Abstract
Congenital disorders of the immune system affecting maturation and/or function of phagocytic leucocytes can result in severe infectious and inflammatory complications with high mortality and morbidity. Further complications include progression to MDS/AML in some cases. Allogeneic stem cell transplantation is the only curative treatment for most patients with these diseases. In this review, we provide a detailed update on indications and outcomes of alloHSCT for congenital neutrophil disorders, based on data from the available literature.
Highlights
Congenital neutrophil disorders as a category of primary immunodeficiency (PID) can be classified in many ways, but a key point of distinction is whether the disorder is quantitative, or qualitative [1]
The 2017 International Union of Immunological Societies (IUIS) Phenotypic Classification for Primary Immunodeficiencies divides neutrophil disorders into four broad categories: congenital neutropenia associated with or without syndromic disease, and functional neutrophil defects with or without syndromic disease [2]
In this review we provide an update on the evidence, indications and modalities of alloHSCT for the various congenital neutrophil disorders based on data from the available literature, excluding Chronic Granulomatous Disease (CGD) which will be discussed elsewhere in this special edition
Summary
Congenital neutrophil disorders as a category of primary immunodeficiency (PID) can be classified in many ways, but a key point of distinction is whether the disorder is quantitative, or qualitative [1]. Some patients develop severe autoinflammatory complications underlining the role of neutrophils in autoinflammatory processes beyond microbial defense [5, 6]. In many of these diseases there is a recognized risk of progression to myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) [1, 2, 7, 8]. Treatment for neutrophil disorders classically comprises anti-microbial therapy, granulocyte-colony stimulating factor (G-CSF), and allogeneic hematopoietic stem cell transplantation (alloHSCT) [8]
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