An international panel of medical experts recently proposed major revisions to how doctors measure the success of treatment for a common – and often serious – skin complication of stem cell transplants.
The National Institutes of Health (NIH) Consensus Project Task Force recently published a report of their sophisticated approach in the journal, Transplantation and Cell Therapy.
CDI faculty member Rachel Rosenstein, MD, Ph.D., co-authored the report. With her colleagues, she helped present ways to develop better response criteria for clinical trials that assess the impact of treatment on skin involvement in graft-versus-host disease.
Cutaneous chronic graft-versus-host disease (cGVHD) occurs when cells derived from stem cell donor immune cells attack the recipient’s skin. This is a debilitating complication that affects about half of patients on at least a mild basis.
The condition can cause painful or itchy rashes, slow-healing sores, and possibly a progressive, deep hardening of the skin known as scleroderma.
The report argues that current assessment standards are too biased to capture improvements that are clinically meaningful to patients. Under old criteria from 2014, a patient’s skin could become significantly smoother and more flexible, but if the total affected area did not exceed a broad statistical threshold, the treatment would not be considered officially successful in clinical trials.
The proposed improvements from 2025 will stimulate innovation in the process by introducing a more sensitive and patient-focused approach.
Key changes include:
- Separate scoring rubrics for rash-like (epidermal) and sclerosing (sclerotic) symptoms of cGVHD, adapted to their different responses to treatment.
- New clinician rating scales that assess the size of the affected area, the quality of the skin – such as its softness and firmness – and the qualitative impact of the disease on the patient’s daily activities.
Although not specifically included in the new guidelines, more advanced tools such as myotonometry to measure skin stiffness and high-frequency ultrasound (HIFU) for better imaging quality are being evaluated for future use.
As a member of the task force and as a physician-scientist, Dr. Rosenstein and his co-authors called for the urgent development of biomarkers—molecular signals in the blood or skin—to revolutionize patient care.
These biological markers could act as an early warning system, identifying patients at high risk for serious disease before it is fully developed.”
Dr. Rachel Rosenstein, MD, Ph.D., CDI Faculty Member
Dr. Rosenstein accompanied her co-authors and presented the biomarker research at meetings related to the 7th cGvHD International Symposium in Vancouver, British Columbia, as well as the Tandem 2025 Meetings – a joint conference of the American Society for Transplantation and Cell Therapy (ASTCT) and the BTR-International Research Center (ASTCT) in Honolulu, Hawaii.
He urged better collection and analysis of protein markers of fibrosis and inflammation, and cellular immune markers such as T-cells that have all been identified in research but are not yet collected as standard clinical practice during treatment.
Such data will be generated using blood and skin collection. Methods could potentially include innovative, minimally invasive techniques—such as a surface-level tissue harvesting procedure called “skin strip stripping”—to make cGVHD treatment more precise, predictive and effective for each patient.
“Our analysis of the current state of cGVHD biomarkers forms our call to action,” said Dr. Rosenstein. “It demonstrates the need for medicine to move beyond observation with the naked eye and begin to analyze and strategize at the molecular level to truly conquer cGVHD.”
Dr. Rosenstein echoed her colleagues’ argument that more nuanced criteria would better capture true improvements in patients and enhance the quality of results in clinical trials for treatment.
“I am thrilled to be part of the NIH task force,” said Dr. Rosenstein. “By first understanding how to improve patient care at the individual level we can discover and develop more effective drugs and treatments for all patients at the population level.”
Source:
Journal Reference:
