PMID- 34778119
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20240404
IS  - 2296-2360 (Print)
IS  - 2296-2360 (Electronic)
IS  - 2296-2360 (Linking)
VI  - 9
DP  - 2021
TI  - Case Report: Haploidentical Bone Marrow Transplantation in Two Brothers With 
      Wiskott-Aldrich Syndrome Using Their Father as the Donor.
PG  - 647505
LID - 10.3389/fped.2021.647505 [doi]
LID - 647505
AB  - Wiskott-Aldrich syndrome (WAS) is an X-linked genetic disorder with a variable 
      phenotypic expression that includes thrombocytopenia, eczema, and 
      immunodeficiency. Some patients may also exhibit autoimmune manifestations. 
      Patients with WAS are at increased risk of developing malignancies such as 
      lymphoma. Allogeneic hematopoietic cell transplantation remains the only curative 
      treatment. Haploidentical bone marrow transplantation (haplo-BMT) with 
      post-transplant cyclophosphamide (PT-CY) has more recently been applied in WAS. 
      Here, we report two brothers who underwent successful T-cell replete haplo-BMT 
      with PT-CY at ages 9 months and 4 years using their father as the donor. Our 
      myeloablative regimen was well-tolerated with minimal organ toxicity and no acute 
      or chronic graft vs. host disease (GvHD). Haplo-BMT may be considered as a safe 
      and effective option for patients with WAS who do not have available human 
      leukocyte antigen (HLA) matched donors.
CI  - Copyright (c) 2021 Smith, Alfonso, Reddivalla, Angulo and Katsanis.
FAU - Smith, Jasmine
AU  - Smith J
AD  - Department of Pediatrics, University of Arizona, Tucson, AZ, United States.
AD  - Banner University Medical Center, Tucson, AZ, United States.
FAU - Alfonso, Jessica Hass
AU  - Alfonso JH
AD  - Department of Pediatrics, University of Arizona, Tucson, AZ, United States.
AD  - Banner University Medical Center, Tucson, AZ, United States.
FAU - Reddivalla, Naresh
AU  - Reddivalla N
AD  - Department of Pediatrics, University of Arizona, Tucson, AZ, United States.
AD  - Banner Children's at Desert, Mesa, AZ, United States.
FAU - Angulo, Pablo
AU  - Angulo P
AD  - Department of Pediatrics, University of Arizona, Tucson, AZ, United States.
AD  - Banner Children's at Desert, Mesa, AZ, United States.
FAU - Katsanis, Emmanuel
AU  - Katsanis E
AD  - Department of Pediatrics, University of Arizona, Tucson, AZ, United States.
AD  - Banner University Medical Center, Tucson, AZ, United States.
AD  - Department of Immunobiology, University of Arizona, Tucson, AZ, United States.
AD  - Department of Medicine, University of Arizona, Tucson, AZ, United States.
AD  - Department of Pathology, University of Arizona, Tucson, AZ, United States.
AD  - The University of Arizona Cancer Center, Tucson, AZ, United States.
LA  - eng
PT  - Case Reports
DEP - 20211027
PL  - Switzerland
TA  - Front Pediatr
JT  - Frontiers in pediatrics
JID - 101615492
PMC - PMC8578118
OTO - NOTNLM
OT  - Wiskott Aldrich syndrome (WAS)
OT  - cyclophosphamide
OT  - haploidentical hematopoietic stem cell transplantation
OT  - immunodeficiency
OT  - myeloablative allogeneic hematopoietic cell transplantation
COIS- The authors declare that the research was conducted in the absence of any 
      commercial or financial relationships that could be construed as a potential 
      conflict of interest.
EDAT- 2021/11/16 06:00
MHDA- 2021/11/16 06:01
PMCR- 2021/10/27
CRDT- 2021/11/15 07:08
PHST- 2020/12/30 00:00 [received]
PHST- 2021/09/23 00:00 [accepted]
PHST- 2021/11/15 07:08 [entrez]
PHST- 2021/11/16 06:00 [pubmed]
PHST- 2021/11/16 06:01 [medline]
PHST- 2021/10/27 00:00 [pmc-release]
AID - 10.3389/fped.2021.647505 [doi]
PST - epublish
SO  - Front Pediatr. 2021 Oct 27;9:647505. doi: 10.3389/fped.2021.647505. eCollection 
      2021.