Literature DB >> 20493395

Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: advances in biology and future directions for treatment.

Sung-Yun Pai1, Luigi D Notarangelo.   

Abstract

The Wiskott-Aldrich syndrome (WAS) is an X-linked disorder characterized by a triad of diagnostic clinical elements: immunodeficiency, eczema, and hemorrhage caused by thrombocytopenia with small-sized platelets. The formal proof that hematopoietic cell transplantation (HCT) could be used to cure WAS revealed a requirement for both immunosuppression and myelosuppression that still underlies the standard approach to curative therapy today. The current short- and long-term toxicities of HCT are the main stumbling block for the ability to cure every patient with WAS and X-linked thrombocytopenia, and much remains to be done. Copyright (c) 2010 Elsevier Inc. All rights reserved.

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Year:  2010        PMID： 20493395      PMCID： PMC2930258          DOI： 10.1016/j.iac.2010.02.001

Source DB:  PubMed          Journal:  Immunol Allergy Clin North Am        ISSN： 0889-8561            Impact factor:   3.479


  81 in total

1.  An international study examining therapeutic options used in treatment of Wiskott-Aldrich syndrome.

Authors:  Mary Ellen Conley; Delphine Saragoussi; Luigi Notarangelo; Amos Etzioni; Jean Laurent Casanova
Journal:  Clin Immunol       Date:  2003-12       Impact factor: 3.969

2.  Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99.

Authors:  Corinne Antoine; Susanna Müller; Andrew Cant; Marina Cavazzana-Calvo; Paul Veys; Jaak Vossen; Anders Fasth; Carsten Heilmann; Nicolas Wulffraat; Reinhard Seger; Stéphane Blanche; Wilhelm Friedrich; Mario Abinun; Graham Davies; Robert Bredius; Ansgar Schulz; Paul Landais; Alain Fischer
Journal:  Lancet       Date:  2003-02-15       Impact factor: 79.321

3.  Wiskott-Aldrich syndrome protein is required for NK cell cytotoxicity and colocalizes with actin to NK cell-activating immunologic synapses.

Authors:  Jordan S Orange; Narayanaswamy Ramesh; Eileen Remold-O'Donnell; Yoji Sasahara; Louise Koopman; Michael Byrne; Francisco A Bonilla; Fred S Rosen; Raif S Geha; Jack L Strominger
Journal:  Proc Natl Acad Sci U S A       Date:  2002-08-12       Impact factor: 11.205

4.  Mixed chimera status of 12 patients with Wiskott-Aldrich syndrome (WAS) after hematopoietic stem cell transplantation: evaluation by flow cytometric analysis of intracellular WAS protein expression.

Authors:  Koji Yamaguchi; Tadashi Ariga; Masafumi Yamada; David L Nelson; Ryouji Kobayashi; Chie Kobayashi; Yasushi Noguchi; Yasuhiko Ito; Kenji Katamura; Yoshihisa Nagatoshi; Satoshi Kondo; Hiroyuki Katoh; Yukio Sakiyama
Journal:  Blood       Date:  2002-08-15       Impact factor: 22.113

5.  Autoimmunity in Wiskott-Aldrich syndrome: risk factors, clinical features, and outcome in a single-center cohort of 55 patients.

Authors:  Sophie Dupuis-Girod; Jacques Medioni; Elie Haddad; Pierre Quartier; Marina Cavazzana-Calvo; Françoise Le Deist; Geneviève de Saint Basile; Jean Delaunay; Klaus Schwarz; Jean-Laurent Casanova; Stephane Blanche; Alain Fischer
Journal:  Pediatrics       Date:  2003-05       Impact factor: 7.124

6.  Clinical course of patients with WASP gene mutations.

Authors:  Kohsuke Imai; Tomohiro Morio; Yi Zhu; Yinzhu Jin; Sukeyuki Itoh; Michiko Kajiwara; Jun-Ichi Yata; Shuki Mizutani; Hans D Ochs; Shigeaki Nonoyama
Journal:  Blood       Date:  2003-09-11       Impact factor: 22.113

7.  Umbilical cord blood transplantation in Wiskott Aldrich syndrome.

Authors:  Alan P Knutsen; Marsha Steffen; Kris Wassmer; Donna A Wall
Journal:  J Pediatr       Date:  2003-05       Impact factor: 4.406

Review 8.  Autoimmunity in Wiskott-Aldrich syndrome.

Authors:  Shepherd H Schurman; Fabio Candotti
Journal:  Curr Opin Rheumatol       Date:  2003-07       Impact factor: 5.006

9.  Successful unrelated cord blood transplantation in an infant with Wiskott-Aldrich syndrome following recurrent cytomegalovirus disease.

Authors:  Michiya Kaneko; Tsutomu Watanabe; Hiroyoshi Watanabe; Masahiro Kimura; Hiroko Suzuya; Yasuhiro Okamoto; Ryuji Nakagawa; Yasuhiro Kuroda
Journal:  Int J Hematol       Date:  2003-12       Impact factor: 2.490

10.  Early deficit of lymphocytes in Wiskott-Aldrich syndrome: possible role of WASP in human lymphocyte maturation.

Authors:  J Y Park; M Kob; A P Prodeus; F S Rosen; A Shcherbina; E Remold-O'Donnell
Journal:  Clin Exp Immunol       Date:  2004-04       Impact factor: 4.330
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  14 in total

1.  Successful HLA haploidentical HSCT with post-transplant cyclophosphamide in Wiskott-Aldrich syndrome.

Authors:  P Kreetapirom; S Hongeng; W Manuyakorn; U Anurathapan; S Pakakasama; N Sirachainan; B S Andersson
Journal:  Bone Marrow Transplant       Date:  2017-03-13       Impact factor: 5.483

2.  Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.

Authors:  Salima Hacein-Bey Abina; H Bobby Gaspar; Johanna Blondeau; Laure Caccavelli; Sabine Charrier; Karen Buckland; Capucine Picard; Emmanuelle Six; Nourredine Himoudi; Kimberly Gilmour; Anne-Marie McNicol; Havinder Hara; Jinhua Xu-Bayford; Christine Rivat; Fabien Touzot; Fulvio Mavilio; Annick Lim; Jean-Marc Treluyer; Sébastien Héritier; Francois Lefrère; Jeremy Magalon; Isabelle Pengue-Koyi; Géraldine Honnet; Stéphane Blanche; Eric A Sherman; Frances Male; Charles Berry; Nirav Malani; Frederic D Bushman; Alain Fischer; Adrian J Thrasher; Anne Galy; Marina Cavazzana
Journal:  JAMA       Date:  2015-04-21       Impact factor: 56.272

3.  Treatment of primary immunodeficiency with allogeneic transplant and gene therapy.

Authors:  Sung-Yun Pai
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2019-12-06

4.  Wiskott-Aldrich syndrome protein-deficient hematopoietic cells can be efficiently mobilized by granulocyte colony-stimulating factor.

Authors:  Sabine Charrier; Michael Blundell; Gregory Cédrone; Fawzia Louache; William Vainchenker; Adrian J Thrasher; Anne Galy
Journal:  Haematologica       Date:  2013-02-26       Impact factor: 9.941

Review 5.  Genetics of familial forms of thrombocytopenia.

Authors:  Carlo L Balduini; Anna Savoia
Journal:  Hum Genet       Date:  2012-08-11       Impact factor: 4.132

Review 6.  Clinical and imaging considerations in primary immunodeficiency disorders: an update.

Authors:  Eveline Y Wu; Lauren Ehrlich; Brian Handly; Donald P Frush; Rebecca H Buckley
Journal:  Pediatr Radiol       Date:  2016-09-22

7.  Otitis media in children with congenital immunodeficiencies.

Authors:  Simon Urschel
Journal:  Curr Allergy Asthma Rep       Date:  2010-11       Impact factor: 4.806

8.  Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study.

Authors:  Daniele Moratto; Silvia Giliani; Carmem Bonfim; Evelina Mazzolari; Alain Fischer; Hans D Ochs; Andrew J Cant; Adrian J Thrasher; Morton J Cowan; Michael H Albert; Trudy Small; Sung-Yun Pai; Elie Haddad; Antonella Lisa; Sophie Hambleton; Mary Slatter; Marina Cavazzana-Calvo; Nizar Mahlaoui; Capucine Picard; Troy R Torgerson; Lauri Burroughs; Adriana Koliski; Jose Zanis Neto; Fulvio Porta; Waseem Qasim; Paul Veys; Kristina Kavanau; Manfred Hönig; Ansgar Schulz; Wilhelm Friedrich; Luigi D Notarangelo
Journal:  Blood       Date:  2011-06-09       Impact factor: 22.113

9.  Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult.

Authors:  Emma C Morris; Thomas Fox; Ronjon Chakraverty; Rita Tendeiro; Katie Snell; Christine Rivat; Sarah Grace; Kimberly Gilmour; Sarita Workman; Karen Buckland; Katie Butler; Ronnie Chee; Alan D Salama; Hazem Ibrahim; Havinder Hara; Cecile Duret; Fulvio Mavilio; Frances Male; Frederick D Bushman; Anne Galy; Siobhan O Burns; H Bobby Gaspar; Adrian J Thrasher
Journal:  Blood       Date:  2017-07-17       Impact factor: 22.113

Review 10.  Lentiviral vectors for the treatment of primary immunodeficiencies.

Authors:  Giada Farinelli; Valentina Capo; Samantha Scaramuzza; Alessandro Aiuti
Journal:  J Inherit Metab Dis       Date:  2014-03-12       Impact factor: 4.982
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