Efficacy and Safety of Subcutaneous Efgartigimod <scp>PH20</scp> in Adults With Primary Immune Thrombocytopenia ( <scp>ADVANCE SC</scp> ): A Multicenter, Randomized, Double‐Blinded, Placebo‐Controlled, Phase 3 Trial
/L for at least four of the six visits during weeks 19-24): -13.7% [17/124] versus 16.2% [11/68], respectively; p = 0.51; adjusted difference in proportions, -3.5% [95% CI, -14.7-7.0]. Efgartigimod PH20 SC was well tolerated: most adverse events were mild to moderate and comparable between treatment groups. Platelet count increases from baseline were higher than expected with placebo and lower than expected with efgartigimod PH20 SC. Clinical Trial Registration: The ADVANCE SC trial is registered on ClinicalTrials.gov (NCT04687072).
Authors
- Nichola Cooper (ORCID: https://orcid.org/0000-0002-9845-8292)
- J BUSSEL
- Catherine M. Broome (ORCID: https://orcid.org/0000-0003-1507-2851)
- Abderrahim Khélif (ORCID: https://orcid.org/0000-0003-0220-3592)
- Waleed Ghanima (ORCID: https://orcid.org/0000-0003-2225-6165)
- Rui Yang (ORCID: https://orcid.org/0000-0002-9384-8398)
- F Rodeghiero
- Hanny T. Al-Samkari (ORCID: https://orcid.org/0000-0001-6175-1383)
- Jaume Ayguasanosa
- Giorgia Ciurlia
- Vickie McDonald (ORCID: https://orcid.org/0000-0003-4392-8223)
- Filip Matthijssens (ORCID: https://orcid.org/0000-0002-6499-4572)
- Domenica Gandini
- Kristof De Beuf
- Anna Hultberg
- Yoshitaka Miyakawa (ORCID: https://orcid.org/0000-0002-4429-7227)
- Ségolène Pastouret
- the ADVANCE SC Investigator Trial Group
- Spero R. Cataland
- Wilma Barcellini
Institutions
- Oslo University Hospital (NO)
- Georgetown University (US)
- Chinese Academy of Medical Sciences & Peking Union Medical College (CN)
- Cornell University (US)
- Barts Health NHS Trust (GB)
- MedStar Georgetown University Hospital (US)
- The Ohio State University Wexner Medical Center (US)
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT)
- Massachusetts General Hospital (US)
- Hôpital Farhat Hached (TN)
- Saitama Medical University Hospital (JP)
- Wuhan Union Hospital (CN)
- Institute of Hematology & Blood Diseases Hospital (CN)
- Ospedale San Bortolo (IT)
- Argenx (Belgium) (BE)
- Union Hospital (CN)
- Imperial College London (GB)
Publication Details
- Journal
- American Journal of Hematology
- Published
- 2026-04-06
- DOI
- https://doi.org/10.1002/ajh.70303
- Primary Topic
- Platelet Disorders and Treatments
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Argenx