Neurocognitive Function of Patients with Severe Combined Immunodeficiency

Shah, Ami J.; Kohn, Donald B.

doi:10.1016/j.iac.2009.10.003

Cited by 2 publications

(2 citation statements)

References 32 publications

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“…Fortunately, outcomes appear to be quite good, and future work to optimize HSCT will focus largely on the quality of the graft and the quality of life. Emerging data on neurocognitive effects will require specific attention, for example (46, 47). This study highlights the frequent need for re‐transplantation after MMRD and confirms a potential interaction between a disease and type of transplant.…”

Section: Discussionmentioning

confidence: 99%

A single‐center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD)

DiNardo¹,

Brown²,

Perez

et al. 2011

Pediatric Transplantation

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PIDD are rare inherited disorders that can result in life-threatening infections. Allogeneic HSCT is the only cure for many primary immune deficiencies; however, the specific diseases and optimal type(s) of transplants are not clear. This study compares transplant outcomes in a large cohort with a relatively uniform pre- and post-transplant management strategies. We conducted a retrospective analysis of 39 pediatric patients who underwent HSCT for SCID (n = 25) or other immune deficiencies (n = 14) from 1986 to 2010. A structured case report form was used to collect clinical information. The outcomes of survival, immune reconstitution, engraftment, incidence of GvHD and IVIG dependency were tabulated. Overall survival rates were 88% for SCID and 86% for other primary immune deficiencies, which are high compared to other historical series. No single variable was associated with mortality. Immunoglobulin dependence occurred only in patients who had X-linked SCID and a parental donor haploidentical transplant. Because of improved supportive care and use of alternative donors and conditioning regimens, HSCT has become an acceptable option for an increasing number of PIDD subtypes not previously transplanted with high frequency. This study encourages greater use of transplantation.

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Section: Discussionmentioning

confidence: 99%

A single‐center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD)

DiNardo¹,

Brown²,

Perez

et al. 2011

Pediatric Transplantation

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“…Cognitive effects are related to many factors, including the intrinsic genetic defect (e.g., ADA-SCID and some types of RS-SCID), psychosocial factors such as socioeconomic status, the duration and severity of illness / hospitalization, and possibly direct CNS toxicity from conditioning. (45, 46) Given the general observation that the incidence and severity of cognitive deficits increases when radiation is given to very young infants,(47) alkylating chemotherapy such as busulfan, which readily crosses the blood-brain-barrier, should be administered with caution. Close follow-up, including a full battery of age-appropriate neurocognitive testing, should be done biannually through childhood.…”

Section: Toxicitymentioning

confidence: 99%

Hematopoietic Stem Cell Transplantation for Severe Combined Immunodeficiency

2015

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Hematopoietic stem cell transplantation (HSCT) is an effective approach for the treatment of severe combined immunodeficiency (SCID). However, SCID is not a homogeneous disease, and the treatment required for successful transplantation varies significantly between SCID subtypes and the degree of HLA mismatch between the best available donor and the patient. Recent studies are beginning to more clearly define this heterogeneity and how outcomes may vary. With a more detailed understanding of SCID, new approaches can be developed to maximize immune reconstitution, while minimizing acute and long-term toxicities associated with chemotherapy conditioning.

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Neurocognitive Function of Patients with Severe Combined Immunodeficiency

Cited by 2 publications

References 32 publications

A single‐center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD)

A single‐center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD)

Hematopoietic Stem Cell Transplantation for Severe Combined Immunodeficiency

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