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Review
. 2019 Nov 18;19(11):52.
doi: 10.1007/s11882-019-0883-1.

Conditioning Regimens for Hematopoietic Cell Transplantation in Primary Immunodeficiency

S H Lum  1 , M Hoenig  2 , A R Gennery  1   3 , M A Slatter  4   5
Affiliations
Review

Conditioning Regimens for Hematopoietic Cell Transplantation in Primary Immunodeficiency

S H Lum et al. Curr Allergy Asthma Rep. .

Abstract

Purpose of review: Hematopoietic cell transplantation (HCT) is an established curative treatment for children with primary immunodeficiencies. This article reviews the latest developments in conditioning regimens for primary immunodeficiency (PID). It focuses on data regarding transplant outcomes according to newer reduced toxicity conditioning regimens used in HCT for PID.

Recent findings: Conventional myeloablative conditioning regimens are associated with significant acute toxicities, transplant-related mortality, and late effects such as infertility. Reduced toxicity conditioning regimens have had significant positive impacts on HCT outcome, and there are now well-established strategies in children with PID. Treosulfan has emerged as a promising preparative agent. Use of a peripheral stem cell source has been shown to be associated with better donor chimerism in patients receiving reduced toxicity conditioning. Minimal conditioning regimens using monoclonal antibodies are in clinical trials with promising results thus far. Reduced toxicity conditioning has emerged as standard of care for PID and has resulted in improved transplant survival for patients with significant comorbidities.

Keywords: HCT outcome; Hematopoietic cell transplantation; Primary immunodeficiency; Reduced toxicity conditioning; Transplant-related survival.

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Conflict of interest statement

The authors declare no conflicts of interest relevant to this manuscript.

Figures

Fig. 1
Fig. 1
Intensity of conditioning regimen according to chemotherapy, pharmacokinetic guided dosing, timing of serotherapy, and combination of chemotherapy
See this image and copyright information in PMC

References

    1. Gatti RA, Meuwissen HJ, Allen HD, Hong R, Good RA. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet. 1968;2(7583):1366–1369. - 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):1364–1366. - PubMed
    1. Slatter MA, Gennery AR. Hematopoietic cell transplantation in primary immunodeficiency - conventional and emerging indications. Expert Rev Clin Immunol. 2018;14(2):103–114. - PubMed
    1. Slatter MA, Gennery AR. Advances in hematopoietic stem cell transplantation for primary immunodeficiency. Expert Rev Clin Immunol. 2013;9(10):991–999. - PubMed
    1. Shah RM, Elfeky R, Nademi Z, Qasim W, Amrolia P, Chiesa R, et al. T-cell receptor alphabeta(+) and CD19(+) cell-depleted haploidentical and mismatched hematopoietic stem cell transplantation in primary immune deficiency. J Allergy Clin Immunol. 2018;141(4):1417–1426. - PubMed

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