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

Dan Tomomasa; Claire Booth; Jack J Bleesing; Takeshi Isoda; Chie Kobayashi; Kazutoshi Koike; Takeshi Taketani; Akihisa Sawada; Akihiro Tamura; Rebecca A Marsh; Tomohiro Morio; Andrew R Gennery; Hirokazu Kanegane

doi:10.1016/j.clim.2022.108993

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

Clin Immunol. 2022 Apr:237:108993. doi: 10.1016/j.clim.2022.108993. Epub 2022 Mar 30.

Authors

Affiliations

¹ Department of Pediatrics and Developmental Biology, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan.
² Molecular and Cellular Immunology Section, UCL Great Ormond Street Institute of Child Health, London, UK; Department of Pediatric Immunology, Great Ormond Street Hospital, London, UK.
³ Division of Bone Marrow Transplantation and Immune Deficiency, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, United States of America.
⁴ Division of Pediatric Hematology and Oncology, Ibaraki Children's Hospital, Mito, Japan.
⁵ Department of Pediatrics, Shimane University Faculty of Medicine, Izumo, Japan.
⁶ Department of Hematology/Oncology, Osaka Women's and Children's Hospital, Izumi, Japan.
⁷ Department of Hematology & Oncology, Children's Cancer Center, Hyogo Children's Hospital, Kobe, Japan.
⁸ Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK; Paediatric Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne, UK.
⁹ Deparment of Child Health and Development, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan. Electronic address: hkanegane.ped@tmd.ac.jp.

PMID: 35367395
DOI: 10.1016/j.clim.2022.108993

Abstract

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.

Keywords: Preemptive hematopoietic cell transplantation; X-linked lymphoproliferative syndrome type 1.

MeSH terms

Hematopoietic Stem Cell Transplantation* / methods
Humans
Lymphoproliferative Disorders* / diagnosis
Lymphoproliferative Disorders* / therapy
Prognosis
Retrospective Studies
Siblings
Transplantation, Homologous
United States