Evidence map›Paper›PMID 40251904›Full record

Trial reportAllergy2025

Patient-Reported Outcomes in the Phase III OASIS-HAE Study of Donidalorsen for Hereditary Angioedema.

Marc A Riedl, Aaron Yarlas, Laura Bordone, Sabrina Treadwell, Sophie Wang, Kenneth B Newman, Danny M Cohn

Registry-linked trialAbstract readClinical Trial, Phase IIIRandomized Controlled TrialMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Allergy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05139810 (A Phase 3 Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients With Hereditary Angioedema), which is not on this map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT05139810 phase3completednot on this map

A Phase 3 Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients With Hereditary Angioedema (HAE)

TypeinterventionalSponsorIonis Pharmaceuticals, Inc.Ran2021 to 2023Enrolled91ConditionsHereditary AngioedemaArmsDonidalorsen, Placebo
3 · Its place in the literature

Who cites it

7 citing papers in PubMed.

  1. Trial
  2. Article
  3. Review
  4. Article
  5. Review
  6. Review
  7. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors.

Marc A RiedlDivision of Allergy and Immunology, University of California San Diego School of Medicine, La Jolla, California, USA.
Aaron YarlasIonis, Carlsbad, California, USA.
Laura BordoneIonis, Carlsbad, California, USA.
Sabrina TreadwellIonis, Carlsbad, California, USA.
Sophie WangIonis, Carlsbad, California, USA.
Kenneth B NewmanIonis, Carlsbad, California, USA.
Danny M CohnDepartment of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands.ORCID https://orcid.org/0000-0002-5067-7533

Funding

Ionis Pharmaceuticals
6 · The paper itself

Abstract

backgroundHereditary angioedema (HAE) is a rare disease characterized by unpredictable, frequently severe swelling that negatively impacts patients' quality of life (QoL). In the phase III OASIS-HAE study (NCT05139810), donidalorsen reduced HAE attack rate, increased disease control, and improved QoL. Here, we report further analysis of donidalorsen's impact on QoL and other patient-reported outcomes (PROs).

methodsThis double-blind, placebo-controlled study randomized patients with HAE to donidalorsen 80 mg or placebo once every 4 (Q4W) or 8 weeks (Q8W) over 24 weeks. PROs included Angioedema (AE)-QoL Questionnaire, Angioedema Control Test (AECT), Patient Global Impression of Severity (PGIS), and Work Productivity and Activity Impairment Questionnaire plus Classroom Impairment Questions (WPAI+CIQ).

resultsNinety patients received donidalorsen Q4W (n = 45), donidalorsen Q8W (n = 23), or placebo (n = 22). A larger percentage of the donidalorsen Q4W group (88%) achieved clinically meaningful improvement (≥ 6-point reduction) in AE-QoL total score vs. placebo (45%). Both donidalorsen groups reported larger least-squares mean (LSM) changes from baseline to week 24 vs. placebo in AE-QoL functioning (difference: Q4W, -24.5; Q8W, -16.1), fears/shame (Q4W, -23.9; Q8W, -20.1), and nutrition (Q4W, -15.7; Q8W, -10.7) domains. Donidalorsen improved AECT scores vs. placebo (difference: Q4W, 6.0; Q8W, 4.1). A greater proportion of the donidalorsen Q4W group reported decreased disease severity vs. the placebo group (PGIS; 82% vs. 44%). Donidalorsen Q4W showed benefits vs. placebo in the presenteeism, overall work/school impairment, and activity impairment domains of the WPAI+CIQ.

conclusionsDonidalorsen significantly improved QoL and other PROs vs. placebo in patients with HAE.

Indexed as

Angioedemas, HereditaryPatient Reported Outcome MeasuresAdultAgedDouble-Blind MethodFemaleHumansMaleMiddle AgedOligonucleotidesQuality of LifeSeverity of Illness IndexSurveys and QuestionnairesTreatment OutcomeYoung AdultdonidalorsenOligonucleotidesdisease controldonidalorsenhereditary angioedemapatient‐reported outcomesquality of life

Identifiers

PMID40251904
PMCPMC12368753

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.