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Evolving Treatment Goals and Strategies in Primary Immune Thrombocytopenia

Clinical Thought

As the treatment landscape for primary immune thrombocytopenia (ITP) continues to expand, healthcare professionals face increasingly complex decisions regarding treatment goals, therapy selection, and sequencing. This expert commentary reviews the evolving management of ITP, with emphasis on the limitations of corticosteroid-based therapy, the challenges of treatment selection and sequencing, and the growing importance of durable disease control and patient-reported outcomes. The activity examines updated 2026 ASH guideline recommendations for initial and subsequent therapy, including the roles of thrombopoietic agents, rituximab, BTK inhibition, and other treatment classes, as well as recent phase III data from VAYHIT2 and LUNA3. Emerging therapies such as ianalumab, efgartigimod, and mezagitamab are also discussed. Together, these data highlight the need to individualize treatment based on clinical factors, comorbidities, treatment goals, and patient preferences while moving beyond short-term platelet response toward more sustained and patient-centered outcomes.

Released: September 18, 2026

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Faculty

Hanny Al-Samkari

Hanny Al-Samkari, MD

The Peggy S. Blitz Endowed Chair in Hematology/Oncology
Co-Director, Hereditary Hemorrhagic Telangiectasia Center
Massachusetts General Hospital
Associate Professor of Medicine
Harvard Medical School
Boston, Massachusetts
Adam Cuker

Adam Cuker, MD, MS

Professor of Medicine
Professor of Pathology & Laboratory Medicine
Chief, Section of Classical Hematology
Director, Penn Comprehensive Hemophilia and Thrombosis Program
Perelman School of Medicine
University of Pennsylvania
Philadelphia, Pennsylvania

Provided by

Provided by Clinical Care Options, LLC dba Decera Clinical Education.

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Supporters

This activity is supported by an educational grant from Novartis Pharmaceuticals Corporation.

Novartis Pharmaceuticals Corporation

Target Audience

This is intended for hematologists, physician associates, nurses, nurse practitioners, clinical nurse specialists, pharmacists, and other healthcare professionals who manage patients with ITP.

Learning Objectives

Upon completion of this activity, participants should be able to:

  • Apply recent clinical trial and real-world evidence to optimize therapy selection across early and later lines, including appropriate integration of novel immune-targeting mechanisms

  • Incorporate durability-focused treatment goals, including sustained response off treatment, treatment-free remission, and time to treatment failure, into individualized care plans

  • Implement patient-centered shared decision-making strategies, including use of validated patient resources and quality-of-life assessments, to improve adherence, satisfaction, and long-term outcomes

Financial Disclosures

Primary Author

Hanny Al-Samkari, MD: consultant/advisor/speaker: Agios, Alnylam, Alpine, Amgen, Atavistik, Diagonal, Novartis, Pharmacosmos, Sanofi, Sobi, Takeda, Tectonic, Terremoto, Vaderis; researcher: Agios, Alnylam, Amgen, Atavistik, Novartis, Sobi, Vaderis.

Adam Cuker, MD, MS: consultant/advisor/speaker: Incyte, Pfizer.

Additional Disclosures

Generative artificial intelligence (AI) tools were used for data analysis or summarization influencing content. All AI-assisted content underwent human review, editing, and validation by qualified faculty and accredited education staff to ensure scientific accuracy, balance, and compliance with the ACCME Standards for Integrity and Independence in Accredited Continuing Education.