Outcomes following hematopoietic cell transplantation for Wiskott–Aldrich syndrome

@article{Shin2012OutcomesFH,
  title={Outcomes following hematopoietic cell transplantation for Wiskott–Aldrich syndrome},
  author={Chu Ri Shin and M-O Kim and D L Li and Jack J H Bleesing and Richard E. Harris and Pooja Mehta and Sonata Jodele and Michael B. Jordan and Rebecca A. Marsh and Stella M. Davies and Alexandra H. Filipovich},
  journal={Bone Marrow Transplantation},
  year={2012},
  volume={47},
  pages={1428-1435}
}
HLA-identical sibling donor transplantation remains the treatment of choice for Wiskott–Aldrich Syndrome (WAS). Since 1990, utilization of alternative donor sources has increased significantly. We report the hematopoietic cell transplantation (HCT) outcomes of 47 patients with WAS treated at a single center since 1990. Improved outcomes were observed after 2000 despite the increased number of alternative donors. Five-year OS improved from 62.5% (95% CI: 34.9% to 81.1%) to 90.8% (95% CI: 67.7… 
Excellent Outcomes Following Hematopoietic Cell Transplantation for Wiskott-Aldrich Syndrome: A PIDTC Report.
TLDR
HCT outcomes for WAS have improved since 2005 compared to prior reports, and HCT at a younger age continues to be associated with superior outcomes supporting the recommendation for early HCT.
Impact of Conditioning on Outcome of Hematopoietic Stem Cell Transplantation for Wiskott-Aldrich Syndrome
TLDR
All children who underwent HSCT at 4 Israeli centers from 1996 to 2011 survived with complete clinical, immunologic, and hematologic recovery and graft failure was related to the use of conditioning regimens other than full dose Bu/Cy and not to the donor source.
Successful Allogeneic Peripheral Blood Stem Cell Transplantation in 4 Wiskott-Aldrich Syndrome Patients
TLDR
This limited study with high-dose PBSC transplantation approach for WAS demonstrated a safe and effective treatment option, with rapid engraftment, without complications, excellent long-term outcomes, independent of conditioning regimen.
Hematopoietic Stem Cell Therapy for Wiskott–Aldrich Syndrome: Improved Outcome and Quality of Life
TLDR
After trials and errors, inactivating lentiviral vectors carrying the WAS gene were successfully evaluated in clinical trials, demonstrating cure of the disease except for insufficient resolution of the platelet defect.
A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome
TLDR
Umbilical cord blood transplantation is a good alternative option for young children with Wiskott-Aldrich syndrome lacking an HLA identical stem cell donor and overall survival tended to be better in patients transplanted after 2007.
High Incidence of Autoimmune Disease after Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease.
  • Asaf D Yanir, I. C. Hanson, C. Martinez
  • Medicine, Biology
    Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
  • 2018
Transplantation of Hematopoietic Stem Cells for Primary Immunodeficiencies in Brazil: Challenges in Treating Rare Diseases in Developing Countries
TLDR
Although transplant for PID patients in Brazil has evolved since its beginning, it still faces some challenges like delayed diagnosis and referral, severe infections before transplant, a limited number of transplant centers with expertise, and resources for more advanced techniques.
Autoimmune thyroiditis following HLA‐matched sibling hematopoietic stem cell transplantation for Wiskott‐Aldrich syndrome
TLDR
The case of a male patient who underwent HLA‐matched HSCT for WAS at the age of 5 months, with his sister acting as his donor and subsequently developed AITD 12 months post‐transplant, with marked elevation of antithyroid peroxidase antibody titer is described.
A Case of Two Adult Brothers with Wiskott-Aldrich Syndrome, One Treated with Gene Therapy and One with HLA-Identical Hematopoietic Stem Cell Transplantation
TLDR
The early and medium-term follow-up after two adult siblings with the same WAS gene mutation and similar clinical phenotype, who underwent HSPC-GT and HSCT are described, focusing on safety, clinical outcome, and quality of life (QoL) of the two subjects.
Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome
TLDR
A clinical protocol based on lentiviral vector (LV) gene transfer into autologous hematopoietic stem/progenitor cells (HSCs) resulted in robust, stable, and long-term engraftment of gene-corrected HSCs in the patients’ bone marrow, and the findings support the use of LV gene therapy to treat patients with WAS.
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References

SHOWING 1-10 OF 20 REFERENCES
Stem cell transplantation for the Wiskott–Aldrich syndrome: a single-center experience confirms efficacy of matched unrelated donor transplantation
TLDR
The safety and efficacy of MUD BMT when performed early in the clinical course of WAS is emphasized, further emphasizing the importance of closely matched unrelated donor bone marrow transplant in patients who lack an human leukocyte antigen-identical sibling.
Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation.
TLDR
The most striking finding was the observation of cGVHD-independent autoimmunity in 20% of patients strongly associated with a mixed/split chimerism status, suggesting that residual-host lymphocytes can mediate autoimmune disease despite the coexistence of donor lymphocytes.
Complete correction of the Wiskott-Aldrich syndrome by allogeneic bone-marrow transplantation.
TLDR
Two patients with the Wiskott-Aldrich syndrome had complete donor lymphoid and hematopoietic engraftment after successful allogeneic bone-marrow transplantation, which may be a model for the correction of other genetically determined immune and Hematologic bone- marrow disorders.
Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program.
TLDR
The best transplantation outcomes in Wiskott-Aldrich syndrome are achieved with HLA-identical sibling donors, and equivalent survivals are possible with unrelated donors in young children.
CLINICAL MANIFESTATIONS OF GRAFT‐VERSUS-HOST DISEASE IN HUMAN RECIPIENTS OF MARROW FROM HL‐A-MATCHED SIBLING DONOR,S
TLDR
The results show that despite histocompatibility matching and methotrexate therapy, GVHD remains a serious and often fatal complication of marrow transplantation.
Clinical course of patients with WASP gene mutations.
TLDR
It is demonstrated that WAS protein expression is a useful tool for predicting long-term prognosis for patients with WAS/XLT and hematopoietic stem cell transplantation should be considered, especially for WASP-negative patients, while the patients are young to improve prognosis.
A MULTI-INSTITUTIONAL SURVEY OF THE WISKOTT-ALDRICH SYNDROME
TLDR
Two high-risk subgroups were identified in the study population: patients with platelet counts < 10 000/mm3 at the time of diagnosis were at high- risk of bleeding, and patients with autoimmune disorders were at increased risk of having a malignancy.
National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report.
  • A. Filipovich, D. Weisdorf, M. Flowers
  • Medicine
    Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
  • 2005
TLDR
This consensus document standardizes the criteria for diagnosis of chronic graft-versus-host disease (GVHD) and proposes a new clinical scoring system (0-3) that describes the extent and severity of chronic GVHD for each organ or site at any given time, taking functional impact into account.
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