PMID- 27715384
OWN - NLM
STAT- MEDLINE
DCOM- 20170324
LR  - 20220331
IS  - 1521-0669 (Electronic)
IS  - 0888-0018 (Print)
IS  - 0888-0018 (Linking)
VI  - 33
IP  - 6
DP  - 2016 Sep
TI  - Allogeneic hematopoietic stem cell transplantation for nonmalignant hematologic 
      disorders using chemotherapy-only cytoreductive regimens and T-cell-depleted 
      grafts from human leukocyte antigen-matched or -mismatched donors.
PG  - 347-358
AB  - Nonmalignant hematologic disorders (NMHD) of childhood comprise a variety of 
      disorders, including acquired severe aplastic anemia and inherited marrow failure 
      syndromes. Patients with high-risk NMHD without matched related donors fare 
      poorly with allogeneic hematopoietic alternative donor stem cell transplantation 
      (allo-HSCT) and are at high risk for developing graft-versus-host disease 
      following unmodified grafts. The authors retrospectively analyzed data on 18 
      patients affected by NMHD, lacking a human leukocyte antigen (HLA)-identical 
      sibling donor, who underwent an alternative donor allo-HSCT at their institution 
      between April 2005 and May 2013. Fifty percent of the patients had received prior 
      immunosuppressive therapy, 72% had a history of infections, and 56% were 
      transfusion dependent at the time of transplant. Cytoreduction included a 
      combination of 3 of 5 agents: fludarabine, melphalan, thiotepa, busulfan, and 
      cyclophosphamide. Grafts were T-cell depleted. All evaluable patients engrafted. 
      Five died of transplant complications. The cumulative incidence of 
      graft-versus-host disease was 6%. No patient had recurrence of disease. Five-year 
      overall survival was 77%. Age at transplant <6 years was strongly associated with 
      better survival. Based on these results, transplant with chemotherapy-only 
      cytoreductive regimens and T-cell-depleted stem cell transplants could be 
      recommended for patients with high-risk NMHD, especially at a younger age.
FAU - Mussetti, Alberto
AU  - Mussetti A
AD  - a Dipartimento di Ematologia e Onco-Ematologia Pediatrica , Fondazione IRCCS 
      Istituto Nazionale dei Tumori , Milan , Italy.
FAU - Kernan, Nancy A
AU  - Kernan NA
AD  - b Bone Marrow Transplant Service, Department of Pediatrics , Memorial Sloan 
      Kettering Cancer Center , New York , New York , USA.
FAU - Prockop, Susan E
AU  - Prockop SE
AD  - b Bone Marrow Transplant Service, Department of Pediatrics , Memorial Sloan 
      Kettering Cancer Center , New York , New York , USA.
FAU - Scaradavou, Andromachi
AU  - Scaradavou A
AD  - b Bone Marrow Transplant Service, Department of Pediatrics , Memorial Sloan 
      Kettering Cancer Center , New York , New York , USA.
FAU - Lehrman, Rachel
AU  - Lehrman R
AD  - b Bone Marrow Transplant Service, Department of Pediatrics , Memorial Sloan 
      Kettering Cancer Center , New York , New York , USA.
FAU - Ruggiero, Julianne M
AU  - Ruggiero JM
AD  - b Bone Marrow Transplant Service, Department of Pediatrics , Memorial Sloan 
      Kettering Cancer Center , New York , New York , USA.
FAU - Curran, Kevin
AU  - Curran K
AD  - b Bone Marrow Transplant Service, Department of Pediatrics , Memorial Sloan 
      Kettering Cancer Center , New York , New York , USA.
FAU - Kobos, Rachel
AU  - Kobos R
AD  - b Bone Marrow Transplant Service, Department of Pediatrics , Memorial Sloan 
      Kettering Cancer Center , New York , New York , USA.
FAU - O'Reilly, Richard
AU  - O'Reilly R
AD  - b Bone Marrow Transplant Service, Department of Pediatrics , Memorial Sloan 
      Kettering Cancer Center , New York , New York , USA.
FAU - Boulad, Farid
AU  - Boulad F
AD  - b Bone Marrow Transplant Service, Department of Pediatrics , Memorial Sloan 
      Kettering Cancer Center , New York , New York , USA.
AD  - c Division of Pediatric Hematology/Oncology , New York Presbyterian Hospital, 
      Weill Cornell Medical College , New York , New York , USA.
LA  - eng
GR  - P30 CA008748/CA/NCI NIH HHS/United States
PT  - Journal Article
DEP - 20161007
PL  - England
TA  - Pediatr Hematol Oncol
JT  - Pediatric hematology and oncology
JID - 8700164
RN  - 0 (Myeloablative Agonists)
SB  - IM
MH  - Adolescent
MH  - Allografts
MH  - Child
MH  - Child, Preschool
MH  - Disease-Free Survival
MH  - Female
MH  - Graft vs Host Disease/mortality/therapy
MH  - Hematologic Diseases/*mortality/*therapy
MH  - *Hematopoietic Stem Cell Transplantation
MH  - Histocompatibility Testing
MH  - Humans
MH  - Infant
MH  - *Lymphocyte Depletion
MH  - Male
MH  - Myeloablative Agonists/administration & dosage
MH  - Survival Rate
MH  - *T-Lymphocytes
MH  - *Transplantation Conditioning
MH  - *Unrelated Donors
PMC - PMC5175271
MID - NIHMS831613
OTO - NOTNLM
OT  - Anemias
OT  - bone marrow failure
OT  - bone marrow transplant
OT  - chemotherapy
COIS- The authors declare no conflict of interest.
EDAT- 2016/11/01 06:00
MHDA- 2017/03/25 06:00
PMCR- 2017/10/07
CRDT- 2016/10/08 06:00
PHST- 2016/11/01 06:00 [pubmed]
PHST- 2017/03/25 06:00 [medline]
PHST- 2016/10/08 06:00 [entrez]
PHST- 2017/10/07 00:00 [pmc-release]
AID - 10.1080/08880018.2016.1204399 [doi]
PST - ppublish
SO  - Pediatr Hematol Oncol. 2016 Sep;33(6):347-358. doi: 
      10.1080/08880018.2016.1204399. Epub 2016 Oct 7.