A patient-reported outcome (PRO) measure has been developed for patients with chronic graft-versus-host disease (cGVHD)-associated sclerosis (cGVHD-Scl), with the hope that it will lead to more meaningful clinical trials for the condition and for which more effective therapies are sorely needed.
Because objective measures like CT scans and organ function frequently do not account for how a patient is feeling, PROs are a vital element in determining how well treatments work, said Stephanie Lee, MD, MPH, professor of hematology at Fred Hutchinson Cancer Center in Seattle and senior author on a recent paper on the new tool, known as the Lee Symptom Scale-Skin Sclerosis. The findings from Dr. Lee and colleagues were published in Blood.
An existing PRO for cGVHD was not optimal for gauging responses for sclerotic GVHD, she said.
“It’s not detailed enough or specific enough to this particular form of GVHD to do a great job of capturing it,” Dr. Lee said. “We wanted to set the stage for other trials looking at this complication, and to do that, we felt we needed a better, more precise instrument.”
After an allogeneic hematopoietic cell transplant, up to half of patients suffer from cGVHD, and sclerosis is a particularly problematic form. Patients experience pain, burning, tingling, and aching, and the thickened skin that develops can open up when stretched, leading to sores, said first author Emily Baumrin, MD, MSCE, assistant professor of dermatology at the University of Pennsylvania.
“A major problem with cGVHD-Scl is that once the skin and fascia become fibrotic, it is very hard to reverse with our currently available therapies,” Dr. Baumrin said. “The symptoms and functional limitations often last for months to years, which can be debilitating for patients. Many patients feel frustration, anger, and hopelessness regarding their condition, and better therapies are desperately needed.”
In a round of interviews with patients, chosen to capture the wide array of cGVHD-Scl manifestations, researchers continued interviewing until no new themes emerged, eventually interviewing 36 patients. Characteristics and symptoms of the disease were grouped into five categories: skin changes, symptoms, emotional and social role functioning, mobility restrictions, and activity limitations.
The draft of the PRO instrument was evaluated in interviews with 25 of the patients, and it received positive feedback, with only minor addition or elimination of items. One item added was “twist at the waist,” which is an indication of the kind of detail the instrument wants to capture, Dr. Lee said.
“That’s something that a doctor may or may not talk to the patient about, it may or may not be a huge deal to the patient or not involved at all,” she said. “But if it is an issue, and the patient’s not able to do it, just imagine what your life would be like if you can’t turn at your waist or bend down or pick up something from the floor.”
Dr. Lee said the instrument will be included in clinical trials at her center, but it still needs to be applied to many more patients and assessed for its sensitivity to change to be sure it is picking up on changes in treatment.
“By its design,” Dr. Baumrin said, “we expect this tool to improve on existing PROs in measuring response to treatment over time. Since it is often difficult for the clinician to assess sclerosis change over time, this PRO measure will enhance our ability to determine whether treatments are working in patient care and research in a way that is meaningful to patients.”
Limitations of the study included the small cohort size and the use of a sample that was drawn from a relatively homogenous population of outpatients at a single center, leading to underrepresentation of racial and ethnic subgroups.
Any conflicts of interest declared by the authors can be found in the original article.
Reference
Baumrin E, Pidala JA, Mitchell S, et al. Development of the Lee Symptom Scale-Skin Sclerosis for chronic GVHD-associated sclerosis [published online ahead of print, 2025 Jan. 14]. Blood. doi: 10.1182/blood.2024027334.
