Early Treatment of Severe Combined Immunodeficiency May Be Beneficial in Newborns

Laurie Barclay, MD

April 23, 2010

April 23, 2010 — Early treatment of severe combined immunodeficiency (SCID) may be beneficial, and population-based newborn screening may be possible, according to the results of a systematic evidence review reported online April 19 and to be published in the May issue of Pediatrics.

"...SCID is a group of disorders that leads to early childhood death as a result of severe infections," write Ellen A. Lipstein, MD, MPH, from Cincinnati Children's Hospital Medical Center in Cincinnati, Ohio, and colleagues. "Recent research has addressed potential newborn screening for SCID."

To perform a systematic review of the evidence for newborn screening for SCID, including test characteristics, treatment efficacy, and cost-effectiveness, the investigators searched MEDLINE and the OVID In-Process & Other Non-Indexed Citations databases. Reviews, editorials or other opinion pieces, case series of fewer than 4 patients, studies of only adult subjects, and studies on nonhuman data were excluded.

After systematic assessment of the remaining articles, 2 independent reviewers used standardized tools to abstract data. The investigators also interviewed appropriate experts for topics lacking published evidence. Of 719 articles initially identified, 26 met inclusion criteria.

Screening for SCID is possible, based on evidence from several small studies, and experts interviewed indicated that 2 US states have begun pilot screening programs. However, there are few data regarding the cost-effectiveness of a SCID screening program.

Compared with children who were treated later, children receiving early stem-cell transplant for SCID had better outcomes, according to findings of large case series. Evidence regarding the need for donor-recipient matching and use of pretransplant chemotherapy is inconclusive.

"Evidence indicates the benefits of early treatment of SCID and the possibility of population-based newborn screening," the study authors write. "Better information on optimal treatment and the costs of treatment and screening would benefit policy makers deciding among competing health care priorities."

Limitations of this review include those inherent in the included studies and inability to perform meta-analysis.

"As tests and treatments are developed for disorders that are even less common than SCID, the questions, particularly with regard to treatment efficacy, screening accuracy, and cost-effectiveness, will likely become even more difficult to answer," the study authors write. "Establishing a precedent and a system for careful evaluation of the evidence, before widespread implementation of such screening, should be a priority for policy makers and child health advocates alike and may help develop a system for comparing the value of screening for diverse disorders."

Dr. Lipstein's effort was supported by the US Department of Health and Human Services, Health Resources and Services Administration. The other study authors have disclosed no relevant financial relationships.

Pediatrics. Published online April 19, 2010. Abstract

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