Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia

SCIENTIFIC ABSTRACT Severe combined immunodeficiency (SCID) due to deficiency of the purine metabolic enzyme adenosine deaminase (ADA) is a fatal childhood immunodeficiency disease. Immune reconsti...

The ADA human gene therapy clinical protocol: Points to Consider response with clinical protocol, July 6, 1990.

Primary immunodeficiencies (PIDs) primarily affecting the phagocytes (neutrophils and macrophages) typically predispose patients to infections. However, one of the most clinically important features of these disorders is their relatively narrow spectrum of disease-specific infections. Invasive aspergillosis in the absence of immune suppression is essentially seen only in chronic granulomatous disease; disseminated nontuberculous mycobacterial infection in the absence of immune suppression is seen predominantly in patients with defects of the IFN-gamma/IL-12 axis. In contrast, infections that are relatively common in some of the PIDs affecting the lymphoid system (Pneumocystis jiroveci and Streptococcus pneumoniae) are extremely uncommon in PIDs affecting phagocytes. Therefore careful attention to the microbiology laboratory early in the course of evaluation of a patient with recurrent infections and suspected of having a PID will help steer the workup in the appropriate direction. Over the last few years, there have been major advances in the molecular and cellular understandings of PIDs affecting phagocytes. As the field of PIDs becomes broader and more clinical and molecular definition becomes available, it is increasingly important to be able to identify likely pathways for investigation early in the evaluation. Here we have updated some of the more rapidly evolving aspects of PIDs affecting phagocytes, with a special emphasis on the associated microbiology.

https://www.jacionline.org/article/S0091-6749(04)00933-9/pdf

Phagocyte immunodeficiencies and their infections

Bone marrow transplantation (BMT) in Europe for primary immunodeficiencies other than severe combined immunodeficiency: a report from the European Group for BMT and the European Group for Immunodeficiency

Reduction of Graft Failure by a Monoclonal Antibody (Anti-LFA-1 CD11a) After HLA Nonidentical Bone Marrow Transplantation in Children With Immunodeficiencies, Osteopetrosis, and Fanconi’s Anemia: A European Group for Immunodeficiency/European Group for Bone Marrow Transplantation Report

Successful HLA nonidentical bone marrow transplantation in three patients with the leukocyte adhesion deficiency.

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Successful HLA nonidentical bone marrow transplantation in three patients with the leukocyte adhesion deficiency.