Post-transplantation Cyclophosphamide as GVHD Prophylaxis After HSCT

Phase 2 Completed 200 enrolled Ivan S Moiseev

Non-randomizedParallel-groupOpen-labelTreatment

Summary

This study evaluates the efficacy of high-dose post-transplantation cyclophosphomide as graft-versus-host disease (GVHD) prophylaxis after allogeneic stem cell transplantation in patients with different risk of GVHD. The risk-adapted strategy involves using single-agent cyclophosphomide in recipients of matched bone marrow graft, and combining cyclophosphomide with tacrolimus and mycophenolate mofetil in recipients of matched peripheral blood stem cells and mismatched bone marrow.

Timeline

Start: 2014-10
Primary completion: 2016-11
Completion: 2017-11

Outcome

Met primary endpoint

paper The study met the primary endpoint to demonstrate equivalent rates of acute GVHD grade II-IV (11%, 17%,19%, P= .46), III-IV (7%, 2%, 6%, P= .41), and moderate and severe chronic GVHD (22%, 11%, 15%, P= .23). PMID 29360184 ↗

Publications

Moiseev IS, Pirogova OV, Alyanski AL, Babenko EV, Gindina TL, Darskaya EI, Slesarchuk OA, Bykova TA, Chukhlovin AB, Pevtcov DE, Bondarenko SN, Afanasyev BV. Risk-adapted GVHD prophylaxis with post-transplantation cyclophosphamide in adults after related, unrelated, and haploidentical transplantations. Eur J Haematol. 2018 May;100(5):395-402. doi: 10.1111/ejh.13030. Epub 2018 Mar 1.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Cyclophosphamide	Other / unclassified	50 mg/kg	Intravenous
Subject	Mycophenolate Mofetil	Small molecule	30 mg/kg	Intravenous
Subject	Mycophenolate Mofetil	Small molecule	45 mg/kg	Intravenous
Subject	Tacrolimus	Small molecule	0.03 mg/kg	—
Background	Busulfan	Small molecule	1 mg/kg	Oral
Background	Fludarabine	Small molecule	30 mg/m2	Intravenous