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Biol Blood Marrow Transplant. 2015 Apr;21(4):720-8. doi: 10.1016/j.bbmt.2014.12.025. Epub 2014 Dec 30.

Single and multiple dose MultiStem (multipotent adult progenitor cell) therapy prophylaxis of acute graft-versus-host disease in myeloablative allogeneic hematopoietic cell transplantation: a phase 1 trial.

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Knight Cancer Institute, Center for Hematologic Malignancies, Oregon Health and Science University, Portland, Oregon. Electronic address:
Department of Microbiology & Immunology, Katholieke Universiteit Leuven, Leuven, Belgium.
Adult Blood & Marrow Stem Cell Transplant Program, Texas Transplant Institute, San Antonio, Texas.
University of Pennsylvania, Philadelphia, Pennsylvania.
Internal Medicine, Bone Marrow Transplant Program, Mayo Clinic Hospital, Phoenix, Arizona.
Internal Medicine, Bone Marrow Transplant Program, Ohio State University, Columbus, Ohio.
Knight Cancer Institute, Center for Hematologic Malignancies, Oregon Health and Science University, Portland, Oregon.
Athersys, Inc., Cleveland, Ohio.
University Hospitals Case Medical Center, Case Western Reserve University, Cleveland, Ohio.


We conducted a multicenter, phase 1 dose escalation study evaluating the safety of the allogeneic multipotent adult progenitor cell (MAPC, MultiStem, Athersys, Inc., Cleveland, OH) stromal product administered as an adjunct therapy to 36 patients after myeloablative allogeneic hematopoietic cell transplantation (HCT). Patients received increasing doses of MAPC (1, 5, or 10 million cells per kilogram recipient weight) as a single i.v. dose on day +2 after HCT (n = 18), or once weekly for up to 5 doses (1 or 5 million cells per kilogram; n = 18). Infusional and regimen-related toxicities were assessed for 30 days after the last MAPC dose. Of 36 allogeneic HCT donors (17 related and 19 unrelated), 35 were 6/6 HLA matched. MAPC infusions were well tolerated without associated infusional toxicity, graft failure, or increased incidence of infection. Median times to neutrophil (n = 36) and platelet (n = 31) engraftment were 15 (range, 11 to 25) and 16 (range, 11 to 41) days, respectively. The overall cumulative incidences of grades II to IV and III and IV acute graft-versus-host disease (GVHD) at day 100 were 37% and 14%, respectively (n = 36). In the group that received the highest single MAPC dose (10 million cells/kg), day 100 incidence of grade II to IV GVHD was 11.1% (1 of 9) with no observed cases of grade III and IV GVHD. We found no evidence for MHC class II allogeneic antibody induction, although some patients showed an increase in serum anticlass I titers compared with baseline. MAPC contribution to blood chimerism was negligible. These phase I data support the safety of stromal stem cell therapy and suggest that MAPC should be tested prospectively as a novel therapeutic option for GVHD prophylaxis after HCT.


Graft-versus-host disease; Mesenchymal stromal cells; Multipotent adult progenitor cells; Myeloablative allogeneic hematopoietic cell transplantation; Phase 1

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