PMID- 34295770
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20220425
IS  - 2224-4344 (Print)
IS  - 2224-4344 (Electronic)
IS  - 2224-4336 (Linking)
VI  - 10
IP  - 6
DP  - 2021 Jun
TI  - Hematopoietic stem cell transplantation for thalassemia major using HLA 
      fully-matched and mismatched donor grafts.
PG  - 1552-1565
LID - 10.21037/tp-20-415 [doi]
AB  - BACKGROUND: Until now, allogeneic hematopoietic stem cell transplantation 
      (allo-HSCT) is the only effective method to cure Thalassemia major. However, it 
      has not been determined whether similar results can be obtained with the same 
      conditioning regimen for both fully-matched and mismatched donors grafts. We 
      hypothesized that using modified NF-08-TM conditioning regimen could achieve 
      similar results for both fully and mismatched donors grafts. METHODS: This 
      retrospective cohort study included patients with beta-thalassemia major who 
      underwent HSCT with modified NF-08-TM conditioning regimen at Guangzhou Women and 
      Children's Medical Centre between January 2013 and January 2019. RESULTS: Among 
      the 257 patients (172 males) included in this study, 3 had two transplantations. 
      Totally 193 and 67 had fully-matched and mismatched donors were examined, 
      respectively. The median follow-up was 29 months; 6-year overall survival (OS), 
      thalassemia-free survival (TFS), graft rejection (GR) and transplantation-related 
      mortality (TRM) were 92.08%, 90.89%, 1.24% and 8.01%, respectively. Multivariate 
      analysis showed that human leukocyte antigen (HLA) compatibility between patient 
      and donor was not independently associated with OS, TFS, GR or TRM. Mismatched 
      donor graft transplantation for beta-thalassemia major is associated with similar 
      survival outcomes and incidences of complications (except for acute GVHD) to 
      fully-matched donor graft transplantation based on modified NF-08-TM conditioning 
      regimen. CONCLUSIONS: In conclusion, based on the modified NF-08-TM regimen, 
      certain mismatched donor transplantations for thalassemia major patients could 
      achieve similar results as fully-matched donor transplantations.
CI  - 2021 Translational Pediatrics. All rights reserved.
FAU - Huang, Chuwen
AU  - Huang C
AD  - Hematology and Oncology Department, Guangzhou Women and Children Medical Center, 
      Guangzhou, China.
FAU - Qu, Yuhua
AU  - Qu Y
AD  - Hematology and Oncology Department, Guangzhou Women and Children Medical Center, 
      Guangzhou, China.
FAU - Liu, Sha
AU  - Liu S
AD  - Hematology and Oncology Department, Guangzhou Women and Children Medical Center, 
      Guangzhou, China.
FAU - Nie, Shushan
AU  - Nie S
AD  - Hematology and Oncology Department, Guangzhou Women and Children Medical Center, 
      Guangzhou, China.
FAU - Jiang, Hua
AU  - Jiang H
AD  - Hematology and Oncology Department, Guangzhou Women and Children Medical Center, 
      Guangzhou, China.
LA  - eng
PT  - Journal Article
PL  - China
TA  - Transl Pediatr
JT  - Translational pediatrics
JID - 101649179
PMC - PMC8261584
OTO - NOTNLM
OT  - HLA antigens
OT  - Thalassemia
OT  - hematopoietic stem cell transplantation
OT  - transplant donors
COIS- Conflicts of Interest: All authors have completed the ICMJE uniform disclosure 
      form (available at http://dx.doi.org/10.21037/tp-20-415). The authors have no 
      conflicts of interest to declare.
EDAT- 2021/07/24 06:00
MHDA- 2021/07/24 06:01
PMCR- 2021/06/01
CRDT- 2021/07/23 06:51
PHST- 2020/12/02 00:00 [received]
PHST- 2021/04/21 00:00 [accepted]
PHST- 2021/07/23 06:51 [entrez]
PHST- 2021/07/24 06:00 [pubmed]
PHST- 2021/07/24 06:01 [medline]
PHST- 2021/06/01 00:00 [pmc-release]
AID - tp-10-06-1552 [pii]
AID - 10.21037/tp-20-415 [doi]
PST - ppublish
SO  - Transl Pediatr. 2021 Jun;10(6):1552-1565. doi: 10.21037/tp-20-415.