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INTRODUCTION: Hereditary angioedema (HAE) is a rare genetic disorder caused by deficiency or dysfunction of C1-esterase inhibitor that is characterized by recurrent episodes of bradykinin-mediated edema. Lanadelumab has been the only available first-line therapy for long-term prophylaxis (LTP) of HAE in China since its approval in 2020. The present study aimed to investigate the clinical efficacy and safety of lanadelumab for LTP in Chinese patients. METHODS: A retrospective clinical data were collected for the 6 patients and used to examine the frequency of attack symptoms, disease-related loss of work days, and quality of life before and after LTP with lanadelumab. Health-related quality of life was assessed using the Dermatology Life Quality Index (DLQI) and the Angioedema Quality of Life Questionnaire (AE-QoL). RESULTS: Lanadelumab led to reductions of 97.8% and 98.5% in the attack rate and treated attack rate, respectively. All patients exhibited significant improvements in AE-QoL and DLQI scores (100% reduction rates) during the early treatment period (4 weeks and 2 weeks, respectively) and in missed work days/year (98.9% reduction rate). The efficacy of lanadelumab remained stable during COVID-19 vaccination and infection. No serious/severe treatment-emergent adverse events occurred during lanadelumab treatment. CONCLUSION: This study is the first report that demonstrates the clinical efficacy of lanadelumab and safety of LTP in HAE patients from Chinese mainland. A reasonable dosage plan can ensure a quick and long-lasting protective role of lanadelumab against HAE attacks, during COVID-19 pandemic period.
Lanadelumab led to reductions of 97.8% and 98.5% in the attack rate and treated attack rate, respectively.
uncontrolled six-patient pre/post design limits causal attribution despite internally consistent attack totals
All patients exhibited significant improvements in AE-QoL and DLQI scores (100% reduction rates) during the early treatment period (4 weeks and 2 weeks, respectively) and in missed work days/year (98.9% reduction rate).
complete within-cohort reductions are reported, but self-reporting and the absence of a control limit inference
The efficacy of lanadelumab remained stable during COVID-19 vaccination and infection.
uncontrolled observations plus an undocumented untreated comparator limit attribution during vaccination and infection
No serious/severe treatment-emergent adverse events occurred during lanadelumab treatment.
91 injections support the observed absence, but the sample is too small for general safety inference
Derived from the full evaluation — not a separate score.
Strengths
Tables 1–3 report diagnostic characteristics, genetic findings, injection schedules, and individual attack and work-loss counts, allowing the principal totals to be reconstructed.
↳ Tables 1–3, pp. 224–226
The Introduction distinguishes the absence of mainland-Chinese experience from existing randomized and regional evidence, including reports from Hong Kong and Japan.
↳ Introduction, pp. 222–223; Discussion, p. 227
The Discussion identifies economic and access barriers and proposes explicit criteria for considering prophylaxis, giving Chinese HAE clinicians a recognizable decision context.
↳ Introduction, p. 223; Discussion, p. 226
Limitations
An uncontrolled retrospective series of six selected patients cannot establish that lanadelumab caused the full observed reductions or that the proposed dose-spacing regimen ensures protection.
↳ Abstract, p. 221; Methods, pp. 222–223; Discussion, pp. 228–229
The Discussion reports a 23.1% attack rate among untreated hospital patients without defining that cohort, its sample size, ascertainment, or analysis, weakening the COVID-related claim.
↳ Discussion, COVID-19 paragraph, p. 229
Recruitment began in April 2022, while Approval No. 20230929 appears later; although approval and consent are declared, the retrospective oversight timeline is not explained.
↳ Results, Patient Characteristics, p. 223; Statement of Ethics, p. 229
Tables 1–4 provide unusually granular reporting for a six-patient case series, and the principal attack totals reconcile with the stated reductions. The study also occupies a genuine mainland-China evidence gap and situates its observations against randomized and regional real-world studies. The main score constraint is that an uncontrolled retrospective series is used to claim demonstrated efficacy and to recommend dose-spacing schedules that “can ensure” protection. The COVID-19 interpretation is further weakened by an untreated hospital comparator introduced only in the Discussion without methods or cohort details.
Nabu’s assessment, alongside the field’s view.
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Limited2.8
Confidence mediumThe study adds the first reported mainland-Chinese clinical series to an established international evidence base and contributes local access and dose-spacing observations. Its advance remains geographically narrow and confirmatory because it includes only six patients.
“clinical application of lanadelumab in Chinese HAE patients has not been reported till now”
Tables 1–4 provide reconstructable patient characteristics, injection schedules, attack counts, work loss, and adverse events. The uncontrolled retrospective design does not exclude natural variation or reporting effects, yet the Discussion advances causal efficacy and dose-spacing conclusions.
“extension of the treatment interval after 4 months was safe”
The article follows a conventional methods-to-results structure and presents the numerical outcomes clearly. Central wording that the study “demonstrates” efficacy and that a regimen “can ensure” protection conveys greater certainty than the design supports.
“A reasonable dosage plan can ensure a quick and long-lasting protective role”
The Discussion compares the findings with pivotal, extension, Western, Hong Kong, and Japanese studies and names several genuine limitations. Contextual discipline is reduced by untested cross-study superiority language and by specific dosing guidance despite acknowledging that definitive conclusions require more data.
“The treatment course and reduction regimen for lanadelumab require more data to reach definitive conclusions.”
Caveats4 of 4 checks
The principal patient-level totals reconcile with the reported reductions, but the COVID-19 comparison introduces an undocumented untreated cohort. This affects the COVID-related interpretation rather than the core attack-count arithmetic.
Ethics approval, written consent, conflicts, funding roles, and data availability are declared. The approval number appears to postdate the start of recruitment, and the retrospective timing is not explained.
Flags: 3 declared / 5 total
37 of 37 checkable references verified
39 references in manuscript 2 are books, websites or datasets — counted, but not index-checkable
No retraction notice found in Retraction Watch.
Sources: Retraction Watch ✓
Where this paper’s evidence sits on the path from initial observation to real-world use.
The treatment was observed in routine hospital practice, and the article supplies concrete interval-extension schedules. The evidence remains preliminary because those schedules derive from six uncontrolled cases and are acknowledged as requiring more data.
“require more data to reach definitive conclusions”
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