PMID- 27537474
OWN - NLM
STAT- MEDLINE
DCOM- 20171024
LR  - 20180819
IS  - 1531-7048 (Electronic)
IS  - 1065-6251 (Linking)
VI  - 23
IP  - 6
DP  - 2016 Nov
TI  - Transplantation for thalassemia major: alternative donors.
PG  - 515-523
AB  - PURPOSE OF REVIEW: Lack of a human leukocyte antigen (HLA)-matched family donor 
      is a major obstacle limiting the use of allogeneic hematopoietic stem cell 
      transplantation (HSCT) to cure thalassemia major. Use of alternative donors, if 
      found well tolerated enough, may be a viable option for transplantation in this 
      setting, allowing to expand the number of patients treatable by HSCT. This review 
      will provide an overview of alternative HSCT approaches in thalassemia major. 
      RECENT FINDINGS: With the introduction of high-resolution molecular HLA-typing 
      techniques, the outcome of matched unrelated donor HSCT recipients has become 
      comparable with that of patients given matched family donor HSCT. On the 
      contrary, the use of unrelated cord blood transplantation has been limited by the 
      risk of graft failure and slow hematopoietic recovery, although novel techniques 
      of ex vivo graft manipulation are emerging as promising strategies for overcoming 
      these obstacles. Although experience with haploidentical HSCT in thalassemia 
      major is still limited, currently explored platforms hold the potential to extend 
      the access to HSCT to thalassemia major patients lacking an HLA-matched either 
      related or unrelated donor. SUMMARY: The significant advances achieved in 
      transplantation techniques, both in the matched unrelated and in the 
      haploidentical setting, are expected to significantly broaden the applicability 
      of HSCT to patients with thalassemia major.
FAU - Locatelli, Franco
AU  - Locatelli F
AD  - aDipartimento di Oncoematologia Pediatrica, IRCCS, Ospedale Pediatrico Bambino 
      Gesu, Roma bDipartimento di Scienze Pediatriche, Universita degli Studi di Pavia, 
      Pavia, Italy.
FAU - Merli, Pietro
AU  - Merli P
FAU - Strocchio, Luisa
AU  - Strocchio L
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Review
PL  - United States
TA  - Curr Opin Hematol
JT  - Current opinion in hematology
JID - 9430802
RN  - 0 (HLA Antigens)
SB  - IM
MH  - Cord Blood Stem Cell Transplantation
MH  - Graft Rejection
MH  - Graft vs Host Disease/diagnosis/etiology/prevention & control
MH  - HLA Antigens/genetics/immunology
MH  - *Hematopoietic Stem Cell Transplantation/adverse effects/methods
MH  - Histocompatibility Testing
MH  - Humans
MH  - *Tissue Donors
MH  - Transplantation Conditioning/adverse effects/methods
MH  - Transplantation, Homologous
MH  - Treatment Outcome
MH  - beta-Thalassemia/complications/diagnosis/mortality/*therapy
EDAT- 2016/10/18 06:00
MHDA- 2017/10/25 06:00
CRDT- 2016/08/19 06:00
PHST- 2016/10/18 06:00 [pubmed]
PHST- 2017/10/25 06:00 [medline]
PHST- 2016/08/19 06:00 [entrez]
AID - 10.1097/MOH.0000000000000280 [doi]
PST - ppublish
SO  - Curr Opin Hematol. 2016 Nov;23(6):515-523. doi: 10.1097/MOH.0000000000000280.