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Review
. 2023 Apr 5:14:1162605.
doi: 10.3389/fimmu.2023.1162605. eCollection 2023.

Personalized hematopoietic stem cell transplantation for inborn errors of immunity

Mary Slatter  1   2 , Su Han Lum  1   2
Affiliations
Review

Personalized hematopoietic stem cell transplantation for inborn errors of immunity

Mary Slatter et al. Front Immunol. .

Abstract

Patients with inborn errors of immunity (IEI) have been transplanted for more than 50 years. Many long-term survivors have ongoing medical issues showing the need for further improvements in how hematopoietic stem cell transplantation (HSCT) is performed if patients in the future are to have a normal quality of life. Precise genetic diagnosis enables early treatment before recurrent infection, autoimmunity and organ impairment occur. Newborn screening for severe combined immunodeficiency (SCID) is established in many countries. For newly described disorders the decision to transplant is not straight-forward. Specific biologic therapies are effective for some diseases and can be used as a bridge to HSCT to improve outcome. Developments in reduced toxicity conditioning and methods of T-cell depletion for mismatched donors have made transplant an option for all eligible patients. Further refinements in conditioning plus precise graft composition and additional cellular therapy are emerging as techniques to personalize the approach to HSCT for each patient.

Keywords: SCID-severe combined immunodeficiency; T cell depletion; conditioning; hematopoietic stem cell transplantation; inborn errors of immunity.

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Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

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References

    1. Gatti RA, Meuwissen HJ, Allen HD, Hong R, Good RA. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet (1968) 2(7583):1366–9. doi: 10.1016/S0140-6736(68)92673-1 - DOI - PubMed
    1. de Koning J, van der Waay D, Vossen JM, Versprille A, Dooren LJ. Barrier nursing of an infant in a laminar cross-flow bench. Maandschr Kindergeneeskd (1970) 38(1):1–13. doi: 10.1007/978-3-662-30498-3_8 - DOI - PubMed
    1. Bach FH, Albertini RJ, Joo P, Anderson JL, Bortin MM. Bone-marrow transplantation in a patient with the wiskott-Aldrich syndrome. Lancet (1968) 2(7583):1364–6. doi: 10.1016/S0140-6736(68)92672-X - DOI - PubMed
    1. Tangye SG, Al-Herz W, Bousfiha A, Chatila T, Cunningham-Rundles C, Etzioni A, et al. Human inborn errors of immunity: 2019 update on the classification from the international union of immunological societies expert committee. J Clin Immunol (2020) 40(1):24–64. doi: 10.1007/s10875-019-00737-x - DOI - PMC - PubMed
    1. Day JW, Elfeky R, Nicholson B, Goodman R, Pearce R, Fox TA, et al. Retrospective, landmark analysis of long-term adult morbidity following allogeneic HSCT for inborn errors of immunity in infancy and childhood. J Clin Immunol (2022) 42(6):1230–43. doi: 10.1007/s10875-022-01278-6 - DOI - PMC - PubMed

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