Allogeneic peripheral blood stem cell transplantation in a Wiskott-Aldrich syndrome patient

Hongeng, Suradej; Pakakasama, Samart; Hathirat, P; Chaisiripoomkere, W; Ungkanont, Artit

doi:10.1038/sj.bmt.1701927

Cited by 2 publications

(4 citation statements)

References 2 publications

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“…Bone marrow is the primary source in the majority of the clinical experience in HSCT for patients with WAS. There are limited published outcome data with PBSC transplantation for WAS 19,21,26,29,30. The use of PBSC in our patients with a median CD34 + cell count: 13.6×10 6 /kg (8 to 24.9×10 6 /kg) offers a well-tolerated conditioning regimen, rapid donor myeloid engraftment and platelet recovery (Table 2).…”

Section: Discussionmentioning

confidence: 99%

“…There are limited published outcome data with PBSC transplantation for WAS. 19,21,26,29,30 The use of PBSC in our patients with a median CD34 + cell count: 13.6×10 6 /kg (8 to 24.9×10 6 /kg) offers a well-tolerated conditioning regimen, rapid donor myeloid engraftment and platelet recovery (Table 2). The higher levels of donor engraftment observed with PBSCs are likely to reflect an increased alloreactive effect with reduce the risk of autologous reconstitution.…”

Section: Discussionmentioning

confidence: 99%

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Successful Allogeneic Peripheral Blood Stem Cell Transplantation in 4 Wiskott-Aldrich Syndrome Patients

Benakli¹,

Nacer²,

Mehdid³

et al. 2021

Journal of Pediatric Hematology/Oncology

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Background: Allogeneic hematopoietic stem cell transplantation is a potential curative treatment in Wiskott-Aldrich syndrome (WAS). Here, we analyzed the outcomes in 4 WAS patients who underwent this procedure with peripheral blood stem cell (PBSC) in our center.Patients and Methods: Four patients with severe WAS phenotype have received allogeneic hematopoietic stem cell transplantation between January 2014 and December 2019 from matched sibling donors with PBSC. Two different preparative conditioning regimens were provided: the first associated busulfan-cyclophosphamide (2 patients) and the second with busulfan-fludarabine administered to the others. Cyclosporine gave as preferred graftversus-host disease prophylaxis with a short course of methotrexate.Results: All patients achieved engraftment after PBSC with a median CD34 + cell count: 13.6×10 6 /kg (8 to 24.9×10 6 /kg). Chronic graft-versus-host disease developed in 2 patients treated by cyclosporine-steroids with complete resolution. Chimerism for all the patients was fully donor ( > 95% donor). After a median follow-up of 41 months (8 to 74 mo), all patients (100%) are alive, healthy, with complete clinical, immunologic, and hematologic recovery, without signs of WAS. Conclusion: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.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Successful Allogeneic Peripheral Blood Stem Cell Transplantation in 4 Wiskott-Aldrich Syndrome Patients

Benakli¹,

Nacer²,

Mehdid³

et al. 2021

Journal of Pediatric Hematology/Oncology

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“…This gene was found to encode WAS protein, a component of the cytoskeletal surface membrane complex of hematopoietic cells. This syndrome especially affects morphology and function of platelets and lymphocytes (1, 2).…”

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confidence: 99%

“…Untreated patients with typical WAS have poor prognosis with the major causes of death being infection, bleeding, lymphoproliferative disorders, and malignancy (3). Allogenic HSCT is the only curative treatment which can correct both lymphoid and platelet abnormalities (2, 4). The five yr probabilities of survival have been reported to be 87% (74–94%) with HLA identical sibling donors, 52% (37–65%) with other related donors and 71% (58–80%) with unrelated donors (1).…”

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confidence: 99%

Hematopoietic stem cell transplantation from a donor with Klinefelter syndrome for Wiskott–Aldrich syndrome

Balcı¹,

Turul

Daar³

et al. 2008

Pediatric Transplantation

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WAS is a rare X-linked recessive disorder characterized by primary progressive T cell immunodeficiency, impaired antipolysaccharide antibody response, thrombocytopenia with small platelet, and eczematoid dermatitis. Untreated patients with typical WAS have poor prognosis with the major causes of death being infection, bleeding, lymphoproliferative disorders, and malignancy. Due to the increased risk of infectious and hemorrhagic episodes the best results with HSCT are achieved in patients less than five yr of age and are recommended as early as possible. Here, we report a three-yr-old boy with WAS who underwent UCB and BMT from his genotypically identical brother with Klinefelter syndrome.

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Allogeneic peripheral blood stem cell transplantation in a Wiskott-Aldrich syndrome patient

Cited by 2 publications

References 2 publications

Successful Allogeneic Peripheral Blood Stem Cell Transplantation in 4 Wiskott-Aldrich Syndrome Patients

Successful Allogeneic Peripheral Blood Stem Cell Transplantation in 4 Wiskott-Aldrich Syndrome Patients

Hematopoietic stem cell transplantation from a donor with Klinefelter syndrome for Wiskott–Aldrich syndrome

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