ASH shifts ITP treatment strategy beyond steroids alone

Clinical takeaway: For adults with primary immune thrombocytopenia (ITP) who require treatment, consider moving beyond corticosteroid monotherapy. The updated ASH guideline now favors initial combination therapy with rituximab or a thrombopoietic agent plus corticosteroids and recommends thrombopoietic agents as the preferred second-line option after steroid failure.

The American Society of Hematology (ASH) has updated its 2019 ITP guidance to reflect new evidence and newly available therapies, introducing a notable shift away from corticosteroid-only treatment strategies for many adults with primary ITP.

For initial therapy, the panel suggests either rituximab plus corticosteroids or a thrombopoietic agent plus corticosteroids, with or without IVIG, rather than corticosteroids alone. The recommendation was driven by evidence that combination approaches may improve sustained response off treatment, reduce bleeding and rescue therapy use, and improve quality of life.

For patients who need treatment beyond an initial course of corticosteroids, ASH issued its strongest recommendation in favor of thrombopoietic agents. Rituximab is suggested as an alternative for patients who prefer to avoid chronic medication use or who cannot access or tolerate thrombopoietic agents.

Among patients who are not candidates for thrombopoietic agents or rituximab, the guideline suggests BTK inhibitors, SYK inhibitors, or mycophenolate mofetil (MMF), although evidence was insufficient to rank these options. Azathioprine was considered less favorable because of a poorer balance between benefits and harms but may remain useful in some settings because of its low cost and safety profile during pregnancy.

The panel also issued good-practice statements recommending that splenectomy, when feasible, be delayed for at least 1 year after diagnosis because of the potential for spontaneous remission. Switching between thrombopoietic agents may be considered for inadequate response, adverse effects, adherence challenges, comorbidities, or patient preference.

“Previously published guidelines ... advocate for corticosteroids alone in the first-line setting. In contrast, our guidelines suggest rituximab or a thrombopoietic agent plus corticosteroids as initial therapy,” the authors wrote, citing improved sustained responses, reduced rescue therapy use, and better health-related quality of life with combination treatment.

What's changed

• Initial therapy now favors rituximab plus corticosteroids or a thrombopoietic agent plus corticosteroids over corticosteroids alone.
• Thrombopoietic agents are now the preferred second-line treatment after corticosteroid failure and received the guideline's only strong recommendation.
• Rituximab is ranked behind thrombopoietic agents but remains a recommended option in selected patients.
• BTK inhibitors are included as a second-line option, reflecting the arrival of newer therapies since the 2019 guideline.
• Azathioprine has been deprioritized relative to thrombopoietic agents, rituximab, BTK inhibitors, SYK inhibitors, and MMF.

Source: Cuker A, et al. (2026 Aug 19) Blood Adv. American Society of Hematology 2026 Guidelines for Immune Thrombocytopenia (ITP): Initial and Second-Line Therapy in Adults with Primary ITP