An observational study in Acute Graft-versus-host Disease, sponsored by IRCCS Burlo Garofolo. Completed at 1 site in Italy. Open to participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2023-04-24.
Sponsored by IRCCS Burlo Garofolo · Observational
Despite significant progress in overall survival and event-free survival in Pediatric Hematopoietic Stem Cell Transplant (HSCT), therapeutic options for graft-versus-host disease control remain limited, particularly in steroid-refractory patients. Several strategies have been proposed in the last 20 years but so far, the results have been inconclusive, complicated by the small population afflicted, inconsistent treatment schedules, different disease classifications and diagnosis methods. The number of studies concerning pediatric patients are even smaller. First line therapy for acute graft-versus-host disease (aGVHD) is steroid treatment that achieve partial or complete remission of the disease in a variable percentage of cases (40-60%), depending mainly to severity of GVHD and number of organ involvement, with hepatic and gastrointestinal GVHD particularly refractory to steroid treatment. For second line therapy there is no a standardized strategy with a great variety of immunosuppressive treatment without a real superiority of a drug in comparison to another. Steroid refractory acute GVHD is therefore one of the most important challenges in HSCT field. One of the more promising routes, based on published data and clinical experience, is the off-label use of Infliximab, an anti-Tumor Necrosis Factor α drug (already approved for many rheumatologic and autoimmune diseases) administered as a second line treatment in patients with steroid-refractory aGVHD at the standardized dosage of 10 mg/kg, although limited evidence has been published to validate this subscription. Biological pattern that could explain susceptibly of GVHD to infliximab treatment could lie in physiopathology of acute gastrointestinal GVHD that may resemble ulcerative rectocolitis. In this case, relation to Therapeutic Drug Monitoring (TDM) and Tumor Necrosis Factor α (TNFα) levels could be critical in monitoring the efficacy of the drug and need of further doses. Limited published data and clinical experience show that Infliximab may be able to further control symptoms and inflammatory response in a promising percentage of treated patients, although some have no benefit from the treatment. The aim of this study is to analyze the role of TNFα concentration in aGVHD, its levels fluctuation and clinical response of GVHD to Infliximab treatment in steroid-refractory pediatric patients.
806 studies on the registry are indexed under Graft vs Host Disease; 138 are open to participants now.
This study's enrollment of 28 is below the median of 94 across 102 observational studies indexed under Graft vs Host Disease.
Browse Graft vs Host Disease studies →IRCCS Burlo Garofolo is the lead sponsor of 87 studies on the registry; 32 are open to participants now.
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Patients with steroid-refractory aGVHD and treated with infliximab
Exclusion Criteria:
Correlation between TNFα plasmatic concentration and serum infliximab levels
TNFα levels and infliximab concentration will be measured in peripheral blood sample (serum)
Time frame: At day 56 after starting infliximab treatment
Correlation between TNFα concentration and serum infliximab levels
TNFα levels and infliximab concentration will be measured in peripheral blood sample (serum)
Time frame: At day 7 after starting infliximab treatment
Association between Baseline TNFα concentration and aGVHD overall severity
TNFα levels will be measured in peripheral blood sample (serum)
Time frame: Before starting infliximab treatment
Number of patients who achieved a significant drop of TNFα concentration after infliximab treatment
TNFα levels will be measured in peripheral blood sample (serum)
Time frame: At day 7 after starting infliximab treatment
Number of patients who achieved a significant drop of TNFα concentration after infliximab treatment
TNFα levels will be measured in peripheral blood sample (serum)
Time frame: At day 14 after starting infliximab treatment
Number of patients who achieved a significant drop of TNFα concentration after infliximab treatment
TNFα levels will be measured in peripheral blood sample (serum)
Time frame: At day 28 after starting infliximab treatment
Number of patients who achieved a significant drop of TNFα concentration after infliximab treatment
TNFα levels will be measured in peripheral blood sample (serum)
Time frame: At day 42 after starting infliximab treatment
Number of patients who achieved a significant drop of TNFα concentration after infliximab treatment
TNFα levels will be measured in peripheral blood sample (serum)
Time frame: At day 56 after starting infliximab treatment
Response to infliximab treatment for aGVHD
Number of patients who had Complete Response (CR), Partial Response (PR) or Non-Response (NR). Response is defined as complete resolution of GVHD symptoms and normalization of the biochemical parameters of inflammation.
Time frame: At day 7 after starting infliximab treatment
Response to infliximab treatment for aGVHD
Number of patients who had Complete Response (CR), Partial Response (PR) or Non-Response (NR). Response is defined as complete resolution of GVHD symptoms and normalization of the biochemical parameters of inflammation.
Time frame: At day 14 after starting infliximab treatment
Response to infliximab treatment for aGVHD
Number of patients who had Complete Response (CR), Partial Response (PR) or Non-Response (NR). Response is defined as complete resolution of GVHD symptoms and normalization of the biochemical parameters of inflammation.
Time frame: At day 28 after starting infliximab treatment
Response to infliximab treatment for aGVHD
Number of patients who had Complete Response (CR), Partial Response (PR) or Non-Response (NR). Response is defined as complete resolution of GVHD symptoms and normalization of the biochemical parameters of inflammation.
Time frame: At day 42 after starting infliximab treatment
Response to infliximab treatment for aGVHD
Number of patients who had Complete Response (CR), Partial Response (PR) or Non-Response (NR). Response is defined as complete resolution of GVHD symptoms and normalization of the biochemical parameters of inflammation.
Time frame: At day 56 after starting infliximab treatment
Infliximab serum concentration in patients with clinical CR, PR, NR.
Infliximab concentration will be measured in peripheral blood sample (serum). Response is defined as complete resolution of GVHD symptoms and normalization of the biochemical parameters of inflammation.
Time frame: At day 7 after starting infliximab treatment
Infliximab serum concentration in patients with clinical CR, PR, NR.
Infliximab concentration will be measured in peripheral blood sample (serum). Response is defined as complete resolution of GVHD symptoms and normalization of the biochemical parameters of inflammation.
Time frame: At day 14 after starting infliximab treatment
Infliximab serum concentration in patients with clinical CR, PR, NR.
Infliximab concentration will be measured in peripheral blood sample (serum). Response is defined as complete resolution of GVHD symptoms and normalization of the biochemical parameters of inflammation.
Time frame: At day 28 after starting infliximab treatment
Infliximab serum concentration in patients with clinical CR, PR, NR.
Infliximab concentration will be measured in peripheral blood sample (serum). Response is defined as complete resolution of GVHD symptoms and normalization of the biochemical parameters of inflammation.
Time frame: At day 42 after starting infliximab treatment
Infliximab serum concentration in patients with clinical CR, PR, NR.
Infliximab concentration will be measured in peripheral blood sample (serum). Response is defined as complete resolution of GVHD symptoms and normalization of the biochemical parameters of inflammation.
Time frame: At day 56 after starting infliximab treatment
Percentage of infection during follow-up
Viral reactivation (Cytomegalovirus and Epstein-Barr virus), bacterial and fungal infection will be evaluated by medical records
Time frame: At 6 months after starting infliximab treatment
Percentage of infection during follow-up
Viral reactivation (Cytomegalovirus and Epstein-Barr virus), bacterial and fungal infection will be evaluated by medical records
Time frame: At 12 months after starting infliximab treatment
Percentage of chronic GVHD
Evaluated by medical records
Time frame: At 6 months after starting infliximab treatment
Percentage of chronic GVHD
Evaluated by medical records
Time frame: At 12 months after starting infliximab treatment
Percentage of relapse
Evaluated by medical records
Time frame: At 6 months after starting infliximab treatment
Percentage of relapse
Evaluated by medical records
Time frame: At 12 months after starting infliximab treatment
Transplant-related mortality
Evaluated by medical records
Time frame: At 6 months after starting infliximab treatment
Overall survival
Evaluated by medical records
Time frame: At 6 months after starting infliximab treatment
Transplant-related mortality
Evaluated by medical records
Time frame: At 12 months after starting infliximab treatment
Overall survival
Evaluated by medical records
Time frame: At 12 months after starting infliximab treatment
This study is completed, as verified in Apr 2023. You cannot join it, but the record below documents what was studied.
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IRCCS Burlo Garofolo