Extended clinical and immunological phenotype and transplant outcome in CD27 and CD70 deficiency

Blood. 2020 Dec 3;136(23):2638-2655. doi: 10.1182/blood.2020006738.

Abstract

Biallelic mutations in the genes encoding CD27 or its ligand CD70 underlie inborn errors of immunity (IEIs) characterized predominantly by Epstein-Barr virus (EBV)-associated immune dysregulation, such as chronic viremia, severe infectious mononucleosis, hemophagocytic lymphohistiocytosis (HLH), lymphoproliferation, and malignancy. A comprehensive understanding of the natural history, immune characteristics, and transplant outcomes has remained elusive. Here, in a multi-institutional global collaboration, we collected the clinical information of 49 patients from 29 families (CD27, n = 33; CD70, n = 16), including 24 previously unreported individuals and identified a total of 16 distinct mutations in CD27, and 8 in CD70, respectively. The majority of patients (90%) were EBV+ at diagnosis, but only ∼30% presented with infectious mononucleosis. Lymphoproliferation and lymphoma were the main clinical manifestations (70% and 43%, respectively), and 9 of the CD27-deficient patients developed HLH. Twenty-one patients (43%) developed autoinflammatory features including uveitis, arthritis, and periodic fever. Detailed immunological characterization revealed aberrant generation of memory B and T cells, including a paucity of EBV-specific T cells, and impaired effector function of CD8+ T cells, thereby providing mechanistic insight into cellular defects underpinning the clinical features of disrupted CD27/CD70 signaling. Nineteen patients underwent allogeneic hematopoietic stem cell transplantation (HSCT) prior to adulthood predominantly because of lymphoma, with 95% survival without disease recurrence. Our data highlight the marked predisposition to lymphoma of both CD27- and CD70-deficient patients. The excellent outcome after HSCT supports the timely implementation of this treatment modality particularly in patients presenting with malignant transformation to lymphoma.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Allografts
  • CD27 Ligand / deficiency*
  • Child
  • Child, Preschool
  • Disease-Free Survival
  • Female
  • Genetic Diseases, Inborn* / genetics
  • Genetic Diseases, Inborn* / immunology
  • Genetic Diseases, Inborn* / mortality
  • Genetic Diseases, Inborn* / therapy
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Immunologic Deficiency Syndromes* / genetics
  • Immunologic Deficiency Syndromes* / immunology
  • Immunologic Deficiency Syndromes* / mortality
  • Immunologic Deficiency Syndromes* / therapy
  • Infant
  • Male
  • Retrospective Studies
  • Survival Rate
  • Tumor Necrosis Factor Receptor Superfamily, Member 7 / deficiency*

Substances

  • CD27 Ligand
  • CD70 protein, human
  • Tumor Necrosis Factor Receptor Superfamily, Member 7