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Evolving Treatment Goals and Strategies in Primary Immune Thrombocytopenia

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Released: September 18, 2026

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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.

Hanny Al-Samkari
Hanny Al-Samkari, MD
Adam Cuker
Adam Cuker, MD, MS
Evolving Goals and Strategies in Primary ITP

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Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by a low platelet count and an increased, but variable, risk of bleeding. Many patients with ITP also experience fatigue and impaired health-related quality of life. Increasing numbers of treatments are available for ITP, including corticosteroids, intravenous immunoglobulin (IVIG), thrombopoietin receptor agonists, rituximab, fostamatinib, rilzabrutinib, other immunosuppressive agents, and splenectomy. However, selecting and sequencing these therapies in clinical practice remains challenging.

Current Challenges and Unmet Needs

Adam Cuker, MD, MS:
Traditionally, corticosteroids have served as the backbone of first-line therapy for ITP. Although they can effectively increase platelet counts, their use is limited by significant toxicities, and relapse is common during or after corticosteroid tapering. Thus, new strategies for first-line therapy that minimize corticosteroid exposure, reduce relapse risk, promote durable disease control, and improve the long-term disease course remain an important unmet need.

Beyond the first-line setting, current practice often involves trial and error for patients who require additional treatment—if a patient does not respond to or tolerate one treatment, another is selected. Accordingly, evidence-based, patient-centered approaches to treatment selection and sequencing are essential to guide clinical decision-making.

Evolving Treatment Goals

Hanny Al-Samkari, MD:
As recognition of the patient burden of ITP has increased and treatment options have improved, therapeutic goals have become more ambitious. Many patients seek more than treatments that simply raise the platelet count and prevent bleeding. They also seek treatments that minimize toxicity, improve fatigue and health-related quality of life, and offer the potential for a sustained response off treatment.

Patient-Centered Care and Shared Decision-making

Adam Cuker, MD, MS:
In this context, ITP management involves a partnership between the hematologist and the patient. Hematologists should discuss the benefits and limitations of the available treatment options with their patients and consider their symptoms, comorbidities, lifestyle, and treatment goals, values, and preferences. Shared decision-making is essential to selecting a treatment approach that best aligns with each patient’s priorities.

Practical Considerations for Treatment Selection and Sequencing

Hanny Al-Samkari, MD:
Consistent with these evolving treatment goals, for adults with primary ITP who require initial therapy, the American Society of Hematology (ASH) 2026 ITP guidelines suggest either rituximab plus corticosteroids (with or without IVIG) or a thrombopoietic agent plus corticosteroids (with or without IVIG) rather than mycophenolate mofetil plus corticosteroids (with or without IVIG) or corticosteroids (with or without IVIG) alone. Rituximab-based combination therapy may be preferable for patients who place a high value on avoiding regular medication. By contrast, thrombopoietic agent–based combination therapy may be preferable in patients who place a high value on avoiding immunosuppression. Comorbidities and other patient-specific factors, including thrombotic risk, should also be considered. 

For adults with primary ITP who require additional treatment after initial therapy with corticosteroids (with or without IVIG), the ASH guidelines recommend a thrombopoietic agent or rituximab. For adults who are ineligible for or decline a thrombopoietic agent or rituximab, the guidelines suggest a BTK inhibitor, mycophenolate mofetil, or a SYK inhibitor. Clinical factors and patient preferences should guide treatment selection and sequencing. Rilzabrutinib, a BTK inhibitor, is approved by the FDA for adults with persistent or chronic ITP who have had an insufficient response to a previous treatment.

In addition to these guideline recommendations, recent phase III data highlight the growing importance of durability and patient-reported outcomes. In VAYHIT2, adults with primary ITP who had an insufficient response to or relapsed after first-line corticosteroids therapy received ianalumab or placebo in addition to eltrombopag. Ianalumab 9 mg/kg plus eltrombopag prolonged time to treatment failure compared with placebo plus eltrombopag (HR: 0.55), and stable response at 6 months was 62% vs 39%, respectively. In LUNA 3, previously treated adults with persistent or chronic ITP received rilzabrutinib or placebo; durable platelet response occurred in 23% vs 0% (P < .0001) and platelet response occurred in 65% vs. 33%. Durable platelet response occurred in 23% vs 0% (P <.0001), and rilzabrutinib also improved physical fatigue. Together, these studies illustrate how the treatment landscape is moving beyond short-term platelet response toward more durable, patient-centered outcomes.

Emerging Therapies

Adam Cuker, MD, MS:
Beyond currently available therapies, emerging therapies in ITP include several agents in clinical development. Ianalumab has positive phase III VAYHIT2 data in adults with primary ITP after an insufficient response to or relapse after first-line glucocorticoid therapy, as summarized above, and remains under study for first-line treatment of ITP. Efgartigimod IV met the primary endpoint in ADVANCE IV in adults with persistent or chronic primary ITP and had a platelet response rate of 67% vs 30% with placebo; by contrast, the subcutaneous ADVANCE-SC study did not meet its primary or secondary endpoints, and the phase III ADVANCE-NEXT study is ongoing. In a randomized phase II trial of adults with persistent or chronic ITP, platelet response through week 16 was observed in 91% of patients receiving mezagitamab 600 mg vs 23% with placebo. Phase III development is ongoing. All remain investigational for ITP in the United States.

Your Thoughts
What key points about ITP treatment options do you discuss with patients with primary ITP? What resources or educational tools would make these discussions clearer and more effective? Leave a comment to join the discussion!

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For adults with primary ITP who require initial treatment, how often do you discuss the potential role of combination therapy (corticosteroids plus rituximab or a thrombopoietic agent) with the patient?

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