14–19]. Second, accurate response assessment requires equivalent awareness of current and baseline organ measures.
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.