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The anti-CTLA-4 monoclonal antibody, ipilimumab, has been used to effectively improve overall survival in two phase III studies involving patients with advanced melanoma1. Following on from the success in melanoma, Davids M.S, from the Dana-Farber Cancer Institute et al., hypothesized that immune checkpoint blockade, established by targeting CTLA-4 with ipilimumab, may induce a graft-versus-tumor effect in patients with relapse after allogeneic HSCT and lead to a clinical response.2
Davids MS, et al., conducted a phase 1/1b multicenter, open-label, investigator-initiated study to determine the MTD, the safety and efficacy of ipilimumab in patients with relapsed hematologic cancer (12 AML patients out of 28) after allogeneic hematopoietic stem-cell transplantation (HSCT). The results were published in N Engl J Med in July 2016.
The trial was conducted in accordance with the Declaration of Helsinki and ICH-GCP guidelines and was registered at ClinicalTrials.gov (NCT01822509).
These data warrant the further investigation of the use of ipilimumab for patients with relapsed hematologic malignancies after transplantation, however, monitoring for immune-related adverse events is required.
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