The study plans to treat at least 60 pediatric participants, male and female, between the ages of 2 months and 17 years inclusive with aGVHD following allogeneic hematopoietic stem cell transplant (HSCT) that has failed to respond to treatment with systemic corticosteroid therapy. Participants may have Grades C and D aGVHD involving the skin, liver and/or gastrointestinal (GI) tract or Grade B aGVHD involving the liver and/or GI tract, with or without concomitant skin disease.
Remestemcel-L will be evaluated in pediatric participants with aGVHD following allogeneic HSCT that has failed to respond to treatment with systemic corticosteroid therapy.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
55
Participants were treated with IV remestemcel-L at a dose of 2 x 10\^6 MSC/kg (actual body weight at screening) twice per week for each of 4 consecutive weeks. Infusions were administered at least 3 days apart and no more than 5 days apart for any infusion.
Children's Hospital Los Angeles
Los Angeles, California, United States
Children's Hospital of Orange County
Orange, California, United States
University of California at San Francisco
San Francisco, California, United States
Children's Hospital Colorado Center for Cancer/Blood Disorders
Aurora, Colorado, United States
Alfred I. DuPont Hospital for Children of the Nemours Foundation
Wilmington, Delaware, United States
Miami Children's Research Institute
Miami, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
University of Mississippi Medical Center
Jackson, Mississippi, United States
Washington University
St Louis, Missouri, United States
...and 10 more locations
Overall Response Rate (ORR) at Day 28 Post Initiation of Therapy
ORR was defined as the percentage of participants who had achieved overall response. Overall response was defined as complete response (CR) plus partial response (PR) as per aGVHD response criteria. CR was defined as resolution of aGVHD in all involved organs. PR was defined as organ improvement of at least 1 stage without worsening of any other organ.
Time frame: Day 28
Overall Survival (OS) Rate at Day 100 Post Initiation of Therapy
Overall survival rate was defined as percentage of participants who survived. OS was defined as the time to death from the start of drug therapy.
Time frame: Day 100
OS Rate at Day 100 Post Initiation of Therapy, Stratified by Responder Status at Day 28
Overall survival rate was defined as percentage of participants who survived. OS was defined as the time to death from the start of drug therapy.
Time frame: Day 100
OS Rate at Day 100 Post Initiation of Therapy, Stratified by Baseline aGVHD Grade
OS rate was defined as percentage of participants who survived. OS was defined as the time to death from the start of drug therapy. Maximum severity of acute GVHD was assessed by using International Bone Marrow Transplant Registry (IBMTR) index. The severity index was defined as: Grade A (skin Stage 1: extent of rash \<25%); Grade B (skin Stage 2: extent of rash 25 to 50% or liver Stage 1 to 2: total bilirubin 34 to 102 micromoles per liter \[mcmol/L\] or intestinal tract Stage 1 to 2: volume of diarrhea 550 to 1500 milliliters per day \[mL/day\]); Grade C (skin Stage 3: extent of rash \> 50% or liver Stage 3: total bilirubin 103 to 255 mcmol/L or intestinal tract Stage 3: volume of diarrhea \>1500 mL/day); Grade D (skin Stage 4: extent of rash bullae or liver Stage 4: total bilirubin \>255 or intestinal tract Stage 4: volume of diarrhea severe pain and ileus).
Time frame: Day 100
OS Rate at Day 100 Post Initiation of Therapy, Stratified by Organ Involvement
OS rate was defined as percentage of participants who survived. OS was defined as the time to death from the start of drug therapy. The data was summarized for organ involvement: skin only, lower GI only, and multi-organ.
Time frame: Day 100
OR Rate at Day 56 and 100 Post Initiation of Therapy
OR rate was defined as the percentage of participants who had achieved overall response. Overall response was defined as CR plus PR as per aGVHD response criteria. CR was defined as resolution of aGVHD in all involved organs. PR was defined as organ improvement of at least 1 stage without worsening of any other organ.
Time frame: Day 56 and Day 100
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