Winst gevend schreef op 7 maart 2024 14:05:
The only potentially curative therapy available for APDS is haematopoietic stem cell transplant (HSCT)
However, HSCT carries a considerable risk of complications including rejections or graft failure.
In a Japanese cohort of 9 patients with APDS receiving HSCT, 91% experienced
adverse complications of HSCT and 36% experienced initial engraftment failure and 2 patients died.
In a US and European APDS cohort of 57 patients receiving HSCT,
2-year overall survival was 86% and 2-year graftfailure free survival was 68%.
Among the 8 patients who died during follow-up, 63% of deaths occurred inthe first 100 days following HSCT.
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