Ianalumab plus Eltrombopag in Immune Thrombocytopenia
1
Issued Date
2026-04-16
Resource Type
eISSN
15334406
Scopus ID
2-s2.0-105035872303
Pubmed ID
41363800
Journal Title
New England Journal of Medicine
Volume
394
Issue
15
Start Page
1503
End Page
1513
Rights Holder(s)
SCOPUS
Bibliographic Citation
New England Journal of Medicine Vol.394 No.15 (2026) , 1503-1513
Suggested Citation
Cuker A., Stauch T., Cooper N., Al-Samkari H., Michel M., Ghanima W., Urban P., Fronczek J., Foster M., Weill M., Zhang L., Hou M., Zander T., Sharif A., Sun J., Nath U.K., Schutgens R., Rossi E., Deleu L., Červinek L., Yoon J.H., Chang H., Ruchutrakool T., Iino M., Goto T., Zaja F. Ianalumab plus Eltrombopag in Immune Thrombocytopenia. New England Journal of Medicine Vol.394 No.15 (2026) , 1503-1513. 1513. doi:10.1056/NEJMoa2515168 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/116385
Title
Ianalumab plus Eltrombopag in Immune Thrombocytopenia
Author's Affiliation
Harvard Medical School
Imperial College London
Universitetet i Oslo
University of Pennsylvania Perelman School of Medicine
Chang Gung Memorial Hospital
Uniklinik Köln
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Hôpital Henri Mondor
Novartis International AG
Nanfang Hospital, Southern Medical University
Qilu Hospital of Shandong University
Universitätsklinikum Jena und Medizinische Fakultät
Siriraj Hospital
The Catholic University of Korea Seoul St. Mary's Hospital
Fakultni Nemocnice Brno
Novartis Pharmaceuticals Corporation
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
All India Institute of Medical Sciences Rishikesh
Chang Gung University School of Medicine
Azienda Sanitaria Universitaria Giuliano Isontina
Japanese Red Cross Nagoya Daiichi Hospital
Yamanashi Prefectural Central Hospital
Van Creveldkliniek
AZ Delta
Østfold Hospital Trust
Sultanah Aminah Johor Bahru
Imperial College London
Universitetet i Oslo
University of Pennsylvania Perelman School of Medicine
Chang Gung Memorial Hospital
Uniklinik Köln
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Hôpital Henri Mondor
Novartis International AG
Nanfang Hospital, Southern Medical University
Qilu Hospital of Shandong University
Universitätsklinikum Jena und Medizinische Fakultät
Siriraj Hospital
The Catholic University of Korea Seoul St. Mary's Hospital
Fakultni Nemocnice Brno
Novartis Pharmaceuticals Corporation
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
All India Institute of Medical Sciences Rishikesh
Chang Gung University School of Medicine
Azienda Sanitaria Universitaria Giuliano Isontina
Japanese Red Cross Nagoya Daiichi Hospital
Yamanashi Prefectural Central Hospital
Van Creveldkliniek
AZ Delta
Østfold Hospital Trust
Sultanah Aminah Johor Bahru
Corresponding Author(s)
Other Contributor(s)
Abstract
BACKGROUND: Current second-line treatments for immune thrombocytopenia (ITP) require long-term administration. Ianalumab, a monoclonal antibody targeting B cells, is being assessed as a short-course second-line therapy in ITP. METHODS: In this phase 3, randomized, double-blind trial, we assigned, in a 1:1:1 ratio, adults with primary ITP and an insufficient response or a relapse after first-line glucocorticoid therapy to receive ianalumab at a dose of 9 mg or 3 mg per kilogram of body weight or placebo once monthly for 4 months. Eltrombopag, an oral thrombopoietin-receptor agonist, was administered once daily in each group according to local prescribing information; the dose was tapered until discontinuation by the end of week 24 in eligible patients. The primary end point was freedom from treatment failure, as determined in a time-to-event analysis, with treatment failure defined by a platelet count of less than 30×109 per liter more than 8 weeks after randomization, initiation of rescue therapy more than 8 weeks after randomization, initiation of new ITP therapy, inability to taper or discontinue eltrombopag because of an inadequate platelet count, or death from any cause, whichever occurred first. The key secondary end point was a stable response at 6 months, defined by a platelet count of at least 50×109 per liter in at least 75% of the measurements between weeks 19 and 25 without use of rescue therapy or new ITP therapy. Safety was assessed. RESULTS: A total of 152 patients underwent randomization: 50 to the 9-mg ianalumab group, 51 to the 3-mg ianalumab group, and 51 to the placebo group. The estimated probability of being free from treatment failure at 12 months was 54% (95% confidence interval [CI], 39 to 67) in the 9-mg group, 51% (95% CI, 36 to 64) in the 3-mg group, and 30% (95% CI, 18 to 43) in the placebo group. The time to treatment failure was significantly longer with ianalumab plus eltrombopag than with placebo plus eltrombopag; the estimated hazard ratio for treatment failure (ianalumab vs. placebo) was 0.55 (P = 0.04) in the 9-mg group and 0.58 (P = 0.045) in the 3-mg group. The percentage of patients with a stable response at 6 months was significantly higher in the 9-mg group than in the placebo group (62% vs. 39%; P = 0.045). The overall frequency of adverse events during the treatment period was generally similar in the three groups. The frequency of serious adverse events was 16% in the 9-mg group, 6% in the 3-mg group, and 4% in the placebo group. CONCLUSIONS: Ianalumab plus eltrombopag led to a longer time to treatment failure than placebo plus eltrombopag. (Funded by Novartis; VAYHIT2 ClinicalTrials.gov number, NCT05653219.).