Stable mixed chimerism after hematopoietic stem cell transplantation in Wiskott-Aldrich syndrome

2006 ◽  
Vol 10 (3) ◽  
pp. 395-399 ◽  
Author(s):  
Figen Doğu ◽  
Melahat Kurtuluş-Ülküer ◽  
Yaşar Bilge ◽  
Günseli Bozdoğan ◽  
Üner Ülküer ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Mixed Chimerism ◽  
Hematopoietic Stem ◽  
Wiskott Aldrich Syndrome

scholarly journals Long-term persistent mixed chimerism in a patient with Wiskott–Aldrich syndrome after allogeneic hematopoietic stem cell transplantation

2021 ◽  
Vol 20 (2) ◽  
pp. 148-155
Author(s):  
V. О. Bludova ◽  
A. L. Laberko ◽  
Yu. A. Rodina ◽  
V. V. Brilliantova ◽  
E. V. Raykina ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Mixed Chimerism ◽  
Normal Numbers ◽  
Hematopoietic Stem ◽  
Wiskott Aldrich Syndrome

The article describes a clinical case of a patient with Wiskott–Aldrich syndrome, in whom long- term persistence of mixed chimerism was determined after hematopoietic stem cell transplantation (HSCT) from a haploidentical donor. Based on the analysis of the patient's clinical picture after HSCT, it was shown that the presence of> 50% of donor cells in the myeloid lineage is necessary for the correction of thrombocytopenia. In addition, the presence of mixed chimerism in B-lymphocytes possibly contributed to the development of autoimmune complications in the patient, as well as to the persistent hypogammaglobulinemia, despite the restoration of the normal numbers of lymphocytes in all main sub-populations. The role of mixed chimerism in the pathogenesis of immune post-transplant complications requires study in large groups of patients with primary immunodeficiencies. The patient's parents agreed to use the information, including the child's photo, in scientific research and publications.

scholarly journals 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

Blood ◽  
2008 ◽  
Vol 111 (1) ◽  
pp. 439-445 ◽  
Author(s):  
Hulya Ozsahin ◽  
Marina Cavazzana-Calvo ◽  
Luigi D. Notarangelo ◽  
Ansgar Schulz ◽  
Adrian J. Thrasher ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Collaborative Study ◽  
Hematopoietic Stem ◽  
Long Term Outcome ◽  
Wiskott Aldrich Syndrome

Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency with microthrombocytopenia, eczema, recurrent infections, autoimmune disorders, and malignancies that are life-threatening in the majority of patients. In this long-term, retrospective, multicenter study, we analyzed events that occurred in 96 WAS patients who received transplants between 1979 and 2001 who survived at least 2 years following hematopoietic stem-cell transplantation (HSCT). Events included chronic graft-versus-host disease (cGVHD), autoimmunity, infections, and sequelae of before or after HSCT complications. Three patients (3%) died 2.1 to 21 years following HSCT. Overall 7-year event-free survival rate was 75%. It was lower in recipients of mismatched related donors, also in relation with an older age at HSCT and disease severity. The most striking finding was the observation of cGVHD-independent autoimmunity in 20% of patients strongly associated with a mixed/split chimerism status (P < .001), suggesting that residual-host lymphocytes can mediate autoimmune disease despite the coexistence of donor lymphocytes. Infectious complications (6%) related to splenectomy were also significant and may warrant a more restrictive approach to performing splenectomy in WAS patients. Overall, this study provides the basis for a prospective, standardized, and more in-depth detailed analysis of chimerism and events in long-term follow-up of WAS patients who receive transplants to design better-adapted therapeutic strategies.

scholarly journals Hematopoietic stem cell transplantation monitoring in childhood. Hematological diseases in Serbia: STR-PCR techniques

2007 ◽  
Vol 59 (1) ◽  
pp. 23-27
Author(s):  
Aleksandra Krstic ◽  
O. Stojkovic ◽  
Marija Guc-Scekic ◽  
Dragana Vujic ◽  
Dragana Jevtic ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Tandem Repeats ◽  
Methodological Approach ◽  
Disease Free Survival ◽  
Mixed Chimerism ◽  
Hematopoietic Stem

Hematopoietic stem cell transplantation (HSCT) is a very successful method of treatment for children with different aquired or inborn diseases. The main goal of post-transplantation chimerism monitoring in HSCT is to predict negative events (such as disease relapse and graft rejection), in order to intervene with appropriate therapy and improve the probability of long-term DFS (disease free survival). In this context, by quantifying the relative amounts of donor and recipient cells present in the peripheral blood sample, it can be determined if engraftment has taken place at all, or if full or mixed chimerism exists. In a group of patients who underwent hematopoietic stem cell transplantation at the Mother and Child Health Care Institute, we decided to use standard human identfication tests based on multiplex PCR analyses of short tandem repeats (STRs), as they are highly informative, sensitive, and fast and therefore represent an optimal methodological approach to engraftment analysis.

scholarly journals Outcome Of Hematopoietic Stem Cell Transplantation For Wiskott-Aldrich Syndrome

2014 ◽  
Vol 20 (2) ◽  
pp. S231
Author(s):  
Sakara Hutspardol ◽  
Adam Gassas ◽  
John Doyle ◽  
Muhammad Ali ◽  
R. Maarten Egeler ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Hematopoietic Stem ◽  
Wiskott Aldrich Syndrome

Correction of the murine Wiskott-Aldrich syndrome phenotype by hematopoietic stem cell transplantation

Blood ◽  
2002 ◽  
Vol 99 (12) ◽  
pp. 4626-4628 ◽  
Author(s):  
Ted S. Strom ◽  
Xiuling Li ◽  
John M. Cunningham ◽  
Arthur W. Nienhuis
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Disease Phenotype ◽  
Hematopoietic Stem ◽  
Wiskott Aldrich Syndrome ◽  
Radiation Induced ◽  
Deficient Mice

Allogeneic hematopoietic stem cell transplantation (HSCT) corrects the Wiskott-Aldrich syndrome (WAS) phenotype. However, the toxicity and mortality frequently associated with this approach warrant the exploration of new therapeutic strategies. Transplantation studies of a murine model of WAS deficiency have been limited by the occurrence of a radiation-induced fatal exacerbation of a pre-existing colitis in the peritransplantation period. Here we demonstrate that when crossed to a C57/B6 background, WAS-deficient males show little if any colitis and reliably survive HSCT. We show that HSCT corrects the hematologic and functional deficiencies of WAS knockout mice. These results strengthen the analogy between murine and human WAS and provide a basis for the use of WAS-deficient mice to explore novel approaches for correction of the disease phenotype.

Treosulfan-based conditioning regimens for hematopoietic stem cell transplantation in children with primary immunodeficiency: United Kingdom experience

Blood ◽  
2011 ◽  
Vol 117 (16) ◽  
pp. 4367-4375 ◽  
Author(s):  
Mary A. Slatter ◽  
Kanchan Rao ◽  
Persis Amrolia ◽  
Terry Flood ◽  
Mario Abinun ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Primary Immunodeficiency ◽  
Occlusive Disease ◽  
Mixed Chimerism ◽  
Hematopoietic Stem

Abstract Children with primary immunodeficiency diseases, particularly those less than 1 year of age, experience significant toxicity after hematopoietic stem cell transplantation, with busulfan- or melphalan-based conditioning. Treosulfan causes less veno-occlusive disease than busulfan and does not require pharmacokinetic monitoring. We report its use in 70 children. Children received 42 g/m2 or 36 g/m2 with cyclophosphamide 200 mg/kg (n = 30) or fludarabine 150 mg/m2 (n = 40), with alemtuzumab in most. Median age at transplantation was 8.5 months (range, 1.2-175 months); 46 (66%) patients were 12 months of age or younger. Donors were as follows: matched sibling donor, 8; matched family donor, 13; haploidentical, 4; and unrelated, 45. Median follow-up was 19 months (range, 1-47 months). Overall survival was 81%, equivalent in those age less or greater than 1 year. Skin toxicity was common. Veno-occlusive disease occurred twice with cyclophosphamide. Eighteen patients (26%) had graft-versus-host disease, and only 7 (10%) greater than grade 2. Two patients rejected; 24 of 42 more than 1 year after transplantation had 100% donor chimerism. The remainder had stable mixed chimerism. T-cell chimerism was significantly better with fludarabine. Long-term follow-up is required, but in combination with fludarabine, treosulfan is a good choice of conditioning for hematopoietic stem cell transplantation in primary immunodeficiency disease.

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