CDI scientist joins NIH group to improve post-stem cell transplant patient evaluation

An international task force of medical experts recently proposed major revisions to the way doctors measure treatment success for a common–and often severe–skin complication of stem cell transplantation.

The National Institutes of Health (NIH) Consensus Project Task Force recently published a report of their refined approach in the journal, Transplantation and Cellular Therapy.

CDI Faculty Member Rachel Rosenstein, M.D., Ph.D., co-authored the report. With her colleagues, she helped present ways to develop better response criteria for clinical trials evaluating 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 affecting about half of all patients on at least a mild basis.

The condition can cause painful or itchy rashes, slow-healing sores, and potentially a progressive, deep-tissue hardening of the skin known as sclerosis.

The report argues today’s assessment standards are too insensitive to capture improvements that are clinically meaningful to patients. Under old criteria from 2014, a patient’s skin could become significantly softer and more flexible, but if the total area affected didn’t cross a broad statistical threshold, the treatment would not be officially deemed successful in clinical trials.

Proposed refinements from 2025 would spur process innovation by introducing a more sensitive and patient-focused approach.

Key changes include:

• Separate scoring rubrics for rash-like (epidermal) and hardening (sclerotic) symptoms of cGVHD, tailoring to their different responses to therapy.
• New clinician-rated scales assessing the size of the affected area, the quality of the skin—such as its softness and tightness—and the qualitative impact of the disease on a patient’s daily functions.

While not specifically included in the new guidelines, more advanced tools such as myotonometry for measuring skin stiffness, and high-frequency ultrasound (HIFU) for better quality imaging, are being evaluated for future use.

As a member of the task force and a physician-scientist, Dr. Rosenstein and co-authors called for the urgent development of biomarkers—molecular signals in the blood or skin—to revolutionize patient care.

“These biological clues could function as an early warning system,” said Dr. Rosenstein, “identifying patients at high risk for severe disease before it fully develops.”

Dr. Rosenstein accompanied her co-authors and presented on biomarker research at meetings associated with the 7th International cGvHD symposium in Vancouver, British Columbia, as well as the 2025 Tandem Meetings–a joint conference of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood and Marrow Transplant Research (CIBMTR)--in Honolulu, Hawaii.

She urged better collection and analysis of protein markers of fibrosis and inflammation, and cellular immunological 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 would be generated using blood and skin collection. Methods could potentially include innovative, minimally invasive techniques—like a surface-level tissue collection process called “skin-tape stripping”---to make cGVHD treatment more precise, predictive, and effective for every patient.

“Our analysis of the current state of cGVHD biomarkers shapes our call to action,” said Dr. Rosenstein. “It illustrates the need for medicine to move beyond naked-eye observation, and to start analyzing and strategizing at a molecular level to truly conquer cGVHD.”

Dr. Rosenstein echoed her colleagues’ argument that more nuanced criteria will better capture real-world improvements in patients, and strengthen the quality of outcomes in clinical trials for treatment.

“I’m thrilled to be a member of the NIH task force,” said Dr. Rosenstein. “It’s by first understanding the improvement of patient care at the individual level that we can discover and develop more effective drugs and therapies for all patients at a population level.”