Blood Podcast How I Treat Wiskott-Alrich syndrome
Oct 2, 2025
Sung-Yun Pai, a clinician-researcher specializing in Wiskott-Aldrich syndrome, shares vital insights on the latest treatment advancements. He discusses the evolving risk-benefit calculus in therapies due to safer options and longer follow-ups. The conversation highlights challenges like late diagnosis, donor matching, and limited access to gene therapy. Pai stresses the urgency of early referrals to specialized centers and deliberates on the decision-making process between transplant and gene therapy, emphasizing the complexities of achieving full correction.
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Changing Risk-Benefit Calculus
- Recent advances and longer follow-up have changed the risk-benefit calculus for treating Wiskott-Aldrich syndrome.
- Safer treatments let clinicians offer curative therapy to a wider range of patients than in the past.
Diagnosis Timing And Donor Gaps Matter
- Late diagnosis and lack of well-matched donors worsen outcomes and complicate curative therapy.
- Gene therapy looks promising but remains unavailable in the U.S. for many patients despite supportive data.
Late-Onset Renal Manifestations
- Renal disease such as IgA nephropathy appears more in older patients with certain WAS variants.
- Kidney manifestations are a later-recognized component, often emerging in teens and young adults.