Matched related donor hematopoietic stem cell transplantation protocol for patients with primary immunodeficiency

The exact role of factors affecting the outcome of hematopoietic stem cell transplantation (HSCT) are difficult to assess. Some determinants like genotype and pathway affected are inherently hard to evaluate in rare disorders, such as severe combined immunodeficiency (SCID). Comparison of outcomes even within an institution has been challenging because of the use of multiple inconsistent protocols. To eliminate this possibility in our cohort of patients, we have established detailed protocols for various indications. For patients with primary immunodeficiency (PID) in whom residual function is present and who have a human leukocyte antigen (HLA) matched related donor (MRD), we use the HSCT protocol that has previously been published for HLA matched unrelated donors (Kim 2016). Here, we present a HSCT protocol for patients with SCID who do not have T cells or NK cells, and who have a MRD (Table 1). We hope this approach could be beneficial, especially to new faculty and budding HSCT programs.

Statement of novelty: This report describes the protocols for HSCT using matched related donors for patients with primary immunodeficiency at our institution.