PMID- 29403023
OWN - NLM
STAT- MEDLINE
DCOM- 20190715
LR  - 20190715
IS  - 1476-5365 (Electronic)
IS  - 0268-3369 (Linking)
VI  - 53
IP  - 7
DP  - 2018 Jul
TI  - Allogenic peripheral stem cell transplantation from HLA-matched related donors 
      for adult sickle cell disease: remarkable outcomes from a single-center trial.
PG  - 880-890
LID - 10.1038/s41409-018-0111-y [doi]
AB  - Adult patients with sickle cell disease (SCD) are highly susceptible to stem cell 
      transplant complications, including drug toxicity, graft versus host disease 
      (GVHD), and graft rejection due to SCD-related tissue damage, endothelial 
      activation, and inflammation. The scarcity of compatible stem cells for 
      transplantation further limits treatment options, with only 43 cases of adult 
      allogeneic peripheral blood stem cell transplantation (allo-PSCT) from human 
      leukocyte antigen (HLA)-identical sibling donors reported in the international 
      registry for the period 1986-2013. Herein we report remarkable outcomes in a 
      cohort of adult SCD patients who underwent allo-PSCT using a fludarabine (Flu), 
      busulfan (Bu), and anti-T-cell lymphocyte globulin (ATG)-based conditioning 
      regimen in combination with very low dose total body irradiation (TBI), followed 
      by post-transplant cyclophosphamide (Cy) and sirolimus as GVHD prophylaxis. We 
      performed a single-center, retrospective study consisting of 20 consecutive 
      patients (mean age 33.4 years) who underwent allo-PSCT from HLA-matched related 
      donors with a conditioning regimen of Flu 150/Bu 3.2/Cy 29/ATG 30 (Fresenius)/TBI 
      200 between September 2013 and September 2017. Data were validated by an 
      independent data audit group of the affiliated JACIE-accredited transplantation 
      center. All patients experienced a sustained donor cell engraftment. Full donor 
      chimerism (total cell) occurred within 180 days in all patients. Mean duration of 
      follow-up was 13.8 months (range: 0.3-50 months), with 12 (60%) patients 
      completing 12 months. No non-relapse mortality or graft rejection occurred. 
      Successful treatment was achieved without the presence of graft loss, grade 
      III-IV acute GVHD, extensive chronic GVHD, or other major complications. 
      Allo-PSCT in combination with Flu 150/Bu 3.2/Cy 29/ATG 30(Fresenius)/TBI 200- 
      Cy/Sirolimus therapy yielded encouraging outcomes with no mortality and low 
      incidence of GVHD. Further controlled studies will be necessary to compare 
      transplant protocols and long-term outcomes.
FAU - Ozdogu, H
AU  - Ozdogu H
AD  - Department of Hematology and Adana Adult Bone Marrow Transplantation Centre, 
      University of Baskent, Ankara, Turkey.
FAU - Boga, C
AU  - Boga C
AD  - Department of Hematology and Adana Adult Bone Marrow Transplantation Centre, 
      University of Baskent, Ankara, Turkey. drcanboga@hotmail.com.
FAU - Yeral, M
AU  - Yeral M
AD  - Department of Hematology and Adana Adult Bone Marrow Transplantation Centre, 
      University of Baskent, Ankara, Turkey.
FAU - Kozanoglu, I
AU  - Kozanoglu I
AD  - Department of Physiology and Adana Adult Bone Marrow Transplantation Centre, 
      University of Baskent, Ankara, Turkey.
FAU - Gereklioglu, C
AU  - Gereklioglu C
AD  - Department of Family Medicine, University of Baskent, Ankara, Turkey.
FAU - Aytan, P
AU  - Aytan P
AD  - Department of Hematology and Adana Adult Bone Marrow Transplantation Centre, 
      University of Baskent, Ankara, Turkey.
FAU - Kasar, M
AU  - Kasar M
AD  - Department of Immunology, University of Baskent, Ankara, Turkey.
FAU - Asma, S
AU  - Asma S
AD  - Department of Family Medicine, University of Baskent, Ankara, Turkey.
FAU - Buyukkurt, N
AU  - Buyukkurt N
AD  - Department of Hematology and Adana Adult Bone Marrow Transplantation Centre, 
      University of Baskent, Ankara, Turkey.
FAU - Solmaz, S
AU  - Solmaz S
AD  - Department of Hematology and Adana Adult Bone Marrow Transplantation Centre, 
      University of Baskent, Ankara, Turkey.
FAU - Korur, A
AU  - Korur A
AD  - Department of Family Medicine, University of Baskent, Ankara, Turkey.
FAU - Sariturk, C
AU  - Sariturk C
AD  - Medical Statistics, Adana Hospital, University of Baskent, Ankara, Turkey.
LA  - eng
PT  - Journal Article
DEP - 20180205
PL  - England
TA  - Bone Marrow Transplant
JT  - Bone marrow transplantation
JID - 8702459
SB  - IM
MH  - Adult
MH  - Anemia, Sickle Cell/pathology/*therapy
MH  - Female
MH  - Humans
MH  - Male
MH  - Middle Aged
MH  - Peripheral Blood Stem Cell Transplantation/*methods
MH  - Retrospective Studies
MH  - Tissue Donors
MH  - Transplantation, Homologous/*methods
MH  - Young Adult
EDAT- 2018/02/07 06:00
MHDA- 2019/07/16 06:00
CRDT- 2018/02/07 06:00
PHST- 2017/11/29 00:00 [received]
PHST- 2018/01/13 00:00 [accepted]
PHST- 2018/01/11 00:00 [revised]
PHST- 2018/02/07 06:00 [pubmed]
PHST- 2019/07/16 06:00 [medline]
PHST- 2018/02/07 06:00 [entrez]
AID - 10.1038/s41409-018-0111-y [pii]
AID - 10.1038/s41409-018-0111-y [doi]
PST - ppublish
SO  - Bone Marrow Transplant. 2018 Jul;53(7):880-890. doi: 10.1038/s41409-018-0111-y. 
      Epub 2018 Feb 5.