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

Bibliographische Detailangaben
Veröffentlicht in:	Clinical immunology (Orlando, Fla.). - 1999. - 237(2022) vom: 01. Apr., Seite 108993
1. Verfasser:	Tomomasa, Dan (VerfasserIn)
Weitere Verfasser:	Booth, Claire, Bleesing, Jack J, Isoda, Takeshi, Kobayashi, Chie, Koike, Kazutoshi, Taketani, Takeshi, Sawada, Akihisa, Tamura, Akihiro, Marsh, Rebecca A, Morio, Tomohiro, Gennery, Andrew R, Kanegane, Hirokazu
Format:	Online-Aufsatz
Sprache:	English
Veröffentlicht:	2022
Zugriff auf das übergeordnete Werk:	Clinical immunology (Orlando, Fla.)
Schlagworte:	Journal Article Preemptive hematopoietic cell transplantation X-linked lymphoproliferative syndrome type 1

Beschreibung
Zusammenfassung:	Copyright © 2022. Published by Elsevier Inc. 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
Beschreibung:	Date Completed 18.04.2022 Date Revised 20.05.2022 published: Print-Electronic Citation Status MEDLINE
ISSN:	1521-7035
DOI:	10.1016/j.clim.2022.108993