Outcomes following hematopoietic cell transplantation for Wiskott–Aldrich syndrome

Cr, Shin; Kim, Miok; D, Li; Jj, Bleesing; Harris, Richard E.; Mehta, Parinda A.; Jodele, Sonata; Mb, Jordan; Ra, Marsh; Sm, Davies; Ah, Filipovich

doi:10.1038/bmt.2012.31

Cited by 74 publications

(63 citation statements)

References 17 publications

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“…Outcomes of patients with WAS who do not undergo HSCT remain poor, with the mean age at death being 20 years in previous reports 9 and with increasing risk of malignancies with age. Several groups 11,15,16 reported successful HSCT results with an OS of up to 88% when using a “gold standard donor”. 14 In the absence of a matched related donor, other groups have reported the successful use of matched unrelated donors with 71% OS.…”

Section: Discussionmentioning

confidence: 99%

“…6,10 Various series of HSCT from HLA-matched related donors have consistently resulted in survival rates above 80% for patients with WAS. 11–15 In the absence of a matched related donor, the OS reported after matched unrelated HSCT has been around 70%. 11 In the last 20 years, unrelated donor umbilical cord blood transplantation (UCBT) has become an option for patients lacking an HLA-matched donor.…”

Section: Introductionmentioning

confidence: 99%

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A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome

et al. 2017

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Wiskott-Aldrich syndrome is a severe X-linked recessive immune deficiency disorder. A scoring system of Wiskott-Aldrich syndrome severity (0.5–5) distinguishes two phenotypes: X-linked thrombocytopenia and classic Wiskott-Aldrich syndrome. Hematopoietic cell transplantation is curative for Wiskott-Aldrich syndrome; however, the use of unrelated umbilical cord blood transplantation has seldom been described. We analyzed umbilical cord blood transplantation outcomes for 90 patients. The median age at umbilical cord blood transplantation was 1.5 years. Patients were classified according to clinical scores [2 (23%), 3 (30%), 4 (23%) and 5 (19%)]. Most patients underwent HLA-mismatched umbilical cord blood transplantation and myeloablative conditioning with anti-thymocyte globulin. The cumulative incidence of neutrophil recovery at day 60 was 89% and that of grade II–IV acute graft-versus-host disease at day 100 was 38%. The use of methotrexate for graft-versus-host disease prophylaxis delayed engraftment (P=0.02), but decreased acute graft-versus-host disease (P=0.03). At 5 years, overall survival and event-free survival rates were 75% and 70%, respectively. The estimated 5-year event-free survival rates were 83%, 73% and 55% for patients with a clinical score of 2, 4–5 and 3, respectively. In multivariate analysis, age <2 years at the time of the umbilical cord blood transplant and a clinical phenotype of X-linked thrombocytopenia were associated with improved event-free survival. Overall survival tended to be better in patients transplanted after 2007 (P=0.09). In conclusion, umbilical cord blood transplantation is a good alternative option for young children with Wiskott-Aldrich syndrome lacking an HLA identical stem cell donor.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome

et al. 2017

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“…The presence of autoimmunity increases disease severity and contributes to the indication for HSCT. Unfortunately, even after HSCT and/or gene therapy autoimmune cytopenias may resurface and become refractory (55-58), as demonstrated by the 55% of WAS patients who developed autoimmune cytopenias in the post-transplant period (59). Thrombopoietin receptor agonists such as romiplostim and eltrombopag are emerging therapies for ITP, mainly by promoting platelet production.…”

Section: Treatment Of Autoimmune Cytopenias In Primary Immunodeficmentioning

confidence: 99%

Mechanism-Based Strategies for the Management of Autoimmunity and Immune Dysregulation in Primary Immunodeficiencies

Walter

Farmer

Foldvari

et al. 2016

The Journal of Allergy and Clinical Immunology: In Practice

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A broad spectrum of autoimmunity is now well described in patients with primary immunodeficiencies (PIDs). Management of autoimmune disease in the background of PID is particularly challenging given the seemingly discordant goals of immune support and immune suppression. Our growing ability to define the molecular underpinnings of immune dysregulation has facilitated novel targeted therapeutics. This review focuses on mechanism-based treatment strategies for the most common autoimmune and inflammatory complications of PID including autoimmune cytopenias, rheumatologic disease, and gastrointestinal disease. We aim to provide guidance regarding the rational use of these agents in the complex PID patient population.

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“…In the setting of WAS, age at the time of HSCT is an important consideration 16,35,40,44. Patients with WAS who undergo unrelated donor HSCT at younger ages (ie, less than 5 years of age) have outcomes that are comparable to those associated with the use of matched-sibling donor HSCT.…”

Section: Transplant Considerationsmentioning

confidence: 99%

Wiskott–Aldrich syndrome: diagnosis, current management, and emerging treatments

Buchbinder¹,

Nugent²,

Fillipovich³

2014

TACG

130

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Wiskott–Aldrich syndrome (WAS) is a rare X-linked primary immunodeficiency disorder characterized by the triad of eczema, thrombocytopenia, and severe and often recurrent infections. Despite the rarity of this disorder, our understanding of the molecular and cellular pathogenesis of WAS has continued to increase. Advances in the use of diagnostic tools, the provision of supportive care, and improvements in allogeneic hematopoietic stem cell transplantation have significantly reduced the morbidity and mortality associated with this disorder. Exciting advancements in the care of patients with WAS have also occurred, including the successful application of autologous gene-modified hematopoietic stem cell transplantation.

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Outcomes following hematopoietic cell transplantation for Wiskott–Aldrich syndrome

Cited by 74 publications

References 17 publications

A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome

A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome

Mechanism-Based Strategies for the Management of Autoimmunity and Immune Dysregulation in Primary Immunodeficiencies

Wiskott–Aldrich syndrome: diagnosis, current management, and emerging treatments

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Outcomes following hematopoietic cell transplantation for Wiskott–Aldrich syndrome

Cited by 74 publications

References 17 publications

A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome

A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome

Mechanism-Based Strategies for the Management of Autoimmunity and Immune Dysregulation in Primary Immunodeficiencies

Wiskott&ndash;Aldrich syndrome: diagnosis, current management, and emerging treatments

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Wiskott–Aldrich syndrome: diagnosis, current management, and emerging treatments