Jesus Ardila Novoa: New Therapies for ITP
Jesus Ardila Novoa, Pediatric Hematologist and Oncologist at Club Noel Children’s Hospital Foundation, shared a post on LinkedIn:
“ISTH hosted a webinar I couldn’t keep to myself.
ITP: Advancing Treatment — New and Emerging Therapies
As in hemophilia, the therapeutic landscape for immune thrombocytopenia is evolving rapidly. And with more options available, shared decision making with the patient becomes not just desirable — but essential.
Three therapies reshaping the field:
- Ianalumab (anti-BAFF) as first-line therapy with corticosteroids — VAYHIT1 measures time to treatment failure. In VAYHIT2, combined with eltrombopag, it significantly prolonged that time vs placebo (HR 0.58 · P=0.045).
- Efgartigimod — blocks FcRN receptors, reducing circulating IgG including autoantibodies. Efficacious in ADVANCE IV. Critical note: ADVANCE SC was NOT superior to placebo — route of administration determines the clinical outcome.
- Rilzabrutinib — BTK inhibitor approved by the FDA in August 2025 (LUNA3). Significant and sustained platelet count increase across multiple patient demographics.
No direct head-to-head comparative trials exist between them. That is precisely why choosing requires understanding the mechanism, the patient’s profile — and above all, their preferences and treatment goals.
Swipe to my notes from the webinar in 5 images.
Special thanks to the speakers for an exceptionally high-level discussion: Dr. Adam Cuker, Dr. Michele Lambert, and Dr. Silvia Zheng.”

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