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Preemptive hematopoietic cell transplantation for asymptomatic patients with X-linked lymphoproliferative syndrome type 1

Lookup NU author(s): Professor Andrew GenneryORCiD

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Abstract

© 2022. Few reports have examined whether prophylactic allogeneic hematopoietic cell transplantation (HCT) for X-linked lymphoproliferative syndrome type 1 (XLP1) improves the prognosis. We compared the prognosis of symptomatic probands and affected siblings in the same family. Twenty-two cases (10 probands and 12 affected siblings) in Japan, the United Kingdom, and the United States were analyzed. The overall survival (OS) rate at 5 years after diagnosis was 70.0% in probands and 91.7% in affected siblings (p = 0.0789). The prognosis of patients who developed symptoms of XLP1 before HCT and those who did not was also compared. The 5-year probability of OS from the time of diagnosis in asymptomatic patients (100%) was significantly better than that in symptomatic patients (66.7%). These results suggested that early HCT as soon as the diagnosis is made improves the prognosis in asymptomatic XLP1 patients.

Publication metadata

Author(s): Tomomasa D, Booth C, Bleesing JJ, Isoda T, Kobayashi C, Koike K, Taketani T, Sawada A, Tamura A, Marsh RA, Morio T, Gennery AR, Kanegane H

Publication type: Article

Publication status: Published

Journal: Clinical Immunology

Year: 2022

Volume: 237

Print publication date: 01/04/2022

Online publication date: 30/03/2022

Acceptance date: 26/03/2022

ISSN (print): 1521-6616

ISSN (electronic): 1521-7035

Publisher: Academic Press Inc.

URL: https://doi.org/10.1016/j.clim.2022.108993

DOI: 10.1016/j.clim.2022.108993

PubMed id: 35367395

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