ArticleBone marrow transplantation2006
Stem cell transplantation for the Wiskott-Aldrich syndrome: a single-center experience confirms efficacy of matched unrelated donor transplantation.
Article in Bone marrow transplantation, 2006. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
25 citing papers in PubMed, 78 citations in OpenAlex.
- Hematopoietic Stem Cell Therapy for Wiskott-Aldrich Syndrome: Improved Outcome and Quality of Life.Journal of blood medicine · 2021Review
- Article
- Wiskott-Aldrich Syndrome (WAS) and Dedicator of Cytokinesis 8- (DOCK8) Deficiency.Frontiers in pediatrics · 2019Review
- Article
- In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases.Cell stem cell · 2016Article
- Engineering Stem Cells for Biomedical Applications.Advanced healthcare materials · 2016Review
- Hematopoietic stem cell transplantation for primary immunodeficiencies.Hematology/oncology clinics of North America · 2014Review
- Recent advances in transplantation for primary immune deficiency diseases: a comprehensive review.Clinical reviews in allergy & immunology · 2014Review
- Wiskott-Aldrich syndrome: diagnosis, current management, and emerging treatments.The application of clinical genetics · 2014Review
- Diagnosis and treatment of gastrointestinal disorders in patients with primary immunodeficiency.Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association · 2013Review
- Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott-Aldrich syndrome.Gene therapy · 2012Article
- Outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome.Bone marrow transplantation · 2012Article
- Warts and all: human papillomavirus in primary immunodeficiencies.The Journal of allergy and clinical immunology · 2012Review
- Autoimmunity in wiskott-Aldrich syndrome: an unsolved enigma.Frontiers in immunology · 2012Article
- Prolonged survival after splenectomy in Wiskott-Aldrich syndrome: a case report.Italian journal of pediatrics · 2011Article
- Article
- Paracrine molecules of mesenchymal stem cells for hematopoietic stem cell niche.Bone marrow research · 2011Article
- Hematopoietic cell transplantation for treatment of primary immune deficiencies.Cellular therapy and transplantation · 2010Article
- Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: advances in biology and future directions for treatment.Immunology and allergy clinics of North America · 2010Review
- Clinical and molecular characteristics of 35 Chinese children with Wiskott-Aldrich syndrome.Journal of clinical immunology · 2009Article
Corrections and comments
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Authors and funding
12 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The treatment of Wiskott-Aldrich syndrome (WAS), a once uniformly fatal disorder, has evolved considerably as the use of hematopoietic stem cell transplant has become more widespread. For the majority of patients who lack an human leukocyte antigen-identical sibling, closely matched unrelated donor bone marrow transplant (MUD BMT) at an early age is an excellent option that nevertheless is not uniformly chosen. We retrospectively analyzed our experience with transplantation in 23 patients with WAS from 1990 to 2005 at the University of Brescia, Italy, of whom 16 received MUD BMT. Myeloablative chemotherapy was well tolerated with median neutrophil engraftment at day 18, and no cases of grade III or IV graft-vs-host disease. Overall survival was very good with 78.2% (18/23) of the whole cohort and 81.2% (13/16) of MUD BMT recipients surviving. Among 18 survivors, full donor engraftment was detected in 12 patients, and stable mixed chimerism in all blood lineages in four patients. Deaths were limited to patients who had received mismatched related BMT or who had severe clinical symptomatology at the time of transplantation, further emphasizing the safety and efficacy of MUD BMT when performed early in the clinical course of WAS.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.