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Immune Thrombocytopenia Treatment: Individualizing Care Beyond Platelet Counts

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Released: September 14, 2026

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The approach to immune thrombocytopenia treatment is evolving as updated guidance and emerging evidence support more individualized care. This faculty-led activity explores strategies to reduce corticosteroid exposure, navigate subsequent treatment options, address fatigue and quality of life alongside platelet response, and assess the potential for treatment tapering and treatment-free response.

Hanny Al-Samkari
Hanny Al-Samkari, MD
Adam Cuker
Adam Cuker, MD, MS
Immune Thrombocytopenia Treatment

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The treatment landscape for immune thrombocytopenia (ITP) is becoming increasingly complex. For many years, management largely centered on corticosteroids, intravenous immunoglobulin (IVIG), rituximab, thrombopoietin receptor agonists (TPO-RAs), and splenectomy with platelet count often guiding treatment decisions. At present, the framework is expanding toward a more individualized model focused on minimizing corticosteroid exposure, selecting therapy based on patient and disease characteristics, incorporating fatigue and quality of life into treatment goals, and considering the potential for sustained response after treatment discontinuation. In this commentary, Adam Cuker, MD, MS, and Hanny Al-Samkari, MD, share their perspectives on these evolving approaches to ITP management.

Minimizing Corticosteroid Exposure in the Initial Management of ITP

Adam Cuker, MD, MS:
Corticosteroids remain useful in ITP, particularly when a rapid initial response is needed. However, repeated courses can create a steroid “roller coaster,” in which the platelet count rises with treatment and falls during tapering, leading to cumulative treatment-related toxicity with each course.

In adults with primary ITP who require initial therapy, the 2026 American Society of Hematology (ASH) guidelines now suggest adding either rituximab or a thrombopoietic agent (eg, a TPO-RA) to corticosteroids, with or without IVIG, rather than corticosteroids with or without IVIG alone. The recommendation to avoid prolonged prednisone exposure remains unchanged; that is, use should be limited to 6 weeks or less.

Prednisone and dexamethasone remain options, with a short course of high-dose dexamethasone potentially preferred when a rapid response is particularly important. The goal is not to eliminate corticosteroids from ITP management but rather to use them purposefully, limit cumulative exposure, and plan the next step rather than return to repeated courses.

Individualizing Subsequent Therapy in ITP

Hanny Al-Samkari, MD:
No single treatment sequence fits every patient. Treatment selection should consider disease duration, bleeding risk, comorbidities, prior responses, treatment burden, route of administration, and patient preferences.

For adults who need additional treatment after initial corticosteroids, with or without IVIG, the 2026 ASH guidelines recommend a thrombopoietic agent (eg, a TPO-RA) or rituximab. For patients who are ineligible for or decline either option, the ASH guidelines suggest a BTK inhibitor (eg, rilzabrutinib), mycophenolate mofetil, or a SYK inhibitor (eg, fostamatinib) as additional options.

An inadequate response to one TPO-RA does not necessarily indicate failure of the entire class; patients may respond to a different thrombopoietic agent. Differences in administration, tolerability, and patient preference may also support switching to another TPO-RA. Rilzabrutinib has further expanded the available treatment options. It is approved by the FDA for adults with persistent or chronic ITP who have had an insufficient response to previous treatment. In the phase III LUNA-3 trial, which enrolled a largely multiagent refractory ITP population, a durable platelet response was achieved by 23% of patients receiving rilzabrutinib compared with 0% receiving placebo. Rilzabrutinib also reduced the need for rescue therapy and improved bleeding scores and physical fatigue compared with placebo. These findings support integrating BTK inhibition into individualized treatment decision-making rather than applying a rigid, one-size-fits-all sequence.

Incorporating Fatigue and Quality of Life Into ITP Treatment Decisions

Adam Cuker, MD, MS:
ITP is more than a bleeding disorder. In the I-WISh survey, 50% of patients reported fatigue at survey completion, and 65% of those experiencing fatigue rated it as severe.

Fatigue should not automatically be attributed to ITP because it is often multifactorial. Potential contributors include iron deficiency or anemia resulting from bleeding, sleep disruption, depression, thyroid disease, deconditioning, medication effects including corticosteroids, and other comorbidities.

At the same time, fatigue should not be dismissed simply because the platelet count is considered “safe.” Platelet count alone does not capture how a patient may be struggling to work, exercise, concentrate, participate socially, or manage everyday activities. Validated tools such as the 10-item ITP Life Quality Index can help assess these effects and systematically incorporate the patient experience into clinical decision-making.

Patient-reported outcomes are increasingly incorporated into clinical trials as prespecified endpoints or exploratory measures. In LUNA 3, rilzabrutinib significantly improved physical fatigue compared with placebo, with improvement sustained from Week 13 (P = .01) through Week 25 (P = .0003). Platelet response remains an important endpoint, but it is not the only outcome that matters. 

Evaluating Treatment-Free Response and the Potential for Durable Disease Control in ITP

Hanny Al-Samkari, MD:
For selected patients with a stable complete response on a TPO-RA, lifelong treatment may not be necessary.

In the prospective, multicenter phase IV STOP-AGO trial of 48 adults with persistent or chronic ITP who had achieved a complete response on TPO-RA therapy, 52% maintained a sustained response off treatment at 52 weeks, and 11 of 12 patients rechallenged with a TPO-RA after relapse regained a complete response. Long-term follow-up subsequently showed that approximately one half of patients remained in sustained response off treatment 4 years after treatment discontinuation.

These findings support a carefully monitored TPO-RA taper in selected patients maintaining a stable platelet count >50,000/µL for at least 6 months without bleeding or rescue therapy. Management should follow a gradual dose reduction before complete discontinuation, accompanied by close subsequent platelet monitoring. However, these findings do not establish whether TPO-RAs induce disease modification or whether treatment-free responses reflect the natural history of ITP.

Ianalumab raises an even more intriguing possibility. In the phase III VAYHIT2 trial, ianalumab plus eltrombopag prolonged time to treatment failure in adults with ITP who had an insufficient response to or relapse after first-line glucocorticoid therapy. At 12 months, the estimated probability of remaining free from treatment failure was 54% with ianalumab 9 mg/kg plus eltrombopag and 51% with ianalumab 3 mg/kg plus eltrombopag, compared with 30% with placebo plus eltrombopag (HR for treatment failure: 0.55 and 0.58, respectively). Stable response at 6 months was also significantly higher with ianalumab 9 mg/kg plus eltrombopag than with placebo plus eltrombopag (62% vs 39%; P = .045).

These findings raise the possibility that a short course of ianalumab could provide durable disease control after treatment withdrawal, although true disease modification remains unproven as of yet (this will be answered with ongoing follow-up of these patients). The phase III VAYHIT1 trial (NCT05653349; active, not recruiting) is evaluating ianalumab with first-line corticosteroids using stable response off treatment at 12 months as the primary endpoint, and the phase II VAY2EXPLORE trial (NCT07421167) is currently recruiting adults with primary ITP to evaluate ianalumab plus investigator-choice TPO-RA followed by TPO-RA tapering.

Your Thoughts
How has the expanding range of ITP treatments changed your approach after initial therapy? When additional treatment is needed, do you typically switch mechanisms of action, try another agent within the same class, or consider tapering therapy in selected patients who have achieved a stable response? What questions or challenges do you have managing your patients with ITP? Leave your question or comment below.

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