4A01.12

Major histocompatibility complex class II deficiency

Deficiência major de classe II do complexo de histocompatibilidade

Category

Definition

Combined immune deficiency associated with the lack of HLA class II molecules is an autosomal recessive inherited disease. Failure to express HLA class II molecules causes a combined deficiency of cell-mediated immune responses (CD4 lymphopenia and impaired response to antigens) and antibody-mediated immune responses (hypogammaglobulinemia). As a result, patients are exposed to recurrent viral and bacterial infections, often leading to chronic diarrhoea and growth retardation. The disease is caused by mutations in four proteins involved in the regulation of HLA class II molecules gene transcription. Less than 100 cases have been reported worldwide. Patients can be cured with bone marrow transplantations of haematopoietic stem cells.

Index Terms

Major histocompatibility complex class II deficiencyImmunodeficiency by defective expression of HLA class 2HLA class 2-negative severe combined immunodeficiencyBare lymphocyte syndrome type 2