TY - JOUR
T1 - Lanadelumab in Patients 2 to Less Than 12 Years Old With Hereditary Angioedema
T2 - Results From the Phase 3 SPRING Study
AU - SPRING Investigators
AU - Maurer, Marcus
AU - Lumry, William R.
AU - Li, H. Henry
AU - Aygören-Pürsün, Emel
AU - Busse, Paula J.
AU - Jacobs, Joshua
AU - Nurse, Christina
AU - Ahmed, Mariam A.
AU - Watt, Maureen
AU - Yu, Ming
AU - Yang, W. H.
AU - Aygören-Pürsün, E.
AU - Martinez-Saguer, I.
AU - Maurer, M.
AU - Farkas, H.
AU - Perosa, M.
AU - Bernstein, J.
AU - Busse, P.
AU - Jacobs, J. S.
AU - Li, H. H.
AU - Lumry, W. R.
AU - Rehman, S. M.
AU - Tachdjian, R.
AU - Wedner, H. J.
AU - Weinstein, M. E.
N1 - Publisher Copyright:
© 2023 The Authors
PY - 2024/1
Y1 - 2024/1
N2 - Background: Symptoms of hereditary angioedema (HAE) often first occur during childhood, and HAE attacks in children can be severe and substantially affect health-related quality of life (HRQoL). However, there are no approved long-term prophylaxis treatments for children aged less than 6 years. Objective: The SPRING Study (NCT04070326) evaluated the safety, pharmacokinetics, and efficacy of lanadelumab and HRQoL in patients aged 2 to less than 12 years. Methods: Over 52 weeks of treatment, patients aged 2 to less than 6 years received lanadelumab 150 mg every 4 weeks (Q4W) and patients aged 6 to less than 12 years received 150 mg every 2 weeks (Q2W) but could switch to Q4W if they were attack-free for 26 weeks. Results: We enrolled 21 patients (aged 2 to less than 6 years: n = 4; aged 6 to less than 12 years: n = 17), 20 of whom completed the study. There were no reported serious treatment-emergent adverse events or discontinuations resulting from such events. Treatment-emergent adverse events were reported for 17 patients (81.0%). The most common TEAE was injection site pain. Overall systemic exposure was comparable for both age groups. The mean (SD) attack rate during treatment decreased by 94.8% from baseline (1.84 [1.53] to 0.08 [0.17] attacks/mo), and 16 (76.2%) patients were attack-free. The attack rate reduction in both age groups was similar during the first 26-week fixed-dosing treatment. Seven patients switched from Q2W to Q4W and remained attack-free. A large, clinically meaningful increase in the Pediatric Quality of Life Inventory Generic Core Scale Total Score and a large increase in the Pediatric Quality of Life Inventory Generic Core Scale–Family Impact Module Total Score from baseline to end of study (better HRQoL) were observed. Conclusions: Findings support safety, efficacy, and improved HRQoL with lanadelumab 150 mg Q2W and Q4W regimens for the prevention of HAE attacks in patients aged 2 to less than 12 years.
AB - Background: Symptoms of hereditary angioedema (HAE) often first occur during childhood, and HAE attacks in children can be severe and substantially affect health-related quality of life (HRQoL). However, there are no approved long-term prophylaxis treatments for children aged less than 6 years. Objective: The SPRING Study (NCT04070326) evaluated the safety, pharmacokinetics, and efficacy of lanadelumab and HRQoL in patients aged 2 to less than 12 years. Methods: Over 52 weeks of treatment, patients aged 2 to less than 6 years received lanadelumab 150 mg every 4 weeks (Q4W) and patients aged 6 to less than 12 years received 150 mg every 2 weeks (Q2W) but could switch to Q4W if they were attack-free for 26 weeks. Results: We enrolled 21 patients (aged 2 to less than 6 years: n = 4; aged 6 to less than 12 years: n = 17), 20 of whom completed the study. There were no reported serious treatment-emergent adverse events or discontinuations resulting from such events. Treatment-emergent adverse events were reported for 17 patients (81.0%). The most common TEAE was injection site pain. Overall systemic exposure was comparable for both age groups. The mean (SD) attack rate during treatment decreased by 94.8% from baseline (1.84 [1.53] to 0.08 [0.17] attacks/mo), and 16 (76.2%) patients were attack-free. The attack rate reduction in both age groups was similar during the first 26-week fixed-dosing treatment. Seven patients switched from Q2W to Q4W and remained attack-free. A large, clinically meaningful increase in the Pediatric Quality of Life Inventory Generic Core Scale Total Score and a large increase in the Pediatric Quality of Life Inventory Generic Core Scale–Family Impact Module Total Score from baseline to end of study (better HRQoL) were observed. Conclusions: Findings support safety, efficacy, and improved HRQoL with lanadelumab 150 mg Q2W and Q4W regimens for the prevention of HAE attacks in patients aged 2 to less than 12 years.
KW - Health-related quality of life
KW - Hereditary angioedema
KW - Lanadelumab
KW - Long-term prophylaxis
KW - Pediatrics
KW - Pharmacodynamic
KW - Pharmacokinetics
UR - http://www.scopus.com/inward/record.url?scp=85173217152&partnerID=8YFLogxK
U2 - 10.1016/j.jaip.2023.09.009
DO - 10.1016/j.jaip.2023.09.009
M3 - Article
C2 - 37730089
AN - SCOPUS:85173217152
SN - 2213-2198
VL - 12
SP - 201-211.e6
JO - Journal of Allergy and Clinical Immunology: In Practice
JF - Journal of Allergy and Clinical Immunology: In Practice
IS - 1
ER -