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Curr Allergy Asthma Rep. 2019 Nov 18;19(11):52. doi: 10.1007/s11882-019-0883-1.

Conditioning Regimens for Hematopoietic Cell Transplantation in Primary Immunodeficiency.

Author information

1
Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, UK.
2
Department of Pediatrics, University Medical Center Ulm, Ulm, Germany.
3
Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, UK.
4
Children's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne Hospital NHS Foundation Trust, Newcastle upon Tyne, UK. mary.slatter@nuth.nhs.uk.
5
Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, UK. mary.slatter@nuth.nhs.uk.

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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