Details

Autor(en) / Beteiligte

• Clément, Marie Caroline, MD
• Mahlaoui, Nizar, MD, MSc, MPH
• Mignot, Cécile, MD, MPH
• Le Bihan, Christine, MD
• Rabetrano, Hasina, MSc
• Hoang, Ly, MSc
• Neven, Bénédicte, MD, PhD
• Moshous, Despina, MD, PhD
• Cavazzana, Marina, MD, PhD
• Blanche, Stéphane, MD
• Fischer, Alain, MD, PhD
• Audrain, Marie, MD, PhD
• Durand-Zaleski, Isabelle, MD, PhD

Titel

Systematic neonatal screening for severe combined immunodeficiency and severe T-cell lymphopenia: Analysis of cost-effectiveness based on French real field data

Ist Teil von

• Journal of allergy and clinical immunology, 2015-06, Vol.135 (6), p.1589-1593

Ort / Verlag

United States: Elsevier Inc

Erscheinungsjahr

2015

Link zum Volltext

Quelle

MEDLINE

Beschreibungen/Notizen

• Background The inclusion of severe combined immunodeficiency (SCID) in a Europe-wide screening program is currently debated. Objective In making a case for inclusion in the French newborn screening program, we explored the costs incurred and potentially saved by early management of SCID. Methods For test costs, a microcosting study documented the resources used in a laboratory piloting a newborn screening test on Guthrie cards using the T-cell receptor excision circle quantification method. For treatment costs, patients with SCID admitted to the national reference center for primary immunodeficiency in France between 2006 and 2010 were included. Costs of admission were estimated from actual national production costs. We estimated the costs for patients who underwent early versus delayed hematopoietic stem cell transplantation (HSCT; age, ≤3 vs >3 months, respectively). Results The unit cost of the test varied between €4.69 and €6.79 for 33,800 samples per year, depending on equipment use and saturation. Of the 30 patients included, 27 underwent HSCT after age 3 months. At 1 year after HSCT, 10 of these had died, and all 3 patients undergoing early transplantation survived. The medical costs for HSCT after 3 months were €195,776 (interquartile range, €165,884-€257,160) versus €86,179 (range, €59,014-€272,577) when performed before 3 months of age. In patients undergoing late transplantation, active infection contributed to high cost and poor outcome. Conclusion Early detection of SCID could reduce the cost of treatment by €50,000-100,000 per case. Assuming a €5 unit cost per test, the incidence required to break even is 1:20,000; however, if the survival advantage of HSCT before 3 months is confirmed, universal screening is likely to be cost-effective.