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, esophagus, 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) |
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|
Organ |
Response measure |
Clinically meaningful change |
Patient-reported outcome |
Clinically meaningful change |
OR, 95% CI, for improvement in PRO predicting NIH response |
p-value |
|
Skin |
NIH Skin score [0-3] |
1 pt |
LSS skin scale [0-100] |
11 pts |
2.16 (1.12-4.16) |
0.021 |
|
|
Sclerotic skin [0-10] |
2 pts |
LSS skin scale [0-100] |
11 pts |
1.66 (0.91-3.01) |
0.098 |
|
|
Sclerotic skin [0-10] |
2 pts |
Skin tightening [0-10] |
2 pts |
2.50 (1.28-4.87) |
0.007 |
|
Joint |
P-ROM [4-25] |
2 pts |
LSS single item joints [0-4] |
1 pt |
1.49 (0.84-2.66) |
0.175 |
|
|
NIH joint score [0-3] |
1 pt |
LSS single item joints [0-4] |
1 pt |
1.26 (0.73-2.20) |
0.408 |
|
Mouth |
Oral mucositis rating scale |
2 pts |
LSS mouth scale [0-100] |
13 pts |
6.36 (2.94-13.74) |
<0.0001 |
|
|
Oral mucositis rating scale |
2 pts |
Mouth sensitivity [0-10] |
2 pts |
4.53 (2.25-9.09) |
<0.0001 |
|
GI |
NIH esophagus score [0-3] |
1 pt |
LSS nutrition scale [0-100] |
7 pts |
1.32 (0.53-3.29) |
0.546 |
|
|
NIH upper GI score [0-3] |
1 pt |
LSS nutrition scale [0-100] |
7 pts |
4.44 (1.74-11.35) |
0.002 |
|
|
NIH lower GI score [0-3] |
1 pt |
LSS nutrition scale [0-100] |
7 pts |
0.91 (0.27-3.05) |
0.878 |
|
Eye |
NIH eye score [0-3] |
1 pt |
LSS eye scale [0-100] |
15 pts |
3.39 (1.98-5.82) |
<0.0001 |
|
|
NIH eye score [0-3] |
1 pt |
Worst eye compliant [0-10] |
2 pts |
2.35 (1.33-4.15) |
0.003 |
|
Lung |
FEV1% |
10% |
LSS lung scale [0-100] |
8 pts |
0.90 (0.43-1.87) |
0.779 |
|
|
NIH lung score [0-3] |
1 pt |
LSS lung scale [0-100] |
8 pts |
2.81 (1.70-4.65) |
<0.0001 |
|
Overall |
NIH responder [CR+PR] vs. nonresponder |
|
LSS summary scale [0-100] |
7 pts |
1.89 (1.17-3.06) |
0.010 |
|
|
NIH responder [CR+PR] vs. nonresponder |
|
Overall cGVHD [0-10] |
2 pts |
2.37 (1.48-3.77) |
<0.001 |
|
|
Overall severity [0-10] |
2 pts |
LSS summary scale [0-100] |
7 pts |
1.84 (1.08-3.11) |
0.024 |
|
|
Overall severity [0-10] |
2 pts |
Overall cGVHD [0-10] |
2 pts |
3.46 (2.17-5.52) |
<0.0001 |
|
Pt, point; LSS, Lee symptom scale; GI, gastrointestinal; P-ROM, photographic range of motion |
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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, esophagus 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.
USE
REZUROCK® (belumosudil) is a prescription medicine used to treat adults and children 12 years of age and older with chronic graft-versus-host disease (chronic GVHD) after you have received at least 2 prior treatments (systemic therapy) and they did not work. It is not known if REZUROCK is safe and effective in children less than 12 years old.
IMPORTANT SAFETY INFORMATION
Before taking REZUROCK, tell your healthcare provider about all of your medical conditions, including if you:
- have kidney or liver problems.
- are pregnant or plan to become pregnant. REZUROCK can harm your unborn baby. If you are able to become pregnant, your healthcare provider will do a pregnancy test before starting treatment with REZUROCK. Tell your healthcare provider if you become pregnant or think you may be pregnant during treatment with REZUROCK.
- Females who can become pregnant should use effective birth control during treatment with REZUROCK and for at least 1 week after the last dose.
- Males with female partners who can become pregnant should use effective birth control during treatment with REZUROCK and for at least 1 week after the last dose.
- are breastfeeding or plan to breastfeed. It is not known if REZUROCK passes into breast milk. Do not breastfeed during treatment with REZUROCK and for at least 1 week after the last dose.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. REZUROCK may affect the way other medicines work, and other medicines may affect the way REZUROCK works.
Know the medicines you take. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.
How should I take REZUROCK?
- Take REZUROCK exactly as your healthcare provider tells you to take it.
- Do not change your dose or stop taking REZUROCK without first talking to your healthcare provider.
- Take REZUROCK 1 time a day with a meal.
- Take REZUROCK at about the same time each day.
- Swallow REZUROCK tablets whole with a glass of water.
- Do not cut, crush, or chew REZUROCK tablets.
- Your healthcare provider will do blood tests to check your liver at least 1 time a month during treatment with REZUROCK.
- If you miss a dose of REZUROCK, take it as soon as you remember on the same day. Take your next dose of REZUROCK at your regular time on the next day. Do not take extra doses of REZUROCK to make up for a missed dose.
- If you take too much REZUROCK, call your healthcare provider or go to the nearest hospital emergency room right away.
What are the possible side effects of REZUROCK? The most common side effects of REZUROCK include:
- infections
- tiredness or weakness
- nausea
- diarrhea
- shortness of breath
- cough
- swelling
- bleeding
- stomach (abdominal) pain
- muscle or bone pain
- headache
- high blood pressure
Your healthcare provider may change your dose of REZUROCK, temporarily stop, or permanently stop treatment with REZUROCK if you have certain side effects.
REZUROCK may affect fertility in males and females. Talk to your healthcare provider if this is a concern for you.
These are not all the possible side effects of REZUROCK. Call your doctor for medical advice about side effects.
Please see full Prescribing Information.
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