PMID- 27404668
OWN - NLM
STAT- MEDLINE
DCOM- 20170525
LR  - 20181113
IS  - 1097-0142 (Electronic)
IS  - 0008-543X (Print)
IS  - 0008-543X (Linking)
VI  - 122
IP  - 21
DP  - 2016 Nov 15
TI  - Results of a 2-arm, phase 2 clinical trial using post-transplantation 
      cyclophosphamide for the prevention of graft-versus-host disease in 
      haploidentical donor and mismatched unrelated donor hematopoietic stem cell 
      transplantation.
PG  - 3316-3326
LID - 10.1002/cncr.30180 [doi]
AB  - BACKGROUND: High-dose, post-transplantation cyclophosphamide (PTCy) to prevent 
      graft-versus-host disease (GVHD) has improved outcomes in haploidentical (HAPLO) 
      stem cell transplantation (SCT). However, it remains unclear whether this 
      strategy is effective in SCT from 1-antigen human leukocyte antigen 
      (HLA)-mismatched unrelated donors (9/10 MUD) and how the outcomes of these 
      patients compare with those of haploidentical transplantation recipients. 
      METHODS: A parallel, 2-arm, nonrandomized phase 2 clinical trial was conducted of 
      melphalan-based reduced-intensity conditioning with PTCy, tacrolimus, and 
      mycophenolate mofetil to prevent GVHD in patients with high-risk hematologic 
      malignancies who underwent HAPLO (n = 60) or 9/10 MUD (n = 46) SCT. RESULTS: The 
      1-year overall and progression-free survival rates were 70% and 60%, 
      respectively, in the HAPLO arm and 60% and 47%, respectively, in the 9/10 MUD 
      arm. The day +100 cumulative incidence of grade II to IV acute GVHD and grade III 
      to IV acute GVHD was 28% and 3%, respectively, in the HAPLO arm and 33% and 13%, 
      respectively, in the 9/10 MUD arm. The 2-year cumulative incidence of chronic 
      GVHD was 24% in the HAPLO arm and 19% in the 9/10 MUD arm. The 1-year cumulative 
      incidence of nonrelapse mortality was 21% in the HAPLO arm and 31% in the 9/10 
      MUD arm, and the 1-year relapse rate was 19% in the HAPLO arm and 25% in the 9/10 
      MUD arm. CONCLUSIONS: Although this was a nonrandomized study and could not serve 
      as a direct comparison between the 2 groups, the authors conclude that PTCy-based 
      GVHD prophylaxis is effective for both HAPLO and 9/10 MUD SCTs. Prospective 
      randomized trials will be required to compare the efficacies of alternative donor 
      options for patients lacking HLA-matched donors. Cancer 2016;122:3316-3326. (c) 
      2016 American Cancer Society.
CI  - (c) 2016 American Cancer Society.
FAU - Gaballa, Sameh
AU  - Gaballa S
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Ge, Isabell
AU  - Ge I
AD  - University Medical Center Freiburg, Freiburg, Germany.
FAU - El Fakih, Riad
AU  - El Fakih R
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Brammer, Jonathan E
AU  - Brammer JE
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Kongtim, Piyanuch
AU  - Kongtim P
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Tomuleasa, Ciprian
AU  - Tomuleasa C
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Wang, Sa A
AU  - Wang SA
AD  - Department of Hematopathology, The University of Texas MD Anderson Cancer Center, 
      Houston, Texas.
FAU - Lee, Dean
AU  - Lee D
AD  - Division of Pediatrics, The University of Texas MD Anderson Cancer Center, 
      Houston, Texas.
FAU - Petropoulos, Demetrios
AU  - Petropoulos D
AD  - Division of Pediatrics, The University of Texas MD Anderson Cancer Center, 
      Houston, Texas.
FAU - Cao, Kai
AU  - Cao K
AD  - Department of Laboratory Medicine, The University of Texas MD Anderson Cancer 
      Center, Houston, Texas.
FAU - Rondon, Gabriela
AU  - Rondon G
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Chen, Julianne
AU  - Chen J
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Hammerstrom, Aimee
AU  - Hammerstrom A
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Lombardi, Lindsey
AU  - Lombardi L
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Alatrash, Gheath
AU  - Alatrash G
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Korbling, Martin
AU  - Korbling M
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Oran, Betul
AU  - Oran B
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Kebriaei, Partow
AU  - Kebriaei P
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Ahmed, Sairah
AU  - Ahmed S
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Shah, Nina
AU  - Shah N
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Rezvani, Katayoun
AU  - Rezvani K
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Marin, David
AU  - Marin D
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Bashir, Qaiser
AU  - Bashir Q
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Alousi, Amin
AU  - Alousi A
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Nieto, Yago
AU  - Nieto Y
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Qazilbash, Muzaffar
AU  - Qazilbash M
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Hosing, Chitra
AU  - Hosing C
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Popat, Uday
AU  - Popat U
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Shpall, Elizabeth J
AU  - Shpall EJ
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Khouri, Issa
AU  - Khouri I
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Champlin, Richard E
AU  - Champlin RE
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas.
FAU - Ciurea, Stefan O
AU  - Ciurea SO
AD  - Department of Stem Cell Transplantation and Cellular Therapy, The University of 
      Texas MD Anderson Cancer Center, Houston, Texas. sciurea@mdanderson.org.
LA  - eng
GR  - P30 CA016672/CA/NCI NIH HHS/United States
PT  - Clinical Trial, Phase II
PT  - Comparative Study
PT  - Journal Article
DEP - 20160712
PL  - United States
TA  - Cancer
JT  - Cancer
JID - 0374236
RN  - 0 (Immunosuppressive Agents)
RN  - 8N3DW7272P (Cyclophosphamide)
RN  - HU9DX48N0T (Mycophenolic Acid)
RN  - WM0HAQ4WNM (Tacrolimus)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Aged
MH  - Cyclophosphamide/*therapeutic use
MH  - Drug Therapy, Combination
MH  - Feasibility Studies
MH  - Female
MH  - Follow-Up Studies
MH  - Graft vs Host Disease/etiology/*prevention & control
MH  - Hematologic Neoplasms/*therapy
MH  - Hematopoietic Stem Cell Transplantation/*adverse effects
MH  - Histocompatibility Testing
MH  - Humans
MH  - Immunosuppressive Agents/*therapeutic use
MH  - Male
MH  - Middle Aged
MH  - Mycophenolic Acid/therapeutic use
MH  - Prognosis
MH  - Prospective Studies
MH  - Tacrolimus/therapeutic use
MH  - Transplantation Conditioning
MH  - Transplantation, Homologous
MH  - *Unrelated Donors
MH  - Young Adult
PMC - PMC5459484
MID - NIHMS860575
OTO - NOTNLM
OT  - 9/10 matched unrelated donors
OT  - graft-versus-host disease
OT  - haploidentical transplantation
OT  - hematologic malignancies
OT  - post-transplantation cyclophosphamide
COIS- CONFLICT OF INTEREST DISCLOSURES Dean Lee reports licensing fees from Ziopharm 
      Oncology and Intrexon Corporation; personal fees from Ziopharm Oncology, Courier 
      Therapeutics, Intellia Therapeutics, Shire, and Sanofi Oncology; and holds 
      ownership and is a board member of Therapeutics, all outside the submitted work. 
      Qaiser Bashir reports research funding from Celgene and Takeda and serves on the 
      Takeda and Spectrum Pharmaceuticals advisory boards, all outside the submitted 
      work. Chitra Hosing reports a grant for clinical trials from Celgene, Inc; and 
      personal fees from Sanofi, Aviara Pharmaceuticals, Seattle Genetics, and Cardinal 
      Health, all outside the submitted work.
EDAT- 2016/10/21 06:00
MHDA- 2017/05/26 06:00
PMCR- 2017/11/15
CRDT- 2016/07/13 06:00
PHST- 2016/03/12 00:00 [received]
PHST- 2016/05/17 00:00 [revised]
PHST- 2016/05/24 00:00 [accepted]
PHST- 2016/10/21 06:00 [pubmed]
PHST- 2017/05/26 06:00 [medline]
PHST- 2016/07/13 06:00 [entrez]
PHST- 2017/11/15 00:00 [pmc-release]
AID - 10.1002/cncr.30180 [doi]
PST - ppublish
SO  - Cancer. 2016 Nov 15;122(21):3316-3326. doi: 10.1002/cncr.30180. Epub 2016 Jul 12.