Immunotherapy shows clinical benefit in relapsed transplant recipients

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A multicenter phase 1 trial of the immune checkpoint blocker ipilimumab found clinical benefit in nearly half of blood cancer patients who had relapsed following allogeneic stem cell transplantation, according to investigators from Dana-Farber Cancer Institute, who developed and lead the study.

The study reported at the American Society of Hematology annual meeting is the first in which ipilimumab was given in multiple doses over an extended time period, the researchers said.

At a median follow-up time of six months, "We have seen less toxicity than expected and a strong efficacy signal, and that’s very encouraging in a population that does not have many therapeutic options," said Matthew S. Davids, MD, medical oncologist at Dana-Farber, the first author on the abstract. Robert J. Soiffer, MD, chief of the Division of Hematologic Malignancies at Dana-Farber is the senior author.

The 13 patients in the study had been treated for a variety of hematological cancers and had suffered relapse a median of 19 months following transplant from a related or unrelated donor. Ipilimumab, which blocks the CTLA4 immune checkpoint to augment the patient’s immune response to the cancer, was given every three weeks for four induction cycles, then every 12 weeks for up to a year.


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