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Conditioning regimens for PID recommended by EBMT/ESID inborn errors working party [14]. Protocol A and B: These are recommended for patients without severe preexisting organ damage and non-SCID diseases where a complete donor chimerism is desired for optimal disease correction. Protocols C and D: These are recommended for patients with preexisting organ damage and/or diseases where engraftment has been shown to reliably occur with reduced intensity conditioning. Mixed donor chimerism is more likely to occur compared to protocols A and B. Protocol E: This may be best suited for patients with preexisting organ damage and/or diseases where full myeloid engraftment is not absolutely required. Higher degrees of chimerism can be achieved when using PBSC. DLI may be required in case of mixed chimerism. Protocol F: To avoid organ toxicity this regimen is only recommended for patients with DNA repair/radio-sensitivity disorders (except Artemis deficiency) in which alkylating agents are used in low dose. PID, primary immune deficiencies; EBMT, European Society for Blood and Marrow Transplantation; ESID, European Society for Immune Deficiencies; SCID, severe combined immunodeficiency; PBSC, peripheral blood stem cell; DLI, donor lymphocyte infusion.
Relationship between age and disease phase. (A) Number of patients by disease phase. (B) Number of patients with disease phase by age group.
Clin Pediatr Hematol Oncol 2023;30:53-104 |
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