2019
DOI: 10.3389/fped.2019.00434
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Conditioning Perspectives for Primary Immunodeficiency Stem Cell Transplants

Abstract: The majority of children undergoing Hematopoietic Stem cell Transplantation (HSCT) require conditioning therapy to make space and prevent rejection of the donor stem cells. The exception is certain children with Severe Combined immune deficiency, who have limited or no ability to reject the donor graft. Transplant conditioning is associated with significant morbidity and mortality from both direct toxic effects of chemotherapy as well as opportunistic infections associated with profound immunosuppression. The … Show more

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Cited by 31 publications
(36 citation statements)
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“…Although the level of chimerism needed to correct IEIs is not precisely defined, most IEIs do not require full donor chimerism, explaining why modified myeloablative and reduced intensity conditioning has become the practice of choice in many centers to reduce acute and long-term toxicity 185 .…”
Section: Conditioningmentioning
confidence: 99%
“…Although the level of chimerism needed to correct IEIs is not precisely defined, most IEIs do not require full donor chimerism, explaining why modified myeloablative and reduced intensity conditioning has become the practice of choice in many centers to reduce acute and long-term toxicity 185 .…”
Section: Conditioningmentioning
confidence: 99%
“…Modern day regimens are typically classified into three categories (3,5,6). Myeloablative regimens typically requires a stem cell graft infusion to reconstitute hematopoiesis.…”
Section: Individual Agents Utilized For Preparative Regimen Designmentioning
confidence: 99%
“…This transformation is a product of many advancements including: (1) Improving our understanding of hematopoiesis and immune reconstitution. (2) Improvements in supportive care, (3) Improvements in the prevention of graft-versus-host disease (GVHD), (4) Expansion of donor pools, (5) Refinements in preparative regimen selection and design. These advancements have produced a steady decline in transplant related mortality rates which now approach 10% in some instances.…”
Section: Introductionmentioning
confidence: 99%
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“…5 However, conditioning regimens may cause acute and chronic toxicity, including organ damage, risk of infections, and infertility. 10 These considerations are particularly important for RAG deficiency manifesting as combined immunodeficiency with immune dysregulation (CID-ID), in which the unusual clinical presentation often leads to delayed diagnosis and progressive development of organ damage. [11][12][13] In these patients, the mortality rate reported after HSCT is higher than the rate observed in patients with typical or atypical SCID.…”
mentioning
confidence: 99%