Costs associated with treatment of severe combined immunodeficiency (SCID) - rationale for newborn screening in Sweden

To the Editor: Severe combined immunodeficiency (SCID) is the most severe form of primary immunodeficiency with profound defects in both the cellular and humoral immune defence.1-3 The affected newborns are highly susceptible to life-threatening opportunistic bacterial, viral, and/or fungal infections.1-5 Early detection of SCID in the preinfectious period followed by hematopoietic stem cell transplantation (HSCT) has been shown to dramatically improve survival.1 This has led to the implementation of national newborn screening (NBS) programs in selected countries and pilot projects ongoing in many additional countries.6 Two studies from the United States and France of real-life costs for HSCT in SCID have been published and both showed that early HSCT resulted in lower treatment-related costs.7,8 The aim of this study was to use patient-unique, individual, real-life cost and care data to calculate costs for children with SCID undergoing HSCT in Sweden, and to identify types and percentage breakdown of the costs. [...]once a child has passed the first post-HSCT year and survived, the costs for health care were limited. The study also demonstrated important health benefits for the children and the families from early HSCT, such as reduction by more than half of in-hospital days 3 6 of 6 15 118 Year +4 0 0 0 0 3 of 3 4 of 6 9 101 Year +5 0 0 0 0 2 of 2 4 of 6 4 73 Total 663 1461 302 787 Table E3Outpatient clinic visits for each child for the entire cost follow-up period for children before and...