# Initial Experience of Long-Term Prophylaxis with Lanadelumab for Hereditary Angioedema in China: A Clinical Observation Study on Six Patients

- **DOI:** [10.1159/000541242](https://doi.org/10.1159/000541242)
- **Rating:** Limited (2.8 out of 5)
- **Verdict:** A useful first mainland Chinese lanadelumab series with detailed patient-level outcomes, but its uncontrolled six-patient design cannot validate efficacy or dose-spacing guidance.
- **Framework version:** v5.9
- **Pipeline version:** v0.4
- **Evaluation date:** 2026-09-18
- **Evaluation page:** https://www.nabu.science/paper/10.1159%2F000541242

## Key claims

1. Lanadelumab led to reductions of 97.8% and 98.5% in the attack rate and treated attack rate, respectively.
   - Support: Tentative
   - Type: Primary finding
   - Basis: uncontrolled six-patient pre/post design limits causal attribution despite internally consistent attack totals
   - Location: Abstract; Results, Table 3 and Figure 1
2. 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).
   - Support: Supported
   - Type: Secondary
   - Basis: complete within-cohort reductions are reported, but self-reporting and the absence of a control limit inference
   - Location: Abstract; Results, Figures 2–4 and Table 3
3. The efficacy of lanadelumab remained stable during COVID-19 vaccination and infection.
   - Support: Tentative
   - Type: Secondary
   - Basis: uncontrolled observations plus an undocumented untreated comparator limit attribution during vaccination and infection
   - Location: Abstract; Results, Interaction with COVID-19; Discussion, p. 229
4. No serious/severe treatment-emergent adverse events occurred during lanadelumab treatment.
   - Support: Supported
   - Type: Secondary
   - Basis: 91 injections support the observed absence, but the sample is too small for general safety inference
   - Location: Abstract; Results, Safety of LTP with Lanadelumab; Table 4

## Strengths

- **Detailed patient-level clinical documentation** Tables 1–3 report diagnostic characteristics, genetic findings, injection schedules, and individual attack and work-loss counts, allowing the principal totals to be reconstructed.
  - Dimension: methodological rigour
  - Location: Tables 1–3, pp. 224–226
- **Documented mainland-China evidence gap** The Introduction distinguishes the absence of mainland-Chinese experience from existing randomized and regional evidence, including reports from Hong Kong and Japan.
  - Dimension: contribution
  - Location: Introduction, pp. 222–223; Discussion, p. 227
- **Visible local clinical application pathway** The Discussion identifies economic and access barriers and proposes explicit criteria for considering prophylaxis, giving Chinese HAE clinicians a recognizable decision context.
  - Dimension: application pathway
  - Location: Introduction, p. 223; Discussion, p. 226

## Limitations

- **Causal conclusions exceed study design** 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.
  - Dimension: methodological rigour
  - Location: Abstract, p. 221; Methods, pp. 222–223; Discussion, pp. 228–229
- **COVID comparator lacks reported methods** 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.
  - Dimension: positioning
  - Location: Discussion, COVID-19 paragraph, p. 229
- **Ethics timing remains unexplained** Recruitment began in April 2022, while Approval No. 20230929 appears later; although approval and consent are declared, the retrospective oversight timeline is not explained.
  - Dimension: methodological rigour
  - Location: Results, Patient Characteristics, p. 223; Statement of Ethics, p. 229

## Reliability checks

Caveats (4 of 4 checks).

- **Internal coherence:** Noted. 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.
- **Research conduct:** Noted. 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.
- **Reference integrity:** Clean. 37 of 37 checkable references verified
  - 39 references in manuscript
  - 2 are books, websites or datasets — counted, but not index-checkable
  - References not verified automatically are resolved manually within 24 hours.

## Post-publication record

- **Status:** Clean
- **Summary:** No retraction or expression of concern found in Crossref (includes Retraction Watch).
- **Last checked:** 2026-10-17