Publication: Long-term outcome of LRBA deficiency in 76 patients after various treatment modalities as evaluated by the immune deficiency and dysregulation activity (IDDA) score
Long-term outcome of LRBA deficiency in 76 patients after various treatment modalities as evaluated by the immune deficiency and dysregulation activity (IDDA) score
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Tesch, V. K., Abolhassani, H., Shadur, B., Zobel, J., Mareika, Y., et al, & Kolios, A. (2020). Long-term outcome of LRBA deficiency in 76 patients after various treatment modalities as evaluated by the immune deficiency and dysregulation activity (IDDA) score. Journal of Allergy and Clinical Immunology, 145(5), 1452–1463. https://doi.org/10.1016/j.jaci.2019.12.896
Abstract
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Abstract
Background: Recent findings strongly support hematopoietic stem cell transplantation (HSCT) in patients with severe presentation of LPS-responsive beige-like anchor protein (LRBA) deficiency, but long-term follow-up and survival data beyond previous patient reports or meta-reviews are scarce for those patients who do not receive a transplant.
Objective: This international retrospective study was conducted to elucidate the longitudinal clinical course of patients with LRBA deficiency who do and do not receive a transplant.
Method: We
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- Tesch, Victoria Katharina
- Abolhassani, Hassan
- Shadur, Bella
- Zobel, Joachim
- Mareika, Yuliya
- et al
- Kolios, Antonios
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Tesch, V. K., Abolhassani, H., Shadur, B., Zobel, J., Mareika, Y., et al, & Kolios, A. (2020). Long-term outcome of LRBA deficiency in 76 patients after various treatment modalities as evaluated by the immune deficiency and dysregulation activity (IDDA) score. Journal of Allergy and Clinical Immunology, 145(5), 1452–1463. https://doi.org/10.1016/j.jaci.2019.12.896
