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Lanadelumab in Patients 2 to Less Than 12 Years Old With Hereditary Angioedema: Results From the Phase 3 SPRING Study
Marcus Maurer1, William R Lumry2, H Henry Li3
1Institute of Allergology, Charité-Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany; Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Immunology and Allergology, Berlin, Germany.
Insights
Lanadelumab significantly reduced hereditary angioedema (HAE) attacks and improved quality of life in children aged 2 to 12 years. The treatment demonstrated a 94.8% decrease in attack rates, with most patients remaining attack-free.
Area of Science:
- Immunology
- Pediatrics
- Pharmacology
Background:
- Hereditary angioedema (HAE) symptoms often begin in childhood, with severe attacks impacting quality of life.
- No long-term prophylaxis treatments are approved for children under 6 years old.
Purpose of the Study:
- To evaluate the safety, pharmacokinetics, and efficacy of lanadelumab in patients aged 2 to less than 12 years.
- To assess the impact of lanadelumab on health-related quality of life (HRQoL) in pediatric HAE patients.
Main Methods:
- The SPRING Study enrolled 21 pediatric patients with HAE.
- Patients aged 2-6 years received lanadelumab 150 mg every 4 weeks (Q4W); patients aged 6-12 years received 150 mg every 2 weeks (Q2W), with an option to switch to Q4W if attack-free for 26 weeks.
- Treatment duration was 52 weeks.
Main Results:
- No serious treatment-emergent adverse events (TEAEs) or discontinuations due to TEAEs were reported.
- A 94.8% reduction in HAE attack rate from baseline was observed (1.84 to 0.08 attacks/month).
- 16 out of 21 patients (76.2%) were attack-free, and significant improvements in HRQoL were noted.
Conclusions:
- Lanadelumab (150 mg Q2W and Q4W) is safe and effective for preventing HAE attacks in children aged 2 to 12 years.
- Treatment with lanadelumab leads to substantial improvements in health-related quality of life in this pediatric population.
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.
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