Media Update: Rezurock® (belumosudil) patient-reported outcomes correlated with clinical response in chronic graft-versus-host disease

Rezurock® (belumosudil) patient-reported outcomes correlated with clinical response in chronic graft-versus-host disease

  • Results demonstrate strong, but not universal, correlation between organ clinical responses and clinically meaningful improvements in patient-reported outcomes from a pooled analysis of ROCKstar and KD025-208 clinical trials

Paris, April 21, 2022. Results from a pooled analysis of Rezurock® (belumosudil) for the treatment of chronic graft-versus-host disease (cGVHD) show certain organ clinical responses correlated with clinically meaningful changes in patient-reported outcomes (PROs). Kadmon, a Sanofi Company, will present the results at the 2022 American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR) Tandem Meetings in Salt Lake City, Utah.

Jonathan Ieyoub
Head of Sanofi US Rezurock Medical
“These Rezurock data reinforce the importance of patient-reported outcome measures by showing their correlation to clinical response. Knowing the impact that chronic graft-versus-host disease has on patients, the results add support for use of patient-reported outcomes to assess responses in clinical care.”

The pooled analysis of the ROCKstar (KD025-213) and KD025-208 studies demonstrates a very strong, but not universal, correlation between organ clinical response measures and clinically meaningful changes in PROs in patients treated with Rezurock. In particular, the clinically meaningful changes in PROs were reported in the skin, mouth, eye, upper GI, lung (clinical lung scale score), and overall measures. These data support use of PROs for response assessment in cGVHD clinical trials and patient care to help capture the patient’s perspective on cGVHD disease activity.

About the Pooled Analysis

The pooled analysis of clinical and PRO data included 170 (91.3%) patients enrolled in two Rezurock studies (n=132, KD025-213; n=54, KD025-208) who had baseline PROs, at least one follow-up PRO, and at least one response assessment. The median age was 54.5 years, 98 (58%) were men, the median number of prior treatments was 3, 70% had severe cGVHD, and the median duration of follow-up at the time of data cut-off was 15 months (range, 0.6-44 months).

Organ responses, defined as complete responses (CR) and partial responses (PR), were 35% (48/135) in skin, 68% (84/124) in joints, 57% (56/99) in mouth, 41% (52/128) in eyes, 73% (19/26) in upper GI, 87% (13/15) in lower GI, 23% (14/61) in lungs, and 77% (130/170) overall. For all organs except for joints, oesophagus, and lower GI there was at least one PRO that was statistically correlated at p<0.05 with National Institutes of Health (NIH) organ response. NIH clinical lung response, but not the lung response based on FEV1% change, was associated with the lung PRO.

Correlation of NIH organ response (CR+PR) with clinically meaningful patient-reported outcomes (PROs)
OrganResponse measureClinically meaningful changePatient-reported outcomeClinically meaningful changeOR, 95% CI, for improvement in PRO predicting NIH responsep-value
SkinNIH Skin score 0-31 ptLSS skin scale 0-10011 pts2.16 (1.12-4.16)0.021
 Sclerotic skin 0-102 ptsLSS skin scale 0-10011 pts1.66 (0.91-3.01)0.098
 Sclerotic skin 0-102 ptsSkin tightening 0-102 pts2.50 (1.28-4.87)0.007
JointP-ROM 4-252 ptsLSS single item joints 0-41 pt1.49 (0.84-2.66)0.175
 NIH joint score 0-31 ptLSS single item joints 0-41 pt1.26 (0.73-2.20)0.408
MouthOral mucositis rating scale2 ptsLSS mouth scale 0-10013 pts6.36 (2.94-13.74)<0.0001
 Oral mucositis rating scale2 ptsMouth sensitivity 0-102 pts4.53 (2.25-9.09)<0.0001
GINIH esophagus score 0-31 ptLSS nutrition scale 0-1007 pts1.32 (0.53-3.29)0.546
 NIH upper GI score 0-31 ptLSS nutrition scale 0-1007 pts4.44 (1.74-11.35)0.002
 NIH lower GI score 0-31 ptLSS nutrition scale 0-1007 pts0.91 (0.27-3.05)0.878
EyeNIH eye score 0-31 ptLSS eye scale 0-10015 pts3.39 (1.98-5.82)<0.0001
 NIH eye score 0-31 ptWorst eye compliant 0-102 pts2.35 (1.33-4.15)0.003
LungFEV1%10%LSS lung scale 0-1008 pts0.90 (0.43-1.87)0.779
 NIH lung score 0-31 ptLSS lung scale 0-1008 pts2.81 (1.70-4.65)<0.0001
OverallNIH responder CR+PR vs. nonresponder LSS summary scale 0-1007 pts1.89 (1.17-3.06)0.010
 NIH responder CR+PR vs. nonresponder Overall cGVHD 0-102 pts2.37 (1.48-3.77)<0.001
 Overall severity 0-102 ptsLSS summary scale 0-1007 pts1.84 (1.08-3.11)0.024
 Overall severity 0-102 ptsOverall cGVHD 0-102 pts3.46 (2.17-5.52)<0.0001
Pt, point; LSS, Lee symptom scale; GI, gastrointestinal; P-ROM, photographic range of motion

About cGVHD

cGVHD is a complication that can occur following allogeneic stem cell transplantation, resulting in significant morbidity and mortality. In cGVHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, oesophagus and gastrointestinal tract. Approximately 14,000 patients in the United States are living with cGVHD.

About Rezurock® (belumosudil)

Rezurock® (belumosudil) is the first and only approved therapy inhibiting Rho-associated coiled-coil kinase 2 (ROCK2). Rezurock is approved in the United States for the treatment of adult and pediatric patients 12 years and older with cGVHD after failure of at least two prior lines of systemic therapy. For more information, visit www.Rezurock.com.

About Sanofi
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
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